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Education Program

Junior Faculty Career Development Education Session


Structured as an education session, although only offered once, this session focuses on important career development lessons and advice. This years' session focuses on considerations for a new job.

Chair:

Yvonne A. Efebera, MD, MPH
OhioHealth
Columbus, OH

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Sessions on Malignant Hematology

Acute Myeloid Leukemia: Improving Outcomes in Challenging Subsets


The therapeutic landscape for Acute Myeloid Leukemia (AML) patients has undergone a remarkable transformation in the past five years.  The addition of small molecule inhibitors, such as BCL-2, IDH, and FLT3 inhibitors, have led to increased treatment options and improved outcomes for many patients.  However, despite these advancements, the majority of patients will not be cured of their disease.  Measurable residual disease (MRD) testing in remission after treatment for AML can identify patients at increased risk of relapse and death. There are also ongoing efforts investigating novel therapeutics for high-risk AML patient subsets to improve current unacceptable poor outcomes.  This educational session will examine the current evidence for integrating MRD into response assessments and as a potential treatment goal for patients.  The session will also discuss ongoing investigational efforts to improve outcomes in high-risk AML patient subsets such as secondary AML and the genomic subsets: MLL rearranged, FLT3-ITD and TP53 mutated.

Dr. Chris Hourigan will outline the evidence and challenges currently for use of MRD testing in AML to inform treatment decision making and management.  Factors such as lack of test harmonization and unclear clinical utility for individual patients have limited widespread adoption of AML MRD testing in clinical practice. He will also discuss the current multiple national-level precision medicine efforts now ongoing to both develop optimal testing and validate AML MRD negativity as a goal of therapy.

Dr. Keith Pratz will present case-based approaches to suggest best practices for current treatment approaches for patients with secondary AML including those with prior chemotherapy exposure or arising from antecedent hematologic malignancy.  He will discuss ongoing opportunities and challenges for these high-risk patient populations and ongoing research efforts to improve upon the current poor outcomes of these patients.  

Dr. Alice Mims will discuss current ongoing investigational efforts to improve patient outcomes in particular high risk AML molecular subsets: TP53 mutated, MLL or KMT2A rearranged, and FLT3-ITD mutated disease.  This talk will examine best current treatment approaches for patients with these specific genomic features along with completed and ongoing research endeavors including novel approaches with immunotherapeutics and targeted small molecule inhibitors.  

Chair:

Alice S. Mims, MD
The Ohio State University
Columbus, OH

Speakers:

Christopher S Hourigan
National Institutes of Health
Bethesda, MD
Achieving MRD Negativity in AML: How Important Is This and How Do We Get There?

Keith W. Pratz, MD
University of Pennsylvania
Philadelphia, PA
Optimizing Outcomes in Secondary AML

Alice S. Mims, MD
The Ohio State University
Columbus, OH
Novel Investigational Approaches for High Risk Molecular Subsets: TP53, MLL, FLT3

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Are Alternative Donors Now Mainstream in Allogeneic Transplant?


Hematopoietic cell transplantation (HCT) is the only potential curative treatment for most of malignant and some of the non-malignant hematological disorders. Recent studies comparing donor to no donor treatment in transplant eligible patients have shown better outcomes in patients undergoing HCT. Donor availability has been one of the important access obstacles to this curative therapy since matched sibling donor availability has been reported to be only ~30%. Matched unrelated donor (MUD) availability is in the 20-40% range for African- and Hispanic- Americans and donor availability is even lower in mixed race and ethnicity which has not been accurately estimated in published data. These estimates are even lower counting to HLA typing, medical deferrals, and suitability related issues. In the absence of a MSD or MUD, alternative donor choices including: haploidentical related (HAPLO), mismatched unrelated (MMUD) and umbilical cord blood (UCB) all have contributed to improve access and outcome of patients who are otherwise eligible for HCT.

Mr. Stephen Spellman will discuss HLA matching and impact on HCT outcomes. He will use a case-based approach focusing on the likelihood of availability by donor type by patient ethnic background considering matched sibling donors and alternative donors, including matched and mismatched unrelated, cord blood and haploidentical related donors. His talk will address complete HLA match by donor type, discussion of areas with limited data available and current controversies warranting future research. 

 

Dr. Monzr Al Malki will discuss the rapid growth on the use of alternative donors in HCT; specifically, in MMUD setting, including historical data and outcome analysis using conventional calcineurin inhibitor (CNI)-based graft-versus-host disease (GvHD) prophylaxis, optimal choice among multiple MMUDs available using different mis/matching criteria, and the importance of avoiding donor-specific HLA antibodies (DSA) in mitigating the risk of graft rejection. Dr. Al Malki later described current novel approaches to improve outcomes in MMUD HCT including post-transplant cyclophosphamide (PTCy) and Abatacept (ABA), discussing some of the main differences between these novel approaches and active clinical trial.

Dr. Arnon Nagler will debate the HAPLO donor vs UCB as graft source options based on the currently available data. This discussion should give some guidance and help the transplant physician to choose among a haploidentical versus a cord blood donor.  Although the number of HAPLO transplants (mainly non-T cell-depleted haplo transplants with post-transplant cyclophosphamide) is increasing while UCB is decreasing worldwide, recent developments in UCB and especially cord blood expansion and other strategies to improve engraftment and immune reconstitution post-UCB make UCB as a valuable option. Given the limited numbers of published or ongoing well-designed randomized controlled trials, the decision to perform haplo transplant or CBT in each patient depends not only on the patient, disease and donor characteristics and availability (although most if not all patients should have in principle an alternative donor) but also on the transplant physician discretion and most importantly center experience, preference and center’s ongoing clinical trials and strategies.

Chair:

Monzr M. Al Malki, MD
City of Hope National Medical Center
Duarte, CA

Speakers:

Monzr M. Al Malki, MD
City of Hope National Medical Center
Duarte, CA
New Strategies for Mismatched Unrelated Donors

Stephen R. Spellman
National Marrow Donor Program
Minneapolis, MN
What Is Complete HLA Match in 2022?

Arnon Nagler, MD
Chaim Sheba Medical Center
Tel Hashomer, Israel
In 2022, Which is Preferred: Haploidentical or Cord Transplant?

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Beyond Routine Frontline Therapy of CML


Despite most chronic phase (CP-) CML patients achieving excellent outcomes with tyrosine kinase inhibitor (TKI) therapy there are still complex challenges confronting the clinician. In this session we will cover three of these specific challenges. For the patient who doesn’t respond well to front-line or second-line therapy further lines of TKI therapy are now available that may still achieve long-term disease control. In some cases, allogeneic stem cell transplantation (allo-HSCT) will need to be considered, to determine whether it provides the best prospect of long-term survival. Many younger women with CP-CML are now incorporating plans to raise a family when they are making treatment decisions. This is now a realistic and relatively safe consideration in women responding well to TKI therapy.

Dr Timothy Hughes will discuss the appropriate assessments that are needed in CP-CML patients being considered for a change in therapeutic approach because of TKI resistance. The choice of optimal TKI and TKI dose for a CP-CML patient with TKI resistance needs to be considered in the context of their prior response and tolerance, mutation profile, co-morbidities, and cardiovascular risk factors. This talk will outline strategies to integrate all these factors to assist in making the best recommendations for the patient to consider.

