American Society of Hematology

Education Program

The following information is preliminary and subject to change.

The ASH Education Program will be held from Saturday, December 1, through Monday, December 3, with most sessions being offered twice. A question-and-answer period will occur at the end of each individual speaker presentation.

Chapters based on these sessions will be published in Hematology 2018, the ASH Education Program.

2018 Education Program Co-Chairs

Mark A. Crowther, MD

Mark A. Crowther, MD
St. Joseph's Hospital, Hamilton, Canada

Mikkael A. Sekeres, MD

Mikkael A. Sekeres, MD
Cleveland Clinic Taussig Cancer Center, Cleveland, OH

Sessions on Malignant Hematology

A Panoply of Immunotherapies for Adult B-Lineage Acute Lymphocytic Leukemia


The management of newly diagnosed and relapsed acute lymphocytic leukemia (ALL) has traditionally relied on combination chemotherapy, but novel immune-based approaches are rapidly changing the treatment landscape. This education session will focus on the role of antibody therapy for ALL and discuss the experience with chimeric antigen receptor (CAR) T cells. Further, this session will address critical challenges associated with the use of these novel therapies including toxicity and cost.

Dr. Michaela Liedtke will discuss monoclonal and bi-specific antibodies including rituximab, inotuzumab ozogamicin and blinatumomab. She will highlight key differences in their mechanism of action and provide an overview of clinical trial results that support their use. Dr. Liedtke will summarize current recommendations and review ongoing clinical studies using antibody-based ALL therapy.

Dr. Michael Pulsipher will review the role of CAR T-cell therapy in ALL and compare this approach with hematopoietic stem cell transplant. CAR T-cell therapy is still early in its development, and Dr. Pulsipher will describe how different CAR T-cell constructs can be used safely and optimally. He will then summarize practical issues associated with successfully getting patients to this therapy, and project how CAR T cells fit into our treatment paradigms now and going forward.

Dr. Mark Litzow will review some of the major toxicities that have arisen in the setting of the new immunotherapies that are revolutionizing the treatment of ALL. He will discuss how these toxicities need to be accounted for as clinicians plan the overall management of their patients. The financial burden of these new immunotherapies will be discussed and placed in the context of the benefits these therapies bring to patients.

Chair:

Michaela Liedtke, MD
Stanford Cancer Institute
Stanford, CA

Speakers:

Michaela Liedtke, MD
Stanford Cancer Institute
Stanford, CA
Antibody-Based Therapies in Patients With ALL

Michael A. Pulsipher, MD
Children's Hospital Los Angeles
Los Angeles, CA
Are CAR T Cells Better Than Antibody or Hematopoietic Cell Transplantation (HCT) Therapy in ALL?

Mark R. Litzow, MD
Mayo Clinic
Rochester, MN
No Free Rides: Management of Toxicities (Including Financial) of Novel Therapies in ALL

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Acute Myeloid Leukemia: Moving Beyond 7+3


After decades of relative stagnation in treatment options, 2017 ushered in the approval of several new therapies for acute myeloid leukemia (AML). With those new therapies comes the hope that AML may be ripe for more precise, rational, and efficacious treatments specific to molecular subtype or biologic mechanism. And yet, in 2018, how and when to test for genetic mutations, when to treat with novel agents, and whether a given patient will benefit from classical induction therapy all remain thorny questions without straightforward answers. This session will focus on how to appropriately utilize next generation sequencing and other molecular testing to characterize AML, how to incorporate novel therapies, and what to expect from traditional chemotherapeutic strategies given our understanding about disease heterogeneity.

Dr. Elizabeth Griffiths will review the role of mutational profiling in helping to define disease biology in AML at diagnosis and relapse. She will describe how mutational data are helping to inform clinical prognosis and provide opportunities for rational targeted therapeutics. Dr. Griffiths will further review which mutational events might be used to alter the approach to patient care by distinguishing those patients likely to benefit from traditional consolidation from those more suitable for inclusion in clinical trials or upfront allogeneic transplantation.

Dr. Daniel Pollyea will discuss what approaches to AML may look like in the near future with newer, molecularly defined therapies that may complement, or replace, intensive induction chemotherapy. He will describe which agents are most promising, when and how they may be used, and outline an approach to AML informed by disease biology rather than convention.

Dr. Laura Michaelis will discuss the role that classical cytotoxic therapy still plays in the treatment of newly diagnosed and relapsed patients with AML. She will summarize what can be expected from cytotoxic induction regimens in patients with good risk and intermediate risk disease and update the audience on options for patients with adverse risk cytogenetic and molecular features. Dr. Michaelis will touch on new developments in relapsed/refractory disease and, finally, will outline a reasonable approach to choosing which patients are least likely to benefit from cytotoxic therapy and should be targeted for clinical trials of less-intensive options.

Chair:

Laura C. Michaelis, MD
Medical College of Wisconsin
Milwaukee, WI

Speakers:

Elizabeth A. Griffiths, MD
Roswell Park Comprehensive Cancer Center
Buffalo, NY
When to Obtain Genomic Data in AML and Which Mutations Matter

Daniel A Pollyea, MD
University of Colorado
Aurora, CO
New Drugs Inspired by Genomics and When to Use Them

Laura C. Michaelis, MD
Medical College of Wisconsin
Milwaukee, WI
Cytotoxic Therapy in AML: Not Quite Dead Yet

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Aggressive Lymphomas


This session on aggressive lymphomas will focus on rapidly expanding genomic knowledge as applied to diagnostics, prognostics, and therapeutics. Large data sets of karyotypes, copy number abnormalities, and mutations are now available in sometimes overwhelming detail. Session 1 will summarize the most important known genomic abnormalities in T cell lymphomas; session 2 will summarize the key genomic changes in B cell lymphomas; and session 3 will focus on how these genetic abnormalities can be used to tailor therapy at relapse.

Dr. David Weinstock will discuss recent advances in our understanding of the pathobiology of T-cell lymphomas, focusing on genetic alterations with diagnostic, prognostic or therapeutic relevance. He will review issues that have complicated the development of precision medicine for patients with T-cell lymphomas and discuss newer approaches for extending genomic, transcriptional and phenotypic platforms for disease assessment.

Dr. Lisa Rimsza will discuss genomic abnormalities in B-cell lymphoma. This talk will cover disease-defining abnormalities including high grade B cell lymphomas with MYC and BCL2 and/or BCL6 translocations, RNA expression patterns in diffuse large B-cell lymphoma, key gene amplifications, prognostic and predictive mutations, and clonal heterogeneity.

Dr. Jonathon Cohen will discuss the development of novel therapies in the management of relapsed/refractory lymphoma. He will review the most promising classes of agents including targeted therapies, antibody-drug conjugates, and cellular therapies, and will review the role of molecular diagnostics in therapy selection.

