Education Program

A Little Less Conversation, a Little More Action: An Outcome Equity Roadmap for Children and AYAs With Leukemia and Lymphoma

Saturday, December 7, 2024, 9:30 a.m. - 10:45 a.m. Marriott Marquis San Diego Marina, Marriott Grand Ballroom 2-4

Centuries of structural racism have contributed to discrimination, environmental and social injustice, and household material hardship among historically marginalized populations. Recent studies have highlighted the impact of factors such as income and insurance status on cancer outcomes and health equity. Under-representation of specific groups has limited our understanding of cancer risk, disease biology, treatment-related toxicities, and patient-reported outcomes. The educational objective of this session is to present the latest evidence regarding modifiable factors that contribute to outcome disparities in childhood/adolescent and young adult (AYA) hematologic malignancies, and to discuss potential targets for interventions that address these disparities. Dr. Ji will present an in-depth overview of public health insurance programs in the U.S. and related implications on outcome disparities among AYAs with blood cancers. Using a case-based approach, Dr. Ji will discuss opportunities to improve access to high-quality public health insurance, subsequent to the Affordable Care Act. Dr. Umaretiya will discuss the role of social determinants of health (SDOH) as modifiable drivers of outcome inequities in pediatric oncology. She will review the prevalence of SDOH in pediatric oncology, present historically marginalized parent perspectives on unmet social needs during cancer care, and highlight novel interventions addressing this population’s SDOH and social needs. Dr. Mittal will discuss factors contributing to low rates of enrollment of AYAs with hematologic malignancies to clinical trials, including a lack of availability and accessibility. She will provide an up-to-date review of collaborative initiatives that are addressing some of these barriers to improve AYA access to cancer clinical trials.

Chair:

Maria Monica Gramatges, MD,PhD
Texas Children's Hospital
Houston,  TX

Speakers:

Xu Ji, PhD
Emory University School of Medicine
Atlanta,  GA
The Lasting Impact of the ACA: How Medicaid Expansion Reduces Outcome Disparities in AYAs With Leukemia and Lymphoma

Puja J. Umaretiya, MD
Childrens Medical Center Dallas, UT Southwestern
Dallas,  TX
Targeting Hardship: Poverty as a Modifiable Risk Factor in Childhood Leukemia and Lymphoma Treatment

Nupur Mittal, MD
Rush University
Chicago,  IL
Sharing is Caring: A Network Collaborative Approach to Identify and Address Barriers in Accessing Clinical Trials in Adolescents and Young Adults with Leukemia and Lymphoma

A Shifting Landscape: Treatment of MCL

Monday, December 9, 2024, 10:30 a.m. - 11:45 a.m. Marriott Marquis San Diego Marina, Marriott Grand Ballroom 2-4

Mantle Cell Lymphoma is a rare subtype of B-cell non-Hodgkin Lymphoma characterized by disease heterogeneity.  The approach to treating patients diagnosed with mantle cell lymphoma has evolved over time with therapeutics including high dose cytarabine containing regimens prior to autologous stem cell consolidation, rituximab maintenance and the oral Bruton’s tyrosine kinase inhibitors for relapsed/refractory disease.  It is recognized that not all patients benefit from these approaches.  This educational session will explore the evolving treatment landscape in mantle cell lymphoma including identification and treatment of patients with high-risk disease, the role of autologous stem cell transplant consolidation in the era of novel therapies and the current and future incorporation of T-cell engaging therapies in the treatment landscape.

Dr. Kami Maddocks will define high-risk features in Mantle Cell Lymphoma associated with inferior prognosis to standard treatment approaches. Clinical trials incorporating Bruton's tyrosine kinase inhibitors into initial therapy will be discussed. The role of targeted and immunotherapy combinations in a “chemotherapy-free” approach will be presented.

Dr. Martin Dreyling will discuss the role of autologous stem cell transplantation and additional consolidation/maintenance therapies in first line treatment. Clinical trials incorporating Bruton's tyrosine kinase inhibitors into initial therapy will be mentioned. Differential treatment strategies according to clinically applicable biological risk factors will be suggested.

Dr. Lia Palomba will review T-cell engaging therapies in relapsed or refractory Mantle Cell Lymphoma, including CAR T-cells and bispecific antibodies. She will review available data on the outcome of these therapies in the general MCL population and in high-risk subgroups.

Chair:

Kami J. Maddocks, MD
The Ohio State University
Columbus,  OH

Speakers:

Kami J. Maddocks, MD
The Ohio State University
Columbus,  OH
Your Chemo is No Good Here: Management of High-Risk MCL

Martin Dreyling, MD
LMU University Hosptial München
Munich,  Germany
To Consolidate or Not to Consolidate: The Role of Autologous Stem Cell Transplantation in MCL

Lia Palomba, MD
Memorial Sloan Kettering Cancer Center
New York,  NY
T-cell–based Therapies for Treating Relapsed or Refractory Mantle Cell Lymphoma

Addressing Unmet Needs in T Cell Lymphomas

Monday, December 9, 2024, 4:30 p.m. - 5:45 p.m. San Diego Convention Center, Ballroom 20AB

The rare nature of peripheral and cutaneous T-cell lymphomas, variable clinical course and only recently evolving understanding of their unique biology make the care of patients with these disease particularly difficult. This educational session will delve into current clinical challenges in management of this difficult to treat population and will focus on the more common subtypes of peripheral T-cell lymphoma and cutaneous T-cell lymphomas.  The session first will discuss management, incorporation of novel agents and consideration of personalized approaches in relapsed/refractory peripheral T-cell lymphomas and cutaneous T-cell lymphomas. We will also discuss the utility and timing of both autologous and allogeneic transplant in cutaneous and peripheral T-cell lymphoma.  

Dr. Mehta-Shah will discuss current clinical challenges in the management of patients with relapsed/refractory T-cell lymphomas.  In the evolving landscape of treatment and biologic considerations, we will discuss the use of both standard agents as well as novel targeted agents in the treatment of this patient population. Given that our understanding of these heterogenous diseases continues to improve, we will discuss strategies for subtypes specific or personalized therapy.

Dr. Horwitz will explore and detail the expanding options for treatment of CTCL.

Dr. Dreger will discuss the role of stem cell transplant (auto and allo) on PTCL and CTCL.

Chair:

Neha Mehta-Shah, MD
Washington University in St. Louis
St Louis,  MO

Speakers:

Neha Mehta-Shah, MD
Washington University in St. Louis
St Louis,  MO
BV and Beyond: How to Incorporate Novel Agents into PTCL Management

Steven Horwitz, MD
Memorial Sloan-Kettering Cancer Center
New York,  NY
Through Thick and Thin: Confronting the Aggressive Cutaneous T-cell Lymphomas

Peter Dreger, MD
Universitätsklinikum
Heidelberg,  Germany
The Role of Stem Cell Transplant (auto and allo) in PTCL and CTCL

Adult ALL Advancements: Optimizing Cure in 2024

Monday, December 9, 2024, 2:45 p.m. - 4:00 p.m. San Diego Convention Center, Ballroom 20AB

Acute lymphoblastic leukemia (ALL) in adults is an aggressive condition with historically few long-term survivors. Fortunately, the outlook for ALL in adults is improving rapidly as years of scientific research and clinical investigation begins to pay dividends. Improved understanding of the heterogeneity and varied biological behavior of ALL subtypes has led to routine immunophenotypic and genetic characterization of individual ALL cases in the clinic. Profiling of ALL at diagnosis as well as better ability to track measurable residual disease (MRD) after therapy now permits personalized, risk-adapted treatment recommendations in ALL including the rational application of allogeneic stem cell transplantation. In addition, multiple new targeted and immune therapies have been approved by regulatory authorities for Philadelphia chromosome-positive (Ph+) and negative B-ALL. The rapid pace of scientific and therapeutic advancement in ALL is challenging established standards of care for upfront treatment of adults with newly diagnosed ALL. In this session, the impact of scientific and therapeutic advancements in the initial treatment approach for ALL will be reviewed, with a focus on recent evidence generation.

Dr. Marlise Luskin will review data supporting the selection of various approaches to initial treatment of Ph+ ALL in adults. She will discuss data justifying the use of specific BCR-ABL1 kinase inhibitors including dasatinib, nilotinib, and ponatinib. In this session, she will also review evidence defining the role of conventional chemotherapy, allogeneic transplantation, and the bi-specific T-cell engager (BiTE) blinatumomab in first-line post-remissiont treatment of Ph+ ALL in adults of different ages.

Prof. Matthias Stelljes will discuss the role of novel antibody-based and cell-based immunotherapies in the treatment of ALL in adults. Blinatumomab, the antibody-drug conjugate inotuzumab ozogamicin, and CD19-directed CAR-T cells are current standard-of-care options for patients with relapsed or refractory B-cell ALL. The CD20-targeting monoclonal antibody rituximab and blinatumomab are established components of frontline therapy for patients with CD20-positive ALL and patients with persistent MRD, respectively. More recently, blinatumomab and inotuzumab have shown promising results in the upfront treatment of patients with B-cell ALL. These data form the basis for evaluating and defining new treatment standards, which will be discussed in this educational session.

Dr. Partow Kebriaei will review risk profiling in adult patients with ALL, and specifically how disease risk informs the decision to recommend transplant in first complete remission. Additionally, she will highlight advancements in GVHD prophylaxis that facilitate greater donor availability and decreased toxicity of transplant. Finally, Dr. Kebriaei will review the optimal sequence for transplant within the context of CAR T therapy.

Chair:

Marlise R Luskin, MD, MSCE
Dana-Farber Cancer Institute
Boston,  MA

Speakers:

Marlise R Luskin, MD, MSCE
Dana-Farber Cancer Institute
Boston,  MA
Ph+ ALL: New Approaches for Upfront Therapy

Matthias Stelljes, MD
University Hospital Muenster
Muenster,  Germany
Ph- ALL: Impact of Immunotherapy in Upfront Treatment

Partow Kebriaei, MD
MD Anderson Cancer Center
Houston,  TX
Transplant in ALL: Who, When, and How?

Adult CAR-T Outcomes: Beyond the Acronyms (OS, PFS, CRS, ICANS)

Monday, December 9, 2024, 4:30 p.m. - 5:45 p.m. San Diego Convention Center, Room 24

CAR T-cell therapy has become a standard treatment option resulting in improved progression-free and overall survival in a variety of relapsed and refractory adult hematologic malignancies. The acute toxicities, particularly CRS and ICANS, and outcomes of CAR T-cell therapy have been extensively described. However, as indications and utilization of this treatment have expanded with increasing reports of long-term survivors, it has become increasingly apparent that there are a number of clinically significant, long-term complications and toxicities that may occur in CAR T-cell recipients. Recognition and understanding of these long-term complications are imperative to patient selection, counseling, and management. This session will review and provide guidelines for monitoring and treatment of long-term complications after CAR T-cell therapy in adult patients.

Measuring disease-related and treatment-driven patient-reported outcomes (PROs) as standard of care in patients undergoing CAR T-cell therapy is both feasible and vital. As has been shown with numerous oncology therapies, the assessment of symptom and functioning PROs is now sufficiently mature to justify their contribution to treatment precision, efficiency, and standardization and national and international collaborations. Nonetheless, collaborative efforts among clinicians, symptom researchers, and electronic health system designers are needed to integrate evidence-based PROs into routine patient care after CAR-T cell therapy. This will help in capturing early toxicity and implement timely interventions with the ultimate goal of improving treatment tolerability related to symptom burden and CAR T-cell clinical outcomes.

Adults receiving chimeric antigen receptor (CAR) T-cell therapy may experience late complications distinct from those in the early CAR T-cell treatment period. Late complications include cytopenias, infections, secondary malignancies, and delayed neurotoxicities. Late cytopenias may be from prolonged recovery after lymphodepleting chemotherapy or arise anew, raising concerns for recurrent disease or a secondary malignancy. Late infections, which are predominantly viral, may require ongoing prophylaxis and immunoglobulin replacement therapy. Both hematologic and solid secondary malignancies are a known risk of CAR T-cell therapy, with recent observations of T-cell cancers. It is essential to monitor for late complications in adult patients receiving CAR T-cells to improve the outcomes and quality of life.

Infections following CAR-T remain a notable cause of morbidity and mortality not only in the early (<30 days) post-CAR-T but also persist through the prolonged (30-90 days) and late (>90 days) follow-up.  The predisposition to infections results from immunosuppression related to underlying disease, prior cytotoxic therapies, lymphodepletion chemotherapy, delayed hematopoietic recovery, B-cell aplasia, and delayed T-cell immune reconstitution. This draft delineates risk factors and epidemiology of infections along the post-CAR-T timeline. Dr. Jain will further discuss our approach to mitigation and management of infections including antibacterial, antiviral, and antifungal prophylaxis, growth factors to expedite count recovery, immunoglobulin replacement therapy, and re-vaccination programs.

Chair:

Michael R Bishop, MD
University of Chicago
Chicago,  IL

Speakers:

Xin Shelley Wang, MD, MPH
The University of Texas MD Anderson Cancer Center
Houston,  TX
Patient-Reported Outcomes After CAR T-cell Therapy?in Patients with Hematological Malignancies

Michael R Bishop, MD
University of Chicago
Chicago,  IL
Late Complications and Long-term Care of Adult CAR T-cell Patients

Tania Jain, MD
Johns Hopkins University
Baltimore,  MD
Mitigating and Managing Infection Risk in Adults Treated with CAR T-Cell Therapy

ALPS, CVID, and LGL: When Autoimmune Cytopenias Are a Marker of Underlying Immune Disease

Saturday, December 7, 2024, 9:30 a.m. - 10:45 a.m. San Diego Convention Center, Room 6CF

Autoimmune cytopenias that are chronic, refractory and multi lineage present a challenge in the clinic. However, recent advances exploring their molecular genetic underpinnings have made them more amenable to rationally targeted therapies and forestall end organ damage in the long run. Dr. V. Koneti Rao will discuss diagnosis and management of ALPS-FAS (Autoimmune Lymphoproliferative Syndrome due to FAS variants) and APDS (Activated PI3Kinase Delta Syndrome due to PIK3CD and PIK3R1 genetic variants) including recently licensed targeted treatment for the latter using a PI3Kinsae inhibitor, leniolisib. Dr.Charlotte Cunnigham Rundles will discuss the impact of genetic disorders presenting as CVID (Common Variable Immune Deficiency) affecting multiple organs due to variants in transcription factors, check point inhibitors, chemokines and cytokines. CVID is one of the most common inborn errors of immunity presenting with infections due to insufficient levels of immune globulins and loss of specific antibodies. However, a significant proportion of these patients develop autoimmune multilineage cytopenias and increased susceptibility to malignancies including lymphoma. These conditions may often be the initial manifestation of the underlying immune defect in a patient who has not had significant infections, and these episodes may recur over time. Treatment of the hematologic complications may require frequent and episodic use of corticosteroids, and/or rituximab; splenectomy is discouraged. Genetic associations noted in CVID patients include CTLA4, LRBA, IKZF1, NFKB1 and NFKB2,STAT3 and STAT1 variants that might provide opportunities for targeted therapeutics. Dr.Thierry Lamy will highlight the impact of clonal evolution of T and Nk cells in LGL (large granular lymphocytosis).Large granular lymphocyte (LGL) leukemia is a rare T/NK cell driven chronic lymphoproliferative disease characterized by neutropenia, anemia and autoimmune disorders such as rheumatoid arthritis, Sjogren syndrome, vasculitis, and autoimmune endocrinopathies. Recent advances in the pathogenesis of LGL leukemia have highlighted the implication of STAT3 genetic variants playing a pivotal role in the leukemic cell proliferation and survival, and autoimmunity. The genetic landscape of LGL is afflicted with gene mutations affecting NF-KB pathway (TNFAIP3), epigenome (TET2, KMT2D), and microenvironment (CCL22). Immunosuppressive agents (cyclophosphamide, methotrexate and cyclosporine) constitute the foundation of first line therapy. Recent advances have led to the development of targeted approaches, including JAK-STAT inhibitors, cytokine targeting and hypomethylating agents, opening novel approaches in an otherwise incurable disease.

Chair:

V. Koneti Rao, MD
National Institutes of Health
Bethesda,  MD

Speakers:

V. Koneti Rao, MD
NIH
Bethesda,  MD
Beyond FAScinating: Advances in Diagnosis and Management of Autoimmune Lymphoproliferative Syndrome and Activated PI3 Kinase d Syndrome

Charlotte Cunningham-Rundles, MD, PhD
Mount Sinai School of Medicine
New York,  NY
Common Variable Immunodeficiency: Hematologic Presentation and Advances in Molecular Diagnosis

Thierry Lamy, MD, PhD
Pontchaillou Hospital Rennes UNIVERSITY HOSPITAL
Rennes,  France
Large Granular Lymphocyte Leukemia: A Clonal Disorder with Autoimmune Manifestations

AML M&Ms: How to Integrate Mutations and MRD Data

Saturday, December 7, 2024, 9:30 a.m. - 10:45 a.m. Manchester Grand Hyatt San Diego, Seaport Ballroom ABCD

The genomic heterogeneity of AML, the clinical variability of patients and patient outcomes to various therapies, and the underlying awareness and evaluation of clonal evolution mandates an integrated approach to AML risk assessment and treatment decision-making.   This session will focus on the clinical management of AML using a comprehensive evaluation including both genetic characterization and MRD-based response assessments during therapy, to optimize the clinical outcomes of patients with AML.

Dr. Jerald Radich will examine the role of MRD as an indicator of treatment response, and help answer the question of why some patients with persistent MRD do not relapse, while others without detectable MRD may still relapse nonetheless.  He will explore how to best use MRD to optimize patient care, and explore how the mutational landscape of AML at diagnosis and during therapy can help to explain not only the clinical utility of MRD but also key features of leukemia biology.

Dr. Michael Heuser will discuss the prognostic impact of MRD status in different genetic risk groups and provide an overview on the available MRD technologies to monitor treatment response in transplant-eligible patients with AML. He will help to unravel the complex matrix of MRD and mutational dependencies for practical clinical application and management decisions in patients with AML.

Dr. Courtney DiNardo will summarize the current treatment landscape of AML in patients ineligible for transplant, and focus on the importance of a comprehensive genomic assessment to identify optimal treatment strategies both at diagnosis and relapse.  She will also highlight the role of MRD assessments and review the role of MRD measurements to guide treatment decisions in patients receiving non-intensive therapies.

Chair:

Courtney D. DiNardo, MD,MSc
MD Anderson Cancer Center
Houston,  TX

Speakers:

Jerald P. Radich, MD
University of Washington School of Medicine
Seattle,  WA
Mutations and MRD: Clinical Implications of Clonal Ontogeny

Michael Heuser, MD
Hannover Medical School
Hannover,  Germany
Mutation-and MRD-informed Treatment Decisions for the Transplant-Eligible AML Patient

Courtney D. DiNardo, MD,MSc
MD Anderson Cancer Center
Houston,  TX
Mutation-and MRD-informed Treatments for Transplant-Ineligible Patients

Anticoagulation SOS: Navigating Monitoring Conundrums

Monday, December 9, 2024, 10:30 a.m. - 11:45 a.m. San Diego Convention Center, Room 28 A-D

Anticoagulation monitoring challenges are encountered commonly in clinical practice. This educational session will present clinically oriented approaches to understanding and managing anticoagulation monitoring challenges associated with direct oral anticoagulant (DOAC) use, heparin monitoring, and anticoagulation for antiphospholipid antibody syndrome.

Dr. Deborah Siegal will review the potential benefits and limitations of DOAC measurement. Clinical situations in which DOAC measurement may be helpful will be discussed and DOAC testing options will be presented based on the goal of measurement.

Dr. Cheryl Maier will review the definition of heparin resistance which varies depending on specific measurements of anticoagulation and the type of assay used. Multiple causes of heparin resistance will be covered using a case-based approach to help providers troubleshoot heparin resistance and develop alternative management strategies when heparin resistance is encountered.

Prof. Hannah Cohen will summarise the use of warfarin in patients with thrombotic antiphospholipid syndrome (APS). Using a case-based approach, she will highlight key considerations in the choice of oral anticoagulant, issues that may impact on accurate monitoring of warfarin anticoagulation in APS patients, and strategies to overcome these. She will also discuss the use and monitoring of low-molecular-weight/unfractionated heparin in APS patients in particular situations.

Chair:

Deborah Siegal, MD,MSc
University of Ottawa
Ottawa,  ON, Canada

Speakers:

Deborah Siegal, MD,MSc
University of Ottawa
Ottawa,  ON, Canada
DOACs: role of anti-Xa and Drug Level Monitoring

Cheryl L Maier, MD, PhD
Emory University
Atlanta,  GA
Troubleshooting Heparin Resistance

Hannah Cohen, MD
University College London Hospitals NHS Foundation Trust
London,  United Kingdom
Warfarin and Heparin Monitoring in Antiphospholipid Syndrome

Beyond TTP: "Atypical" TMAs for the Hematologist

Saturday, December 7, 2024, 2:00 p.m. - 3:15 p.m. San Diego Convention Center, Room 24

The American Society of Hematology educational session will delve into the complexities of thrombotic microangiopathies (TMAs) outside the typical presentation of thrombotic thrombocytopenic purpura (TTP).

