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3 P's In a Pod
| Session Offered Twice |
December 8, 2012, 4:00 PM - 5:30 PM
Georgia World Congress Center (A101, Building A, Level 1, Georgia World Congress Center) | December 10, 2012, 2:45 PM - 4:15 PM
Georgia World Congress Center (A103, Building A, Level 1, Georgia World Congress Center) |
This session will consist of a potpourri of pathologic processes and procedures of particular interest to the practicing hematologist, all involving the letter “P”!
Dr. Gregory Grabowski will review Gaucher disease as the prototype for lysosomal storage diseases (LSD) such as Fabry, Pompe, and Niemann-Pick diseases. He will also discuss the clinical and economic success of enzyme replacement therapy in this disorder-stimulated research, development in these diseases, and the recognition of disruption of autophagy/lysosomal function in other diseases, such as age-related degenerative diseases and idiopathic hypertrophic cardiomyopathy.
Dr. Robert Desnick will review the porphyrias, focusing on congenital erythropoietic porphyria (CEP), erythropoietic protoporphyria (EPP), and the recently identified X-linked protoporphyria (XLP). The diagnosis, treatment, and results of recent clinical trials will be presented. Dr. Desnick will also discuss chronic phlebotomy for Porphyria Cutanea Tarda and hematin and liver transplantation for the acute neurologic attacks in acute hepatic porphyrias.
Dr. Jeffrey Winters will review basic concepts and mechanisms of plasma exchange. He will discuss issues that must be considered when prescribing or performing plasma exchange in order to ensure safe and effective therapy. He will review the “Guidelines on the Use of Therapeutic Apheresis in Clinical Practice–Evidence-Based Approach from the Apheresis Applications Committee of the American Society for Apheresis” as a practical tool for determining the role of plasma exchange in disease treatment.
Chair:
Jeffrey Lawrence Winters, MD
Mayo Clinic, Rochester, MN, USA
Speakers:
Gregory Grabowski, MD
Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA
Philippe Gaucher Disease and Other Storage Disorders
Robert J. Desnick, MD, PhD
Mount Sinai School of Medicine, New York, NY, USA
Porphyrias: Advances in Diagnosis and Treatment of the “Purple” Diseases
Jeffrey Lawrence Winters, MD
Mayo Clinic, Rochester, MN, USA
Plasma Exchange: Concepts, Mechanisms, and an Overview of the American Society for Apheresis Guidelines
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Acute Myeloid Leukemia: Newly Discovered Genes, Screens (for Minimal Residual Disease), and Therapeutic Means
| Session Offered Twice |
December 8, 2012, 9:30 AM - 11:00 AM
Georgia World Congress Center (Thomas Murphy Ballroom 3-4, Building B, Level 5, Georgia World Congress Center) | December 9, 2012, 7:30 AM - 9:00 AM
Georgia World Congress Center (Thomas Murphy Ballroom 3-4, Building B, Level 5, Georgia World Congress Center) |
Recent clinical, genetic, and functional studies have improved our understanding of the biology of acute myeloid leukemia(AML) and allowed for the development of biomarkers to predict relapse and response to therapy, the implementation of approaches to monitor minimal residual disease (MRD) during and after dose-intensive therapy, and the development of novel therapies for patients with refractory AML.
Dr. Ross Levine will review our current understanding of the molecular genetics of AML and how cytogenetic, genetic, and epigenetic studies can be used to improve prognostication in AML and to predict response to therapy.
Dr. Elisabeth Paietta will review the current genetic and flow cytometric approaches to monitoring minimal residual disease in AML and the data indicating how MRD assessment can be used to inform clinical decisions.
Dr. Jeffrey Szer will review the recent development of novel chemotherapeutic and targeted therapies for patients with refractory AML and how biologic insights are being translated to the clinic using novel therapeutic strategies.
Chair:
Ross L. Levine
Memorial Sloan-Kettering Cancer Center, New York, NY, USA
Speakers:
Ross L. Levine
Memorial Sloan-Kettering Cancer Center, New York, NY, USA
How Do Novel Molecular Genetic Markers Influence Treatment Decisions in Acute Myeloid Leukemia?
Elisabeth Paietta, PhD
The North Division of Montefiore Medical Center, Bronx, NY, USA
Minimal Residual Disease in Acute Myeloid Leukemia: Coming of Age
Jeffrey Szer, MBBS
The Royal Melbourne Hospital, Parkville, Victoria, Australia
The Prevalent Predicament of Relapsed Acute Myeloid Leukemia
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Advances in the Pathogenesis and Treatment of Myelodysplastic Syndromes
| Session Offered Twice |
December 8, 2012, 7:30 AM - 9:00 AM
Georgia World Congress Center (Thomas Murphy Ballroom 1-2, Building B, Level 5, Georgia World Congress Center) | December 8, 2012, 2:00 PM - 3:30 PM
Georgia World Congress Center (Thomas Murphy Ballroom 1-2, Building B, Level 5, Georgia World Congress Center) |
During the past decade myelodysplastic syndromes (MDS) have emerged as a major research focus for the global hematology community. In addition to the significant advances in their diagnosis, prognostic evaluation, and treatment, there has been an explosion in the characterization of molecular defects. These include the consequences of RPS14 gene deletions in 5q- syndrome, the significance of p53 aberrations, mutations of spliceosome complex genes (such as SF3B1 in refractory anemia with ring sideroblasts), and a plethora of other genetic abnormalities that either individually or collectively alter the hemopoietic differentiation and propensity to leukemic transformation.
The spectrum of biological significance of the mutations and their genetic and epigenetic consequences will be discussed by Dr. Maria Figueroa.
Relationships between the genetic and epigenetic changes and the effects of DNMT and HDAC inhibition will be discussed by Dr. Valeria Santini.
Allogeneic hemopoietic stem cell transplantation in MDS has come of age. Dr. Ghulam Mufti will discuss how transplant timing, pre-transplant preparative regimens, conditioning regimens, and post-transplant therapies have rapidly evolved to promote an increasing disease-free survival and possibly a cure in up to 40 percent of transplanted patients. He will also discuss the remaining challenges, both in terms of identifying key genetic events and expanding the therapeutic repertoire that will eventually lead to a prolongation in survival beyond what is achieved by currently available treatments.
Chair:
Ghulam J. Mufti, MD, PhD
King's College Hospital NHS Foundation Trust, London, United Kingdom
Speakers:
Maria E. Figueroa, MD
University of Michigan, Ann Arbor, MI, USA
Interpreting New Molecular Genetics in Myelodysplastic Syndromes
Valeria Santini, MD
University of Florence Azienda Ospedaliero-Universitaria Careggi, Florence, Italy; University of Florence, Florence, Italy
Novel Therapeutic Strategies: Hypomethylating Agents and Beyond
Ghulam J. Mufti, MD, PhD
King's College Hospital NHS Foundation Trust, London, United Kingdom
Myelodysplastic Syndromes: Who and When in the Course of Disease to Transplant
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Chronic Lymphocytic Leukemia: Can New Prognostic Factors Guide New Therapeutic Approaches?
| Session Offered Twice |
December 8, 2012, 9:30 AM - 11:00 AM
Georgia World Congress Center (Hall B5, Building B, Level 1, Georgia World Congress Center) | December 9, 2012, 7:30 AM - 9:00 AM
Georgia World Congress Center (Hall B5, Building B, Level 1, Georgia World Congress Center) |
This session will focus on recent advances and new concepts in chronic lymphocytic leukemia (CLL). The existence of several prognostic makers, the advent of targeted therapies, and the possibility of accurately assessing the depth of treatment responses have increased the complexity of managing CLL patients while at the same time providing tools for future progress toward the eradication of the disease.
Dr. Chiorazzi will address the biologic underpinnings of several existing prognostic markers. He will draw these concepts from laboratory and clinical data that have emerged over the past decade.
Dr. Adrian Wiestner will discuss the emerging role of kinase inhibitors in the treatment of CLL. He will introduce the pathogenic pathways targeted by novel small-molecule drugs in current clinical testing. He will then review the results from clinical studies using kinase inhibitors and discuss the promise and challenges of incorporating these agents into treatment strategies.
Dr. Paolo Ghia will discuss the future of CLL prognostication based on evidence that the quality of response to therapy is an independent predictor of overall survival. He will briefly review the methods for quantification of minimal residual disease and will discuss their potential use to predict prognosis and guide treatment.
Chair:
Nicholas Chiorazzi, MD
The Feinstein Institute for Medical Research, North Shore-LIJ Health System, Manhasset, NY, USA; The Feinstein Institute for Medical Research - NSLIJHS, Manhasset, NY, USA
Speakers:
Nicholas Chiorazzi, MD
The Feinstein Institute for Medical Research, North Shore-LIJ Health System, Manhasset, NY, USA; The Feinstein Institute for Medical Research - NSLIJHS, Manhasset, NY, USA
Implications of New Prognostic Markers in Chronic Lymphocytic Leukemia
Adrian Wiestner, MD, PhD
National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD, USA
Emerging Role of Kinase Targeted Strategies in Chronic Lymphocytic Leukemia
Paolo Ghia
Università Vita-Salute San Raffaele and Istituto Scientifico San Raffaele, Milano, Italy
A Look Into The Future: Can Minimal Residual Disease Guide Therapy And Predict Prognosis in Chronic Lymphocytic Leukemia?