Dr Nicolaus Kroeger will discuss the challenge of integrating allo-HSCT as a curative treatment approach for chronic and advanced phases of CML in the treatment beyond frontline therapy. Despite lack of prospective randomised trials, the lecture will provide current results, Pros and Cons and international recommendations regarding allo-HSCT which are supportive and helpful for patient counselling and decision making.

Dr Jane Apperley will address an issue of considerable importance to patients with CML who are of child-bearing age, namely the safety of planned or unplanned pregnancies. She will discuss the management of patients who present during pregnancy, and then of those with established disease who wish to have a child. Decision-making requires sensitive consideration of the competing risks to patient and infant, and involvement of multi-disciplinary teams.

Chair:

Timothy P. Hughes, MD,MBBS,FRACP,FRCPA
South Australian Health and Medical Research Institute SAHMRI
Adelaide, SA, Australia

Speakers:

Timothy P. Hughes, MD,MBBS,FRACP,FRCPA
South Australian Health and Medical Research Institute SAHMRI
Adelaide, SA, Australia
Treatment of TKI resistant chronic phase CML

Nicolaus Kroeger, MD
University Medical Center Hamburg-Eppendorf
Hamburg, Germany
Transplantation in CML in the TKI Era: Who, When, How?

Jane F. Apperley, FRCP, FRCPath, MB
Hammersmith Hospital, Imperial College
London, ENG, United Kingdom
Treatment of CML in Pregnancy

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Controversies in Aggressive NHL


Diffuse large B-cell lymphoma (DLBCL) is the most common lymphoid malignancy worldwide comprising approximately 30% of all lymphomas. Currently, 50-60% of patients diagnosed with DLBCL are alive at 5 years and cured with modern therapy, but approximately 10-15% of patients will be refractory to first-line therapy and an additional 20-30% will relapse following a complete response. For the majority of patients who relapse, this occurs within the first 2 years of initial therapy. Patients who experience early relapse or who have primary refractory disease (less than a complete response or relapse within 3 to 6 months to initial therapy) have poor outcomes.

Dr Kate Cwynarski will outline the recent evidence questioning the efficacy of traditional methods for delivering CNS prophylaxis and the increasing focus on alternative interventions for this important clinical problem. She will also discuss the currently available risk stratification models and methods for identifying patients at high risk of CNS relapse and the potential for novel molecular diagnostics to improve patient selection in the future. She will also outline novel and emerging therapies presently under investigation. The talk will propose a revised approach to this contentious issue. 

Dr. Flowers will use patient scenarios to illustrate some of the key challenges faced by patients with relapsed DLBCL. He will discuss conventional management strategies for patients with relapsed/refractory DLBCL including high dose therapy and autologous stem cell transplantation and chimeric antigen receptor (CAR) T-cell therapy. He will also discuss results of recent trials that may change standard approaches and provide algorithms for sequencing therapy and managing patients in the modern era.

Dr. Anita Kumar will discuss the current guidelines and existing data that support the use of upfront autologous stem cell transplantation (ASCT) in mantle cell lymphoma (MCL). In contrast, she will also present the emerging evidence that challenges the applicability of this approach in the modern era. Additionally, she will discuss risk factors associated with outcome after ASCT and the potential role of minimal residual disease assessment in refining patient selection for ASCT in the future. Finally, she will discuss novel treatment approaches that incorporate biologically targeted therapies and risk-adapted treatment paradigms that are actively under investigation. Using a case-based approach, Dr. Kumar will highlight future opportunities for changing the established treatment paradigm for “transplant-eligible” MCL patients.

Chair:

Christopher R. Flowers
MD Anderson Cancer Center
Houston, TX

Speakers:

Kate Cwynarski, MBBS,PhD,FRCP,FRCPath
University College London
London, ENG, United Kingdom
CNS Prophylaxis in Aggressive B-Cell Lymphoma

Christopher R. Flowers
MD Anderson Cancer Center
Houston, TX
Sequencing Therapy in Relapsed DLBCL

Anita Kumar, MD
Memorial Sloan-Ketter Cancer Center
New York, NY
What Is the Role of Upfront Autologous Stem Cell Transplant in Mantle Cell Lymphoma?

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Immunotherapy in Multiple Myeloma


 

The importance of the immune system in multiple myeloma (MM) treatment was first demonstrated in patients undergoing allogeneic stem cell transplant. Unfortunately this early immunotherapy approach was significantly limited by transplant-associated morbidity and mortality. Since antibody-based immunotherapies have emerged as an important aspect of therapy for all MM patients, positively impacting survival. Novel antibody formats, chimeric antigen receptor (CAR) T-cells and non-cellular immune approaches demonstrate the vast possibilities for further innovation and offer new hope in a yet incurable disease.

Dr. Paula Rodriguez Otero will outline the current state of approved BCMA-directed CAR T-cell therapy in advanced MM as well as future developments focused on optimizing patient care and novel CAR designs. BCMA-directed CAR T-cells have shown unprecedented efficacy in heavily pre-treated MM leading to the approval of two BCMA CAR-T cell products. Still, no plateau is seen in the survival curves and relapses continue to occur. Therefore, further improvement is needed. Potential strategies including earlier use in MM as well as next generation CARs, to further augment efficacy will be discussed.

Dr. Suzanne Trudel will discuss the evolving therapeutic landscape of antibodies in MM including the use of naked antibodies in the frontline setting. Advances in antibody design have resulted in antibodies with improved properties to maximize efficacy that includes bispecific antibodies and antibody drug conjugates (ADCs). Belantamab mafodotin is an approved ADC-targeting BCMA, while BMCA-targeting bispecifics will soon be approved for commercial use. The efficacy of these novel antibodies in advanced MM, as well as the unique safety considerations of these agents will be discussed.

Dr. Sarah Holstein will provide an overview of non-cellular immunotherapy approaches to add to the MM therapeutic armamentarium. While CARs and novel antibodies are rapidly being developed, there continues to be a focus on non-cellular therapies that harness the immune system. The class of drugs that target the cereblon complex has expanded beyond the original immunomodulatory drugs (IMiDs) to include next-generation cereblon E3 ligase modulators (CELMoDs). Ongoing studies are evaluating the potential of adding novel agents including repurposed drugs to IMiDs/CELMoDs. Other novel immunotherapies under development include immunocytokines, immunotoxins and NK cell activators/engagers.

