| Co-Chairs: |
Joseph R. Mikhael, MD, Mayo Clinic Arizona, Scottsdale, AZ
Bradford S. Schwartz, MD, University of Illinois College of Medicine at Urbana-Champaign, Urbana, IL |
The 2010 Education Program will be held Saturday, December 4, and Sunday, December 5. Each session will be offered twice unless otherwise noted. Chapters based on these sessions will be published in the Education Program Book, Hematology 2010. In addition, audio recordings and slides from Education Program presentations will be available on DVD.
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Challenges in Acute Myeloid Leukemia
| Chair: |
Hugo F. Fernandez, MD, Moffitt Cancer Center, Tampa, FL |
| Speakers: |
James M. Foran, MD, University of Alabama at Birmingham, Birmingham, AL
New Prognostic Markers in Acute Myeloid Leukemia |
Selina Luger, MD, Abramson Cancer Center, University of Pennsylvania, Philadelphia, PA
Treating the Elderly Patient With Acute Myeloid Leukemia |
Hugo F. Fernandez, MD, Moffitt Cancer Center, Tampa, FL
New Trends in the Standard of Care for Initial Therapy of Acute Myeloid Leukemia |
This session will focus on recent advances in the treatment of acute myeloid leukemia (AML) for younger and older adults. The session will cover genetic and molecular abnormalities that impact the disease in its initial response to chemotherapy, and leukemia-free and overall survival. The speakers will present recent findings in approaches to initial therapy for younger adults and for the appropriateness of therapy and the best approach for older adults with AML.
Dr. James Foran will review new prognostic markers in AML, including refinements in cytogenetic classification as well as new molecular and flow-cytometric prognostic factors that can affect response and survival. He will discuss the impact of these markers in both younger and older patients, focusing on those with cytogenetically normal AML. He will also discuss the potential role of new prognostic markers in clinical management, including allogeneic transplantation.
Dr. Selina Luger will discuss the treatment of the elderly AML patient, focusing on the prognostic scoring systems that are being used to predict survival, early death, and appropriateness for intensive therapy. She will summarize recent studies that have aggressively treated older patients and new chemotherapeutic combinations that have been designed for those considered unfit for intensive therapy.
Dr. Hugo Fernandez will present recent approaches to induction chemotherapy for younger patients with AML. He will discuss the impact of anthracycline intensification and targeted therapy and compare and contrast the different anthracyclines in their ability to successfully achieve a complete remission.
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Myelodysplastic Syndromes
| Chair: |
William Blum, MD, The Ohio State University, Columbus, OH |
| Speakers: |
Guillermo Garcia-Manero, MD, The University of Texas M. D. Anderson Cancer Center, Houston, TX
Prognosis in Myelodysplastic Syndromes |
Corey Cutler, MD, MPH, Dana-Farber Cancer Institute, Boston, MA
How Does Allogeneic Transplant Fit Into Current MDS Therapy? |
William Blum, MD, The Ohio State University, Columbus, OH
How Much, How Frequent, How Long? A Clinical Guide to New Therapies in MDS |
This session will focus on recent advances in the care of patients with myelodysplastic syndromes (MDS) including prognosis in MDS, the use of allogeneic transplantation in MDS, and endpoints in the management of patients with MDS.
Dr. Guillermo Garcia-Manero will review the state of the art in our understanding of prognosis for patients with MDS. He will describe the relationship of clinical and disease-specific factors in MDS to clinical outcomes and to the selection of appropriate therapies. Further, he will describe advances in molecular understanding of the disease as it relates to clinical outcome and response to treatment.
Dr. Corey Cutler will review the role of allogeneic transplantation in the care of MDS patients. He will review evidence for the clinical benefit of this approach and discuss the selection of appropriate patients for transplantation. He will describe the timing of transplantation relative to clinical factors such as age and prognostic risk. Selection of optimal conditioning regimens will also be discussed.
Dr. William Blum will describe patient-oriented endpoints in the care of patients with MDS. He will review current treatment options and discuss the goals of therapy with each of these various treatment approaches. He will discuss not only long-term goals of treatment but also short-term problems to provide guidance on decision making in the ongoing care of MDS patients. He will then briefly review the current understanding of molecular endpoints/targets in MDS therapy.
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Acute Lymphoid Leukemia
| Chair: |
David I. Marks, MBBS, PhD, University Hospitals Bristol NHS Foundation Trust, Bristol, United Kingdom |
| Speakers: |
Wendy Stock, MD, University of Chicago, Chicago, IL
Treating the Younger Adult With ALL |
David I. Marks, MBBS, PhD, University Hospitals Bristol NHS Foundation Trust, Bristol, United Kingdom
Treating the Older Adult With ALL |
Dario Campana, MD, PhD, St. Jude Children’s Research Hospital, Memphis, TN
Minimal Residual Disease in ALL |
This session will focus on recent advances in the management of older and younger adults with ALL, in particular describing differences in biology, the results of recent trials, and registry-based studies. There will also be a detailed discussion of the methods of detection of minimal residual disease (MRD) and their value in determining therapeutic decisions.
Dr. Wendy Stock will focus on the management of patients up to the age of 30 years focusing on current trials and questions about biological/sociological differences between young adults and younger children with ALL and older adults.
Dr. David Marks will discuss prognostic factors and each phase of therapy including the role of allogeneic and autologous stem cell transplantation. There will be discussions about T-cell disease and relapse as well as a brief description of management in older patients and likely future advances.