The session will address three related disease states: Transplant-Associated Thrombotic Microangiopathy (TA-TMA), atypical Hemolytic Uremic Syndrome (aHUS) and Catastrophic Antiphospholipid Antibody Syndrome (CAPs).

The segment on TA-TMA will cover its incidence in the post-transplant period, the challenges of distinguishing TA-TMA from other post-transplant complications, and the data on emerging treatment approaches. The session will also include a comprehensive review of aHUS, focusing on its genetic underpinnings, atypical presentations, and the latest advancements in targeted therapies, such as complement inhibitors. The discussion will encapsulate the nuances of managing these atypical TMAs, highlighting case studies and clinical trials that underscore the critical role of personalized medicine in improving patient outcomes. This session will also explore Catastrophic Antiphospholipid Syndrome (CAPS), a rare and severe subtype of antiphospholipid syndrome characterized by widespread microvascular thrombi, leading to multiple organ failure. The talk will provide an overview of the pathophysiology, diagnostic criteria, and emerging treatment strategies. Discussion will also focus on distinguishing CAPS from other TMAs through clinical presentation and laboratory findings, emphasizing the importance of timely and accurate diagnosis to mitigate the high mortality rate associated with this condition.

Chair:

Mark Crowther, MS MSc LLM FRCPC FRSC
McMaster University
Hamilton,  ON, Canada

Speakers:

Anuja Java
Washington University in St. Louis
St. Louis,  MO
Atypical Hemolytic Uremic Syndrome: Diagnosis, Management, and Discontinuation of Therapy

Ang Li, MD,MS
Baylor College of Medicine
Houston,  TX
Transplant Associated TMA: The Conundrum of Diagnosis and Treatment

Mark Crowther, MS MSc LLM FRCPC FRSC
McMaster University
Hamilton,  ON, Canada
Catastrophic Antiphospholipid Syndrome: A CAPS-tivating Hematologic Disease

Challenges and Opportunities of Sickle Cell Care in Africa

Monday, December 9, 2024, 10:30 a.m. - 11:45 a.m. San Diego Convention Center, Room 31

This session will discuss the current state of therapies for sickle cell disease in Africa. Access barriers to disease-modifying and curative treatments for sickle cell anemia will be identified, and recommendations of strategies to address some of these barriers will be proposed. The case will be made to prioritize widespread use of hydroxyurea therapy as the bedrock of sickle cell anemia care in Africa.

Chair:

Isaac Odame, MBBCh
The Hospital for Sick Children (SickKids) and the University of Toronto
Toronto,  ON, Canada

Speakers:

Obiageli E Nnodu, MD
University of Abuja
Abuja,  Nigeria
Newborn Screening Initiatives for Sickle Cell Disease in Africa

Isaac Odame, MBBCh
The Hospital for Sick Children (SickKids) and the University of Toronto
Toronto,  ON, Canada
Transfusion, Disease-Modifying Treatments, and Curative Therapies for Sickle Cell Disease in Africa: Where Are We Now?

Kofi A. Anie, PhD, MBE
London North West University Healthcare NHS Trust and Imperial College London
London,  ENG, United Kingdom
The Intersection of Sickle Cell Disease, Stigma, and Pain in Africa

Challenges in Cancer-Associated Thrombosis

Sunday, December 8, 2024, 4:30 p.m. - 5:45 p.m. San Diego Convention Center, Hall B

Patients with cancer have an increased risk of venous thromboembolism (VTE) for which anticoagulation is the treatment of choice. However, they also commonly face complications related to cancer itself or cancer therapies that create unique challenges for the management of cancer-associated thrombosis. This educational session will explore the evidence for best practices in assessing and managing challenging scenarios in patients with cancer-associated thrombosis, including patients with brain metastasis, recurrent venous thromboembolism despite anticoagulation, and thrombocytopenia.

Dr. Leader will summarize the evidence of anticoagulant use in patients with brain metastasis, discuss risks and benefits of anticoagulation in these patients and optimal management strategies in this high-risk population. 

Dr. Zwicker will discuss the challenges of evaluating and managing patients with cancer and new or progressive VTE on anticoagulation. He will discuss diagnostic strategies to identify etiologies for anticoagulant failure and provide therapeutic options in this patient population. 

Dr. Ay will address the challenge of concurrent VTE and thrombocytopenia in patients with cancer. He will summarize data from available literature, review guidance recommendations, and discuss management strategies and future directions in these patients.

Chair:

Tzu-Fei Wang, MD,MPH
Ottawa Hospital Research Institute, University of Ottawa
Ottawa,  ON, Canada

Speakers:

Avi Leader, MD
Memorial Sloan Kettering Cancer Center
New York,  NY
Management of Anticoagulation in Patients With Brain Metastasis

Jeffrey I. Zwicker, MD
Memorial Sloan Kettering
NYC,  NY
Trousseau Syndrome: Management of Refractory VTE

Cihan Ay
Medical University of Vienna
Vienna,  Austria
Treatment of VTE in the Thrombocytopenic Cancer Patient

Chasing Zebras: Navigating Rare Myeloid Neoplasms

Saturday, December 7, 2024, 2:00 p.m. - 3:15 p.m. Manchester Grand Hyatt San Diego, Grand Hall C

It turns out the old adage from our medical school days, “If you hear hoofbeats, think of horses, not zebras,” was not completely correct!  Indeed, the diagnosis of a “rare disease” turns out to be not as rare as we previously thought.  The faculty speaking in this important session have focused their entire careers to patient care and cutting-edge research in areas of rare and even ultra-rare hematologic malignancies. The coupling of increasing pathologic technology and diagnostic methods along with augmented education and awareness have shed light on some of the more rare blood cancers in our field, especially over the past decade.  In particular, in the field of myeloid neoplasms, the emerging importance of dividing these entities into specific subgroups distinguished by unique clinicopathologic characteristics has had a tremendous impact not only on diagnosis, but also on prognosis and development of novel treatment decision approaches.  

In this first talk, Dr Robert P Hasserjian focuses his remarks on understanding the growing field of chronic neutrophilic leukemia (CNL) and CSF3R-related disorders. In the decade since the elucidation by, Maxson et al (Maxson, NEJM 2013), of frequent oncogenic CSF3R mutations in patients with CNL and atypical CML, this field has rapidly expanded as we are beginning to understand that these Philadelphia negative hematologic myeloid malignancies  are more commonly present than we previously thought.

In the second talk, Dr Naveen Pemmaraju highlights the encouraging developments in a rapidly emerging field in hematologic malignancies, that of Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN).  BPDCN is a singularly unique malignancy that is known for involving cutaneous sites, followed by bone marrow/blood lymph nodes, and of interest, a high rate of CNS involvement. Despite many nomenclature and disease category changes over the past three decades, BPDCN is now recognized as its own unique entity which expresses, via flow cytometry and immunohistochemistry: CD123+, along with CD4+, CD56+, TCL-1+ and TCF-4+. The clinical pioneering of the first approved CD123-targeted agent in the field (tagraxofusp, led by Pemmaraju et al NEJM April 2019) has led to the development of a  worldwide clinical/translational interest in BPDCN, targeting CD123 by various therapeutic approaches, and for interest in developing combination approaches and CNS-directed therapies for this rare blood cancer.

In the final talk of this session, Dr Olga Weinberg very nicely draws attention to acute leukemias of ambiguous lineage, a field that is rapidly growing as it benefits from deeper, faster, and more directed targeted sequencing efforts and improved patho-biologic understanding of the various subsets that make up this unique entity. Among the keys to this topic will be understanding several of the newer tests, at the level of the blood, bone marrow, flow cytometry, cytogenetics and molecular next generation sequencing that are available in the pathologists’ and clinicians’ tool kits in searching for identification and diagnosis of particular subtypes of this historically elusive diagnosis that is becoming more and more common to encounter.

Chair:

Naveen Pemmaraju, MD
MD Anderson Cancer Center
Houston,  TX

Speakers:

Robert P Hasserjian, MD
Massachusetts General Hospital
Boston,  MA
The Spectrum of Ph Negative Disease: CNL and CSF3R-Related Disorders

Naveen Pemmaraju, MD
MD Anderson Cancer Center
Houston,  TX
BPDCN State of the Art

Olga K. Weinberg, MD
UT Southwestern
Dallas,  TX
How to Think About Acute Leukemia of Ambiguous Lineage

Follicular Lymphoma: Playing the Long Game

Sunday, December 8, 2024, 4:30 p.m. - 5:45 p.m. Marriott Marquis San Diego Marina, Marriott Grand Ballroom 11-13

The prolonged survival for most patients with newly diagnosed follicular lymphoma in 2024 is a triumph of the introduction of rituximab a quarter of a century ago. Our improved understanding of follicular lymphoma biology, the heterogeneity of both disease and patients, and new therapeutic options help us in “choosing wisely” the sequencing of treatment for a given patient. We can now be confident of a median 10 years between first and second treatments along with an age-matched survival for patients in sustained remission after immunochemotherapy. In this session, we map the remaining challenges of identifying the minority of patients with early progression and a poor prognosis, as well as the sequencing of the highly promising T-cell immunotherapies for patients with early or multiply-relapsed disease.

In this talk, Dr. Trotman will provide the survival data to equip clinicians in framing optimistic initial conversations with most patients at diagnosis of advanced stage FL. She outlines the expectations of longevity and a “functional cure” for many. She will address considerations of both patient and FL heterogeneity when discussing initial therapy of high tumor burden disease. She provides the data to assist patients who have achieved complete metabolic remission with prognostication and life planning after initial immunochemotherapy.

In this talk, Dr. Casulo will review the origins of POD24 and its impact on survival outcomes in follicular lymphoma. She will review currently available biomarkers and risk calculators for POD24, providing updates on the pathobiology influencing follicular lymphoma prognosis and implicated in POD24. This talk will also explore the impact of histologic transformation on survival in POD24 and outline future directions for management of POD24 in the current era.

Dr. Bartlett will discuss the activity and safety of anti-CD3xCD20 bispecific antibody T-cell engagers and anti-CD19 autologous chimeric antigen receptor T-cells for relapsed or refractory follicular lymphoma. She will review the pivotal clinical trial data, highlighting depth and durations of responses as well as toxicities including cytokine release syndrome, infections, and neurologic events. She will describe a framework for sequencing these classes of agents, additionally taking into account patient factors and logistical considerations.

Chair:

Judith Trotman, FRACP
Concord Repatriation General Hospital
Concord,  NSW, Australia

Speakers:

Judith Trotman, FRACP
Concord Repatriation General Hospital
Concord,  NSW, Australia
In Pursuit of a Functional Cure for Follicular Lymphoma

Carla Casulo, MD
James P. Wilmot Cancer Center
Rochester,  NY
The POD24 Challenge: Where Do We Go From Here for Early Progressors?

Nancy L. Bartlett, MD
Washington University School of Medicine
Saint Louis,  MO
Sequencing Bispecific Antibodies and CAR T-cells for FL

Handling Bad News: How to Best Manage TP53 Myeloid Disease

Saturday, December 7, 2024, 4:00 p.m. - 5:15 p.m. Manchester Grand Hyatt San Diego, Grand Hall D

Mutations of TP53 are present in 10-15% of cases of de novo acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) and approximately 30% of cases of therapy-related myeloid neoplasms. TP53-mutated myeloid disease is associated with complex cytogenetic abnormalities and poor outcome, with a median survival of only 5-10 months. In this educational session, we will explore the impact of TP53 mutation type and number on outcomes and clinical management.  We will review current and emerging treatment approaches for this high-risk subtype of AML/MDS.

Dr. Daniel Link will discuss evidence that TP53 hotspot mutations have dominant negative or gain-of function properties and discuss the potential impact on outcome and clinical management. He will review recent data establishing the importance of determining TP53 mutant allele status (monoallelic versus multi-hit). 

Dr. Marina Konopleva will review current non-transplant therapeutic approaches for TP53-mutated myeloid disease and discuss new therapies on the horizon, including chimeric antigen receptor therapies, mutated p53 reactivators, Fc fusion protein and monoclonal antibodies targeting various myeloid antigens.

Dr. Hugo Fernandez will discuss the use of allogenic hematopoietic transplant in the treatment of TP53-mutated myeloid disease, including new approaches to tailor conditioning and use maintenance therapy after transplantation.

Chair:

Daniel C Link, MD
Washington University School of Medicine in St. Louis
Saint Louis,  MO

Speakers:

Daniel C Link, MD
Washington University School of Medicine in St. Louis
Saint Louis,  MO
Are TP53 Mutations All Alike?

Marina Konopleva, MD
Albert Einstein College of Medicine
Houston,  TX
Non-transplant Treatment Approaches for High-risk TP53 Myeloid Disease

Hugo Francisco Fernandez, MD
Moffitt Malignant Hematology and Cellular Therapy
Pembroke Pines,  FL
Transplant Options and Outcomes for TP53 Myeloid Disease

Hematologic Challenges at the End-of-Life

Saturday, December 7, 2024, 2:00 p.m. - 3:15 p.m. San Diego Convention Center, Room 6A

Management of hematologic conditions for patients at the end of life is both a challenge and an opportunity to provide evidence-based, compassionate care. Care of seriously ill patients entails specific considerations—including incorporating life expectancy, addressing symptom burden, and navigating hospice—that apply across the spectrum of hematologic diseases. Further, there is a growing, multidisciplinary evidence base to support treatment decision-making that this session will review. First, we will discuss optimization of end-of-life care in transfusion-dependent hematologic malignancies. We will then focus on a strategy for management of anticoagulant medications in those with limited life expectancy. Finally, we will address prognostication and palliative care in sickle cell disease.

Dr. Loh will highlight challenges and barriers in implementing timely end-of-life-care and potential strategies to overcome these barriers for patients with transfusion-dependent hematologic malignancies.

Dr. Park's talk "Anticoagulation at the End of Life: Whether, When, and How to Treat,"  will focus on state-of-the-art evidence from hematology and palliative care science to inform management of anticoagulant medications in patients with life-limiting illness. Dr. Parks will discuss the complexities of anticoagulation at the end of life, opportunities to enhance our knowledge, and outline an approach to care that is based on patients’ values.  

Dr. Johnston will discuss challenges in providing high quality end-of-life care for people with SCD, including disease-related, communication-related, and systemic-related challenges and discuss strategies to overcome these challenges, including use of primary and specialty palliative care.

Chair:

Anna L. Parks, MD
University of Utah Health
Salt Lake City,  UT

Speakers:

Kah Poh (Melissa) Loh, MD
University of Rochester
Rochester,  NY
Challenges in Hospice and End-of-Life Care in the Transfusion-Dependent Patient

Anna L. Parks, MD
University of Utah Health
Salt Lake City,  UT
Anticoagulation at the End of Life: Whether, When, and How to Treat

Emily E Johnston, MD
University of Alabama At Birmingham
Birmingham,  AL
End-of-life Care for People with Sickle Cell Disease: Barriers to and Facilitators of High-Quality Care

Hematologic Management of the Patient with Heavy Menstrual Bleeding

Saturday, December 7, 2024, 2:00 p.m. - 3:15 p.m. San Diego Convention Center, Room 28 A-D

Heavy menstrual bleeding (HBM) has an important impact on the physical, emotional and social well-being, and in adolescents, can be the only clinical sign of an underlying bleeding disorder. Due to several medical, economic and social factors, it remains a significantly underdiagnosed and undertreated condition. Hematologists play an important role in the evaluation and care of patients with anemia from chronic blood loss, and may be the first clinicians to identify HMB as its cause.  This education session will describe a clinical multidisciplinary approach to the evaluation and treatment of patients presenting with HMB, with a special focus on adolescents. It will provide an overview of diagnostic and management strategies to broaden the tools available to hematologists caring for these patients in different clinical settings.

Dr. Allison P Wheeler will discuss the various tools available for the quantification of menstrual blood loss, including those that evaluate the effect of HMB on quality of life; followed by a description of non-surgical (hormonal and non-hormonal) treatments of HMB, their efficacy, contraindications and selection of the most appropriate therapy using shared decision-making.

Dr. Juliana Perez Botero will present a focused approach to laboratory testing (screening and confirmatory) for patients presenting with HMB in whom von Willebrand Disease is suspected, with emphasis on the effect of pre-analytical variables and testing methodology on the results and their clinical interpretation.

Dr. Janice Staber will discuss the value of multidisciplinary clinics specializing in the care of adolescents with HMB, including their critical components and different care delivery models, and share important lessons learned during the development and implementation of one of these clinics.

Chair:

Juliana Perez Botero, MD
Mayo Clinic
Rochester,  MN

Speakers:

Allison P Wheeler, MD/MSCI
Washington Center for Bleeding Disorders
Seattle,  WA
Quantifying Menorrhagia and Overview of Non-Surgical Management of Heavy Menstrual Bleeding

Juliana Perez Botero, MD
Mayo Clinic
Rochester,  MN
Von Willebrand Disease and Heavy Menstrual Bleeding: When and How to Test

Janice Staber, MD
University of Iowa Stead Family Children's Hospital
Iowa City,  IA
Heavy Menstrual Bleeding Clinics for Adolescents

HIT-and-Run: Challenges in Diagnosis and Management of Heparin-Induced Thrombocytopenia

Saturday, December 7, 2024, 4:00 p.m. - 5:15 p.m. San Diego Convention Center, Room 28 A-D

In hospitalized patients, the diagnosis and management of heparin induced thrombocytopenia (HIT) is a recurrent challenge. To this end, this session will 1) provide evidence-based practical guidance in the diagnosis and management of suspected and confirmed HIT; 2) classify anti-platelet factor 4 (PF4) antibody mediated disorders (autoimmune HIT), their relationship to heparin exposure, and the role of rapid PF4 and platelet activation assays in diagnosis; and 3) review alternative management strategies when heparin cessation and non-heparin anticoagulants are insufficient in patients who have refractory HIT, experience acute bleeding, or are planned to undergo cardiac surgery. Emphasizing evidence-based strategies, this session will provide practical approaches and system-level interventions for high-quality care.

Chair:

Toyosi Onwuemene, MD,MS
Duke University
Durham,  NC

Speakers:

Jori E. May, MD
University of Alabama at Birmingham
Birmingham,  AL
Practical Guide to the Diagnosis and Management of Heparin-Induced Thrombocytopenia

Toyosi Onwuemene, MD,MS
Duke University
Durham,  NC
Approaches to Management of HIT in Complex Scenarios, including Cardiac Surgery

Marie Scully, MD
University College London
London,  ENG, United Kingdom
Demystifying Autoimmune HIT: What It Is, When to Test, and How to Treat

Integrating New Therapies into the Management of Classical Heme Disorders

Saturday, December 7, 2024, 2:00 p.m. - 3:15 p.m. San Diego Convention Center, Room 30

This session will provide an overview on the current evidence of different novel therapies for the management of Classical Heme Disorders including ß-Thalassemia, Acute Intermittent Porphyria?(AIP) and anemia in chronic kidney disease (CKD).

Dr. Volker Haase will outline mechanisms of action and pharmacologic properties of HIF-prolyl hydroxylase inhibitors (HIF-PHIs), a new class of oral anemia drugs approved for the use in patients with chronic kidney disease (CKD). He will discuss nondesirable on-target and off-target effects, cardiovascular and other safety concerns, and provide a benefit/risk-based perspective on how this new class of oral drugs might impact current management of anemia in CKD. A clinical case is presented that highlights the clinical complexities and therapeutic challenges in the management of anemia in patients with CKD.

Prof. Taher will highlight persisting unmet needs in patients with non-transfusion-dependent and transfusion-dependent ß-thalassemia. His presentation will overview the current evidence base for luspatercept as a novel disease-modifying agent targeting ineffective erythropoiesis in ß-thalassemia. He will also provide guidance on best-practices for practical application of luspatercept in the real world setting and identify emerging data gaps.

For Dr. Dickey’s presentation, she will review the givosiran clinical trial data and the available clinical literature on givosiran, as well as highlight important unanswered questions.

Chair:

Rakhi P. Naik, MD
Johns Hopkins Hospital
Baltimore,  MD

Speakers:

Volker Hans Haase, MD
Vanderbilt University
Nashville,  TN
Hypoxia-Inducible Factor Activators: A Novel Class of Oral Drugs for the Treatment of Anemia of Chronic Kidney Disease

Kleber Yotsumoto Fertrin, MD,PhD
University of Washington
Seattle,  WA
Luspatercept: A Treatment for Ineffective Erythropoiesis in Thalassemia

Amy Dickey, MD
Massachusetts General Hospital
Boston,  MA
Givosiran: A Targeted Treatment for Acute Intermittent Porphyria

Ironing Out the Wrinkles: Managing Iron Overload in Different Clinical Scenarios

Saturday, December 7, 2024, 2:00 p.m. - 3:15 p.m. San Diego Convention Center, Room 29

Iron homeostasis and porphyrin metabolism must be closely coordinated to ensure efficient biosynthesis of heme. Disturbed coordination is exemplified by sideroblastic anemias, which are characterized by insufficient incorporation of iron into the end product of the prophyrin biosynthesis pathway, resulting in anemia and mitochondrial as well as systemic iron overload. Conversely, iron overload from other causes, like hereditary hemochromatosis, can have unfavourable effects on porphyrin synthesis, provoking porphyria cutanea tarda. The intricate interplay between iron and porphyrin metabolism can be disturbed by inherited or acquired causes. Therapeutic options to mitigate the ensuing clinical symptoms are highlighted by the three speakers of this session.