Chair:

Lisa M. Rimsza, MD
Mayo Clinic - Scottsdale
Scottsdale, AZ

Speakers:

David M Weinstock, MD
Dana Farber Cancer Institute/Harvard
Boston, MA
T Cell Lymphomas: Advances in Genomics and Classification

Lisa M. Rimsza, MD
Mayo Clinic
Scottsdale, AZ
B Cell Lymphomas: Advances in Genomics and Classification

Jonathon B. Cohen, MD,MS
Winship Cancer Institute of Emory University
Atlanta, GA
Novel Therapies for Relapsed/Refractory Aggressive Lymphomas

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Approach to the Treatment of the Older, Unfit Patient With Myeloma: From Diagnosis to Relapse


There have been several advances in definition, staging, treatment, and response assessment in multiple myeloma (MM). Rather than initiating therapy only when hypercalcemia, renal dysfunction, anemia, or bone disease on bone radiographs are present, MM defining events include: > 60 % bone marrow plasma cells; abnormal free light chain ratio > 100 (involved kappa) or <0.01 (involved lambda); and focal bone marrow lesions on PET-CT and/or MRI. The revised International Staging System based upon serum beta 2 microglobulin and albumin incorporates lactate dehydrogenase and high-risk fluorescence in situ hybridization abnormalities. The International Myeloma Working Group Criteria defines minimal residual disease response as absence of clonal plasma cells (with 10-5 sensitivity) and incorporates sensitive imaging. Combinations of immunomodulatory drugs, proteasome inhibitors, histone deacetylase inhibitor, and monoclonal antibodies achieves high frequency and extent of response, as well as prolonged progression free survival (PFS) and overall survival (OS).

Drs. Thierry Facon and Tanya Wildes will use a case of newly diagnosed MM in an older patient to discuss the international and North American viewpoints, respectively, regarding diagnostic, prognostic, and treatment options. The speakers will highlight the utility of patient related factors (e.g., frailty) and tumor related factors (e.g., high risk genetics) informing choice of initial therapy, management of bone disease, use of maintenance therapy, and treatment options for relapsed disease. In this time of rapid advances, this session describes how North American and international experts treat MM to optimize response and quality of life, decrease adverse events, and prolong PFS and OS.

Chair:

Kenneth C. Anderson, MD
Jerome Lipper Multiple Myeloma Center, LeBow Institute for Myeloma Therapeutics, Dana-Farber Cancer Institute, Harvard Medical School
Boston, MA

Speakers:

Thierry Facon, MD
Hôpital Claude Huriez
Lille, France
Treatment Perspective 1

Tanya Wildes, MD
Washington University
Saint Louis, MO
Treatment Perspective 2

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Approach to the Treatment of the Young, Fit Patient With Myeloma: From Diagnosis to Relapse


In this session, speakers will present a case of a newly diagnosed patient with multiple myeloma, but in otherwise good health, who requests consultation about optimal management. With a back and forth exchange in a debate style format, Drs. Saad Usmani and Sergio Giralt will present their individual recommendations and rationales for induction therapy, stem cell transplantation/consolidation therapy, and maintenance therapy. Each speaker will discuss how new technologies will influence diagnostic testing, such as the role of gene expression profiling and of measuring minimal residual disease. The session will then follow the therapeutic choices and resulting outcomes for this patient with subsequent discussion about the sequencing of the suite of therapies for relapsing disease. Each speaker will discuss his views on the timing for implementation of allogeneic stem cell transplantation and for the emerging CAR T-cell therapies.

Chair:

Eric Seifter, MD
Johns Hopkins University School of Medicine
Baltimore, MD

Speakers:

Saad Z. Usmani, MD
Levine Cancer Institute
Charlotte, NC
Treatment Perspective 1

Sergio A. Giralt, MD
Memorial Sloan Kettering Cancer Center
New York, NY
Treatment Perspective 2

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As If Myeloproliferative Neoplasms Weren't Already Challenging Enough


While the tools of precision medicine have led to transformative progress in myeloproliferative neoplasms (MPNs), these diseases remain at an inflection point, challenged by how to best harness molecular data for optimal patient care. This session will use a case-based approach to highlight how advances in the genetics of MPNs are being assimilated into prognostication and therapeutic decision-making. Clinical perspectives will be provided on managing resistance/intolerance to JAK inhibition and progression to accelerated/blast phase disease, which remain unmet needs in myelofibrosis. Lastly, speakers will discuss challenges related to the diagnosis and treatment of advanced systemic mastocytosis and focus on the new paradigm of KIT inhibition in these neoplasms.

Dr. Ann Mullally will discuss the molecular genetics of MPNs, encompassing both phenotypic driver mutations (JAK2, CALR, MPL) and concomitant high-risk mutations such as ASXL1. She will describe the biology of these mutations and their implications for both prognosis and treatment. Dr. Mullally will also review risk stratification in MPNs more broadly, focusing on recently published models which integrate clinical and molecular variables.

Dr. Ruben Mesa will discuss management of advanced phase MPNs. He will review what constitutes progression, including transformation to acute leukemia. Dr. Mesa will summarize the challenges related to defining resistance and/or intolerance to JAK inhibitors (e.g., ruxolitinib), and management strategies for these patients. Treatment options for accelerated and blast phase disease will be outlined, including both those with and those without a transplant option.

Dr. Jason Gotlib will highlight the challenges related to diagnosis and classification of advanced variants of systemic mastocytosis (advSM). He will review the treatment landscape of KIT inhibition in advSM, including selective KIT D816V inhibitors in clinical trials. Dr. Gotlib will summarize clinical and molecular markers for monitoring response and progression, and will outline a roadmap for addressing the unmet needs of advSM patients.

Chair:

Jason R. Gotlib, MD,MS
Stanford Cancer Institute
Stanford, CA

Speakers:

Ann Mullally, MD
Brigham and Women's Hospital
Boston, MA
JAK2 (and Other Genes): Be Nimble With MPN Diagnosis, Prognosis, and Therapy

Ruben A. Mesa, MD, FACP
Mays Cancer Center at UT Health San Antonio
San Antonio, TX
Advancing Treatments for Advanced Phase MPNs

Jason R. Gotlib, MD,MS
Stanford Cancer Institute
Stanford, CA
The New Tool KIT in Advanced Systemic Mastocytosis

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AYA: Big Children or Small Adults? Leukemia Treatment in Adolescence


Adolescent and young adult (AYA) patients (age range 15-39 years) with acute leukemia are a unique population with specific cancer care needs. Management of AYAs with leukemia requires an understanding of age-related differences in disease biology, identification of optimal treatment regimens based on current evidence, and the ability to provide adequate psychosocial support during and after therapy. In this session, we will continue to explore why outcomes in AYAs remain inferior compared with other age groups. The session will first focus on differences in disease biology between children, AYAs and adults. Specific challenges and long-term morbidities that AYA survivors face will be discussed, and speakers will review factors affecting clinical trial enrollment.

Dr. Kathryn Roberts will summarize recent advances in the genomic profiling of children, AYAs and older adults, and will compare differences in the genomic landscape that may contribute to poor outcome. She will also discuss the identification of new genetic subgroups and their implications for treatment, including Ph-like acute lymphocytic leukemia.

Dr. K. Scott Baker will discuss survivorship issues unique to the AYA population including the physical, social and emotional needs that differ not only from their peers, but also from the needs of younger or older cancer survivors.

Dr. Theresa Keegan will present recent patterns in AYA clinical trial enrollment, will consider variations in enrollment across cancer types, and will discuss barriers and facilitators to clinical trial participation including sociodemographic and treatment-setting characteristics.