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Chronic Myeloid Leukemia: The Pristine Paradigm for Successful Targeted Therapy
| Session Offered Twice |
December 8, 2012, 2:00 PM - 3:30 PM
Georgia World Congress Center (Hall B5, Building B, Level 1, Georgia World Congress Center) | December 9, 2012, 9:30 AM - 11:00 AM
Georgia World Congress Center (Hall B5, Building B, Level 1, Georgia World Congress Center) |
An expanding range of highly effective BCR-ABL1 kinase inhibitors are currently available or are undergoing clinical trial for patients with chronic myeloid leukemia (CML), either as first-line therapy or after treatment failure. While there is now a welcome range of options for clinicians, the question of which inhibitor to choose for individual patients remains unanswered. Long-term follow-up suggests that a small proportion of patients are candidates for treatment discontinuation without disease recurrence. The new inhibitors may increase the frequency of safe discontinuation. As some patients reach their second decade of targeted therapy, long-term effective molecular monitoring remains important to confirm adherence and is essential when considering treatment discontinuation.
Dr. David Marin will outline the therapeutic options for newly diagnosed patients and review the relative merits of the various kinase inhibitors. He will discuss whether more potent inhibitors offer an advantage in terms of transformation-free survival and deeper molecular responses compared with imatinib, which is still the recommended first-line therapy.
Dr. Francois-Xavier Mahon will discuss whether cure of CML is possible for some patients treated with kinase inhibitors. He will describe the new initiatives to increase the frequency of safe treatment discontinuation, and he will outline the risks of stopping therapy and the factors that independently predict for molecular disease recurrence and their potential biological basis.
Dr. Susan Branford will review the role of continued therapeutic response monitoring in optimal responders. She will discuss the difficulties of standardizing the analysis of deep molecular response, whether BCR-ABL1 mutation analysis is necessary in some circumstances, and whether changes in the molecular response can serve as a marker of non-adherence to therapy regimens.
Chair:
Susan Branford, PhD
University of Adelaide, Adelaide, Australia
Speakers:
David Marin
Imperial College, London, United Kingdom
Initial Choice of Therapy among Plenty For Newly Diagnosed Chronic Myeloid Leukemia
Francois-Xavier Mahon, MD, PhD
Hôpital Pellegrin, CHU de Bordeaux, Bordeaux, France
Is Going for Cure in Chronic Myeloid Leukemia Possible and Justifiable?
Susan Branford, PhD
University of Adelaide, Adelaide, Australia
Monitoring After Successful Therapy for Chronic Myeloid Leukemia
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Dynamic Discoveries and Directions in Pediatric Leukemias
| Session Offered Twice |
December 8, 2012, 7:30 AM - 9:00 AM
Georgia World Congress Center (A101, Building A, Level 1, Georgia World Congress Center) | December 10, 2012, 7:00 AM - 8:30 AM
Georgia World Congress Center (A103, Building A, Level 1, Georgia World Congress Center) |
Acute leukemia is one of the most curable malignances in children. While outcomes for newly diagnosed disease have now improved significantly, subgroups of patients with a high risk for treatment failure remain, and challenges include both how to integrate evolving new discoveries into clinical practice to identify these high-risk patients and how to improve their outcomes. Detection of minimal residual disease (MRD) has evolved as one of the most powerful tools in the risk assessment of acute leukemia. Clinical application of this highly sensitive tool, particularly in acute lymphocytic leukemia (ALL) requires reliable, reproducible, and quality-assured methods to ensure patient safety. To that end, the prognostic impact of MRD needs to be established prospectively in the context of clinical protocols.
Dr. Martin Schrappe will provide an overview of existing methods and evaluate the future role of this method in single and multiagent protocols.
Dr. Renier Brentjens will discuss the latest in cellular therapies for leukemia. An emphasis will be placed on adoptive therapy with T cells genetically modified to express tumor-specific chimeric antigen receptors (CARs). An update on currently ongoing clinical trials using this technology for B-cell malignancies will be included.
Dr. Elizabeth Raetz will highlight the outcome disparity that remains for children with relapsed ALL. Current strategies for treating relapsed ALL and the potential for using early measures of disease response to prioritize new agents and to allocate therapy will be discussed. Examples of how laboratory discoveries can inform clinical trial design will also be explored.
Chair:
Elizabeth A. Raetz, MD
New York University Medical Center, New York, NY, USA
Speakers:
Martin Schrappe, MD
University Medical Center Schleswig-Holstein, Kiel, Germany
Minimal Residual Disease: Optimal Methods, Timing, and Clinical Relevance for an Individual Patient
Renier J. Brentjens, MD, PhD
Memorial Sloan Kettering Cancer Center, New York, NY, USA
Novel Cellular Therapies for Leukemia: CAR-Modified T-cells Targeted to the CD19 Antigen
Elizabeth A. Raetz, MD
New York University Medical Center, New York, NY, USA
Where Do We Stand in the Treatment of Relapsed Acute Lymphoblastic Leukemia?
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Everyday Bleeding Disorders
| Session Offered Twice |
December 8, 2012, 7:30 AM - 9:00 AM
Georgia World Congress Center (A411-A412, Building A, Level 4, Georgia World Congress Center) | December 8, 2012, 2:00 PM - 3:30 PM
Georgia World Congress Center (A411-A412, Building A, Level 4, Georgia World Congress Center) |
This session will focus on three commonly encountered challenges in the evaluation of patients with bleeding symptoms. The collection and interpretation of bleeding histories remains a subjective process.
Dr. Sarah O’Brien will describe the development of standardized bleeding scores as tools for a more quantitative assessment of bleeding, and for identification of patients with higher likelihood of developing an underlying bleeding disorder. She will discuss recent research applications of bleeding scores in adult and pediatric hematology, as well as their potential utility in clinical practice. The diagnosis of von Willebrand disease (VWD) remains a challenge because no single test is entirely diagnostic.
Dr. Jorge DiPaola will discuss the molecular interactions of von Willebrand factor (vWF) that have fueled the recent development of tests that allow for more accurate diagnosis. He will describe the utility of genetic testing for VWD type 2 and 3 and new laboratory techniques that improve the accuracy of subtype diagnosis. Finally, he will discuss type 1 VWD and the diagnostic dilemmas that arise from the overlap of borderline low vWF levels and mild bleeding.
Dr. Pier Mannucci will review the mechanism of hemorrhagic complications in liver cirrhosis and renal insufficiency. He will review current evidence that improvement of anemia through use of erythropoietin in renal insufficiency has dramatically reduced bleeding tendencies. In liver disease, bleeding is not explained by abnormalities of routine coagulation tests because a decrease in natural anticoagulants compensates for the decrease of procoagulant factors. In both diseases, thrombosis is actually a problem more pertinent than bleeding.
Chair:
Sarah H O'Brien, MD
Nationwide Children's Hospital, Columbus, OH, USA
Speakers:
Sarah H O'Brien, MD
Nationwide Children's Hospital, Columbus, OH, USA
Bleeding Scores: Are They Really Useful?
Jorge Di Paola, MD
University of Colorado School of Medicine, Aurora, CO, USA
Making a Diagnosis Of Von Willebrand Disease
Pier Mannuccio Mannucci, MD
Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milano, Italy
Hemostatic Defects in Liver And Renal Dysfunction
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Evidence-Based Approaches to Cytopenias
| Session Offered Twice |
December 9, 2012, 7:30 AM - 9:00 AM
Georgia World Congress Center (B405-B407, Building B, Level 4, Georgia World Congress Center) | December 10, 2012, 10:30 AM - 12:00 PM
Georgia World Congress Center (B405-B407, Building B, Level 4, Georgia World Congress Center) |
This session will focus on recent advances on the diagnosis of neutropenia, chronic anemia, and thrombocytopenia. Emphasis will be placed on approaches for diagnostically difficult cases and how to resolve the diagnosis.
Dr. Laurence Boxer will review the approaches to establishing a diagnosis in challenging cases of acute and chronic neutropenia. He will focus on utilizing state-of-the-art cellular and molecular information to arrive at the cause of the neutropenia. Dr. Boxer will also discuss molecular diagnosis, mechanisms underlying neutropenia, cellular metabolism, and immunologic processes that suppress myeloid production or lead to neutrophil destruction.
Dr. Mark Koury will discuss a diagnostic approach to chronic anemia based on the evaluation of complete blood count, reticulocyte count, and red cell indices. The basic pathophysiological causes for the development of chronic anemia will be reviewed, and their effects on each of these laboratory results will be discussed. Examples of patient presentation, evaluations, and treatments will be presented.
Dr. Roberto Stasi will suggest a systematic diagnostic approach to challenging cases of thrombocytopenia. Dr. Stasi will focus on the interpretation of clinical findings as well as the relevance and appropriateness of diagnostic tests.
Chair:
Laurence A. Boxer, MD
University of Michigan, Ann Arbor, MI, USA
Speakers:
Laurence A. Boxer, MD
University of Michigan, Ann Arbor, MI, USA
How to Approach Neutropenia
Mark J. Koury, MD
Vanderbilt University, Nashville, TN, USA
How to Approach Chronic Anemia
Roberto Stasi, MD, PhD
St. George's Hospital, London, United Kingdom
How to Approach Thrombocytopenia
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Hematologic Diseases in Pregnancy
| Session Offered Twice |
December 8, 2012, 7:30 AM - 9:00 AM
Georgia World Congress Center (Sidney Marcus Auditorium, Building A, Level 4, Georgia World Congress Center) | December 8, 2012, 2:00 PM - 3:30 PM
Georgia World Congress Center (Sidney Marcus Auditorium, Building A, Level 4, Georgia World Congress Center) |
Diagnosis and management of hematologic disorders in the pregnant patient present special challenges with consideration for safety of both the woman and her fetus. Exacerbation may occur during pregnancy, and treatment goals change as pregnancy progresses and delivery nears. The fetus may be at risk of complications of the disease and its management, and may also require special monitoring before and after delivery. There is a marked lack of direct evidence and decisions based on extrapolation of data from non-pregnant cohorts and reported experience. New observations are expanding our understanding of pathologic mechanisms in pregnancy, improving management and opportunities for successful outcomes in “high-risk” pregnancies.