Dr. Domenico Girelli will discuss hereditary hemochromatosis according to the recent clinically oriented classification. While hyperferritinemia is common in practice, hemochromatosis accounts for few cases, typically with increased transferrin saturation. HFE-related hemochromatosis has a low penetrance, influenced by lifestyle and dysmetabolic factors. Most patients are currently identified in the early phases before life-threatening manifestations. Phlebotomy remains the mainstay for achieving iron depletion, with blood donation being an option for maintenance. The management of rare, severe non-HFE forms, requiring a comprehensive approach, will also be presented.

Dr. Norbert Gattermann will outline the pathophysiology of systemic iron overload in MDS as well as mitochondrial iron accumulation in patients with acquired sideroblastic anemias. He will explain how SF3B1 mutations and the subsequent missplicing and deficiency of ABCB7 may lead to impaired heme synthesis and excess mitochondrial iron. The rationale for iron chelation therapy in MDS will be considered, together with some of its practical aspects. Finally, Dr. Gattermann will assess the extent to which luspatercept might contribute to the management of iron overload in patients with MDS.

Dr. Rebecca Leaf will focus on porphyria cutanea tarda (PCT), a disorder of heme biosynthesis that arises due to inhibition of hepatic uroporphyrinogen decarboxylase in the setting of increased liver iron content and oxidative stress. Patients with PCT invariably have siderosis on liver biopsy and up to 20% are homozygous for the HFE C282Y mutation. PCT manifests as blistering cutaneous lesions on sun-exposed areas, skin fragility, and elevated plasma and urine porphyrins. Treatment includes therapeutic phlebotomy to decrease total body iron levels and hydroxychloroquine, which reduces hepatic porphyrin content.

Chair:

Norbert Gattermann, MD
Heinrich Heine University Dusseldorf
Dusseldorf,  Germany

Speakers:

Domenico Girelli, MD,PhD
University of Verona
Verona,  Italy
Diagnosis and Management of Hereditary Hemochromatosis: Lifestyle Modification, Phlebotomy, and Blood Donation

Norbert Gattermann, MD
Heinrich Heine University Dusseldorf
Dusseldorf,  Germany
Iron Overload in Acquired Sideroblastic Anemias and MDS: Pathophysiology and Role of Chelation and Luspatercept

Rebecca Karp Leaf, MD
Mass General Hospital Cancer Center
Boston,  MA
Porphyria Cutanea Tarda: A Unique Iron-Related Disorder

It's a Different World: CLL 2024

Monday, December 9, 2024, 10:30 a.m. - 11:45 a.m. San Diego Convention Center, Hall B

The treatment of CLL has significantly advanced over the past decade with the introduction of novel targeted therapies. Inhibitors of Bruton’s tyrosine kinase (BTK) as well as BCL2 have supplanted chemoimmunotherapy in both frontline and relapsed therapy. However, while outcomes have improved in the current era, the disease is not cured, and questions still remain about optimal therapies for frontline and relapsed disease. This education session will focus on unanswered questions in CLL, and how current and upcoming research can answer these questions and continue to move the field forward. Dr Stephan Stilgenbauer will discuss risk stratification in CLL and how genomic features influence treatment choice in the frontline setting. He will discuss the implications of genomic risk on outcomes with currently available therapies. Dr Jennifer Woyach will discuss the evidence surrounding doublets and triplets in the frontline treatment of CLL. She will discuss scenarios in which doublets or triplets might be preferred over other standard options, and current and future trials that will help elucidate the use of these treatment for patients. Dr Arnon Kater will discuss the role of immunotherapies in relapsed/refractory CLL. He will discuss the current evidence surrounding transplant, as well as newer paradigms including cellular therapies and bispecific antibodies.

Chair:

Jennifer A. Woyach, MD
The Ohio State University
Columbus,  OH

Speakers:

Stephan Stilgenbauer, MD
University of Ulm
Ulm,  Germany
Risk-Stratification in Frontline CLL Therapy (Standard of Care)

Jennifer A. Woyach, MD
The Ohio State University
Columbus,  OH
The Evolving Frontline Management of CLL: Are Triplets Better Than Doublets? How Will We Find Out?

Arnon P. Kater, MD,PhD
Amsterdam UMC
Amsterdam,  Netherlands
Relapsed/Refractory CLL: The Role of allo-SCT, CAR-T, and T-cell Engagers

Monoclonal Gammopathies: When the Clone Is More Than a Positive Laboratory Finding

Saturday, December 7, 2024, 4:00 p.m. - 5:15 p.m. Marriott Marquis San Diego Marina, Pacific Ballroom Salons 21-22

On the spectrum of plasma cell dyscrasias monoclonal gammopathy of undetermined significance (MGUS) represents the most commonly encountered condition in clinical practice. While it is clinically silent, the premalignant MGUS clone has the potential to progress to multiple myeloma or a related B-cell lymphoproliferative malignancy usually associated with high tumor burden. In contrast, monoclonal gammopathy of clinical significance (MGCS) is characterized by a small quiescent B-cell clone but significant symptoms caused by the monoclonal immunoglobulin affecting the skin, kidneys and/or nervous system.

Dr. Michaela Liedtke will present recent updates in the epidemiology of MGUS and prevalence of associated conditions based on population screening studies. Recommendations for a cost-effective diagnostic and follow-up approach will be discussed as well as diagnostic advances including mass spectrometry and revised light chain criteria. We will review updates in MGUS risk stratification related to genetics and other clone & host factors and discuss evidence for the emerging role of lifestyle interventions. The presentation will conclude with a brief overview of MGCS and highlight the clinical features and international collaboration that led to the discovery of TEMPI syndrome.

Dr Frank Bridoux will present the main characteristics of the different types of renal disorders associated with MGUS and introduce the concept of monoclonal gammopathy of renal significance (MGRS). The classification of MGRS-related renal disorders will be presented as well as the diagnostic work-up. The principles of hematologic and renal management of MGRS will be described.

Dr. Maite Cibeira will talk to us about monoclonal gammopathy of neurological significance with the aim of simplifying the hematologist’s diagnostic approach, in close collaboration with the neurologist. She will also discuss the available treatment strategies taking into account the rarity of these diseases, which makes it difficult to conduct clinical trials and explains the lack of standardized treatment options, as well as the relevance of supportive care in the context of an essential multidisciplinary management.

Chair:

Michaela Liedtke, MD
Stanford University Cancer Center
Stanford,  CA

Speakers:

Michaela Liedtke, MD
Stanford University Cancer Center
Stanford,  CA
MGUS Where Do We Stand Today?

Frank Bridoux, MD, PhD
CHU Poitiers
Poitiers,  France
Renal Manifestations of MGUS

M Teresa Cibeira López, MD, PhD
Hospital Clinic of Barcelona, University of Barcelona, IDIBAPS
Barcelona,  Spain
Neurological Manifestations of MGUS

Moving the Needle in Hodgkin Lymphoma

Saturday, December 7, 2024, 9:30 a.m. - 10:45 a.m. Marriott Marquis San Diego Marina, Pacific Ballroom Salons 15-17

After decades of small incremental shifts in the management of classic Hodgkin lymphoma (cHL), recent studies have accelerated the pace of change in the treatment of cHL. While brentuximab vedotin (BV) has become a standard part of initial treatment for advanced stage disease, studies using PD-1 blockade in the frontline setting are leading to a re-examination of the role of these novel agents for the treatment of cHL. Meanwhile, the novel agents have led to improved outcomes for patients with relapsed/refractory cHL, leading to questions about the timing and role of stem cell transplantation and whether novel therapies alone can lead to durable remission. Dr. Lynch will address whether recent studies using PD1 blockade in frontline treatment of cHL have established a new standard of care for cHL. Dr. Advani will examine the role of BV in the initial treatment of cHL as well as for relapsed/refractory cHL in light of the recent studies in the frontline and salvage setting. Dr. Perales will discuss the evolving role of stem cell transplantation for cHL, optimal salvage regimens, timing and patient selection.

Chair:

Alex F. Herrera, MD
City of Hope
Duarte,  CA

Speakers:

Ryan C. Lynch, MD
Fred Hutchinson Cancer Research Center
Seattle,  WA
Has PD1 Blockade Changed the Standard of Care for cHL?

Ranjana H. Advani, MD
Stanford University
Stanford,  CA
When Should We Use It? The Role of Brentuximab Vedotin in 2024

Miguel Angel Perales, MD
Memorial Sloan Kettering Cancer Center
New York,  NY
When to Use Stem Cell Transplantation for Classical Hodgkin Lymphoma

MPN Practice Pearls: Profiling, Pegylated IFN and Pregnancy

Sunday, December 8, 2024, 9:30 a.m. - 10:45 a.m. Manchester Grand Hyatt San Diego, Seaport Ballroom EFGH

This MPN session focuses on issues of clinical relevance while emphasizing disease biology. The clinical care of patients with MPN is evolving, with increasing evidence indicating that molecular genetic data is an important component of care, both at diagnosis and during follow up. Approximately three years after the approval of ropeginterferon by the Food and Drug Administration (FDA) for the treatment of Polycythemia Vera (PV), it is timely to review the role of pegylated interferon in the treatment of MPN. Finally, as the obstetric population with MPN increases, understanding how to optimize care for patients with MPN during pregnancy is increasingly relevant. Attendees can expect to learn MPN practice pearls in all three sessions! 

Dr. Mead will discuss current approaches to molecular profiling of MPN patients, using illustrative clinical case histories to demonstrate how genetic analysis is already fully integrated into MPN diagnostic classification and prognostic risk stratification. He will discuss how molecular profiling can also be used in MPN to measure response to therapy both in clinical trials and routine clinical practice. Taking a forward look, he will discuss how molecular profiling in MPN might be used in the future to select specific molecularly targeted therapies and the role of additional genetic methodologies beyond mutation analysis.

 

Dr. Kiladjian will discuss the use of pegylated interferon for the treatment of MPN and review the different types of pegylated interferon currently available. He will review in which MPN subtypes pegylated interferon has the greatest clinical efficacy and discuss both hematological and molecular response. He will review the mechanism of action of pegylated interferon and link this to potential for disease modification and prevention of MPN progression. Finally, he will review the limitations of clinical studies with respect to their ability to measure MPN progression, as well as the need for more comprehensive assessment of molecular response.

Dr. Chi-Joan How will discuss practices around the management of pregnancy in MPN patients. As the obstetric population ages and MPNs are being increasingly diagnosed in younger patients, pregnancy in MPNs will become more frequently encountered. She will discuss current data surrounding pregnancy outcomes in MPNs and current recommendations for the use of anti-platelet agents, anticoagulation, and cytoreduction in the ante- and post-partum period. Using a case-based approach, Dr. How will suggest best practices in areas with limited data.

Chair:

Ann Mullally, MD
Stanford University School of Medicine
Boston,  MA

Speakers:

Adam J Mead, MBBChir
University of Oxford
Oxford,  ENG, United Kingdom
Molecular Profiling in MPN: Who Should Have It and Why?

Jean-Jacques Kiladjian, MD,PhD
Hopital Saint-Louis
Paris,  France
Pegylated Interferon: The Who, Why, and How

Chi-Joan How, MD
Brigham and Women's Hospital
Boston,  MA
Pregnancy: MPN Management Before, During, and After Pregnancy

Newly Diagnosed Multiple Myeloma: Many Choices and More Questions

Saturday, December 7, 2024, 2:00 p.m. - 3:15 p.m. Marriott Marquis San Diego Marina, Pacific Ballroom Salons 24-26

The treatment of newly diagnosed myeloma has evolved rapidly with the new therapies and different combinations of these, resulting in deeper and more durable responses. The use of autologous stem cell transplantation has also evolved with this progress, and increasingly the initial treatment is being determined agnostic of the transplant eligibility and or desirability. In the decision making process, two factors play an important role, namely the underlying disease risk and the patient functional status or frailty. In particular, increasing disase risk calls for more intense treatments with the goal of achieving MRD negativity while frailty often limits the intensity of the combinations. Phase 3 trials continue to explore the role of quadruplet regimes in older patient populations with increasing depth of response and durable responses, and the nature of the combinations will evolve in the coming years with new classes of drugs being introduced, especially immunotherapies.

Autologous stem cell transplant has been an integral part of myeloma therapy for the past three decades. Originally envisaged as consolidation following initial treatment of newly diagnosed MM in those eligible to undergo the procedure, its role has evolved over time with investigations exploring various aspects including the types of conditioning regimens, role of post-transplant maintenance as well as tandem autologous stem cell transplantation. While the original phase 3 trials compared ASCT with no ASCT and demonstrated improved overall survival, subsequent trials studied the paradigm of delayed ASCT at the time of first relapse, demonstrating significant improvement in PFS but no OS advantage. As the initial therapies for treatment of myeloma has improved with high rate of deep responses, the role of ACT have continued ot be debated, but have maintained its position in the treatment with most recent trials continuing to show an improvement in PFS compared with no ASCT. Dr. Perrot's talk will focus on how ASCT is positioned in the treatment paradigm of MM.  

The overall survival in patients with MM over recent decades. This trend is anticipated to further advance with the emergence of T-cell redirecting therapies, including chimeric antigen receptor T-cell (CAR-T) therapy and T-cell engaging bispecific antibodies. Despite these therapeutic advancements, treatment-related adverse events likely impede quality of life. This underscores the imperative of optimizing supportive care strategies to maximize treatment outcomes. Such optimization is crucial not only for patient well-being but also for treatment adherence to sustain long-term disease control. Dr. Zweegmen will discuss treatment and prevention of bone disease, state of the art thrombosis prophylaxis and supportive care during T-cell redirecting therapies targeting BCMA and GPRC5d, amongst which prevention of infections.

Chair:

Shaji Kumar, MD
Mayo Clinic
Rochester,  MN

Speakers:

Shaji Kumar, MD
Mayo Clinic
Rochester,  MN
What Is the Ideal Approach – Doublet, Triplet or Quadruplet(s)?

Aurore Perrot, MD, PhD
CHU de Toulouse, IUCT-O, Université de Toulouse, UPS, Service d’Hématologie
Toulouse,  France
Transplant in Myeloma: Who, When, and Why?

Sonja Zweegman, MD, PhD
Amsterdam University Medical Center, Vrije Universiteit Amsterdam
Amsterdam,  Netherlands
Supportive Care in Myeloma – When Treating the Clone Alone Is Not Enough

Not All Lymphadenopathy is Lymphoma: Lymphoproliferative Diseases of Immune Dysregulation

Saturday, December 7, 2024, 9:30 a.m. - 10:45 a.m. Marriott Marquis San Diego Marina, Pacific Ballroom Salons 21-22

Immune dysregulation often causes lymphadenopathy that requires clinicopathological correlation for accurate diagnosis.  This session will review important causes of lymphadenopathy that fall outside the typical spectrum of malignant lymphoma.  Castleman disease, IgG4-Related disease and the relationship between EBV and Post-transplant Lymphoproliferative Disorder will be reviewed.  A common theme among these sessions is the importance of combining clinical, laboratory, and pathological “clues” to arrive at the best possible diagnosis and management plan.

Dr. Fajgenbaum will provide an overview on the classification, diagnosis, and management of Castleman disease.  Castleman disease is a heterogeneous group of lymphoproliferative disorders, and idiopathic multicentric Castleman disease (iMCD) can be particularly difficult to diagnose and distinguish from other inflammatory causes of lymphadenopathy.  Diagnostic criteria for iMCD and the subtypes of iMCD including thrombocytopenia, anasarca, (reticulin) fibrosis, renal dysfunction and organomegaly (iMCD-TAFRO), idiopathic plasmacytic lymphadenopathy (iMCD-IPL) and not otherwise specified (iMCD-NOS) will be reviewed.

Dr. Chen will review IgG4-related disease (IgG4-RD), a disease of immune dysregulation with protean manifestations including autoimmune pancreatitis, sclerosing cholangitis, lacrimal and salivary gland swelling, and tubulointerstitial nephritis.  The session will focus on an approach to four key manifestations for Hematologists:  Polyclonal hypergammaglobulinemia, IgG4-positive plasma cell enriched lymphadenopathy, eosinophilia, and retroperitoneal fibrosis.  Practical tips for differentiating IgG4-RD from key mimickers such as Castleman disease, eosinophilic vasculitis, and histiocyte disorders will be highlighted.

Dr. Rouce will discuss the interplay between Epstein-Barr virus (EBV) and Post-tranplant lymphoproliferative disorder (PTLD).  The spectrum of PTLD includes three variations of lymphoproliferation:  lymphoid hyperplasia, neoplasia and malignancy.  Lymphoid hyperplasia is characterized by polyclonal proliferation and non-destructive lesions whereas lymphoid malignancy features monoclonal proliferation and destructive lesions. EBV is the main driver of PTLD, and management ranges from reduction in immunosuppression to intensive, lymphoma-directed therapy.

Chair:

Luke Y. C. Chen, MD,MEd
Dalhousie University
Halifax,  NS, Canada

Speakers:

David C. Fajgenbaum, MD
University of Pennsylvania
Philadelphia,  PA
Identifying Castleman Disease from Non-Clonal Inflammatory Causes of Generalized Lymphadenopathy

Luke Y. C. Chen, MD,MEd
Dalhousie University
Halifax,  NS, Canada
IgG4-Related Disease for the Hematologist

Rayne Rouce, MD
Texas Children's Hospital
Houston,  TX
EBV and Post-transplant Lymphoproliferative Disorder (PTLD): A Complex Relationship

Optimizing Non-Curative Therapies for Sickle Cell Disease

Saturday, December 7, 2024, 9:30 a.m. - 10:45 a.m. San Diego Convention Center, Room 33

Despite expanding curative options, ineffective disease modifying medication use, acute pain management, and care delivery remain the primary challenges for the vast majority of individuals with sickle cell disease (SCD). This educational session will provide a practical guide for effectively using disease modifying medications, the best practices on delivering multi-modal acute pain management, and examples of successful, real-world adaptive clinical care models. Finally, it will discuss the remaining research questions to answer in these areas to optimize the outcomes of individuals with SCD.

Dr. Creary will discuss the disease modifying medications for SCD. The talk will offer practical guidance on how to overcome common treatment barriers and to use disease modifying medications in a way that balances the unique characteristics of these medications and the patients’ desired outcomes.

Dr. Brandow will discuss alternative methods for acute vaso-occlusive pain management. The talk will review recent research that support using non-opioid and non-pharmacologic interventions and the care delivery processes that have been shown to improve acute SCD pain outcomes

Dr. Bartolucci will discuss updating the existing clinical care models to be more personalized, adaptive, and collaborative, in response to the diverse experiences for those with SCD. The talk will discuss challenges in different income countries and the need for appropriate biomarkers and predictive algorithms.

Chair:

Susan Elizabeth Creary, MD,MSc
Ohio State University College of Medicine
Columbus,  OH

Speakers:

Susan Elizabeth Creary, MD,MSc
Ohio State University College of Medicine
Columbus,  OH
Practical Guide for Disease-Modifying Medication of Children and Adolescents with Sickle Cell Disease

Amanda M. Brandow, DO,MS
Medical College of Wisconsin
Milwaukee,  WI
Beyond IV Push: Alternative Methods for Management of Acute Pain in SCD

Pablo Bartolucci, MD,PhD
APHP UPEC IMRB
Créteil,  France
Novel Clinical Care Models for Patients with Sickle Cell Disease

Overcoming Post-transplant Relapse: How to Win the Battle Royale

Sunday, December 8, 2024, 9:30 a.m. - 10:45 a.m. San Diego Convention Center, Room 6CF

Over the last few decades, significant progress in reducing infectious and immune-related complications of allogeneic hematopoietic cell transplantation have improved the feasibility and overall outcome of this complex procedure. Still, in patients patients transplanted for malignant diseases, reappearance of the original cancer, or relapse, continue to represent a crucial clinical issue. This educational session will present current knowledge on the biology of relapse, on how to tailor the transplant protocol to reduce its incidence, and on the use of novel drugs to prevent or treat recurrence.

Dr. Luca Vago will outline available evidence on the dynamic changes occurring in cancer cells and the patient immune system upon allogeneic hematopoietic cell transplantation, with a particular focus on how key aspects of the transplant such as donor choice and use of specific drugs impact on disease evolution, and on how state-of-the-art profiling of relapsed disease can help selecting appropriate countermeasures.

Dr. Ito's session begins by highlighting the current evidence of maintenance therapy for post-transplant relapse prevention, including targeted therapies, hypomethylating agents, venetoclax, and immunotherapies. The talk then shifts the focus to the role of relapse monitoring, emphasizing the importance of measurable residual disease and donor chimerism. Finally, the session will review the salvage therapies for overt relapse, including donor lymphocyte infusion, second allogeneic stem cell transplantation, and selected agents under investigation.

Dr. Pierini will speak on novel conditioning and prophylaxis regimens for relapse prevention.

