The 2011 Education Program will be held Saturday, December 10, and Sunday, December 11. Each session will be offered twice unless otherwise noted. Chapters based on these sessions will be published in the Education Program Book, Hematology 2011. In addition, audio recordings and slides from Education Program presentations will be available on DVD and webcasts.

• Acquired Hematopoietic Disorders: Complement-Mediated Blood Disorders
• Acute Myeloid Leukemia in the Age of Genomics
• Antiplatelet Therapy
• Aplastic Anemia: Bone Marrow Failure
• Chronic Lymphocytic Leukemia: From Biology to Targeted Therapy
• Chronic Myeloid Leukemia: Managing a Chronic Disease
• Consultative Hematology I: Common Questions in Thrombosis Consults
• Consultative Hematology II: Difficult Pediatric Consultations
• Controversies and Updates in Multiple Myeloma
• Current Issues in Myeloproliferative Neoplasms
• Current Management Issues in Acute Lymphocytic Leukemia
• Diagnosis and Management of Myeloproliferative Neoplasm Variants
• Grant Writing for Junior Faculty: Biomedical Research Funding
• Hematopoietic Stem Cell Transplantation I: Transplant in Benign Hematology
• Hematopoietic Stem Cell Transplantation II: Controversies and Challenges in HSCT
• Hodgkin Lymphoma
• Junior Faculty Development Program: Mentorship: How to Receive It, How to Provide It, How to Optimize It
• Laboratory Medicine: Genetics of Lymphoid Neoplasms: What You Need To Know For Your Current (and Future) Daily Clinical Practice
• Pediatric Malignancies: Clinical Implications of Advances in Leukemia Biology
• Platelet Drugs and Disorders
• Possible Game-Changers in Hemophilia
• Recent High-Impact Clinical Trials in Sickle Cell Disease: Where Do They Leave the Field?
• Strategies for Optimal Management in Thalassemia - Now and In the Future
• Transfusion Medicine I: Adverse Complications of Stored Blood
• Transfusion Medicine II: Hematology and Transfusion Medicine in Sub-Saharan Africa
• Understanding and Management of Inherited Bone Marrow Failure Syndromes
• Update on Aggressive Lymphomas - New Agents and Current Management
• Updates on Disorders of Iron Utilization and Distribution
• Updates on Treatment Strategies in Myelodysplastic Syndromes

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Acquired Hematopoietic Disorders: Complement-Mediated Blood Disorders

Is it possible to have a concurrent working knowledge of both the coagulation and complement systems? Most hematologists would say no. This education session is not intended to displace the coagulation system from memory, but rather to invigorate interest in an arcane but fascinating area of hematology by highlighting exciting new advances in understanding the genetics, pathophysiology, and therapy of complement-mediated blood disorders. Dr. John Atkinson will discuss the pathophysiology of hemolytic uremic syndrome and related thrombomicroangiopathies. The concept will be espoused that renal endothelial cell stress/injury in an individual who is heterozygous for a dysfunctional genetic variant (haploinsufficiency) leads to overactivity of an ancient, innate immune system known as the alternative complement pathway. A procoagulant environment is thus created that predisposes to the formation of clots in the microvasculature. Dr. Timothy Goodship will discuss the inherited and acquired factors that predispose to development of atypical hemolytic uremia syndrome, current recommendations for investigation of the disease, and recent developments in its management. Dr. Charles Parker's lecture will focus on paroxysmal nocturnal hemoglobinuria (PNH) with an emphasis on disease management. The complement inhibitor eculizumab has had a resounding effect on the natural history of PNH by providing a safe, effective therapeutic mechanism for inhibiting intravascular hemolysis, but owing to the heterogeneous nature of the disease that is further complicated by elements of bone failure and thrombophilia, management remains complex. Dr. Parker will present an overview of disease classification that provides a framework for individualizing treatment of patients with PNH.

Chair(s):

Charles J. Parker, MD
University of Utah School of Medicine
Salt Lake City, UT, USA

Speaker(s):

John P. Atkinson, MD
Washington University School of Medicine 
St. Louis, MO, USA
Too Much of a Good Thing At a Site of Tissue Injury: The Instructive Example of Endothelial Cells, the Complement System and Blood Clots In Small Blood Vessels

Timothy H. J. Goodship, MD
Newcastle University 
Newcastle upon Tyne, England
Atypical Hemolytic Uremic Syndrome, Genetic Basis and Clinical Manifestations

Charles J. Parker, MD
University of Utah School of Medicine 
Salt Lake City, UT, USA
Still Confusing After All These Years: Management of Paroxysmal Nocturnal Hemoglobinuria In the Era of Complement Inhibitory Therapy

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Acute Myeloid Leukemia in the Age of Genomics

Recent technological advances, such as whole genome sequencing, have revolutionized our ability to analyze cancer cells at a genome-wide level. In particular, genomic studies in acute myeloid leukemia (AML) are providing major new insights into AML biology. This session will focus on the impact of genomic studies in AML and their application to the management of patients with AML, including their use in treatment decisions and the development of novel therapies. Dr. Daniel Link will review emerging technologies that are being used to analyze cancer genomes. He will summarize major recent findings in AML, with an emphasis on whole genome sequencing. Dr. Link will also discuss the current and potential future clinical application of these studies in AML. Dr. Hartmut Döhner will review the genetic alterations in AML, with an emphasis on recent mutations identified by whole genome sequencing. He will then discuss the impact of these genetic alterations on prognosis and their role in directing treatment decisions, including allogeneic transplantation. Dr. Gail Roboz will review emerging therapeutic approaches in AML. She will discuss new strategies in induction, consolidation, and non-transplant postremission treatment. Dr. Roboz will also discuss novel agents and combinations. Finally, she will discuss the rapidly evolving areas of treating minimal residual disease and targeting leukemia stem cells in AML.

Chair(s):

Daniel C. Link, MD
Washington University
St. Louis, MO, USA

Speaker(s):

Daniel C. Link, MD
Washington University 
St. Louis, MO, USA
Genomics of AML: Clinical Applications

Hartmut Döhner, MD
University of Ulm 
Ulm, Germany
Impact of Genetic Features on Treatment Decisions in AML

Gail J. Roboz, MD
Weill Medical College of Cornell University 
New York, NY, USA
Novel Approaches to the Treatment of AML

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Antiplatelet Therapy

This session will focus on the role of platelets in atherothrombosis, recent advances in antiplatelet therapy, and the clinical relevance of response variability to antiplatelet therapy. Dr. Shaun Jackson will review the role of platelets in atherothrombosis. The discussion will focus on the role of platelets in enhancing leukocyte interactions with the vessel wall, leading to enhanced "thrombo-inflammatory" responses. Dr. Alan Michelson will discuss the benefits and limitations of currently available and investigational antiplatelet therapy. The discussion will focus on novel P2Y12 antagonists (prasugrel, ticagrelor, cangrelor, elinogrel) and protease-activated receptor 1 (PAR1) antagonists. Dr. Marco Cattaneo will discuss the clinical relevance of response variability to antiplatelet therapy, including aspirin and clopidogrel. The discussion will focus on the magnitude, causes, clinical consequences, and management, if any, of response variability to antiplatelet therapy.