Chair:

Suzanne Trudel
McLaughlin Centre for Molecular Medicine
Toronto, ON, Canada

Speakers:

Paula Rodriguez Otero, MD, PhD
Servicio de Hematología y Hemoterapia, Clínica Universidad de Navarra
Pamplona, Spain
Cellular Therapy for Multiple Myeloma: What’s Now and What’s Next

Suzanne Trudel
McLaughlin Centre for Molecular Medicine
Toronto, ON, Canada
Antibodies and Bispecifics for Multiple Myeloma: Effective Effector Therapy

Sarah A. Holstein, MD, PhD
University of Nebraska Medical Center
Omaha, NE
Beyond the Cell: Novel Non-Cellular Immunotherapy Approaches to Multiple Myeloma

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Improving Outcomes in ALL


The treatment of acute lymphoblastic leukemia (ALL) is changing rapidly with the application of intensive pediatric regimens for adolescents and young adults, improved risk stratification with sensitive techniques for detection of measurable residual disease, and the application of novel, highly-active targeted therapies for Ph-negative and Ph-positive B-cell ALL. The purpose of this session is to understand current treatment approaches and ongoing clinical trials intended to improve cure rates and survival while reducing toxicity for older patients.

Nicolas Boissel, MD, PhD will discuss current approaches and ongoing clinical trials integrating novel agents for adolescents and young adults with ALL.

Kristen O'Dwyer, MD will provide an update of the treatment of T-cell ALL and future directions in the treatment of this rare disease.

Matthew Wieduwilt, MD, PhD will give an overview of front-line therapy for adults with Ph+ ALL including how highly-active targeted therapies have improved the prognosis of this disease over the last two decades.

Chair:

Matthew J. Wieduwilt, MD, PhD
University of California, San Diego
La Jolla, CA

Speakers:

Nicolas Boissel, MD, PhD
Saint-Louis Hospital
Paris, France
New Developments in ALL in AYA

Kristen M. O'Dwyer, MD
University of Rochester Medical Center
Rochester, NY
Optimal Approach to T-Cell ALL

Matthew J. Wieduwilt, MD, PhD
University of California, San Diego
La Jolla, CA
Ph+ ALL in 2022: Is There an Optimal Approach?

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JAK/STAT Inhibition and Beyond in Ph Negative MPNs


Myeloproliferative neoplasms (MPNs), including essential thrombocytosis (ET), polycythemia vera (PV), and myelofibrosis (MF) are hematopoietic stem cell neoplasms with heterogeneous clinical features and outcomes, but a common pathogenic driver:  JAK/STAT pathway activation.  Despite the development of JAK pathway inhibitors, many clinical challenges and unmet needs persist in the MPN field.  This educational session will focus on actively evolving areas of MPN clinical management and research, including molecular prognostication across MPN subtypes, management of cytopenic MF, and approaches to accelerated or blast phase MPNs.  

Dr. Alessandro Maria Vannucchi will outline the most frequent chromosomal and mutation abnormalities detected in chronic MPNs and discuss the evidence, in some instances still limited, that such knowledge might inform clinical decision making. By using an instructive clinical case, he will highlight how molecular-integrated risk score systems might enhance the identification of patients with myelofibrosis and poor prognosis, as compared to conventional clinical scores, facilitating their referral to stem cell transplantation.

Dr. Kristen Pettit will focus on a phenotypically distinct subset of MF, termed "cytopenic MF."  She will present the clinical features, outcomes, and limitations unique to cytopenic MF.  She will also discuss new and emerging strategies to treat this distinct and challenging disease state.  

Dr. Raajit Rampal will review the clinical features of accelerated or blast phase MPNs, and discuss pathologic drivers of MPN evolution towards these more aggressive disease states.  He will discuss current treatment options for accelerated of blast phase MPNs, highlighting novel therapeutic options as well as the need for more effective therapies.

Chair:

Kristen M. Pettit
University of Michigan
Ann Arbor, MI

Speakers:

Alessandro Vannucchi, MD
University of Florence, Azienda Ospedaliero Universitaria Careggi, CRIMM
Florence, Italy
Molecular Prognostication in Ph Negative MPNs in 2022

Kristen M. Pettit
University of Michigan
Ann Arbor, MI
New Approaches to Tackle Cytopenic Myelofibrosis

Raajit Rampal, MD, PhD
Memorial Sloan Kettering Cancer Center
New York, NY
Management of Accelerated and Blast Phase MPN

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Late Effects Monitoring for Survivors of Pediatric Hematologic Malignancies


Survivors of pediatric hematologic malignancies are at increased risk of long-term medical and psychosocial late effects as a consequence of their treatment, and in some instances, also secondary to underlying genetic predisposition. This educational session will review the evidence for three particularly significant toxicity categories: cardiovascular, neurocognitive, and secondary malignant neoplasms. The session will also review current screening and intervention strategies that may mitigate the development of these late effects. Finally, presenters will also highlight gaps and areas of future research.   

 

Dr. Eric Chow will provide background on the epidemiology of long-term treatment associated cardiovascular toxicity. He will then review current prevention and screening strategies, and touch on key management considerations.

 

Dr. Kevin Krull will outline the risk factors that are associated with neurocognitive deficits after therapy. In this session Dr. Krull also will review potential screening options, along with primary and secondary prevention options under current study.

 

Dr. Smita Bhatia will review the growing data informing genetic risk factors for secondary malignant neoplasms following cancer therapies, and the potential pathophysiologic pathways they reveal. Finally, she will discuss how these data can be incorporated into future screening and prediction algorithms.

Chair:

Eric J Chow, MD, MPH
Seattle Children's Hospital
Seattle, WA

Speakers:

Eric J Chow, MD, MPH
Seattle Children's Hospital
Seattle, WA
Mitigating, Monitoring, and Managing Long-Term Chemotherapy- and Radiation-Induced Cardiac Toxicity

Kevin R. Krull, PhD
St. Jude's
MEMPHIS, TN
Risk Factors and Screening for Neurocognitive Impacts of Therapy

Smita Bhatia, MD,MPH
University of Alabama at Birmingham
Birmingham, AL
Germ Line Risk Factors for Second Malignant Neoplasms After Treatment for Pediatric Hematologic Malignancies

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Maximizing Outcomes in CLL


The therapy of CLL has undergone a revolution in the last decade with the availability of highly effective targeted therapies.  The options continue to expand with next generation inhibitors and combination therapy.  The variety of available options can be confusing and their optimal use over a patient’s entire disease course is still often unclear.  More and more patients are developing disease progression after both BTK inhibitors and venetoclax and represent a new significant unmet need.  Additionally, Richter’s syndrome, the transformation of CLL to an aggressive lymphoma, remains a major clinical challenge with poor outcomes and is also a significant unmet need. 

Dr. Jennifer Brown will discuss the selection of initial therapy in CLL, including BTK inhibitors with or without anti-CD20 antibody and venetoclax-obinutuzumab, as well as recent data on combination therapy, with a focus on BTK inhibitor-venetoclax based combinations.  She will discuss the key unanswered questions and challenges in frontline therapy.

Dr. Lydia Scarfo will focus on the growing population of patients with CLL who have been treated with both BTK inhibitors and venetoclax, a population which represents a major unmet clinical need.  Her discussion will include the role of non-covalent BTK inhibitors, and cellular therapy (mainly CAR T cell approaches), as well as novel promising investigational agents and strategies.   

Dr. Tanya Siddiqi will discuss Richter’s syndrome, the transformation of CLL to an aggressive lymphoma, an event which occurs typically in patients with high risk features.  There remains no known effective standard of care and hence clinical trial enrollment is preferred when possible. Novel treatment approaches using combinations of small molecule targeted agents, antibody-based therapy, and/or CAR-T cell therapy appear to be more beneficial than chemoimmunotherapy alone.