Chair:

Luca Vago, MD,PhD
IRCCS Ospedale San Raffaele
Milano,  Italy

Speakers:

Luca Vago, MD,PhD
IRCCS Ospedale San Raffaele
Milano,  Italy
Biology of Transplant Relapse: Actionable Features

Antonio Pierini, MD,PhD
University of Perugia
Perugia,  Italy
Novel Conditioning and Prophylaxis Regimens for Relapse Prevention

Sawa Ito, MD,PhD
University of Pittsburgh
Pittsburgh,  PA
Maintenance Strategies for Relapse Prevention and Treatment

Sex Hormones, Contraceptives, and Thrombotic Risk: Where Are We Now?

Monday, December 9, 2024, 2:45 p.m. - 4:00 p.m. San Diego Convention Center, Room 29

Venous thromboembolism is a common disease, with an incidence of one to three per 1000 individuals per year. Numerous risk factors are known, which can be divided into genetic and acquired. One of the most well-known acquired risk factors is exposure to endogenous (i.e. pregnancy) and exogenous female hormones, including oral contraceptive use, hormone substitution therapy, and gender-affirming hormone therapy. Thrombosis specialists are frequently consulted about optimal hormonal therapy in individuals with an increased risk of venous thromboembolism, as well as optimal management of pregnancy-related venous thromboembolism.

Dr Leslie Skeith will cover contraceptive-related VTE risk by agent and by clinical scenario, including in patients with inherited thrombophilia, systemic lupus erythematosus with or without antiphospholipid antibodies or antiphospholipid syndrome, and sickle cell disease considering contraception. Relevant clinical practice guidelines are reviewed. A multidisciplinary approach to counselling is needed for patient-focused decision-making.

Dr Joseph Shatzel will explore the hematologic complications of gender-affirming therapy, with a particular focus on managing and mitigating the thrombotic risks linked to exogenous estrogen use. He will detail existing data on thrombotic risks reported in transgender individuals and provide insights from studies involving cisgender women and men who receive exogenous estrogen and testosterone, providing important contextual background. He will delve into strategies for addressing testosterone-induced erythrocytosis and iron deficiency in transgender individuals undergoing hormone therapy.

Dr Saskia Middeldorp will present clinical cases of VTE related to hormonal contraceptive use and pregnancy to illustrate key considerations for clinical practice. Practice points for primary VTE treatment and the evidence on the risk of recurrent VTE and bleeding in this population are detailed. The potential value of thrombophilia testing is described including the: “who, why, when, what and how”. We also discuss key aspects of shared decision making for anticoagulant duration, including a reduced dose anticoagulant strategy in hormone-related VTE.

Chair:

Saskia Middeldorp, MD, PhD
Radboud university medical center
Nijmegen,  Netherlands

Speakers:

Leslie Skeith, MD
University of Calgary
Calgary,  AB, Canada
Estrogen, Progestin, and Beyond: Thrombotic Risk and Contraceptive Choices

Joseph J. Shatzel, MD
Oregon Health and Science University
Portland,  OR
Gender-Affirming Hormone Therapy in the Transgender Patient: Influence on Thrombotic Risk

Saskia Middeldorp, MD, PhD
Radboud university medical center
Nijmegen,  Netherlands
Hormone-Related Thrombosis: Duration of Anticoagulation, Risk of Recurrence, and the Role of Hypercoagulability Testing

The Expanding Landscape of Treatment for ITP

Saturday, December 7, 2024, 9:30 a.m. - 10:45 a.m. San Diego Convention Center, Room 28 A-D

The management of Immune Thrombocytopenia (ITP) has seen a renaissance in the available therapies and in the approach to patients with the diagnosis in recent years. This has lead to an expanding number of therapeutic options for patients and physicians to consider as they embark on their therapeutic journey with ITP. Dr. Waleed Ghanima will discuss the new approaches incorporating combination strategies in management of patients with ITP including suggestions for when to consider moving beyond mono therapy for ITP. Dr. Annemarie Fogerty will then discuss updates on ITP in pregnancy, a special category of ITP with implications for both maternal and fetal health and provide the most information on both treatments and outcomes. Finally, Dr. Michele Lambert will then describe current clinical trials (or recently completed) and provide information on novel therapeutics that are in development as well as the current treatment gaps where additional study is needed.

Chair:

Michele P. Lambert, MD
Children's Hospital of Philadelphia
Philadelphia,  PA

Speakers:

Waleed Ghanima, MD,PhD
Østfold Hospital
Gralum,  Norway
Insights on Treatment of Adult ITP: Algorithm for Management and Role of Multimodal Therapy

Annemarie E Fogerty, M.D.
Massachusetts General Hospital
Boston,  MA
ITP in Pregnancy: Diagnostics and Therapeutics in 2024

Michele P. Lambert, MD
Children's Hospital of Philadelphia
Philadelphia,  PA
On the Horizon: Upcoming New Agents for the Management of ITP

Vascular Anomalies, Vascular Malformations, and the Role of the Hematologist

Monday, December 9, 2024, 4:30 p.m. - 5:45 p.m. San Diego Convention Center, Room 25

Vascular anomalies have traditionally been managed by surgical and interventional radiology colleagues. However, an evolving understanding of the pathophysiology, genetic landscape and hematologic outcomes will necessitate involvement from hematologists and oncologists for comprehensive care. This session will explore the molecular underpinnings and genetic factors of these conditions, highlighting the shift towards targeted medical therapies informed by recent discoveries in crucial signaling pathways. The talks will also cover the management of hemostasis and thrombosis risks, stressing the need for a multidisciplinary approach to diagnostics and treatment to enhance patient safety and outcomes. Together, these talks will shed light on the latest scientific advancements and their translation into clinical practices highlighting the pivotal role hematologists and oncologists play in this specialized care.

Dr. Vikkula will present an overview of the current understanding of the pathophysiological foundations of vascular anomalies. He will examine how genetic associations across various clinical cases inform the diagnostic work-up and classification of these conditions. Additionally, he will discuss the pathophysiological effects of the characterized pathogenic variants, which underlie the basis of developing targeted medical therapies for vascular anomalies.

Dr. Borst’s talk will review currently available medical therapies for vascular anomalies, review guidelines for drug dosing and monitoring, and introduce some novel therapeutics. The discovery of somatic pathogenic variants in the PI3K/AKT/mTOR and Ras/MAPK intracellular signaling pathways as drivers of vascular anomalies has led to a new era of precision medicine for these malformations and tumors. Hematologists/oncologists are critical to the care of patients with vascular anomalies, owing to expertise in managing targeted medical therapies.

Dr. Crary’s talk will focus on the evolving role of hematologists in diagnosing and managing consumptive coagulopathy associated with vascular anomalies such as kaposiform hemangioendothelioma and slow-flow vascular malformations She will emphasize the importance of interdisciplinary approaches, especially in peri-operative settings, to prevent complications like bleeding and venous thromboembolism. The talk illustrates the use of clinical signs, imaging, laboratory findings, and anticoagulation therapy to improve patient outcomes.

Chair:

Shelley E Crary, MD,MS
Arkansas Children's Hospital
Little Rock,  AR

Speakers:

Miikka Vikkula, MD, PhD
Universite Catholique De Louvain
Brussels,  Belgium
Molecular Landscape and Classification of Vascular Anomalies

Alexandra Jane Borst, MD
The University of North Carolina at Chapel Hill
Chapel Hill,  NC
Targeted Medical Therapies for Vascular Anomalies

Shelley E Crary, MD,MS
Arkansas Children's Hospital
Little Rock,  AR
Hemostasis and Thrombosis Risks and Management in Vascular Anomalies

Education Spotlight Sessions

Curative Therapies for Sickle Cell Disease: Option for Some but Not Quite All

Sunday, December 8, 2024, 4:30 p.m. - 5:45 p.m. San Diego Convention Center, Room 30

Despite substantial improvements in care, individuals with sickle cell disease continue to experience substantial morbidity and a shortened life expectancy. With recent advances in the development of curative therapies, including allogeneic stem cell transplantation and gene therapy, it is important to identify barriers to accessing these treatments and adequately define suitable candidates for curative therapies.

In this session, Dr. Santosh Saraf will review the current guidelines for curative therapies, discuss the potential benefits and risks of the different curative therapies, and provide insight on when to refer patients for curative therapy.

In the second presentation, Dr. Payal Desai will discuss the various barriers to accessing curative therapies as well as the need for development of an integrated care approach.

Chair:

Payal Desai, MD
Atrium Health
Charlotte,  NC

Speakers:

Santosh L. Saraf, MD
University of Illinois Chicago
Chicago,  IL
Curative Therapies in Sickle Cell Disease: Which Patient, What Modality, and When to Refer

Payal Desai, MD
Wake Forest University
Charlotte,  NC
Systemic Barriers to Curative Therapy and Need for Integrated Care

Gene Therapy and Hemophilia A: What Is the Future of Curative Therapy in the Age of Emicizumab?

Monday, December 9, 2024, 2:45 p.m. - 4:00 p.m. San Diego Convention Center, Room 24

There have been marked advancements in the treatment of hemophilia A over the past decades.  Adoption of routine prophylaxis to prevent bleeding as the standard of care has led to many children growing into adulthood with normal joint function and adults with improved function and quality of life.  However, there remains a continued treatment burden and bleeding events, including spontaneous hemorrhage. Newer therapeutics with regulatory approval and in late phase clinical trial may further decrease the treatment burden and improve efficacy.  This session will provide an update on gene therapy for hemophilia and discuss gene therapy in the context of new and coming therapies. 

Frank W.G. Leebeek, MD, PhD
The Promise of Gene Therapy for Severe Hemophilia A

In the past decade enormous improvements have been made in delivering AAV-based gene therapy in patients with hemophilia A. In several phase 1 and 3 studies patients reached normal levels of FVIII after a single dose of AAV-delivered FVIII-gene construct, leading to reduced bleeding rates and patients could stop regular FVIII prophylaxis. During follow up of more than five years FVIII activity declined over time, however the majority of patients still have residual FVIII expression enabling them to refrain for prophylaxis. These promising results led to FDA and EMA approval of gene therapy for hemophilia A in 2022.  Longer term follow-up is needed to show us the duration of benefits and (potential) drawbacks of gene therapy compared to current and other novel treatments.

Christine L Kempton, MD, MSc
Unknowns of Gene Therapy and Successes of Medical Therapies for Hemophilia A

Although gene therapy has held the promise of a cure, a consistent cure has yet to be realized. Additionally, the beneficial results seen in hemophilia A gene therapy clinical trials have occurred with meaningful challenges. This talk will review the risks and benefits of gene therapy for hemophilia A and consider them within the context of therapies (emicizumab and Fc-VWF-XTEN fusion protein-eht) that have shown consistent benefit compared with previously available factor VIII products as well as other promising therapies (Mim8, fitusiran, concizumab, and marstacimab) in late-stage clinical trials.

Chair:

Barbara A. Konkle, MD
Bloodworks Northwest
Seattle,  WA

Speakers:

Frank W.G. Leebeek, MD, PhD
Erasmus university Medical Center
Rotterdam,  Netherlands
The Promise of Gene Therapy for Severe Hemophilia A

Christine L Kempton, MD
Emory University
Atlanta,  GA
Unknowns of Gene Therapy and Successes of Medical Therapies for Hemophilia A

Making a Match: Optimal Donor HLA and Beyond

Monday, December 9, 2024, 10:30 a.m. - 11:45 a.m. San Diego Convention Center, Room 24

Selection of the optimal donor for allogeneic hematopoietic cell transplantation (allo-HCT) involves a comprehensive evaluation of several critical factors. Human leukocyte antigen (HLA) matching has proved to be the most likely crucial parameter in this process. However, several additional criteria can significantly influence allo-HCT outcomes.

Dr. Heather Stefanski will discuss the patient’s likelihood and probability of having a fully HLA-matched donor and finding a suitable donor prior to allo-HCT. She will summarize the historical barriers to transplant, especially for racially and ethnically diverse patients. A special focus will be put on new strategies, including post-transplant cyclophosphamide and abatacept, which can allow for safe and effective use of HLA-mismatched donor sources such as haploidentical donors and mismatched unrelated donors.  Dr. Stefanski will highlight the research evidence that shows alternative donors are a viable and safe option for many allo-HCT recipients.

Dr. Mohamad Mohty will discuss the non-HLA factors which can contribute to further optimizing the selection of a donor prior to allo-HCT. These factors include the age of the donor (especially data favoring younger donors), recipient-donor gender mismatch and its impact on graft-versus-host disease, cytomegalovirus serostatus, and blood type compatibility. Dr Mohty will also briefly discuss the potential role of killer immunoglobulin-like receptor (KIR) matching, and donor-recipient kinship.

By integrating HLA and non-HLA variables into the donor selection process, one can significantly enhance the outcome of patients who are candidates for allo-HCT.

Chair:

Mohamad Mohty, MD,PhD
Saint-Antoine Hospital
Paris,  France

Speakers:

Heather E. Stefanski, MD,PhD
CIBMTR® (Center for International Blood and Marrow Transplant Research), NMDP
Minneapolis,  MN
HLA: Optimizing the ’Full HLA Match’

Mohamad Mohty, MD,PhD
Saint-Antoine Hospital
Paris,  France
Beyond HLA: Optimizing Non-HLA Variables

Smoldering Myeloma: A Case for Early Intervention?

Monday, December 9, 2024, 10:30 a.m. - 11:45 a.m. Marriott Marquis San Diego Marina, Pacific Ballroom Salons 18-19

Smoldering Multiple Myeloma (SMM) is a precursor plasma cell disorder characterized by the presence of an M-component concentration equal to or greater than 3 g/dL and/or bone marrow plasma cell infiltration of 10-59%, without any myeloma-defining events. The outcome is heterogeneous, especially regarding progression to active disease such as Multiple Myeloma or other plasma-cell related disorders. The current standard of care is observation, but ongoing and completed research suggests it is possible and straightforward to identify SMM patients at high risk of progression to Multiple Myeloma (50% at 2 years).

This session will feature two clinical cases that highlight the issues involved in determining whether early treatment is the optimal approach for managing this precursor plasma cell disorder and if early treatment is the only way to potentially cure Multiple Myeloma.

Dr. Mateos will discuss the role of early treatment in Smoldering Myeloma as a strategy to cure the disease. She will present evidence supporting the benefits of early intervention and its impact on long-term outcomes.

Dr. Ramasamy will provide a counterpoint to this approach, addressing the costs and toxicities associated with early treatment. He will discuss the potential downsides and argue for a more conservative management approach based on observation.

We look forward to a dynamic and educational session that will provide valuable insights into the management of Smoldering Multiple Myeloma

Chair:

Maria-Victoria Mateos, MD, PhD
University Hospital of Salamanca/IBSAL/Cancer Research Center-IBMCC (USAL-CSIC)
Salamanca,  Spain

Speakers:

Maria-Victoria Mateos, MD, PhD
University Hospital of Salamanca
Salamanca,  Spain
Point: Yes, That is the Only Way We Can Cure Myeloma?

Karthik Ramasamy, MD,PhD
University of Oxford
Oxford,  United Kingdom
Counterpoint: No, It Just Adds Cost and Toxicity?

The Clone Wars: Myeloid Neoplasia Risk in Clonal Hematopoiesis

Sunday, December 8, 2024, 4:30 p.m. - 5:45 p.m. Manchester Grand Hyatt San Diego, Grand Hall C

Clonal expansions in the hematopoietic system, referred to as clonal hematopoiesis (CH), is common throughout life and associated with significant clinical outcomes, including increased risk of hematologic cancers, all-cause mortality and nonmalignant conditions. Recently, clinical definitions of disorders within the spectrum of CH have been introduced, including clonal hematopoiesis of indeterminate potential (CHIP) and clonal cytopenia of undetermined significance (CCUS), providing the framework for harmonized classification and registration. However, while clone features and dynamics have been well described, relevant key issues remain to be clarified.

In the first presentation, Dr. Malcovati will provide an overview of biological underpinnings and clinical implications of CH. In addition, the spectrum of clonal cytopenias, as well as current classification framework and its pitfalls, will be critically discussed. Finally, the contribution of intra- and extra-clonal factors to clonal evolution, and the models developed for predicting the risk of developing myeloid neoplasms and the trajectory of evolution will be reviewed, as a basis for informing early-detection and prevention strategies.

In the second presentation, Dr Grønbæk will discuss the clinical management of CH in everyday clinical practice, focusing on how individual/patients with CH are identified, including the incidental finding, which emphasizes the need for informed consent before sequencing. She will elaborate on the importance of identifying and following high-risk individuals/patients while sparing low risk individuals unnecessary worries. The importance of enrolling these individuals/patients in clinical trials will be discussed including the optimal trials designs. Examples of ongoing trials will be presented and critically appraised.

Chair:

Luca Malcovati, MD
University of Pavia and S. Matteo Hospital
Pavia,  Italy

Speakers:

Luca Malcovati, MD
University of Pavia
Piazzale Golgi 2,  Italy
Predicting Risk of MN in CH

Kirsten Gronbaek, MD, PhD, PR
Kirsten Grønbæk
Copenhagen N,  Denmark
How to Follow CH Patients

How I Treat

How I Treat: A Personal Journey with Castleman Disease (ticketed session for in-person participants)

Sunday, December 8, 2024, 8:00 a.m. - 9:00 a.m. Hilton San Diego Bayfront, Aqua 300

How I Treat: Acute Lymphoblastic Leukemia Incorporating New Treatment Options (ticketed session for in-person participants)

Saturday, December 7, 2024, 8:00 a.m. - 9:00 a.m. Hilton San Diego Bayfront, Aqua 314

How I Treat: Acute Myeloid Leukemia with Targetable Mutations (ticketed session for in-person participants)

Sunday, December 8, 2024, 8:00 a.m. - 9:00 a.m. Hilton San Diego Bayfront, Aqua 314

How I Treat: Adult Outpatient CAR T Therapy (ticketed session for in-person participants)

Sunday, December 8, 2024, 8:00 a.m. - 9:00 a.m. Hilton San Diego Bayfront, Aqua 311

How I Treat: Challenges and Management of Cancer Associated Thrombosis (ticketed session for in-person participants)

Saturday, December 7, 2024, 8:00 a.m. - 9:00 a.m. Hilton San Diego Bayfront, Aqua 300

How I Treat: Follicular Lymphoma Incorporating New Treatment Options (ticketed session for in-person participants)

Saturday, December 7, 2024, 8:00 a.m. - 9:00 a.m. Hilton San Diego Bayfront, Aqua 313

How I Treat: Gene Therapy in Sickle Cell Disease (ticketed session for in-person participants)

Saturday, December 7, 2024, 8:00 a.m. - 9:00 a.m. Hilton San Diego Bayfront, Aqua 311

How I Treat: Newly Diagnosed Multiple Myeloma (ticketed session for in-person participants)

Sunday, December 8, 2024, 8:00 a.m. - 9:00 a.m. Hilton San Diego Bayfront, Aqua 313

How I Treat: Options for Long-Term Management of Hemophilia (ticketed session for in-person participants)

Saturday, December 7, 2024, 8:00 a.m. - 9:00 a.m. Hilton San Diego Bayfront, Aqua 310

How I Treat: Sickle Cell Disease in Africa (ticketed session for in-person participants)

Sunday, December 8, 2024, 8:00 a.m. - 9:00 a.m. Hilton San Diego Bayfront, Aqua 310

Marquee Sessions

2024 Ham-Wasserman Lecture

Saturday, December 7, 2024, 12:30 p.m. - 1:30 p.m. San Diego Convention Center, Hall B

Sickle cell disease (SCD) is an inherited red blood cell (RBC) disorder caused by either homozygous inheritance of the hemoglobin S mutation (HbSS), or compound heterozygous inheritance of the hemoglobin S mutation with another ß-globin chain abnormality. India has among the highest hemoglobin S allele frequencies in the world and the third-highest birth rate for babies born with HbSS. 

The Indian sickle cell haplotype is associated with the Arab-Indian or Asian haplotype and is associated with high fetal hemoglobin (HbF) levels and hence, is believed to have mild clinical presentation as compared to other populations.  

 

In this lecture Dr. Dipty Jain will discuss the status of frequency, comorbidities, and management of sickle cell disease (SCD) in India, which is characterized by relatively high percentages of fetal hemoglobin, accompanied by mild to severe complications observed globally. Additionally she will talk about targeted newborn screening and fixed low dose of hydroxyurea in managing children with sickle cell disease in India. 

Dr. Jain will discuss the milestones and progress in understanding sickle cell disease (SCD) phenotype accelerated by the Government of India’s ambitious ‘’National Sickle Cell Anemia Elimination Mission 2023’’ with comprehensive guidelines and implementation of screening, treat, counsel, educate, development of technology and novel therapies including gene therapy specific to the requirement of India’s population with sickle cell disease (SCD). 

Chair:

Mohandas Narla, DSc
New York Blood Center
New York,  NY

Speaker:

Dipty Lalit Jain, MD,MBBS
Government Medical College and Hospital, Nagpur
NAGPUR,  Maharastra, India
Sickle Cell Disease in Developing Countries

2024 Presidential Symposium

Tuesday, December 10, 2024, 9:45 a.m. - 11:15 a.m. San Diego Convention Center, Hall B

Red cells constitute ~70% of all cells in the human body and in healthy individuals their circulatory life span is ~ 120 days. At steady state the bone marrow produces 2.5 million red cells per second to maintain normal hemoglobin levels. Anemia characterized by decreased hemoglobin levels is a major global health problem affecting nearly 2 billion individuals. Decreased red cell life span and/or decreased red cell production in the bone marrow account for anemia in various inherited and acquired hemolytic anemias. Significant progress has been and is continuing to be made in our understanding of the mechanistic basis for anemia. The three presentations in the symposium will highlight recent advances in our understanding of red cell biology and of anemia.