Chair(s):

Alan D. Michelson, MD
Children's Hospital Boston, Dana-Farber Cancer Institute, Harvard Medical School
Boston, MA, USA

Speaker(s):

Shaun P Jackson, PhD
Monash University 
Prahran, Australia
Role of Platelets in Atherogenesis

Alan D. Michelson, MD
Children's Hospital Boston, Harvard Medical School 
Boston, MA, USA
Advances in Antiplatelet Therapy

Marco Cattaneo, MD
University of Milan, Azienda Ospedaliera San Paolo 
Milan, Italy
The Clinical Relevance of Response Variability to Antiplatelet Therapy

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Aplastic Anemia: Bone Marrow Failure

Short- and long-term outcome for young patients with aplastic anemia (AA) has improved greatly over the past decade, most notably due to improvement in supportive care, bone marrow transplantation (BMT), and clinical trials for immunosuppressive therapy (IST). Reflecting the immune-mediated nature of the disease in most patients, pretreatment clinical and laboratory findings influencing response to IST have recently been identified. Comprehensive diagnostic guidelines, including histopathology and diagnostic tests to rule out inherited bone marrow failure disorders and low-grade myelodysplastic syndrome (MDS), are required to further rationalize therapy in individual patients. Dr. Eva Guinan will discuss the diagnostic procedure and management in AA. Indications for matched sibling- and alternative-donor BMT, IST with cyclosporine and antithymocyte globulin, and standards of supportive care will be outlined. Dr. Charlotte Niemeyer will review the classification of AA and MDS in children. The clinical and morphological criteria for diagnosing acquired AA, refractory cytopenia of childhood, or inherited bone marrow failure disorders will be explored. The implications for patient management and therapeutic options will also be considered. Dr. Jaroslaw Maciejewski will discuss clonal evolution in AA. Array-based DNA technologies can allow the detection of unbalanced genomic aberrations and thus are likely to improve diagnostic precision and better distinction of hypocellular bone marrow disorders.

Chair(s):

Charlotte M. Niemeyer, MD
Children's Hospital, University of Freiburg
Freiburg, Germany

Speaker(s):

Eva C. Guinan, MD
Dana-Farber Cancer Institute 
Boston, MA, USA
Diagnosis and Management of Aplastic Anemia

Charlotte M. Niemeyer, MD
Children's Hospital, University of Freiburg 
Freiburg, Germany
Classification of Childhood Aplastic Anemia/MDS

Jaroslaw P. Maciejewski, MD, PhD
Cleveland Clinic 
Cleveland, OH, USA
Clonal Evolution in Aplastic Anemia

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Chronic Lymphocytic Leukemia: From Biology to Targeted Therapy

This session will focus on recent advances in our understanding of the pathophysiology of chronic lymphocytic leukemia (CLL) that have revealed new insights into the development and proliferation of the disease as well as the common mechanisms of resistance to conventional therapy. Dr. Jan Burger will review the role of the interaction between the microenvironment and the leukemic cell and how signaling through the B-cell receptor pathway, among others, leads to the proliferation of CLL cells and the progression of the disease. He will also review the down-stream signaling after the B-cell receptor engagement and how this stimulus is transmitted into a proliferative signal. Dr. Peter Hillmen will review the use of conventional molecular markers, such as fluorescent in situ hybridization (FISH) and immunoglobulin mutation analysis, to influence treatment decisions. He will review how this information is being used to stratify patients into different treatment approaches and will introduce the concept that understanding the pathophysiology of CLL is now leading to novel therapies with extremely promising efficacy, even in the most refractory patients. Dr. Jennifer Brown will review the current approaches to the management of patients with CLL who have failed to respond to conventional immunochemotherapy or have relapsed soon after receiving it. Dr. Brown will also discuss the role of conventional therapies, such as alemtuzumab, the increasing evidence and use of allogeneic stem cell transplant in these patients, and the promise of the new wave of novel therapies currently in trials for CLL.

Chair(s):

Peter Hillmen, MBChB, PhD
St. James's University Hospital
Leeds, United Kingdom

Speaker(s):

Jan A. Burger, MD, PhD
The University of Texas MD Anderson Cancer Center; Leukemia Department 
Houston, TX, USA
CLL and the Micro-Environment

Peter Hillmen, MBChB, PhD
St. James's University Hospital 
Leeds, United Kingdom
Using the Biology of CLL to Choose Treatment

Jennifer R Brown, MD, PhD
Dana Farber Cancer Institute 
Boston, MA, USA
The Treatment of Relapsed and Refractory CLL

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Chronic Myeloid Leukemia: Managing a Chronic Disease

The nature of chronic myeloid leukemia (CML) has changed dramatically in the last decade. With the advent of targeted tyrosine kinase inhibitors (TKIs), it was transformed from an invariably fatal disease in its natural evolution to one compatible with normal life expectancy if patients comply with daily oral therapy. Although CML is a relatively rare malignancy, effective treatment has significantly changed its prevalence and raised new problems of management. In fact, for an increasingly large population of patients, CML has become a chronic illness, like hypertension, diabetes, or AIDS. Dr. Neil Shah will describe definitions of response and impact of response on outcomes, and update data on TKIs employed in newly diagnosed patients and those with resistance to first-line therapy. Common causes of resistance will be discussed, as well as factors that may affect the choice of preferred second-line treatment. Dr. Andreas Hochhaus will discuss CML as a chronic disease and will focus on the management of treatment-related adverse effects, considering the favorable data on survival. Topics to be discussed include adherence to therapy, pharmacoeconomic parameters, CML and pregnancy, implementation of harmonized surveillance strategies, and cooperation with patient advocacy groups. Dr. Junia Melo will address issues associated with the possibility of safely discontinuing TKI treatment for patients in continuous "remission," how to identify those who will benefit from such discontinuation, and how to monitor these patients. She will speculate on the possibility that CML can now be "cured" and will discuss alternative definitions or expectations for a "cure" in this type of disease.

Chair(s):

Junia V. Melo, MD, PhD
Centre for Cancer Biology
Adelaide, Australia

Speaker(s):

Neil Shah, MD, PhD
University of California San Francisco 
San Francisco, CA, USA
Optimal Front-Line Therapy and Options for Patients Resistant to First-Line TKI Therapy

Andreas Hochhaus, MD
Universitätsklinikum Jena 
Jena, Germany
Managing CML as a Chronic Disease

Junia V. Melo, MD, PhD
Centre for Cancer Biology 
Adelaide, Australia
Minimal Residual Disease and Discontinuation of Therapy: Can We Aim at a Cure?

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Consultative Hematology I: Common Questions in Thrombosis Consults

Dr. Theodore Warkentin will review the detection of platelet-activating anti-PF4/heparin antibodies and will discuss pitfalls leading to heparin-induced thrombocytopenia (HIT) "overdiagnosis." Since only approximately 10 percent of patients who undergo serologic investigation actually have HIT, management strategies should be appropriate for both HIT and non-HIT thrombocytopenia. Dr. Warkentin will describe his personal management strategy, which emphasizes use of indirect factor Xa inhibitors (danaparoid, fondaparinux), based upon safety/efficacy, availability of prophylactic- and therapeuticdose regimens, and avoidance of problems with PT/PTT monitoring. Thrombophilia testing identifies abnormalities in about 50 percent of patients with venous thromboembolism. Dr. Saskia Middeldorp will discuss the association of thrombophilia with thrombosis and pregnancy complications, and she will assess benefits of testing in terms of numbers needed to test to prevent negative clinical outcomes. Dr. Middeldorp will present evidence that testing for hereditary thrombophilia generally does not alter the clinical management of patients with venous or arterial thrombosis or pregnancy complications, and that informed decisions can be made based on current knowledge. The post-thrombotic syndrome (PTS) impairs quality of life for 25 percent to 50 percent of patients with deep-vein thrombosis. Dr. Suresh Vedantham will discuss the limitations of standard therapy (anticoagulation and elastic compression stockings) for prevention of PTS. He will review recent advances in catheter-directed thrombolysis and summarize the evidence that these procedures reduce the incidence of PTS. He will describe high-risk conditions in which endovascular procedures may be indicated for first-line therapy of acute deep-vein thrombosis and will discuss the use of salvage procedures such as iliac vein stenting to treat patients with severe established PTS.