Dr. Terry Gernsheimer will discuss pathogenic mechanisms of thrombocytopenia and differential diagnosis of this common complication of pregnancy. Safety of the fetus limits therapeutic options and goals may be different than in the non-pregnant patient. A rational approach to risk assessment and management of both the patient and her offspring during pregnancy and delivery will be outlined.
Dr. Sophie Lanzkron will review maternal and fetal risks of pregnancy for individuals with ß thalassemia major and sickle cell disease. As treatment for individuals with hemoglobinopathies has improved and life expectancy has increased, many people with these disorders are opting to have children. Treatment strategies to minimize complications during pregnancy will be discussed.
Dr. Ian Greer will discuss the implications of venous thromboembolic disease for pregnancy as well as for the long-term health of the mother. The diagnosis of thrombosis and the safety and efficacy of available anticoagulants in the gravid patient will be reviewed.
Chair:
Terry B. Gernsheimer, MD
Puget Sound Blood Center, Seattle, WA, USA
Speakers:
Terry B. Gernsheimer, MD
Puget Sound Blood Center, Seattle, WA, USA
Thrombocytopenia in Pregnancy: Is This Immune Thrombocytopenia or...?
Sophie Lanzkron, MD, MHS
The Johns Hopkins School of Medicine, Baltimore, MD, USA
Baby on Board: What You Need to Know about Pregnancy in the Hemoglobinopathies
Ian A. Greer, MD
University of Liverpool, Liverpool, United Kingdom
Thrombosis in Pregnancy: Updates in Diagnosis and Management
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Hematopoietic Stem Cell Transplantation I: Exploiting Alternative Donors
| Session Offered Twice |
December 8, 2012, 7:30 AM - 9:00 AM
Georgia World Congress Center (A103, Building A, Level 1, Georgia World Congress Center) | December 8, 2012, 9:30 AM - 11:00 AM
Georgia World Congress Center (A103, Building A, Level 1, Georgia World Congress Center) |
This session will discuss the state of the art in allogeneic stem cell transplant medicine, demonstrating that with the exciting advances in the field, almost every patient who needs a transplant will now have access to a stem cell donor. The speakers will focus on donor selection, the optimal strategies for allograft preparation, and the clinical results for each of the major stem sources in use today.
Dr. Elizabeth Shpall will review the latest results in cord blood transplantation for high-risk hematologic cancers and genetic diseases. She will discuss the use of single versus double cord blood transplants, myeloablative versus nonmyeloablative preparative regimens, and the generation of cord blood immune cells to enhance immune recovery and anti-tumor responses.
Dr. Andrea Bacigalupo will review the clinical outcome of patients transplanted with matched siblings or unrelated donors; separate analyses will be presented for acute leukemias, myelodysplasic syndromes, myelofibrosis, aplastic anemia, and thalassemia. Comparisons will consider predictive factors such as donor age, stem cell source, transplant protocol, and graft-versus-host disease (GVHD) prophylaxis.
Dr. Ephraim Fuchs will review the use of human leukocyte antigen (HLA)-haploidentical stem cell transplantation in patients with high-risk hematologic malignancies. This session will discuss approaches to prophylaxis of GVHD, selection of HLA-haploidentical donors, immune reconstitution, and applications to treat nonmalignant diseases.
Chair:
Elizabeth J. Shpall, MD
The University of Texas MD Anderson Cancer Center, Houston, TX, USA
Speakers:
Elizabeth J. Shpall, MD
The University of Texas MD Anderson Cancer Center, Houston, TX, USA
Umbilical Cord Blood Transplantation: A Maturing Technology
Andrea Bacigalupo, MD
Ospedale San Martino, Genova, Italy
Matched and Mismatched Unrelated Donor Transplantation: Is the Outcome the Same as Matched-Sibling Donor?
Ephraim J. Fuchs, MD
The Johns Hopkins University School of Medicine, Baltimore, MD, USA
Haploidentical Transplantation for Hematologic Malignancies: Where Do We Stand?
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Hematopoietic Stem Cell Transplantation II: Towards Safer Allogeneic Transplantation
| Session Offered Twice |
December 9, 2012, 7:30 AM - 9:00 AM
Georgia World Congress Center (A101, Building A, Level 1, Georgia World Congress Center) | December 10, 2012, 10:30 AM - 12:00 PM
Georgia World Congress Center (A101, Building A, Level 1, Georgia World Congress Center) |
This session will focus on recent advances in allogeneic stem cell transplantation, providing an overview of conditioning regimens and graft-versus-host disease (GVHD) management, as well as challenging modifications to both that have been introduced in the last decade. Both aspects have conducted to better initial survival, probably due to longer exposure to immunosuppression, which leads to a higher exposure to late infections.
Dr. Didier Blaise will briefly review the concept of reduced-toxicity conditioning approaches. He will challenge their impact on transplant-related mortality with their ability to exert an adequate disease control as compared to usual standard myeloablative conditioning in different pathologies and populations. His presentation will seek to define the present challenges of the area for future development.
Dr. Steven Pavletic will review the achievements in acute and chronic GVHD management and how they have contributed to improved initial survival. He will also cover the long-term management of GVHD and its implication in disease control, and address the challenge represented by an older population in this context.
Dr. Kieren Marr will review the pattern and characteristics of delayed infections. She will question the reasons for their emergence and will address recent advances in diagnosis, prophylaxis, and treatment.
Chair:
Didier Blaise, MD
Institut Paoli-Calmettes, Marseille, France
Speakers:
Didier Blaise, MD
Institut Paoli-Calmettes, Marseille, France
Do Different Conditioning Regimens Really Make a Difference?
Steven Z. Pavletic, MD
National Cancer Institute, Bethesda, MD, USA
Are We Making Progress in Graft-Versus-Host-Disease Prophylaxis and Treatment?
Kieren A Marr, MD
The Johns Hopkins Hospital, Baltimore, MD, USA; Sidney Kimmel Cancer Center, Baltimore, MD, USA
Delayed Opportunistic Infections in Hematopoietic Stem Cell Transplant Patients: A Surmountable Challenge
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Hemoglobinopathies: New Frontiers and Insights
| Session Offered Twice |
December 8, 2012, 9:30 AM - 11:00 AM
Georgia World Congress Center (B405-B407, Building B, Level 4, Georgia World Congress Center) | December 10, 2012, 7:00 AM - 8:30 AM
Georgia World Congress Center (B405-B407, Building B, Level 4, Georgia World Congress Center) |
This session will focus on emerging therapies in the treatment of hemoglobinopathies and the potential of quality-of-life instruments to measure their efficacy.
Dr. Leboulch will discuss curative treatments focusing on gene therapy. The only available curative therapy for the hemoglobinopathies is allogeneic hematopoietic stem cell transplantation. However, a general lack of available donors, transplant rejection, and the development of graft-versus-host disease are significant problems. Gene therapy, designed to compensate for the defective ß–globin genes or inhibit HbS polymerization, has now reached phase I/II clinical trials. Five years after
lentiviral ß-globin gene therapy, a severe ß-E/ß-0 thalassemia patient has remained transfusion independent. Protocol details along with alternative approaches to increase safety and efficacy will be discussed.
Dr. Elliott Vichinsky will discuss novel therapies to address the pathophysiology of hemoglobinopathies. These strategies include improvement of nitric oxide availability (arginine), decreased inflammation and hypoxia reperfusion injury (5-lipoxygenase inhibitors and adenosine 2A agonists), decreased cellular adhesion and endothelial dysfunction (propanolol, pan-selectin inhibitors, statins), and the effects of antioxidant therapy (glutamine, alpha lipoic acid, acetyl-Lcarnitive, curcuminoids) on tissue injury. The use of hepcidin agonists in thalassemia will also be reviewed.
Dr. Julie Panepinto will review what is known regarding patient-reported outcomes (PROs) in hemoglobinopathies. She will discuss the role of PROs within clinical trials in hemoglobinopathies and their benefits in measuring the effects of treatment. Dr. Panepinto will also review U.S. Food and Drug Administration guidelines on PRO instruments to measure efficacy endpoints in a clinical trial. Lastly, she will highlight potential uses for PROs in a clinical setting.
Chair:
Elliott P. Vichinsky, MD
Children's Hospital and Research Center, Oakland, Oakland, CA, USA
Speakers:
Philippe Leboulch, MD
Brigham and Women's Hospital, Harvard Medical School, Boston, MA, USA
Advances In Gene Therapy and Stem Cell Transplantation In Hemoglobinopathies: Is the Future Here?