Chair:

Kathryn G. Roberts, PhD
St. Jude Children's Research Hospital
Memphis, TN

Speakers:

Kathryn G. Roberts, PhD
St. Jude Children's Research Hospital
Memphis, TN
Genetics and Prognosis in Children Versus Adults

K. Scott Baker, MD, MS
Fred Hutchinson Cancer Research Center
Seattle, WA
Long-Term Complications in Adolescent and Young Adult (AYA) Leukemia Survivors

Theresa H.M. Keegan, PhD, MS
University of California – Davis
Sacramento, CA
Adolescent Angst: Enrollment in Clinical Trials

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Chronic Myeloid Leukemia: With Great Success Comes Great Responsibility


In the 20 years since imatinib was first used in chronic myeloid leukemia (CML), we have witnessed a remarkable change in the outcome of this previously fatal disease, a change achieved through a combination of the use of highly effective tyrosine kinase inhibitors (TKIs) and rigorous molecular monitoring to assess response and change treatment accordingly. For the small group of patients who fail TKIs through resistance or intolerance, allogeneic stem cell transplantation (allo-SCT) remains a highly effective option.

Jane Apperley will describe the outcome of newly diagnosed patients newly treated with first or second generation TKIs, discuss the balance between the desire to achieve the deep molecular responses that permit trials of TKI discontinuation and the potential adverse effects of the early use of the more potent TKIs, and provide pragmatic advice regarding treatment decisions.

Tim Hughes will explore the appropriate time-dependent molecular targets for CML patients on TKI and the reasons why those targets differ, especially when considering treatment discontinuation. He will discuss the factors that influence the choice of TKI when molecular responses are not optimal and will highlight the critical role of molecular monitoring in managing treatment-free remission.

Charles Craddock will summarise the results of alloSCT in all phases of CML and discuss factors affecting decision-making now that increased donor availability and reduced intensity conditioning regimens have increased the number of transplant eligible patients. As disease relapse remains a major cause of treatment failure he will focus on emerging peri and post-transplant strategies to improve outcome.

Chair:

Jane Apperley, MB ChB MD
Imperial College London
London, United Kingdom

Speakers:

Jane Apperley, MB ChB MD
Imperial College London
London, United Kingdom
The Argument for Using Imatinib in CML

Timothy P. Hughes, MD
South Australian Health and Medical Research Institute
Adelaide, Australia
Molecular Monitoring in CML: How Deep, How Often, and How Should It Influence Therapy?

Charles Craddock, FRCP, FRCPath
Queen Elizabeth Hospital
Birmingham, United Kingdom
We Do Still Transplant CML, Don't We?

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Follicular Lymphoma: Have We Made Progress?


Follicular lymphoma (FL) is an incurable but treatable disease with vast array of treatment options. Despite the abundance of efficacious options, there is no universally agreed upon standard approach to treatment in the first-line or the relapsed/refractory setting. This education session will review existing data supporting the optimal approaches to the management of patients with FL in the first-line and relapsed setting with a focus on novel immune-based therapies for FL.

Dr. John P. Leonard will discuss new developments in the first line management of FL. He will address approaches to risk stratify patients, indications for treatment, and options for first line therapy. Dr. Leonard will present the risks and benefits for first line single agent therapy, immunomodulatory drugs with rituximab, chemoimmunotherapy, and maintenance rituximab.

Dr. Christopher Flowers will discuss treatment options for patients with relapsed FL. He will address strategies for sequencing of therapy in the management of relapsed patients and personalized approaches for balancing patient characteristics, preferences, and comorbidities with treatment-related factors such as efficacy, toxicity profile, and mechanisms of action to optimize outcomes.

Dr. Loretta Nastoupil will discuss current approaches using immune-based therapies for patients with FL and the biological rationale for future strategies. She will address ongoing and emerging clinical trials using novel immunotherapy approaches aimed at improving outcomes.

Chair:

Christopher R Flowers, MD,MS
Winship Cancer Institute of Emory University
Atlanta, GA

Speakers:

John P. Leonard, MD
Weill Cornell Medical College/The NewYork-Presbyterian Hospital
New York, NY
Where to Start? Upfront Therapy for Follicular Lymphoma in 2018

Loretta J. Nastoupil, MD
The University of Texas MD Anderson Cancer Center
Houston, TX
Sequencing of Therapies in Relapsed Follicular Lymphoma

Christopher R Flowers, MD,MS
Winship Cancer Institute of Emory University
Atlanta, GA
Novel Immunotherapy Approaches to Follicular Lymphoma

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Hodgkin Lymphoma: Beyond ABVD for Everyone


In North America, ABVD has been a standard approach in Hodgkin lymphoma therapy since the 1970s; however, three recent advances – PET imaging, brentuximab vedotin, and PD-1 blockade – are significantly altering treatment strategies. Recognition of the prognostic significance of interim PET has led to PET-adapted treatment approaches, which identify patients who might benefit from therapy escalation or de-escalation. Brentuximab vedotin and PD-1 blockade have improved outcomes in the relapsed and refractory setting, but their ideal place in the management of Hodgkin lymphoma is unclear. This session will focus on PET-adapted therapy and incorporation of the newer drugs for Hodgkin lymphoma, in both frontline and relapsed/refractory settings.

Dr. Ranjana Advani will summarize recent advances in the frontline treatment of advanced-stage Hodgkin lymphoma. She will discuss response-adapted therapy and the role of novel agents in patients with newly diagnosed advanced stage disease.

Dr. Alison Moskowitz will discuss the ideal settings for use of brentuximab vedotin in Hodgkin lymphoma. She will review the data supporting approved and investigational uses.

Dr. Alex Herrera will discuss the role of PD-1 inhibitors in the treatment of Hodgkin lymphoma. He will review the data supporting approved indications for PD-1 blockade and discuss investigational combinations.

Chair:

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

Speakers:

Ranjana H. (MD) Advani, MD
Stanford Cancer Center
CC-2338, CA
Risk-Adapted Upfront Therapy for Hodgkin Lymphoma in 2018

Alison J. Moskowitz, MD
Memorial Sloan Kettering Cancer Center
New York, NY
Optimizing the Role of Brentuximab in Hodgkin Lymphoma Therapy

Alex F. Herrera, MD
City of Hope
Duarte, CA
Where Do PD-1 Inhibitors Fit in Hodgkin Lymphoma Therapy?

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Reining in Graft-Versus-Host Disease (GVHD) After Allogeneic Hematopoietic Cell Transplantation


Allogeneic hematopoietic cell transplantation (alloHCT) is a potentially curative treatment for different hematological malignancies. A major life-threatening complication is acute graft-versus-host disease (GVHD). This session will focus on the biology of GVHD in the intestinal tract and the ability of serum biomarkers that predict long-term GVHD outcomes in comparison to clinical GVHD staging. Current strategies to prevent and treat GVHD in alloHCT and their limitations and challenges will be presented. Lastly, new therapies will be reviewed that do not target individual surface molecules such as chemokine or cytokine receptors, but instead inhibit signaling pathways downstream of these molecules.

Dr. James Ferrara’s talk will deal with the biology of GVHD in the intestinal tract which is the most difficult organ to treat. The ability of serum biomarkers that predict long term GVHD outcomes, which relate primarily to the intestinal tract, will also be discussed, including new biologic insights offered by the biomarkers.