Chair(s):

Douglas M. Tollefsen, MD, PhD
Washington University Medical School
St. Louis, MO, USA

Speaker(s):

Theodore E. Warkentin, MD
Michael G. DeGroote School of Medicine, McMaster University 
Hamilton, ON, Canada
How I Diagnose and Manage Heparin-Induced Thrombocytopenia

Saskia Middeldorp, MD
Academic Medical Center, University of Amsterdam 
Amsterdam, Netherlands
Is Thrombophilia Testing Useful?

Suresh Vedantham, MD
Washington University School of Medicine 
St. Louis, MO, USA
Endovascular Procedures In the Management of Deep-Vein Thrombosis

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Consultative Hematology II: Difficult Pediatric Consultations

This session will focus on three separate areas that are difficult hematologic consultations in pediatrics. Dr. Brian Wicklund will review the pathophysiology of bleeding and thrombosis associated with liver disease in pediatric patients. He will review the diagnostic evaluation of these conditions and the available data on treatment to either allow diagnostic or surgical procedures to be done or to control bleeding episodes. This will include the use of standard blood component therapy and recombinant factor VIIa. Dr. Cliff Takemoto will discuss the challenges to the diagnosis and management of heparin-induced thrombocytopenia in children. He will review the available data on the epidemiology in pediatric patients and discuss the use of laboratory testing and clinical scoring models for diagnosis. Dr. Takemoto will also describe approaches to management and review the clinical experience with direct thrombin inhibitors in children. Dr. Sheila Weitzman will briefly review the underlying genetic and secondary causes of hemophagocytic lymphohistiocytosis (HLH), when to suspect this diagnosis, the changing criteria used to make the diagnosis, and practical clinical and laboratory findings that are helpful in distinguishing HLH from other conditions. This will include commonly asked questions with case histories that illustrate important points in management.

Chair(s):

Brian M. Wicklund, MDCM
Children's Mercy Hospital
Kansas City, MO, USA

Speaker(s):

Brian M. Wicklund, MDCM
Children's Mercy Hospital 
Kansas City, MO, USA
Bleeding and Clotting Disorders in Pediatric Liver Disease Patients

Clifford M. Takemoto, MD
The Johns Hopkins University 
Baltimore, MD, USA
Heparin-Induced Thrombocytopenia Screening and Management in Pediatrics

Sheila Weitzman, MD
The Hospital for Sick Children 
Toronto, ON, Canada
Diagnostic Evaluation of Suspected Hemophagocytic Disorders

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Controversies and Updates in Multiple Myeloma

This session will describe oncogenomic studies and model systems of myeloma in the bone marrow microenvironment, which have allowed for identification of current therapeutic targets as well as validation of targeted therapies. Advances in genomic profiling to identify signatures of response, develop next-generation targeted therapies, inform scientifically based combination therapies, and personalize immune therapies will be discussed. The speakers will show how novel therapies have been integrated into the high-dose therapy and stem cell transplant paradigm in myeloma as induction, consolidation, and maintenance therapy to achieve unprecedented rates and durability of response. Given the high extent and frequency of response observed, the role of transplantation in the era of combination novel therapies will be described, including both autologous and allogeneic stem cell transplantation. Finally, as patients achieve sustained responses to novel therapies and live longer, adverse events associated with novel therapies may be observed. In this context, the significance of secondary cancers observed in myeloma patients receiving novel therapies will be critically examined. This session will therefore characterize bench-to-bedside progress in targeted therapies to date and, importantly, outline how future treatments for myeloma will be affected by these scientific advances.

Chair(s):

Kenneth C. Anderson, MD
Dana-Farber Cancer Institute
Boston, MA, USA

Speaker(s):

Kenneth C. Anderson, MD
Dana-Farber Cancer Institute 
Boston, MA, USA
New Insights Into Therapeutic Targets in Myeloma

Sergio Giralt, MD
Memorial Sloan-Kettering Cancer Center 
New York, NY, USA
Optimal Role for Bone Marrow Transplant

Donna E. Reece, MD, FRCPC
Princess Margaret Hospital 
Toronto, ON, Canada
Post-Transplant Maintenace Therapy in Myeloma/Optimal Frontline Therapy

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Current Issues in Myeloproliferative Neoplasms

This session will focus initially on the molecular genetics of myeloproliferative neoplasms (MPNs) and then on the diagnosis, stratification, and clinical management of patients with essential thrombocythemia (ET) and myelofibrosis (MF). Dr. Nicholas Cross will review the central role of abnormalities that impact on JAK2 signaling in MPNs and will discuss other somatically acquired mutations that impact on the epigenetic control of gene expression and other cellular processes. Dr. Cross will also review recent progress in our understanding of inherited variants that predispose to development of disease and discuss current areas of scientific controversy. Dr. Francisco Cervantes will briefly review the molecular basis and diagnostic criteria of ET and its clinical aspects, primarily the vascular complications and their pathogenesis. As ET usually affects patients' quality of life rather than survival, emphasis will be made on the importance of a risk-adapted treatment strategy. Current treatment options for ET will be discussed, as well as the results of newer therapies such as pegylated interferon and JAK2 inhibitors. Dr. Alessandro Vannucchi will briefly review the diagnostic criteria for primary myelofibrosis and post-PV/post-ET myelofibrosis, and will then focus on established criteria for patients’ risk stratification as well as on novel biomarkers associated with prognosis. Conventional treatment options will be presented in a patient's need-oriented approach, while results of novel therapies, including JAK1 and JAK2 inhibitors, immunomodulators, and other agents, will be critically discussed.

Chair(s):

Nicholas C.P. Cross, PhD
University of Southampton
Southamptom, United Kingdom

Speaker(s):

Nicholas C.P. Cross, PhD
University of Southampton 
Southamptom, United Kingdom
Genetic and Epigenetic Complexity in Myeloproliferative Neoplasms

Francisco Cervantes, MD, PhD
Hospital Clínic 
Barcelona, Spain
Management of Essential Thrombocythemia

Alessandro M. Vannucchi, MD
University of Florence 
Florence, Italy
Management of Myelofibrosis

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Current Management Issues in Acute Lymphocytic Leukemia

Acute lymphocytic leukemia (ALL) affects children more commonly than adults. For affected individuals of any age, treatment is long and complex and brings about considerable acute and chronic toxicity. Successive intensifications of therapy have now resulted in long-term, disease-free survival - equated to cure - as the norm for children with ALL. For many pediatric hematologists, dealing with long-term treatment-related morbidity is now as pressing an issue as that of successfully treating the underlying disease. For adults, in whom disease-free survival has yet to emulate that seen in children, effective novel approaches that do not recapitulate the acute toxicities of currently available multi-agent chemotherapy are still eagerly sought. However, timely evaluation of their real contribution to outcome is not always straightforward, due to the relative rarity of the disease. In this session, the speakers will touch upon the topical issue of the balance between effectiveness and toxicity of ALL therapy in three ways. Dr. Leslie Robison will examine the impact of long-term toxicities of treatment in younger patients and discuss strategies for minimizing their impact; Dr. Dieter Hoelzer will evaluate the evidence for if, how, and when monoclonal antibodies should be added to our current therapeutic strategies for ALL to provide additional benefit; and Dr. Adele Fielding will discuss the management of Philadelphia-chromosomepositive ALL with particular reference to whether tyrosine kinase inhibitors (TKIs) have rendered any of our current approaches to therapy dispensable.