Elliott P. Vichinsky, MD
Children's Hospital and Research Center, Oakland, Oakland, CA, USA
Emerging "A" Therapies in Hemoglobinopathies: Agonists, Antagonists, Antioxidants, and Arginine
Julie A. Panepinto, MD
Medical College of Wisconsin, Children's Hospital of Wisconsin, Milwaukee, WI, USA
Health-Related Quality of Life in Patients with Hemoglobinopathies
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Immune Dysregulation
| Session Offered Twice |
December 8, 2012, 7:30 AM - 9:00 AM
Georgia World Congress Center (B405-B407, Building B, Level 4, Georgia World Congress Center) | December 8, 2012, 2:00 PM - 3:30 PM
Georgia World Congress Center (B405-B407, Building B, Level 4, Georgia World Congress Center) |
Immune dysregulation can lead to several hematologic disorders that include aplastic anemia, immune thrombocytopenia (ITP), and common variable immunodeficiency (CVID). An aberrant immune response can lead to auto-reactivity and cytopenia(s) in patients with aplastic anemia and ITP, or an inability to mount an effective immune response with susceptibility to infections in patients with CVID.
Dr. Phillip Scheinberg will briefly summarize recent insights into the pathogenesis of aplastic anemia. He will discuss the results of recent immunosuppression trials using alemtuzumab, horse and rabbit antithymocyte globulin (ATG), and how these studies are likely to affect clinical practice. He will then discuss transplantation outcomes in aplastic anemia in recent years and how the role of this therapeutic modality is evolving in aplastic anemia.
Dr. Charlotte Cunningham-Rundles will be discussing the presentations of CVID consisting of two phenotypes: one in which only infections are the characteristic; and another in which inflammatory and/or hematologic complications develop, including lymphadenopathy, splenomegaly, autoimmune cytopenias, enteropathy, and granulomatous disease. She will discuss how these phenotypes appear to be stable, how they are related to both immunologic and inflammatory markers, and how they are predictive of outcomes.
Dr. Jill Johnsen will present an overview of our current understanding of the pathogenesis of ITP. She will summarize the immune dysregulation observed in ITP and the associated phenotypes of accelerated platelet destruction and reduced platelet production. She will also discuss the implications of these evolving mechanistic models in the diagnosis and treatment of ITP.
Chair:
Phillip Scheinberg, MD
Hospital Sao Jose - Hospital Beneficencia Portuguesa de Sao Paulo, Sao Paulo, Brazil
Speakers:
Phillip Scheinberg, MD
Hospital Sao Jose - Hospital Beneficencia Portuguesa de Sao Paulo, Sao Paulo, Brazil
Aplastic Anemia: Therapeutic Updates in Immunosuppression and Transplantation
Charlotte Cunningham-Rundles, MD, PhD
Mount Sinai School of Medicine, New York, NY, USA
The Many Faces of Common Variable Immunodeficiency
Jill M. Johnsen, MD
University of Washington, Seattle, WA, USA; Puget Sound Blood Center, Seattle, WA, USA
Pathogenesis of Immune Thrombocytopenia: New Insights
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Insights into Biology and Refinement of Treatment Strategies in Hodgkin Lymphoma
| Session Offered Twice |
December 9, 2012, 7:30 AM - 9:00 AM
Georgia World Congress Center (A103, Building A, Level 1, Georgia World Congress Center) | December 10, 2012, 10:30 AM - 12:00 PM
Georgia World Congress Center (A103, Building A, Level 1, Georgia World Congress Center) |
This session will focus on recent advances in our understanding of the biology of Hodgkin lymphoma and important clinical issues related to the management of patients with the disease.
Dr. Ralf Kuppers will review current concepts related to the biology of Hodgkin lymphoma. He will discuss the histogenesis of Hodgkin/Reed-Sternberg cells and lymphocyte predominant cells, including their cellular origin and relationship to putative precursor cells; the genetic lesions that have been identified; and the deregulated transcription factor networks and signaling pathways that contribute to the survival and proliferation of these cells.
Dr. Martin Hutchings will review the multi-faceted roles of positron emission tomography (PET) imaging in Hodgkin lymphoma. He will discuss its applications in staging, early assessment of chemosensitivity, treatment response assessment, and post-treatment surveillance. He will also note important current clinical trials that incorporate PET imaging in defining patient management.
Drs. Ralph Meyer and Richard Hoppe will debate the advantages and disadvantages of incorporating radiation therapy into the management of patients with early-stage Hodgkin lymphoma. They will include a discussion of clinical trials that have tested the concept of Hodgkin lymphoma management with chemotherapy alone, as well as trials that have incorporated radiation therapy routinely. They will note the importance of different outcome measures for defining efficacy and identify opportunities for new clinical trials.
Chair:
Richard T. Hoppe, MD
Stanford University School of Medicine, Stanford, CA, USA
Speakers:
Ralf Küppers, PhD
University of Duisburg-Essen, Essen, Germany
New Insights in the Biology of Hodgkin Lymphoma
Martin Hutchings
Rigshospitalet - Copenhagen University Hospital, Copenhagen, Denmark
How Does PET/CT Help in Selecting Therapy for Patients with Hodgkin Lymphoma?
Richard T. Hoppe, MD
Stanford University School of Medicine, Stanford, CA, USA
Point-Counterpoint: Early Stage Hodgkin Lymphoma: The Role of Radiation Therapy
Ralph M. Meyer, MD
Queen's University, Kingston, ON, Canada
Point-Counterpoint: Early Stage Hodgkin Lymphoma: The Role of Radiation Therapy
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International Hematology: Limitations and Accomplishments in Sickle Cell Disease
| Session Offered Once |
December 10, 2012, 2:45 PM - 4:15 PM
Georgia World Congress Center (Georgia Ballroom 2, Building C, Level 3, Georgia World Congress Center) |
THIS SESSION IS OFFERED ONCE.
This session will highlight the global burden of sickle cell disease (SCD) and steps being taken by an international village to advance research and clinical care in countries with few health resources. Speakers will describe a network that aims to increase awareness of and treatment for the disease, the challenges of providing care, research projects and opportunities where disease prevalence is high, and efforts to establish a new clinical care center in one of the largest hospitals in Nigeria.
Dr. Isaac Odame will provide an overview of the global burden of SCD and discuss steps that need to be taken to begin to address the problem. He will discuss the formation and role of a network of SCD clinicians and scientists committed to fostering North-South and South-South collaborations aimed at furthering research and advancing clinical care for patients with SCD, particularly in low-income countries with the highest disease burden.
Dr. Julie Makani will provide an overview of the challenges in delivering comprehensive care for SCD in Africa within the context of limited resources and high disease burden. She will discuss opportunities presented by high patient numbers and the steps taken by African investigators and their collaborators in promoting and performing SCD research in sub-Saharan Africa through the establishment of active regional research networks.
Dr. Zakari Aliyu will discuss an effective model of partnerships involved in building sustainable capacity through health system strengthening to advance clinical care and promote research in low-income settings. He will describe his collaborative efforts to establish a new blood, sickle cell, cancer, and palliative care institute within one of the largest hospitals in Nigeria.
Chair:
Griffin P. Rodgers, MD
National Institute of Diabetes, Digestive and Kidney Diseases, National Institutes of Health, Bethesda, MD, USA
Speakers:
Isaac Odame, MD
The Hospital for Sick Children, Toronto, ON, Canada
Global Perspectives on Sickle Cell Disease
Julie Makani, MD, PhD
Muhimbili University of Health and Allied Sciences, Dar es Salaam, Tanzania
Sickle Cell Disease in Sub-Saharan Africa: Challenges, Progress and Next Steps
Zakari Aliyu, MD
Taraba State Specialist Hospital, Taraba, Nigeria
Private-Public Partnerships: Model for Finding Solutions to Sickle Cell Disease in Low-Income Countries
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Junior Faculty Development Education Program: How to be Successful in Your First “Real” Job
| Session Offered Once |
December 8, 2012, 4:00 PM - 5:30 PM
Georgia World Congress Center (B206, Building B, Level 2, Georgia World Congress Center) |
THIS SESSION IS OFFERED ONCE.
This session will focus on issues critical to career development of junior faculty members.
Dr. Donald Small will review how to define realistic research goals based on a research effort assignment, how to establish effective research collaborations, and how to obtain critical early career extramural funding.
Dr. Mignon Loh will discuss why it is important to identify a niche, how to decide on an appropriate niche and become the local expert in this area, and how to move from being a local expert to a national/international expert.
Dr. Barbara Konkle will illustrate the advantages and disadvantages of transitioning from trainee to junior faculty member at the same institution, how to identify potential issues, and how to remedy problem situations in constructive ways. She will also discuss appropriate considerations when things are going well and you are approached with other career opportunities.
Chair:
Stephen P. Hunger, MD
University of Colorado School of Medicine, Aurora, CO, USA
Speakers:
Donald Small, MD, PhD
The Johns Hopkins University School of Medicine, Baltimore, MD, USA
Developing a Productive Research Program
Mignon Loh, MD
University of California - San Francisco, San Francisco, CA, USA
Developing a Focused Area of Clinical Expertise
Barbara A. Konkle, MD
Puget Sound Blood Center, Seattle, WA, USA; University of Washington, Seattle, WA, USA
Considering a New Job: Should I Stay or Should I Go?
