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Education Program

Junior Faculty Career Development Education Session


Chair:

Leslie Renee Ellis, MD, MSHPEd
Wake Forest University School of Medicine
Winston Salem, NC

Speakers:

Marvin T. Nieman, PhD
Case Western Reserve University
Cleveland, OH
Leading Yourself

Alison W. Loren, MD, MSCE
University of Pennsylvania
Philadelphia, PA
Leading Others

Linda J. Burns, MD
Center for International Blood and Marrow Transplant Research
Milwaukee, WI
Leading Organizations

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Sessions on Malignant Hematology

A Map for the Changing Landscape of CLL


Hematologists treating a patient with chronic lymphocytic leukemia (CLL) face the difficult task of choosing the most appropriate therapeutic regimen in a rapidly evolving field. There has been significant progress in the treatment of patients with CLL with improved knowledge of disease biology leading to the introduction of targeted agents and immunotherapies. While chemo-immunotherapy (CIT) regimens such as fludarabine-cyclophosphamide-rituximab (FCR) remain a reasonable treatment option for the frontline management of a young, fit patient with mutated IGHV without a 17p deletion or TP53 mutation, its use is decreasing due to the recent approval of several novel targeted drugs. The Bruton’s tyrosine kinase inhibitors (BTKi: ibrutinib and acalabrutinib) along with the BCL-2 inhibitor venetoclax are currently available for use in the frontline setting with or without the addition of a monoclonal antibody. These novel agents are becoming the preferred therapeutic interventions due to their remarkable efficacy, remission duration, and safety profile. With a growing armamentarium of available drugs, proper selection of the optimal treatment strategy is crucial to achieve long-term remissions.

 Dr. Carol Moreno will outline the therapeutics available to treat patients with relapsed/refractory CLL after initial use of CIT and review the challenges of choosing the right regimen. She will highlight some of the key recent findings in relapsed CLL management strategies and discuss important selection factors in particular settings (high risk prognostic markers, comorbidities, fixed duration vs continuous duration).

 Dr. Jacqueline Barrientos will discuss the challenge of selecting initial frontline therapy in a patient with CLL including optimized patient selection and toxicity mitigation. Using a case-based approach, Dr. Barrientos will suggest best practices in challenging cases due to comorbidities, side effects, and patient preference.

 Dr. Anthony Mato will discuss and compare treatment strategies for patients requiring therapy after relapse following initial therapy with novel targeted agents with a review on current sequencing data  post initial frontline therapy with novel agents. He will discuss different treatment approaches for a patient after the development of BTKi intolerance vs. true progression. Treatment recommendations based on clinical evidence and expert opinion will be reviewed for patients following discontinuation of venetoclax who progress while on therapy or relapse after fixed-duration regimen. He will also describe areas for future research.

Chair:

Jacqueline C Barrientos, MD, MS
Zucker School of Medicine at Hofstra/Northwell
Lake Success, NY

Speakers:

Carol Moreno, MD, PhD
Hospital de la Santa Creu i Sant Pau, Autonomous University of Barcelona
Barcelona, Spain
Standard Approaches to Relapsed CLL after Frontline Chemoimmunotherapy

Jacqueline C Barrientos, MD, MS
Department of Medicine, Hofstra North Shore - LIJ School of Medicine
Great Neck, NY
Chemotherapy-Free Frontline Therapy for CLL: Is It Worth It?

Anthony R. Mato, MD
Memorial Sloan Kettering Cancer Center
New York, NY
Approaches for Relapsed CLL After Chemotherapy-Free Frontline Regimens

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Acute Myeloid Leukemia - So Many Treatment Options; How Do You Decide?


Until recently, treatment options for patients with newly diagnosed and relapsed/refractory acute myeloid leukemia (AML) were limited to cytotoxic chemotherapeutic agents that possessed little specificity for the cytogenetic and molecular mutations known to risk stratify patients with this disease. Furthermore, treatment was often started very soon after the diagnosis was made with limited consideration of these disease specific characteristics. The approval of the multi-targeted kinase inhibitor midostaurin for patients with FLT3 mutated AML in 2017 heralded the approval of eight additional agents and quickly ushered in a new age for the therapeutic management of this disease. Outside of emergent presentations, most patients with a new diagnosis of AML now undergo a complete molecular and cytogenetic evaluation that in many cases decides the treatment plan. As a result, not only have the agents that we treat patients with changed, the way that we talk about these options with patients, decide on, and manage therapy has been transformed.

Dr. Jacqueline S. Garcia will discuss one of the most debatable questions in the field, how does one determine whether a patient is “fit” for a therapy? Underlying fitness has been a central factor in determining initial treatment choice for patients diagnosed with AML. However, relatively effective non-intensive therapies are now available for patients with co-morbidities or those who are older than 75 years, which may change our approach. Genetic and functional determinants of response have become increasingly important in treatment decision making irrespective of chronological age. Dr. Garcia will provide insights and recommendations on how to approach fitness and treatment choice in patients newly diagnosed with AML.

Dr. Alison R. Walker will discuss recommendations for best practices to help guide the increasingly complex treatment discussions that are needed for patients at each stage of their disease. Once a complete remission has been achieved, the road to maximizing disease free and overall survival for AML patients may take different paths. How can we help patients make an informed decision, weighing the risk of relapse with patient wishes and desired quality of life? Dr. Walker will be discussing approaches to shared decision making in determining consolidation, maintenance, and transplantation for patients with AML.

Dr. Eunice S. Wang will discuss recommendations for the ongoing management of patients who are receiving these new treatments. While the advent of targeted therapies for AML has led to new hope for our patients, it has also introduced new challenges in management. Which patients benefit most from a specific therapy and what side effects should we watch for? When should you consider discontinuation of therapy? Are there patients who should move on to allogeneic stem cell transplantation rather than continue with the targeted agent? Dr. Wang will discuss the unique toxicities associated with mutation specific and non-specific agents and the risk/benefit ratio in decision making for individual patients.

Chair:

Alison R. Walker, MD
The Ohio State University Medical Center
Columbus, OH

Speakers:

Jacqueline S. Garcia, MD
DFCI
Boston, MA
Does Patient Fitness Play a Role in Determining First-Line Treatment for AML

Alison R. Walker, MD
The Ohio State University Medical Center
Columbus, OH
How to Approach Shared Decision Making in Determining Maintenance, Consolidation Therapy and Transplant

Eunice S. Wang, MD
Roswell Park Comprehensive Cancer Center
Buffalo, NY
Management of Toxicities Associated with Targeted Therapies: When to Push Through and When to Stop

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Aggressive Lymphomas: What Novel Approaches Are Ready for Prime Time?


As our understanding of the molecular underpinnings of aggressive lymphomas has grown, the identification of novel therapies targeting biological drivers of disease have also been recognized. It remains to be understood however, how these novel therapies will fit into practice. Should our efforts be focused on specific drivers for individual cell of origin subtypes, reversing chemoresistance, or harnessing antitumor immunity? Despite the fast pace nature of the scientific advances in this field, we are still waiting for breakthrough treatment strategies for aggressive lymphoma.

This education session will explore the emerging evidence of epigenetic drivers of disease and how epigenetics relates to lymphomas that become refractory to chemotherapy. In addition, this session will dissect how epigenetic therapies can be utilized to overcome chemoresistance and enhance immunotherapy. Finally, cutting-edge novel therapeutics and strategies for targeting aggressive lymphomas will be discussed in detail.

Dr. Michael Green will discuss recent advances in our understanding of the role of epigenetics in the development of lymphoma. Focusing on follicular and diffuse large B-cell lymphomas, he will discuss the cellular and molecular mechanisms by which somatic mutations alter the lymphoma epigenome and drive lymphomagenesis. The discussion will be centered on how this knowledge can be leveraged to design rational therapeutic strategies for patients with lymphoma.

Dr. Jennifer Amengual will discuss principles of chemotherapy resistance and insights into how epigenetics has been implicated in the emergence of resistant clones. She will discuss how epigenetics relates to immune evasion in aggressive lymphomas. Finally, Dr. Amengual will review how epigenetic modulation can resensitize lymphoma to chemotherapy and novel agents as well as enhance antitumor immunity to potentiate immunotherapy.

Dr. Kami Maddocks will discuss novel approaches in the treatment of aggressive lymphomas. She will discuss potential targets for therapy in aggressive non-Hodgkin lymphoma including various immunological, signaling and mutation targets. Focusing on aggressive B-cell lymphomas, she will review emerging therapies in development for the treatment of relapsed/refractory NHL including results from the most promising clinical trials.

Chair:

Jennifer E. Amengual, MD
Columbia University
Scarsdale, NY

Speakers:

Michael R Green, PhD
UT M. D. Anderson Cancer Center
Houston, TX
Epigenetics of Lymphomas

Jennifer E. Amengual, MD
Columbia University
New York, NY
Can We Use Epigenetics to Prime Chemoresistant Lymphomas?

Kami J. Maddocks, MD
Ohio State University Hospital
Upper Arlington, OH
Novel Targets in Aggressive Lymphomas

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Caring for Patients with Acute Leukemia in Community Hospitals: Who, What, and When to Refer?