Dr. Patrick Gallagher will discuss recent advances which have expanded understanding of the many roles of the erythrocyte membrane. An update on the diagnosis and treatment of membrane disorders and potential emerging therapies will be discussed.

Dr. Naomi Taylor will highlight the pivotal role of metabolite transporters and fuel choice in erythroid lineage commitment and terminal erythroid differentiation. The importance of metabolic reprogramming in diseases of disordered and ineffective erythropoiesis will be discussed.

Dr. Olivier Hermine will explain the mechanisms that control red cell production during terminal erythroid differentiation, focusing on the processes that determine the fate of erythroid precursors between differentiation or cell death. The discussion will also touch upon how these mechanisms are impacted in hemoglobinopathies and myelodysplastic disorders, which can result in ineffective erythropoiesis and anemia. Additionally, therapeutic implications arising from these insights will be emphasized.

Chair:

Mohandas Narla, DSc
New York Blood Center
New York,  NY

Speakers:

Patrick G. Gallagher, MD
Nationwide Childrens, Ohio State University
Columbus,  OH
Mature Red Cell Membrane Disorders (including genome-wide screening and PK activators)

Naomi Taylor, MD
National Institutes of Health
Bethesda,  MD
Metabolism and Erythropoiesis (including metabolism, IDH mutations and relevance to MDS, sideroblastic anemia)

Olivier Hermine, MD,PhD
IMAGINE institute Paris France
Paris,  France
Ineffective Erythropoiesis in Thalassemia and Sickle Cell Disease

ASH-EHA Joint Symposium

Sunday, December 8, 2024, 12:30 p.m. - 1:30 p.m. San Diego Convention Center, Hall B

Session Overview

The ASH-EHA Joint Symposium is intended to address global issues in hematology from both the North American and European perspectives and provide insight on how international collaboration can help confront these issues. The first of these sessions was held at the ASH Annual Meeting in December 2005, and has taken place twice each year since at the European Hematology Association’s (EHA) Annual Congress in June and the ASH Annual Meeting in December. The leadership of both societies determine the recommended topics and speakers for these events each year, with the society presidents co-chairing the event. This year's topic is Myelodysplasia.  

Myelodysplasia

Myelodysplasia and Myeloproliferative syndromes are complex and heterogeneous clinical entities with varying progression rates. Significant progress is being made in our understanding of the molecular and genetic basis for these disorders. These insights are being used to develop new strategies for optimal patient-specific treatment modalities for effective and optimal clinical management of these disorders.  Dr. Amy DeZern will discuss prognostication and risk stratification strategies for patients with myelodysplastic syndromes. Dr. Matteo Della Porta will discuss the targeting of cytopenias in low-risk MDS.

CoChair:

Speakers:

Amy E. DeZern, MD
Johns Hopkins University
Baltimore,  MD
Prognostication and risk stratification strategies for patients with myelodysplastic syndromes

Matteo Giovanni Della Porta, MD
IRCCS Humanitas Research Hospital
Rozzano,  Milan, Italy
Targeting of Cytopenias in Low-Risk MDS

E. Donnall Thomas Lecture and Prize

Monday, December 9, 2024, 9:00 a.m. - 10:00 a.m. San Diego Convention Center, Hall B

In studying the intrinsic and extrinsic mechanisms that regulate hematopoietic stem cell (HSC) maintenance and the roles these mechanisms play in cancer, Dr. Morrison and others identified a series of self-renewal regulators, revealing important principles. First, stem cell self-renewal is mechanistically distinct from restricted progenitor proliferation. Second, self-renewal mechanisms are conserved among stem cells in different tissues. Third, these mechanisms comprise networks of proto-oncogenes and tumor suppressors that are dysregulated in cancer. These networks change over time, conferring temporal changes in stem cell properties that match the changing growth and regeneration demands of tissues. Tumor suppressor expression increases with age in stem cells, suppressing cancer development but also reducing tissue regenerative capacity during aging.

In terms of cell-extrinsic mechanisms, HSC niches in adult bone marrow and spleen have been identified. They are closely associated with sinusoidal blood vessels in both tissues. Leptin Receptor-expressing (LepR⁺) perivascular stromal cells and endothelial cells are the sources of factors required for HSC maintenance in the bone marrow. The identification of these cells has revealed new mechanisms that regulate hematopoiesis in the bone marrow. There are distinct subsets of LepR⁺ cells that create perivascular niches for HSCs and restricted hematopoietic progenitors throughout the bone marrow. LepR⁺ cells also synthesize growth factors that regulate osteogenesis, vascular regeneration, and peripheral nerve maintenance in the bone marrow. A subset of LepR⁺ cells are the skeletal (mesenchymal) stem cells that give rise to the osteoblasts and adipocytes that are produced in adult bone marrow.

This lecture will focus on new insights that have been gained into the mechanisms that regulate HSC maintenance, hematopoiesis, and osteogenesis in the bone marrow as a result of identifying LepR⁺ stromal cells, as well as the implications of these findings for clinical bone marrow transplantation.

Chair:

Mohandas Narla, DSc
New York Blood Center
New York,  NY

Speaker:

Sean J. Morrison, PhD
University of Texas Southwestern Medical Center
Dallas,  TX
Hematopoietic Stem Cell Maintenance

Ernest Beutler Lecture and Prize

Monday, December 9, 2024, 1:30 p.m. - 2:30 p.m. San Diego Convention Center, Hall B

Studies of hemoglobin, and its associated disorders, sickle cell disease (SCD) and the thalassemias, have written much of the history of the development of molecular biology and genetic medicine. More than 70 years ago, Dr. Linus Pauling dubbed SCD the first “molecular disease,” and seven years later Dr. Vernon Ingram identified the single amino acid difference between normal and sickle hemoglobin within the adult b-globin protein. With the advent of gene cloning and DNA sequencing, subsequent research in the 1980s elucidated the myriad mutations in the b-globin gene causing reduced or absent b-globin production in b-thalassemias. The benefits of increased expression of fetal hemoglobin (HbF) in individuals with SCD or b-thalassemia were recognized by astute clinicians based on of the lack of symptoms in the neonatal period and in the benign hereditary persistence of fetal hemoglobin (HPFH) condition. This stimulated interest in understanding the intricacies of globin gene regulation and how the switch from fetal (g)-to adult (b)-globin is normally controlled. In parallel, advances in gene transfer fueled efforts to introduce normal globin genes into hematopoietic stem cells of patients with mutant b-globin genes as curative therapy, an autologous approach built on decades of improvements in allogeneic bone marrow transplantation. Finally, remarkable advances in the modification of genes with CRISPR over the past decade have provided the means to define with precision the function of specific DNA sequences and develop gene editing approaches for therapy.

These research threads have converged to bring the first successive clinical trials forward for patients with SCD and b-thalassemia. In the last year, both globin gene addition (lovo-cel) and CRISPR gene editing (exa-cel) therapies received FDA approval. This lecture will describe the background and results of these groundbreaking studies, and discuss the challenges faced in bringing therapies to the many patients with SCD and b-thalassemia, particularly in resource-limiting geographic regions.

Dr. Stuart Orkin will describe current perspectives on the transcriptional control of globin genes, focusing on the direct role of the repressor protein BCL11A in silencing of HbF production and how CRISPR editing of an erythroid enhancer within the BCL11A gene forms the basis of the approved editing therapy, exa-cel, developed by CRISPR Therapeutics and Vertex Pharmaceuticals.

Dr. John Tisdale will summarize recent advances in ex vivo modification of hematopoietic stem cells for both lentiviral gene and CRISPR gene editing therapies for the hemoglobin disorders, concentrating on clinical parameters and prospects for improving preconditioning and moving toward strategies that would allow application without the need for hospitalization and intensive supportive care.

Chair:

Mohandas Narla, DSc
New York Blood Center
New York,  NY

Speakers:

Stuart H Orkin, MD
Harvard Medical School
Boston,  MA
Basic Science

John Tisdale, MD
National Institutes of Health
Bethesda,  MD
Clinical/Translational Science

Meet the Scientist

Meet the Scientist: CAR-T and CAR-NK in Therapy of Hematologic Malignancies (Ticketed session for in-person participants)

Saturday, December 7, 2024, 8:00 a.m. - 9:00 a.m. Hilton San Diego Bayfront, Aqua Salon F

Meet the Scientist: Epigenetic Regulation in Bone Marrow Failure (Ticketed session for in-person participants)

Saturday, December 7, 2024, 8:00 a.m. - 9:00 a.m. Hilton San Diego Bayfront, Aqua Salon E

Meet the Scientist: Mechanisms of Drug Resistance in Acute Myeloid Leukemia (Ticketed session for in-person participants)

Sunday, December 8, 2024, 8:00 a.m. - 9:00 a.m. Hilton San Diego Bayfront, Aqua Salon AB

Meet the Scientist: Microenvironment Regulation of Hematopoietic Stem Cells (Ticketed session for in-person participants)

Sunday, December 8, 2024, 8:00 a.m. - 9:00 a.m. Hilton San Diego Bayfront, Aqua Salon D

Meet the Scientist: Pluripotent Stem Cell-Based Modeling of Hematologic Malignancies (Ticketed session for in-person participants)

Sunday, December 8, 2024, 8:00 a.m. - 9:00 a.m. Hilton San Diego Bayfront, Aqua Salon E

Meet the Scientist: Production of Functional Hematopoietic Stem Cells from Pluripotent Cells (Ticketed session for in-person participants)

Saturday, December 7, 2024, 8:00 a.m. - 9:00 a.m. Hilton San Diego Bayfront, Aqua Salon D

Meet the Scientist: Therapy-Related Myeloid Neoplasms (Ticketed session for in-person participants)

Saturday, December 7, 2024, 8:00 a.m. - 9:00 a.m. Hilton San Diego Bayfront, Aqua Salon AB

Meet the Scientist: Transferrin Receptor Physiology and Iron Metabolism (Ticketed session for in-person participants)

Saturday, December 7, 2024, 8:00 a.m. - 9:00 a.m. Hilton San Diego Bayfront, Aqua Salon C

Meet the Scientist: What Does the Future Hold for Management of Hemophilia? (Ticketed session for in-person participants)

Sunday, December 8, 2024, 8:00 a.m. - 9:00 a.m. Hilton San Diego Bayfront, Aqua Salon C

Scientific Program

Crosstalk Between Iron Homeostasis and Metabolism

Scientific Committee on Iron and Heme

Monday, December 9, 2024, 4:30 p.m. - 5:45 p.m. San Diego Convention Center, Room 29

Maintaining iron balance is fundamental to preserve metabolic functions and energy requirements. This session reflects the current understanding of the reciprocal relationship existing between iron homeostasis and energetic metabolism. It aims to highlight the regulatory role of heme and iron on glucose and lipid metabolism as well as the impact of metabolism on the modulation of the iron status. Novel findings will be presented about the physiologic and pathologic implications of iron-mediated regulation of metabolism, and metabolite-driven control of iron and heme homeostasis. The session will focus on b-thalassemia, sickle cell disease and obesity, and will have broad relevance to all hematologic and non-hematological diseases.

Dr. Antonella Nai will discuss the role of Transferrin Receptor2 (TFR2) in the reciprocal regulation between iron homeostasis, erythropoiesis, and glucose metabolism. TFR2 is an iron sensor acting as a brake of erythropoietin signaling in erythroid cells. Its genetic inactivation in the hematopoietic compartment enhances erythropoiesis and improves anemia in ß-thalassemia. She will present unpublished data demonstrating that Tfr2 deficiency increases the metabolic activity of erythroid cells, thus promoting glucose consumption and reducing blood glycemia. This is particularly relevant in ß-thalassemia, whereby glucose intolerance and diabetes are common and invalidating complications.

Dr. Wei Ying will focus on the connection between iron homeostasis and lipid metabolism. He will present findings related to the role of iron homeostasis in regulating hepatocyte lipogenesis and neighboring cell responses, including hepatic stellate cells, and describe how alterations of these mechanisms drive non-alcoholic fatty liver disease, steatohepatitis and obesity.

Dr. Leitinger will share how heme triggers a unique bioenergetic switch in macrophages, characterized by a metabolic shift from oxidative phosphorylation towards glucose consumption, and its critical role in the effective clearance and detoxification of heme in sickle cell disease. Based on the previous identification of the phosphofructokinase PFKFB3 as key mediator of the metabolic switching in heme-detoxifying macrophages, he will present data about a potential therapeutic strategy involving the inhibition of PFKFB3 in a mouse model of sickle cell disease. Furthermore, the general clinical relevance of these processes for patients with hemolytic disorders will be discussed.

Chair:

Francesca Vinchi, PhD
New York Blood Center
New York,  NY

Speakers:

Antonella Nai, PhD
IRCCS San Raffaele Scientific Institute
Milan,  Italy
Iron, Erythropoiesis, and Glucose Metabolism

Wei Ying, PhD
University of California San Diego
La Jolla,  CA
Iron and Lipid Metabolism

Norbert Leitinger
University of Virginia
Charlottesville,  VA
Heme and Macrophage Metabolism.

Host Factors Driving Anti-Leukemia Immunotherapy Outcomes

Scientific Committee on Immunology and Host Defense

Saturday, December 7, 2024, 2:00 p.m. - 3:15 p.m. Marriott Marquis San Diego Marina, Pacific Ballroom Salons 18-19

This session will cover how the immune microenvironment of the host interacts with leukemia cells and how the donor immune system can exert graft-versus-leukemia (GVL) effects. The speakers will focus on the characterization of immune effector cells and leukemia cells using high resolution RNA and protein analysis, the characterization of metabolites that impact the GVL effect and kinase inhibition strategies to overcome immune escape mechanisms of leukemia cells.

Dr. Catherine Wu will discuss the components critical to devising effective immunotherapy for the treatment of myeloid leukemia, namely the identification of myeloid malignancy antigen targets, and the detection of critical immune cell effectors. The analysis of GvL responses following allogeneic stem cell transplant for AML/MDS, through single cell transcriptome and spatial analysis of marrow specimens, is a particularly instructive setting from which to detect key antigens and cell subpopulations implicated in anti-leukemia responses. The insights gained from the study of such GVL settings promise to provide a roadmap for devising new immunotherapeutic approaches for myeloid malignancies.

Dr. Takanori Teshima will discuss mechanisms of leukemia relapse after allogeneic hematopoietic stem cell transplantation and novel strategies to prevent relapse by inhibiting immune escape of leukemic cells, restoring immune cell dysfunction, and turning “COLD” into “HOT” immune microenvironment. In addition, novel strategies to separate GVL from GVHD by controlling migration and activation of donor T cells in the vicinity of leukemic cells.

Chair:

Robert Zeiser, MD
Universitätsklinikum Freiburg
Freiburg,  Germany

Speakers:

Catherine J. Wu, MD
Harvard Medical School
Boston,  MA
Immune Microenvironment and GVL Effects

Takanori Teshima, MD,PhD
Hokkaido University
Sapporo,  Hokkaido, Japan
Firing Up COLD Tumor Microenvironment to Facilitate GVL

Franziska Blaeschke, MD,PhD
German Cancer Research Center (DKFZ)
Heidelberg,  Germany
Knockin Sequences to Program Durable Cell Therapies Acting Against Leukemia Cells

Ineffective Erythropoiesis: Insights Into Molecular Mechanisms and Disease Pathophysiology

Scientific Committee on Red Cell Biology

Saturday, December 7, 2024, 9:30 a.m. - 10:45 a.m. Manchester Grand Hyatt San Diego, Grand Hall D

Ineffective erythropoiesis is the leading cause of red cell-related diseases, including anemia, which stands as the most prevalent hematologic disease, affecting millions of individuals worldwide. Understanding the underlying mechanisms of ineffective erythropoiesis is pivotal for advancing diagnostic and therapeutic approaches in hereditary anemias and adult-onset conditions. This session will explore recent breakthroughs in research related to ineffective erythropoiesis, focusing on areas of hemoglobinopathies, malaria infection, and myeloid neoplasms. By bringing together experts at the forefront of these investigations, the session will not only enhance understanding of the pathogenesis of ineffective erythropoiesis but also catalyze the development of innovative approaches to treat related diseases.

Dr. El Nemer will discuss the molecular mechanisms underlying ineffective erythropoiesis in beta thalassemia (bTM) major and sickle cell disease (SCD). The talk will draw a parallel between bTM and SCD to describe the cellular dysfunctions observed in both pathologies involving the chaperon protein HSP70 and oxidative stress. The talk will also discuss the impact of ineffective erythropoiesis on the erythroid and hematopoietic niches in SCD.

Dr. Egan will discuss the impact of Plasmodium falciparum malaria on erythropoiesis.  In malaria infection, P. falciparumparasites invade and replicate within human erythrocytes in the peripheral blood, leading to anemia, microvascular congestion, and multi-organ dysfunction. Emerging evidence suggests that erythroblastic islands in the bone marrow also serve as a critical reservoir for parasites, which may contribute to malaria anemia. Recent investigations into host cell responses to P. falciparum infection of erythroid precursor cells derived ex-vivo from primary human hematopoietic stem/progenitor cells (HSPCs) identified stage-specific transcriptional changes in infected cells, including in genes involved in cell cycle regulation, stress responses, and key developmental processes.  Efforts employing scRNA-seq and mass cytometry to elucidate molecular interactions between P. falciparum and the hematopoietic niche at single-cell resolution and their potential implications for erythropoiesis will be discussed.   

Dr. Doulatov will discuss advances in understanding how splicing factor mutations in SF3B1 drive ineffective erythropoiesis and hematopoiesis in myelodysplastic syndromes. Recent development of induced pluripotent stem cell models and precise gene editing of primary human hematopoietic stem cells have provided insights into splicing alterations induced by mutant SF3B1 and their functional impact on hematopoiesis and erythropoiesis. Due to its essential role in disease pathophysiology, therapeutic strategies for targeting mutant SF3B1 will also be discussed.

Chair:

Peng Ji, MD,PhD
Northwestern University Medical School
Chicago,  IL

Speakers:

Wassim El Nemer, PhD
French Blood Establishment (EFS)
Marseille,  France
Post-Transcriptional and Signaling Mechanisms in Ineffective Erythropoiesis in Hemoglobinopathies

Elizabeth S. Egan, MD, PhD
Stanford University School of Medicine
Stanford,  CA
Plasmodium Falciparum Influences Erythropoiesis

Sergei Doulatov, PhD
University of Washington School of Medicine
Seattle,  WA
Ineffective Erythropoiesis in Myeloid Neoplasms

JOINT SESSION: Emerging Therapeutic Strategies Targeting Epigenetic, Transcriptomic, and Metabolic Mechanisms

Scientific Committee on Epigenetics and Genomics + Scientific Committee on Myeloid Neoplasia

Sunday, December 8, 2024, 9:30 a.m. - 11:05 a.m. San Diego Convention Center, Room 24

Recent developments in therapeutic targeting of various mechanisms of epigenetic, transcriptomic, and metabolic vulnerabilities in cancer utilize novel technologies and may provide future breakthroughs. Given the extensive amount of knowledge derived from high throughput methodologies in the last decade, rational targeting is now possible, with sophisticated mechanistic studies to study the effects of targeting. This session will focus on novel research aimed at identifying and targeting these molecules, including novel targeting approaches such as heterobifunctional molecules and others.

Dr. Crabtree will discuss a new approach to make cancer therapeutics by rewiring cancer drivers to activate po pathways of apoptosis) (Gourisankar et al., Nature 2023, PMID: 37495688 ; Sarott et al., Science 2024, PMID: 39361741).  The approach uses chemically induced proximity (CIP) and to kill the cancer cell with its driver. The molecules, called TCIPs (Transcriptional/Epigenetic Chemical Inducers of Proximity) rapidly reverse epigenetic repression of cell death genes and eliminates lymphoma in several CDX and PDX mouse models.

Dr. Cravatt will discuss novel small molecules that target RNA binding proteins.  Traditionally considered undruggable, RNA binding proteins may be targetable through the use of chemo proteomic approaches to identify ligandable sites.  Dr. Cravatt’s group has successfully employed this strategy to develop chemical probes that can engage cysteine residues in RNA binding proteins and regulate their activity in gene regulation.

Dr. Uckelmann will discuss novel inhibitors of transcriptional mechanisms. Previous work of Dr. Uckelmann has focused on elucidating the role of mutant NPM1 (NPM1c) in regulating oncogenic gene expression in acute myeloid leukemia (AML) cells. Her work showed that the chromatin specific function of the NPM1c oncoprotein can be targeted by Menin-MLL inhibitors which are currently in clinical trials for treatment of NPM1c leukemia. In her talk she will discuss emerging developments in our mechanistic understanding and therapeutic targeting of epigenetic regulators in AML.

Dr. Jones will discuss targeting metabolic dependencies in leukemia. Acute myeloid leukemia (AML) cells and leukemia stem cells (LSCs) have unique metabolic properties that can be leveraged to develop new therapeutic strategies. In this presentation, she will discuss complementary approaches to target metabolism in AML and LSCs.  These approaches include mechanisms to target energy metabolism, metabolite regulated protein synthesis and post-translational modifications, as well as the potential for using metabolite abundance to predict resistance to therapy.