Chair:

Jennifer R. Brown, MD, PhD
Dana-Farber Cancer Institute
Boston, MA

Speakers:

Jennifer R. Brown, MD, PhD
Dana-Farber Cancer Institute
Boston, MA
Selecting Initial Therapy in CLL

Lydia Scarfò
Ospedale San Raffaele
Milano, Italy
Novel Therapies and Combinations in CLL Refractory to BTK Inhibitors and Venetoclax

Tanya Siddiqi, MD
City of Hope National Medical Center
Duarte, CA
Treatment of Richter's Syndrome

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Multiple Myeloma: Assessing the Patient and the Disease


The treatment paradigm of multiple myeloma (MM) has dramatically changed over the past decade, offering many patients the chance for excellent disease control and longer survival. However, not every patient can tolerate the intensity of some interventions, while a subset of MM patients with high-risk disease likely should receive escalated therapy. In addition, the proper and complete assessment and categorization of patients is therefore mandatory.  This educational session will explore emerging evidence in the assessment and management of newly diagnosed and relapsed MM patients and cover clinical, diagnostic, and radiographic evaluations.

 

 Dr. Ciara Freeman will focus on the older, transplant ineligible myeloma patient population. Providers who treat myeloma will see an increasing volume of elderly patients for initial evaluation and subsequent lines of therapy. Older MM adults are more likely to be under- than over-treated, and therefore more objective (and ideally straightforward) ways to evaluate their fitness and ability to tolerate therapy will increasingly assist in decision making. The aim of this review is to highlight some of the approaches possible, how results might inform treatment selection, and illustrate ways that patients can be optimized for, rather than excluded from, the more complex therapies newly available.

Dr. Timothy Schmidt will discuss the challenges of defining and identifying high risk myeloma. A significant number of MM patients experience dramatically shorter disease-free intervals compared to “standard risk” patients. Dr. Schmidt will cover the development of both various biologic and molecular markers, as well as clinical data that can be used to define risk. In addition, he will review various treatment strategies that are explicitly employed for this group of patients.  

 

Dr. Taxiarchis Kourelis will explore recent developments in minimal residual disease testing, imaging, new biomarkers and their roles in risk assessment. The incorporation of more modern techniques such as 18FDG-positive emission tomography and magnetic resonance imaging, in various iterations, have led to an appreciation of the spacial heterogeneity of myeloma tumor deposits encountered among myeloma patients and to the realization of the inadequacy of traditional bone surveys. Dr. Kourelis will also review recent data that underscore the predictive power of such techniques to stratify risk and help guide treatment decision. He will also consider the role of MRD testing and the use of mass spectrometry to predict risk of progression and relapse. 

 

Chair:

Natalie Callander, MD
University of Wisconsin
Madison, WI

Speakers:

Ciara L. Freeman, MSc,FRCPath,MBBChir,MRCP
Moffitt Cancer Center
Tampa, FL
Fitness and Frailty in Myeloma

Timothy Martin Schmidt, MD
University of Wisconsin
Madison, WI
High or Low? Assessing Disease Risk in Multiple Myeloma

Taxiarchis Kourelis, MD
Mayo Clinic
Rochester, MN
The Burden of Myeloma: Novel Approaches to Disease Assessment

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Novel Approaches in MDS


Myelodysplastic syndromes (MDS) are characterized clinically by a hyperproliferative bone marrow, reflective of ineffective hematopoiesis, and usually accompanied by one or more peripheral cytopenias. This session will focus on the rapid advances in both diagnosis and treatment in the entire spectrum of the clinical presentation of MDS. The identification of various molecular mutations that impact the pathogenesis of the disease has resulted in new molecularly defined subtypes of MDS and a new prognostic classification, the IPSS-M. This has also led to advances in our understanding of the evolution of cytopenias to low risk MDS and the role of clonal hematopoiesis (CH) and inflammation in this process. This is an emerging area in the field of MDS where identification, close observation, and possibly early treatment of these entities may form part of the future strategy of the care of the MDS patient.Lastly, this session will detail the innovative therapies in combination with hypomethylating gents that are moving forward in the treatment of high risk MDS and their role in the treatment of MDS with various molecular mutations.

Dr. Mario Cazzola will discuss the increasing importance of gene sequencing  and the role of molecular mutational data in the diagnosis and  risk stratification of MDS. New molecularly-defined MDS entities in the updated pathology classifications will be discussed. The Molecular International Prognostic Scoring System for MDS (IPSS-M) which incorporates molecular mutational data into MDS risk classification, will also be illustrated, using a case-based approach.

Dr. Amit Verma will discuss the role of inflammation in the pathogenesis of ineffective hematopoiesis and cytopenias in MDS. He will outline the various inflammatory mediators that are elevated in MDS, including the TGF-beta pathways as well as inflammatory cascades triggered by spliceosome mutations in MDS. He will also summarize the translational/clinical efforts targeting these pathways in proposed and ongoing clinical trials.

Dr. Borate will discuss the various advances in clinical trials looking at different combinations with hypomethylating agents that are moving forward in high risk MDS. She will focus on agents that target  various dysregulated pathways in MDS including the BCL-2 pathway, various immunotherapy targeting agents including CD47 and TIM-3 as well as other biomarker and mutation specific agents being explored in high risk MDS. Lastly she will discuss the role of traditional AML chemotherapy agents in this high risk population.

Chair:

Uma Borate, MD
The Ohio State University
Columbus, OH

Speakers:

Mario Cazzola, MD
University of Pavia
Pavia, Italy
Risk Stratifying MDS in the Time of Precision Medicine

Amit Verma, MD
Albert Einstein College of Medicine
Bronx, NY
Targeting Inflammation in Lower Risk MDS

Uma Borate, MD
The Ohio State University
Columbus, OH
New Investigational Combinations For Higher-Risk MDS

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To Transplant or Not to Transplant in Active or High Risk Myeloid Disease


Chair:

Coleman Lindsley, MD, PhD
Dana-Farber Cancer Institute
Boston, MA

Speakers:

Daniel J. Weisdorf, MD
University of Minnesota
Minneapolis, MN
Transplant in Those with High Burden Disease: Refractory AML and High Risk MDS

Charles Craddock
Queen Elizabeth Hospital
Birmingham, ENG, United Kingdom
Transplant in AML with Measurable Residual Disease: Proceed or Defer?

Coleman Lindsley, MD, PhD
Dana-Farber Cancer Institute
Boston, MA
Transplant for TP53-Mutated MDS and AML: Because We Can or Because We Should?

