Education Program

Sessions on Classical Hematology

Arterial Disease and the Hematologist

Arterial Disease and the Hematologist

Chair:
Jori May, MD, University of Alabama at Birmingham, Birmingham, AL

Speakers:
William Powers, MD, FACP, FAHA, Duke University, Durham, NC
Diagnosis and management of cryptogenic stroke 

Amy Pollack, MD, Mayo Clinic, Jacksonville, FL
Updates in antithrombotic therapy in coronary and peripheral artery disease 

Freda Passam, MD, PhD, University of Sydney, Camperdown, Australia
Managing patients with a history of arterial disease and new venous thromboembolism

Although Hematologists primarily care for patients with venous disease, we are increasingly called upon to assist in the care of patients complex arterial disease and/or a combination of venous and arterial thromboembolism.  This can be challenging, given the evidence for the diagnosis and management of common sites of arterial disease rapidly evolves, but also in patients without atherosclerotic or embolic risk factors, there is little data to guide management.  To address this need, this session will explore key clinical challenges and recent advances in the diagnosis and treatment of cryptogenic stroke, coronary and peripheral artery disease, and discuss complex cases involving both arterial and venous thromboembolic events.

Dr. William Powers will examine the concept of cryptogenic stroke and the narrower category of embolic stroke of undetermined source (ESUS), emphasizing their roles as diagnoses of exclusion. He will explore the clinical and demographic characteristics that define these entities and present findings from randomized controlled trials that inform current management strategies.

 Dr. Amy Pollak will provide a comprehensive update on antithrombotic therapy for coronary and peripheral artery disease. Her presentation will cover emerging evidence and updated guidelines in the management of antithrombotic therapy in coronary artery disease (CAD) and peripheral artery disease (PAD), with an emphasis on individualized patient care.  This case-based session will equip clinicians with actionable knowledge to improve patient outcomes while navigating antithrombotic treatment decisions in CAD and PAD.

Dr. Freda Passam will address the increasingly common challenge of managing patients with pre-existing arterial disease who present with new venous thromboembolism. Through a series of illustrative case scenarios, she will identify clinical dilemmas and examine the latest evidence to inform antithrombotic therapy and long-term risk reduction strategies. Her talk will also provide a practical framework to support therapeutic decision-making in complex vascular patients with overlapping thrombotic conditions.

Chair:

Jori May
University of Alabama at Birmingham
Birmingham, AL, United States

Speakers:

William Powers
Duke University
Durham, NC, United States
Cryptogenic Stroke: Definitions and Management

Freda Passam
University of Sydney
Sydney, NSW, Australia
Managing Patients with a History of Arterial Disease and New Venous Thromboembolism

Amy Pollak
Mayo Clinic
Jacksonville, FL, United States
Updates in Antithrombotic Therapy in Coronary and Peripheral Artery Disease

Between Clots and Complications: A New Era of Anticoagulation in Cancer

Chair:

Marc Carrier
The Ottawa Hospital
Ottawa, ON, Canada

Speakers:

Damon Houghton
Mayo Clinic Rochester
Rochester, MN, United States
Anticoagulants in Hematologic Malignancies: What is the Data?

Marc Carrier
The Ottawa Hospital
Ottawa, ON, Canada
Implementation Failure: Thromboprophylaxis in Ambulatory Patient with Cancer

Michael Jaglal
Moffitt Cancer Center
Tampa, FL, United States
No Shots Needed: Direct Oral Anticoagulants in Cancer Associated Thrombosis- 10 Year Follow Up

Biology Based Management of Iron Toxicity and Overload in Hematology Practice

Regardless of the underlying diagnosis, organ damage from iron results from the magnitude and duration of exposure to reactive ferrous iron (Fe²?). Successful treatment depends on reducing this ferrous (Fe²?) iron all the time. This can be achieved rapidly but requires continuous exposure to an effective chelator, making adherence to therapy a pivotal strategy.  In contrast, removal of stored non-reactive ferric (Fe³?) iron, which—unlike ferrous iron—can be detected by magnetic resonance imaging, takes much longer.

Iron in hemoglobinopathies:  Our understanding of clinical iron biology is rooted in decades of observation in patients with transfusion-dependent hemoglobinopathies. The principles derived from this experience have informed the modern management of iron toxicity and will be the focus of the first talk in this session.

Iron in hematopoietic stem cell transplant: Management of iron toxicity in the context of hematopoietic stem cell transplantation involves unique challenges related to pre-transplant exposure to ferrous iron,  marked exacerbation of ferrous (Fe²?) iron during the transplant process, and residual iron exposure post-transplant. These transplant-specific considerations will be the focus of the second talk of this session.

Iron in myelodysplastic syndrome:  Iron dysregulation can precede the onset of cytopenia in MDS, leading to chronic exposure to toxic iron that may contribute to marrow failure and malignant transformation. Key clinical challenges include determining who should be treated, when to initiate therapy, and how long to continue it, particularly in the context of evolving risk. The role of toxic iron species in the pathophysiology of MDS, and strategies for their management, will be discussed in the third talk of this session.

In the end, exposure to toxic iron depends on whether the iron can be used as fast as it enters the body, whether by absorption or transfusion.  The mechanism of organ toxicity is the same, but the balance of iron entry and utilization depends on the individual iron loading disorder.  

Chair:

Thomas Coates
Children's Hospital of LA
Los Angeles, CA, United States

Speakers:

Heather Leitch
St. Paul's Hospital University of British Columbia
Vancouver, BC, Canada
Management of Iron Overload in Adult Myelodysplastic Syndrome

Emanuele Angelucci
Unita Operativa Ematologia Ospedale 'A. Businco'
Genova, Italy
Management of Iron Overload in the Setting of Hematopoietic Stem Cell Transplant

Thomas Coates
Children's Hospital of LA
Los Angeles, CA, United States
From Treatment to Biology and Back: Managing Iron Overload in Transfused Hemoglobinopathies

Bleeding Disorder of Uncertain Cause: What CAN We Be Certain About?

This session is focused on BDUC (Bleeding Disorder of Unknown Cause) including its diagnosis, risk of future bleeding and how to manage surgery and procedures.  

In the first presentation, Dr. James will discuss the diagnosis of BDUC.  Despite careful clinical assessment of patients referred for a possible bleeding disorder (including the use of a validated Bleeding Assessment Tool and appropriate hemostatic laboratory testing) it is well recognized that only a minority of patients receive a clear diagnosis such as von Willebrand disease (VWD) or a platelet function disorder (PFD).   The remainder of those with a clearly abnormal bleeding history but normal hemostatic work up can be classifed as having Bleeding Disorder of Unknown Cause (BDUC).  BDUC is a diagnosis of exclusion and recommendations for the clinical assessment and laboratory work up will be reviewed in this presentation.

In the second presentation, Dr. Ingrid Pabinger will discuss the important clinical issue of predicting future bleeding in BDUC patients.  Patients with bleeding disorder of unknown cause show an increased bleeding phenotype. Still, there is scarce data on the risk of bleeding in patients with BDUC on future bleeding manifestations, specifically during child-birth, surgery and other invasive procedures. The lecture will present available data and experience from clinical research and observations.