The availability of multiple new therapeutic options has led to fundamental shift in the treatment landscape of acute leukemias. With the availability of new oral agents and parenteral drugs suitable for outpatient administration, especially in acute myeloid leukemia (AML), there is increased interest in treating acute leukemia patients in the community rather than referring them to academic centers. Thus, this education session will explore best practices for treating acute leukemia patients in the community, in which situations these patients should be referred for treatment, or co-management, at academic centers, and provide an update on issues in infection prophylaxis and treatment.

First, Dr. Halpern will discuss practice patterns and outcomes for patients treated in the community compared with academic centers and examine potential reasons for differences in outcomes. She will review which specific patient subgroups might benefit most from treatment at academic centers, and finally will explore how expanding diagnostic methods and the changing therapeutic landscape in AML might affect differences in patient outcomes at different types of institutions.

Dr Jillella will then discuss the importance of community hospitals in the management of patients with acute leukemia. She will discuss the role of community and academic collaboration as an additional step to improving patient outcomes in the community and will also explore the barriers to clinical trials in the community and a collaborative means to improve accrual.

Finally, Dr. Taplitz will review important issues in prophylaxis and treatment of infections in patients with acute leukemia in the community, with a special focus on newer data on antimicrobial prophylaxis, treatment of febrile neutropenia including de-escalation of antibiotics, and infection risk with some of the newer therapies for treatment of leukemia.

Chair:

Anna B. Halpern, MD
University of Washington
Seattle, WA

Speakers:

Anna B. Halpern, MD
University of Washington
Seattle, WA
Practice patterns and Outcomes for Patients with Acute [Myeloid] Leukemia

Anand P Jillella, MD
Medical College of Georgia At Augusta University
Augusta, GA
Optimizing [AML] Management in Community Centers and When to Refer

Randy Taplitz, MD
City of Hope National Medical Center
Duarte, CA
Supportive Care for Patients with Acute Leukemias

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Challenging Situations for Patients with Aggressive Lymphomas


Although there have been recent advances in the management of patients with aggressive lymphomas ranging from chimeric antigen receptor T-cell therapy to combination of antibody-drug conjugates with chemotherapy, significant challenges still exist in care delivery for this patient population. Such challenges include appropriate application of treatment advances to older patients with significant comorbidities, selection of effective treatments for patients with relapsed/refractory T-cell lymphomas, and timely integration of palliative care with disease-directed care. This educational session will explore the evidence for selecting treatments that balance efficacy with tolerability for less fit patients. It will also describe how to apply an understanding of subtypes of relapsed/refractory T-cell lymphoma to make effective treatment recommendations. Lastly, the session will discuss strategies to incorporate palliative care for patients with aggressive lymphomas.

Dr. Nancy Bartlett will outline the challenges of providing intensive treatment for patients with aggressive B-cell lymphomas who are over the age of 80 or have significant comorbidities. She will discuss evidence-based approaches of less-intensive yet effective treatments for this population. This talk will outline strategies by which to select treatment options that optimize efficacy while minimizing toxicity for less fit patients with aggressive B-cell lymphomas.

Dr. Lauren Pinter-Brown will outline recent advances in the treatment of aggressive T-cell lymphomas in the front-line setting. She will also discuss challenges regarding selecting patients who might benefit from consolidative autologous hematopoietic stem-cell transplant and treatment selection in the relapsed/refractory setting. Using a case-based format, Dr. Pinter-Brown will discuss how to apply an understanding of the subtypes of aggressive T-cell lymphomas to inform decision-making regarding consolidative transplant in the first-line setting and selection of treatment in the relapsed/refractory setting.

Dr. Oreofe Odejide will discuss the growing prognostic uncertainty that accompanies recent advances in the treatment of relapsed/refractory aggressive lymphomas and the challenge such uncertainty poses to integration of palliative care. This talk will discuss approaches for hematologic oncologists to provide primary palliative care for patients with relapsed/refractory disease and published tools to guide timely goals of care conversations. Dr. Odejide will also discuss approaches to integrate specialty palliative care for this patient population.

Chair:

Oreofe O. Odejide, MD,MPH
Dana-Farber Cancer Institute
Boston, MA

Speakers:

Nancy Bartlett
Washington University Sch. of Med. Siteman Cancer Center
Saint Louis, MO
Approaches for Unfit Patients with Aggressive Lymphomas

Lauren C. Pinter-Brown, MD
University of California - Irvine
Orange, CA
Strategies for T-Cell Lymphomas

Oreofe O. Odejide, MD,MPH
Dana-Farber Cancer Institute
Boston, MA
Strategies for Management of Relapse after Aggressive B-cell and T-cell Lymphomas and How to Introduce Palliative Care

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Handling Challenging Questions in the Management of CML


In 2020 for the great majority of chronic phase chronic myeloid leukemia (CML) patients, life expectancy is unaffected by a diagnosis of CML because of the unparalleled efficacy of ABL targeted tyrosine kinase inhibitors (TKIs) in halting disease progression. In daily practice CML patients frequently have medical comorbidities that can lead to an increased risk of adverse events with TKI therapy. A key goal is to optimize patient outcomes weighing CML disease risk factors, patient age and medical history. Using a clinical case-based and evidence-based approach, this educational session will explore how disease-specific risk factors and patient comorbidities influence TKI selection and how we promote safe management in patients with comorbidities.

The paradigm for long-term CML management continues to evolve. Treatment-free remission (TFR), successful discontinuation of TKI therapy while maintaining major molecular response, is among the most important patient-described goals and is a strategy, if successful, that can limit healthcare costs. The session will also examine when TKI discontinuation may be considered.

Dr. Vivian Oehler will discuss the evidence guiding selection between the 1st generation TKI imatinib and the 2nd generation TKIs bosutinib, dasatinib, and nilotinib first-line in the context of patient-specific CML disease risk factors, therapy-related risks, and treatment goals. The impact of 1st generation vs. 2nd generation TKI selection on treatment response and outcomes, the ability to achieve TFR and the impact of specific TKIs on longer-term health will be reviewed.

Dr. Delphine Rea will discuss how patients who may safely discontinue TKI therapy are selected. Using the latest available evidence this talk will describe expected outcomes and factors that promote successful TFR including depth of response and timing of discontinuation. How specific TKI selection and molecular responses influence the ability to discontinue therapy will be reviewed, as well as risks associated with TKI discontinuation.

Dr. Jorge Cortes will examine how patient comorbidities, particularly cardiovascular risk factors, impact the risk for serious adverse events and subsequent monitoring and management. Recognizing that patients with comorbidities were frequently excluded from clinical trials, available evidence will be reviewed regarding specific TKI risks and optimized management to ensure good overall treatment outcomes while minimizing more serious consequences

Chair:

Vivian G. Oehler, MD
Fred Hutchinson Cancer Research Ctr.
Seattle, WA

Speakers:

Vivian G. Oehler, MD
Fred Hutchinson Cancer Research Ctr.
Seattle, WA
First Generation vs. Second Generation TKI - Which is Best At Diagnosis of Chronic Phase CML?

Delphine Rea, MD,PhD
Hopital Saint-Louis
Paris, France
When is it safe to stop TKIs?

Jorge E. Cortes, MD
Georgia Cancer Center, Augusta University
Augusta, GA
How do manage CML patients with comorbidities?

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Immunotherapy in Multiple Myeloma


While the Nobel prize for development of monoclonal antibodies by fusion of multiple myeloma (MM) cells with mouse derived spleen cells was awarded in 1984, there were no therapeutic antibodies for the treatment of myeloma for over 30 years. Since 2015, there has been an explosion in the investigation of immunotherapeutic approaches – both humoral and cellular strategies. In spite of an arsenal of 7 currently available classes of anti-MM therapies, there remain many unmet medical needs (eg patients who are genomically and functionally high risk, multidrug refractory, frail elderly) and it will be exciting to see if immunotherapeutic strategies can help fill these voids. 

Dr Niels Van de Donk will discuss treatment selection and sequencing, which becomes increasingly complex with the increasing number of therapeutic options. The choice of treatment is dependent on both patient- and tumor-related factors. Treatment-related factors, such as type and response to prior therapy are also critical in terms of the selection of a new treatment regimen. The increasing use of CD38 antibodies in newly diagnosed and early relapsed/refractory MM, raises questions, including subsequent retreatment with CD38 antibodies. With the introduction of new immunotherapies with novel modes of action, sequencing should also take into account the effect of prior therapy on immune function.

Dr Eric Smith will outline B Cell membrane antigen (BCMA) targeted chimeric antigen receptor (CAR)-T cell therapy for MM, which induces frequent and deep responses in heavily pre-treated patients, however relapse is still far too common. After reviewing emerging clinical data, potential mechanisms of resistance will be explored, including T cell intrinsic and tumor cell mediated. Some of these resistance factors will be specific to targeting BCMA, while others are broadly applicable. Potential strategies in late pre-clinical or early clinical development aimed at addressing the underlying biology that leads to sub-optimal responses will also be discussed. 

Dr Chari will discuss other known MM antigens that may serve as effective therapeutic targets for naked antibodies and several such agents are in development. Some of these cell surface antigens are also internalized, making them ideal candidates for antibody–drug conjugates (ADC) such as belantamab mafodotin, conjugated with auristatin F and targeting BCMA. An entirely novel class of agents are the T cell redirecting agents with the majority being bispecific monoclonal antibodies that simultaneously bind CD3 on T cells and a myeloma antigen. Bispecifics targeting the MM antigens BCMA and GPRC5d have already demonstrated deep and durable responses in heavily pretreated patients. Bispecifics targeting CD38 and FCHR5 as well as trispecifics are under investigation.