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Treatment Approaches for the Multiple Myeloma Patient in 2022


Chair:

Yvonne A. Efebera, MD, MPH
Ohio Health
Columbus, OH

Speakers:

Sigrun Thorsteinsdottir, MD,PhD
Rigshospitalet
København, Copenhagen, Denmark
The Consultant’s Guide to Smoldering Multiple Myeloma

Caitlin Costello, MD
University of California, San Diego
La Jolla, CA
Newly Diagnosed Multiple Myeloma: Making Sense of the Menu

Yvonne A. Efebera, MD, MPH
Ohio Health
Columbus, OH
The First Relapse in Multiple Myeloma – How to Pick the Next Best Thing

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Updates in Targeted Therapy in Pediatric Leukemia


In this case-based session, Dr Sarah Tasian will review the biology of Philadelphia chromosome-like acute lymphoblastic leukemia (Ph-like ALL) and current genetic testing approaches to identify patients with this high-risk leukemia subtype. She will then discuss indications for integration of tyrosine kinase inhibitors (TKIs) into the therapy of patients with Ph-like ALL. Finally, she will highlight recently-completed, current, and planned TKI-based clinical trials for children and adolescents/young adults with Ph-like ALL.

Dr. Katherine Tarlock will discuss targeted therapies in children with AML including targeted small molecule inhibitors as well as immunotherapeutic agents.  She will focus on single gene mutations and aberrantly activated oncogenic pathways, and review some of challenges of mutation targeting and drug development in pediatric versus adult AML. She will also discuss the evolution of targeting cell surface antigens with a variety of immunotherapeutic strategies in pediatric AML. This talk will focus on targeted agents currently available and with active clinical trials, and will also touch on those in clinical trial development.

Dr. Rishi Kotecha will highlight the challenges of treating infants with acute lymphoblastic leukemia (ALL). This talk will focus on targeted agents that are being primed for clinical use and provide insight into the landscape of clinical trials that are currently in development for infants with ALL. In addition, Dr. Kotecha will review the outcomes from preclinical studies that have investigated novel targeted agents for infants with KMT2A-rearranged ALL and their potential for future clinical translation.   

Dr. Katherine Tarlock will discuss targeted therapies in children with AML including targeted small molecule inhibitors as well as immunotherapeutic agents.  She will focus on single gene mutations and aberrantly activated oncogenic pathways, and review some of challenges of mutation targeting and drug development in pediatric versus adult AML. She will also discuss the evolution of targeting cell surface antigens with a variety of immunotherapeutic strategies in pediatric AML. This talk will focus on targeted agents currently available and with active clinical trials, and will also touch on those in clinical trial development.

Chair:

Sarah K Tasian, MD
Children's Hospital of Philadelphia
Philadelphia, PA

Speakers:

Sarah K Tasian, MD
Children's Hospital of Philadelphia
Philadelphia, PA
Clinical Screening for Ph-like ALL and the Developing Role of TKIs

Katherine Tarlock, MD
Seattle Children's Hospital
Seattle, WA
The Evolution of Targeted Therapy in Pediatric AML: Small Molecule Inhibitors and Immunotherapeutic Strategies

Rishi Sury Kotecha, PhD,FRACP,MBBChir,MRCP
Perth Children's Hospital
Perth, Australia
Updates in Infant Leukemia and the Potential for Targeted Therapy

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What's New in Indolent Lymphoma


Session is designed to expire the biopsy of low grade lymphoma and link how this has lead to the introduction of several new agents in the management of patients. Dr. Jessica Okosun will discuss the pathogenesis of follicular lymphoma and how this has lead to development of several novel agents. Dr. Luca Arcaini will discuss options for patients with marginal zone lymphoma and its varied subtypes.

Chair:

Tycel J. J. Phillips, MD
University of Michigan
Ann Arbor, MI

Speakers:

Tycel J. J. Phillips, MD
University of Michigan
Ann Arbor, MI
Management of Early Relapsed Follicular Lymphoma

Jessica Okosun, MB BChir PhD
Barts Cancer Institute, Queen Mary University of London
London, United Kingdom
Pathogenesis of Follicular Lymphoma

Luca Arcaini
Department of Molecular Medicine, University of Pavia and Division of Hematology, Fondazione IRCCS Policlinico San Matteo
Pavia, Italy
Management of Marginal Zone Lymphoma

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Where Are We Headed in Hodgkin Lymphoma?


Classic Hodgkin lymphoma (cHL) and nodular lymphocyte predominant Hodgkin lymphoma (NLPHL) are distinct diseases with different treatment paradigms.  cHL is an aggressive disease that is highly curable, therefore current research is focused on developing regimens that maintain or improve upon efficacy and also minimize treatment-related toxicity.  Studies are beginning to demonstrate that incorporation of  brentuximab vedotin (BV) or PD-1 blockade into the front-line or second line setting for cHL may aid in achieving this goal.  NLPHL is typically an indolent disease associated with favorable outcomes; however there are certain features that can indicate risk for a more aggressive course.  Given its variable presentation, treatment for NLPHL is individualized.   

Dr. Alison Moskowitz will review the management of primary refractory/first relapsed Hodgkin lymphoma.  She will discuss data supporting the use of novel agents in second-line treatment and studies exploring transplant-free approaches for relapsed and refractory disease.

Dr. Sven Borchmann will review the management of Nodular lymphocyte predominant Hodgkin lymphoma (NLPHL). He will put emphasis on the need for an individualized approach considering patient preferences, disease aggressiveness and individual host factors.

Dr. Alex Herrera will present the available data regarding the use of brentuximab vedotin and PD-1 blockade in the frontline management of Hodgkin lymphoma. He will discuss the current and potential future role of the novel agents in the initial management of HL and ongoing clinical trials that seek to define those roles.

Chair:

Alison J. Moskowitz, MD
Memorial Sloan Kettering Cancer Center
New York, NY

Speakers:

Alex F. Herrera, MD
City of Hope
Los Angeles, CA
Incorporating Novel Agents in Frontline Treatment of HL

Sven Borchmann, MD
University of Cologne
Cologne, Germany
Management of Nodular Lymphocyte Predominant HL

Alison J. Moskowitz, MD
Memorial Sloan Kettering Cancer Center
New York, NY
Do All Patients with Primary Refractory/First Relapse of HL Need Autologous Stem Cell Transplant?

Sessions on Non-Malignant Hematology

Anxiety Provoking Consultations: Mast Cells and Eosinophils


Dr. Cem Akin will discuss various clinical presentations and referral scenarios for mast cell disorders in the hematology practice, and when and how to initiate further diagnostic workup and how to interpret the results. He will review differential diagnosis of a patient with elevated tryptase level including hereditary alpha tryptasemia, and mastocytosis. He will discuss treatment options for mast cell activation symptoms including new tyrosine kinase inhibitors in indolent systemic mastocytosis.

Dr. Amy Klion will review the differential diagnosis of marked eosinophilia and classification of hypereosinophilic syndromes.  She will provide a general approach to treatment of patients with idiopathic and lymphocyte-driven hypereosinophilic syndromes, focusing on eosinophil-targeted biologics. Lastly, Dr. Klion will briefly discuss the potential risks of eosinophil depletion.

Dr. Jason Gotlib will navigate the treatment of advanced systemic mastocytosis (AdvSM) and primary eosinophilic neoplasms including chronic eosinophilic leukemia (CEL) and myeloid/lymphoid neoplasms with fusion tyrosine kinases (MLN-eo).  He will review the latest data and future directions of using KIT inhibition in AdvSM, with an emphasis on the challenge of SM with an associated hematologic neoplasm.  Lastly, Dr. Gotlib will survey the molecular landscape of primary eosinophilic neoplasms and opportunities for targeted therapy, including tyrosine kinase inhibitors in MLN-eo with rearranged PDGFRA/B, FGFR1, JAK2, FLT3, and ABL1.