In the final presentation, Dr. Callie Berkowitz will provide a general management approach to surgeries and procedures in patients with BDUC, including a review of available hemostatic therapies. Relevant research and knowledge gaps will be highlighted.

Chair:

Paula James
Queen's University
Kingston, ON, Canada

Speakers:

Callie Berkowitz
University of North Carolina at Chapel Hill
Chapel Hill, NC, United States
Buckle Up! Managing Surgery in Patients with Bleeding Disorder of Unknown Cause (BDUC)

Paula James
Queen's University
Kingston, ON, Canada
Diagnosis of Bleeding Disorder of Unknown Cause: How many tests is enough to diagnose BDUC?

Ingrid Pabinger
Hematology & Hemostaseology
Vienna, Austria
Predictors for Future Bleeding in Bleeding Disorder of Unknown Cause

Challenges in Diagnosing and Managing Mucocutaneous Bleeding Disorders

This session will focus on diagnosing and managing mucocutaneous bleeding disorders. First, Dr. Veronica Flood will discuss the challenges inherent in diagnosis of von Willebrand disease (VWD). Particular attention will be paid to the challenges in the pediatric population and the complications inherent in laboratory testing. Subsequently the problem of "undiagnosing" VWD will be discussed, with attention to the importance of bleeding history for each individual patient.

Next Dr. Catherine Hayward will discuss diagnosis of qualitative platelet disorders that are not Glanzmann thrombasthenia or Bernard Soulier Syndrome. This talk will discuss diagnostic testing for platelet defects, including a discussion of the utility of genetic testing. The different ways to assess platelet function will be examined, and the typical workup outlined along with the requirement for inclusion of patient bleeding history. Finally, this presentation will discuss therapeutic options for patients with platelet function defects.

Lastly Dr. Raj Kasthuri will discuss what's new in diagnosis of HHT. This presentation will cover the genetics of HHT and clinical manifestations, including issues surrounding iron deficiency. Other complications of HHT will be discussed as well as typical treatment options. Evidence to support current therapies will be presented, and new and upcoming therapies for HHT will be discussed as well.

Chair:

Veronica Flood
Medical College of Wisconsin
Milwaukee, WI, United States

Speakers:

Veronica Flood
Medical College of Wisconsin
Milwaukee, WI, United States
(Un) Diagnosing von Willebrand Disease

Catherine Hayward
McMaster University
Hamilton, ON, Canada
When It’s Not Glanzmann Thrombasthenia or Bernard Soulier Syndrome: Diagnosing Other Qualitative Platelet Disorders

Raj Kasthuri
Univ. of North Carolina at Chapel Hill Division of Hematology
Chapel Hill, NC, United States
What's New in Hereditary Hemorrhagic Telangiectasia?

Complementopathies: Modern Diagnosis and Management

Therapeutic complement inhibition has been available for nearly two decades. Paroxysmal nocturnal hemoglobinuria (PNH) was the first disease to garner attention, with clear benefits noted by blocking terminal complement. Novel therapeutics have been developed which have different targets, alternate modes of administration, and reduced treatment burden. Atypical hemolytic uremic syndrome (aHUS) or complement-mediated thrombotic microangiopathy (CM-TMA) is another disease of complement dysregulation. Complement inhibition in such patients has shown improved outcomes compared to plasma therapy. Unlike PNH, however, establishing a diagnosis of aHUS/CM-TMA is less straightforward, and cautious drug discontinuation is possible after careful clinical and risk factor assessment. Once any patient is started on complement inhibition, they must be monitored closely for drug response and disease control; however, many such monitoring strategies are in their infancy or not regularly available. Across PNH, aHUS/CM-TMA, and an ever-expanding list of diseases with complement-mediated pathology, we have learned much about the risks and benefits of complement inhibition and, more broadly, new insights into complement’s role in health and disease. This session will address diagnostic, monitoring, and treatment strategies for patients with PNH and aHUS/CM-TMA.    

Dr. Cole will present an approach to the diagnosis and monitoring of patients with aHUS/CM-TMA. A comprehensive initial assessment will be discussed, including the utility of genetic testing, and functional assays of complement activation. Particular emphasis will be placed on critically evaluating the existing evidence regarding complement inhibitor use in cases of secondary TMA.

Dr. Afshar-Kharghan will discuss the long-term management of patients with aHUS/CM-TMA. Use of complement inhibitors has improved outcomes for such patients, but chronic treatment means persistent infection risk and economic burden among other challenges. Drug discontinuation is possible in some patients, and this session will review clinical, genetic, and other considerations to guide such decisions.

Dr. Patriquin will review the current state of complement inhibition and treatment strategies for patients with PNH. Inhibitors targeting different complement molecules and pathways are available for both first- and second-line therapy in many countries. An overall approach to treatment initiation, monitoring, and considerations of special populations will be reviewed.

Chair:

Christopher Patriquin
University Health Network, Toronto
Toronto, ON, Canada

Speakers:

Vahid Afshar-Kharghan
MD anderson
Houston, TX, United States
Long-term Outcomes in Complement-Mediated Thrombotic Microangiopathy/aHUS

Christopher Patriquin
University Health Network, Toronto
Toronto, ON, Canada
The Varieties of Therapeutic Experience: Navigating Treatment Options for Patients with PNH

Michael Cole
Johns Hopkins University
Baltimore, MD, United States
Update in the Diagnosis of Complement-Mediated Thrombotic Microangiopathy/Atypical Hemolytic Uremic Syndrome

Decoding the Enigma: Challenges in Classical Hematology Consultations

Chair:

Tulika Seth
All India Institute of Medical Sciences (AIIMS)
New Delhi, Delhi, India

Speakers:

Thomas Ortel
Duke University Medical Center
Durham, NC, United States
Anticoagulation in Malignancy - The Patient Who Bleeds and Clots Simultaneously

Naseema Gangat
Mayo clinic, Minnesota
Rochester, MN, United States
JAK2 Wild-Type Erythrocytosis: Concept, Differential Diagnosis, Diagnostic Steps and Treatment Approaches

Kathryn Webert Jr
Canadian Blood Services
Ancaster, Ontario, Canada
The Untransfusable Patient

Hemoglobinopathies and Increased Risk of Venous Thrombo-Embolism

The primary clinical manfestations of hemoglobinopathies and thalassemia are anemia as result of ineffective erythropoiesis and/or hemolytic anemia, and additionally ischemia-reperfusion injury and infarction in the case of sickle cell diseaes. Downstream consequences include increased incidence of both venous and arterial thronmbotic events due to perturbations of the coagulation system, platelet activation, and endothelial dysfunction. Though the vast majority of people who are heterozygous for the sickle mutation are asynmptomatic, recent epidemiological and translational studies suggest sickle cell trait is associated with a modestly increased risk of venous thrombosis. In this session, we will present the pathophysiology that underlies the hypercoagulability associated with thalassemia, sickle cell disease, and sickle cell trait. 