Dr. Dario Campana will discuss the rationale and methods for MRD monitoring, the clinical significance of MRD in adult and childhood ALL, and the relation between biologic and genetic features of leukemia with treatment response as measured by MRD assays.
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Multiple Myeloma
| Chair: |
Sagar Lonial, MD, Emory University School of Medicine, Atlanta, GA |
| Speakers: |
Ola Landgren, MD, PhD, National Cancer Institute, National Institutes of Health, Bethesda, MD
MGUS: New Insights Into Pathophysiology and Epidemiology |
Raymond L. Comenzo, MD, Tufts Medical Center, Boston, MA
Amyloidosis: Novel Therapies |
Sagar Lonial, MD, Emory University School of Medicine, Atlanta, GA
Relapsed Therapy in Myeloma |
This session will focus on recent advances in the basic science of plasma cell disorders focusing specifically on disease pathogenesis from the MGUS/smoldering state, the light chain deposition state of amyloidosis, and management and concepts for relapsed multiple myeloma.
Dr. Ola Landgren will review new insights into the basic biology and pathophysiology of MGUS and smoldering myeloma, with specific emphasis on new epidemiological data tracking of the frequency of MGUS and risk factors for progression. He will review the genes and pathways involved in the transition from MGUS to myeloma, as well as genetic changes that are common to MGUS, smoldering myeloma, and symptomatic myeloma.
Dr. Raymond Comenzo will review the diagnostic criteria for amyloidosis, as well as the many different presenting symptoms associated with the diagnosis of this challenging disease. He will also review new diagnostic tests that can be used to confirm the different types of amyloidosis. Finally, he will discuss standard and newer treatment approaches for amyloidosis that specifically target the etiology and pathology of the disease.
Dr. Sagar Lonial will describe the myriad of new treatments and approaches for the management of relapsed disease, including single agent, combination new targets, and risk-based approaches for relapsed disease.
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Supportive Care in Plasma Cell Dyscrasias
| Chair: |
Pieter Sonneveld, MD, PhD, Erasmus University Medical Center, Rotterdam, Netherlands |
| Speakers: |
Pieter Sonneveld, MD, PhD, Erasmus University Medical Center, Rotterdam, Netherlands
Dealing With Neuropathy |
Meletios A. Dimopoulos, MD, University of Athens School of Medicine, Alexandra General Hospital, Athens, Greece
Renal Insufficiency and Failure |
Sigurdur Y. Kristinsson, MD, PhD, Karolinska University Hospital and Karolinska Institutet, Stockholm, Sweden
Thrombotic Issues in Myeloma |
This session reviews clinical aspects of plasma cell dyscrasias, concentrating on morbidity at presentation and treatment-related complications in multiple myeloma.
Dr. Pieter Sonneveld will review the etiology and presenting symptoms of neuropathy in monoclonal plasma cell diseases. He will review predisposing factors and clinical characteristics of treatment-related neuropathy with a focus on bortezomib and thalidomide. Practical recommendations for prevention and treatment of treatment-associated neuropathy will be discussed.
Dr. Meletios Dimopoulos will describe the pathogenesis and clinical presentation of renal insufficiency in multiple myeloma. He will then discuss various diagnostic and therapeutic approaches for the patient presenting with renal failure. Finally, he will review the treatment options with a focus on the use of novel agents, the preferred regimens, and the impact on prognosis in this group of patients.
Dr. Sigurdur Kristinsson will discuss the mechanisms of thrombosis in multiple myeloma. He will describe epidemiological factors as well as disease-related activation of hemostasis. He will then review the incidence of thrombosis in myeloma and risk factors that are associated with specific treatment choices. Finally, recommendations for thrombosis prophylaxis will be discussed.
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Lymphoma: Translating Basic Science Into Therapy
| Chair: |
AlexThomas E. Witzig, MD, Mayo Clinic, Rochester, MN |
| Speakers: |
Thomas E. Witzig, MD, Mayo Clinic, Rochester, MN
Signal Transduction Pathway Therapy in Lymphoma |
Sandeep Dave, MD, Duke University, Durham, NC
Host Factors for Risk and Survival in Lymphoma |
John P. Leonard, MD, Weill Cornell Medical College, New York, NY
Novel Agents in Follicular Lymphoma |
Significant progress is being made in the understanding of the pathogenesis of non-Hodgkin lymphoma. New insights into the mechanisms of resistance of tumor cells to common agents and the role of tumor microenvironment have led to the development of new agents for the treatment of lymphoma. These treatments have moved from the laboratory through phase I and phase II and now are being applied upfront.
Dr. Thomas Witzig will discuss the various signal transduction pathways involved in the growth and resistance of lymphoma cells and how these pathways are being targeted. He will also discuss some of the mechanisms of resistance that develop when these new agents are used.
Dr. Sandeep Dave will describe the importance of host factors and the risk of lymphomagenesis and prognosis. These host factors and microenvironmental factors can play an important role in the development and maintenance of lymphoma. They also provide unique targets for therapy.
Dr. John Leonard will describe how these novel agents have entered standard care for lymphoma patients. He will also discuss new agents being tested in clinical trials. This session will provide important information for the practicing hematologist/oncologist that can be readily applied to the care of patients. In addition, the session will provide information relevant to translational research scientists who are investigating new ways of targeting these pathways in lymphoma.