Dr. Betty Hamilton will summarize the current strategies to prevent and treat GVHD in alloHCT. She will discuss the limitations and challenges to these approaches and focus on the review of novel prophylactic strategies under development and used in clinical trials for the prevention of GVHD.

Dr. Robert Zeiser will discuss novel therapeutic strategies to treat GVHD with a focus on kinase inhibition. He will also summarize features of the pathomechanism of acute GVHD and strategies that target pathways involved in immune cell activation. Dr. Zeiser will also review kinase inhibition strategies that could enhance graft-versus-leukemia effects.

Chair:

Robert Zeiser, MD
University of Freiburg Medical Center
Freiburg, Germany

Speakers:

James L. Ferrara, MD,DSc
Icahn School of Medicine at Mount Sinai
New York, NY
What Causes Graft-Versus-Host Disease? Biology Matters

Betty K. Hamilton, MD
Cleveland Clinic Foundation
Cleveland, OH
Current Approaches to Prevent and Treat GVHD Following Allogeneic Stem Cell Transplantation

Robert Zeiser, MD
University of Freiburg Medical Center
Freiburg, Germany
Innovative Approaches to Treat GVHD Following Allogeneic Stem Cell Transplantation

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The Future of Chronic Lymphocytic Leukemia (CLL) Therapy


This session will highlight the recent advances in chronic lymphocytic leukemia (CLL) therapy through illustrative case studies. The discussion will cover the status of risk-stratified upfront CLL therapy as well as the ongoing trials that may change this standard of care, the available options for relapsed therapy now and in the future, including combinations and sequencing, and what is known about the biology and therapy of Richter transformation in the era of novel agents.

Dr. Nitin Jain will review the risk-stratified frontline therapy of fit and unfit CLL patients in current practice, including the important considerations influencing that choice. He will further discuss the potential implications of ongoing clinical trials that are incorporating novel agents in frontline therapy.

Dr. Jennifer Brown will discuss relapsed therapy with a focus on sequencing in clinical practice, including the management of patients progressing on BTK inhibitors, as well as combination therapies in development and newer agents that may alter the treatment landscape. She will further discuss some of the theoretical considerations and unknowns in this rapidly evolving field.

Dr. Wei Ding will review and discuss the current data on Richter transformation, its biology and therapy in the era prior to novel agents, and whether and how its manifestations are changing in the era of novel agents.

Chair:

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

Speakers:

Nitin Jain, MD
MD Anderson Cancer Center
Houston, TX
Selecting Frontline Therapy for CLL in 2018

Jennifer R. Brown, MD, PhD
Dana-Farber Cancer Institute
Boston, MA
Relapsed CLL: Sequencing, Combinations, and Novel Agents

Wei Ding, MD, PhD
Mayo Clinic
Rochester, MN
Richter Transformation in the Era of Novel Agents

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The Molecular Maelstrom of Myelodysplastic Syndromes (MDS)


The myelodysplastic syndromes (MDS) are associated with dozens of recurrent somatic gene mutations, which influence disease biology, prognosis, and response to therapy. None of these mutations are present in more than 25 percent of patients, which results in tremendous molecular heterogeneity compared to many other neoplasms. A subset of these mutations can also be detected in a large proportion of the healthy older population and could confer a risk of MDS. This session will focus on understanding the implications of mutations in the context of MDS and age-associated clonal hematopoiesis.

Dr. David Steensma will review the clinical challenge of the common aging-associated neoplasm precursor state of clonal hematopoiesis of indeterminate potential (CHIP), including potential mechanisms of clonal progression to MDS or other hematological neoplasms, as well as risk of cardiovascular events due to circulating clonally-derived monocytes. He will also discuss management of patients with CHIP.

Dr. Aziz Nazha will review established prognostic models in MDS and their strengths and weaknesses when applied in clinical practice. He will also discuss the prognostic impact of somatic mutations in MDS, the addition of these mutations to current models, and newer approaches of incorporating the clinical and mutational data into novel prognostic models.

Dr. Amy DeZern will discuss recent developments in molecular characterization of MDS, which are improving diagnostic accuracy, providing insights into pathogenesis, and refining treatment options. She will summarize the status of our biologic knowledge of the mutational profiles in this heterogenous disease. Dr. DeZern will then outline optimal strategies for therapeutic management and highlight the current potential and challenges for targeting molecular mutations in patients with MDS.

Chair:

David P. Steensma, MD
Dana-Farber Cancer Institute
Boston, MA

Speakers:

David P. Steensma, MD
Dana-Farber Cancer Institute
Boston, MA
How CHIP Flips Into MDS

Aziz Nazha
Cleveland Clinic
Cleveland, OH
The MDS Genomics-Prognosis Symbiosis

Amy E. DeZern, MD
The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Baltimore, MD
Treatments Targeting MDS Genetics: A Fool's Errand?

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Update in Transfusion Therapy for Hematologic Malignancies: Transfusion Support – TACO and TRALI


Transfusion therapy for patients with hematological malignancies represents a unique set of circumstances and challenges. Collaboration between transfusion medicine specialists and hematologists is critical to the best patient outcome. The speakers in this session will further our understanding of opportunities to improve transfusion therapy for our patients from transfusion triggers through most appropriate product selection to managing transfusion complications.

Dr. Christine Cserti-Gazdewich will focus her presentation on inconsistencies and gaps in care as they relate to the two-dimensional theme of quantity and quality of our blood ordering, particularly as it relates to red blood cells. She will address blood compatibility testing, matching logistics in hematological malignancies and the most common hazards and impacts that are cumulatively peculiar to hematological malignancies.

Dr. Nadine Shehata will discuss managing patients who are refractory to platelet transfusions. She will address platelet refractoriness, its causes and clinical significance. Dr. Shehata will also present non-immune mediated platelet refractoriness. Finally, she will provide clinically relevant information and suggest approaches to caring for patients with platelet refractoriness. The practicing hematologist will find this presentation highly relevant to daily challenges.

Dr. Nareg Roubinian will discuss pathophysiology of transfusion associated circulatory overload (TACO) and transfusion related acute lung injury (TRALI) based on recent data from in vivo models and clinical investigations. He will present transfusion and recipient risk factors for TACO and TRALI with an emphasis on patients with hematological malignancies. Dr. Roubinian will update the audience on contemporary incidence and prevention of these transfusion complications.

Chair:

Zbigniew Szczepiorkowski, MD, PhD
Dartmouth-Hitchcock Medical Center
Lebanon, NH

Speakers:

Christine M. Cserti-Gazdewich, BSc, MD, FRCPC
University Health Network, University of Toronto
Toronto, ON, Canada
Shifting Ground and Gaps in Transfusion Support of Patients With Hematologic Malignancies

Nadine Shehata, MD,FRCPC,MSc
Mount Sinai Hospital
Toronto, ON, Canada
Managing the Patient: Refractory to Platelets

Nareg Roubinian, MD
University of California – San Francisco
San Francisco, CA
TACO and TRALI: Biology and Current Therapy

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Using Genomics to Risk Stratify and Treat Kids


As next-generation sequencing becomes a commonly available test, those caring for children with leukemia need to incorporate a rapidly changing genomic landscape into their daily practice. Such results potentially impact frontline treatment as well as approaches to relapse, as identified targets may have therapeutic implications. In addition, approximately 9 percent of children are estimated to have a germline predisposition to developing cancer and, thus, the ethics surrounding testing, counseling, and screening other family members have become critical considerations. 