Chair:

Ted Wun
University of California, Davis
Sacramento, CA, United States

Speakers:

Ted Wun
University of California, Davis
Sacramento, California, United States
Sickle Cell Disease: Managing Thromboembolism

Maria Domenica Cappellini
Foundation IRCCS Ca' Granda Policlinico Milano - University of Milan
Milano, Italy
Thalassemia and Hypercoagulability

Rakhi Naik Jr
Johns Hopkins University School of Medicine
Baltimore, Maryland, United States
When Sickle Trait Is Not Just Trait: Risk of VTE

Immune Cytopenias: Addressing Challenges and Advancing Treatments

Immune thrombocytopenia (ITP), warm and cold autoimmune hemolytic anemia (AIHA) represent sporadic but serious hematologic disorders for which management paradigms are continuously evolving. Based on our expanding insights into disease pathophysiology, we are witnessing a burgeoning rise in newer therapies for ITP, warm AIHA and cold agglutinin disease (CAD).  This educational session will summarize the current evidence for assessing and managing patients with CAD, wAIHA and refractory ITP.

Dr. Sigbjorn Berentsen will provide a comprehensive summary of CAD, a disorder of clonal B-cells.  CAD is primarily mediated by classical complement activation, leading to hemolytic anemia, fatigue, and cold-induced circulatory symptoms.  Non-complement mechanisms, such as erythrocyte agglutination, also contribute to symptoms. Tailoring therapies based on predominant symptoms is proving to be an important consideration for these patients. Dr. Berentsen will present a management algorithm, including newer therapies and early phase trials.

Dr. Wilma Barcellini will discuss advances in our understanding of pathophysiology and treatment for warm autoimmune hemolytic anemia (wAIHA). While there are no US-FDA approved therapies for wAIHA, several drugs show promise in the management of this rare but potentially life-threatening condition. Novel agents in development for wAIHA, encompassing drugs targeting B-cells, plasma cells, spleen tyrosine kinase, and the neonatal Fc receptor will be highlighted. Using a case-based approach, Dr. Barcellini will suggest best practices in areas with limited data.

Dr. Sandhya Panch will evaluate evolving definitions, pathophysiology of refractory ITP and highlight various conditions which may be misdiagnosed and mistreated as refractory ITP. In patients with ITP secondary to underlying causes such as benign or malignant lymphoproliferative disease, rheumatologic disease or infections, treating the underlying condition often mitigates thrombocytopenia. This talk will address treatment paradigms for primary refractory ITP and secondary ITP, carefully considering both entities prior to sequencing/combining therapies for this difficult to treat subset of patients.

Chair:

Sandhya Panch
National Institutes of Health
Seattle, WA, United States

Speakers:

Sigbjorn Berentsen
Dept of Research and Innovation, Haugesund Hospital
Haugesund, n.a., Norway
Diagnosis and Management of Cold Agglutinin Disease

Wilma IRCCS Ca' Granda Ospedale Maggiore Barcellini
Fondazione IRCCS Ospedale Maggiore Policlinico Mangiagalli e Regina Elena
Milano, Italy
Management of Autoimmune Hemolytic Anemia

Sandhya Panch
University of Washington/Fred Hutch Cancer Center
Seattle, Washington, United States
Refractory ITP: Revisiting Definitions, Diagnostics and Management Paradigms

Molecular Diagnostics for Clinical Classical Hematologists: When to Order, What to Order, What it Means, and When to Refer to Genetics?

Molecular diagnostics have enhanced our understanding of diseases and their pathophysiology, have improved accuracy and timeliness of diagnosis, and not infrequently have offered surprising insights, revising with newfound clarity previous misinterpretations of clinical and laboratory data. This educational session will explore the options of genetic testing available for diseases treated by the classical hematologists, including cytopenias due to clonal hematopoiesis or inherited bone marrow failure syndromes and hereditary red cell disorders. The indications for genetic testing, the current options and limitations in technology and analysis, and the benefits of collaboration with Genetics will be discussed.

Chair:

Theodosia Kalfa
Cincinnati Children's Hospital Medical Center
Cincinnati, OH, United States

Speakers:

Uma Borate
The Ohio State University
Columbus, OH, United States
Molecular Approach to Cytopenia and Bone Marrow Failure

Xiao Peng
The Children's Hospital At Montefiore
Bronx, NY, United States
Molecular Diagnostics 101: How to Use Genetic Tests in Classical Hematology

Theodosia Kalfa
Cincinnati Children's Hospital Medical Center and University of Cincinnati Medical School,
Cincinnati, OH, United States
Molecular Surprises in Evaluations of Red Cell Disorders

Our Patient is Pregnant: Co-managing Obstetric Patients with Complex Hematologic Issues During their Pregnancy and Delivery

Pregnant women comprise a population with exceptionally challenging sets of problems for the consulting hematologist.  This session, held in conjunction with the Foundation for Women and Girls with Blood disorders will focus on three sets of hematologic problems faced by pregnant women. In many cases, there are no evidence-based guidelines for optimal management, but only expert opinion and case series, making management decisions even more challenging.

Dr. Bethany Samuelson Bannow will present the risks and hemostatic changes associated with pregnancy in individuals with and without bleeding disorders.   Essential components of delivery planning in patients with bleeding disorders will be discussed, including mode of delivery and neuraxial anesthesia.

Dr. Donald Arnold will review management of immune thrombocytopenia (ITP) in pregnancy, including management of refractory ITP and what is known about use of thrombopoietin receptor agonists in pregnancy.

Dr. Lydia Pecker will present best practices for management of high-risk pregnancies in patients with sickle cell disease and how to improve proactively strategize to best improve outcomes for both moms and babies.

Chair:

Alice Ma
University of North Carolina at Chapel Hill
Chapel Hill, NC, United States

Speakers:

Bethany Samuelson Bannow
Oregon Health & Science University
Portland, OR, United States
Pregnancy Management for Patients with Bleeding Disorders

Donald Arnold
McMaster University
Hamilton, ON, Canada
A Practical Approach to Immune Thrombocytopenia in Pregnancy

Lydia Pecker
Johns Hopkins University
Baltimore, MD, United States
Proactive Management to Improve Outcomes in High-Risk Sickle Cell Disease Pregnancy

Sickle Cell Therapies - The Who, What, When, and Where

This session will focus on the Optimization of Treatments for individuals with Sickle Cell Disease, by exploring the who, what, when and where.
Raffaella Colombatti will address the “who” question in optimizing patient selection for treatment in Sickle Cell Disease: which patients are best suited for disease-modifying therapies, combination approaches, or curative treatments? Her talk will explore how clinical evidence, patient profiles and preferences, and real-world data can guide these decisions across different ages and geographical settings.

Seethal Jacob will focus on the “when” question: when is the optimal time to initiate disease-modifying or curative therapies in Sickle Cell Disease? She will examine key factors influencing treatment timing, integration of therapies, and how evolving therapeutic options can help address persistent unmet needs- utilizing a prevention framework and a multimodal risk-to-action approach.