Chair(s):

Adele Fielding, MB BS, PhD
University College London
London, United Kingdom

Speaker(s):

Leslie L. Robison, PhD
St. Jude Children's Research Hospital 
Memphis, TN, USA
Late Effects of ALL Therapy In the Young

Dieter Hoelzer, MD PhD
Goethe University Hospital 
Frankfurt, Germany
New Antibody Therapies in ALL

Adele Fielding, MB BS, PhD
University College London 
London, United Kingdom
(Ph+) ALL Therapy

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Diagnosis and Management of Myeloproliferative Neoplasm Variants

This session will focus on recent advances in our understanding of the molecular basis and clinical management of myeloproliferative neoplasm variants, i.e., myeloproliferative disorders other than chronic myeloid leukemia, polycythemia vera, essential thrombocythemia, and primary myelofibrosis. Dr. James Vardiman will introduce the session by discussing the rationale for the World Health Organization (WHO) classification of the myelodysplastic/myeloproliferative neoplasms and of the variant neoplasms with abnormalities of PDGFRA, PDGFRB, and FGFR1 that are associated with a prominent eosinophil component. The discussion will illustrate how, in some cases, genetic abnormalities have influenced the WHO nomenclature and classification of neoplasms, particularly when genetic information helps to define specific entities for which targeted therapy may be available. Dr. Amy Klion will discuss myeloid neoplasms and myeloproliferative disorders whose presentations overlap with those of hypereosinophilic syndrome. These disorders include chronic eosinophilic leukemia (CEL) associated with PDGFRA rearrangement and D816KIT-positive systemic mastocytosis with eosinophilia. Dr. Mario Cazzola will examine clonal myeloid neoplasms that, at the time of initial presentation, have some clinical, laboratory, or morphologic findings that support a diagnosis of myelodysplastic syndrome, and other findings more consistent with myeloproliferative neoplasm. These disorders include chronic myelomonocytic leukemia (CMML), atypical chronic myeloid leukemia (aCML, BCRABL1 negative), and refractory anemia with ringed sideroblasts associated with marked thrombocytosis (RARS-T).

Chair(s):

Mario Cazzola, MD
Fondazione IRCCS Policlinico San Matteo, University of Pavia
Pavia, Italy

Speaker(s):

James W. Vardiman, MD
University of Chicago 
Chicago, IL, USA
World Health Organization Classification and Genetics

Amy D. Klion, MD
National Institute of Allergy and Infectious Diseases, National Institutes of Health 
Bethesda, MD, USA
Eosinophilic Myeloproliferative Neoplasms

Mario Cazzola, MD
Fondazione IRCCS Policlinico San Matteo, University of Pavia 
Pavia, Italy
Myelodysplastic/Myeloproliferative Neoplasms

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Grant Writing for Junior Faculty: Biomedical Research Funding

THIS SESSION IS OFFERED ONCE. This session will address approaches to obtaining research funding, with a focus on providing tips for junior investigators. The session will cover tapping into philanthropic sources, funding from disease-oriented foundations, effective use of the new, short format for National Institutes of Health (NIH) grants, and how to write a career-development plan. Dr. Donald Feinstein will discuss obtaining financial support from grateful patients, funding from disease-specific organizations, such as the Leukemia & Lymphoma Society, the Myelodysplastic Syndromes (MDS) Foundation, and the Multiple Myeloma Research Foundation, and research support from other charitable foundations and internal funding unique to the individual's institution. Dr. Peter Klein will discuss how the new NIH funding format changes the approach to grant writing, while offering tips on organization and presentation of NIH grant applications and providing insights into how the study section reviews grant applications in the context of the new format. Award applicants to NIH and other funding organizations may have excellent research proposals but can stand or fall by their career-development plans. Dr. Beverly Mitchell will share insights into how to write a compelling career-development plan.

Chair(s):

Peter S. Klein, MD, PhD
University of Pennsylvania
Philadelphia, PA, USA

Speaker(s):

Donald I. Feinstein, MD
Keck School of Medicine of the University Of Southern California 
Los Angeles, CA, USA
Funding Through Philanthropy and Disease Advocacy Groups: Charitable Foundation and Venture Philanthropy

Peter S. Klein, MD, PhD
University of Pennsylvania 
Philadelphia, PA, USA
The Short National Institutes of Health Grant

Beverly S. Mitchell, MD
Stanford University 
Stanford, CA, USA
Career Development Plan: Expectations and Reality

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Hematopoietic Stem Cell Transplantation I: Transplant in Benign Hematology

This session will focus on hematopoietic stem cell transplantation (HSCT) for non-malignant disorders and discuss innovations targeting successful outcomes as the field moves toward HSCT for benign disorders. Dr. Alan Tyndall will present updates on the international program using immunoablation with HSCT in patients with severe autoimmune disease. Though final efficacy data from prospective randomized trials are not yet available, much has been learned concerning disease mechanisms and toxicity reduction. Emerging data regarding mesenchymal multipotent stromal cells in the treatment of inflammatory disorders including a concept of "homing and healing" will be summarized. Dr. Robert Wynn will review the current status of HSCT for inborn errors of metabolism (IEM) and alternative therapies, including how they complement or compete with HSCT. The outcome of children with Hurler syndrome has improved as a result of concerted international efforts to understand factors that predict for successful graft outcome. Improved outcomes have led to a reappraisal of HSCT in other IEM where hitherto its application has been limited by perceived risks. The potential roles in future years of multi-modality therapy including gene therapy, enzyme replacement therapy, and substrate reduction therapy will be discussed. Dr. Shalini Shenoy will review progress in understanding the natural history of sickle cell disease and discuss emerging therapies. HSCT as treatment with curative intent will be compared with supportive care and curative strategies to define patients best served by transplant. The pros and cons of HSCT and progress made in the field will also be discussed, focusing on innovative approaches targeted at improved outcomes.