Chair:

Ajai Chari, MD
Mt. Sinai School of Medicine
New York, NY

Speakers:

Niels W. C. J. Van De Donk, MD
VU University Medical Center
Amsterdam, NH, Netherlands
Sequencing Multiple Myeloma Therapies With and After Antibody Therapies

Eric L Smith, MD,PhD
Dana Farber Cancer Institute
Boston, MA
The Future of CAR T Cells in Multiple Myeloma

Ajai Chari, MD
Mt. Sinai School of Medicine
New York, NY
Talk 3 Bispecific, Trispecific, and Other Novel Immune Treatments in Myeloma

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Improving Symptom Control for Children with Hematological Malignancies


Dr. Lillian Sung will describe the importance of symptom control in children with cancer, and approaches to identify symptoms amenable to clinical implementation. Topics will include child self-report, parent proxy-report and a novel collaborative approach involving child and parent dyads. Recent research will be reviewed along with approaches toward clinical implementation that allow program evaluation.

Dr. Tamara P. Miller will discuss the current method of capturing treatment toxicities on pediatric hematology clinical trials, under-reporting of toxicities using the existing system, and the specific challenges related to pediatric trials. Potential solutions to improve adverse event ascertainment, including using electronic health record data, will be described.

Dr Robert Phillips will discuss approaches to control toxicity and aversive symptoms, from preventative strategies to therapeutic approaches. The importance of patient and parent involvement and the trade-offs we must make in such strategies will be considered, contextualizing recent research and outlining how future research could be improved.?"

Chair:

Lillian Sung, MD,PhD
Division of Hematology/Oncology, Department of Pediatrics, Hospital for Sick Children
Toronto, Canada

Speakers:

Lillian Sung, MD,PhD
Division of Hematology/Oncology, Department of Pediatrics, Hospital for Sick Children
Toronto, Canada
Symptom Screening in Routine Care - Time to Move Beyond Research?

Tamara P. Miller, MD,MSc
Emory University School of Medicine/Children's Healthcare of Atlanta
Atlanta, GA
Capturing Treatment Toxicities in Clinical Practice

Robert Phillips, MD
Leeds Children's Hospital
Leeds, United Kingdom
Interventions to Improve Symptoms

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Indolent Lymphomas: Answers to Smoldering Questions


Many therapeutic progresses have been made in recent years in the field of indolent lymphoma. Beside different classical cytotoxic agents, immunotherapies have continued to develop, with new agents and new combinations recently approved. Several kinase inhibitors have been evaluated in follicular and marginal zone lymphomas, and are now available for patients, and other forms of targeted therapies are currently being investigated. All of these approaches may have specific side effects, potentially impairing patient quality of life. While these diseases are often chronic, patients overall survival has been significantly improved in the last 20 years and some of them may enjoy long periods without any treatment, and eventually be considered as cured. Others present with difficult to treat disease, refractory to multiple therapies, and histologic transformation has been usually considered as a turning point in the disease history, when other therapeutic approaches need to be implemented.

Dr. Gilles Salles will review different therapeutic options currently available for the first line or subsequent progressions in patients with follicular lymphoma, and discuss how to optimize the use of the different approaches.

Dr. Pier Luigi Zinzani will discuss the different clinical presentations of marginal zone lymphoma and address their therapeutic management. He will also address how new agents or combinations developed in the field could be used for those patients.

Dr. Sonali Smith will review the current data available for patients with transformed indolent lymphoma, and discuss the respective role of cytotoxic agents, transplant and CAR-T cells in this setting.

Chair:

Gilles Salles, MD, PhD
Centre Hospitalier Lyon-Sud
Lyon, France

Speakers:

Gilles Salles, MD, PhD
Centre Hospitalier Lyon-Sud
Lyon, France
How Do I Sequence Therapy for Follicular Lymphoma?

Pier Luigi Zinzani, MD, PhD
University of Bologna
Bologna, Italy
How Do I Sequence Therapy for Marginal Zone Lymphomas?

Sonali M. Smith, MD
University of Chicago
Chicago, IL
Transformed Lymphoma - What Should I Do Now?

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Managing Toxicities of Targeted Therapies in CLL


The last 5-10 years have been an incredible time for patients with CLL given the advent of novel agents such as venetoclax, BTK and PI3K inhibitors. The use of these therapies has resulted in high response rates and long progression-free survival even in patients with high-risk, relapsed or refractory disease. As life-changing as these therapies have been in the care of CLL patients however, these agents have unique side effects which have required providers to gain familiarity and adapt their practice accordingly. This educational session will review the toxicity profiles of the most commonly used novel agents currently used to treat CLL patients, and will address the challenges and potential strategies to assess and manage real-world toxicity of these agents. Dr. Kirsten Fischer will summarize recent clinical advances with regards to preventing and monitoring for tumor lysis syndrome and other toxicities of venetoclax in therapy of CLL. She will discuss the opportunities and challenges associated with the treatment of venetoclax and will provide recommendations on how to optimize therapy with venetoclax. Dr. Lamanna will review the clinical data with regards to the incidence and management of toxicities related to Bruton’s tyrosine kinase (BTK) inhibitors with a focus on clinical implications and applicability. She will discuss next generation BTK inhibitors as well as potential combination strategies that are being evaluated. Dr. Danielle Brander will discuss the identification and management of the early and late onset toxicities that can arise with the use of phosphoinositide-3-kinase (PI3K) inhibitors for treatment of patients with CLL. This will include a review of clinical trials investigating next generation PI3K inhibitors, combination therapy, and/or dosing schedules that may mitigate toxicity risk.

Chair:

Nicole Lamanna, MD
Columbia University Medical Center
New York, NY

Speakers:

Kirsten Fischer, MD
University Hospital Cologne
Cologne, Germany
Preventing and Monitoring For Tumor Lysis Syndrome and Other Toxicities of Venetoclax

Nicole Lamanna, MD
Columbia University Medical Center
New York, NY
Managing Toxicities of Bruton Tyrosine Kinase Inhibitors

Danielle M. Brander, MD
Duke University
Durham, NC
Managing Toxicities of PI3K Inhibitors

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Monoclonal Gammopathies of Determined Significance


In the last few years, the clinical significance of rare monoclonal gammopathies that are caused by non-plasmacellular clones or by small plasma cell clones that cause severe organ damage has been fully recognized. Better understanding of mechanisms of disease, new diagnostic tools and novel therapeutic agents tested in ongoing or recently concluded clinical trials can remarkably improve patient management and outcome. Still, these diseases remain difficult to recognize and their treatment is challenging. This educational session will focus on Waldenström macroglobulinemia, light chain amyloidosis, and other monoclonal gammopathies of clinical significance. The session will discuss the diagnostic workup, risk assessment, personalized therapy and monitoring of patients suffering from these diseases in light of the results of recent clinical trials.

Dr. Giovanni Palladini will review the state of the art and recent advancements in the diagnostic workup, staging, and monitoring of patients with light chain (AL) amyloidosis. The results of recent controlled clinical trials and placement of novel therapeutic options in the treatment of newly-diagnosed and relapsed/refractory patients will be discussed.

Dr. Jorge Castillo will review recent advances on the management of patients with Waldenström macroglobulinemia (WM), including assessment of risk of progression in patients with asymptomatic WM, indications to treat and how to personalize treatment options based on the patient's clinical features and genomic profile. The results of recent and ongoing clinical trials and the application of these emerging data on patients care will also be discussed.

Dr. Angela Dispenzieri will review the spectrum of monoclonal gammopathies that are of clinical significance (MGCS) including those that cause dermopathy, neuropathy, and/or nephropathy. She will discuss how to rule in or rule out MGCS in a busy clinic and share what is known to date about pathophysiology and management of these rare conditions.

Chair:

Giovanni Palladini, MD, PhD
University of Pavia and Foundation 'IRCCS Policlinico San Matteo'
Pavia, PV, Italy

Speakers:

Giovanni Palladini, MD, PhD
Università Degli Studi Di Pavia
Pavia, Italy
Management of AL Amyloidosis in 2020

Jorge J. Castillo, MD
Dana Farber Cancer Institute
Boston, MA
Management of Waldenström Macroglobulinemia in 2020

Angela Dispenzieri, MD
Mayo Clinic
Rochester, MN
Monoclonal Gammopathies of Clinical Significance

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Myelodysplastic Syndromes: What We Have and What We Want


Myelodysplastic syndromes (MDS) constitute a heterogeneous set of disorders for which the management remains complex and is challenged by a variety of clinical presentations with mostly broad based approaches for treatment. Advances in understanding the pathobiology of myelodysplasias are driven by improved invent of mouse models that recapitulate the MDS phenotype rather than using leukemia-derived models. Furthermore, identification of somatic mutations in MDS have generated enthusiasm for the hope of targeted therapies, even if for only small subsets of patients. This educational session will explore the evidence for best practices for risk stratification for patients diagnosed with MDS. Treatment options for both lower risk and higher risk MDS patients will be discussed in detail and will include existing therapies, novel agents as well as hematopoietic stem cell transplant approaches.