Sheinei Alan will explore the “what”—identifying the most appropriate treatment options for adults with Sickle Cell Disease. He will discuss individualized strategies, criteria for treatment selection, timing considerations, and best practices for shared decision-making in adult care

Chair:

Raffaella Colombatti
University of Padova
PADOVA, Italy

Speakers:

Raffaella Colombatti
University of Padova
PADOVA, Italy
Optimizing the "Right" Patient Selection for Treatment for Sickle Cell Disease

Seethal Jacob
Indiana University School of Medicine
Indianapolis, IN, United States
Optimizing the Right Time to Start Sickle Cell Therapies

Sheinei Alan
Inova Medical Center
Fairfax, VA, United States
The Right Patient, the Right Treatment: Tailoring Sickle Cell Disease Care

The Changing Landscape of the Care of Patients with Immune-Mediated and Congenital TTP

Thrombotic thrombocytopenic purpura (TTP), once almost universally fatal, is now a condition with markedly improved survival due to advancements in diagnostics, therapeutic plasma exchange, immunosuppressive therapies, and novel therapies like caplacizumab and recombinant ADAMTS13. However, the care of patients with both immune-mediated and congenital TTP continues to evolve, with new challenges emerging across the disease spectrum. This session will highlight key areas of progress and ongoing gaps in the management of TTP, including novel approaches to acute treatment, long-term surveillance and complication management in immune TTP, and evolving standards for care in congenital TTP.

Dr. Linus Voelker will review contemporary treatment strategies for acute immune-mediated TTP, with a focus on individualizing therapy. He will discuss the integration of caplacizumab, corticosteroids, rituximab, and other emerging agents, including strategies to balance rapid disease control with long-term remission goals. His presentation will highlight the evolving role of caplacizumab as font-line therapy, including with plasma exchange-free approaches in selected patients. 

Dr. Senthil Sukumar will address the increasing recognition of long-term morbidity in immune TTP survivors. His presentation will cover strategies for monitoring and managing disease relapses, cardiovascular risk, and neurocognitive complications, as well as patient-centered approaches to survivorship care. He will also discuss approaches to managing persistent ADAMTS13 activity deficiency to standard immunosuppression, including considerations for second-line therapy and beyond.

Dr. Alice Taylor will outline current updates on treatment strategies in congenital TTP, illustrating the evolution of treatment options in congenital TTP in recent years, from plasma and plasma-derived products to recombinant ADAMTS13. The classical textbook description of congenital TTP describes diagnosis in early childhood, but there is growing recognition of incidence in adulthood with associated morbidity. In an era of improved diagnostics and treatment options, she will clarify the role of prophylaxis and how this is delivered. Beyond this, Dr. Taylor will discuss how we need to examine what clinical outcomes we are targeting beyond prevention of acute TTP relapse.

Chair:

Senthil Sukumar
Baylor College of Medicine
Houston, TX, United States

Speakers:

Linus Voelker
Department II of Internal Medicine and Center for Molecular Medicine Cologne, Faculty of Medicine and University Hospital Cologne, University of Cologne, Cologne, Germany.
Cologne, NRW, Germany
Tailored Treatment of Acute Immune-Mediated Thrombotic Thrombocytopenic Purpura

Senthil Sukumar
Baylor College of Medicine
Houston, TX, United States
The Changing Landscape of the Care of Patients with Immune-Mediated and Congenital TTP

Alice Maria Taylor, MBBS BSc (Hons) MD
Great Ormond Street Hospital
London, ENG, United Kingdom
Modern Management of Congenital Thrombotic Thrombocytopenic Purpura (cTTP)

Update in Hemophilia Across the Lifespan: Novel therapies/Gene Therapy/Issues in Elderly Patients

Driven by remarkable scientific achievements over the last half-century, the life expectancy of individuals with severe hemophilia has reached that of the general population. Tailoring therapy to each patient’s deficiency, age, clinical circumstances, and lifegoals requires treating clinician to be knowledgeable about therapeutic options to support shared decision-making.

This session provides an update in hemophilia across the lifespan covering the broad array of current therapies, their mechanism of action and application in clinical practice.

The first talk provides an overview of gene therapy for hemophilia which has arrived in the clinic with two approved medications, one for hemophilia A and B respectively.  The basics of gene therapy and their application to clinical practice will be addressed. Dr. Guy Young will "deconstruct" gene therapy by explaining the various components and delivery systems, followed by a brief review of approved products, clinical trial results, and a discussion of novel approaches for hemophilia gene therapy under study. Lastly, gene therapy center logistics and patient selection will be presented. In the second talk, Dr. Mark Reding will discuss the evolution of the treatment landscape over the last decade, and how the introduction of several therapies with novel mechanisms of action has created new challenges for clinicians to select individual optimal treatment.  The development of extended half-life factor products, factor VIII mimetics, and rebalancing therapies will be reviewed.  This talk emphasizes the treatment consideration complexity and the need for a nuanced approach to patient/family education to align with the shared decision-making model of care. The third talk provides a discussion on issues related to aging patients with hemophilia.  Medical conditions affecting the general population are observed in aging hemophilia patients; these conditions include but are not limited to obesity, diabetes, cardiovascular disease and malignancy. Monitoring, preventative care, and treatment of these conditions often represent gaps in care for the hemophilia population; primary care providers may be hesitant to provide needed interventions and/or patients may have difficulty identifying a consistent primary care provider.  Specific issues related to hemophilia sequela including liver and bone health, gait instability, risk for falls and fractures must be considered.  The need for adequate support for aging individuals and the potential development of frailty is amplified when continued underlying bleeding disorder treatment is required with other medical conditions.  Through current treatment advances that support decreased risk of hemophilia associated sequela, and increased general health preventative measures, the hemophilia population may realize improved longevity while maintaining “wellness”, while also achieving personal goals and best outcomes.

Chair:

Amy Shapiro
Indiana Hemophilia & Thrombosis Center
Indianapolis, IN, United States

Speakers:

Mark Reding
M. Health Fairview Center for Bleeding and Clotting disorders, Professor, University of Minnesota Medical School
Minneapolis, MN, United States
New Therapies in Hemophilia: Extend the Half-Life, Bypass, or Rebalance?

Guy Young
Children's Hospital Los Angeles
Los Angeles, CA, United States
Deconstructing Gene Therapy in Hemophilia for the Clinician

Suman Sood
Regents of The University of Michigan
Ann Arbor, MI, United States
The Ageing Hemophilia Patient

Sessions on Malignant Hematology

B-cell Malignancies Common and Rare: What's New in Chronic Lymphocytic Leukemia and Rare B-cell Disorders

Over the past decade, the treatment landscape for B-cell non-Hodgkin lymphomas (B-NHLs) has transformed dramatically. The introduction of targeted therapies, including BTK inhibitors, BCL2 inhibitors, and immunotherapies such as CAR T-cell therapy, has led to unprecedented improvements in outcomes. These advances have been particularly impactful in more common B-cell malignancies such as chronic lymphocytic leukemia (CLL), where multiple large-scale, randomized trials have established highly effective therapies. However, the abundance of options has created a new challenge: determining the optimal sequence and combination of agents to maximize long-term benefit while minimizing toxicity and preserving future options.