CoChair:

Speakers:

Hannah J Uckelmann, PhD
Frankfurt Cancer Institute
Frankfurt,  Germany
Novel Inhibitors of Transcriptional Mechanisms

Gerald R. Crabtree, MD
Stanford University
Stanford,  CA
Heterobifunctional Molecules Targeting Epigenetic Mechanisms

Benjamin Cravatt, PhD
The Scripps Research Institute
San Diego,  CA
Inhibitors of RNAs and RNA Binding Proteins

Courtney Jones, PhD
Cincinnati Children's Hospital
Cincinnati,  OH
Targeting Metabolic Vulnerabilities

JOINT SESSION: Molecular Characterization of Lymphomas in Children, Adolescents, and Young Adults

Scientific Committee on Blood Disorders in Childhood + Scientific Committee on Lymphoid Neoplasia

Saturday, December 7, 2024, 4:00 p.m. - 5:35 p.m. Marriott Marquis San Diego Marina, Marriott Grand Ballroom 2-4

Dynamic advances in the molecular characterization of lymphomas continue to shape how these malignancies are classified and inform therapeutic targeting. This joint session will provide a state-of-the-science update on next-generation technologies used to explore that special group of lymphoma subtypes that are most prevalent in pediatric, adolescents, and young adults. The speakers represent experts in these methods and will highlight exciting applications in elucidating lymphoma biology (especially age-related differences in pathogenesis), including enhancing clinical management.

Dr. Lisa Roth will discuss ongoing efforts to delineate the genomic landscape of classic Hodgkin lymphoma (cHL). Historically, genetic evaluation of cHL has been limited by the scarcity of Hodgkin and Reed-Sternberg (HRS) cells in cHL tumors. This has been overcome in recent years using fluorescence-activated cell sorting to isolate HRS cells from cHL tissues and evaluation of circulating tumor DNA, which have revealed tumors with high mutational burden, complex structural variants, and genomic subtypes with distinct clinical and molecular characteristics. The applicability of these findings to develop prognostic biomarkers and novel therapeutics will be discussed.

Dr. Tomohiro Aoki will discuss emerging single-cell technologies that have enabled better understanding of the comprehensive interactions within the cHL tumor microenvironment at an unprecedented resolution. Considering current pathogenesis models where HRS cells “recruit and re-educate” their microenvironment to create an immuno-suppressive milieu, cHL has emerged as one of the most exciting fields for application of these cutting-edge technologies. Dr. Aoki will summarize new insights gained using CyTOF, single-cell RNA sequencing and multiplexed imaging techniques, and discuss potential implications for clinical decisions.

Dr. Birgit Burkhardt will provide a comprehensive update of recurrent genetic variants reported in Burkitt lymphoma (BL) in several recent studies. Covering both endemic and sporadic BL, Dr. Burkhardt will highlight the impact of patient age at diagnosis on molecular characteristics and its implications for pathogenesis. Incorporation of specific variants as potential biomarkers for risk group stratification in upcoming BL clinical trials will also be discussed.

Dr. Björn Chapuy will discuss novel (epi)genomic analyses employed to define molecular heterogeneity in B-cell lymphoma subtypes and identify clinically actionable alterations, with a focus on primary mediastinal large B-cell lymphomas (PBMLs). With biometric features that often overlap with cHL, PMBLs are distinguished by their typical manifestation in young female patients and sensitivity to PD-1 blockade. Dr. Chapuy will present evolving approaches to comprehensive genetic analysis used to inform and test response to therapies that target such mechanisms of immune escape.

CoChair:

Speakers:

Lisa Giulino Roth, MD
Weill Cornell Medicine
New York,  NY
Genomic Landscape in Hodgkin Lymphoma

Tomohiro Aoki, MD, PhD
Princess Margaret Cancer Center
Toronto,  ON, Canada
Sequencing Technologies in Hodgkin Lymphoma

Birgit Burkhardt, MD,PhD
University Hospital Münster
Muenster,  Germany
Age-Related Molecular Changes in Burkitt Lymphoma

Björn Chapuy
Charité - University Medical Center Berlin, Campus Benjamin Franklin
Berlin,  Germany
Functional Genomics of Non-Hodgkin Lymphoma in Children, Adolescents, and Young Adults

JOINT SESSION: Newly Described Functions in the Vascular Space

Scientific Committee on Megakaryocytes and Platelets + Scientific Committee on Thrombosis &amp; Vascular Biology

Saturday, December 7, 2024, 4:00 p.m. - 5:35 p.m. San Diego Convention Center, Room 25

Dysregulated interactions between platelets, immune cells, endothelial cells, and their plasma environment underlie numerous age-related diseases. This joint session will provide an integrated view of the vascular space and will highlight the cellular and molecular mechanisms underlying diseases that primarily afflict the elderly. Specifically, the session will focus on novel discoveries addressing how platelet and endothelial aging may affect neurodegenerative disorders and thrombotic disease, and immune thrombocytopenia (ITP), and will shed light into the role of fibrinolytic factors in shaping the pro-atherogenic plasma lipid profile.

Dr. Nerlov, will discuss how aging affects hematopoietic stem cells (HSCs). He will describe how increased HSC platelet bias due to how age-related increases in TGFbeta signaling is an evolutionarily conserved feature of murine and human hematopoiesis. The role of HSC platelet bias in suppressing lymphoid cell production during aging and how TGFbeta receptor inhibition can increase the ability of aged HSCs to support antiviral immunity will also be discussed.  

Dr. Cooper, will discuss the current understanding of ITP pathogenesis and how the results from novel treatments in ITP may depict different disease types in ITP. ITP is a common bleeding disorder with an increase in incidence after age 60. Understanding the disease phenotype and evolution is needed to improve patient outcomes. However, the diagnosis remains presumptive, and treatment is largely empiric, with many patients rotating through several treatments before having a platelet response.

Dr. Lesniewski will discuss the phenotype of the aging vasculature and the hematopathology underlying mechanisms and potential therapeutic strategies being explored to reverse arterial aging to reduce cardiovascular (CVD) risk in older adults. This is important since several factors contributing to dysfunction of the endothelium (i.e., oxidative stress and inflammation, deregulated nutrient sensing, and cellular senescence) increase with aging and could impact CVD risk.

Dr. Zheng will discuss research demonstrating that hepatocytes are an unappreciated source of tissue-type plasminogen activator (tPA), sense metabolic stresses, and impact the production of tPA and its inhibitor PAI1, thereby influencing the extent of fibrinolysis in obesity. She will introduce findings that tPA binds directly to hepatocyte apoB, preventing it from being loaded with lipids and assembled into lipoproteins. Conversely, tPA's inhibitor, PAI1, has the opposite effect, sequestering free tPA and preventing its interaction with apoB to promote VLDL assembly and increase apoB-lipoprotein cholesterol levels.

 

CoChair:

Speakers:

Claus Nerlov, PhD
University of Oxford
Oxford,  United Kingdom
Platelets and Aging

Nichola Cooper, MD
Imperial College
London,  ENG, United Kingdom
New Insights in MK/PLT Functions and Diseases.

Lisa Lesniewski, PhD
University of Utah Health
Salt Lake City,  UT
Age-Related Endothelial Function

Ze Zheng, MBBS,PhD
Medical College of Wisconsin; Versiti Blood Research Institute
Milwaukee,  WI
Novel Functions of Fibrinolytic Factors in Shaping the Pro-Atherogenic Lipid Profile in Plasma.

JOINT SESSION: The Fuel & Physiology of Regeneration

Scientific Committee on Hematopoiesis + Scientific Committee on Stem Cells and Regenerative Medicine

Saturday, December 7, 2024, 4:00 p.m. - 5:35 p.m. San Diego Convention Center, Room 24

The regulation of hematopoietic stem cell (HSC) regeneration involves a sophisticated interplay between signals from the niche and the nuclear regulatory machinery. Extensive work has been done to identify many key players in both areas. However, conveying the signal to the nucleus is not sufficient without the “doers,” the machineries that execute the cellular processes critical for regeneration. Recent studies have identified several vulnerable processes in HSC physiology, such as proteostasis and energy metabolism, which become imbalanced during HSC exhaustion or dysfunction, i.e., processes involved in HSC culture expansion or aging. This session will closely examine the critical physiological processes and their interplay with other regulatory cues, including nutrient-derived signals (minerals and vitamins) crucial for maintaining healthy regeneration without malignant transformation.

Dr. Robert Signer, will discuss how protein homeostasis has emerged as fundamentally and preferentially important in HSCs. He will review how the protein homeostasis network is uniquely configured to promote hematopoietic stem cell self-renewal. Dr. Signer will further discuss how mechanisms of translational control, protein folding, and protein degradation are rewired throughout life to preserve stem cell fitness and will evaluate consequences of protein homeostasis disruption.

Dr. Marie-Dominique Filippi, will discuss the role of metabolism in HSC functions and how HSCs are highly responsive to changes in metabolite availability. New mechanisms will be presented on how HSC metabolic needs are remodeled during regenerative conditions. Additionally, Dr. Filippi will discuss the usage of branch chain amino acids as a new cell-autonomous metabolic checkpoint that influences HSC replicative lifespan.

Dr. Nina Cabezas-Wallscheid, will discuss how mouse and human HSCs and multipotent progenitors are metabolically regulated from intrinsic to dietary-derived metabolites. Through her work using integrated low-input multi-layer omics data, she will highlight research demonstrating distinct metabolic and epigenetic hubs that are essential in HSCs (and not for their downstream progenitors)

Dr. Britta Will, will discuss integrating nutrients into HSC regulation and iron homeostasis in HSCs. Her lab has uncovered a key role of the readily accessible intracellular labile iron pool in instructing HSC self-renewal. More recently, she has focused on identifying the molecular mechanisms of action, particularly focusing on metabolic and non-enzymatic pathways that rely on iron.

CoChair:

Speakers:

Robert Signer, PhD
University of California San Diego
La Jolla,  CA
Proteomic Health in Hematopoietic Stem Cells

Marie-Dominique Filippi, PhD
Cincinnati Children's Hospital Research Foundation
Cincinnati,  OH
Energy Management in Hematopoietic Stem Cells

Nina Cabezas-Wallscheid
ETH Zürich
Zürich,  Switzerland
Integrating Nutrients to Hematopoietic Stem Cell Regulation

Britta Will, PhD
Albert Einstein College of Medicine
Bronx,  NY
Iron Homeostasis in Hematopoietic Stem Cells.

JOINT SESSION: Therapeutic Gene Editing of Stem Cells in Classical and Malignant Hematology

Scientific Committee on Bone Marrow Failure + Scientific Committee on Transplantation Biology &amp; Cellular Therapies

Sunday, December 8, 2024, 9:30 a.m. - 11:05 a.m. San Diego Convention Center, Room 29

Gene editing is a novel technology to change specific nucleotides or knockouts in cells, and these are starting to be used clinically. This joint-scientific committee session will include 4 talks that span from basic science to clinical use of gene-edited stem cell products and CAR-T products in malignant and classical hematology.

Dr. Justin Eyquem will discuss his latest work using novel gene editing technologies to improve CAR T-cell function and facilitate their manufacturing. He will present new gene edits associated with improved CAR T-cell functional persistence and novel types of vectors for the generation of CAR T cell in vivo.

Dr. Paulo Rio will present the latest results in optimizing novel genome editing tools, including prime editing, to correct hematopoietic stem cells (HSCs) from Fanconi Anemia (FA) patients. She will discuss the latest advances from her lab in optimizing prime editing technology to efficiently target long-term repopulating HSCs and correct the most common FA mutations in both cell lines and primary HSCs from FA patients.

Dr. Pietro Genovese will present the innovative “epitope editing” strategy for overcoming on-target/off-tumor toxicities in acute myeloid leukemia (AML) immunotherapy. Epitope mapping and library screenings have identified specific amino acid changes that have avoided antibody binding. These have now been introduced into HSPCs via base-editing techniques. Epitope-edited HSPC’s were resistant to CAR-T cell therapy, and enabled specific eradication of patient-derived AML xenografts. Dr. Genovese will discuss the potential of precision immunotherapy for relapsed/refractory AML, non-genotoxic conditioning approaches, and advances in multiplex engineering of HSPCs.

Dr. Julia Skokowa will review recent efforts to establish clinical genome editing to correct severe congenital neutropenia-associated mutations as well as gene editing efforts to inhibit mutated genes in patients' HSCs that may be applicable to other bone marrow failure syndromes. She will also discuss key considerations that are particularly important for gene therapy of pre-leukemic bone marrow syndromes, where gene editing of HSCs should be extremely safe so as not to potentiate the leukemogenic transformation of hematopoiesis. Finally, Dr. Skokowa will present her thoughts on the ethics of gene editing for pre-leukemic bone marrow failure.

CoChair:

Speakers:

Justin Eyquem, PhD
University of California San Francisco
San Fransisco,  CA
New Gene Editing Technologies

Paula Rio, PhD
IIS-FJD, UAM
Madrid,  Spain
Gene Editing for Fanconi Anemia Bone Marrow Failure

Pietro Genovese, PhD
Boston Children's Hospital
Boston,  MA
Stem Cell Gene Editing

Julia Skokowa, MD, PhD
University Hospital Tübingen
Tübingen,  Germany
Gene Editing for Severe Congenital Neutropenia – Current Advances and Critical Considerations

Leveraging Single Cell Multi-Omics to Understand Mechanisms of Myeloid Lineage Expansion in Aging and Disease.

Scientific Committee on Myeloid Biology

Monday, December 9, 2024, 10:30 a.m. - 11:45 a.m. San Diego Convention Center, Room 6DE

Single cell multi-omic studies of normal and malignant hematopoiesis highlight how hematology remains an ideal testing ground for technologic innovation. Continued advancements in single cell sequencing and the analysis of these complex datasets have informed functional experiments that have enabled the field to improve our understanding of hematopoietic stem cell behavior and myeloid differentiation in response to aging, inflammation, and malignant transformation. This session will showcase innovative transcriptomic and functional genomic studies of the pathways that govern myeloid differentiation in response to aging, inflammation, and malignancy.

Dr. Laurenti will focus on how mutations at the DNMT3A R882 hotspot impact differentiation into mature myeloid cell types in steady-state and inflammatory contexts. She will report evidence implicating neutrophils in an inflammatory vicious circle triggered by the acquisition of DNMT3A R882 mutations in hematopoietic stem cells. She will also discuss findings which could contribute to explain the high risk of leukemia associated with DNMT3A R882 mutations.

Dr. Dick will explore how aging and inflammatory conditions impact myelopoiesis and pre-leukemic clonal expansions. He will focus on the identification of HSC-inflammatory memory (HSC-iM), a new HSC population that remembers prior inflammatory stress and adapts to chronic inflammation through dormancy and impaired differentiation output. He will present evidence that selective advantage of CH-mutant HSCs arise as a consequence of relieving the increased dormancy and decreased differentiation output of HSC-iM. Through scRNA/ATAC analysis, combined with novel lineage trace analysis, evidence will be presented that HSC-iM transmit their activated programs to their myeloid progeny resulting in their pro-inflammatory properties.

Dr. Klein will examine natural variation in human bone marrow biopsies from over 70 individuals with a representation of ages, genders and ethnicities. He will report on the stereotyped covariation identified between certain progenitor and mature cell type frequencies, and present evidence of both constrained and divergent gene expression programs across the 70 individuals. He will also discuss how the tumor microenvironment can impact the function of mature myeloid cells.

Chair:

Esther A. Obeng, MD,PhD
St. Jude Children's Research Hospital
Memphis,  TN

Speakers:

Elisa Laurenti, PhD
University of Cambridge
Cambridge,  ENG, United Kingdom
Hematopoietic Stem Cell Commitment to the Myeloid Lineage

John E. Dick, PhD
Princess Margaret Cancer Centre, University Health Network
Toronto,  ON, CAN
Hematopoietic Stem Cell and Myeloid Progenitor Cell Response to Inflammation

Allon Moshe Klein, PhD
Harvard Medical School
Boston,  MA
Myeloid Progenitor and Mature Neutrophil Responses in Cancer

Metabolic Deregulation in Hematopoietic Neoplasia: Tumor, The Environment, and Therapies.

Scientific Committee on Hematopathology and Clinical Laboratory Hematology

Sunday, December 8, 2024, 9:30 a.m. - 10:45 a.m. Marriott Marquis San Diego Marina, Pacific Ballroom Salons 15-17

Cellular metabolism and its role in the pathogenesis of hematologic malignancies is an emerging area of research. Recent studies implicate various cellular mechanisms by which oncogenic activation of metabolic pathways contribute to lymphoid and myeloid malignancies as well as their respective tumor environment. In this session, the speakers will discuss the role of deregulated cellular metabolism that contributes to lymphoid and myeloid malignancies and how they may provide novel insights with therapeutic relevance. The metabolic control of T cell differentiation and their function in immunity will also be discussed. This session highlights the opportunities for gaining novel mechanistic insights of hematologic malignancies through global metabolomic studies that has potential to impact the field of T-cell immunology, immunotherapies and facilitate translation to clinical practice.

Chair:

Megan S. S Lim, MD,PhD
Memorial Sloan Kettering Cancer Center
New York City,  NY

Speakers:

Jürgen Maximilian Ruland
Technical University of Munich
Munich,  Germany
Novel Insights in Metabolic Deregulation in Lymphoid Malignancies

Paolo Gallipoli, MD
Queen Mary University of London
London,  United Kingdom
Targeting Metabolic Deregulation in Acute Myeloid Leukemia

Ananda Goldrath, PhD
Allen Institute
Seattle,  WA
Metabolic Control of T Cell Differentiation and Function in Tumor Immunity

Proteo-Genomics to Better Study Multiple Myeloma Biology and Evolution

Scientific Committee on Plasma Cell Neoplasia

Saturday, December 7, 2024, 9:30 a.m. - 10:45 a.m. Marriott Marquis San Diego Marina, San Diego Ballroom AB

Cancer proteogenomics incorporates methods that integrate mass spectrometry-based measurements of protein abundance and post-translational modifications with genomic, epigenomic and transcriptomic data from preclinical cancer models and patients samples. Such comprehensive multi-omic data provides new biological and diagnostic knowledge that can improve our understanding of malignant transformation and therapeutic outcomes.

This area is new in the field of Plasma Cell Neoplasia and in this session we will discuss how proteogenomic can lead to a better understanding of multiple myeloma (MM) biology and support the development of novel anti-MM therapeutic targets.

Dr. Krönke will discuss the impact of proteogenomic studies on understanding disease progression in plasma cell malignancies. His comprehensive analysis reveals protein-level changes that are not always predictable from genomic or transcriptomic data alone, highlighting the importance of examining protein dynamics in understanding MM pathogenesis. He will further outline how proteogenomic analysis enables the development of new risk stratification models and reveal deregulated proteins and pathways that can be exploited for new therapeutic approaches in MM.

Dr. Maura will discuss how whole genome sequencing can be utilized to reconstruct the genomic landscape and track the temporal evolution of multiple myeloma. These aspects are becoming increasingly relevant, as the timing and mechanisms of plasma cell transformation in the germinal center, along with their progression to MGUS, SMM, and MM, can significantly impact clinical outcomes. He will also explore how comprehensive genomic profiling can identify mechanisms of resistance and predict responses, particularly in the context of immunotherapy using bispecific antibodies and CAR T cells.

Dr. Perna will discuss an integrated pipeline for probing the MM surface proteome (surfaceome) with mass-spectrometry and transcriptomic analyses to identify novel immunotherapeutic targets. She will discuss the contribution that surfaceome makes to MM biology and the pre-clinical development of novel chimeric antigen receptor platforms for MM based on in vitro and in vivo models including patient samples and BCMA-escaped disease. This work may provide a rationale for developing innovative CAR T cells for MM patients.

Chair:

Paola Neri, MD,PhD
Arnie Charbonneau Cancer Institute, University of Calgary
Calgary,  AB, Canada

Speakers:

Jan Kronke, MD
Charité – Universitätsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt Universität zu Berlin, and Berlin Institute of Health
Berlin,  Germany
Defining the Proteomic Landscape of Multiple Myeloma

Francesco Maura, MD
University of Miami
Palmetto Bay,  FL
Genomic Drivers Involved in Myeloma Progression and Development of Resistance

Fabiana Perna, MD, PhD
H. Lee Moffitt Cancer Center and Research Institute
Tampa,  FL
Proteo-Genomics to Better Study Cell Surfaceome in Multiple Myeloma

Understanding the Impact of Donor and Recipient Metabolic Variability on Blood Transfusion Outcomes

Scientific Committee on Transfusion Medicine

Saturday, December 7, 2024, 9:30 a.m. - 10:45 a.m. San Diego Convention Center, Room 7

Blood transfusion requires the donation of blood products from a diverse array of individuals with unique genetics and environmental inputs. This results in the quality and overall behavior of the blood products being less standardized and predictable, with direct consequences on transfused recipients. This session will focus on how recently recognized unique genetic and environmental backgrounds of both the blood donor and the transfusion recipient influence the outcomes of patients. Newly applied metabolic tools reveal metabolic variability in donors and recipients, shedding light on fundamental aspects of these differential responses of transfusion in patients, which could guide future strategies for more personalized and effective blood transfusions.