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Issues of Toxicity in Allogeneic Bone Marrow Transplantation
| Chair: |
Mohamad Mohty, MD, PhD, CHU de Nantes, Nantes, France |
| Speakers: |
Mohamed L. Sorror, MD, MSc, University of Washington, Fred Hutchinson Cancer Research Center, Seattle, WA
Predicting Toxicity Based on Patient Comorbidities |
Mohamad Mohty, MD, PhD, CHU de Nantes, Nantes, France
Long-Term Physiological Side Effects After Allo-BMT |
Margaret F. Bevans, RN, PhD, National Institutes of Health, Bethesda, MD
Quality of Life and Psychosocial Issues With Allogeneic BMT |
Several types of late sequelae from allogeneic bone marrow or hematopoietic stem cell transplantation (allo-BMT) have been noted, and awareness of these complications is important in minimizing late morbidity and mortality. This session will review different aspects related to the role of comorbidities in predicting toxicity after allo-BMT and features of long-term side effects, with a particular emphasis on quality-of-life and psychosocial issues.
Dr. Mohamed Sorror will briefly review the initial steps of adopting systematic assessment of pre-BMT comorbidities in academic research and clinical practice, touching on the summation of multiple comorbidities into a single risk factor. He will then describe the vital role of comorbidities in predicting regimen-related morbidity and mortality after different settings of allogeneic and autologous BMT and across different institutions. Future incorporation of comorbidities and other traditional prognostic factors in redefining risk-benefit assessment for late BMT sequels will be discussed.
Dr. Mohamad Mohty will review late effects after allo-BMT, a heterogeneous group of complications that can include toxicities from the preparative regimen that may cause organ damage with permanent sequel, infections from immunodeficiency, endocrine disturbances, secondary malignancies, and chronic graft-versus-host disease with its corollary of altered immune reconstitution.
Dr. Margaret Bevans will describe common dimensions of health-related quality-of-life and psychosocial outcomes across the trajectory of allo-BMT. She will discuss key considerations when interpreting these findings and highlight the recipient and caregiver outcomes that are most amenable to clinical intervention. Gaps in the evidence will be briefly reviewed, and opportunities for future research will be identified.
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Pediatric Malignancies
| Chair: |
Stella M. Davies, MBBS, PhD, Cincinnati Children’s Hospital Medical Center, Cincinnati, OH |
| Speakers: |
Mignon L. Loh, MD, University of California - San Francisco, San Francisco, CA
Pediatric Myeloproliferative Disorders |
Meinolf Suttorp, MD, Universitätsklinikum Carl Gustav Carus, Dresden, Germany
Treatment of Pediatric CML in 2010: Use of TKIs and BMT |
Stella M. Davies, MBBS, PhD, Cincinnati Children’s Hospital Medical Center, Cincinnati, OH
Pediatric Regimens for ALL |
Chemotherapy and transplantation are important modalities in the treatment and cure of pediatric hematologic malignancies. The appropriate selection of transplant- or non-transplant-based therapies for treatment depends on careful knowledge of the biology of the disorder and of expected outcomes of each therapy.
In this session Dr. Mignon Loh will describe new developments in the biology of myeloproliferative disorders in children, with a particular focus on familial susceptibility.
Dr. Meinolf Suttorp will then explore the applicability of transplantation in children with chronic myelogenous leukemia in the era of ever-improving tyrosine kinase inhibitors.
Finally, Dr. Stella Davies will discuss the question of whether there is still a role for transplantation in children with acute lymphoblastic leukemia, as outcomes of chemotherapy improve and our understanding of biology and prognosis progresses.
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Chronic Lymphocytic Leukemia
| Chair: |
Richard R. Furman, MD, Weill Medical College of Cornell University, New York, NY |
| Speakers: |
Mark C. Lanasa, MD, PhD, Duke University Medical Center, Durham, NC
New Insights Into the Biology of CLL |
Alessandra Ferrajoli, MD, The University of Texas M. D. Anderson Cancer Center, Houston, TX
Treatment of Younger Patients With CLL |
Richard R. Furman, MD, Weill Medical College of Cornell University, New York, NY
Prognostic Markers and Stratification of CLL |
This session will focus on recent advances in chronic lymphocytic leukemia (CLL), including the biology of CLL, special considerations for the treatment of younger patients with CLL, and the application of prognostic markers to the clinical care of patients with CLL.
Dr. Mark Lanasa will broadly review some of the recent advances in the understanding of the biology of CLL. The goal of this review is to provide a model of disease pathogenesis that integrates inherited, acquired, environmental, and immunologic factors. Topics to be covered will include: the contribution of genetic factors in CLL pathogenesis, the central role of the B-cell receptor and B-cell receptor signaling in CLL, and how interactions between CLL cells and the tumor microenvironment promote CLL cell survival and chemo-resistance.
Dr. Alessandra Ferrajoli will discuss the issues that make the treatment of younger patients with CLL unique. She will focus on differences in the biology of their disease and the goals of therapy.
Dr. Ferrajoli will then discuss treatment options, including allogeneic stem cell transplantation, and factors to consider in treatment planning. Dr. Richard Furman will briefly review the classic and novel prognostic markers and the biological basis for these prognostic markers. The talk will then focus on how these prognostic markers should be used to guide therapy for patients with CLL.