Dr. Uwe Platzbecker will review the specifics of risk stratification for patients diagnosed with MDS. MDS are associated with varying degrees of cytopenias and clinical presentations. This poses a particular challenge to providers when counseling patients regarding prognosis and subsequent treatment. This educational session will review the established scoring systems and their advantages/disadvantages. Important considerations on use of the scoring systems include assessment of intermediate risk MDS as well as CMML. Current strategies to improve future precision of risk stratification will also be described.

Dr. Hetty Carraway will discuss the treatment options for patients diagnosed with lower risk MDS. She will review the approaches for patients with isolated cytopenias as well as the management of those MDS patients with multiple cytopenias. Therapeutic considerations regarding presence of somatic mutations will also be reviewed with regard to best treatment option (either now or in development).

Dr. Bart Scott will address the established and newer treatment options for higher risk patients diagnosed with MDS. While hypomethylating therapy has been the mainstay of therapy for this population, he will address the upcoming novel combination therapies that challenge this status quo. Dr. Scott will also provide an update on the use of hematopoietic stem cell transplant and the current approaches for patients eligible for this curative approach.

Chair:

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

Speakers:

Uwe Platzbecker
University Hospital Leipzig
Leipzig, Germany
Risk Stratification in MDS

Hetty E. Carraway, MD, MBA
Cleveland Clinic
Cleveland, OH
Therapy for Lower Risk MDS

Bart L. Scott, MD
Fred Hutchinson Cancer Center
Seattle, WA
Existing Agents, Novel Agents, or Transplantation for High Risk MDS

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Myeloproliferative Disorders: Too Many Cells, Too Few Therapies - How Do We Choose?


The genomic revolution has transformed our understanding of the pathophysiology of the myeloproliferative neoplasms (MPN), and has redefined MPN diagnostic criteria. The myeloproliferative neoplasms are indolent diseases where survival is measured in decades, yet morbidity and mortality vary widely. In addition to transforming diagnostic criteria, genomics have informed nearly every aspect of MPN. This educational session will explore the evidence for comprehensive genomic assessment during stages of MPN from initial evaluation, through evolution and transformation.

Dr. Alison Moliterno will outline the evidence for use of genomic testing in evaluating MPN presentation across varied clinical contexts. She will discuss the evidence supporting the use of clonal genomic burden in determining MPN subtypes and assessing thrombosis risk, and its use in clinical decision making.

Dr. Olatoyosi Odenike will discuss how genomics informs the evaluation and management of MPN progression. The therapies for MPN have expanded, and the genomics of clonal progression and transformation can be a guiding light in assessing suitability for therapies to meet treatment goals.

Dr. Mrinal Patnaik will discuss the opportunities and challenges that genomics offers in the management of myeloproliferative/myelodysplastic syndromes. He will discuss the insights that genomics has produced in illuminating these complex syndromes, and will highlight how genomic assessment will light the way to targeted therapy.

Chair:

Alison R. Moliterno, MD
Johns Hopkins University School of Medicine
Baltimore, MD

Speakers:

Mrinal M. Patnaik, MD,MBBS
MAYO
Rochester, MN
Myeloproliferative/Myelodysplastic Overlap Syndromes: Diagnosis and Treatment

Alison R. Moliterno, MD
Johns Hopkins University School of Medicine
Baltimore, MD
Applied Genomics in MPD

Olatoyosi Odenike, MD
University of Chicago
Chicago, IL
Myelofibrosis: When to Refer for Allogeneic Transplantation

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Pediatric Hematological Malignancies: CARs for Kids


The remarkable success of CD19-targeted CAR-T cells for pediatric Pre-B acute lymphoblastic leukemia (B-ALL) has not only changed how practitioners approach relapsed, refractory disease, but also has raised several important, if controversial, questions. For decades, allogeneic hematopoietic stem cell transplant (allo-HSCT) has been the gold standard salvage therapy for pediatric hematological malignancies. With continuing improvements to efficacy and the accumulation of more data on safety, CAR-T cells have emerged as a probable, less toxic replacement for allo-HSCT. This educational session will explore current thinking regarding use of CAR T cells earlier in therapy for patients with poor prognostic features who would historically have been relegated to allo-HSCT in first remission. Importantly, despite their resounding successes in B-ALL, whether CD19 CAR T cells will have similarly striking results in pediatric patients with mature B-cell lymphomas including Burkitt Lymphoma remains an open area of investigation. Beyond CD19, we will discuss current clinical and pre-clinical CAR-T cell strategies targeting an array of other antigens for pediatric patients with lymphoid and myeloid malignancies, including T-lineage malignancies.

 

Dr. Maude will outline the evidence for use of CD19 CAR T cells as an alternative to allo-HSCT, both in patients with relapsed, refractory Pre-B ALL and in patients with poor-prognostic features who have achieved first remission. She will discuss evidence suggesting CAR T cells are a feasible alternative to allo-HSCT, as well as how patient and clinical characteristics impact clinical decision-making.

 

Dr. Rouce will discuss the use of CAR T cells for mature B cell and Burkitt lymphoma, reviewing current clinical trials testing different CD19 CAR constructs in this patient population, as well as special challenges in treating these diseases.  She will review efficacy data and discuss broader application and best practices that can be extrapolated from adult data.  

 

Dr. Gardner will discuss the challenges preventing the rapid translation of CAR T cell strategies for T lineage and myeloid malignancies, as well as strategies to overcome them. She will provide a comprehensive overview of CAR targets for these malignancies, as well as safety concerns for CAR T cell therapies for this pediatric patient population and strategies to overcome them.

Chair:

Rayne H. Rouce, MD
Texas Children's Hospital
Houston, TX

Speakers:

Shannon L. Maude, MD, PhD
Children's Hospital of Philadelphia
Philadelphia, PA
CAR-T Cells vs. Allogeneic HSCT for Poor Risk ALL

Rayne H. Rouce, MD
Texas Children's Hospital
Houston, TX
CAR-T Cells for Mature B Cell Lympohoma and Burkitt Lymphoma

Rebecca A Gardner, MD
Seattle Children's Hospital
Seattle, WA
CAR-T Cells for Other Pediatric Hematological Malignancies Including AML

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The Emerging Role of Targeted Therapies and Cell Therapy in Transplant


Considered the original form of cell therapy, the field of stem cell transplantation has undergone a paradigm shift since Dr. Don Thomas’ seminal publication in 1957. Advances in our basic understanding of the graft-versus-leukemia effect have paved the way for novel engineered cell-based therapeutics with target antigen specificity and without the toxicity associated with graft-versus-host disease. In this session we will provide an overview of the past, present and future of this exciting field.

Dr. Stephan Mielke will provide a historical overview of the evolution of allogeneic stem cell transplantation and emerging cell therapies, such as CAR T cells from experimental therapies to standard of care. He will present data to support the role of immunotherapy, both as a definitive treatment approach as well as a complement to allogeneic stem cell transplantation in the context of different malignancies. He will challenge the definitions of clinical success from the perspectives of regulatory hurdles, value-based healthcare and patient access to treatment.

Dr. Jesus G. Berdeja will discuss the unique safety profile and logistical aspects that pose challenges and opportunities for the safe and successful delivery of these therapies. Close interaction, communication, and established partnerships between the community practitioner, the disease specialist and the transplant physician will be required to provide the optimal care longitudinally for any one patient. Dr. Berdeja will discuss practical aspects for programs to deliver these therapies and how future advances may ultimately widen availability beyond just a few centers.

Dr. Katayoun Rezvani will provide an overview of next-generation cell therapies beyond T cells, including the application of natural killer (NK) cells, invariant NKT cells, macrophages and other immune subsets as platforms for CAR engineering. She will also discuss the application of gene editing technologies such as CRISPR-Cas9 to further enhance the safety and potency of engineered cells for the immunotherapy of cancer.

Chair:

Katayoun Rezvani, MD, PhD
MD Anderson Cancer Center
Houston, TX

Speakers:

Stephan Mielke, MD
Karolinska Institute & University Hospital
Stockholm, Sweden
The Evolving Field of Hematopoietic Stem Cell Transplantation: The Emerging Role of Cell Therapy

Jesus G. Berdeja, MD
Sarah Cannon Research Institute and Tennessee Oncology
Nashville, TN
Practical Aspects of Building a New Immunotherapy Program: The Future of Cell Therapy

Katayoun Rezvani, MD, PhD
MD Anderson Cancer Center
Houston, TX
Next Generation cell therapies: beyond CAR T

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Understanding How to Manipulate the Immune System in Immunotherapy for Lymphoma


The anti-tumor immune response in lymphoma is typically compromised, exhausted or suppressed. Treatment strategies are being developed to manipulate the immune system and optimize immune function to generate a more effective anti-tumor immune response. In this session, we will discuss the fundamentals of immunotherapy for lymphoma, as well as strategies using checkpoint blockade, bispecific antibodies, novel drugs and cellular therapies to improve immune function.

Dr. Stephen Ansell will provide a general overview of the components that are necessary for an optimal anti-tumor immune response and highlight the immunological barriers to an adequate immune response that exist in patients with lymphoma.

Dr. Catherine Diefenbach will discuss the role of drugs in optimizing immune function in lymphoma. She will review clinical results with immune checkpoint blockade, bispecific antibodies, as well as small molecule inhibitors.