Dr. Charlotte Niemeyer will review the genetics and approaches to risk stratification of patients with juvenile myelomonocytic leukemia (JMML). She will also discuss the common congenital syndromes that can be associated with JMML, such as Noonan syndrome and neurofibromatosis type 1, along with the complexities of managing such patients when they develop JMML.

Dr. Mignon Loh will review the broad landscape of somatic genetics of childhood acute lymphocytic leukemia and acute myeloid leukemia and will discuss how risk stratification has been influenced by these alterations. She will also present examples of how targeted therapies have been introduced for relevant patients.   

Using a case-based approach, Dr. Eric Kodish will review dilemmas raised by genomic testing from the perspective of pediatric ethics. Issues discussed will include but not be limited to parental permission, assent, informed consent, incidental findings, research ethics, and managing uncertainty.

Chair:

Mignon L. Loh, MD
Benioff Children's Hospital and Helen Diller Family Comprehensive Cancer Center, University of California San Francisco
San Francisco, CA

Speakers:

Mignon L. Loh, MD
Benioff Children's Hospital and Helen Diller Family Comprehensive Cancer Center, University of California San Francisco
San Francisco, CA
Using Genomics to Define Pediatric Blood Cancers and Inform Practice

Eric Kodish, MD
Cleveland Clinic
Cleveland, OH
Ethical Conundrums in Pediatric Genomics

Charlotte Niemeyer, MD
University of Freiburg
Freiburg, Germany
Juvenile Myelomonocytic Leukemia Genomics and Decisions

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Sessions on Non-Malignant Hematology

Anxiety-Provoking Consults: Macrophage Activation Syndrome, Castleman Disease, and Hypereosinophilia


This session will present a case-based approach to rare, potentially life-threatening syndromes that raise the anxiety of consulting hematologists.

Dr. Amy Klion will discuss the rationale for a personalized approach to the treatment of hypereosinophilic syndromes, including the use of conventional and novel targeted therapies. She will review a case highlighting the relationship between clinical features, molecular abnormalities, and response to therapy.

Dr. David Fajgenbaum will summarize recent advances in the diagnosis and treatment of Castleman disease, particularly the idiopathic multicentric Castleman disease (iMCD) subtype. He will review a case highlighting diagnostic challenges, currently available therapies, and the discovery of novel precision therapies for iMCD as a paradigm for other enigmatic disorders.

Dr. Nancy Berliner will review the diagnosis and treatment of two life-threatening complications of systemic autoimmune disease. She will discuss a complex patient with catastrophic antiphospholipid antibody syndrome (APS), with consideration of potential novel approaches to therapy. Dr. Berliner will also discuss a second case that highlights the unique features of macrophage activation syndrome that distinguish it from other hemophagocytic syndromes.

Chair:

Nancy Berliner, MD
Brigham and Women's Hospital
Boston, MA

Speakers:

Amy Klion, MD
National Institutes of Health
Bethesda, MD
Hypereosinophilic Syndrome: Approach to Treatment in the Era of Precision Medicine

David Fajgenbaum, MD
University of Pennsylvania
Philadelphia, PA
Novel Insights and Therapeutic Targets in Idiopathic Multicentric Castleman Disease

Nancy Berliner, MD
Brigham and Women's Hospital
Boston, MA
CAPS and MAS: The Life-Threatening Hematology/Rheumatology Interface

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Delivering High-Quality Oral Anticoagulant Therapy: State of the Art in 2018


Oral anticoagulants are both one of the most commonly prescribed classes of medication and one associated with the high risk of major complications. This session will focus on the optimal management of this class of medication. It will include discussions of clinically important drug interactions with oral anticoagulants, the role of specialized anticoagulation services, and tips for using vitamin K antagonists.

Dr. Vittorio Pengo will discuss the exclusive use of warfarin treatment in some patient categories despite its decrease after the entry of direct oral anticoagulants (DOACs). The beginning of treatment is of fundamental importance as thrombotic and hemorrhagic complications occur soon after starting treatment as a consequence of poor maintenance of international normalized ratio in the therapeutic range. Dr. Pengo will discuss how to treat an excess or a deficit of anticoagulation after stabilization of treatment, and how to handle bridging therapy in the occasion of surgery or invasive maneuvers. The use of dedicated software helps to perform correct treatment in these patients.

Dr. Sara Vazquez will highlight clinically relevant drug interactions involving both warfarin and DOACs. She will discuss monitoring and management strategies for various types of interactions in addition to providing helpful drug interaction resources for clinicians.

Dr. Nathan Clark will discuss the opportunities for anticoagulation monitoring services to incorporate management of DOACs to optimize anticoagulant drug therapy. Key clinical activities and interventions will be discussed. Dr. Clark will also describe published quality improvement efforts of anticoagulant monitoring service in this setting.

Chair:

David R. Anderson, MD
Dalhousie University
Halifax, NS, Canada

Speakers:

Vittorio Pengo, MD
University of Padua
Padua, Italy
Optimizing Quality Care for the Oral VKAs

Sara Vazquez, PharmD
University of Utah Health
Salt Lake City, UT
Drug-Drug Interactions in an Era of Multiple Anticoagulants: A Focus on Clinically Relevant Interactions

Nathan Clark, PharmD
Kaiser Permanente
Denver, CO
Role of the Anticoagulant Monitoring Service in 2018: Beyond Warfarin

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Hemolytic Anemia: A Cornucopia of Causes


Hemolytic anemias present interesting diagnostic and therapeutic challenges due to their rarity and multiplicity of causes. This session will focus on diagnosis, pathophysiology and treatment of a variety of hemolytic anemias not caused by hemoglobinopathies.

Dr. Robert Brodsky will discuss a variety of complement-driven hemolytic anemias including atypical hemolytic uremic syndrome, HELLP syndrome, and paroxysmal nocturnal hemoglobinuria. He will also discuss indications for therapy, need for continuation of therapy, and the next generation of exciting complement inhibitors in clinical development.

Dr. Mohandas Narla will summarize recent advances in the diagnosis and clinical management of inherited red cell membrane disorders. Membrane structural defects lead to hereditary spherocytosis (HS) and hereditary elliptocytosis (HE), while altered membrane transport function accounts for hereditary overhydrated stomatocytosis and hereditary xerocytosis. The degrees of membrane loss and resultant increases in cell sphericity determine the severity of anemia in HS and HE and splenectomy leads to amelioration of anemia by increasing the circulatory red cell life span. Splenectomy is not beneficial in these latter two membrane transport disorders and in fact contraindicated due to severe post-splenectomy thrombotic complications.

Dr. Anita Hill will discuss both warm autoimmune hemolytic anemias and cold agglutinin disease. Complications, such as thrombosis, are underestimated and contribute to morbidity as well as mortality. Guidelines are now available for the first time and a pathway for diagnosis and management will be discussed. The future for patients with primary cold agglutinin disease is promising with the trial results using a complement inhibitor in this disease.