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Clinical Advances in Hodgkin Lymphoma
| Chair: |
Nancy Bartlett, MD, Washington University School of Medicine, St. Louis, MO |
| Speakers: |
Peter Borchmann, PhD, MD, University of Cologne, Cologne, Germany
The Past: What We Have Learned in the Last Decade |
Nancy Bartlett, MD, Washington University School of Medicine, St. Louis, MO
The Present: Optimizing Therapy – Too Much or Too Little? |
Kristie A. Blum, MD, The Ohio State University, Columbus, OH
The Future: Upcoming Diagnostic and Therapeutic Developments |
Despite the overall favorable prognosis of Hodgkin lymphoma, efforts to improve therapy for high-risk patients and to decrease long-term toxicity for low-risk patients continue. New methods of risk stratification, including FDG-PET and molecular profiling, as well as new therapies with non-overlapping toxicity profiles, have made these goals a reality.
Dr. Peter Borchmann will describe the results of several key randomized trials for Hodgkin lymphoma that have been published in the last decade. Large trials comparing standard therapies with more aggressive regimens for patients with an unfavorable prognosis and less intense treatments for patients with a favorable prognosis have shaped our current approaches to Hodgkin lymphoma.
Dr. Nancy Bartlett will discuss practical approaches to choosing therapy for patients with Hodgkin lymphoma based on current available data, including how to utilize FDG-PET to guide therapy. Ongoing clinical trials aimed at optimizing therapy with the dual goal of maximal efficacy and minimal toxicity will also be summarized.
Dr. Kristie Blum will summarize the encouraging preliminary results of several phase I and II trials of new agents for Hodgkin lymphoma, including second generation anti-CD30 antibodies, the CD30 targeted antibody drug conjugate brentuximab vedotin, lenalidomide, and MTOR and HDAC inhibitors. Ongoing efforts to incorporate these agents into earlier lines of therapy, either as single agents or in combination, will be described.
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Clinical Impact of Targeted Therapies in Myeloproliferative Neoplasms
| Chair: |
Ruben A. Mesa, MD, Mayo Clinic, Scottsdale, AZ |
| Speakers: |
Jerald Radich, MD, Fred Hutchinson Cancer Research Center, Seattle, WA
CML 2010: Where Are We Now and Where Can We Go? |
Claire N. Harrison, DM, Guy’s and St. Thomas NHS Foundation Trust, London, United Kingdom
ET/PV: Rethinking Disease Definitions and Therapeutic Strategies |
Ruben A. Mesa, MD, Mayo Clinic, Scottsdale, AZ
Assessing New Therapies and Their Overall Impact in Myelofibrosis |
A half-century has passed since the discovery of the Philadelphia chromosome in chronic myeloid leukemia (CML), and we are now five years after the discovery of JAK2 V617F in Philadelphia chromosome-negative myeloproliferative neoplasms (MPNs). What impact are those seminal discoveries having on the care of MPN patients in 2010? In CML, the BCR-ABL tyrosine kinase inhibitors led to hematologic and molecular response in most patients. In contrast, we are still in early phases of targeted therapy with BCR-ABL-negative MPNs (essential thrombocythemia [ET], polycythemia vera [PV], and myelofibrosis [MF]). Initial results with JAK2 inhibitors suggest some clinical benefits, but responses are incomplete. The parallel therapeutic developments in these two related groups of disorders highlight the opportunities and challenges of targeted therapies for hematologic neoplasms.
Dr. Jerald Radich will discuss the optimization of tyrosine kinase inhibition in chronic-phase CML, what we should do for patients who need second-line therapy, and lastly, what the unanswered questions are in CML in 2010.
Dr. Claire Harrison will discuss the impact of molecular markers upon the current classification of ET and PV, the indications for cytoreduction, the potential role of interferon as a disease-modifying therapy, and what impact JAK2 inhibitors are currently having in these disorders.
Dr. Ruben Mesa will discuss the modern assessment of prognosis in MF, the indications for allogeneic stem cell transplant and whether the JAK2 inhibitors affect this decision, and the impact of investigative therapies and how we can achieve complete remission in MF.
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Understanding and Managing Ultra High-Risk Hematologic Malignancies
| Chair: |
Elihu H. Estey, MD, University of Washington, Fred Hutchinson Cancer Research Center, Seattle, WA |
| Speakers: |
Hervé Avet-Loiseau, MD, PhD, University Hospital of Nantes, Nantes, France
Ultra High-Risk Multiple Myeloma |
Stephan Stilgenbauer, MD, University of Ulm, Ulm, Germany
Ultra High-Risk CLL |
Elihu H. Estey, MD, University of Washington, Fred Hutchinson Cancer Research Center, Seattle, WA
High Cytogenetic/Molecular Genetic Risk AML |
Dr. Hervé Avet-Loiseau will discuss identification of the 15-20 percent of myeloma patients who can be classified as ultra high-risk, based for example on high b2-microglobulin, renal insufficiency, proliferative disease, specific chromosomal changes such as t(4;14) and del(17p), and genomic aberrations. He will review recent data suggesting that specific management may at least improve progression-free survival. Dr. Stephan Stilgenbauer will review current data on molecular mechanisms underlying refractory chronic lymphocytic leukemia (CLL). He will discuss how to use our understanding of the biology of refractory CLL (e.g., with p53 defects) to develop targeted treatment options.
Dr. Stilgenbauer will also address the practical problem of defining “ultra high-risk CLL” clinically, for example, based on response to therapy (including minimal residual disease) in the era of modern CLL treatment, including chemoimmunotherapy (e.g., FCR). Lastly, he will review practical aspects of management of these patients, focusing on novel biological agents and stem cell transplantation.