Dr. David Porter will review the use of cellular therapies in treating patients with lymphoma. He will discuss the biology, efficacy and side effects associated with cell-based approaches and the impact they are making on lymphoma management.

Chair:

Stephen M. Ansell, MD, PhD
Mayo Clinic
Rochester, MN

Speakers:

Stephen M. Ansell, MD, PhD
Mayo Clinic
Rochester, MN
Fundamentals of Immunology for Understanding Immunotherapy

Catherine Diefenbach, MD
New York University School of Medicine, NYU Cancer Institute
New York, NY
Immunotherapy with Drugs

David L. Porter, MD
University of Pennsylvania
Philadelphia, PA
Immunotherapy with Cells

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Sessions on Non-Malignant Hematology

Advances in the Laboratory Assessment of Hemostatic and Thrombotic Disorders


Laboratory assessment of hemostatic and thrombotic disorders is a diverse and changing field. Because disorders involving bleeding and clotting range from complex, multisystem disorders, to monogenic disorders of a single coagulation factor, the diagnosis of these disorders can be difficult and time consuming. In this session, we will address three common modern conundrums in laboratory testing of hemostatic and thrombotic disorders and discuss the challenges and novel techniques developing to meet these challenges.

Dr. Johanna Kremer Hovinga will discuss the diagnosis of microangiopathic hemolytic anemia starting with the determination of schistocytes on the peripheral blood smear in the setting of thrombocytopenia as the key findings in all forms of thrombotic microangiopathy. She will then summarize key features of patient history and standard laboratory tests which can be used to make a first evaluation and describe specialized testing that can be used to establish the final diagnosis and to guide treatment and follow-up.

Dr. Michele Lambert will then discuss the current efforts ongoing to standardize and improve reporting of genetic variants and determine pathogenicity of novel genes in the hereditary hemorrhagic, thrombotic and platelet disorders. She will summarize efforts by ClinGen and the role of the American Society of Hematology and the International Society of Thrombosis and Haemostasis to drive standardization of terminology and reporting.

Dr. Rita Selby will introduce and briefly describe viscoelastic (VE) assays in hemostasis which includes both Thromboelastography (TEG) and Rotational Thromboelastometry (ROTEM) assays, comparing them to standard laboratory based routine hemostasis assays. She will discuss the evidence supporting TEG and ROTEM in common clinical scenarios requiring assessment of hemostasis and management guidance. She will outline strategies by which clinicians and the hemostasis laboratory can work collaboratively to successfully implement this testing at point of care or in the laboratory, based on clinical need, and will discuss appropriate institutional based algorithms, quality assurance and cost.

Chair:

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

Speakers:

Johanna A Kremer Hovinga Strebel
Department of Hematology and Central Hematology Laboratory, Inselspital, Bern University Hospital, University of Bern
Bern, Switzerland
The Laboratory Evaluation of Patients with Microangiopathic Hemolytic Anemia

Michele P. Lambert, MD
Children's Hosp. of Phila.
Philadelphia, PA
Improving Interpretation of Genetic Testing for Hereditary Hemorrhagic, Thrombotic, and Platelet Disorders

Rita Selby, MBBS, FRCPC
University Health Network and Sunnybrook Health Sciences Centre
Toronto, ON, Canada
Expanding Clinical Roles for Viscoelastic Testing

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Anxiety Provoking Hematology Consults, Second Edition


There is an array of complex and somewhat obscure conditions, seen but not always recognised by haematologists. Many of these provide enough diagnostic difficulty to meet the definition of anxiety-provoking consults. Three case-based examples of such conditions will be examined in detail.

Dr Kenneth L. McClain will unravel the mysteries of histiocytic disorders including Langerhan-cell and non-Langerhan cell types.

Dr D  Montgomery Bissell will unravel the complexities of acute intermittent and congenital erythropoietic porphyrias.

Dr Ari Zimran will dissect why Gaucher disease is so commonly ignored in the differential diagnosis of difficult clinical scenarios.

Chair:

Jeffrey Szer, MBBS
Royal Melbourne Hospital
Melbourne, VIC, Australia

Speakers:

Kenneth L. McClain, MD, PhD
Texas Children's Hospital
Houston, TX
Histiocytic Disorders: Novel Insights into Biology and Implications for Therapy (Langerhans and Erdheim-Chester)

Michael Linenberger, MD
University of Washington, Fred Hutchinson Cancer Research Center
Seattle, WA
Updates on the Diagnosis and Management of the Most Common Porphyrias – AIP and EPP

Ari Zimran, MD
Gaucher Unit
Jerusalem, Israel
Gaucher Disease

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Beyond the Marrow: Major Non-Hematologic Complications of Inherited Bone Marrow Failure Syndromes


Progressive bone marrow failure, myelodysplastic syndrome or acute myeloid leukemia are hematologic complications in patients with inherited bone marrow failure and related hematopoietic malignancy predisposition syndromes that are potentially curable with hematopoietic stem cell transplantation (HCT). However, a myriad of age-related and syndrome specific non-hematologic complications can develop over time in transplanted and non-HCT patients with these syndromes and pose unique challenges that can impact the quality and duration of life. Our understanding of the biology of life-threatening non-hematologic complications such as hepatopulmonary syndrome, arterio-venous malformations, pulmonary or liver fibrosis, and their association with age, disease phenotype and HCT in short telomere syndromes is evolving and is integral to inform treatment decision and management. Another major concern is an inordinately high risk of solid tumors, particularly squamous cell carcinoma of the head and neck and anogenital region in Fanconi anemia (FA) and dyskeratosis congenita (DC). This risk increases with age, and cancers tend to develop earlier in patients who have received HCT. This education session will address the non-hematologic complications in short telomere syndrome, age-associated predisposition to specific cancers in inherited marrow failure syndromes and in relation to HCT. The session will also discuss specific pre- and post-HCT considerations to reduce transplant-associated complications.

Dr. Kristen Schratz will outline the extra hematopoietic manifestations of short telomere syndromes in children and adults. She will discuss the natural histories of these complications and their relevance to diagnostic and treatment decisions for patients with bone marrow failure with a particular emphasis on pulmonary, hepatic and immune complications. This talk will also address the indications for telomere length testing beyond hematologic indications.

Dr. Neelam Giri will discuss the non-hematopoietic malignancies in the classic IBMFS and other hematopoietic malignancy predisposition syndromes with special focus on FA and DC and in relation to HCT or no HCT. She will use case-based approach to highlight the implication of biallelic FANCD1/BRCA2 or FANCN/PALB2 mutations and the risks of specific cancers in children versus the risk of squamous cell carcinomas in adults with FA due to mutations in other FA genes.

Dr. Carmen Bonfim will discuss the need for careful pre- and post-transplant considerations that must be given to patients with IBMFS and hematological malignancies predisposition syndromes. Early toxicities and long-term complications may be related to a combination of the underlying disease and exposures to pre, peri and post transplantation risk factors. Maintaining a high index of suspicion for these late effects is essential for the development of more effective surveillance strategies and prevention of sequels.

Chair:

Neelam Giri, MD
National Cancer Institute
Bethesda, MD

Speakers:

Kristen E Schratz, MD
Johns Hopkins University School of Medicine
Baltimore, MD
The Many Sequelae of Telomere Biology Disorders

Neelam Giri, MD
National Cancer Institute
Bethesda, MD
Non-Hematologic Cancer in Marrow Failure

Carmem M. S. Bonfim, MD PhD
Federal University of Parana
Curitiba, PR, Brazil
Special Considerations for Pre-and Post-Transplant in Inherited Marrow Failure Syndromes

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Chronic Transfusion Support: Challenging Cases


Blood transfusion therapy is a critical component of the care of patients with a variety of hematologic disorders. This educational session will review ways to optimize transfusion therapy in an outpatient setting, focusing on patients with myelodysplastic syndromes, thalassemias and sickle cell disease. Transfusion thresholds and targets will be evaluated in the context of challenging adult and pediatric patient cases. Best-practices around clinical coordination and logistics will be reviewed. The role of concomitant medical therapies to support patients receiving transfusions will also be assessed. The social, emotional and financial burdens of chronic transfusion therapy can weigh heavily on patients. Through a multidisciplinary approach, there may be opportunities to alleviate those obstacles and optimize patient adherence and outcomes. Dr. Erica Wood will present current recommendations for transfusion therapy for patients with myelodysplastic syndromes. Recent data on transfusion thresholds will be reviewed in the context of patient preference and clinical outcomes. Dr. Ashutosh Lal will discuss challenges in managing transfusion therapy for patients with hemoglobin E/beta thalassemia, beta thalassemia intermedia, and alpha thalassemia major, where traditional transfusion goals may not apply. Dr. Jennifer Webb will examine the social burdens of transfusion therapy, focusing on pediatric patients with sickle cell disease and their families.

Chair:

Jennifer Webb, MD
Children's National Hospital
Washington, DC

Speakers:

Ashutosh Lal, MD
Children's Hospital & Research Center Oakland
Oakland, CA
Chronic Transfusion for Patients with Hemoglobinopathies

Erica M. Wood, MBBS, FRACP, FRCPA
Monash University
Melbourne, VIC, Australia
Outpatient Transfusions for Myelodysplastic Syndrome

Jennifer Webb, MD
Children's National Hospital
Washington, DC
The Social Aspects of a Chronic Transfusion Program

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Diagnostic and Prognostic Models in VTE Management: Ready for Primetime?