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Keeping Pace with Advances in Myeloma
| Session Offered Twice |
December 8, 2012, 7:30 AM - 9:00 AM
Georgia World Congress Center (Hall B5, Building B, Level 1, Georgia World Congress Center) | December 8, 2012, 4:00 PM - 5:30 PM
Georgia World Congress Center (Hall B5, Building B, Level 1, Georgia World Congress Center) |
This session will focus on recent advances in the understanding of multiple myeloma biology and the changes in the treatment of this disease after the introduction of the immunomodulatory drugs thalidomide and lenalidomide and the proteasome inhibitor bortezomib. The role of transplantation in the era of novel agents will be addressed, and future therapeutic directions will be discussed. This program will report on the most effective treatment strategies available today and outline how to properly integrate them in the management of multiple myeloma.
Dr. Gareth Morgan will review the biological features of multiple myeloma. He will describe the physiological immune system development, explain the mechanisms determining molecular alterations in myeloma, and correlate these abnormalities with the evolution of the disease. He will also comment on the clinical implications of these alterations in myeloma therapy.
Dr. S. Vincent Rajkumar will review the different doublet, triplet, or quadruplet drug combinations used for the treatment of newly diagnosed myeloma. He will provide efficacy and safety results of various studies and compare these approaches. He will also present preliminary results from recent studies introducing the concept of second-generation proteasome inhibitors.
Dr. Antonio Palumbo will review the role of stem cell transplantation in myeloma and the clinical benefit achieved after the introduction of novel induction, consolidation, and maintenance approaches. He will discuss whether drug combinations have replaced stem cell transplantation, the risk/benefit ratio of these approaches, and the role of maintenance treatment.
Chair:
Antonio Palumbo, MD
University of Torino, Torino, Italy
Speakers:
Gareth J. Morgan, MD PhD
Royal Marsden Hospital, Sutton, United Kingdom
How to Use New Biology to Guide Therapy in Myeloma
S. Vincent Rajkumar
Mayo Clinic, Rochester, MN, USA
Doublet, Triplets, or Quadruplets of Novel Agents in Newly Diagnosed Myeloma?
Antonio Palumbo, MD
University of Torino, Torino, Italy
Have Drug Combinations Supplanted Stem Cell Transplantation in Myeloma?
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Landscape Changes and Challenges In Hemophilia
| Session Offered Twice |
December 9, 2012, 9:30 AM - 11:00 AM
Georgia World Congress Center (A411-A412, Building A, Level 4, Georgia World Congress Center) | December 10, 2012, 7:00 AM - 8:30 AM
Georgia World Congress Center (B207-B208, Building B, Level 2, Georgia World Congress Center) |
The hemophilia landscape is changing and will continue to undergo dramatic changes in the next several years. After a relatively quiet period of novel treatment development and the initial setbacks with gene therapy, it is clear that innovation in hemophilia care is now moving more rapidly. There are a host of new treatments for factor VIII and IX deficiency as well as for inhibitor patients that are in late stages of clinical trials. Additional research regarding the management of acquired hemophilia has also provided new evidence to assist in the management of this rare disorder. Finally, the prospects for gene therapy appear to be much brighter given the recent breakthrough in the clinical trial of factor IX-deficient patients.
Dr. Guy Young will review the current challenges in the management of hemophilia and will discuss long-term outcomes and complications, including the management of inhibitor patients. He will also discuss the changing landscape of hemophilia given the new agents that are on the horizon.
Dr. Peter Collins will review the current status of the management of acquired hemophilia and discuss the results of recent studies and their impact on the care of these patients.
Dr. Katherine High will review the most recent results of gene therapy trials in humans as well as explore the different techniques that are being studied for the treatment of both factor VIII and factor IX deficiencies.
Chair:
Guy Young, MD
Children's Hospital Los Angeles, Los Angeles, CA, USA
Speakers:
Guy Young, MD
Children's Hospital Los Angeles, Los Angeles, CA, USA
New Challenges In Hemophilia: Long-Term Outcomes And Complications
Peter W Collins, MD
Cardiff University School of Medicine, Cardiff, United Kingdom
Therapeutic Challenges In Acquired Factor VIII Deficiency
Katherine A. High, MD
Children's Hospital of Philadelphia, Philadelphia, PA, USA
The Gene Therapy Journey For Hemophilia: Are We There Yet?
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New Insights into the Genetic Pathogenesis of Acute Lymphocytic Leukemia and New Treatment Strategies
| Session Offered Twice |
December 8, 2012, 9:30 AM - 11:00 AM
Georgia World Congress Center (Thomas Murphy Ballroom 1-2, Building B, Level 5, Georgia World Congress Center) | December 9, 2012, 7:30 AM - 9:00 AM
Georgia World Congress Center (Thomas Murphy Ballroom 1-2, Building B, Level 5, Georgia World Congress Center) |
The session will focus on recent advances in the basic biology and clinical features of acute lymphocytic leukemia (ALL) with an emphasis on the molecular genetics of this malignancy and treatment approaches to both Philadelphia chromosome-positive (Ph+) and negative (Ph-) disease. Dr. Charles Mullighan will review the genomic analyses that have provided important insights into the biologic basis of ALL. He will also discuss the approaches used to profile ALL genomes, including microarrays and next generation sequencing, and review findings of recent studies in childhood B- and T-lineage ALL that have identified new ALL subtypes, characterized alterations in multiple key pathways that contribute to leukemogenesis, and identified previously unsuspected targets of mutation. Notably, these studies contribute to development of new tests for diagnosis and risk stratification and have identified new therapeutic targets in high-risk disease.
Dr. Deborah Thomas will discuss the changing paradigms for treatment of the genetically distinct subtype of Ph+ or BCR-ABL-positive B-lymphocytic leukemia in the era of second- and later-generation tyrosine kinase inhibitors (TKIs), inclusive of emerging data regarding TKI therapy after allogeneic HCT. She will then review the prevalence and dynamics of tyrosine kinase domain mutations in the context of novel agents and potential therapeutic strategies designed to circumvent these and other resistance mechanisms.
Dr. Hillard Lazarus will review indications for when to consider proceeding with hematopoietic cell transplantation (HCT) in Ph-ALL in first complete remission compared to conventional approaches. Next, he will review patient outcomes obtained with myeloablative versus reduced-intensity conditioning regimens. Finally, he will review HCT results using different donor sources, including matched-related versus alternative donor sources.
Chair:
Hillard M. Lazarus, MD
University Hospitals Case Medical Center, Cleveland, OH, USA
Speakers:
Charles G. Mullighan, MBBS MSc MD
St. Jude Children's Research Hospital, Memphis, TN, USA
The Molecular Genetic Makeup of Acute Lymphocytic Leukemia
Deborah A. Thomas, MD
The University of Texas, M. D. Anderson Cancer Center, Houston, TX, USA
Philadelphia-Chromosome-Positive Acute Lymphocytic Leukemia: It Was the Worst of Times, It Is the Best of Times
Hillard M. Lazarus, MD
University Hospitals Case Medical Center, Cleveland, OH, USA
When, How, and What Cell Source For Hematopoietic Cell Transplantation In First Complete Remission Adult Acute Lymphoblastic Leukemia
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Non-Hodgkin Lymphoma I: Changing Therapeutic Strategies in Aggressive Lymphomas
| Session Offered Twice |
December 8, 2012, 7:30 AM - 9:00 AM
Georgia World Congress Center (Thomas Murphy Ballroom 3-4, Building B, Level 5, Georgia World Congress Center) | December 8, 2012, 4:00 PM - 5:30 PM
Georgia World Congress Center (Thomas Murphy Ballroom 3-4, Building B, Level 5, Georgia World Congress Center) |
This session will focus on three of the most common consults an expert lymphoma physician sees during the course of current management of patients with diffuse large B-cell lymphoma (DLBCL).
Dr. Laurie Sehn will review the important pre-treatment, clinical, immunohistochemical, and molecular prognostic factors that may help guide primary therapy in the rituximab era. Importantly, she will help determine if any of these factors are potential targets for novel therapy and if they are additive to the international prognostic index.
Dr. Craig Moskowitz will review the use and abuse of fluorodeoxyglucos positron emission tomography (FDG-PET) imaging in the management of DLBCL, focusing on its utility as a prognostic tool for risk-adapted therapy in the primary as well as the relapsed setting.
Dr. Christian Gisselbrecht will describe why we may be fighting a losing battle once a patient relapses after primary therapy. He will use the results of the Collaborative Trial in Relapsed Aggressive Lymphoma (CORAL) study as a platform for new treatment strategies in patients with primary refractory and relapsed DLBCL in the rituximab era.
Chair:
Craig H. Moskowitz, MD
Memorial Sloan-Kettering Cancer Center, New York, NY, USA
Speakers:
Laurie H. Sehn, MD
British Columbia Cancer Agency, Vancouver, BC, Canada
Paramount Prognostic Factors Which Guide Therapeutic Strategies in Diffuse Large B-Cell Lymphoma
Craig H. Moskowitz, MD
Memorial Sloan-Kettering Cancer Center, New York, NY, USA
Interim PET-CT (I-PET) in the management of Diffuse Large B-cell Lymphoma
Christian Gisselbrecht, MD
Hopital Saint-Louis, Paris, France
Is There Any Role for Transplantation in the Rituximab Era for Diffuse Large B-Cell Lymphoma?
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Non-Hodgkin Lymphoma II: Understanding the Indolent Lymphomas
| Session Offered Twice |
December 8, 2012, 2:00 PM - 3:30 PM
Georgia World Congress Center (Thomas Murphy Ballroom 3-4, Building B, Level 5, Georgia World Congress Center) | December 10, 2012, 7:00 AM - 8:30 AM
Georgia World Congress Center (Georgia Ballroom 2, Building C, Level 3, Georgia World Congress Center) |
The indolent lymphomas comprise several subtypes, including the marginal zone lymphomas and follicular lymphoma. Indolent lymphomas possess variable clinical characteristics and pathologic features, and a consensus has not yet been reached regarding their optimal treatment, which includes observation, radiation therapy, chemoimmunotherapy, and hematopoietic stem cell transplantation (HSCT).