Chair(s):

Shalini Shenoy, MD
Washington University School of Medicine
St. Louis, MO, USA

Speaker(s):

Alan Tyndall, MD
Felix-Platter Spital 
Basel, Switzerland
Successes and Failures of Stem Cell Transplant In Autoimmune Disease

Robert Wynn, MD
Royal Manchester Children's Hospital 
Manchester, United Kingdom
Stem Cell Transplanation for Inherited Metabolic Disorders

Shalini Shenoy, MD
Washington University School of Medicine 
St. Louis, MO, USA
Hematopoietic Stem Cell Transplantation for Sickle Cell Disease: Current Practice and Emerging Trends

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Hematopoietic Stem Cell Transplantation II: Controversies and Challenges in HSCT

This session will focus on recent advances and concepts aimed at improving efficacy and minimizing toxicity of allogeneic stem cell transplantation (SCT). Dr. David Porter will discuss strategies to enhance the graft-versus-tumor (GVT) effects of allogeneic SCT. He will discuss the use of donor leukocyte infusions (DLI) as adoptive immunotherapy to prevent and treat relapse, and the role of DLI compared to second transplant for relapse will also be explored. New approaches using manipulated immune effector cells for relapse and novel strategies to enhance specificity and maximize GVT activity of donor lymphocytes will be highlighted. Ultimately, the most effective approach to relapse is prevention. Dr. Katayoun Rezvani will focus on the use of targeted anti-tumor immunotherapy. He will discuss methods to boost GVT effects with leukemia vaccines or the adoptive transfer of leukemiaspecific lymphocytes. The unique posttransplant milieu, which is characterized by lymphopenia, regulatory T-cell depletion, and the release of growth factors, provides a unique opportunity for effective antitumor immunotherapy and augmenting specific GVT responses. Approaches to increase GVT activity may be improved further by combining vaccination with adoptive transfer of donor-derived vaccineprimed lymphocytes. Dr. Michael Boeckh will describe changes over the last several years in the prevention and treatment of cytomegalovirus (CMV) infection. He will provide an overview of CMV disease in patients with hematologic malignancies, with and without hematopoietic cell transplantation, and summarize current diagnostic approaches to CMV infection in blood and tissues. Dr. Boeckh will also review treatment strategies for CMV disease caused by wild-type and drug-resistant viruses and discuss current and future prevention strategies.

Chair(s):

David L. Porter, MD
University of Pennsylvania
Philadelphia, PA, USA

Speaker(s):

David L. Porter, MD
University of Pennsylvania 
Philadelphia, PA, USA
Allogenetic Immunotherapy to Optimize Graft-Versus-Host Tumor Effect: Concepts and Controversies

Katayoun Rezvani, MD, PhD
Imperial College at Hammersmith Hospital 
London, United Kingdom
Post-Transplant Vaccination: Concepts Today and On the Horizon

Michael Boeckh, MD
Fred Hutchinson Cancer Research Center 
Seattle, WA, USA
Complications, Management, Diagnosis and Prevention of CMV Infections, Current and Future

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Hodgkin Lymphoma

Advanced Hodgkin lymphoma (HL) is curable in the majority of patients diagnosed with the malignancy. The challenge is to strike a balance between achieving high cure rates with primary therapy and optimizing survivorship for patients by minimizing risks of early and late adverse effects. There is controversy about what is considered "optimal" front-line therapy, and recommendations vary significantly in North America and Europe. Interim and post-chemotherapy positron emission tomography (PET) scans have emerged as powerful tools for predicting outcome and are the basis for risk stratification in ongoing clinical trials to identify patients who may benefit from radiation therapy (RT) or from treatment intensification. In the last three decades RT has evolved with significant changes in field size and doses aimed at reducing late effects. The major challenge to the clinician in 2011 is how to interpret and adapt emerging data into decision making for an individual patient. Dr. Ranjana Advani will focus on the optimal management strategies for front-line therapy of advanced HL. She will review recent clinical trial results regarding choice of chemotherapy and role of radiation therapy. Dr. Joseph Connors will discuss the emerging role of PET in risk stratification and management decisions in early and advanced HL. He will also briefly review the use of PET in surveillance. Dr. David Hodgson will discuss the impact of modern RT, such as the use of lower doses and involved fields on long-term effects. He will also review how recent and anticipated changes in HL management are expected to alter late effects of treatment.

Chair(s):

Ranjana H. Advani, MD
Stanford University
Stanford, CA, USA

Speaker(s):

Ranjana H. Advani, MD
Stanford University 
Stanford, CA, USA
Advanced Stage: Optimizing Initial Treatment

Joseph M. Connors, MD
British Columbia Cancer Agency 
Vancouver, BC, Canada
Risk Adapted Therapy and the Role of PET

David C. Hodgson, MD
Princess Margaret Hospital, University of Toronto 
Toronto, ON, Canada
Long-Term Effects In the Era of Modern Radiotherapy

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Junior Faculty Development Program: Mentorship: How to Receive It, How to Provide It, How to Optimize It

THIS SESSION IS OFFERED ONCE. The theme of this year's Junior Faculty Development Program is mentoring. Topics to be addressed include negotiating the transition from fellowship to faculty, including how to establish a "niche," and how the mentee role changes as one progresses from trainee to attending. The speakers will discuss how to become a good mentor, including addressing the challenges of a junior faculty member serving as a mentor and how junior faculty can best support their mentees. Finally, a successful senior mentor will discuss how to identify problems in the mentoring relationship, with advice on how to talk to one's mentor to remedy these issues and, if necessary, how to transition to a different mentor.

Chair(s):

Alison W. Loren, MD
University of Pennsylvania
Philadelphia, PA, USA

Speaker(s):

Corey S. Cutler, MD, MPH, FRCPC
Dana-Farber Cancer Institute 
Boston, MA, USA
Cutting the Apron Strings: How to Become Independent and Develop a Niche

Stephanie J. Lee, MD, MPH
Fred Hutchinson Cancer Research Center 
Seattle, WA, USA
Transitioning From Being Mentored to Becoming a Mentor

Mary M. Horowitz, MD, MS
Medical College of Wisconsin, Center for International Blood and Marrow Transplant Research 
Milwaukee, WI, USA
The Warning Signs of a Problematic Mentoring Relationship and How to Fix It

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Laboratory Medicine: Genetics of Lymphoid Neoplasms: What You Need To Know For Your Current (and Future) Daily Clinical Practice

The genetic underpinnings of lymphoid neoplasms are being unraveled at a rapid pace, using a variety of technologies that span the spectrum of conventional metaphase karyotypic analysis: through fluorescent in situ hybridization (FISH) and polymerase chain reaction (PCR) to more nascent approaches. This has led to exciting discoveries that have honed our understanding of these neoplasms; however, this vast amount of new and old information does not always translate into meaningful or immediate clinical applications. The purpose of this session is to inform the practicing hematologist of what testing is likely to be most pertinent to the management of their patients. Dr. Adam Bagg will provide an overview of the genetic basis of some B-cell lymphomas, highlighting the advantages and limitations of a variety of different testing strategies. This will include a discussion of the role of PCR vs. FISH, as well as the use of immunophenotypic approaches that may potentially provide more userfriendly surrogates for underlying genetic aberrations. Dr. Laurence de Leval will describe the molecular pathogenesis of peripheral T-cell lymphomas, with a focus on the recent discoveries. She will review the current classification of these heterogeneous tumors, the insights gained from genome-wide profiling studies, and their implications for the development of novel therapeutic approaches and clinical management. Dr. Leif Bergsagel will discuss the molecular pathogenesis of plasma cell (multiple) myeloma, focusing on the genetic events important for prognosis and selection of therapy. He will review different methods of detection, including RT-PCR, immunophenotyping, FISH, array comparative genomic hybridization (aCGH), and gene expression profiling, and present a risk-adapted approach to the management of myeloma.

