2016 Bone Marrow Failure Disease Scientific Symposium

Aplastic Anemia & Mds International Fdn, Rockville MD

Investigators

Abstract

? DESCRIPTION (provided by applicant): Aplastic anemia (AA), myelodysplastic syndromes (MDS), and paroxysmal nocturnal hemoglobinuria (PNH) are rare diseases that result in failure of the bone marrow fails to produce blood cells. The resulting anemia, thrombocytopenia (low numbers of platelets in the bloodstream), and neutropenia (low numbers of neutrophils) lead to debilitating symptoms and death. Once considered distinct, these three diseases are now thought to be linked by similar pathophysiologies. Research on AA, MDS, and PNH would greatly benefit from increased collaboration between basic, clinical, and translational scientists and scientists studying the individual diseases. Increased understanding of the molecular events driving these diseases and of responses to treatment are needed to define at-risk populations and improve current therapies. The Aplastic Anemia & MDS International Foundation, Inc. (AA&MDSIF) proposes to hold its fifth Bone Marrow Failure (BMF) Disease Scientific Symposium on March 17-18, 2016, in Rockville, Maryland, with Richard M. Stone, M.D., from the Dana-Farber Cancer Institute, as Principal Investigator and Co-Chair of the Symposium and Neal S. Young, M.D., from the National Heart, Lung, and Blood Institute, as Co-Chair. The Symposium will bring together physicians treating these diseases; laboratory researchers studying the genetics, genomics, and pathophysiology of BMF; and clinical investigators to discuss current areas of consensus and controversy, share recent research results, and identify the highest-priority directions for basic and clinical research to advance th field. The format will consist of five main sessions: (1) Genetics and Genomics of BMF, (2) Pathophysiology and New Molecular Targets for BMF, (3) Immunobiology of BMF, (4) Transplant Treatments for BMF, and (5) Non-Transplant Treatments for BMF/Immunosuppression in BMF. Two special sessions will feature a roundtable on evolving therapies for the developing world and on the future of BMF studies in the clinic and the laboratory. Each session will offer time for questions and discussions as well as for capturing recommendations for future research. The two days of the conference will also include posters, discussion sessions and networking opportunities. The conference planners will expand on previous efforts to attract young investigators to the Symposium (and to the BMF disease research field) by offering poster sessions, travel support, extended discussion opportunities with experts, and, if funds permit, a Young Investigators Prize. AA&MDSIF will publish lay versions of the proceedings, including research recommendations, on its website, and meeting leaders will publish a summary of the proceedings for researchers and clinicians in a peer-reviewed journal. Narrative: Although rare, aplastic anemia, myelodysplastic syndromes (MDS), and paroxysmal nocturnal hemoglobinuria cause significant effects on health and can lead to death. Collaborative efforts between physicians treating these diseases and scientists studying the development of bone- marrow failure are needed to speed the development of effective new therapies. The Aplastic Anemia & MDS International Foundation, Inc. will hold its fifth Bone Marrow Failure Disease Scientific Symposium on March 17-18, 2016, to bring together physicians and researchers to discuss current areas of consensus and controversy, share recent research results, and propose recommendations to advance the field.