Dr. Elihu Estey will discuss how cytogenetics (particularly the new category “monosomal karyotype”) and, with increasing frequency, various molecular genetic (e.g., FLT3, NPM1, CEBPA, WT1, miR-181a) or epigenetic markers interact with age to define ultra high-risk acute myelogenous leukemia. After reviewing the current role of allogeneic hematopoietic cell transplant (HCT) in ultra high-risk patients, he will discuss newer therapies that may modulate these markers, particularly when combined with chemotherapy or used to prevent relapse after HCT.
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Parenteral Iron Therapy - Taking the Rust Off
| Chair: |
John W. Adamson, MD, University of California - San Diego, San Diego, CA |
| Speakers: |
Michael Auerbach, MD, Auerbach Hem.-Onc. Associates, Inc., Baltimore, MD
Preparations and Safety |
David H. Henry, MD, Pennsylvania Hospital, Philadelphia, PA
Use in Cancer-Associated Anemia |
Jeffrey Carson, MD, University of Medicine and Dentistry of New Jersey’s Robert Wood Johnson Medical School, Piscataway, NJ
Role in Heart Failure |
Several new parenteral iron preparations have become available in the United States and Europe over the past few years, and others are being studied in clinical trials. This has provided clinicians with multiple options for treating iron deficiency or other disorders in which parenteral iron has been shown to be of benefit – including potential new indications that draw on properties of iron not yet fully understood. These developments have led to questions about the relative safety of the available products, how iron might best be used in combination with erythropoiesis-stimulating agents (ESAs) to possibly ameliorate the risks of ESA treatment, and novel clinical settings in which the effect of iron goes beyond hemoglobin production. This session will provide an update on available iron products and their application for both recognized and novel clinical indications.
Dr. Michael Auerbach will discuss the formulations of the available iron products, their safety profiles, evolving patterns of their clinical use, and other products that are under development.
Dr. David Henry will discuss the use of parenteral iron products in combination with ESAs and how this might affect the risk/benefit profile in patients with chemotherapy-induced anemia.
Dr. Jeffrey Carson will discuss the recent use of parenteral iron in patients with iron deficiency and congestive heart failure and the possible broader applications of parenteral iron treatment.
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Marrow Responses to Aging and Inflammation
| Chair: |
Nancy Berliner, MD, Brigham and Women's Hospital, Boston, MA |
| Speakers: |
Cindy N. Roy, PhD, The Johns Hopkins School of Medicine, Baltimore, MD
Anemia of Inflammation |
Nancy Berliner, MD, Brigham and Women’s Hospital, Boston, MA
Anemia of Aging |
Eileen P. Smith, MD, City of Hope National Medical Center, Duarte, CA
Hematologic Disorders After Solid Organ Transplantation |
This session will focus on new insights into the pathogenesis of hematologic abnormalities associated with aging and inflammation. Special emphasis will be placed on advances in our understanding of iron metabolism and its relationship to anemia in the settings of inflammatory disease and aging, as well as the impact of both inflammatory and non-inflammatory complications of organ transplantation on hematologic outcomes.
Dr. Cindy Roy will review the role of hepcidin in the pathogenesis of the anemia of inflammation. Hepcidin is a peptide hormone produced by the liver that regulates iron absorption and utilization by restricting iron egress from enterocytes, macrophages, and hepatocytes. She will discuss how hepcidin is regulated in response to numerous physiologic factors, especially inflammation, and how this peptide is transforming our understanding of the anemia of inflammation.
Dr. Nancy Berliner will review the epidemiologic features of anemia in the elderly. There is considerable evidence that aging is associated with increased pro-inflammatory cytokine expression and that genetic variation in the expression of these pro-inflammatory cytokines may influence many aspects of the aging process. The potential role of these pathways in unexplained anemia in the elderly will be discussed.
Dr. Eileen Smith will discuss hematologic disorders that arise as a consequence of solid organ transplantation. Although there are a myriad of hematologic abnormalities and complications that can occur in the transplant setting, she will concentrate on the unique issues influencing anemia and other cytopenias, as well as some of the more common coagulation disorders that may complicate solid organ transplantation.
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Consultative Hematology: Hemostasis and Thrombosis
| Chair: |
Agnes Y. Lee, MD, MSc, University of British Columbia, Vancouver, BC, Canada |
| Speakers: |
Andreas Greinacher, MD, Institute for Immunology & Transfusion, Greifswald, Germany
Thrombocytopenia in the ICU |
Agnes Y. Lee, MD, MSc, University of British Columbia, Vancouver, BC, Canada
Thrombosis in Cancer Patients |
Lawrence Goodnough, MD, Stanford University School of Medicine, Palo Alto, CA
Use of Factor VIIa |
This session will focus on three common challenges faced by the consultant hematologist: thrombocytopenia in the intensive care unit, thrombosis in cancer patients, and the use of factor VIIa. Practical and evidence-based approaches will be presented to highlight new evidence and controversies. Thrombocytopenia occurs in 35-45 percent of patients admitted to the intensive care unit, and the patient’s platelet count correlates inversely with the risk of adverse outcomes. The etiology ranges from sepsis, massive hemorrhage and transfusion, thrombotic thrombocytopenic purpura, and heparin-induced thrombocytopenia.
Dr. Andreas Greinacher will provide a framework – based on the dynamic changes in the platelet count – to address the major diagnostic and therapeutic challenges the hematology consultant faces in these patients. Venous thrombosis is associated with morbidity and increased mortality in patients with cancer. Recent research has focused on development of risk-assessment models to identify patients who may benefit from primary prophylaxis, treatment of recurrent thrombosis, and potential antineoplastic effects of anticoagulants.