In contrast, for rare B-cell lymphomas and leukemias, such as hairy cell leukemia, splenic marginal zone lymphoma, and others, fewer prospective studies exist. Nevertheless, progress continues, with translational research, real-world data, and selected clinical trials helping to define best practices in the absence of large randomized datasets. New biologic insights are also driving innovation in these understudied diseases.

This session will provide a comprehensive overview of both common and rare B-cell malignancies. Experts will present the latest clinical and translational research, offer evidence-based treatment recommendations, and highlight emerging strategies in disease management. Attendees will gain insights into how to apply recent data in clinical practice, tailor therapy to individual patient profiles, and approach rare B-cell disorders where evidence may be limited but is rapidly evolving.

Chair:

Mazyar Shadman
Fred Hutchinson Cancer Center
Seattle, WA, United States

Speakers:

Tanya Siddiqi
City of Hope Orange County
Irvine, CA, United States
The Role of MRD Monitoring and Options for CLL Management in Relapsed/Refractory Disease

Andrea Sitlinger
Duke University
Durham, NC, United States
Updates in the Management of Newly Diagnosed CLL

Salman Fazal Jr
Allegheny Health Network Cancer Institute
Pittsburgh, Pennsylvania, United States
What to Know About Rare B-Cell Malignancies in 2025

Down the Rabbit Hole: An Update on Histiocytic Disorders

Chair:

Gaurav Goyal
University of Alabama At Birmingham
Birmingham, AL, United States

Speakers:

Gaurav Goyal
University of Alabama At Birmingham
Birmingham, AL, United States
The Xanthogranuloma Family of Histiocytic Neoplasms in the Molecular Era: Erdheim-Chester Disease and Beyond

Paul La Rosee
Klinik fur Innere Medizin II
Villingen-Schwenningen, Germany
Hemophagocytic Lymphohistiocytosis: Do We Have a Solution for TMI (Too much Inflammation)?

Olive Eckstein
Texas Children's Hospital
Houston, TX, United States
Langerhans Cell Histiocytic and Rosai Dorfman: Combination Therapies, Response Criteria and Biomarkers

Frontline incorporation of immune targeting agents in B-ALL: Triumphs and Challenges

The introduction of immunotherapies in the last decade has fundamentally changed the treatment of B-ALL, first in the setting of relapse and now in the setting of newly diagnosed disease. In this session, the evidence supporting this change will be reviewed. However, this session will also focus on what comes next. Drs. Kristen O’Dwyer and Sumit Gupta will look at the promise and challenges in de-intensifying treatment by removing elements of traditional therapy in adult and pediatric populations respectively. Dr. Ryan Cassaday will then close the session by focusing specifically on the challenge posed by preventing extramedullary relapses in an era of immunotherapy which may or may not have extramedullary activity.

Chair:

Sumit Gupta
Hospital for Sick Children
Toronto, ON, Canada

Speakers:

Kristen O'Dwyer
University of Rochester Medical Center
Rochester, NY, United States
Immune targeting in Ph neg and Ph+ B-ALL: Improving outcomes and minimizing chemotherapy

Sumit Gupta
Hospital for Sick Children
Toronto, ON, Canada
The Challenge of De-Intensifying Chemotherapy for Children and Adolescents With B-ALL in The Immunotherapy Era

Ryan Cassaday
University of Washington and Fred Hutchinson Cancer Center
Seattle, WA, United States
Sanctuary Sites and Extramedullary relapses in the chemo-free world: insights from immunotherapies in B-ALL

Genomics in Pediatric Acute Leukemia Risk Stratification

Outcomes for patients with acute leukemias have improved through the introduction of targeted therapies, immunotherapies and advances in risk-stratification. Risk-stratification involves the integration of clinical and demographic features such as age and presence or absence of central nervous system (CNS) disease, treatment response via monitoring of measurable residual disease (MRD), and sentinel genomic alterations, with the goal of allocating patients to the appropriate intensity of treatment based on risk of relapse.

Recently, genomic technologies have allowed for the integration of prognostic indels (insertions/deletions), single nucleotide variants (SNVs), transcriptional signatures and fusion oncoproteins. Improvements in statistical modeling allow for the integration of cooperating genomic lesions in risk algorithms and for age and MRD to be considered as continuous variables instead of dichotomous. This session will discuss recent advances in risk-stratification in childhood leukemias.

Dr. Mignon Loh will discuss B-cell acute lymphoblastic leukemia (B-ALL) risk-stratification. B-ALL risk-stratification has been robust for decades as many prognostic genomic alterations, including BCR::ABL1 and ETV6::RUNX1 have been identified.  Recently, more complex genomic algorithms that integrate cooperating lesions are being included. Historically, B-ALL risk-stratification was driven by biomarkers that correlate to response/resistance to cytotoxic chemotherapy. However, many patients are now treated with backbones that integrate immunotherapies. The prognostic significance of many genomic alterations likely differs with chemoimmunotherapy regimens and risk-stratification algorithms will need revision as therapy continues to evolve.

Dr. Martina Pigazzi will discuss risk-stratification in acute myelogenous leukemia (AML). AML risk stratification was formerly limited to a small number of genomic alterations, including Monosomy 7 and RUNX1::RUNX1T1. Currently, multiple groups integrate dozens of prognostic genomic alterations into risk-stratification, identifying high-risk patients likely to benefit from stem cell transplant or targeted therapies and low-risk patients who may benefit from strategies to de-intensify treatment to reduce treatment-related mortality.

Dr. David Teachey will discuss risk-stratification in T-cell ALL (T-ALL). T-ALL risk-stratification has been rudimentary as most groups only consider MRD and CNS status. Unfortunately, the majority of patients who relapse are considered favorable/low risk at diagnosis. Recent large-scale genomic profiling efforts have identified alterations that are prognostic independent of MRD. Genomic T-ALL risk-stratification is finally becoming a reality.

Chair:

David Teachey
Children's Hospital of Philadelphia
Philadelphia, PA, United States

Speakers:

Mignon Loh
Seattle Children's Hospital
Seattle, WA, United States
Revisiting Novel Genomic Classifiers in the Era of Immunotherapy for Pediatric B-ALL

David Teachey
Children's Hospital of Philadelphia
Philadelphia, PA, United States
The Genomics of Pediatric T-ALL

Martina Pigazzi
University of Padova
Padova, Padova, Italy
Using Genomics to Refine Pediatric AML Risk Stratification

Invasion of the Immunotherapies: Where We Are and Where We Are Going in Leukemia, Lymphoma and Myeloma

In less than a decade, immunotherapy has revolutionized the treatment approaches and algorithms for hematologic malignancies. Rapid progress using engineered autologous chimeric antigen receptor T-cells, development of a new generation of antibody-drug conjugates, and the advent of bispecific antibodies with improved delivery features have all had major impact. The first generation of trials with these agents were conducted primarily in the relapsed and refractory setting. However, impressive efficacy and growing experience with managing adverse effects support earlier integration of immunotherapy in treatment paradigms of several blood cancers. There are many remaining challenges and opportunities, and this session features three speakers who will highlight the next chapter for immunotherapies in myeloma, non-Hodgkin lymphoma, and acute leukemias.