Chair:

Robert A. Brodsky, MD
Johns Hopkins University
Baltimore, MD

Speakers:

Robert A. Brodsky, MD
Johns Hopkins University
Baltimore, MD
Complement Driven Anemia: More Than Just Paroxysmal Nocturnal Hemoglobinuria (PNH)

Mohandas Narla, DSc
New York Blood Center
New York, NY
Inherited Hemolytic Anemia: A Possessive Beginner's Guide

Anita Hill, MD, PhS
Leeds Teaching Hospitals NHS Trust
Leeds, United Kingdom
Auto-Immune Hemolytic Anemia

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Hemostatic and Thromboembolic Challenges in the Young


While there is much overlap in the disorders of hemostasis and thrombosis seen in pediatric and adult hematology, these symptoms and diseases in children and adolescents often have unique epidemiology, natural history, and management considerations. This session will follow a community hematologist/oncologist as she encounters several adolescent consults during her busy day in the clinic and hospital, including patients with heavy menstrual bleeding, venous thromboembolism (VTE), and immune thrombocytopenia

Dr. Sarah O’Brien will review the relevance of existing bleeding assessment tools in the evaluation of heavy menstrual bleeding in adolescents, and the difficulties in differentiating anovulatory bleeding from bleeding due an underlying hemostatic defect. Adolescent-specific considerations for each of the most commonly utilized therapies for menorrhagia will be reviewed, as well as the importance of identification and management of iron deficiency anemia.

Dr. Paul Monagle will highlight the fundamental differences in venous thrombosis between children and adults. His presentation will discuss the natural history of VTE in children and adolescents, followed by a brief review of the use of anticoagulants in young patients. Dr. Monagle will finish with a discussion of the current evidence-based guidelines for management of VTE in neonates and children.

Dr. Jenny Despotovic will describe important differences in the biology and natural history of immune thrombocytopenia (ITP) between children and adults. She will review differences in the presentation, pathophysiology, management and outcomes of pediatric and adult ITP, with attention to areas in which the available clinical practice guidelines differ in the management recommendations

Chair:

Sarah H. O'Brien, MD, MSc
Nationwide Children's Hospital
Columbus, OH

Speakers:

Sarah H. O'Brien, MD, MSc
Nationwide Children's Hospital
Columbus, OH
Managing Heavy Menstrual Bleeding in Young Women

Paul Monagle, MB, M
University of Melbourne Royal Children's Hospital
Parkville, Australia
Managing Thrombosis in Neonates, Children, and Adolescents

Jenny M. Despotovic, DO, MS
Texas Children's Hospital/Baylor College of Medicine
Houston, TX
Pediatric Immune Thrombocytopenia (ITP): Is It Different Than Adult ITP?

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High-Performance Hematology: Elite Athletes and Weekend Warriors


Hematologists may be asked to consult on high level athletes with hematologic issues. Clinical decision-making may hugely impact the athlete’s professional career or passionate hobby. Solid knowledge of the various issues involved is key in being able to give the best management advice possible. The first two speakers in this session will review the evidence for best management of venous thromboembolism (VTE), anticoagulation, sickle cell trait and sickle cell disease, and discuss reasonable management approaches where insufficient evidence exists. The third speaker will provide an overview and details about the practice of “blood doping” as worthwhile and interesting general background information for the hematologist and, for that matter, any clinician, policymaker, athlete, or member of the general public.

Dr. Stephan Moll will discuss the complexity of the management and return-to-play decisions of high-level athletes with VTE, with particular focus on long-term anticoagulation. A personalized pharmacokinetic/pharmacodynamic study of a direct oral anticoagulant may allow athletic participation when plasma drug concentration is minimal and resumption of treatment after the risk of bleeding sufficiently normalizes. Scientific data and uncertainties regarding this approach, as well as practical challenges in the implementation, will be discussed.

Dr. Robert Liem will discuss how evidence for exertional rhabdomyolysis, now termed exertional collapse associated with sickle cell trait (SCT), raises concerns for high-intensity exercise in patients with SCT and has resulted in controversial screening practices for athletes and military recruits. In sickle cell anemia, unsubstantiated concerns exist for an association between high-intensity exercise and vaso-occlusive complications. This talk will address three key questions: What is the evidence that high intensity exercise is associated with harm? What are the pathophysiologic processes that could predispose to adverse events? And what are preventive strategies to alleviate risk?

Dr. Don Siegel will discuss that despite the banning of blood doping with drugs such as erythropoietin or through blood transfusion, a cat-and-mouse game continues between perpetrators of blood doping and anti-doping agencies, as the testing becomes more and more complex to combat the athlete’s progressively more sophisticated methods of avoiding being caught. This talk will review the illicit practice of blood doping that has now led to the Athlete Biological Passport, a concept designed to monitor selected biological variables over time that indirectly reveal the effects of doping rather than attempting to detect the doping substances or methods themselves.

Chair:

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

Speakers:

Stephan Moll, MD
University of North Carolina School of Medicine
Chapel Hill, NC
Venous Thromboembolism and Anticoagulant Therapy in Athletes

Robert Liem, MD
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, IL
Exercise in Sickle Cell Trait and Sickle Cell Disease

Don L. Siegel, MD, PhD
University of Pennsylvania
Philadelphia, PA
Blood Doping in High-Performance Sport: A Chemical and Transfusion Medicine Arms Race

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Junior Faculty Career-Development Session


During this session, junior faculty will cover career-development topics, including mentorship. Speakers will discuss how to receive, provide, and optimize mentorship.

Chair:

Rebecca L. Olin, MD
University of California, San Francisco
San Francisco, CA

Speakers:

Nathan T. Connell, MD, MPH
Brigham and Women's Hospital
Boston, MA
The Difference Between a Coach and Mentor

Alison W. Loren, MD, MS
Perelman Center For Advanced Medicine
Philadelphia, PA
The Warning Signs of a Problematic Mentoring Relationship and How to Fix It

Charalambos Andreadis, MD
UCSF
San Francisco, CA
What Works: Transitioning From Being a Trainee to Becoming a Coach

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Long-Term Management of VTE: The First Six Months and Beyond


Patients who have completed some initial course of anticoagulant therapy for venous thromboembolism (VTE) often ask for advice about when or if they should discontinue therapy. The presentations in this session will help physicians consider a wide range of evidence about risks, benefits, and lifestyle burdens that could be relevant to this decision. There will also be a valuable, comprehensive review of three highly pro-thrombotic states that, although rare, can present significant (and often unique) challenges with respect to diagnosis and management.

Dr. Marc Rodger will address common questions such as "who can stop anticoagulants after short-term therapy for VTE?" and "which patients with VTE should continue anticoagulants indefinitely?" He will review the risks and benefits of ongoing anti-coagulation for VTE and provide rational thresholds above or below which he would suggest that a patient continue or discontinue anticoagulation. Dr. Rodger will also review the pros and cons of various anti-thrombotic options that can be used for indefinite secondary VTE prevention.

Dr. Walter Ageno will provide an overview of all complications associated with the long-term use of oral anticoagulant drugs in order to assist clinicians when determining the optimal duration of the secondary prevention of VTE. First, the burden of hemorrhagic complications will be reviewed. Second, the major risk factors for bleeding (along with bleeding risk scores) and their possible impact on therapeutic decisions will be reviewed. Dr. Ageno will also discuss the economic burden of anticoagulant therapy as well as the possible impact that other considerations (e.g., sport activities for young patients and falls for older patients) may have on clinical decision-making.