Chair(s):

Adam Bagg, MD
Hospital of the University of Pennsylvania
Philadelphia, PA, USA

Speaker(s):

Adam Bagg, MD
Hospital of the University of Pennsylvania 
Philadelphia, PA, USA
B-Cells Behaving Badly: A Better Basis to Behold Belligerence in B-Cell Lymphomas

Laurence de Leval, MD, PhD
Institut Universitaire de Pathologie 
Lausanne, Switzerland
Tricky and Terrible T-Cell Tumors: These Are Thrilling Times for Testing

P. Leif Bergsagel, MD
Mayo Clinic Arizona 
Scottsdale, AZ, USA
Many and Multiple Myelomas: Making More Out of the Molecular Mayhem

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Pediatric Malignancies: Clinical Implications of Advances in Leukemia Biology

This session will focus on recent advances in molecular understanding of specific subsets of high-risk pediatric acute leukemias and describe how this information is being used to develop new therapeutic approaches. Dr. Scott Armstrong will review the molecular pathogenesis of leukemias with chromosomal translocations involving the mixed lineage leukemia (MLL) gene and describe aberrant epigenetic programs found in these leukemias. He will discuss how this information is being used to develop new therapeutic approaches to treat this subtype of high-risk leukemia. Dr. Stephen Hunger will review outcomes for BCR-ABL1- positive ALL treated with chemotherapy and/or stem cell transplantation without tyrosine kinase inhbitors (TKIs) and data on outcomes with chemotherapy plus imatinib. He will review emerging data on treatment with chemotherapy plus second-generation TKIs. Dr. Stuart S. Winter will review several recently described genetic aberrations found in high-risk acute myeloid and lymphocytic leukemias. He will also briefly review the signaling pathways that are subsequently disrupted by these molecular aberrations and how they are being targeted for therapeutic intervention.

Chair(s):

Scott A. Armstrong, MD, PhD
Children's Hospital Boston and Dana-Farber Cancer Institute
Boston, MA, USA

Speaker(s):

Scott A. Armstrong, MD, PhD
Children's Hospital Boston and Dana-Farber Cancer Institute 
Boston, MA, USA
Targeting Epigenetic Programs In MLL-Rearranged Leukemias

Stephen Hunger, MD
University of Colorado School of Medicine 
Aurora, CO, USA
First- and Second-Generation Tyrosine Kinase Inhibitors for BCR-ABL-Rearranged Pediatric ALL

Stuart S. Winter, MD
University of New Mexico Health Sciences Center 
Albuquerque, NM, USA
Pediatric Acute Leukemia Therapies Informed by Molecular Analysis of High-Risk Disease

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Platelet Drugs and Disorders

This session will focus on challenges in the diagnosis and treatment of platelet disorders, including inherited and acquired disorders of platelet function and number. Key management issues in pediatric and adult patients will be addressed. Dr. Michele Lambert will review the presentation of inherited platelet disorders, including key history and laboratory findings in children and adults. She will review recent advances in our understanding of the genetics and pathophysiology of these disorders. Dr. Lambert will then present a diagnostic algorithm and discuss treatment indications, approaches, and consequences. Dr. Howard Liebman will begin by briefly reviewing the pathophysiology of immune thrombocytopenia (ITP) and then discuss consensus recommendations for the diagnosis of ITP. He will review treatment approaches, including recent data on therapeutic efficacy of the thrombopoietin mimetics. Risks associated with these and other treatments for ITP will be discussed. Finally, an integrated approach to treatment will be presented. Dr. Barbara Konkle will discuss the varied presentations of acquired platelet dysfunction. She will briefly review drugs and their effects on platelet function in different clinical settings. Dr. Konkle will also discuss effects of cardiopulmonary bypass on platelet function and implications for post-operative bleeding. She will then present data on various medical conditions associated with platelet dysfunction, including infections, renal failure, and underlying hematologic disorders such as myelodysplasia, and provide an approach to treatment.

Chair(s):

Barbara A Konkle, MD
Puget Sound Blood Center
Seattle, WA, USA

Speaker(s):

Michele Lambert, MD
The Children's Hospital of Philadelphia 
Philadelphia, PA, USA
What to Do When You Suspect An Inherited Platelet Disorder

Howard A Liebman, MD
University of Southern California Norris Cancer Hospital 
Los Angeles, CA, USA
Thrombopoietin Mimetics: Emerging Niche in the Thrombocytopenia Armamentarium

Barbara A Konkle, MD
Puget Sound Blood Center 
Seattle, WA, USA
Acquired Platelet Functional Disorders

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Possible Game-Changers in Hemophilia

This session will focus on recent advances and controversies in the treatment of hemophilia. Dr. Patrick Fogarty will discuss the clinical impact of a congenital deficiency of factor VIII (hemophilia A) or factor IX (hemophilia B) in children and adults. He will focus on strategies to correct the deficiency using clotting factor concentrate and the limitations of currently available therapies. Novel approaches to providing enhanced hemostasis in affected individuals will be reviewed, along with a discussion of potential benefits to patients' joint health, tolerability of medical and invasive treatments, and quality of life. Dr. Rebecca Kruse-Jarres will elaborate on some controversies in the management of hemophilia. Her discussion will focus on inhibitor development and treatment and will include the possible impact of intensity, timing, and product choice of early treatment. Further, this talk will concentrate on immune tolerance as a treatment for inhibitor eradication and include possible immune modulation. Discussion will take into account the difficulty in conducting trials in this population. Dr. Kathleen Johnson will describe the cost of care of hemophilia and how health-care reform might affect both consumers and providers. Specifically, this session will describe the components utilized to assess the cost of care and how they are measured. Additionally, this program will review key components of health-care reform and implications for individuals with hemophilia and providers in the future.

Chair(s):

Rebecca Kruse-Jarres, MD
Tulane University School of Medicine
New Orleans, LA, USA

Speaker(s):

Patrick F. Fogarty, MD
University of Pennsylania 
Philadelphia, PA, USA
Biological Rationale for New Drugs in the Bleeding Disorders Pipeline

Rebecca Kruse-Jarres, MD
Tulane University School of Medicine 
New Orleans, LA, USA
Current Controversies in Hemophilia Management

Kathleen A. Johnson, PharmD, PhD
University of Southern California School of Pharmacy 
Los Angeles, CA, USA
Costs of Care in Hemophilia and Possible Implications of Health Care Reform

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Recent High-Impact Clinical Trials in Sickle Cell Disease: Where Do They Leave the Field?

This session will focus on the results of recent major clinical studies in sickle cell disease and their impact on the management of patients, as well as on insights gained into complex pathobiological processes leading to clinical manifestations. Dr. Kathryn Hassell will briefly review the role of hemolysis, nitric oxide, and related factors that may affect the cardiopulmonary system in sickle cell disease. She will discuss the impact of interventional studies, including the Treatment of Pulmonary Hypertension and Sickle Cell Disease with Sildenafil Trial (Walk-PHaSST), on recommendations for screening and treatment of cardiopulmonary complications. Dr. Michael DeBaun will briefly review the current understanding of the pathobiology of neurological complications in sickle cell disease, including stroke and silent ischemia. He will discuss the potential implications of clinical studies, including the Stroke with Transfusion Changing to Hydroxyurea (SWiTCH) study, for the management of acute and progressive neurological complications. Dr. Alexis Thompson will briefly review aspects of sickle cell disease that may be prevented and/or modified by hydroxyurea therapy, based on its mechanisms of action. She will discuss available findings from clinical studies, including the Pediatric Hydroxyurea Phase III Clinical Trial (Baby HUG), and their impact on the use of hydroxyurea in sickle cell disease, including for the primary prevention of complications.