Chair:

Sonali Smith
University of Chicago
Chicago, IL, United States

Speakers:

Doris Hansen
H. Lee Moffitt Cancer Center & Research Institute
Tampa, FL, United States
CAR-T cell and Bispecific Antibodies in the Management of Multiple Myeloma

Sonali Smith
University of Chicago
Chicago, IL, United States
The Latest and Greatest in Immunotherapy for Non-Hodgkin Lymphomas (Engaging T cells in the fight)

Nicolas Boissel
Hôpital Saint-Louis
Paris Cedex 10, France
Where Do Immunotherapies Stand in Management of Acute Leukemia in Adults?

Monoclonal Gammopathies of Clinical Significance

The field of plasma cell disorders has dramatically changed over the last decade with a much greater understanding of pathophysiology, the development of novel therapies and significant revisions to the classifications of these complex diseases. Building on the foundational phrase Monoclonal Gammopathy of Undetermined Significance, other conditions have now been more fully described.  In this session, three such conditions will be explored with practical advice provided for optimal diagnosis and therapy.

Dr. Angela Dispenzieri will review the latest evidence for the proper diagnosis of POEMS syndrome and the key criteria required to recognize this often missed disease.  She will also discuss the clinical management of patients with POEMS syndrome with an emphasis on the extent of understanding of the disease and gaps of knowledge.  

Dr Arnaud Jaccard will explore the complex landscape of peripheral neuropathies associated with monoclonal gammopathies (MGNS). He will present the clinical, electrophysiological, and immunological hallmarks of key MGNS entities, emphasizing practical clues for diagnosis. Through a case-based approach, he will illustrate how integrating targeted laboratory tests can guide clinical reasoning. He will highlight the pivotal role of collaboration between neurologists and hematologists in tailoring treatment strategies, combining immunotherapy and clone-directed therapies.

Dr. Heather Landau will discuss the complex topic of Monoclonal Gammopathy of Renal Significance.  These disorders have variable presentations and are often missed due to the lack of an overt hematologic clone.  She will outline a diagnostic algorithm to AL Amyloidosis and its updated management.  She will also provide insight into the approach to other MGRS conditions and strategies to confirm the diagnosis and preserve renal function. 

Chair:

Joseph Mikhael
City of Hope
Phoenix, Arizona, United States

Speakers:

Angela Dispenzieri
Mayo Clinic
Rochester, MN, United States
POEMS: Syndrome Diagnosis, Treatments, and Outcomes

Heather Landau
Weill Cornell Medical Center
New york, New York, United States
Monoclonal Gammopathy of Renal Significance from a Hematologic Perspective

Arnaud Jaccard
University Hospital
Limoges, Nouvelle Aquitaine, France
Monoclonal Related Neuropathies: Diagnosis, Prognosis, and Outcomes

Multiple Myeloma: What Is the Best Induction, Consolidation, and Maintenance for FIT vs. Non Fit

Chair:

Noopur Raje
Mass General Hospital Cancer Center
Boston, MA, United States

Speakers:

Cesar Rodriguez
ICANS school of medicine
New York, NY, United States
Transplant in Myeloma: What is its Role?

Jonathan Kaufman
Emory Winship
Atlanta, GA, United States
What is the Best Induction for Myeloma for the FIT patient?

Noopur Raje
Mass General Hospital Cancer Center
Boston, MA, United States
Choosing the Optimal Maintenance Strategy in Multiple Myeloma

Navigating Immunotherapies in Pediatric Leukemia and Lymphoma: CAR-Ts, allo-HSCT - How, When and Why?

Chair:

Shannon Maude
The Children's Hospital of Philadelphia
Philadelphia, PA, United States

Speakers:

Adam Lamble
Seattle Children's Hospital
Seattle, WA, United States
Have CARs Stalled for Non-B Cell Malignancies? Where Are We, and Where Are We Going?

Maria Gabelli
Great Ormond Street Hospital for Children
London, ENG, United Kingdom
Maintenance after CAR-T? Are We There Yet? Reducing the risk of relapse after loss of anti-CD19 CAR T-cell persistence

Shannon Maude
The Children's Hospital of Philadelphia
Philadelphia, PA, United States
With BiTEs at the Kiddie Table, Where do CARs Come in for Pediatric B-ALL?

New Age Hematopoietic Stem Cell Transplantation: Same Donors, New Prophylaxis, Novel Engineering (Adult and Pediatric Perspectives)

This session explores innovative advancements in allogeneic hematopoietic cell transplantation (HCT) from both adult and pediatric perspectives. The focus is on evolving strategies in donor selection, graft engineering, prophylaxis for graft-versus-host disease (GVHD), and conditioning regimens.

Dr. Talano will discuss donor selection and graft engineering. While matched sibling and unrelated donors remain the gold standard, alternative sources such as haploidentical donors, mismatched unrelated donors, and umbilical cord blood result in improved outcomes in the modern era. Donor selection considers not only HLA compatibility but non-HLA factors such as donor age which is increasingly important in improving outcomes of HCT. Graft engineering techniques such as CD34+ selection, CD45RA and αβ T-cell depletion, and cord blood expansion are enhancing transplant outcomes. Additionally, optimizing graft cell dosing is critical for improving engraftment and reducing complications.

Dr. John Koreth presents on modern GVHD prophylaxis regimens. He highlights the effectiveness of PTCy, Abatacept, and Vedolizumab in preventing both acute and chronic GVHD. Therapies, including JAK inhibitors, Sirolimus, Alpha-1 antitrypsin, ATG, and Obinutuzumab maintenance, are also reviewed, offering tailored options based on donor type and patient risk profiles.

Dr. Brenda Sandmaier addresses the evolution of conditioning regimens, which are essential for successful engraftment and disease eradication. Conditioning regimens play an essential role in HCT by facilitating engraftment and eradicating malignancies. The landscape of conditioning regimens has undergone an evolution in the concept of intensity. Novel conditioning strategies aim to provide highly efficacious regimens with improved toxicity profiles. Integrating disease specific chemotherapy and targeted agents into conditioning regimens provides enhanced disease elimination and relapse prevention. Agents like Treosulfan provide safer conditioning with a favorable toxicity profile for individuals who are older, pediatric patients, or those with medical comorbidities. Additionally, methodologies for precise and targeted radiation delivery with minimal off-target effects are emerging. These advancements necessitate re-examination and harmonization of conditioning intensity stratification schemes for a more personalized and selective approach.

Chair:

Julie-An Talano
Medical College of WI
Milwaukee, WI, United States

Speakers:

Julie-An Talano
Medical College of Wisconsin
Milwaukee, WI, United States
Donor Selection, Graft Engineering and Dosing Oh My!

John Koreth
Dana Faber Cancer Institute & Harvard Medical School
Boston, MA, United States
New Age GVHD Prophy Regimens: what works for what donor and why

Brenda Sandmaier
University of Washington School of Medicine
Seattle, Washington, United States
New Age SCT Conditioning Regimens: What Works and Why?