Dr. Pier Zinzani will outline the three distinct entities of marginal zone lymphomas (MZLs): extranodal MZL of mucosa-associated lymphoid tissue (MALT) type, splenic MZL, and nodal MZL, all of which originate from post-germinal center marginal zone B cells. Pathogenic mechanisms and therapeutic advances will also be presented.
Dr. Brad Kahl will discuss the role of observation versus rituximab in indolent lymphoma. The “watch and wait” approach remains an acceptable treatment option for asymptomatic indolent non-Hodgkin lymphoma. However, this paradigm has been challenged with the advent of rituximab due to its low toxicity profile and its efficacy in improving progression-free survival.
Dr. Ginna Laport will present data regarding the role of HSCT in advanced follicular lymphoma. Both autologous and allogeneic HSCT can prolong progression-free survival. However, allogeneic HSCT remains the only known cure, and reduced-intensity conditioning regimens have broadened eligibility to older patients and can confer long-term remissions.
Chair:
Ginna G. Laport, MD
Stanford University, Stanford, CA, USA
Speakers:
Pier Luigi Zinzani, MD
Institute of Hematology, Bologna, Italy
The Many Faces of Marginal Zone Lymphoma
Brad Kahl, MD
University of Wisconsin, Madison, WI, USA
Is there a Role for "Watch and Wait" in Follicular Lymphoma in the Rituximab Era?
Ginna G. Laport, MD
Stanford University, Stanford, CA, USA
Changing Role of Stem Cell Transplantation in Follicular Lymphoma
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Not So “Benign” Hematologic Issues in Children
| Session Offered Twice |
December 9, 2012, 7:30 AM - 9:00 AM
Georgia World Congress Center (Sidney Marcus Auditorium, Building A, Level 4, Georgia World Congress Center) | December 10, 2012, 2:45 PM - 4:15 PM
Georgia World Congress Center (Thomas Murphy Ballroom 2-3, Building B, Level 5, Georgia World Congress Center) |
This session will focus on three different pediatric hematologic issues that, although generally appearing to be somewhat benign, indeed cause significant morbidity and mortality. These topics will be explored through review of the current findings and projection of potential directions in care.
Dr. Janna Journeycake will review current evidence in the use of rituximab, thrombopoeitin-receptor agonists, and other new therapeutics in the management of childhood chronic immune thrombocytopenia.
Dr. Anthony Chan will review the progress that has been made in the area of venous thromboembolism in children over the last 20 years.
Dr. Shoshana Revel-Vilk will discuss the developmental hemostatic system that is seemingly abnormal compared to adults and whether neonates are more prone to bleeding or to thrombotic problems. She will also describe approaches to diagnose coagulopathy in neonates.
Chair:
Anthony K.C. Chan, MBBS, FRCPC, FRCPath
McMaster University, Hamilton, ON, Canada
Speakers:
Janna M. Journeycake, MD
University of Texas Southwestern Medical Center at Dallas, Dallas, TX, USA
Childhood Immune Thrombocytopenia: Role of Rituximab, Recombinant Thrombopoietin, and Other New Therapeutics
Anthony K.C. Chan, MBBS, FRCPC, FRCPath
McMaster University, Hamilton, ON, Canada
Updates in Thrombosis in Pediatrics: Where Are We After 20 Years?
Shoshana Revel-Vilk, MD
Hadassah Hebrew-University Medical Center, Jerusalem, Israel
The Conundrum of Neonatal Coagulopathy
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Pearls and Pitfalls in the Hematology Lab: Clotting and Bleeding
| Session Offered Twice |
December 8, 2012, 4:00 PM - 5:30 PM
Georgia World Congress Center (Sidney Marcus Auditorium, Building A, Level 4, Georgia World Congress Center) | December 10, 2012, 10:30 AM - 12:00 PM
Georgia World Congress Center (Sidney Marcus Auditorium, Building A, Level 4, Georgia World Congress Center) |
Hematologist-oncologists are often asked to see patients for a variety of blood coagulation issues. These consultations usually require the consultant to make decisions about ordering and interpreting coagulation assays – an area in which practicing physicians have had progressively less direct contact. This session is designed to provide clinicians with current information on the latest guidelines and also with handy tips and practical advice on how to best utilize these assays.
Dr. Dorothy Adcock will discuss the current approaches to deciding when and how to monitor anticoagulant therapies. The discussion will include recommendations on the latest group of anticoagulants: the oral direct inhibitors of thrombin and factor Xa.
Dr. Jacob Rand will review the current diagnostic criteria for antiphospholipid syndrome, describe the clinical indications for testing, and offer recommendations on how the tests should be interpreted.
Dr. Teresa Quiroga will provide insight on the current approaches to identifying and evaluating patients who are suspected to have bleeding disorders.
Chair:
Jacob H. Rand, MD
Montefiore Medical Center, Albert Einstein College of Medicine, Bronx, NY, USA
Speakers:
Dorothy Marie Adcock, MD
Esoterix, Inc., Englewood, CO, USA
Coagulation Assays and Anticoagulant Monitoring
Jacob H. Rand, MD
Montefiore Medical Center, Albert Einstein College of Medicine, Bronx, NY, USA
Dos and Don'ts in Diagnosing Antiphospholipid Syndrome
Teresa Quiroga, MD
Pontifical Catholic University of Chile School of Medicine, Santiago, Chile
Is My Patient a Bleeder? A Diagnostic Framework for Mild Bleeding Disorders
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Pearls and Pitfalls in the Hematology Lab: Updates on Cellular Diagnostics
| Session Offered Twice |
December 9, 2012, 9:30 AM - 11:00 AM
Georgia World Congress Center (A101, Building A, Level 1, Georgia World Congress Center) | December 10, 2012, 2:45 PM - 4:15 PM
Georgia World Congress Center (A101, Building A, Level 1, Georgia World Congress Center) |
This session will focus on laboratory testing in hematology including new diagnostic assays, the interpretation of diagnostic laboratory tests, and problems associated with such testing.
Dr. Peter Valent will discuss the diagnostic interface in mild cytopenias, ranging from low-risk myelodysplastic syndromes to immune-mediated cytopenias. He will review available clinical and laboratory markers and assays useful to delineate the mechanism(s) of cytopenia, with special reference to bone marrow function-related parameters. Dr. Valent will also discuss available criteria and diagnostic algorithms through which a provisional or a final diagnosis can be established in these patients.
Dr. Tracy George will focus on the evaluation of leukocytosis, describing useful laboratory assays that separate malignant from benign proliferations. She will discuss practical aspects of cellular diagnostics, including examination of the peripheral blood smear, when flow cytometry immunophenotyping is appropriate and when it can be misleading, and when molecular genetic testing is helpful.
Dr. Ralph Green will present a practical approach to the diagnosis of anemia. He will discuss general principles including the use and limitations of red cell indices, testing algorithms, and discriminant functions. With the backdrop of the changing spectrum of the more and less commonly encountered anemias and through illustrative case examples from the obvious to the obscure, Dr. Green will focus on when to order what tests, how to interpret them, and what’s new and interesting beyond the commonplace.
Chair:
Tracy I. George, MD
Stanford University School of Medicine, Stanford, CA, USA
Speakers:
Peter Valent, MD
Medical University of Vienna, Vienna, Austria
Low Blood Counts: Immune, Idiopathic or Myelodysplasia
Tracy I. George, MD
Stanford University School of Medicine, Stanford, CA, USA
Malignant or Benign Leukocytosis
Ralph Green, MD, PhD
UC Davis Medical Center, Sacramento, CA, USA
Anemias Beyond B12 and Iron Deficiency: The Buzz about Other B's, "Elementary" and "Non-Elementary" Problems
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Pediatric Issues in Platelet Transfusions
| Session Offered Twice |
December 9, 2012, 9:30 AM - 11:00 AM
Georgia World Congress Center (Sidney Marcus Auditorium, Building A, Level 4, Georgia World Congress Center) | December 10, 2012, 10:30 AM - 12:00 PM
Georgia World Congress Center (A411-A412, Building A, Level 4, Georgia World Congress Center) |
THIS SESSION IS JOINTLY SPONSORED WITH AABB.
This session will be devoted to recent advances in the basic science of developmental platelet production and function and implications for therapy. It will also discuss thrombocytopenia, transfusion thresholds, and bleeding in sick neonates and pediatric oncology/hematopoietic stem cell transplant patients.
Dr. Sola-Visner will review the main developmental differences in platelet production, survival, and function between neonates and adults, and describe some of the key molecular mechanisms underlying these differences. She will then discuss the unique characteristics of the neonatal hemostatic system and the potential impact of transfusing “developmentally mismatched” adult platelets on neonatal physiology.
Dr. Simon Stanworth will briefly review severity and clinical characteristics of neonates that develop thrombocytopenia. He will then discuss prevalence and patterns of bleeding in neonates with severe thrombocytopenia. Finally, Dr. Stanworth will review surveys and clinical studies that focus on the effectiveness of prophylactic platelet transfusions and platelet transfusion thresholds in this vulnerable population.