Chair(s):

Kathryn L. Hassell, MD
University of Colorado Denver Health Sciences Center
Denver, CO, USA

Speaker(s):

Kathryn L. Hassell, MD
University of Colorado Denver Health Sciences Center 
Denver, CO, USA
Pulmonary Hypertension, Tricuspid Regurgitant Jet Velocity Screening, and the Nitric Oxide Pathway

Michael R. DeBaun, MD, MPH
Monroe Carell Jr. Children’s Hospital at Vanderbilt 
Nashville, TN, USA
Stroke Prophylaxis, Transfusions, and Iron Overload

Alexis A. Thompson, MD
Children's Memorial Hospital 
Chicago, IL, USA
Hydroxyurea for Prophylaxis In Asymptomatic Children with Sickle Cell Disorders

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Strategies for Optimal Management in Thalassemia - Now and In the Future

This session will provide an update on advances in magnetic resonance imaging (MRI) of organ-specific iron loading and the clinical use of chelation therapy to manage iron overload. Strategies for fetal hemoglobin induction and their application to potential new treatments of thalassemia will be discussed. Dr. John Wood will discuss the use of MRI to measure tissue iron stores. His presentation will focus on the indications, methods, and interpretation of the results of liver, heart, and endocrine iron assessments in thalassemia. Dr. Janet Kwiatkowski will briefly review data on the efficacy and adverse effects of the currently available chelators. She will discuss the clinical use of chelators both as single agents and in combination. Dr. Kwiatkowski will also describe the challenges associated with randomized clinical trials of chelators and address potential research design strategies to overcome these obstacles. Dr. Vijay Sankaran will discuss recent studies that have led to the identification of molecular regulators of the fetal-to-adult hemoglobin switch and silencing of fetal hemoglobin (HbF) in adults. He will describe how such work can lead to targeted therapeutic strategies for HbF induction in patients with hemoglobinopathies.

Chair(s):

Janet L. Kwiatkowski, MD
The Children's Hospital of Philadelphia
Philadelphia, PA, USA

Speaker(s):

John C Wood, MD, Ph.D
Children's Hospital Los Angeles 
Los Angeles, CA, USA
Impact of Iron Assessment by Magnetic Resonance Imaging

Janet L. Kwiatkowski, MD
The Children's Hospital of Philadelphia 
Philadelphia, PA, USA
Real World Use of Iron Chelators (Combination Agents, Comparative Effectiveness Strategies)

Vijay G. Sankaran, MD, PhD
Children's Hospital Boston, Harvard Medical School 
Boston, MA, USA
Induction of Fetal Hemoglobin by BCL11A Manipulation - Basic Science and Therapeutic Outlook

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Transfusion Medicine I: Adverse Complications of Stored Blood

THIS SESSION IS JOINTLY SPONSORED WITH AABB. Clinical studies suggest that storage of blood products is associated with increased morbidity and mortality in some recipients. This session will give an overview of our current understanding of the immunomodulatory and vasoregulatory effects of blood products and how their storage may affect clinical outcomes in transfusion. Dr. Craig Morrell will review immune responses to platelets and discuss how the platelet storage lesion can affect immune responses. Platelets have a major role in initiating and accelerating immune responses, and platelet transfusion may be associated with adverse inflammatory outcomes manifested as fever, discomfort, tachycardia, and respiratory issues. The role of platelet-derived immune mediators in driving this response will be highlighted in this presentation in addition to an overview of related ongoing clinical and animal model studies. Dr. Karina Yazdanbakhsh will discuss immunoregulation in response to red blood cell transfusions, focusing on the role of regulatory T and B cell compartments. She will briefly review transfusion-related immunomodulation and discuss how regulatory circuits may be altered when "aged" red blood cell units are transfused, leading to immune perturbations with adverse clinical outcomes. Dr. John Roback will review vasoactive effects of blood transfusion, with a focus on alterations in vascular responsiveness with increased storage intervals prior to transfusion. In vitro studies using aortic rings, as well as in vivo studies in transfused patients and volunteers, will be discussed. Studies to understand recipient factors, as well as mechanisms of the storage effect, will be highlighted as a way to improve the transfusion effects of stored blood.

Chair(s):

Karina Yazdanbakhsh, PhD
New York Blood Center
New York, NY, USA

Speaker(s):

Craig Morrell, DVM, PhD
University of Rochester Medical Center 
Rochester, NY, USA
Age-Related Immunomodulatory Effects of Platelets

Karina Yazdanbakhsh, PhD
New York Blood Center 
New York, NY, USA
Immunoregulatory Effects of Stored Red Cells

John Roback, MD, PhD
Emory University Hospital 
Atlanta, GA, USA
Vascular Effects of the Red Cell Storage Lesion

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Transfusion Medicine II: Hematology and Transfusion Medicine in Sub-Saharan Africa

THIS SESSION IS JOINTLY SPONSORED WITH AABB. THIS SESSION IS OFFERED ONCE. In this session hematologists and transfusion medicine experts will focus on the clinical transfusion medicine and blood supply in sub-Saharan Africa. Dr. Troy Lund will discuss what a hematologist-oncologist encounters working in sub-Saharan Africa, covering the global epidemic of both benign and malignant hematologic disorders. He will discuss potential diagnostic and treatment strategies in Africa and the practicalities of working in sub-Saharan Africa. Dr. Heather Ann Hume will discuss hospital transfusion practices in Uganda's largest publicly funded, universitybased referral hospital. Some of the problems encountered are similar to those found in high-income countries, including proper patient identification, avoiding hemolytic transfusion reactions, and appropriate use of blood and components. However, the setting also presents challenges, both cultural and financial, that demand creative solutions. Some of the particular issues related to the transfusion support of patients with sickle cell anemia in this setting will be discussed. Dr. Jean-Pierre Allain will discuss blood transfusion safety in sub-Saharan Africa as characterized by high prevalence of infectious agents, chronic blood shortage, and lack of resources. Considerable pressure is applied by richer countries and international transfusion bodies to establish voluntary, non-remunerated blood donors (VNRDs) as the only source of blood, excluding the traditional family/replacement donors on the basis of a higher level of safety. Such policies increase the cost of blood and, in some cases, can paradoxically deepen the pre-existing blood shortage. First-time VNRDs are no safer than family/replacement donors; only repeat donation provides improved blood safety. Dr. Allain will discuss strategies to increase the supply of safe blood.

Chair(s):

Jeffrey McCullough, MD
University of Minnesota
Minneapolis, MN, USA

Walter Sunny Dzik, MD
Harvard University
Boston, MA, USA

Speaker(s):

Troy Lund, MD, PhD
University of Minnesota 
Minneapolis, MN, USA
The Hematologist-Oncologist in Sub-Saharan Africa

Heather Ann Hume, MD
CHU Sainte-Justine, University of Montreal 
Montreal, QC, Canada
Transfusion Medicine at the Bedside In Uganda

Jean-Pierre Allain, MD, PhD
University of Cambridge 
Cambridge, United Kingdom
National Blood Services in Sub-Saharan Africa

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Understanding and Management of Inherited Bone Marrow Failure Syndromes

This session will provide an update on these heterogeneous multi-system bone marrow (BM) failure syndromes. This will include the recent advances in their genetics and the important links to fundamental biological pathways: telomere maintenance in dyskeratosis congenita (DC), DNA repair in Fanconi anemia (FA), and ribosome biogenesis in Diamond-Blackfan anemia (DBA). These advances are improving our understanding, providing new diagnostic tests, and facilitating management. Dr. Inderjeet Dokal will give an update on DC including the "cryptic" presentations that have been recognized in recent years. The genetic basis of a significant subset of DC patients has been elucidated with seven out of the eight characterized genes having an important role in telomere maintenance. This is facilitating diagnosis and management, particularly when presentation is atypical. Dr. Sarah Ball will give a clinical and genetic review of DBA. Nine DBA genes have been identified, and these are all important in ribosome biogenesis. These advances are enabling accurate diagnosis and have shown how defects in ribosome function provide an important link between a number of inherited (DBA, Shwachman-Diamond syndrome) and acquired (5q- syndrome) BM failure syndromes. Dr. Jean Soulier will give a presentation on FA. Fourteen FA subtypes are currently recognized, and their associated proteins are important in DNA repair/genomic stability. The diagnosis of FA has evolved considerably in recent years as has our understanding of its natural history, including clonal progression to MDS/AML (with implications for disease monitoring).