Dr. Agnes Lee will provide an update of the evidence and apply it to everyday clinical challenges. Recombinant factor VIIa (rFVIIa) is FDA-approved for use in the setting of hemorrhage associated with factor VIII or factor IX inhibitors in patients with congenital or acquired hemophilia. Prominent off-label indications include the management of patients experiencing massive and uncontrolled hemorrhage, non-traumatic intracranial hemorrhage, cardiothoracic surgery, organ transplantation, and general surgery.
Dr. Lawrence Goodnough will review existing patterns of off-label rFVIIa use and address the evidence regarding the efficacy and safety of this important, but controversial, hemostatic agent.
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Consultative Hematology: The Pregnant Patient
| Chair: |
Marc A. Rodger, MD, The Ottawa Hospital Research Institute, Ottawa, ON, Canada |
| Speakers: |
Marc A. Rodger, MD, The Ottawa Hospital Research Institute, Ottawa, ON, Canada
Venous Thromboembolism |
Shannon M. Bates, MD, MSC, McMaster University, Hamilton, ON, Canada
Recurrent Pregnancy Loss |
Gideon Koren, MD, University of Toronto, Toronto, ON, Canada
Pregnancy and Commonly Used “Hematologic Drugs” |
This session will focus on recent advances in the area of consultative hematology of the pregnant patient. The level of consultative complexity is increased in pregnancy by evolving physiologic changes in expectant mothers, the effects of diagnostic and therapeutic management on the unborn child, and, often, a lack of validated management strategies in pregnancy.
Dr. Marc Rodger will review developments in the pathophysiology, diagnosis, and treatment of venous thromboembolism (VTE) in pregnancy. He will highlight the poverty of research upon which to base clinical recommendations for this common problem in pregnancy, yet offer practical approaches to prevention, diagnosis, and therapeutic management of pregnancyassociated VTE.
Dr. Shannon Bates will review the hematologist’s role in the etiologic and therapeutic management of pregnancy loss. She will delve into the controversial association between acquired and inherited thrombophilias and pregnancy loss. She will also review the evidence supporting or refuting the role of antithrombotics in the prevention of pregnancy loss.
Dr. Gideon Koren will describe how the safety of medication used in pregnancy is assessed, reported, and used in clinical practice. He will specifically review commonly used hematologic medications and diagnostic and therapeutic radiation in women of childbearing potential and in pregnancy. He will focus on common hematologic scenarios in pregnancy and common medications used in these scenarios (e.g., antithrombotics, corticosteroids, hydroxyurea, analgesics, and antibiotics). He will touch on high-stakes, but thankfully rarely used, medications in pregnancy (e.g., chemotherapy, tyrosine kinase inhibitors).
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Sickle Cell
| Chair: |
Wally R. Smith, MD, Virginia Commonwealth University, Richmond, VA |
| Speakers: |
Nigel S. Key, MBChB, University of North Carolina, Chapel Hill, NC
Sickle Trait: Novel Clinical Significance |
Wally R. Smith, MD, Virginia Commonwealth University, Richmond, VA
Sickle Cell Pain: Current Therapy |
Z. Jim Wang, PhD, University of Illinois at Chicago, Chicago, IL
Novel Mechanisms of Sickle Cell Pain |
This session will focus on recent advances in our understanding of the clinical manifestations of sickle cell trait and sickle cell disease, with a particular emphasis on new understandings of the significance of sickle cell trait and new clinical and basic science revelations about pain in sickle cell disease.
Dr. Nigel Key will review the clinical and public health implications of complications of sickle cell trait prompted by a new assessment of these risks.
Dr. Wally Smith will review important new epidemiologic data on the most important symptom of sickle cell disease, pain, taken from his and others’ studies, and discuss their implications for research, clinical care, and public health and public policy.
Dr. Jim Wang will describe novel research showing mechanisms of pain that have not previously been seen as applicable to sickle cell patients, but which may be applicable.
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Thalassemia
| Chair: |
Ellis J. Neufeld, MD, PhD, Children's Hospital, Boston, MA |
| Speakers: |
Ellis J. Neufeld, MD, PhD, Children’s Hospital, Boston, MA
Iron Chelators |
Emanuele Angelucci, MD, Ospedale Oncologico “Armando Businco,” Cagliari, Italy
Transplantation |
Punam Malik, MD, Cincinnati Children’s Hospital, Cincinnati, OH
Gene Therapy |
This session will focus on recent advances in clinical and experimental therapies in thalassemia. Details on current and future curative therapies, including hematopoietic stem cell transplant (HSCT) and gene therapy, will be featured, along with an update on iron chelation.
Dr. Ellis Neufeld will provide an update on current use of the iron chelators deferoxamine, deferasirox, and deferiprone. Although several recent clinical trials further elucidate the potential strengths of these drugs, there remain major challenges to understanding optimal chelator usage. Iron-related end-organ damage and cardiac mortality remain major problems. Inadequate response to deferasirox is a common problem with a possible biological basis.
After a brief review of the history of HSCT in thalassemia, Dr. Emanuele Angelucci will critically review recent results obtained by several groups worldwide, using both experimental approaches and current standard care models. Different preparative regimen strategies and outcomes will be described for the several different categories of eligible patients. Information derived from the HSCT experience will inform future gene therapy approaches. Finally, the role of HSCT in the era of oral chelators will be discussed with particular attention to different countries’ thalassemia epidemiology and resource availability.