Dr. Leslie Skeith will summarize the available evidence and provide recommendations for the management of thrombosis in three acquired thrombophilias: antiphospholipid syndrome, heparin-induced thrombocytopenia, and paroxysmal nocturnal hemoglobinuria. She will discuss what is currently known about the use of direct oral anticoagulants for these conditions.

Chair:

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

Speakers:

Marc A. Rodger, MD, MSc
Ottawa Hospital
Ottawa, ON, Canada
Who Should Get Long-Term Therapy and With What?

Walter Ageno, MD
University of Insubria
Varese, Italy
Breadth of Complications (Including Financial) of Long-Term Oral Anticoagulant Care

Leslie Skeith, MD
University of Calgary
Calgary, AB, Canada
Anticoagulating Patients With High-Risk Acquired Thrombophilias: APS, HIT, and PNH

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Severe Aplastic Anemia: Diagnosis and Management in an Era of Effective Therapies


Significant progress has recently been made in the field of acquired severe aplastic anemia (AA). This session will discuss clonal hematopoiesis and its prognostic significance, both at diagnosis and during follow up. This session will also provide updates on recent trials using the triple combination of anti-thymocyte globulin, cyclosporin and eltrombopag. Finally, the session will demonstrate that transplants from donors other than human leukocyte antigen (HLA)-identical siblings are increasingly used worldwide and result in very encouraging cure rates.

Dr. Daria Babushok will summarize the latest data on clonal hematopoiesis in acquired AA, with an emphasis on the relationship between the immune physiology of AA and the prototypical somatic changes in the bone marrow of patients with AA. Recent data on somatic loss of HLA alleles in AA and on the characteristics of post-AA myelodysplastic syndromes will be discussed. Finally, Dr. Babushok will present a practical approach for incorporating studies of clonal hematopoiesis into a diagnostic evaluation of a patient presenting with suspected AA, as well as the current data and remaining uncertainties on the prognostic significance of somatic mutations in AA.

Dr. Phillip Scheinberg will review the development of eltrombopag as a single agent in patients with severe AA (SAA) refractory to initial immunosuppressive therapy (IST). These results will be placed in context of overall SAA management and how these findings are being applied to current treatment algorithms. Continued development on the combination of eltrombopag and IST in frontline management will be presented and longer-term results will be discussed. Insights into the mechanism of action of eltrombopag in SAA will be presented. These data will be placed in context of other treatment approaches in SAA.

Dr. Andrea Bacigalupo will analyze data of allogeneic hemopoietic stem cell transplantation from donors other than HLA identical siblings. This will include transplants from unrelated donors, HLA haploidentical family members, and unrelated cord blood units. Indications, age limits, transplant platforms, and prognostic factors will be discussed.

Chair:

Andrea Bacigalupo, MD
Gemelli University Hospital
Rome, Italy

Speakers:

Phillip Scheinberg, MD
Hospital A Beneficencia Portuguesa
São Paulo, Brazil
Eltrombopag or Other Therapies Added to Immunosuppression for Treatment-Naïve Severe Aplastic Anemia

Daria V. Babushok, MD, PhD
University of Pennsylvania
Philadelphia, PA
A Brief But Comprehensive Guide to Clonal Evolution in Aplastic Anemia

Andrea Bacigalupo, MD
Gemelli University Hospital
Rome, Italy
Alternative Donor Transplants for Aplastic Anemia

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Sickle Cell Disease: New Frontiers


Recent progress in genetic epidemiology, molecular diagnostics, and targeted therapeutics has led to a surge of new knowledge and developments in sickle cell disease (SCD) and sickle cell trait. This session will focus on recent advances in the understanding of clinical implications of sickle cell trait, as well as promising diagnostic approaches in clinical care and research of SCD. Lastly, this session will review exciting progress in the development of therapeutics for SCD.

Dr. Rakhi Naik will review the current landscape of research in clinical complications of sickle cell trait, including exertion-related injury, chronic kidney disease, and venous thromboembolism. She will discuss the role of epidemiology and precision medicine in evaluating the risk of complications in individuals with sickle cell trait. Dr. Naik will also highlight opportunities for further discovery and guideline development in this emerging field.

Dr. Enrico Novelli will summarize existing evidence and promising data on biomarkers for clinical care and research in SCD. He will discuss pitfalls in biomarker development and the recent progress in molecular and imaging diagnostics in the new era of personalized medicine. Dr. Novelli will also review how these modalities can be applied to a precision medicine approach for both the care of patients and in the design of clinical trials in SCD.

Dr. Deepa Manwani will discuss the considerable progress in the development of treatments for SCD. She will outline the complex pathophysiology of SCD, including blood cell adhesion, inflammation and hemostatic system activation, as potential targets for therapeutics. Dr. Manwani will also highlight the challenges in selection of study end points in the development of novel therapeutic strategies in SCD.

Chair:

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

Speakers:

Rakhi P. Naik, MD
Johns Hopkins University
Baltimore, MD
The Current State of Sickle Cell Trait

Enrico Novelli, MD
University of Pittsburgh
Pittsburgh, PA
Measuring Success: Utility of Biomarkers in Sickle Cell Disease Clinical Trials and Care

Deepa Manwani, MD
Children's Hospital at Montefiore
Bronx, NY
New Insights Into the Pathophysiology and Development of Novel Therapies for Sickle Cell Disease

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Thalassemia Syndromes: Diagnosis and Management in a Changing World


The management of patients with alpha and beta thalassaemia has been improving over the past 30 years. This session will review our understanding of the factors which determine whether patients require regular blood transfusion, how to reduce transfusion requirements, and the role of bone marrow transplantation and gene therapy in the future management of patients with these common forms of inherited anemia.

Dr. Douglas Higgs will discuss new observations made on individuals with the Hemoglobin (Hb) Bart’s Hydrops Fetalis Syndrome (BHFS), the most severe form of a-thalassaemia. BHFS is most often seen in patients from Southeast Asia (SEA) who are homozygotes for the common z SEA/haplotype and who inherit no a globin genes. Untreated, BHFS is inevitably fatal and affected infants die of a severe anemia. Over the past 30 years, there has been an increasing number of reports describing infants who have been treated with intra-uterine transfusion, or transfusion at the time of birth and who have survived. While the overwhelming priority with this disease is prevention, Dr. Higgs will discuss how it may be possible to re-activate the embryonic z gene, which is normally fully silenced after 12 weeks of gestation, and thereby ameliorate the anemia in affected individuals.

Dr. Vip Viprakasit will summarize the complexity of clinical phenotypes found in beta thalassemia using Hb E/beta-thalassemia and others as his model. Due to a recent classification of thalassemia syndromes into transfusion-dependent (TD) versus non-transfusion-dependent (NTD), Dr. Viprakasit will review the key challenges facing clinicians on how to define the correct classification and its impact on clinical management. Finally, he will present his latest data using genomic approaches to demonstrate that the complicated genotype-phenotype correlation in thalassemia is beyond globin genes and their regulation.