Chair:

Jason Gotlib, MD, MS
Stanford Cancer Center
Stanford, CA

Speakers:

Cem Akin, MD
University of Michigan
Ann Arbor, MI
How to Evaluate the Patient with a Suspected Mast Cell Disorder and How/When to Manage Symptoms

Amy D Klion, MD
National Institutes of Health
Bethesda, MD
How to Evaluate the Patient with a Suspected Eosinophil Disorder and How/When to Manage Symptoms

Jason Gotlib, MD, MS
Stanford Cancer Center
Stanford, CA
Available and Emerging Therapies for Bona Fide Advanced Systemic Mastocytosis and Primary Eosinophilic Neoplasms

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Autoimmune Hemolytic Anemias (AIHA)


The purpose of this session is to provide an overview of the diagnosis and treatment of autoimmune hemolytic anemias (AIHA). These disorders may be primary or secondary to other disorders including medications, infection, autoimmune and lymphoproliferative disorders. The underlying pathophysiology and diagnostic criteria for the warm and cold AIHAs will be reviewed. An important focus will be on the laboratory testing required for a proper diagnosis. Using illustrative clinical cases, the indications for therapy will be assessed and the available standard initial therapies reviewed. Since many patients with these disorders will require subsequent treatment, the indications and efficacy of second- and third-line treatments will be reviewed. There will be a review of novel treatments in development for both cold and warm AIHA. Since red blood cell support is often required and complicated in both types of AIHA, attention will be paid additionally to the transfusion medicine support required for these patients.

Chair:

David J. Kuter, MD,DPhil
Massachusetts General Hospital, Harvard Medical School
Boston, MA

Speakers:

David J. Kuter, MD,DPhil
Massachusetts General Hospital, Harvard Medical School
Boston, MA
Warm AIHA and the Best Treatment Strategies

Jenny M. Despotovic, DO,MS
Texas Children's Hospital/Baylor College of Medicine
Houston, TX
Cold AIHA and the Best Treatment Strategies

Sue T Johnson, MSTM, MLS(ASCP)SBB
Versiti Blood Center of Wisconsin
Milwaukee, WI
Evaluate the Difficulties of Evaluating AIHA Patients in the Reference Lab of the Blood Bank and the Best Transfusion Management Strategies

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Late Effects of Curative Therapy for Sickle Cell Disease


Chair:

Courtney D. Fitzhugh, MD
National Institutes of Health
Bethesda, MD

Speakers:

Courtney D. Fitzhugh, MD
National Institutes of Health
Bethesda, MD
Knowledge to Date on Secondary Malignancy

Shalini Shenoy, MD
Washington University School of Medicine
Saint Louis, MO
Organ Function Indications and Potential Improvements

Debra Friedman, MD
Vanderbilt-Ingram Cancer Center
Nashville, TN
Long-term health effects of curative therapies for SCD

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Managing Thrombocytopenia in Challenging Situations


Thrombocytopenia is one of the most common reasons for both inpatient and outpatient hematology consultation. Chemotherapy-induced thrombocytopenia, thrombocytopenia of chronic liver disease, and thrombocytopenia in the pregnant patient are frequently encountered and often challenging clinical situations, and optimal management in these situations can be elusive. New data for the use of thrombopoietin receptor agonists in these patients in specific situations offers opportunities for updated and modernized treatment paradigms but also raise many questions.

Dr. Hanny Al-Samkari will discuss new data for the use of the thrombopoietin receptor agonists to treat patients with chemotherapy-induced thrombocytopenia and how this data guides optimal and safe use of these agents in clinical practice.

Dr. Adam Cuker will describe the clinical consequences and optimal management of thrombocytopenia of chronic liver disease, including a discussion of the thrombopoietin receptor agonists now approved to treat these patients in the periprocedural setting.

Dr. Allyson Pishko will discuss the diagnostic challenges of thrombocytopenia in the pregnant patient as well as new data available regarding the management of pregnant patients with immune thrombocytopenia.

Chair:

Hanny Al-Samkari, MD
Massachusetts General Hospital, Harvard Medical School
Boston, MA

Speakers:

Hanny Al-Samkari, MD
Massachusetts General Hospital, Harvard Medical School
Boston, MA
Chemotherapy-Induced Thrombocytopenia

Adam Cuker, MD, MS
University of Pennsylvania
Philadelphia, PA
Thrombocytopenia in Liver Disease

Allyson M Pishko, MD
University of Pennsylvania
Philadelphia, PA
Thrombocytopenia in Pregnancy

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Obstetric Management and Complications in Sickle Cell Disease in High and Low Income Countries


Pregnancy in women with sickle cell disease (SCD) is a life-threatening condition. In a pooled analysis from recent obstetric and hematology studies conducted in low- and middle-income settings, maternal death in women with SCD is approximately 2,393 and 4,300 deaths per 100,000 live births with and without multidisciplinary care, respectively. In comparison, the USA and Northern Europe's general maternal mortality rate is approximately 23.8 and 8 deaths per 100,000 live births, respectively.


Dr. Eugenia Vicky Asare will highlight the added value of a combined obstetric and sickle cell disease outpatient and inpatient care program to integrate evidence based-management for SCD-related acute pain, acute chest syndrome, and pulmonary thromboembolism. Using the multidisciplinary SCD obstetrics care approach in Ghana, she will highlight best-integrated clinical practices to decrease SCD-related morbidity and mortality in pregnant women with SCD.


Dr. Bosede Afolabi will speak on the difficulties in diagnosing complications in pregnant women with SCD in LMICs, emphasizing Nigeria, the country with the most significant number of individuals living with SCD worldwide. She will discuss the evidence-based preventive and therapeutic options available to treat pregnant women with SCD and the innovations and adaptations used in LMICs to substitute for limited resources.


Dr. Eugene Oteng-Ntim will discuss the Evidence-Based Management of Pregnant Women with SCD in High-Income Countries.

Chair:

Eugenia Vicky Vicky Asare, BSc
Ghana Institute of Clinical Genetics
Accra, Ghana

Speakers:

Eugenia Vicky Vicky Asare, BSc
Ghana Institute of Clinical Genetics
Accra, N/A, Ghana
Epidemiology of Obstetric Complications Among women with Sickle Cell Disease

Bosede Afolabi, DM
University of Lagos College of Medicine
Idi-Araba, Nigeria
Evidence-Based Obstetric Management of Women with Sickle Cell Disease in Low Income Countries

Eugene Oteng-Ntim, PhD, MBBS
Guy's and St Thomas' NHS Foundation Trust
London, ENG, United Kingdom
Evidence-Based Management of Pregnant Women with Sickle Cell Disease in High Income Countries

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Prophylactic Platelet Transfusions


Platelet transfusions have shown to decrease the risk of spontaneous bleeding in patients with severe thrombocytopenia, and are a frequent intervention in patients with hematologic malignancies. In 2019, more than 2.3 million platelets were transfused in United States, representing a 0.9% increase from 2017. As the number of platelet transfusion increases, and other factors (such as the pandemic, staff shortages, and others) impact blood collections, it is critical for blood centers to have systems in place to ensure adequate supply to meet the increasing demand. Similarly, hospital-based transfusion services should monitor appropriate clinical use, and have optimal inventory management strategies to minimize outdates. In regards of platelet safety and purity, the FDA recently issued new guidelines on bacterial testing to mitigate the risk of septic reactions; and in regards of potency, new platelet products such as cold stored platelets and frozen platelets are currently being evaluated as a resource for bleeding patients while offering the flexibility of prolonged storage minimizing wastage and reducing shortages.