Chair:

Cassandra Dorothy Josephson, MD
Johns Hopkins All Children’s Hospital
St. Petersburg,  FL

Speakers:

Angelo D'Alessandro, PhD
University of Colorado Anschutz Medical Campus
Aurora,  CO
Blood Donor Genetics and Biology Impact Red Blood Cell Transfusion Outcomes

Moritz Stolla, MD
Bloodworks and University of Washington, Pathology and Laboratory Medicine
Seattle,  WA
Unveiling Platelet Metabolism: Implications for Storage and Transfusion

Cheryl Lobo, PhD
Laboratory of Blood-Borne Parasites, Lindsley F. Kimball Research Institute, New York Blood Center
New York,  NY
Impact of RBC Metabolism on the Disease Outcomes of Transfusion Dependent Patients

Scientific Spotlight Sessions

Fulfilling the Promise: Pluripotent Stem Cells as Models for Human Blood Development and Disease

Monday, December 9, 2024, 2:45 p.m. - 4:00 p.m. San Diego Convention Center, Room 31

(hPSCs) for transplantation and disease modeling has been a long-standing goal in the field. In recent years there has been tremendous progress in the field, and investigators have been able to define the developmental cues to direct mesodermal differentiation to definitive HSC fate rather than to embryonic yolk sac-type progenitors. These studies have been largely enabled by the advances in single-cell transcriptomic technologies, permitting comparison of in vitro HSC derivation to human HSCs developing in situ in the embryo. The ability to translate our better appreciation of HSC development in the human embryo into the development of in vitro differentiation protocols has resulted in the generation of multilineage engrafting HSCs (iHSCs) from human induced pluripotent stem cells (iPSCs), a recent and significant advance in the field. In parallel, in recent years, a variety of hPSC models, mainly based on iPSC, have been developed for a variety of non-malignant and malignant blood diseases. These have been shown to recapitulate key disease features both in vitro and in vivo in xenograft assays and have empowered unprecedented interrogation into the pathogenesis and therapeutic targeting of hematologic diseases. Advances in these areas of research will be highlighted in this session.

CoChair:

Speakers:

Elizabeth S. Ng, PhD
Murdoch Childrens Research Institute
Parkville, Melbourne,  VIC, Australia
Hematopoietic Stem Cell Development from Human Pluripotent Stem Cells

Eirini Papapetrou, MD,PhD
Icahn School of Medicine at Mount Sinai
New York,  NY
Modeling Blood Disorders Using Human Pluripotent Stem Cells

Biology Underlying Disparities in Lymphoid Malignancies

Sunday, December 8, 2024, 4:30 p.m. - 5:45 p.m. Marriott Marquis San Diego Marina, Pacific Ballroom Salons 18-19

This session will focus on uncovering how disparities in lymphoid cancers may be influenced by both the tumor microenvironment and genetic sequencing. By examining different types of lymphomas, the speakers will highlight how biological and genetic factors contribute to the disparities observed in Hodgkin lymphoma, multiple myeloma, and non-Hodgkin lymphoma for specific populations. This session aims to provide a comprehensive understanding of how these factors interact and shape treatment responses, survival rates, and disease progression, offering insights into reducing healthcare inequities. Together, these presentations will underscore the importance of a multifaceted approach—encompassing both the tumor environment and genetic profiling—to tackling disparities in lymphoma treatment and outcomes.

Dr. Cozen will discuss the critical role the tumor microenvironment plays in influencing disease outcomes, specifically focusing on how immune cells, stromal cells, and signaling molecules create conditions that vary across multiethnic cohorts. The presentation will explore how these variations may contribute to the disparities seen in survival and treatment response in both Hodgkin lymphoma and multiple myeloma. She will also present recent findings that suggest the microenvironment's interaction with genetic and socioeconomic factors, shaping future approaches for research and interventions for these populations.

Dr. Flowers will delve into the role of tumor sequencing in identifying genetic mutations and biomarkers that may be disproportionately prevalent across ancestry groups. The presentation will also discuss the relationships between genomic data and disease phenotypes that can be modeled and may impact treatment efficacy and outcomes. He will discuss the implications of these findings for precision medicine, including the need for more inclusive genetic research to better understand and address these disparities.

CoChair:

Speakers:

Wendy Cozen, DO, MPH
UCI, School of Medicine
Orange,  CA
Tumor Microenvironment Profiling to Investigate Disparities in Hodgkin Lymphoma and Multiple Myeloma

Christopher R. Flowers, MD, MS
The University of Texas MD Anderson Cancer Center
Houston,  TX
Tumor Sequencing to Investigate Disparities in Non-Hodgkin Lymphoma

Inflammation as a Regulator of Hematopoietic Homeostasis in Disease and Clonal Selection

Sunday, December 8, 2024, 4:30 p.m. - 5:45 p.m. San Diego Convention Center, Room 7

Hematopoietic stem and progenitor cells (HSPCs) are responsible for sensing and integrating infectious/inflammatory cues into hematopoietic responses in bone marrow and producing hemato-immune cells at high demand in peripheral tissues. This session will describe how the pro-inflammatory environment/response and systemic inflammation act on and subsequently shape hematopoietic development, homeostasis and diseases. 

Dr. Serine Avagyan will discuss the role of inflammatory pathways in developmental origins of HSPCs, and in shaping postnatal hematopoiesis in the context of aging and genetic blood disorders. This talk will highlight recent studies investigating how inflammation affects the fate of normal and mutant HSPCs, and the pathways by which inflammatory exposure affects the selection of mutant HSPC clones due to differential clonal fitness.   

Dr. Hitoshi Takizawa will discuss an interplay between hematopoiesis and commensal bacteria, a symbiont increasingly recognized as an important modulator for hematopoiesis in health and disease. This session will highlight recent cutting-edge research deciphering pathways by which commensal bacteria and their-derived signals influence the hematopoietic programs, hemato-immune aging, stem cell clonal diseases, and thereby, discuss future preemptive/therapeutic strategies.

CoChair:

Speakers:

Hitoshi Takizawa, PhD
Kumamoto University
Kumamoto,  Japan
Inflammation As a Regulator of Hematopoietic Homeostasis in Disease

Serine Avagyan, MD,PhD
University of California San Francisco
San Francisco,  CA
Inflammation As a Regulator of Hematopoietic Homeostasis in Clonal Selection

Iron at the Crossroads Between Erythropoiesis and Megakaryopoiesis

Monday, December 9, 2024, 2:45 p.m. - 4:00 p.m. San Diego Convention Center, Room 30

Iron balance is critical for healthy and effective erythropoiesis and thrombopoiesis. All mammalian cells rely on iron and the erythropoietic compartment consumes especially high quantities of iron for hemoglobin synthesis. Effective crosstalk is essential for optimal red cell and platelet production. This session will discuss how heme and iron deficiency, excess, trafficking, and processing contribute to ineffective erythropoiesis and anemia and contribute to red cell and platelet production in JAK2 mutant myeloproliferative neoplasms.

Dr. Jan Abkowitz will present single cell transcriptomic and CITE-seq analyses of effective (normal) erythropoiesis and of the ineffective erythropoiesis seen in Diamond Blackfan anemia and MDS-5q. Most erythropoiesis takes place within erythroblastic islands (EBIs) comprised of a central macrophage (“nurse cell”) and 10-50 adjacent erythroid precursors. Dr. Abkowitz’s lab has recently isolated EBIs from human marrow and characterized their central macrophages at single cell resolution. She will describe how this cell normally functions as a safe and ecologically-efficient heme-iron recycler and how its dysfunction may contribute to MDS-5q anemia.

Dr. Radek Skoda will discuss the effects of iron availability on the phenotype of mouse models of myeloproliferative neoplasms (MPNs) driven by either JAK2-V617V or a N542-E543del mutation in JAK2 exon 12 (E12). While JAK2-V617F can manifest as polycythemia vera (PV), essential thrombocythemia (ET), or primary myelofibrosis, expression of E12 results in PV, often with pure erythrocytosis phenotype. At baseline, PV patients with JAK2-V617V and JAK2-V617V mouse models with PV phenotype display iron deficiency, while ET patients and mice with an ET-like phenotype have normal iron stores. Dr. Skoda will describe the effects of iron deficiency and iron overload on the iron-responsive progenitor stages that decide between erythroid and megakaryocytic lineage choices and compare the effects of orally administered ferroportin inhibitor vamifeport and the minihepcidin PR73 on hemoglobin and iron parameters in JAK2-V617F and E12 mutant mouse models.

CoChair:

Speakers:

Janis L. Abkowitz, MD
University of Washington
Seattle,  WA
Role of Heme and Iron in Effective and Ineffective Erythropoiesis

Radek C. Skoda, MD
Baylor College of Medicine
Houston,  TX
Impact of Iron Availability on Megakaryopoiesis

Scientific Symposia

Interface of Complement and Coagulation

Saturday, December 7, 2024, 4:00 p.m. - 5:15 p.m. San Diego Convention Center, Room 33

The complement system and coagulation pathways are the main pillars of immunity and hemostasis respectively that undergo significant activation with injury. While appearing distinct, it is recognized that significant crosstalk exists between the two systems and recently established molecular mechanisms provide insights into a wide variety of disorders. This session will focus on new molecular links identified in complement activation in specific and highly relevant clinical disorders.

Chair:

David Kavanagh
New Castle University
Newcastle Upon Tyne,  United Kingdom

Speakers:

David Kavanagh
New Castle University
Newcastle Upon Tyne,  United Kingdom
Complement and the Prothrombotic State

Efthymia Vlachaki, Consultant Hematologist
Hippokrateon Hospital
Thessaloniki,  Greece
Complement Activation and Preclampsia

Lubka T Roumenina, PhD
INSERM UMRS1138 Cordeliers Research Center
Paris,  France
Sickle Cell Disease and Complement Activation

Interrogating the Impact of the Intestinal Microbiome on Transplant and Cellular Therapies

Sunday, December 8, 2024, 9:30 a.m. - 10:45 a.m. Marriott Marquis San Diego Marina, Marriott Grand Ballroom 11-13

Alterations in the intestinal microbiome are linked to responses and outcomes following allogeneic hematopoietic cell transplantation (allo-HCT) and chimeric antigen receptor (CAR) T-cell therapy. This session will highlight emerging research investigating the host-microbiome axis in this context. We will assess factors that alter the intestinal microbiome in recipients of these cellular therapies, including diet and antibiotic exposure. Further, we will evaluate interventions to modulate the microbiome in this setting, namely fecal microbiota transplant.

Dr. Malard will discuss the role of fecal microbiota transplant in hematological malignancies, focusing on allo-HCT. He will provide an overview of the available clinical data on fecal microbiota transplant for treating graft-versus-host disease after allo-HCT. Dr. Malard will also discuss the potential of this procedure to prevent graft-versus-host disease and mitigate infection. He will also address the role of fecal microbiota transplant as a strategy to eradicate multidrug-resistant bacteria in patients who have undergone allo-HCT. Finally, the potential mechanism of action of fecal microbiota transplant and the implications of fecal microbiota transplant for CAR-T will be discussed.

Dr. David will share an approach for using metagenomic sequencing of fecal samples to track dietary intake in recipients of allo-HCT. He will illustrate how this method, FoodSeq, was initially tested using hospital-based foods. Dr. David will also show how FoodSeq was applied to a patient population, revealing reductions in food intake after allo-HCT. Notably, he will also demonstrate how these changes in food intake were linked to aspects of patient outcomes and microbiome changes following allo-HCT.

Dr. Andermann will explore how antibiotics influence the gut microbiome, the antimicrobial resistome (the collection of resistance genes in the gut), and immunologic/infectious outcomes in patients undergoing allo-HCT and CAR-T. She will review previous research findings and describe how anaerobically active antibiotics alter taxonomic and resistance gene composition in the gut during allo-HCT. Additionally, she will compare the spectrum of activity of antibiotics commonly used for febrile neutropenia, emphasizing the need for further research on how these antibiotics distribute throughout the gut lumen and drive changes in the abundance of commensal gut bacteria in recipients of cellular therapy.

Chair:

Melody Smith, MD,MS
Stanford University School of Medicine
Stanford,  CA

Speakers:

Florent Malard, M.D., Ph.D.
Sorbonne Université, Hôpital Saint-Antoine, AP-HP, Service d'Hématologie Clinique et Thérapie Cellulaire,Centre de Recherche Saint-Antoine UMRs938
Paris,  France
Fecal Microbiota Transplant As a Strategy to Mitigate Infection and Gvhd Following TCT

Lawrence A David, PhD
Duke University
Durham,  NC
Deciphering Nutritional Intake from Metagenomic Data Using Computational Tools

Tessa Andermann, MD,MPH
University of North Carolina, Chapel Hill
Chapel Hill,  NC
Impact of Antibiotic Exposure on Outcomes Following TCT

Newly Discovered Functions of Megakaryocytes and Platelets

Sunday, December 8, 2024, 4:30 p.m. - 5:45 p.m. San Diego Convention Center, Room 29

This session will explore cutting-edge discoveries in platelet biogenesis and the pathological mechanisms underlying platelet-related disorders.

Key topics include the role of glycosylation in megakaryocyte (Mk) and platelet (PLT) functions and their involvement in disease. Focus will be placed on the role of sialic acid and galactose modifications in regulating megakaryocyte-primed hematopoietic stem cell function and megakaryocyte activity, especially in relation to thrombocytopenia, including immune thrombocytopenias. Additionally, novel insights into thrombo-inflammation will be discussed, highlighting how inflammatory processes contribute to thrombosis and platelet dysfunction. Emerging research on the role of megakaryocytes and platelets in bone marrow fibrosis will also be featured, shedding light on their dual function in both hematopoiesis and pathological fibrosis. These topics are highly relevant, particularly with ongoing advancements in understanding the basic mechanisms of platelet production, glycosylation, and the interaction between thrombosis and inflammation.

Dr. Karin Hoffmeister will discuss the role of sialic acid and galactose modifications in regulating megakaryocyte activity. These glycan decorations are crucial for maintaining proper megakaryocyte function and platelet production, with disruptions contributing to platelet clearance and immune-mediated destruction in conditions like immune thrombocytopenia (ITP).

Dr. Tobias Petzold will discuss novel insights on natural occurring resilience mechanism to prevent thromboinflammation as key pathomechanism in thrombotic diseases. By introducing the concept of long-term immobility induced thromboprotection we will learn from a novel animal model- the hibernating brown bear - that is protected from developing venous thrombosis during 6 months of immobility in winter.  These findings will be translated into the clinical context.

Dr. Anna Nam will discuss the impact of ER stress and the unfolded protein response on megakaryopoiesis and platelets due to mutations in CALR in essential thrombocythemia and myelofibrosis. Using advanced single-cell multi-omics approaches, we show that CALR mutations elicit the unfolded protein response in human. Targeting ER stress-related pathways leads to reduction in platelet burden in mouse models.

Chair:

Alessandra Balduini, MD
Tufts University
Medford,  MA

Speakers:

Karin M. Hoffmeister, MD
Versiti Blood Research Institute, Milwaukee, WI
Milwaukee,  WI
Unveiling the Sweet Side of Platelet Formation: Exploring the Pivotal Role of Glycans

Tobias Petzold, MD,MSc
Medizinische Klinik Und Poliklinik I
München,  BAY, Germany
Regulation of Platelet Function during Immobility

Anna S. Nam, MD
Weill Cornell Medicine
New York,  NY
ER Stress in Megakaryopoiesis

Placing the Brakes on Accelerated Aging

Monday, December 9, 2024, 4:30 p.m. - 5:45 p.m. San Diego Convention Center, Room 30

Hematologic disorders disproportionately affect older adults; despite this, our understanding of the implications of age on the pathogenesis and treatment of these diseases remains limited. The geroscience field is actively investigating geriomics to aid in distinguishing biological and chronological aging. This session will highlight biological aging assessments for hematologic disease which will be crucial for the identification of targets and development of therapies for this specific population.

Chair:

Ashley Rosko, MD
The Ohio State University
Columbus,  OH

Speakers:

Els Mansell, PhD
Erasmus Medical Center
Rotterdam,  Netherlands
Aging of Hematopoietic Stem Cells and Their Niche

Christin E Burd, PhD
Ohio State
Columbus,  OH
Geroscience and Geri-Omics in Hematologic Malignancies

Marcel R.M. van den Brink, MD, PhD
City of Hope National Medical Center
Duarte,  CA
Targeting Rejuvenation Therapies to Decelerate Aging

Special Symposium on the Basic Science of Hemostasis and Thrombosis

Monday, December 9, 2024, 4:30 p.m. - 6:00 p.m. San Diego Convention Center, Room 31

This session will highlight four emerging topics in hemostasis and thrombosis research, each providing new and significant insights into their respective fields. The first presentation will discuss the hemostatic role of extravascular FIX in hemophilia B prophylaxis. The second presentation will focus on the conformational regulation of integrin aIIbß3 during platelet activation and the implications for anti-platelet therapies. The third presentation will introduce a novel immune regulatory function of platelets. The fourth presentation will report findings on how F12 haploinsufficiency provides protection against venous thromboembolism.

Dr. Dargaud will discuss the role of extravascular FIX in hemophilia B. His recent findings demonstrate that, in individuals with severe hemophilia B receiving regular prophylaxis with four different FIX formulations, the progressive accumulation of FIX in the extravascular space significantly enhances coagulation capacity in specific target tissues. This process offers substantial protection against bleeding events.

Dr. Zhu will present on the central role of platelet integrin aIIbß3 (GPIIbIIIa) in thrombosis and thrombocytopenia. His presentation will provide an overview of current knowledge regarding the conformational regulation of integrin aIIbß3 during platelet activation and its inhibition by anti-platelet therapies. Additionally, he will delve into the structural basis of aIIbß3 as an alloantigen in immune thrombocytopenia.

Dr. Hou will describe a novel immune regulation function of platelets. Her findings reveal that treatment with thrombopoietin receptor agonists induces platelet-derived TGF-ß1, which reprograms myeloid-derived suppressor cells via the TGF-ß/Smad pathway. This study aligns with growing evidence that TPO-RA treatment triggers plausible treatment-free remission in steady responders, indicating a common mechanism of immune regulation in ITP-specific therapies mediated by platelet-derived TGF-ß.

Dr. Haj will report on the first large-scale analysis of the differential thrombotic risk associated with germline loss of function in the gene encoding FXII. Her research involving 415,921 individuals reveals that F12 haploinsufficiency is protective against venous thromboembolism. These findings suggest that therapeutic blockade of FXII in humans is likely to be both safe and effective.

If you are attending the meeting in San Diego, following this session, please join ASH leadership and your colleagues at the ASH Networking Reception for the Hemostasis and Thrombosis Community, taking place across the hallway, outside on the East Terrace at the San Diego Convention Center, from 6:00 p.m. - 7:30 p.m.

CoChair:

Speakers:

Yesim Dargaud, MD, PhD
Hopital Edouard Herriot Pav. E
Lyon,  France
Role of Extravascular FIX in Hemophilia

Jieqing Zhu, PhD
Versiti Blood Research Institute
Milwaukee,  WI
Platelet Integrin Structure, Function and Role in Disease

Yu Hou, PhD
Qilu Hospital, Cheeloo College of Medicine, Shandong University
Jinan,  CHN
Platelet TGFb1 in the Pathogenesis of Immune Thrombocytopenia Purpura

Amelia K Haj, MD,PhD
Massachusetts General Hospital
Cambridge,  MA
Coagulation Factor XII (F12) Haploinsufficiency Is Protective Against Venous Thromboembolism

Special-Interest Sessions

Artificial Intelligence in Hematology: From Generative AI to Ethics and Applications

Saturday, December 7, 2024, 4:00 p.m. - 5:30 p.m. San Diego Convention Center, Hall B

The session will address important and timely topics related to the application of artificial intelligence (AI) to hematology, ranging from generative AI's potential and pitfalls, modern ethical consideration for AI in biomedical research and examples of practical use of AI in hematology research and clinical care.

Chair:

Olivier Elemento, PhD,MS,BS
Weill Cornell Medicine
New York,  NY

Speakers:

James Zou, PhD
Stanford University
Stanford,  CA
The Potential and Pitfalls of Generative AI in Basic and Clinical Research

Camille Nebeker, EdD
University of California San Diego
San Diego,  CA
From Principles to Practice: Ensuring Ethical Integrity in AI-Driven Biomedical Research

Claudia Haferlach, MD
MLL Munich Leukemia Laboratory
Munich,  Germany
The Application of Artificial Intelligence in Hematological Diagnostics

ASH Clinical Practice Guidelines on Acute Lymphoblastic Leukemia (ALL) in Adolescents and Young Adults

Sunday, December 8, 2024, 9:30 a.m. - 11:00 a.m. Marriott Marquis San Diego Marina, Marriott Grand Ballroom 2-4

This special education session will preview recommendations for the management of both newly diagnosed and relapsed/refractory ALL in adolescents and young adults.