Dr. John Porter will present the state of the art in clinical trials using new therapies for TD and NTD thalassemias.

Chair:

Douglas Higgs, DSc
MRC Weatherall Institute of Molecular Medicine
Oxford, United Kingdom

Speakers:

Vip Viprakasit, MD, DPhil
Siriraj Hospital, Mahidol University
Bangkok, Thailand
Natural History and Phenotypic Heterogeneity in Hemoglobin E Beta-thalassemia Worldwide

Douglas Higgs, DSc
MRC Weatherall Institute of Molecular Medicine
Oxford, United Kingdom
Genetic Variations and Role of Modifiers in E Thalassemia and Other Syndromes

John Porter, MA MD FRCP FRCPath
University College London
London, United Kingdom
Beyond Transfusion Therapy: New Therapies in Thalassemia, Including Drugs, Alternate Donor Transplant, and Gene Therapy

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The Interface Between Man and Machine: Managing Hemostasis and Thrombosis in the Plastic and Metal Circulation


Patient survival during open heart surgery and with severe heart failure is possible because of cardiopulmonary bypass and mechanical circulatory support (MCS) devices. However, thrombotic and bleeding complications are common. This session will provide an overview of MCS, discuss point-of-care testing to monitor anticoagulation, and outline prevention and management strategies for hemorrhage.

Dr. Lisa Baumann Kreuziger will translate the world of MCS to the practicing hematologist through an overview of MCS devices used in adults and children and the alterations to the coagulation system caused by MCS. She will also discuss how to prevent, diagnose, and manage thrombosis during extracorporeal membrane oxygenation and ventricular assist device support.

Dr. Rachel Bercovitz will discuss common point-of-care tests that can provide real-time information to clinicians to optimize the management of their use of anticoagulants and hemostatic therapies for patients on extracorporeal circuits, including extracorporeal membrane oxygenation, cardiopulmonary bypass, and ventricular assist devices. She will compare and contrast the different assays, including activated clotting time, thromboelastometry, and whole blood aggregometry, and summarize evidence regarding their use in clinical care. Dr. Bercovitz will also highlight the gaps in our current knowledge and areas for future investigation.

Patients requiring extracorporeal circulation are at increased risk for catastrophic bleeding due to a combination of pre-existing risk factors, anticoagulant therapy, and the deleterious effects of the mechanical circuit on the hemostatic system. Dr. Keyvan Karkouti will discuss a targeted algorithmic approach for the prevention and management of catastrophic bleeding with a focus on cardiopulmonary bypass.

Chair:

Lisa Baumann Kreuziger, MD, MS
BloodCenter of Wisconsin
Milwaukee, WI

Speakers:

Lisa Baumann Kreuziger, MD, MS
BloodCenter of Wisconsin
Milwaukee, WI
The Adult and Pediatric Mechanical Circulation: A Guide for the Hematologist

Rachel Bercovitz, MD
Northwestern University
Chicago, IL
An Introduction to Point-of-Care Testing in Extracorporeal Circulation and LVADs

Keyvan Karkouti, MD
Toronto General Hospital
Toronto, ON, Canada
Preventing and Managing Catastrophic Bleeding During Extracorporeal Circulation

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Thrombotic Thrombocytopenic Purpura (TTP) 2019: State of the Art


Recent years have seen dramatic advances in our understanding of the pathophysiology and clinical manifestations of thrombotic thrombocytopenic purpura (TTP), as well as its diagnosis and management. In this session, an integrated approach to the clinical and laboratory diagnosis of TTP will be presented. Current and emerging therapies for TTP and long-term sequelae of TTP will also be reviewed.

Dr. Adam Cuker will discuss the epidemiology of TTP, its clinical presentation, and its differentiation from other forms of thrombotic microangiopathy. He will also review the role of ADAMTS13 testing in the diagnosis of TTP and the clinical implications of ADAMTS13 deficiency and inhibitors to ADAMTS13.

Dr. Shruti Chaturvedi will discuss evolving paradigms of TTP management with a focus on novel targeted therapies. She will review the clinical data supporting rituximab as an adjunct to plasma exchange as upfront therapy for acute TTP, for refractory TTP, and as monotherapy for relapse prevention. Dr. Chaturvedi will discuss recent clinical trial data on caplacizumab and will briefly touch upon the other targeted therapies including bortezomib and recombinant ADAMTS13 and discuss their potential role in clinical care. She will summarize the available evidence and discuss strategies for optimal management of TTP during an acute episode and in remission.

Although risk for relapse may be the greatest concern following recovery from an initial episode of acquired autoimmune TTP, Dr. James George will discuss other major health issues that occur with increased frequency in this population and must be recognized and appropriately addressed. These include depression, mild cognitive impairment, development of other autoimmune disorders (e.g., systemic lupus erythematosus), hypertension, and pregnancy complications. He will also discuss survival, which is shortened following recovery from an acute episode of TTP.

Chair:

Adam Cuker, MD, MS
University of Pennsylvania
Philadelphia, PA

Speakers:

Adam Cuker, MD, MS
University of Pennsylvania
Philadelphia, PA
Clinical and Laboratory Diagnosis of TTP: An Integrated Approach

Shruti Chaturvedi, MBBS,MS
Johns Hopkins University
Baltimore, MD
TTP Beyond Plasma Exchange

James George, MD
University of Oklahoma Health Sciences Center
Oklahoma City, OK
TTP: More Than an Acute Disease

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What's Hot in Immune Thrombocytopenic Purpura (ITP)?


The diagnosis of immune thrombocytopenia (ITP) is difficult because of the many underlying causes that can lead to thrombocytopenia. This session will focus on the diagnostic standards and guidelines for ITP, as well as the challenges physicians face in establishing the diagnosis of ITP. Two important causes of thrombocytopenia, drug-induced ITP (DITP) and heparin-induced thrombocytopenia (HIT), will also be discussed.

Dr. John G. Kelton will review the various approaches to the diagnosis of ITP and address parallels between autoimmune hemolytic anemia and ITP. He will discuss the clinical and laboratory diagnosis of ITP, with an emphasis on anti-platelet glycoprotein-specific antibody assays. Recent results of autoantibody testing in ITP will be discussed.

Dr. Cindy Neunert will provide an update on evidence-based guidelines for ITP. During this talk, she will review the current and historical guideline process and update the audience on current recommendations for the management of ITP in adults and children. Areas lacking evidence will be reviewed, and research priorities will be highlighted.

Dr. Tamam Bakchoul will discuss drug-induced ITP, including HIT. The diagnosis of DITP is often challenging since most hospitalized patients take multiple medications and have comorbidities that can also cause thrombocytopenia. Dr. Bakchoul will provide an update on the pathophysiology, diagnosis and management of DITP and HIT.

Chair:

John G. Kelton, MD
McMaster University
Hamilton, ON, Canada

Speakers:

John G. Kelton, MD
McMaster University
Hamilton, ON, Canada
How Do We Diagnose ITP in 2018?

Cindy Neunert, MD
Columbia University
New York, NY
Evidence-Based Management of ITP: ASH Guideline Update

Tamam Bakchoul, MD, PhD
Eberhard Karls University
Tuebingen, Germany
Drug-Associated Thrombocytopenia

Other Education Sessions

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