Now is the Time to Improve Outcomes in Diffuse Large B Cell Lymphoma

The treatment of diffuse large B-cell lymphoma (DLBCL) is evolving as multiple new agents have been FDA-approved for this disease in recent years. A nuanced understanding of molecular subtypes using genomic profiling has been described. Monitoring techniques including 18F-fluorodeoxyglucose positron emission tomography (PET) and evaluation of minimal residual disease (MRD) are being explored to aid in treatment decision making. Immunotherapy approaches, including chimeric antigen receptor (CAR) T-cells and bispecific antibodies (BsAbs), are now being investigated in front line and used as standard of care in relapsed/refractory settings. 

Dr. Sarah Rutherford will discuss the historical and current understanding of molecular subtypes of DLBCL, and will describe existing data and future efforts to specifically target these subtypes in the front line and relapsed/refractory settings. She will also examine the role of subtype-agnostic therapies in this context. 


Dr. Jennifer Crombie will review current mechanisms of evaluating early response assessment in DLBCL, including use of PET and MRD. She will discuss the prognostic impact of these tools and clinical trials that incorporate interim evaluation to either escalate or de-escalate therapy. This session will highlight that while personalized treatment that incorporates interim response holds great promise in the future of DLBCL management, further validation is required in clinical practice. 

Dr. Franck Morschhauser will provide an overview of the novel options which have been incorporated into the DLBCL treatment algorithm these past years, primarily focusing on sequencing of CAR T-cells and BsAbs according to most recent knowledge, but also reviewing alternative targeted strategies which are available for this patient population. After this talk, attendees are expected to have the necessary tools to make a reasoned treatment choice in different clinical scenarios but also anticipate the challenges to overcome in the near future while navigating through this highly dynamic T-cell redirecting era.

Chair:

Sarah Rutherford
Weill Cornell Medicine
New York, NY, United States

Speakers:

Jennifer Crombie
Dana Farber Cancer Institute
Boston, MA, United States
Could Treatment Modification Based on Early Response Assessment Improve Results in DLBCL?

Franck Morschhauser
Centre Hospitalier Régional Universitaire De Lille
Nord, France
Selecting the Best Treatment Approach and Optimizing Sequencing Strategies in Large B-cell Lymphoma

Sarah Rutherford
Weill Cornell Medicine
New York, NY, United States
Molecular Subtypes of DLBCL– Are We Ready to Translate Our Knowledge Into the Change of Treatment Paradigms?

Post-SCT Maintenance Strategies in Leukemia: Who, When and Why?

Allogeneic hematopoietic cell transplantation (HCT) is a central component for the curative strategy for many patients with hematologic malignancies.  Disease relapse after HCT still occurs, for some diseases with distressing regularity.  Therefore, post-HCT maintenance therapies have become increasingly common in both adult and pediatric patients.  Knowing which patients might benefit from maintenance therapy, what to use, and when to start such therapy is not well-defined and often controversial.  This session will review the data for post-HCT maintenance for acute leukemia, with a particular focus on measurable residual disease (MRD), and offer practical guidance to clinicians on this topic.

Chair:

Mark Levis, MD PhD
Johns Hopkins University
Baltimore, MD, United States

Speakers:

Mary-Elizabeth Percival
Fred Hutchinson
Seattle, WA, United States
Peri-transplant Conundrums: Optimizing Maintenance Therapy Using MRD-Directed Approaches

Mark Levis, MD PhD
Johns Hopkins University
Baltimore, MD, United States
Post-HCT Maintenance Therapy for AML: Who, When, Why, How Long?

Jurgen Kuball Jr
UMC Utrecht
UMC Utrecht, Utrecht, Netherlands
Post-transplant cells for the win? DLI and adoptive cell therapy to eradicate MRD

Rare aggressive B-cell Lymphomas: Challenge for a Pathologist, Challenge for a Clinician

The recently published 5th edition of the World Health Organization (WHO) classification of haematolymphoid tumors (WHO-HAEM5) recognizes 18 different subtypes of large B-cell lymphoma (LBCL), based on morphology, genetic abnormalities, site of origin, and association with immunodeficiency and viral infection. In this educational session, three rare and distinct subtypes of LBCL will be discussed, which can be challenging in terms of differential diagnosis, are currently still associated with an inferior prognosis, and may benefit from intensified and/or targeted therapy.

Plasmablastic lymphoma (PBL) is a rare, aggressive CD20-negative B-cell lymphoma with a poor prognosis that poses important diagnostic and therapeutic challenges, given intermediate features between diffuse large B-cell lymphoma and multiple myeloma. Dr. Castillo will review the epidemiology, biology, clinical features, diagnosis, and treatment options in patients with PBL. The current data supporting the use of novel agents such as proteasome inhibitors and anti-CD38 monoclonal antibodies will be discussed, as well as ongoing clinical trials that can potentially be practice-changing.

Mediastinal gray zone lymphoma (MGZL) is defined in the WHO classification as a B-cell lymphoma with overlapping clinical, morphological, immunophenotypic, and molecular features of both primary mediastinal B-cell lymphoma (PMBL) and classical Hodgkin lymphoma (CHL). Due to the paucity of dedicated clinical trials, it remains unclear whether novel immune-based therapeutic options available for PMBL and NSCHL will also apply to MGZL. Dr. Sarkozy will review the molecular data that led to the recent updates in classification and the precise diagnostic criteria for MGZL. Using a clinical case, she will then discuss the initial workup and the different treatment options for MGZL, both in the first-line and relapsed settings. This case will highlight challenges in treatment decision-making, including response assessment.

High-grade B-Cell Lymphomas, including double-hit and triple-hit lymphomas (with BCL2 and MYC or BCL2, BCL6, and MYC translocations), typically have an aggressive disease course and do worse when treated with standard immunochemotherapy. In this presentation, Dr. Davies will apply concepts from both routine molecular diagnostics and the framework of deeper biological insight from genomic studies to understand the modern classification of high-grade B-cell lymphomas. Controversies in the  clinical phenotype and optimal therapeutic approach, both at presentation and relapse,  of high-grade B-cell lymphomas will be explored. 

Chair:

Marie Jose Kersten
Academic Medical Center
Amsterdam, Netherlands

Speakers:

Clementine Sarkozy
Curie
Saint Cloud, France
How to Diagnose and Treat Mediastinal Gray Zone Lymphoma in the Era of Targeted Agents?

Andrew Davies
University of Southampton
Southampton, Hampshire, United Kingdom
High-grade B-Cell Lymphomas: High Difficulties to Diagnose and Treat?

Jorge J Castillo, MD
Dana-Farber Cancer Institute
Boston, MA, United States
When Immature Plasma Cells Form Lymphoma: How to Improve on Diagnostics and Treatment of Plasmablastic Lymphoma?

Targeted Therapy for AML (Triplets and More)

Over the last decade, the understanding of molecular and other drivers of AML has led to the development of novel agents that are utilized in relapsed/refractory and newly diagnosed AML. While the wide adoption of hypomethylating agents with venetoclax is a major advance for the treatment of adults with newly diagnosed AML for whom intensive therapy is inappropriate, significant improvements are still needed. This session will focus on three areas with major promise: the addition of approved targeted agents (FLT3 and IDH1 inhibitors) to standard backbones; the emerging data with the newest class of targeted agents, menin inhibitors; and mechanisms to improve outcomes for adults with AML without a targetable mutation.