Chair:

Sumit Gupta, MD, PhD
Hospital for Sick Children
Toronto,  ON, Canada

Speakers:

Julie Wolfson, MD, MSHS
The University of Alabama at Birmingham
Birmingham,  AL
Management of Frontline ALL in AYA

Jennifer L. McNeer, MD
University of Utah/Primary Children's Hospital
Salt Lake City,  UT
Management of Frontline ALL in AYA

Kristen M. O'Dwyer, MD
University of Rochester
Rochester,  NY
Management of Refractory and Relapse of ALL in Adolescents and Young Adults

Lena E Winestone, MD
University of California San Francisco
San Francisco,  CA
Management of Refractory and Relapse of ALL in Adolescents and Young Adults

ASH Clinical Practice Guidelines on Amyloidosis

Sunday, December 8, 2024, 4:30 p.m. - 6:00 p.m. Manchester Grand Hyatt San Diego, Grand Hall B

This session will review the scope and goals of the upcoming guidelines on amyloidosis, and will provide insights on risk factors, selecting a target site versus a surrogate site for diagnostic biopsies, and using Congo Red stain screening.

Chair:

Vishal Kukreti, MD,MSc
Princess Margaret Cancer Centre
Toronto,  ON, Canada

Speakers:

Joselle Cook, MBBS
Mayo
Rochester,  MN
Increasing Awareness of Risk Factors for AL Amyloidosis

Angela Dispenzieri, MD
Mayo Clinic
Rochester,  MN
Target Site vs. Surrogate Site Biopsy for Diagnosis

Hira Shaikh, MD
University of Iowa Caver College of Medicine
Coralville,  IA
Target Site Vs. Surrogate Site Biopsy for Diagnosis

Naresh Bumma, MD
The Ohio State University
Columbus,  OH
Congo Red Stain Bone Marrow Screening for AL Amyloidosis

ASH Clinicians in Practice (ACIP) Lunch - The Role of Artificial Intelligence in Hematology Practice: The Good, the Bad, and the Ugly

Sunday, December 8, 2024, 11:15 a.m. - 12:30 p.m. Marriott Marquis San Diego Marina, Marina Ballroom DE

Artificial Intelligence (AI) has the potential to transform health care by enhancing diagnostic accuracy, predicting patient outcomes, and developing personalized treatment plans. Machine learning algorithms can analyze vast amounts of medical data, leading to more efficient and precise health care interventions, which could improve patient care and reduce costs. However, implementation of AI in health care also poses challenges related to data privacy concerns, ethical considerations, and the need for robust regulatory frameworks to ensure the responsible and secure use of sensitive medical information. In this session, panelists will speak on the recent advances and use of AI in health care, how it’s integrated into hematology practice by providers, and the use of AI by patients.

Chair:

Mary-Elizabeth M. Percival, MD
Seattle Cancer Care Alliance
Seattle,  WA

Speakers:

Jim Whitfill, MD MBA FACP FSIIM
HonorHealth
Phoenix,  AZ
Artificial Intelligence in Health Care: Benefits and Pitfalls

Christopher Manz, MD, MSHP
Dana-Farber Cancer Institute
Boston,  MA
Artificial Intelligence as a Clinical Tool: Hacks in Practice

Gwen L Nichols, MD
The Leukemia and Lymphoma Society
Rye Brook,  NY
Patient Facing Artificial Intelligence: Uses and Ethical Implications

ASH ISTH Clinical Practice Guidelines on Treatment of Venous Thromboembolism (VTE) in Pediatric Patients

Saturday, December 7, 2024, 9:30 a.m. - 11:00 a.m. San Diego Convention Center, Room 25

This special education session will give a preview on the revision background, which includes asymptomatic VTE, duration of therapy for provoked and unprovoked VTE, and use of anticoagulants (especially DOACs) in pediatric patients.

Chair:

Paul Monagle, MD,BS,MSc,FRACP,FRCPA,MB
University of Melbourne Royal Children's Hospital
Parkville,  VIC, Australia

Speakers:

Leslie Raffini, MD
Children's Hospital of Philadelphia
Philadelphia,  PA
Why Revise the Guidelines?

Rukhmi Bhat, MD,MS
Lurie Children's Hospital
Chicago,  IL
Asymptomatic VTE in Pediatric Patients

Marisol Betensky, MD,MPH
Johns Hopkins All Children's Hospital
Saint Petersburg,  FL
Duration of Therapy for Provoked and Unprovoked VTE in Pediatric Patients

Tina Biss
The Newcastle upon Tyne Hospitals NHS Trust
Newcastle Upon Tyne,  ENG, United Kingdom
Use of Anticoagulants in Children

ASH Quality Improvement Champions

Monday, December 9, 2024, 2:45 p.m. - 4:15 p.m. San Diego Convention Center, Room 7

This new session merges the ASH Choosing Wisely Champions and the ASH Guideline Implementation Champions into a single, 90-minute session. Speakers will be selected from a pool of applicants. In addition to projects that combat overuse (previously the focus of the Choosing Wisely Champions session) and projects that highlight successful guideline implementation strategies (previously the focus of the Guideline Implementation Champions session), the call for applications for this session considers any quality improvement project (e.g., combating underuse, improving stewardship of resources, improving processes, reducing or eliminating healthcare disparities, etc.). The goal of this change is to expand opportunities to highlight high-quality quality improvement work while concentrating the focus into a single session.

CoChair:

Speakers:

Josaura Fernandez Sanchez, MD
Texas Children’s Hospital, Baylor College of Medicine
Houston,  TX
Standardizing Emergency Department Management of Iron Deficiency Anemia from Heavy Menstrual Bleeding

Paolo Lopedote, MD
City of Hope National Medical Center
Monrovia,  CA
Reduced Blood Transfusions in Patients with Chronic Liver Disease Undergoing Paracentesis

Asinamai M Ndai, MS,BPharm
University of Florida Health Physicians
Gainesville,  FL
Optimized Management of Hemophilia in the Emergency Department

ASH-FDA Joint Symposium I: Accelerating Drug Development for Hematologic Malignancies

Monday, December 9, 2024, 10:30 a.m. - 12:00 p.m. San Diego Convention Center, Room 11

Chair:

Cara Rabik, MD,PhD
Food and Drug Administration
Silver Spring,  MD

Speakers:

Maryam Sarraf Yazdy, MD
Food and Drug Administration
Silver Spring, 
From Detection to Development: A Regulatory Perspective on Measurable Residual Disease

Emily Jen, MD,PhD
US Food and Drug Administration
Silver Spring,  MD
Development of Residual Disease as a Biomarker in Acute Leukemia

Christopher S. Hourigan, DM DPhil
Virginia Tech FBRI Cancer Research Center
Washington,  DC
Measurable Residual Disease Testing in AML: Challenges and Progress

'Lola Fashoyin-Aje, MD, MPH
US Food and Drug Administration
Silver Spring,  MD
Recent Approvals of CAR T Therapy for Hematologic Malignancies

Najat Bouchkouj, MD
US Food and Drug Administration
Silver Spring,  MD
Obecabtagene Autoleucel for Treatment of Adults with Relapsed/Refractory B-Cell Precursor Acute Lymphoblastic Leukemia

Hetty E. Carraway, MD,MBA
Cleveland Clinic Taussig Cancer Institute
Cleveland,  OH
Clinical Discussant for Acute Lymphoblastic Leukemia

Cara Rabik, MD,PhD
Food and Drug Administration
Silver Spring,  MD
Panel Discussion

ASH-FDA Joint Symposium II: Challenges and Innovations in Rare Diseases in Non-Malignant Hematology

Monday, December 9, 2024, 4:30 p.m. - 6:00 p.m. San Diego Convention Center, Room 7

Chair:

Donna A. Whyte-Stewart, MD,MSc
Food and Drug Administration
Silver Spring,  MD

Speakers:

Alison R. Moliterno, MD
Food and Drug Administration
Silver Spring,  MD
Mavorixafor for Treatment of WHIM Syndrome

Hanny Al-Samkari, MD
Massachusetts General Hospital
Cambridge,  MA
Mavorixafor, a Novel CXC4 Agonist for WHIM Syndrome: Challenges in Successful Drug Development in Rare Hematologic Disorders

Roma Rajput, MD
Food and Drug Administration
Silver Spring,  MD
Marstacimab-hncq for Treatment of Hemophilia A or Hemophilia B

Megha Kaushal, MD, MSc
US Food and Drug Administration
Silver Spring,  MD
Fidanacogene Elaparvovec-dzkt for Treatment of Adults with Moderate to Severe Hemophilia B

Robert F Sidonio Jr., MD
Emory University School of Medicine
Atlanta,  GA
Clinical Discussant for Hemophilia

Janet L. Kwiatkowski, MD
Children's Hospital of Philadelphia
Philadelphia,  PA
Clinical Discussant for Thalassemia

Donna A. Whyte-Stewart, MD,MSc
Food and Drug Administration
Silver Spring,  MD
Panel Discussion

Clinical Care and Research Implications Following Voxelotor Withdrawal

Monday, December 9, 2024, 10:30 a.m. - 12:00 p.m. San Diego Convention Center, Room 33

Chair:

John J Strouse, MD
Duke University
Durham,  NC

Speakers:

John J Strouse, MD
Duke University
Durham,  NC
Immediate Fall Out Post-Voxelotor Withdrawal

Maureen Okam Achebe, MD, MPH
Brigham and Women's Hospital, Harvard Medical School
Boston,  MA
Implications for Ongoing Clinical Research in SCD and Drug Development

Alexis Thompson, MD, MPH
Perelman School of Medicine, University of Pennsylvania
Philadelphia,  PA
Landscape of SCD Treatment Options and New Therapies

Early Faculty Career Development Session

Monday, December 9, 2024, 10:30 a.m. - 12:00 p.m. San Diego Convention Center, Room 25

In this Junior Faculty Career Development Session, attendees will hear a series of lectures focused on Mentorship. These didactic lectures will feature mentoring experts sharing their experiences in how to receive, provide, and optimize mentoring. Mentor/Mentee relationships can be hard to foster, and in this session, we will focus on empowering junior faculty with the tools to get the most out of this relationship. Junior faculty will hear from mentors and mentees both in the clinical and basic research fields and gain a better understanding on what it is to be a good mentor and mentee.

CoChair:

Speakers:

Betty S. Pace, MD
Augusta University
Augusta,  GA
Identifying a Mentor and Optimizing the Mentor/Mentee Relationship, How to be a Good Mentor?

Adam Scott Kittai, MD
The Ohio State University
Columbus,  OH
Identifying a Mentor and Optimizing the Mentor/Mentee Relationship, How to be a Good Mentor?

Joseph R. Mikhael
Translational Genomics Research Institute
Phoenix,  AZ
The Warning Signs of a Problematic Mentoring Relationship and How to Fix it

Shannon Elf, PhD
University of Utah
Salt Lake City,  UT
The Warning Signs of a Problematic Mentoring Relationship and How to Fix it

Leidy L. Isenalumhe, MD,MS
Moffitt Cancer Center
Lutz,  FL
Transitioning from Being a Mentee to Becoming a Mentor

Raquel Espin Palazon, PhD
Iowa State University
Ames,  IA
Transitioning from Being a Mentee to Becoming a Mentor

Health Equity Symposium: Race, Structural Racism and the Social Determinants of Health in Hematology

Sunday, December 8, 2024, 9:30 a.m. - 11:00 a.m. San Diego Convention Center, Room 6DE

The misuse of race in medicine has far-reaching implications across practice, teaching, and research in hematology. This session is intended to explore what race is and what it is not, and illustrate how systemic factors, including racism, influence health care access and outcomes. The session will also explore strategies to tackle systemic racism in patient care and how to create conditions for equitable care for patients with blood diseases. Dr. Joseph L. Graves Jr. will provide a summary of the history of biological conceptions of race and describe modern evolutionary conceptions of race. He will then address how this understanding can be useful in employing the power of modern genomic tools applied to human biological diversity, including how the use of these tools completely unravels the false premises of racial medicine. Dr. Chanita Hughes Halbert will define health care disparities and describe the nature and distribution of social determinants of disparities in disease risk and outcomes. She will also provide an overview of structural racism and its impact on health care outcomes in diverse populations.

Chair:

Deirdra R Terrell, PhD
University of Oklahoma Health Sciences Center
Oklahoma City,  OK

Speakers:

Joseph L Graves Jr., PhD
North Carolina A&T State University
Greensboro,  NC
The Myth of Race in Medical Science

Chanita Hughes Halbert, PhD
University of Southern California
Los Angeles,  CA
Structural Racism, the Social Determinants of Health, and Healthcare Outcomes – Understanding Inequity and What to Do About It

Medical Educators Symposium

Sunday, December 8, 2024, 7:30 a.m. - 9:30 a.m. Marriott Marquis San Diego Marina, Marina Ballroom FG

The Medical Educators Symposium (previously called the Hematology Course Directors Workshop) is open to all medical educators across the medical student, resident, and fellow continuum. The Medical Educators Symposium highlights cutting edge teaching techniques and theories while also showcasing participants of the ASH Medical Educators Institute. The symposium is two hours long and will begin with an hour of general sessions focused on medical education principles and then followed by simultaneous breakout sessions that happen twice.

Chair:

Hetty E. Carraway, MD,MBA
Cleveland Clinic
Cleveland,  OH

Speakers:

Alison W Loren, MD
University of Pennsylvania
Philadelphia,  PA
General Session 1: Paving a Career in Medical Education & Scholarship

Megan Murray Dupuis, MD, PhD
Vanderbilt University Medical Center
Nashville,  TN
General Session 2: Utilizing Humanities to Influence and Tailor Hematology Curriculum for Adult Learners

Layla N Van Doren, MD, MBA
Yale University
New York,  NY
General Session 3: Incorporating Health Equity Education in UME/GME Curriculum

Alexander A Boucher, MD
University of Minnesota
Minneapolis,  MN
General Session 3: Incorporating Health Equity Education in UME/GME Curriculum

Beverly Schaefer, MD
University of Buffalo
Buffalo,  NY
Breakout 1: Challenges of Course Development

Ariela L. Marshall, MD
University of Minnesota
Rochester,  MN
Breakout 2: Developing Your Teaching Portfolio

Jason A Freed, MD
Beth Israel Deaconess
Boston,  MA
Breakout 3: Cutting-Edge Technology in Education Delivery

Thomas W. LeBlanc, MD,MA,MS
Duke Cancer Institute
Hillsborough,  NC
Breakout 4: Developing a Palliative Care/Difficult Discussion Curriculum

Sickle Cell Disease Centers Session

Sunday, December 8, 2024, 9:30 a.m. - 11:00 a.m. San Diego Convention Center, Room 33

To ensure high-quality care for patients with sickle cell disease (SCD), it is imperative that providers and SCD care leaders have the tools to establish comprehensive clinical care centers for patients with SCD. Over the last several years, ASH has developed a program and blueprint for the creation of sickle cell disease centers. This special interest session is designed to help sickle cell disease care centers understand the development of CMS quality metrics and how to leverage these metrics to support delivery of high-quality care.

CoChair:

Speakers:

Alexandra Power-Hays, MD
Cincinnati Children's Hospital
Cincinnati,  OH
The Importance of Recognition of Pediatric Quality Metrics in SCD: The Role of US News and World Report

Titilope Fasipe, MD,PhD
Texas Children's Cancer and Hematology Centers
Houston,  TX
The Creation of CMS Quality Metrics in the Treatment of Painful Episodes in the Emergency Department

Julie Kanter, MD
University of Alabama at Birmingham
Birmingham,  AL
Leveraging Quality Metrics to Maintain Support of Your Sickle Cell Disease Center

Symposium for Mid-Career Hematologists

Sunday, December 8, 2024, 4:30 p.m. - 6:00 p.m. San Diego Convention Center, Room 24

This symposium aims to guide the audience through the common challenges faced by mid-career hematologists. Some examples of unique challenges for mid-career hematologists include negotiating the first leadership position, principles of leadership, promotion, strategies for managing individuals and teams, conflict management, setting up a successful research program, time management, funding beyond career development awards, performance and compensation metrics, self-care and opportunities to contribute to the Society’s mission. Speakers will include a diverse pool of successful physicians and scientists from academia, regulatory agencies, and the pharmaceutical industry. It is envisioned that this educational session will be organized by a steering committee of Society members who will identify a longitudinal curriculum for the session and speakers and topics.

Chair:

Juan Pablo Alderuccio, MD
Sylvester Comprehensive Cancer Center University of Miami
Miami,  FL

Shruti Chaturvedi, MBBS
Johns Hopkins University
Baltimore,  MD

Speakers:

Ruben A. Mesa, MD
Atrium Health Wake Forest Baptist
Winston-Salem,  NC
Negotiating Your First Leadership Position

Allison A. King, MD,PhD,MPH
Washington University School of Medicine
Saint Louis,  MO
Negotiating Your First Leadership Position

Robert F. Sidonio, Jr., MD, MSc
Emory University
Atlanta,  GA
Your Next Job - How to Identify Academic Opportunities with the Best Payback

Jeffrey D Lebensburger, DO
University of Alabama at Birmingham
Birmingham,  AL
Your Next Job - How to Identify Academic Opportunities with the Best Payback

David P. Steensma, MD
Ajax Therapeutics
Cambridge,  MA
Making the Jump - Careers Outside Academia

Nina Shah, MD
AstraZeneca
South San Francisco,  CA
Making the Jump - Careers Outside Academia

Symposium on Quality: Treating Fairly - The Role of Quality Improvement in Combating Health Care Disparities

Saturday, December 7, 2024, 2:00 p.m. - 3:30 p.m. San Diego Convention Center, Room 11

This year's Quality Symposium will focus on practical strategies for combatting healthcare disparities. First, Dr. Melissa Creary will discuss the Michigan Social Health Interventions to Eliminate Disparities (MSHIELD) program, a statewide data-driven, community-partnered, and equity-centered quality improvement effort. Then Dr. Michelle Sholzberg will review the development and impact of the "Raise the Bar" project, which aims to eliminate disparities in the recognition and management of iron deficiency by adjusting reference ranges. The session will conclude with a talk by Dr. Thomas Greg Knight on mitigating the impact of financial toxicity in patients with hematologic malignancies.

CoChair:

Speakers:

Melissa Creary
University of Michigan School of Public Health
Ann Arbor,  MI
MSHIELD:  A Statewide Approach to Improving Equity Centered Quality Improvement

Michelle Sholzberg, MDCM, MSc
University of Toronto
Toronto,  ON, Canada
Raise the Bar - Combatting Disparities in the Recognition and Management of Iron Deficiency

Thomas G. Knight, MD
Atrium Health Levine Cancer Institute, Wake Forest University School of Medicine
Charlotte,  NC
Cancer and Poverty - Mitigating the Impact of Financial Toxicity in Patients with Hematologic Malignancies

Systems-Based Hematology Education and Networking Session: Anticoagulation Stewardship and AI-Assisted Management

Sunday, December 8, 2024, 4:30 p.m. - 6:30 p.m. San Diego Convention Center, Room 25

This session will feature two talks on systems-level approaches to anticoagulation management, followed by a networking session where attendees will meet with the speakers and with established systems-based hematologists over light hors d'oeuvres and drinks. The first talk with feature Dr. Greg Barnes discussing how to improve systems-level management of anticoagulants within and across care settings. For the second talk, Dr. Damon E. Houghton will discuss the use of artificial intelligence (AI) and machine learning (ML) in the context of anticoagulation management.

Chair:

Camila Masias, MD,MPH
Miami Cancer Institute
Miami,  FL

Speakers:

Geoffrey D Barnes, MD, MSc
University of Michigan
Ann Arbor,  MI
Improving Systems-Level Management of Anticoagulants Within and Across Care Settings

Damon E. Houghton, MD,MS
Mayo Clinic Rochester
Rochester,  MN
Artificial Intelligence (AI) and Machine Learning (ML) in the Context of Anticoagulation Management

Training Program Directors Workshop

Friday, December 6, 2024, 9:00 a.m. - 12:00 p.m. Hilton San Diego Bayfront, Indigo 204

The Training Program Director’s Workshop is held each year before the ASH annual meeting and is designed for training program directors, associate program directors, and others involved with hematology training. The workshop is designed for hematology program directors to share lessons learned, best practice, and explore opportunities to improve their leadership skills. Through experiential learning techniques, breakout sessions, and interactive lectures, program directors can learn from peers and build their network. At this year’s TPDW, we will focus on three opportune topics. First, we will discuss how to design and implement innovative curriculums which can prepare trainees to excel at various aspects of hematology oncology practice beyond just knowledge of the subject matter. Examples of such curriculums include ‘leadership training’, ‘how to be a good educator’, and ‘business of hematology-oncology’. Second, we will focus on best practices for a training program to leverage social media and build their ‘brand’. Lastly, we will discuss practical ways in which programs can help trainees land their dream job.

Chair:

Pallawi Torka
Roswell Park Cancer Center
Buffalo,  NY

Speakers:

Scott Moerdler, MD
Rutgers Cancer Institute
New Brunswick,  NJ
Innovative Fellowship Curriculum

Layla N Van Doren, MD, MBA
Yale School of Medicine
New Haven,  CT
Innovative Fellowship Curriculum

Angela C Weyand, MD
University of Michigan
Ann Arbor,  MI
Leveraging and Enriching Social Media

Gerald Hsu, MD,PhD
University of California, San Francisco
San Francisco,  CA
Best Practices to Support Fellows in the Job Search

Sunandana Chandra, MD
Northwestern University
Chicago,  IL
Best Practices to Support Fellows in the Job Search