Dr. Cassandra Josephson will review the historic data and studies that focus on platelet transfusion thresholds, dosing of platelets, and bleeding incidence in pediatric oncology and hematopoietic stem cell transplant patients. She will then explore the lack of standardization of bleeding assessment tools for reporting bleeding outcomes and examine the clinical relevance of bleeding in this population. Finally, Dr. Josephson will discuss the pediatric sub-analysis of the National Heart, Lung, and Blood Institute and Transfusion Medicine/Hemostasis Clinical Trial Network-sponsored trial on platelet dosing and bleeding outcomes in patients with hypoproliferative thrombocytopenia, the Prophylactic PLAtelet DOse (PLADO) trial.
Chair:
Cassandra Josephson, MD
Emory University, Atlanta, GA, USA
Speakers:
Martha Sola-Visner, MD
Boston Children's Hospital, Harvard Medical School, Boston, MA, USA
Platelets in the Neonatal Period: Developmental Differences in Platelet Production, Function, and Hemostasis, and the Potential Impact of Therapies
Simon J. Stanworth, MRCP, FRCPath, DPhil
NHS Blood and Transplant, Oxford University Hospital NHS Trust, Oxford, United Kingdom
Thrombocytopenia, Bleeding and Platelet Transfusions in Sick Neonates
Cassandra Josephson, MD
Emory University, Atlanta, GA, USA
Thrombocytopenia and Bleeding in Pediatric Oncology Patients
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Perioperative Hematology: To Bleed or Not To Bleed
| Session Offered Twice |
December 8, 2012, 9:30 AM - 11:00 AM
Georgia World Congress Center (Sidney Marcus Auditorium, Building A, Level 4, Georgia World Congress Center) | December 9, 2012, 7:30 AM - 9:00 AM
Georgia World Congress Center (A411-A412, Building A, Level 4, Georgia World Congress Center) |
This session will provide a review and update on the management of patients in association with surgery, focusing on blood coagulation.
Successful surgery often requires an appropriate balance between avoidance of thrombosis and of excessive bleeding. Although preventive planning is the foundation for safe invasive procedures, the hematologist should also give advice when bleeding becomes uncontrollable.
Dr. Ortel will address the management of patients who are receiving chronic anticoagulant therapy and require an elective surgery or procedure. Consideration must be given to the indication for anticoagulant therapy and associated thrombotic risk, as well as to the potential hemorrhagic risks in the post-operative setting. Dr. Ortel will discuss options for various clinical scenarios and will also address the role of “bridging” therapy in high-risk situations.
Dr. Jeannie Callum will discuss the available laboratory tests for the massively bleeding patient, the transfusion strategies available, and the alternatives to transfusion. The discussion will include the evidence for near-patient testing, 1:1:1 formula resuscitation, antifibrinolytics, recombinant factor VIIa, and coagulation concentrates.
Dr. Sam Schulman will highlight the transfusion-saving role of antifibrinolytic therapy, particularly with tranexamic acid. He will also review the possibility, in the era of several new antithrombotic agents without available antidotes, of assessing the risk of bleeding or the contribution of such an agent to actual bleeding, as well as methods to reverse or mitigate the anti-hemostatic effect.
Chair:
Sam Schulman, MD, PhD
McMaster University, Hamilton, ON, Canada
Speakers:
Thomas L. Ortel, MD, PhD
Duke University Medical Center, Durham, NC, USA
Perioperative Management of Patients on Chronic Antithrombotic Therapy
Jeannie Callum, MD
Sunnybrook Health Sciences Centre, Toronto, ON, Canada
Assessing Perioperative Bleeding
Sam Schulman, MD, PhD
McMaster University, Hamilton, ON, Canada
Pharmacological Tools to Reduce Bleeding in Surgery
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The New Era in Antithrombotic Therapy
| Session Offered Twice |
December 10, 2012, 7:00 AM - 8:30 AM
Georgia World Congress Center (Sidney Marcus Auditorium, Building A, Level 4, Georgia World Congress Center) | December 10, 2012, 2:45 PM - 4:15 PM
Georgia World Congress Center (Sidney Marcus Auditorium, Building A, Level 4, Georgia World Congress Center) |
For more than 60 years, the only orally available anticoagulants were the vitamin K antagonists, such as warfarin. The recent introduction of new oral anticoagulants that target thrombin or factor Xa have changed the current landscape. For example, dabigatran etexilate, an oral thrombin inhibitor, and rivaroxaban, an oral factor Xa inhibitor, are licensed as alternatives to warfarin for stroke prevention in patients with atrial fibrillation. Apixaban, another oral factor Xa inhibitor, is likely to follow soon. These agents also show promise for treatment of venous thromboembolism, and, when given in conjunction with antiplatelet therapy in stabilized acute coronary syndrome patients, rivaroxaban reduces cardiovascular death, stroke, and recurrent infarction compared with placebo. The new oral anticoagulants are more convenient than warfarin because they can be given in fixed doses without routine coagulation monitoring and there are few drug interactions. In addition, the new agents are associated with less intracranial bleeding than warfarin. With these advantages, the new oral anticoagulants are poised to replace warfarin for many indications.
Dr. Jeffrey Weitz will compare the pharmacological properties of the new oral anticoagulants and discuss the pros and cons of each drug for the various indications.
Dr. Alan Jacobson will highlight the opportunities and challenges of the new oral anticoagulants as replacements for warfarin.
Dr. Jessica Mega will outline the role of rivaroxaban as an adjunct to dual antiplatelet therapy to reduce the risk of recurrent ischemia in patients with acute coronary syndromes.
Chair:
Jeffrey I. Weitz, MD
Thrombosis & Atherosclerosis Research Institute, McMaster University, Hamilton, ON, Canada
Speakers:
Jeffrey I. Weitz, MD
Thrombosis & Atherosclerosis Research Institute, McMaster University, Hamilton, ON, Canada
Novel Anticoagulants: Which One Should My Patient Use?
Alan K. Jacobson, MD
VA Loma Linda Healthcare System, Loma Linda, CA, USA
Is There A Role For Warfarin Anymore?
Jessica L. Mega, MD
Brigham and Women's Hospital, Harvard Medical School, Boston, MA, USA
Triple Therapy Or Triple Threat?
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The Spectrum of JAK2-Positive Myeloproliferative Neoplasms: Complications and Therapeutic Advances
| Session Offered Twice |
December 8, 2012, 4:00 PM - 5:30 PM
Georgia World Congress Center (Thomas Murphy Ballroom 1-2, Building B, Level 5, Georgia World Congress Center) | December 9, 2012, 9:30 AM - 11:00 AM
Georgia World Congress Center (Thomas Murphy Ballroom 1-2, Building B, Level 5, Georgia World Congress Center) |
The purpose of the session is to present the spectrum of JAK2 V617F-positive myeloproliferative neoplasms (MPNs), to review thrombotic complications, and to then identify targets for inhibition. It is expected that the signaling by JAK2 mutant will be presented in the context of the array of MPNs, how this signaling contributes to thrombotic complications, and which signaling proteins and pathways should be targeted by screens. Inhibition of JAK2 kinase activity is just one avenue to take; other pathways contribute to phenotype, complications, clonal dominance, and evolution to leukemia.
Chair:
Stefan N. Constantinescu, MD, PhD
Ludwig Institute for Cancer Research, Brussels, Belgium
Speakers:
Jean-Jacques Kiladjian, MD, PhD
Hôpital Saint-Louis et Université Paris Diderot, Paris, France; French Intergroup of Myeloproliferative neoplasms (FIM), Paris, France
The Spectrum of JAK2-Positive Myeloproliferative Neoplasms
Anna Falanga, MD
Ospedali Riuniti di Bergamo, Bergamo, Italy
Thrombotic Disease in the Myeloproliferative Neoplasms
Stefan N. Constantinescu, MD, PhD
Ludwig Institute for Cancer Research, Brussels, Belgium
Small-Molecule Inhibitors in Myeloproliferative Neoplasms: Are We Aiming for the Right Targets?
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The Spectrum of Plasma Cell Dyscrasias
| Session Offered Twice |
December 8, 2012, 9:30 AM - 11:00 AM
Georgia World Congress Center (A101, Building A, Level 1, Georgia World Congress Center) | December 10, 2012, 7:00 AM - 8:30 AM
Georgia World Congress Center (A101, Building A, Level 1, Georgia World Congress Center) |
This session will focus on the complex diagnostic challenges that are associated with plasma cell dyscrasias. The focus will be on those disorders that can cause diagnostic confusion and can potentially result in the incorrect treatment selection for patients. The session is aimed at busy practicing clinicians and is focused on making the correct diagnosis, determining the appropriate time to treat, and determining when to observe.
Dr. Irene Ghobrial will review the diagnostic criteria for Waldenström macroglobulinemia. While a number of disorders are associated with the monoclonal IgM protein, they are not Waldenström macröglobulinemia, underscoring the importance of taking specific, required considerations. The management of IgM and neuropathy will be discussed.
Dr. Giampaolo Merlini will review the differential diagnosis of monoclonal gammopathy of undetermined significance (MGUS). Many patients with small monoclonal gammopathies are often assumed to have MGUS when in fact they fulfill the criteria of a dangerous small B-cell clone. New classification schemes for MGUS and smoldering multiple myeloma focusing on criteria for starting treatment will be reviewed.
Dr. Morie Gertz will review the records of seven patients that were initially incorrectly diagnosed. He will present a detailed schema to recognize how to not overlook these entities.