Dr. Jessica Altman will outline the evidence for the use of targeted triplet therapies for the treatment of newly diagnosed AML. Regimens combining hypomethylating agents, venetoclax, and targeted inhibitors of FLT3 and IDH are emerging as a promising therapies for patients with AML unfit for intensive chemotherapy. However, there are many remaining questions including applicability and optimal dosing. Dr. Altman will review the therapeutic options, toxicities, knowledge gaps, and ongoing clinical trials to address areas of unmet need.

Dr. Eunice Wang will describe the clinical data supporting the advent of menin inhibitors as a novel class of targeted therapy for KMT2Ar and NPM1 mutant acute leukemias. She will discuss the efficacy, key toxicities, and resistance mechanisms associated with currently approved and investigational agents. Dr. Wang will also highlight the potential of these agents to transform standard of care therapy in upfront triplet regimens in upcoming clinical trials.

Dr Andrew Wei will address the topic of genomically agnostic triplet combinations. In discussing this subject, Dr Wei will review what is meant by “standard” azacitidine + venetoclax (AZA-VEN) and the evidence supporting/challenging truncated dosing schedules. Clinical limitations associated with AZA-VEN outcomes in older AML populations will be addressed and the scientific rationale for developing more effective triplet combinations. Dr Wei will also consider challenges and options for demonstrating enhanced efficacy with new triplet combinations and discuss genomically agnostic triplet strategies currently in early stages of development that have future promise.

Chair:

Jessica Altman
Northwestern University
Chicago, IL, United States

Speakers:

Jessica Altman
Roswell Park Comprehensive Cancer Center
Buffalo, New York, United States
Targeted Triplet Therapies incorporating FLT3 or IDH Inhibitors: Ready for Prime Time?

Eunice Wang
Roswell Park Comprehensive Cancer Center
BUFFALO, NY, United States
The Promise of Menin Inhibitors: From Approval to Triplet Regimens

Andrew Wei
The Walter and Eliza Hall Institute of Medical Research
1g Royal Parade, Parkville , VIC, Australia
Triplet Regimens for All: Genomically Agnostic Approaches to Improve on HMA + VEN in AML?

Treatment Refinement in Multiple Myeloma

The myeloma treatment armamentarium has undergone an unprecedented evolution, with 19 new drugs or combinations approved over the past two decades. This progress has been associated with a significant improvement in patient survival. However, it also presents new challenges, including optimal treatment sequencing, the debate between fixed-duration versus continuous therapy, and the management of high-risk patients. 

In this educational sesión Dr. Suzanne Lentzsch will review evidence-based treatment sequencing in multiple myeloma. She will discuss the integration of anti-CD38 monoclonal antibody-based quadruplet regimens for transplant-eligible and -ineligible patients, and the emergence of CAR T-cell therapy as a preferred option in early relapse. Given the growing use of bispecific antibodies and other novel agents in later lines, Dr. Lentzsch will explore the complexities of optimizing sequencing strategies and the rationale for prioritizing certain modalities,.

Dr. Ben Derman will discuss the evolving role of maintenance therapy in multiple myeloma, with a focus on emerging data supporting treatment cessation guided by measurable residual disease (MRD).? While indefinite maintenance with lenalidomide has remained a standard of care, can treatment safely be stopped in patients with myeloma, especially those with MRD negativity? Dr. Derman will  highlight the patient-centered benefits of treatment discontinuation, including improved quality of life, reduced toxicity, and financial relief, while acknowledging ongoing challenges surrounding cessation, and finally, he will outline a practical framework for MRD-guided decision-making.

Dr Weisel will speak on how to define and characterize high-risk multiple myeloma. The main focus of her talk will be on the optimal strategy of treating patients with high-risk myeloma showing the data on the current first-line treatment approaches base on the most recent trials. The talk will be complemented by reviewing data on treatment of functional high-risk disease characterized by early relapses despite optimal first-line treatment.

Chair:

Jesus San-Miguel
Cancer Center Clínica Universidad de Navarra
Pamplona, Navarra, Spain

Speakers:

Suzanne Lentzsch
Columbia University
New York, NY, United States
“Endless” Possibilities and How to Exploit Them? What Is the Optimal Treatment Sequence?

Benjamin Derman, MD
University of Chicago
CHICAGO, IL, United States
Can I Stop My Treatment, Doctor? Is MRD-Guided Therapy Ready for Prime Time?

Katja Weisel
University Medical Center Hamburg-Eppendorf
Hamburg, Hamburg, Germany
How to Approach the High-Risk Myeloma from Induction through Relapse?

What's New and Current in the World of Bone Marrow Failure

The field of bone marrow failure syndrome and myelodysplastic syndrome (MDS) diagnosis and treatment is changing rapidly. With evolving definitions and increased detection through next-generation sequencing, distinguishing between ICUS, CCUS, and CHIP requires careful integration of clinical, morphologic, and molecular data. Recent updates in the management of low and intermediate-risk MDS focus on improving outcomes through refined risk stratification and novel therapeutic approaches. At the same time, with the recent development of reduced-toxicity conditioning regimens and the use of post-transplant cyclophosphamide as prophylaxis for graft-versus-host disease alternative donors are now increasingly being used for hematopoietic cell transplantation (HCT) for patients with severe aplastic anemia (SAA).

Dr. Zhuoer Xie will tackle the growing clinical challenges of identifying and managing clonal and at-risk hematologic conditions. This talk will review updated diagnostic frameworks, clarify key distinctions, and provide practical guidance on evaluating somatic mutations in the absence of overt malignancy. She will use real-world cases to illustrate common pitfalls and decision points, including when to pursue bone marrow biopsy and how to monitor for progression.

Dr. Hetty Carraway will discuss how the integration of genomic information has enhanced the accuracy of diagnosis and prognosis of MDS using refined scoring and classification systems. She will further outline how treatment strategies are increasingly being personalized, balancing transfusion dependence, cytopenias and quality of life.

Dr. Akshay Sharma will discuss the changes in the field of HCT for SAA, and how the use of novel HCT regimens has improved the survival after HCT over the last few decades and. He will discuss how these recent advances in the field of HCT for SAA are changing the paradigm of various therapeutic strategies, including timing of HCT with respect to immunosuppressive therapy. We will apply data from the recent clinical trials and emerging concepts to the treatment of patients with SAA to evaluate what the future might look like.

Chair:

Akshay Sharma Jr
St. Jude Children's Research Hospital
Memphis, Tennessee, United States

Speakers:

Zhuoer Xie
Moffitt Cancer Center
Tampa, FL, United States
Demystifying the Diagnosis and Management of ICUS, CHIP, and CCUS

Akshay Sharma Jr
St. Jude Children's Research Hospital
Memphis, Tennessee, United States
The Latest on Aplastic Anemia Management

Hetty Carraway
Cleveland Clinic
Cleveland, OH, United States
Updates on Low/Intermediate-Risk MDS