Chair(s):

Inderjeet Dokal, MD
Barts and The London School of Medicine and Dentistry
London, United Kingdom

Speaker(s):

Inderjeet Dokal, MD
Barts and The London School of Medicine and Dentistry 
London, United Kingdom
Dyskeratosis Congenita

Sarah Ball, DM
St George's University of London 
London, United Kingdom
Diamond-Blackfan Anemia

Jean Soulier, MD PhD
Saint-Louis Hospital and University Paris-Diderot 
Paris, France
Fanconi Anemia

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Update on Aggressive Lymphomas - New Agents and Current Management

This session will focus on recent advances in diagnosis and treatment of aggressive lymphomas, emphasizing new diagnostic entities and novel targeted therapeutic agents for both T- and B-cell histologies. Dr. Elaine Jaffe will review new lymphoma entities in the recently updated World Health Organization (WHO) classification, focusing on the challenge of differentiating Burkitt lymphoma from diffuse large B-cell lymphoma, the unclassified lymphomas intermediate between Burkitt lymphoma and diffuse large B-cell lymphoma, and Epstein-Barr virus-driven lymphomas in individuals of advanced age. In addition to morphological criteria, Dr. Jaffe will review cytogenetic and molecular studies that aid in these diagnostic categories. A key focus will be on the "double hit" lymphomas and the role of C-MYC in defining these entities. Dr. Jonathan Friedberg will then review the challenge of relapsed diffuse large B-cell lymphoma. He will cover new genetic and molecular findings that explain why patients relapse and define novel targeted therapeutic strategies. Dr. Friedberg will then cover the standard treatment approach to relapsed disease in the rituximab era, focusing on new datasets. The talk will conclude with a survey of clinical trials focused on this disease entity. Dr. Kerry J. Savage will conclude the session with a focus on current therapies for peripheral T-cell lymphomas (PTCLs). In this talk, she will provide a brief overview of the challenges with standard CHOP chemotherapy and discuss new treatment directions, including the role of transplant in the therapy of PTCLs. Dr. Savage will then focus her discussion on recently approved agents in relapsed PTCLs, including antifolate agents and histone deacetylase inhibitors, as well as other novel agents under investigation with high promise in the therapy of PTCLs.

Chair(s):

Jonathan Friedberg, MD
University of Rochester
Rochester, NY, USA

Speaker(s):

Elaine S. Jaffe, MD
National Cancer Institute 
Bethesda, MD, USA
New Entities in Aggressive B-Cell Lymphoma World Health Organization Classification

Jonathan Friedberg, MD
University of Rochester 
Rochester, NY, USA
Relapsed Diffuse Large B-Cell Lymphoma

Kerry J. Savage, MD MSc
British Columbia Cancer Agency 
Vancouver, BC, Canada
Therapies for Peripheral T-Cell Lymphomas, Including Novel Agents

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Updates on Disorders of Iron Utilization and Distribution

This session will focus on recent advances in the basic science of iron metabolism and congenital sideroblastic anemias, as well as the potential for molecularly targeted clinical therapeutics for iron disorders. Dr. Karin Finberg will discuss the role of the peptide hormone hepcidin in the regulation of systemic iron homeostasis. New insights into the way hepcidin expression is modulated in response to several physiological factors will be presented. Emphasis will be placed on insights gained from animal models of iron deficiency and iron overload as well as human disorders of iron balance. Dr. Tomas Ganz will discuss how progress in our understanding of systemic iron regulation by hepcidin and ferroportin is translated into new therapeutic approaches for iron overload states and iron restricted anemias. Dr. Mark Fleming will review the diagnosis, classification, molecular characterization, and pathogenesis of congenital sideroblastic anemias and will discuss how insight into these disorders may have an impact on therapy and our understanding of acquired forms of the disease.

Chair(s):

Mark D. Fleming, MD, DPhil
Children's Hospital Boston, Harvard Medical School
Boston, MA, USA

Speaker(s):

Karin E. Finberg, MD, PhD
Duke University Medical Center 
Durham, NC, USA
Unraveling Iron Control Mechanisms

Tomas Ganz, MD, PhD
University of California Los Angeles 
Los Angeles, CA, USA
Therapeutic Outlook for Modulation of Hepcidin System

Mark D. Fleming, MD, DPhil
Children's Hospital Boston, Harvard Medical School 
Boston, MA, USA
Genetic Dissection of Congenital Sideroblastic Anemias

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Updates on Treatment Strategies in Myelodysplastic Syndromes

Treatment of myelodysplastic syndromes (MDS) has recently changed with the approval of the hypomethylating agents azacitidine and decitabine, and of an immunomodulating agent, lenalidomide. Meanwhile, thanks to refinement of molecular analyses, biological advances have been made allowing for identification of new genes involved in MDS pathophysiology. Dr. Timothy Graubert will review recently discovered genetic abnormalities in MDS consisting of gene point mutations, especially in genes coding for proteins involved in epigenetic modifications and, in MDS with 5q deletion, of gene haploinsufficiency. Those mutations help our understanding of MDS, and many of them may have prognostic value, including in response to hypomethylating agents. Dr. Steven Gore will then review how to further improve the survival benefit obtained with hypomethylating agents, especially through trial of different treatment regimens and by combining hypomethylating agents with other drugs. Candidates for those combinations are selected through in vitro and ex vivo testing and will be confirmed by large-scale clinical trials where survival will likely be the major endpoint, given the frequent dissociation between survival and response with hypomethylating agents. Dr. Pierre Fenaux will focus mainly on results obtained with lenalidomide in low- risk MDS with 5q deletion, addressing issues about dosing, prognostic factors of response, and management of side effects. He will also present available results of lenalidomide in lower-risk MDS without 5q deletion and share perspectives opened by lenalidomide in higher-risk MDS (and AML) with 5q deletion.

Chair(s):

Pierre Fenaux, MD, PhD
Hôpital Avicenne, AP-HP, Université Paris 13
Bobigny, France

Speaker(s):

Timothy Graubert, MD
Washington University School of Medicine 
Saint Louis, MO, USA
Genetics of MDS: New Insights

Steven D. Gore, MD
Sidney Kimmel Cancer Center, The Johns Hopkins University 
Baltimore, MD, USA
New Ways to Use DNA Hypomethylating Agents

Pierre Fenaux, MD, PhD
Hôpital Avicenne, AP-HP, Université Paris 13 
Bobigny, France
Immunomodulating Drugs In MDS

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