Chair:
Morie Abraham Gertz, MD
Mayo Clinic, Rochester, MN, USA
Speakers:
Irene Ghobrial, MD
Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA, USA
Are You Sure This is Waldenström Macroglobulinemia?
Giampaolo Merlini, MD
Fondazione IRCCS Policlinico San Matteo, Pavia, Italy
Differential Diagnosis of Monoclonal Gammopathy of Undetermined Significance
Morie Abraham Gertz, MD
Mayo Clinic, Rochester, MN, USA
Case Vignettes and Other Brain Teasers of Monoclonal Gammopathies
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The Thrombotic Thrombocytopenic Purpura and Hemolytic Uremic Syndromes: New Insights and New Treatments
| Session Offered Twice |
December 8, 2012, 4:00 PM - 5:30 PM
Georgia World Congress Center (B405-B407, Building B, Level 4, Georgia World Congress Center) | December 10, 2012, 2:45 PM - 4:15 PM
Georgia World Congress Center (B405-B407, Building B, Level 4, Georgia World Congress Center) |
This session will focus on understanding the evaluation and management of syndromes diagnosed as thrombotic thrombocytopenic purpura (TTP) or hemolytic uremic syndromes (HUS) in adults and children. New diagnostic studies, such as measurement of ADAMTS13 activity or assessment of complement system abnormalities, may be important for establishing etiologies that may require specific treatments. For patients with TTP associated with severe ADAMTS13 deficiency, rituximab may be an important adjunctive treatment for selected patients. For patients described as atypical HUS (aHUS) resulting from an abnormality of complement regulation, treatment with eculizumab may be important.
Dr. James George will describe the diversity of the TTP and HUS syndromes that are encountered in clinical practice, reflecting several different etiologies and distinct long-term outcomes following recovery.
Dr. Johanna Kremer Hovinga will describe the role of ADAMTS13 deficiency in the pathogenesis of TTP and the importance and limitations of ADAMTS13 measurements for evaluation of patients with suspected TTP. She will also address problems specific to the rare families with hereditary ADAMTS13 deficiency (Upshaw-Schulman syndrome).
Dr. Carla Nester will describe the clinical features of patients with abnormalities of complement regulation, resulting in excessive complement activation and the syndrome described as aHUS. She will describe the clinical features that may allow recognition of aHUS and consideration of intervention with the recently approved treatment, eculizumab.
Chair:
James N. George, MD
University of Oklahoma Health Sciences Center, Oklahoma City, OK, USA
Speakers:
James N. George, MD
University of Oklahoma Health Sciences Center, Oklahoma City, OK, USA
Diagnostic and Therapeutic Challenges in the Thrombotic Thrombocytopenic Purpura and Hemolytic Uremic Syndromes
Johanna A. Kremer Hovinga, MD
Bern University Hospital and University of Bern, Bern, Switzerland; Inselspital, Bern University Hosptial and University Bern, Bern, Switzerland
ADAMTS13: Its Roles in Pathogenesis, Diagnosis and Treatment in Thrombotic Thrombocytopenic Purpura
Carla M. Nester, MD
University of Iowa, Iowa City, IA, USA
Atypical Hemolytic Uremic Syndrome: What Is It, How Is It Diagnosed, and How Is It Treated?
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The Trade Secrets of A Successful Academic
| Session Offered Once |
December 8, 2012, 9:30 AM - 11:00 AM
Georgia World Congress Center (B206, Building B, Level 2, Georgia World Congress Center) |
THIS SESSION IS OFFERED ONCE.
This session will explore the secrets behind three critical factors in attaining academic success: achieving leadership through mentoring and being mentored; conducting research studies of value supported by appropriate knowledge of statistics, as well as engaging statisticians as team members; and creating and delivering presentations that captivate your audiences.
Dr. Barbara Alving will provide case studies of highly successful mentors and then examine questions that arise in considering mentorship. What defines a successful mentor? Is mentoring an altruistic activity? What is the difference between mentoring and coaching? When does a professional outgrow the need for mentoring, and are mentors needed in this highly connected, mobile world?
Dr. Sara Vesely will discuss how to interact with a biostatistician to ensure an appropriate study design and utilization of resources and clinical research participants. She will explain how the power of statistics is available to every investigator, regardless of career level.
Dr. Douglas Rizzo will discuss preparing dynamic, innovative presentations based on an ability to understand a variety of potential audiences, including community members, clinical trial participants, potential donors, management, academic colleagues, or international groups with language challenges. He will also describe effective use of presentation formats, such as PowerPoint, and delivery techniques to engage the audience and overcome anxiety.
Chair:
Barbara M. Alving, MD
Former Director of National Center for Research Resources, National Institutes of Health, Uniformed Services University of the Health Sciences, Bethesda, MD, USA
Speakers:
Barbara M. Alving, MD
Former Director of National Center for Research Resources, National Institutes of Health, Uniformed Services University of the Health Sciences, Bethesda, MD, USA
Secrets of Successful Mentors
Sara K. Vesely, PhD
University of Oklahoma Health Sciences Center, Oklahoma City, OK, USA
Secrets of Statistics: What You Need to Know but Were Afraid To Ask
J. Douglas Rizzo, MD
Medical College of Wisconsin, Milwaukee, WI, USA
Secrets of a Great Talk: How to Create and Deliver Effective Presentations
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Thrombosis in Challenging Populations
| Session Offered Twice |
December 8, 2012, 9:30 AM - 11:00 AM
Georgia World Congress Center (A411-A412, Building A, Level 4, Georgia World Congress Center) | December 8, 2012, 4:00 PM - 5:30 PM
Georgia World Congress Center (A411-A412, Building A, Level 4, Georgia World Congress Center) |
Thrombosis remains an important cause of morbidity and mortality among patients with medical illnesses in a variety of settings. Conflicting results from recent large randomized trials and contradictory recommendations from guidelines panels have led to controversy about the best approach to prophylaxis and treatment.
Dr. Alok Khorana will discuss emerging data and controversies in the field of cancer-associated thrombosis. Novel risk-assessment tools, biomarkers, and prophylaxis strategies, including results of recent randomized studies, will be outlined.
Dr. Michael Streiff will provide insight into results of large thromboprophylaxis studies conducted in medical and other populations, including the Prophylaxis of Thromboembolism in Critical Care (PROTECT) and the Study to Evaluate the Mortality Reduction of Enoxaparin in Hospitalized Acutely Ill Medical Receiving Enoxaparin (LIFENOX) trials. Controversial recommendations from recent guidelines panels will also be discussed.
Dr. Pieter Kamphuisen will explore optimal diagnostic and management strategies for catheter-related thrombosis in the contemporary era, with updates from recent studies.
Chair:
Alok A. Khorana, MD
University of Rochester, Rochester, NY, USA
Speakers:
Alok A. Khorana, MD
University of Rochester, Rochester, NY, USA
Cancer-Associated Thrombosis: Updates and Controversies
Michael B. Streiff, MD
The Johns Hopkins Hospital, Baltimore, MD, USA
Thromboprophylaxis in Non-Surgical patients
Pieter W. Kamphuisen, MD, PhD
University Medical Center Groningen, Groningen, Netherlands
Life Line or a Pain in the Neck: Catheter-Related Thrombosis
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Untangling Uncommon Lymphoproliferative Disorders
| Session Offered Twice |
December 9, 2012, 9:30 AM - 11:00 AM
Georgia World Congress Center (Thomas Murphy Ballroom 3-4, Building B, Level 5, Georgia World Congress Center) | December 10, 2012, 10:30 AM - 12:00 PM
Georgia World Congress Center (Georgia Ballroom 2, Building C, Level 3, Georgia World Congress Center) |
This session will present recent advances in the molecular pathogenesis and management of three rare and distinct lymphoproliferative disorders: large granular lymphocyte (LGL) leukemia, hairy cell leukemia (HCL), and prolymphocytic leukemia (PLL). There will be a focus on the understanding of underlying mechanisms of disease and how these can be exploited to develop effective therapy.
Dr. Thomas Loughran will review the molecular pathogenesis of LGL leukemia focusing on global dysregulation of apotoposis and survival pathways which are constitutively activated in leukemic LGL. He will then discuss clinical manifestations, indications for treatment, and therapeutic options for this disease.
Dr. Robert Kreitman will describe the clinical development of immunoconjugates for HCL, including the results of clinical trials using agents that target CD22 and CD25. He will also discuss the current understanding of the molecular pathogenesis of HCL and HCL-variant, including the role of the BRAF gene and the prognostic impact of specific immunoglobulin gene usage, such as VH4-34.
Dr. Claire Dearden will discuss PLL of B- and T-cell sub-types, briefly outlining the clinical presentation and diagnosis before concentrating on management of these disorders with an emphasis on the use of monoclonal antibody therapy.
Chair:
Claire E. Dearden, MD
The Royal Marsden Hospital, London, United Kingdom
Speakers:
Thomas P. Loughran Jr., MD
Pennsylvania State Hershey Cancer Institute, Hershey, PA, USA
Large Granular Lymphocytic Leukemia: Molecular Pathogenesis, Clinical Manifestations and Treatment
Robert J. Kreitman, MD
National Cancer Institute, National Institutes of Health, Bethesda, MD, USA
Immunoconjugates and New Molecular Targets In Hairy Cell Leukemia
Claire E. Dearden, MD
The Royal Marsden Hospital, London, United Kingdom
B- and T-Cell Prolymphocytic Leukemia: Antibody Approaches
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