Venous thromboembolism is responsible for a considerable morbidity and mortality burden world-wide, contributing to potentially preventable hospital-associated and pregnancy-related death. Conversely, development of validated algorithms to permit identification of patients who are potentially suitable for home treatment or those for whom diagnostic imaging may not be necessary is valuable, as these strategies may avoid competing risks of hospital admission and radiation exposure. Moreover, prediction of patients at high and low risk of VTE recurrence could permit refining of treatment duration and optimize targeting of treatment to those most likely to benefit. This session will explore the state of the art in all of these areas and address whether diagnostic and prognostic models in VTE diagnosis and management are indeed “ready for primetime”.

Dr. Erik Klok will discuss under which conditions patients with proven acute pulmonary embolism may be treated at home. He will highlight the different risk stratification tools tested in prospective studies and discuss the best clinical practice of follow-up of patients treated at home

Dr. Fionnuala Ní Áinle will discuss the circumstances in which VTE events may occur and how these risk factors determine future VTE recurrence risk. She will review the evidence for which circumstances are associated with highest predicted VTE recurrence risk. She will discuss gender-based differences in recurrence risk including considerations while caring for women with hormone or pregnancy-associated VTE and people who identify as transgender. She will provide an update on risk-prediction algorithms and will address whether they are “ready for prime time” or incorporation into guidelines.

Dr. Wee Shian Chan will review recent management protocols using ancillary tests in diagnostic algorithms for Pulmonary embolism in pregnancy. The strengths and limitations of these individual approaches—combination of predictions rules and D-dimer testing, to minimize the need for objective testing will be discussed. Using a case-based approach, a sensible algorithm will be explored.

Chair:

Fionnuala Ni Ainle, MD, PhD
University College Dublin
Dublin, Ireland

Speakers:

Frederikus A Klok, MD, PhD
Leiden University Medical Center
2300 RC Leiden, Netherlands
When Is it Safe to Treat PE at Home?

Fionnuala Ni Ainle, MD, PhD
University College Dublin
Dublin, Ireland
Who Are the Patients at High Risk of Recurrent Thrombosis?

Wee Shian Chan, MSc, MD, FRCPC, FACP
BC Children's Hospital
Toronto, ON, Canada
Can Pregnancy-Adapted Algorithms Avoid Diagnostic Imaging for Pulmonary Embolism?

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Genetic Testing for Heritable Hematologic Disorders 101


Germline variants underlie a host of hematologic disorders: inherited neutropenias, anemias, thrombocytopenias, erythrocytosis, thrombophilia, and predisposition to hematopoietic malignancies (HMs). Academic and commercial laboratories offer clinical testing for many of these conditions, but health care providers need to be careful about the precise test(s) being ordered; the capabilities of the test(s); and inherent limitations. Standard molecular profiling of HMs using next-generation sequencing (NGS)-based panel testing of malignant cells performed for diagnostic and prognostic purposes often cannot distinguish somatic from germline alleles. This case-based session will cover genomic sequencing methodologies in clinical use, especially focused on those designed for detection of inherited bone marrow failure and HMs, and the nuances of this testing relevant to the practicing provider. Topics will include: consistent variant interpretation; detection of potential germline alleles from NGS assays performed on malignant cells; and the ethical challenges for clinicians and patients in testing for germline predisposition.

Dr. David Wu will present a case-based discussion of the clinical interpretation of variants identified in testing for hematologic disorders and the current international efforts to provide uniform variant interpretation based on standardized curation rules. Curation of gene variants is the systematic evidenced-based process leading to a designation of clinical significance based on a five-tier system. Unfortunately, increasing numbers of genes variants are classified with uncertain or conflicting classifications. Dr. Wu will explain common NGS testing platforms, their assumptions and capabilities, and how to use current curation guidelines in daily practice to allow for optimal clinical care.

Dr. Lucy A. Godley will explain the complexities of data interpretation when NGS panels are used on blood and bone marrow samples, especially regarding the identification of somatic variants/clonal hematopoiesis versus germline variants and the limitations of most NGS assays from ‘tumor-only’ sequencing. She will discuss the frequency of detecting incidental germline findings from ‘tumor-only’ sequencing and the impact of identifying such variants. Dr. Godley will also emphasize the usefulness in comparing NGS data across time as a means of identifying individuals likely to have an inherited predisposition variant.

Dr. Jonathan Marron will discuss the numerous ethical challenges inherent in germline testing for HMs. He will focus on how informed consent for NGS testing performed for diagnostic and prognostic purposes differs from consent for other tests and procedures, given that it can allow for discovery of additional incidental germline alleles and other complex phenomena. Dr. Marron will also explain approaches to minimize anxiety for patients and families, particularly in light of the significant uncertainty related to NGS-based assays. Finally, he will examine several ethically challenging but common cases involving germline testing for HMs.

Chair:

Lucy A. Godley
The University of Chicago
Chicago, IL

Speakers:

David Wu, MD, PhD
University of Washington
Seattle, WA
Next Generation Sequencing and Variant Interpretation for Germline Hematologic Disorders for the Practicing Provider

Lucy A. Godley
The University of Chicago
Chicago, IL
Somatic Mutation Panels: Recognizing Germline Findings in Tumor Testing

Jonathan M Marron, MD, MPH
Dana Farber/Boston Children's Cancer and Blood Disorders Center
Boston, MA
Informed Consent for Genetic Testing in Hematology

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Infection Risk, Immunization Recommendations, and Antimicrobial Prophylaxis Needs when Treating Non-Malignant Hematologic Disorders - Wash Your Hands and What Else?


The hematologist uses immunosuppressive treatments (steroids, rituximab, eculizumab, cytoxan and others) and splenectomy in the management of non-malignant hematologic disorders - immune thrombocytopenia purpura (ITP), thrombotic thrombocytopenia purpura (TTP), autoimmune hemolytic anemia (AIHA, antiphospholipid antibody syndrome (APS), acquired factor deficiencies, paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) that put patients at risk for bacterial and viral infections or viral reactivation. Similarly, the natural course of hemogoblinopathies can lead to autosplenectomy and an increased risk for potentially life-threatening infections. The risk for infections can be minimized by applying appropriate immunizations strategies and using prophylactic antibiotics and antivirals in appropriate patients. Familiarity with existing immunization and prophylactic treatment strategies is key to prescribe the immunosuppressive therapies as safely as possible. This session with will present clinical-practical issues, with each talk discussing (a) the type of infections that occur with the various immunosuppressive treatments, (b) appropriate immunization strategies (vaccine choices, boosters, vaccine response monitoring), and (c) prophylactic use of antibiotics and antivirals.

Dr. Grace Lee will discuss the risk and type of infections that may occur after surgical splenectomy and autosplenectomy when treating non-malignant hematologic disorders. She will address appropriate immunization strategies - vaccine choices, need for booster vaccinations, need for vaccine response monitoring -, as well as whether anti-microbials should be given prophylactically to certain patients. Existing immunizations guidelines for the Centers for Disease Control (CDC) and other national agencies and societies will be hihglighted.

Dr. Luis E. Malpica will discuss the current evidence base detailing the risk of infections in patients with non-malignant immune-mediated hematologic diseases treated with corticosteroids and/or other oral immunosuppressants (e.g. antimetabolite, calcineurin inhibitor, cyclophosphamide). Using a case-based approach, Dr. Malpica will outline the different preventive strategies used to mitigate infectious complications such as laboratory screening, immunization, and antimicrobial prophylaxis. In addition, management suggestions will be discussed where no formal guidelines exist.

Dr. Jolan Walter will discuss infection risk, immunization recommendations, and antimicrobial prophylaxis needs when treating patient with rituximab or eculizumab. She will address the infectious risks with these treatments, appropriate immunization strategies (vaccine choices, boosters, vaccine response monitoring), the management of the patient who needs rituximab who has positive hepatitis serologies and is at risk for viral hepatitis reactivation, the risk of infection with rituximab-induced neutropenia and whether GCSF has a benefit in this situation, and whether rituximab-associated hypogammaglobulinemia should be treated with IVIG.

Chair:

Stephan Moll, MD
University of North Carolina School of Medicine
Chapel Hill, NC

Speakers:

Grace Lee
Lucile Packard Children's Hospital Stanford
Stanford, CA
Surgical splenectomy and Autosplenectomy when Treating Non-Malignant Hematologic Disorders: Infection Risk, Immunization Recommendations, Antimicrobial Prophylaxis Needs

Luis E Malpica Castillo, MD
University of North Carolina at Chapel Hill
Chapel Hill, NC
Practical Approach to Monitoring, Prevention, and Management of Infectious Complications Associated with Systemic Steroid and Other Immunosuppressive Agent Therapies in Non-Malignant Hematology

Jolan E. Walter, MD, PhD
Johns Hopkins All Children's Hospital
St Petersburg, FL
Rituximab and Eculizumab when Treating Non-Malignant Hematologic Disorders: Infection Risk, Immunization Recommendations, Antimicrobial Prophylaxis Needs

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Out of Balance: Anemias Due to Disordered Iron Homeostasis


Disorders of iron homeostasis can cause iron overload as well as iron deficiency. Iron deficiency anaemia (IDA) remains amongst the five leading causes of years lived with disability in humans, It is a global health concern affecting children, women and the elderly, whilst also being a common comorbidity in multiple medical conditions including chronic inflammatory disorders.