 

Dr. Zbigniew “Ziggy” M. Szczepiorkowski will review and discuss the new FDA guidelines for bacterial testing of platelet products and its implementation in transfusion services.

 

Dr. John D. Roback will describe several strategies to increase the donor pool and prevent severe and prolonged platelet shortages that could impact patient care.

 

Dr. Dana V. Devine will review the hemostatic capacity of cold stored and frozen platelets based on invitro data and clinical studies.

Chair:

Monica B. Pagano, MD
University of Washington Medical Center
Seattle, WA

Speakers:

Zbigniew Macdonald Szczepiorkowski, MD, PhD
Dartmouth-Hitchcock Med. Ctr.
Lebanon, NH
Evaluate how hospitals are approaching the recent FDA guidance to reduce the risk of bacterial contamination, including pathogen reduction technology and large volume delayed sampling

John D. Roback, MD, PhD
Emory University School of Medicine
Atlanta, GA
Donor Pool

Dana V. Devine, PhD
Canadian Blood Services
Vancouver, BC, Canada
Assess New Alternatives for Platelets, including cold (4o) platelets

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Reproductive and Sexual Health in Sickle Cell Disease


Reproductive and sexual health challenges associated with sickle cell disease (SCD) are common but under-recognized morbidities. Both males and females with SCD suffer from the disease- or therapy-related challenges impacting negatively on their reproductive and sexual health. Priapism, otherwise rare in the general population, is common among adolescent and adult males with SCD with attendant sequelae of erectile and sexual dysfunctions. Treatment with a disease-modifying agent, hydroxyurea, potentially damages the gonads (testes and ovaries) and could result in infertility. Both SCD and hydroxyurea therapy could damage ovaries, resulting in diminished ovarian reserve and infertility. Curative treatments such as stem cell transplants and gene therapy also pose a significant risk of infertility to both males and females. These challenges have made decision-making on fertility choices difficult for the affected individuals.

 

In this educational session, we will discuss the epidemiology and trends in the management of priapism. We will elaborate on fertility challenges affecting individuals, most especially females, with SCD and provide a template of strategies for engaging patients and their families to guide them in making an informed decision about their treatment choices, mainly as they affect fertility.

 

Dr. Ibrahim Idris will review the clinical epidemiology of priapism and treatments of priapism. He will highlight the burden of priapism and associated mental duress among affected individuals. An aspect of his talk will address priapism treatment gaps and the role of chronic morning dosing with phosphodiesterase type 5 (PDE-5) inhibitors in the secondary prevention of priapism. He will highlight a new therapy with the potential to prevent priapism recurrence.

 


Women with sickle cell disease have complex preconception care needs and at least some have infertility risk factors. Using a case-based approach, Dr. Pecker will address preconception care needs, measures of ovarian reserve and the role of assisted reproductive technologies in the contemporary care of adult women with sickle cell disease.

 

Dr. Lillian Meacham will dive into the unusually explored territory of decision-making in a pediatric setting regarding uncomfortable issues like gonadal and sexual health. Her talk will demonstrate how and when to communicate sensitive topics in clinic settings. She will explain the type of topics to be handled by the hematologist in a regular clinic visit and the sensitive issues requiring a referral to fertility specialists, especially when a decision to offer treatments that are potentially damaging to the gonads is on the table. The engaging talk will highlight the teaching tools available to ease this communication between providers and patients.

Chair:

Ibrahim Musa Idris, MBBS,MPH,FMCPath
Aminu Kano Teaching Hospital
Kano, Nigeria

Speakers:

Ibrahim Musa Idris, MBBS,MPH,FMCPath
Aminu Kano Teaching Hospital/Bayero University Kano
Kano, Nigeria
Epidemiology and Treatment of Priapism in Sickle Cell Disease

Lydia H. Pecker, MD, MHS
Johns Hopkins University
Baltimore, MD
Fertility of Women with Sickle Cell Disease

Lillian R Meacham, MD
Emory University School of Medicine
Atlanta, GA
Decision Making for Reproductive Health in Sickle Cell Disease

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Thrombosis and Anticoagulation: Clinical Considerations in Selected Populations


Chair:

Alfred I Lee, MD,PhD
Yale University
New Haven, CT

Speakers:

Bethany T. Samuelson Bannow, MD
Oregon Health %26 Science University
Portland, OR
Anticoagulant Therapy for Women

Pantep Angchaisuksiri, MD
Ramathibodi Hospital, Mahidol University
Bangkok, Thailand
Thrombosis and Anticoagulation: Clinical Issues of Special Importance to Hematologists Who Practice in Asia

Alfred I Lee, MD,PhD
Yale University
New Haven, CT
Thrombosis Questions from the Inpatient Wards

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Thrombosis Prevention and Treatment


Venous thromboembolism (VTE) is a leading cause of cardiovascular diseases and related mortality. The optimal prevention and treatment strategies for VTE are essential. The development and generalization of direct oral anticoagulants have significantly advanced the field in recent years. Novel anticoagulants that could potentially provide improved safety are on the horizon. This educational session will first address common questions regarding frequently used oral factor Xa inhibitors by reviewing current literature and providing evidence-based suggestions. Next, controversies and recommendations of optimal VTE prevention strategies for hospitalized medical patients including those with COVID-19 will be discussed. Lastly, the session will explore the upcoming new anticoagulants and strategies for prevention and treatment of VTE, including factor XI inhibitors.

Dr. Tzu-Fei Wang will address five commonly asked questions regarding the use of oral factor Xa inhibitors for the treatment of VTE, including obesity, renal impairment, gastrointestinal malignancy, catheter-related thrombosis, and drug-drug interactions. For each topic, the scope of the problem and pertinent literature will be reviewed. Available guidelines and practice approaches will be discussed.

Dr. Alex Spyropoulos will discuss four controversial topics in thromboprophylaxis of hospitalized acutely-ill medical patients: 1) clinical relevance of key efficacy and safety outcomes incorporated into randomized trials but not incorporated into antithrombotic guidelines on the topic, 2) establishment of individual risk factors or risk models of low bleed risk and high thrombotic risk subgroups of medically ill inpatients that benefit  from extended thromboprophylaxis, 3) the use of mechanical thromboprophylaxis in this population, and 4) thromboprophylaxis of hospitalized COVID-19 patients, a high thrombotic risk subset of medically-ill patients. The clinical and populational health effects of these issues will be discussed, as well implications for future antithrombotic guidelines on these topics.