Dr. Punam Malik will review the development of additive genetic therapy approaches using lentivirus vectors, the research developments in laboratories that are close to translating the research into the clinic, the currently open clinical trial, and alternative approaches to achieving genetic correction, including homologous recombination with reprogramming to iPS cells and back.
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Aplastic Anemia
| Chair: |
Neal S. Young, MD, National Institutes of Health, Bethesda, MD |
| Speakers: |
Neal S. Young, MD, National Institutes of Health, Bethesda, MD
Etiology |
Jakob R. Passweg, MD, MS, Hopitaux Universitaire de Genève, Geneva, Switzerland
Immunosuppression Versus Transplant |
Mary M. Horowitz, MD, MS, Center for International Blood and Marrow Transplant Research, Medical College of Wisconsin, Milwaukee, WI
Transplantation With Alternative Donors |
Current concepts of pathophysiology and results of definitive therapies for aplastic anemia will be reviewed. For mechanisms, emphasis will be on the role of genetic defects in telomere maintenance and repair and the relationship of telomere attrition to immune-mediated hematopoietic cell destruction and to late clonal evolution. For treatments, recent results of clinical trials and retrospective series for various immunosuppressive regimens will be compared, as well as prognostic indicators related to outcomes. The current state of alternative donor sources for transplantation to cure aplastic anemia, with their promises and problems, will be critically assessed. Speakers will speculate on future directions in aplastic anemia research in the laboratory and clinic.
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Issues in Thrombosis Management and Anticoagulation
| Chair: |
Kenneth A. Bauer, MD, Beth Israel Deaconess Medical Center, Boston, MA |
| Speakers: |
Jack Ansell, MD, Lenox Hill Hospital, New York, NY
Warfarin Versus New Agents: Interpreting the Data |
Susan Kahn, MD, MSc, Jewish General Hospital, Montreal, QC, Canada
Post-Thrombotic Syndrome |
Kenneth A. Bauer, MD, Beth Israel Deaconess Medical Center, Boston, MA
Duration of Anticoagulation |
The approval of new oral anticoagulants that do not require laboratory monitoring will open a new era in the prevention and treatment of venous thromboembolism (VTE) and stroke prevention in atrial fibrillation. Challenges will occur when recurrences or major bleeds occur on the new agents. The post-thrombotic syndrome (PTS) is a common, chronic, burdensome, and costly complication of deep-vein thrombosis (DVT). There remains a continuing controversy regarding the need for long-term anticoagulation for patients with a first episode of unprovoked VTE.
Dr. Jack Ansell will provide an update on the most recent results of phase III studies with new oral anticoagulants. Their significance and potential impact compared with standard warfarin therapy will be discussed along with how these new agents will affect the everyday life of physicians and patients.
Dr. Susan Kahn will discuss the relationship between DVT and PTS, highlighting recent work on its epidemiology, classification, prevention, and management. Ongoing trials and areas for future research will also be discussed.
Dr. Kenneth Bauer will present data regarding the recurrence risk in patients with a first episode of VTE and the benefits/risks of long-term warfarin therapy. Data regarding the use of the laboratory to risk-stratify patients with respect to recurrence will be discussed along with the impact of residual thrombus on imaging studies or having a non-removable inferior vena cava filter.
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Hemophilia
| Chair: |
Claire S. Philipp, MD, University of Medicine and Dentistry of New Jersey's Robert Wood Johnson Medical School, New Brunswick, NJ |
| Speakers: |
Claire S. Philipp, MD, University of Medicine and Dentistry of New Jersey’s Robert Wood Johnson Medical School, New Brunswick, NJ
Aging Hemophiliacs |
Steven W. Pipe, MD, University of Michigan, Ann Arbor, MI
New Protein Therapeutics |
Robert R. Montgomery, MD, Blood Research Institute, Medical College of Wisconsin, Milwaukee, WI
Gene Therapy |
Advances in coagulation protein replacement therapy for people with congenital hemophilia A and B have led to progressive reductions in morbidity and increases in life expectancy. Recombinant and high purity plasma-derived factor products are currently safe and effective for the management of hemophilia. Modification of recombinant factors with enhanced biologic properties are being explored and show promise to improve hemophilia treatment. Gene therapy strategies to safely modify the hemophilia phenotype are in investigation and remain a long-term goal for clinical application. This session will focus on complications and comorbidities in the aging hemophilia patient, advances in coagulation protein therapies, and targeted gene therapy for hemophilia A and B.
Dr. Claire Philipp will describe the changing demographics of hemophilia A and B and the impact of increased life expectancy on hemophilia-related complications and age-associated comorbidities. Challenges in hemophilia treatment and management of comorbidities in the older adult hemophilia patients will be discussed.
Dr. Steven Pipe will provide an overview of new coagulation protein therapeutics bioengineered to increase circulating half-life, reduce immunogenicity, and improve the efficiency of biosynthesis, as well as other novel areas of investigation examining alternative therapeutic agents.
Dr. Robert Montgomery will discuss recent data on targeted gene therapy of hemophilia A and B. Initial strategies focusing on the restoration of plasma factor VIII and factor IX and novel approaches targeting expression of factor VIII and factor IX in megakaryocytes and endothelial cells will be discussed. Studies in hemophilia mice demonstrating differences in clinical efficacy will be presented.