 

Dr. Kleber Fertrin will discuss the challenge of diagnosing and managing iron deficiency in patients with chronic inflammatory conditions. He will review the evidence for how biomarkers of iron status behave in true iron deficiency in the setting of low grade inflammation and how this can be applied to identify patients more likely to benefit from iron supplementation. Dr. Fertrin will also highlight risks and benefits of oral and intravenous iron supplementation in chronic inflammatory conditions. 

 

Dr. M.Domenica Cappellini will discuss the inherited microcytic anemias which can be broadly classified into three subgroups: (i) defects in globin chains (hemoglobinopathies or thalassemias), (ii) defects in heme synthesis, and (iii) defects in iron availability or iron acquisition by the erythroid precursors. Besides the very common microcytic anemias due to acquired iron deficiency, a range of hereditary abnormalities that result in actual or functional iron deficiency are now being recognized and they will be discussed in this presentation.

 

Dr. Michael Zimmermann will discuss the global problem of nutritional and functional iron deficiency presenting the experience in Africa. He found that Iron given to infants in Africa can reduce anemia, but can also increase gut inflammation, dysbiosis and diarrhea. Iron deficiency anemia in African infants may impair adaptive immunity and may be a previously unrecognized contributor to vaccine failure.

Chair:

Maria Domenica Cappellini
University of Milan
Milan, MI, Italy

Speakers:

Kleber Yotsumoto Fertrin, MD, PhD
University of Washington
Seattle, WA
Iron Deficiency Across Chronic Inflammatory Conditions

Maria Domenica Cappellini
University of Milan
Milan, MI, Italy
Congenital Microcytic Anemias and Their Treatments

Michael Bruce Zimmermann, MD
ETH Zurich
Zurich, Switzerland
Global Look at Nutritional and Functional Iron Deficiency

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Platelet Transfusions for Hematology / Oncology Patients: Taking a More Granular Look


This session will address key issues in the use of platelet transfusion therapy to prevent and treat bleeding in patient with hematology oncology disorders. Prophylactic platelet transfusions are widely used in patients with severe thrombocytopenia however significant breakthrough bleeding occurs.

Dr Darrell Triulzi will present data supporting this practice, limitations of prophylactic platelet transfusions, and new approaches to reduce breakthrough bleeding. A complication of platelet transfusion therapy is refractoriness characterized by a suboptimal response to platelet transfusion.

Dr Nancy Dunbar will explore the role of platelet ABO and RhD matching in platelet transfusions. Specifically we will explore the impact of major ABO incompatibility on count increments, the risks for hemolysis associated with minor ABO incompatibility and the risk of Rh alloimmunization when Rh negative patients receive Rh positive platelet transfusions.

Dr Claudia Cohn will cover the diagnosis of a platelet-refractory patient and an exploration of the immune-mediated causes for this state. Issues related to patient management will include selection of compatible platelets, laboratory tests related to platelet selection and the various clinical and laboratory factors that may influence the patient's response to platelet transfusions.Patients with immune-mediated platelet refractoriness require special management strategies.

Chair:

Darrell J Triulzi, MD
Institute for Transfusion Medicine
Pittsburgh, PA

Speakers:

Darrell Triulzi
UPMC
Pittsburgh, PA
How Well Do Platelets Prevent Bleeding?

Nancy M. Dunbar, MD
Dartmouth
Lebanon, NH
Does ABO and RhD Matching Matter for Platelet Transfusion?

Claudia S. Cohn, MD,PhD
University of Minnesota
Minneapolis, MN
Platelet Transfusion Refractoriness - How Do I Diagnose and Manage?

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Selected Hemostasis and Thrombosis Topics in Women


Women face unique haemostatic and thrombotic challenges during their life relating mainly to menstruation and pregnancy and childbirth. In women with inherited or acquired bleeding disorders these events can be even more challenging. These important clinical issues are often overlooked and underestimated in clinical practice and clinical research but in recent years it is gratifying that attention is being directed to them. This educational session will use clinical case studies to explore and discuss issues to identify best practice for assessing and managing clinical bleeding issues in women with inherited or acquired bleeding disorders and identify areas for future research to continue to improve care.

Dr Paula James will explore the significant barriers faced by women with bleeding disorders in terms of recognition and attention to their symptoms. It has been reported that women can wait up to 15 years from the onset of bleeding symptoms to an accurate diagnosis. Barriers include lack of recognition of normal vs. abnormal bleeding (especially gynecologic and obstetric), challenges in terms of laboratory testing and issues around disease classification and nomenclature. Efforts to raise awareness about the challenges facing women with bleeding disorders will be discussed as will international efforts to improve diagnosis and access to care.

Dr Bethany Samuelson Bannow will review the incidence of heavy menstrual bleeding as a complication of anticoagulation in menstruating women. She will discuss mainstays of therapy including hormonal therapies, procedure-based interventions and supportive care, as well as iron supplementation. She will review the impact of approaches including modifying the duration of anticoagulation or skipping doses and switching oral anticoagulants.

Dr Claire McLintock will review the risk factors, prevalence and management of postpartum hemorrhage (PPH). She will specifically address emerging evidence directing changes in the management of women with PPH highlighting the importance of rapid identification of women with low fibrinogen levels who require urgent fibrinogen replacement as well as optimal transfusion practice. She will also review the evidence relating to tranexamic acid in treatment of PPH.

Chair:

Claire McLintock, MD
Auckland City Hospital
Auckland, New Zealand

Speakers:

Claire McLintock, MD
Redhealth
Auckland, New Zealand
Prevention and Treatment of Postpartum Hemorrhage

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

Bethany T. Samuelson Bannow, MD
Oregon Health & Science University
Portland, OR
Management of Heavy Menstrual Bleeding on Anticoagulation

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The Brain and Pain in Sickle Cell Disease: Understanding the Role of Sensory, Cognition and Neuropathic Pathways in the SCD Chronic Pain Experience


In the US chronic pain remains the most common and costly medical condition, yet strategies to effectively treat chronic pain are fraught with frustration and dissatisfaction for both the patient and provider. Chronic pain affects over 50% of adults living with sickle cell disease (SCD) and imparts a tremendous burden on the individual and society. The etiology of chronic pain in SCD is complex and represents a convergence of several pathophysiological mechanisms that are still being explored. Evidence remains poor for the use of opioids in managing chronic pain in all populations. Despite the significant risks associated with this practice, optimal evidence-based effective alternatives are lacking, and chronic opioid therapy remains the mainstay for treating chronic sickle cell pain. This session will review the epidemiology and pathophysiology of the various chronic pain syndromes seen in SCD and the impact of chronic pain and its current treatment on the sickle cell brain. The session will additionally provide a practical approach to managing chronic pain in SCD.

Dr. Amanda Brandow will cover the epidemiology, underlying biology and therapeutic interventions for diagnosis and treatment of chronic neuropathic pain in SCD. This talk will describe the alterations of functional connectivity in the brain among individuals with SCD suffering from chronic pain, highlighting the role of central sensitization in the etiology of SCD chronic pain.

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Dr. Ifeyinwa Osunkwo will discuss the known complications of chronic long-term opioid use with a specific focus on opioid induced hyperalgesia that is commonly seen among individuals receiving chronic opioid therapy. She will discuss the long-term consequences of chronic opioid use on the brain, cognition and mood and share potential mitigation strategies proven successful in the general chronic pain literature that could be considered in SCD. This talk will also compare the effects of chronic opioids seen in the general population to potential presentations (physical, cognitive, behavioral) seen in SCD.

 

Dr. Lawrence Long will build on the previous two presentations by providing a step-wise approach to the practicing provider on how to establish a comprehensive chronic pain management strategy for individuals living with SCD in their practice including information on how to use opioids judiciously, assessing opioid risk, and other non-opioid options for chronic pain management. He will also address how to standardize one’s clinical practice to ensure patient and provider safety and adherence to best practices. This session will include a discussion of the recent chronic pain guidelines (ASH, CDC, and NHLBI) with a focus on practical aspects of managing chronic pain in SCD but will also include how to develop acute pain management care plans.

Chair:

Ifeyinwa Osunkwo, MD, MPH
Levine Cancer Institute
Charlotte, NC

Speakers:

Amanda M. Brandow, DO, MS
Medical College of Wisconsin
Milwaukee, WI
Neuropathic Pain in Sickle Cell Disease: Measurement and Management

Ifeyinwa Osunkwo, MD, MPH
Levine Cancer Institute
Charlotte, NC
Optimizing the Management of Chronic Pain in Sickle Cell Disease

Lawrence Long
UCSF Benioff Children's Hospital
Oakland, CA
Building a Contemporary Pain Management Strategy for SCD Patients in Your Practice in 2021

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Updates on the Role of Non-Anticoagulant Interventions in Venous Thromboembolism


Venous thromboembolism (VTE) remains a leading cause of significant morbidity and mortality. Anticoagulants have changed the natural history of untreated VTE in the majority of patients with over 95% success in reducing recurrence. However, there will always remain a small population of patients that either cannot receive anticoagulation, fail appropriate anticoagulation, or require adjunctive therapy to anticoagulation. This educational session will outline the role of non-anticoagulant interventions used in the management of VTE and clinically important outcomes associated with these strategies.

