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ASH Annual Meeting and Exposition

Education Program

Education Program sessions will take place in person and stream simultaneously on the virtual platform (the recording will be available on demand). Sessions will consist of didactic presentations followed by panel discussions and a question-and-answer period with all speakers. Session descriptions for this program will be available in the Annual Meeting App.

Sessions on Malignant Hematology

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


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

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A Shifting Landscape: Treatment of MCL


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
Ohio State University Hospital
Columbus, OH

Speakers:

Kami J. Maddocks, MD
Ohio State University Hospital
Columbus, OH
Your Chemo Is No Good Here – Management of “High-Risk” MCL: TP53mut, Blastoid, CK+ Disease etc

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

Maria Lia Palomba, MD
Memorial Sloan Kettering
New York, NY
The Challenges of Cellular and Other Immunotherapy for MCL (Late Line MCL)

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Addressing Unmet Needs in T Cell Lymphomas


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
Expanding Options for Treatment of CTCL

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

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Adult ALL Advancements: Optimizing Cure in 2024


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?

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


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
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

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


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
Fred Hutchinson Cancer Center
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 Patient

Courtney D. DiNardo, MD,MSc
MD Anderson Cancer Center
Houston, TX
Incorporating Mutational Data and MRD Assessments into Treatment Decisions for Transplant-Ineligible Patients

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Chasing Zebras: Navigating Rare Myeloid Neoplasms


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

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Follicular Lymphoma: Playing the Long Game


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
Follicular Lymphoma: In Pursuit of a Functional Cure

Carla Casulo, MD
James P. Wilmot Cancer Center
Rochester, NY
The POD24 Challenge: Where Do We Go From Here in Follicular Lymphoma?

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

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Handling Bad News: How to Best Manage TP53 Myeloid Disease?


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 Y. Konopleva
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

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It's a Different World: CLL 2024


The treatment paradigm of chronic lymphocytic leukemia (CLL) has been transformed over the past decade and CLL can serve today as a model system for chemotherapy-free cancer therapy. This is based on better understanding of disease biology, in particular regarding molecular genetic drivers and relevant signaling pathways. The latter has led to the development of agents targeting B cell receptor (in particular BTK) and apoptosis (in particular BCL2) pathways which have replaced chemoimmunotherapy (CIT) as the treatment standard. BTK and BCL2 inhibitors, when used alone or in combination or with CD20 antibodies have shown improved progression-free survival (PFS) and in some instances even improved overall survival (OS) against CIT in frontline phase 3 trials. This improvement is particularly pronounced in high-risk CLL subgroups defined by unmutated IGHV, deletion 17p and/or mutation of TP53, making CIT in these subgroups essentially obsolete. Despite these remarkable advances, these risk markers retain some prognostic impact also in the context of targeted therapy making the stratification in frontline management a relevant topic. Furthermore, BTK and BCL2-targeting agents differ in their adverse event profile, making evaluation and treatment guidance based on patient characteristics such as coexisting conditions, comedication and delivery-of-care aspects important in treatment choice.

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

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 SCT, Cellular and Non-cellular Immunotherapy

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


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
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

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Moving the Needle in Hodgkin Lymphoma


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 Herrera
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 Advani
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 cHL

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MPN Practice Pearls: Profiling, Pegylated IFN and Pregnancy


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
Stanford, CA

Speakers:

Adam J Mead, MD PhD
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 IFN: The Who, Why, and How

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

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Newly Diagnosed Multiple Myeloma: Many Choices and More Questions


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 Kunnathu Kumar, MD
Mayo Clinic
Rochester, MN

Speakers:

Shaji Kunnathu 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

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


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
Post-transplant Strategies for Relapse Prevention and Treatment

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Sessions on Non-Malignant Hematology

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


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 (ALPS)

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

Thierry Lamy, MD
Pontchaillou Hospital Rennes UNIVERSITY HOSPITAL
Rennes, France
Large Granular Lymphocytosis (LGL): A Clonal Disorder with Autoimmune Manifestations

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Anticoagulation SOS: Navigating Monitoring Conundrums


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
Measuring the effect of DOACs: Why, When, and How?

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

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

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Beyond TTP: “Atypical” TMAs for the Hematologist


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 HUS: Diagnosis, Management, and Discontinuation 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 (CAPS) and Anticoagulant-Refractory APS

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Challenges and Opportunities of Sickle Cell Care in Africa


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, MD
The Hospital For Sick Children, 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, MD
The Hospital For Sick Children, 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
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

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Challenges in Cancer-Associated Thrombosis


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
The Ottawa Hospital and the 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 Zwicker, MD
Memorial Sloan Kettering
NYC, NY
Trousseau’s Syndrome: Management of Refractory VTE

Cihan Ay, MD
Universidad de Medicina de Viena
Vienna, Austria
Treatment of VTE in the Thrombocytopenic Cancer Patient

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Hematologic Challenges at the End-of-Life


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
University of Alabama At Birmingham
Birmingham, AL
End-of-Life in the Sickle Cell Patient: Life Expectancy and the Role of Palliative Care

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Hematologic Management of the Patient with Heavy Menstrual Bleeding


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
Versiti
Milwaukee, WI

Speakers:

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

Juliana Perez Botero, MD
Versiti
Milwaukee, WI
Von Willebrand's Disease and Heavy Menstrual Bleeding: When to Test, How to Test, and Use of Targeted Therapies

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

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


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 Diagnosis and Management of “Typical” HIT(T)

Toyosi Onwuemene, MD,MS
Duke University
Durham, NC
Alternative 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

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Integrating New Therapies into the Management of Classical Heme Disorders


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:

Ali T Taher, MD,PhD,FRCP
American University of Beirut Medical Center
Beirut, Lebanon

Speakers:

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

Ali T Taher, MD,PhD,FRCP
American University of Beirut Medical Center
Beirut, Lebanon
Luspatercept: A Treatment for Ineffective Erythropoesis in Thalassemia

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

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


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
Management of Iron Overload in Acquired Sideroblastic Anemias and MDS: Role of Chelation and Luspatercept

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

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Not All Lymphadenopathy is Lymphoma: Lymphoproliferative Diseases of Immune Dysregulation


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,MMEd
Dalhousie University
Halifax, NS, Canada

Speakers:

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

Luke Y. C. Chen, MD,MMEd
Dalhousie University
Halifax, NS, Canada
IgG-4 Disease for the Hematologist

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

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Optimizing Non-Curative Therapies for Sickle Cell Disease


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 Management of Children and Adolescents with SCD

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

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

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


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
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

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The Expanding Landscape of Treatment for ITP


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 ITP: Algorithm for Management and Role of Multi-Modal Therapy

Annemarie E Fogerty, M.D.
Massachusetts General Hospital
Boston, MA
Management of ITP during Pregnancy

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

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


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 Children's Hospital of Philadelphia
Philadelphia, PA
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