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Platelet Disorders
| Chair: |
Douglas B. Cines, MD, Hospital of the University of Pennsylvania, Philadelphia, PA |
| Speakers: |
Douglas B. Cines, MD, Hospital of the University of Pennsylvania, Philadelphia, PA
ITP |
Keith R. McCrae, MD, Case Western Reserve University, Cleveland, OH
Platelets in Pregnancy – Numbers and Function |
Lawrence F. Brass, MD, PhD, University of Pennsylvania, Philadelphia, PA
Platelet Function Disorders |
This session will focus on recent advances in the pathophysiology, assessment, and management of common disorders of platelet numbers and function, including immune thrombocytopenic purpura (ITP), thrombocytopenia during pregnancy, and qualitative platelet disorders.
Dr. Douglas Cines will review recent developments in ITP, including changes in nomenclature and demographics, diversity in pathogenesis (including the role of infection, T-cells, and inhibition of platelet production), and recommendations from the International Consensus Report on diagnosis. He will review attempts to alter the natural history of ITP with new approaches to initial therapy, revisit splenectomy, and provide an update on the results of trials evaluating thrombopoietin receptor agonists.
Dr. Keith McCrae will review the differential diagnosis of thrombocytopenia in pregnancy, including gestational thrombocytopenia, ITP, preeclampsia, HELLP syndrome, thrombotic microangiopathies, and less common disorders with a focus on recent developments in the pathogenesis of preeclampsia.
Dr. Lawrence Brass will describe emerging ideas about platelet activation and thrombus formation at sites of vascular injury and disease. Focus will be on molecules on the platelet surface (some well known, others newly described) that regulate thrombus growth and stability. New ideas about the impact of antiplatelet agents will be discussed, as will congenital defects in platelet function and the evaluation of platelet function in the clinical setting.
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Transfusion Medicine: Transfusion Support in Trauma - Military and Civilian Approaches
This session is jointly sponsored with AABB.
| Chair: |
Thomas G. Deloughery, MD, Oregon Health and Science University, Portland, OR |
| Speakers: |
John B. Holcomb, MD, University of Texas Medical School at Houston, Houston, TX
Transfusion in Polytrauma |
Timothy Hannon, MD, MBA, St. Vincent Hospital, Indianapolis, IN
Avoiding the Collateral Damage of Trauma Resuscitation Protocols |
Thomas G. Deloughery, MD, Oregon Health and Science University, Portland, OR
Transfusion Support for Trauma Services |
Traditionally, the approach of the transfusion service in blood support of the massive transfusion trauma patient has been based on blood product selection dictated by laboratory testing. This method has been challenged in recent years by data suggesting that a “fixed-ratio” approach – for example, giving one unit of plasma for every unit of red cells – may result in improved outcomes. This session will attempt to summarize the latest thinking on transfusion support for trauma patients and provide guidance to hematologists who are assisting their institutions in starting/improving massive transfusion protocols.
Dr. John Holcomb will review both military and trauma data and give evidence to support a more aggressive approach to plasma and platelet therapy in blood resuscitation of trauma patients.
Dr. Timothy Hannon will supply evidence for a more cautious approach to transfusion therapy and review complications of transfusion such as transfusion-related lung injury and immune modulation.
Dr. Thomas DeLoughery will discuss the importance of every institution – large or small – having massive transfusion protocols, and how to provide laboratory and transfusion services to support the trauma patient.
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Grant Writing for Junior Faculty
This session is only offered once.
| Chair: |
David A. Williams, MD, Children's Hospital Boston, Boston, MA |
| Speakers: |
David A. Williams, MD, Children’s Hospital Boston, Boston, MA
Grant Writing: General Programmatic Grants |
Stephanie J. Lee, MD, MPH, Fred Hutchinson Cancer Research Center, Seattle, WA
Grant Writing: Clinical Applications |
Jeffrey J. Molldrem, MD, The University of Texas M. D. Anderson Cancer Center, Houston, TX
Grant Writing: Basic/Translational Applications |
This program will feature expert faculty presentations concerning optimal approaches to successful preparation of clinical, individual basic/translational research grants, and programmatic grants. The faculty will present short talks outlining their own successes in previous applications and noting the “new” National Institutes of Health format for grant applications. Representatives of the National Institutes of Health will be available to answer questions. Ample time for questions and answers will be provided.
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Junior Faculty Development: The Economics of Academic Hematology
This session is only offered once.
| Chair: |
Navneet S. Majhail, MD, MS, University of Minnesota, Minneapolis, MN |
| Speakers: |
Ellis J. Neufeld, MD, PhD, Children’s Hospital, Boston, MA
The Costs and Benefits of Your Grant to Your Institution |
Stephen Hunger, MD, University of Colorado College of Medicine, Aurora, CO
Show Me the Money: Negotiating Your Salary and Benefits |
Robert F. Todd III, MD, PhD, Baylor College of Medicine, Houston, TX
Justifying Your Existence: It’s More Than Your Salary |
In addition to excellence in clinical, research, and teaching pursuits, a good understanding of the economic aspect of academic medicine is necessary for personal satisfaction and a successful career in academic hematology.
Dr. Ellis Neufeld will provide his perspective on the costs and benefits of grants to a grantee’s institution. He will review the streams of expenses and revenues associated with grants and the direct and indirect cost components of a grant budget.
Dr. Stephen Hunger will review components of a start-up and compensation package for junior faculty hires and will discuss strategies to negotiate salary, benefits, and effort.
Dr. Robert Todd will review various revenue streams for generating and maintaining salary support and will discuss other service and leadership roles that faculty may pursue to support their academic mission.
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