Dr. Bauer will discuss the role of thrombolytic agents in the treatment of acute pulmonary embolism and deep venous thrombosis. While systemic thrombolysis has not been shown to improve overall outcomes in comparison to anticoagulation alone, this is due to difficulties in performing randomized trials in severely affected patients and the occurrence of intra-cranial hemorrhage in a small percentage of patients. To address these issues, algorithms for identifying patients at high-risk for poor outcomes and data on the use of catheter-directed or reduced systemic doses of thrombolytic agents will be presented. The role of multi-disciplinary pulmonary embolism response teams (PERT) in the management of severely affected patients will also be examined.

Dr. Rajasekhar will examine trends in IVC filter utilization over the past two decades. Using a case-based approach she will outline the evidence for (and against) IVC filter use in various common clinical scenarios. Where possible, evidence-based recommendations on appropriate use of these devices will be outlined. In areas where a lack of strong evidence exists, expert opinion will be highlighted. Short-term and long-term complications associated with IVC filters and suggestions for IVC filter retrieval will be discussed. The presentation will conclude with a holistic approach on best practices surrounding IVC filters.

Dr. Breen will discuss the role of venous stenting in patients with acute and chronic presentations of venous thromboembolism. She will present a clinical case and discuss how stenting may be considered as an option in addition to current practices of anticoagulation, thrombolysis or compression hosiery in patients who have post- thrombotic syndrome or who have had lysis for acute deep vein thrombosis. She will discuss the current evidence base for the use of venous stenting and address some of the challenges that may influence patient outcomes

Chair:

Anita Rajasekhar, MD
University of Florida - Shands Hospital
Gainesville, FL

Speakers:

Kenneth A. Bauer, MD
Harvard Medical School
Boston, MA
Thrombolytic Therapy in Patients with VTE

Anita Rajasekhar, MD
University of Florida - Shands Hospital
Gainesville, FL
Inferior Vena Cava Filters: A Framework for Evidence-based Use

Karen A Breen, MD
Guys and St Thomas; NHS Foundation Trust
London, United Kingdom
Role of Venous Stenting for VTE

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What Hematologists Need to Know About Giving and Stopping Aspirin


In the past 10 years, there have been major changes in the available evidence related to aspirin for the prevention and treatment of both arterial and venous thrombosis. Although hematologists do not typically initiate or discontinue aspirin, they are often asked about risk-benefit trade-offs associated with various anti-thrombotic medications, including aspirin. Clinical questions about the "net benefit" of adding anti-platelet therapy are especially complex in patients who need to take an anticoagulant medication. This session will help the attendee to better estimate the risks and benefits of combination anti-thrombotic therapy for the prevention of arterial thrombosis. The session will also include an overview of the evidence pertinent to whether (and to what degree) aspirin can reduce the risk of venous thrombosis in various settings.

Dr. Michos will discuss the role of aspirin in primary prevention of atherosclerotic cardiovascular disease (ASCVD) in current era. In primary prevention, the absolute risks of vascular events are lower than in secondary prevention; however the complication rates (i.e. bleeding) are comparable. Recent evidence from randomized clinical trials have shown less benefit for prophylactic aspirin when used in combination with other contemporary ASCVD preventive therapies. The 2019 Primary Prevention Guideline from the American College of Cardiology  (ACC) and American Heart Association (AHA) state most healthy people do not need to take aspirin (IIb indication). However, there may still be select patients aged 40 to 70 who have a very high risk of ASCVD who may benefit from aspirin if at low risk for bleeding; the role of aspirin for primary prevention in these patients will be reviewed in this talk.  For example, one might still consider low dose aspirin (75 to 100 mg/day) among current smokers, those with a strong family history of premature ASCVD, those with very elevated cholesterol sub optimally treated with statins, those with subclinical atherosclerosis such as a coronary artery calcium scores >100, and select patients with diabetes at high ASCVD risk. However, these decisions are needed in the context of a clinician-patient risk discussion.

Dr. Barnes will discuss the role of combination aspirin and anticoagulation therapy in two key patient groups: those with dual indications (e.g., atrial fibrillation and coronary artery disease), and the emerging indication of atherosclerotic disease (stable coronary artery disease/peripheral artery disease and following peripheral artery disease revascularization). The talk will summarize key trial data and provide practical strategies to minimize bleeding risk with maximizing thrombotic risk reduction.

Aspirin, a cornerstone therapy for arterial thrombotic disease, has historically been overlooked or considered ineffective for preventing thrombosis on the venous side of the circulatory system.  In this lecture, Dr. Garcia will discuss the use of aspirin for both the primary and secondary prevention of deep vein thrombosis and pulmonary embolism.  He will examine published evidence that informs questions about the relative safety and efficacy of aspirin when compared to placebo or to anticoagulant medications as a strategy to prevent venous thromboembolism.

Chair:

David A. Garcia, MD
University of Washington
Seattle, WA

Speakers:

Erin Michos, MD
Johns Hopkins
Baltimore, MD
Does ASA Still Have a Role as Primary Prevention for Acute Coronary Syndrome

Geoffrey D. Barnes, MD, MSc
University of Michigan
Ann Arbor, MI
Combining Antiplatelet and Anticoagulant Therapy in Cardiovascular Disease

David A. Garcia, MD
University of Washington
Seattle, WA
ASA to Treat or Prevent VENOUS Thrombosis: Is There a Role in 2020?

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Yin and Yang of Autoimmunity and Immunodeficiencies in Hematology


There are over 400 monogenic disorders of the immune system, which predispose to infection, immune dysregulation/autoimmunity and malignancy. In the most recent International Union of Immunological Societies (IUIS) classification of inborn errors of immunity (primary immunodeficiencies and immune dysregulatory diseases, PIDDs), there are 45 genes associated with immune dysregulation and 43 genes associated with bone marrow failure syndromes, besides another 124 genes associated with combined immunodeficiencies (T and B cell), with or without syndromic features. These diseases present a broad spectrum of clinical phenotypes, including autoimmune cytopenias and/or hematopoietic defects. Though these are germline defects, several of them are diagnosed in adulthood or if diagnosed earlier, persist into and through adulthood. There have been several advances in management of adult and pediatric patients with PIDDs, especially in the last few years. For approximately three decades, allogeneic hematopoietic cell transplantation has been considered ‘standard of care’, and the major therapeutic option for children with inherited primary immunodeficiencies and immune dysregulatory disorders (PIDDs). Early allo-HCT is particularly important in infants or children presenting with serious or life-threatening infections, as without definitive treatment, patients with severe forms of PID, such as severe combined immune deficiency (SCID), rarely survive beyond 1 year of age.

While early allo-HCT is preferred for PIDDs, this is often not possible. An initial milder clinical phenotype, delayed diagnosis, late presentation, lack of a genetic diagnosis or an inability to identify a suitable stem cell donor may result in patients surviving to adulthood without having undergone allo-HSCT. Recent evidence has demonstrated that carefully selected adults with PIDDs can achieve equivalent outcomes following HCT compared to that routinely achieved in pediatric cohorts. Therefore, adult and pediatric hematologists need to be aware of the diagnosis of these diseases, their clinical spectrum, treatment options and the role of HCT in management of these diseases.

Dr. Abraham’s talk will focus on the diagnostic laboratory evaluation of immune dysregulatory, combined immunodeficiencies and bone marrow failure syndromes with specific case-based examples with discussion of basic and advanced testing for evaluation of specific immune pathways. Though the case examples will focus on adult patients, the overall discussion will cover the age spectrum of patients affected with inborn errors of immunity (primary immunodeficiencies).

Dr. Seidel’s talk will focus on the relevance of a molecular diagnosis in developing personalized treatment plans for patients with autoimmune cytopenias and immune dysregulation. He will discuss the role of immunophenotypic biomarkers in contributing to therapeutic decision-making, and share data from a prospective study on severe immune cytopenias in treatment stratification.

Dr. Morris’ talk will focus on HCT for these disorders, and discuss specific issues, which make transplanting adult PIDD patients particularly challenging, including: understanding the natural history of rare diseases and predicting outcome with conservative management alone; optimal timing of transplant; conditioning regimens; donor selection; pre, peri- and post-transplant management; and late effects. The role of gene therapy and or gene editing approaches as an alternative to allo-HCT in high risk, monogenic PIDDs will also be discussed.

Chair:

Roshini Sarah Abraham, PhD
Nationwide Childrens Hospital
Columbus, OH

Speakers:

Roshini Sarah Abraham, PhD
Nationwide Childrens Hospital
Columbus, OH
How to Evaluate for Immunodeficiency in Patients with Cytopenias

Markus G Seidel, MD
Medical University of Graz
GRAZ, Austria
Treatment of Immune-Mediated Cytopenias in Adults with PIDs

Emma Morris, FRCPath, MA, MBBChir, PhD, MRCP
Univ. Coll. London Royal Free Hosp.
London, United Kingdom
Allogeneic hematopoietic stem cell transplantation in adults with primary immunudeficiency