Dr. Walter Ageno’s talk will initially focus on the unmet clinical needs in the prevention and treatment of venous and arterial thromboembolic disorders. In particular, the difficult management of patients at high risk of bleeding will be briefly discussed. This will provide the background for the development of new classes of anticoagulant drugs, aimed at maximizing antithrombotic efficacy while minimizing the risk of bleeding. Molecules targeting Factor XI and Factor XII currently under development will be presented and the results of the first published studies will be commented. Finally, potential implications for clinical practice and additional areas of research for these new drugs will be discussed.

Chair:

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

Speakers:

Tzu-Fei Wang, MD,MPH
The Ottawa Hospital, University of Ottawa
Ottawa, ON, Canada
The 5 Most-Frequently-Asked Questions About Fxa Inhibitors (examples: weight, HIT, renal impairment, catheter-associated thrombosis, GI cancers)

Alex C. Spyropoulos, MD
Northwell
New York, NY
Controversies in VTE Prevention for Medical Inpatients (TID vs. BID heparin, extremes of body weight, post-discharge AC, marginal benefit of pharmacologic prophy over SCDs alone)

Walter Ageno, MD
University
Varese, Italy
Coming Soon to a Pharmacy Near You? FXIa Inhibitors and Other Novel Strategies to Prevent or Treat Venous Thromboembolism

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Update in Hemophilia


Chair:

Rebecca Kruse-Jarres, MD, MPH
University of Washington School of Medicine
Seattle, WA

Speakers:

Marilyn J Manco-Johnson, MD
University of Colorado
Aurora, CO
Long-Term Prophylaxis - What Are Our Options and How to Define Success?

Rebecca Kruse-Jarres, MD, MPH
WACBD
Seattle, WA
Peri-Operative Hemostasis

Amit C. Nathwani, MD,PhD
University College London
London, ENG, United Kingdom
Gene Therapy

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Von Willebrand Disease


Von Willebrand disease (VWD) is the most common inherited bleeding disorder. It is caused by deficiency or dysfunction of von Willebrand factor (VWF) a multimeric glycoprotein that plays critical roles in the circulation and is characterized by excessive mucocutaneous bleeding. There have been significant recent advances in our understanding of the biology of VWD and also in the attention being paid to the clinical impact of the disease.   This session will highlight ongoing diagnostic challenges and special management situations. It will also present the patient perspective with a view towards future needs and opportunities to improve care. 

During this session, Dr. Lavin will discuss challenges in the diagnosis of VWD, focusing on the most common subtype, type 1 VWD with levels in the 30-50 IU/dL range. She will suggest approaches for a frequent clinical dilemma, those presenting with bleeding symptoms and borderline plasma VWF levels. Age-related increases in plasma VWF levels complicates assessment of type 1 VWD. The resulting impact on diagnosis will be explored in the context of both adolescence and advanced age.



Dr. James will review the clinical scope of gastrointestinal bleeding in von Willebrand disease (VWD) patients, including current challenges in diagnosis and treatment.  The biology of angiodysplasia and predisposition in patients with abnormal von Willebrand factor (VWF) will be discussed as well as therapeutic options for both acute management and longer term treatment of the vascular abnormalities.



Dr. Sholzberg will discuss what we have learned about the patient's experience with VWD, access and barriers to care. She will describe how structural sexism, stigmatization of vaginal bleeding, delayed diagnosis, and lack of timely access to care result in an increased frequency of bleeding events, iron deficiency, anemia, and  decreased quality of life in patients with VWD. She will describe the positive shift in guidelines, and research prioritization that should promote health equity, and advancement of compassionate and patient centered care.

Chair:

Paula D. James, MD,FRCPC
Queen's University
Kingston, ON, Canada

Speakers:

Michelle Lavin
Haemostasis Research Lab
Dublin, Ireland
Diagnostic Pitfalls and Conundrums

Paula D. James, MD,FRCPC
Queen's University
Kingston, ON, Canada
Special Considerations in GI Bleeding

Michelle Sholzberg, MD
[email protected]
Toronto, ON, Canada
What Have We Learned About the Patient's Experience with von Willebrand Disease?

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What is New in Classical Bone Marrow Failure Syndromes? (focus on management)


Inherited bone marrow failure syndromes are a complex set of disorders characterized by single or multilineage cytopenias and elevated risk of hematopoietic and other malignancies. Patients may present with classic phenotypes, such dysmorphology in Fanconi anemia, but the increased use of germline genetic testing and other diagnostics have led to a growing appreciation of a wide spectrum of IBMFS clinical phenotypes and ages at onset ranging from children to adults. This educational session will explore the clinical manifestations, genetic etiologies, and new avenues for the management of severe congenital neutropenia, Fanconi anemia, and telomere biology disorders.

Dr. Jean Donadieu will discuss therapeutic options for severe congenital neutropenias (SCNs) beyond granulocyte colony stimulating factor (GCSF) and hematopoietic cell transplantation. This talk will describe the rational for the use of inhibitor of sodium glucose cotransporter (ISGTL2), an anti-diabetic drug, in glycogen-storage disease type-IB and glucose-6-phosphatase catalytic subunit-3 (G6PC3) neutropenias and a potential role of C-X-C chemokine-receptor-4 inhibitors in warts, hypoglobulinemia, infections and myelokathexis (WHIM) syndrome. It will also discuss the concept of stimulating somatic genetic rescue, a physiological process that might limit the risk of leukemic transformation, like EIF6 inhibitors in Shwachman Diamond Syndrome.

 

 

Dr. Carlo Dufour will focus on modern management of Fanconi Anemia and its clinical complications starting with presenting a case report that outlines the importance of a long-term specific monitoring plan in this setting. The talk will outline monitoring strategies tailored to timely detect hematological complications in order to perform hematopoietic cell transplantation (HCT) in optimal conditions. The most recently adopted HCT platforms will be discussed. This talk will also address surveillance approaches to identify early cancers, especially epithelial cancers of head and neck and urogenital regions, that currently have no satisfactory treatment.

 

 

Dr. Sharon Savage will discuss the numerous genetic discoveries and the advent of clinical telomere length testing that have led to the recognition of a spectrum of telomere biology disorders (TBDs), beyond classic dyskeratosis congenita (DC). While hematopoietic cell transplantation and androgen therapy are effective for bone marrow failure in TBDs, there is a paucity of options for the other manifestations, such as pulmonary fibrosis, liver disease or cancer. This talk will highlight areas in need of additional clinical and basic science research while providing the background for clinical diagnosis and management.

Chair:

Sharon A. Savage, MD
National Cancer Institute
Rockville, MD

Speakers:

Jean Donadieu
Hopital Trousseau
Paris, Cedex, France
Severe Congenital Neutropenia

Carlo Dufour
G.Gaslini Chidlren's Hospital
Genova, Italy
Fanconi Anemia

Sharon A. Savage, MD
National Cancer Institute
Rockville, MD
Dyskeratosis Congenita & Telomere Biology Disorders

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