Aplastic anemia (AA), the myelodysplastic syndromes (MDS), and paroxysmal nocturnal hemoglobinuria (PNH) are rare diseases that all result in bone marrow failure. When the marrow fails to produce blood cells, anemia, thrombocytopenia, and neutropenia can lead to debilitating symptoms and death. Once considered distinct, these three diseases are now believed to be linked by similar pathophysiologies. Exploration of current research issues in AA, MDS, and PNH would greatly benefit from increased collaboration between basic and clinical scientists, and between scientists studying the individual diseases. Increased understanding of the molecular events driving these diseases and of the response 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 second Bone Marrow Failure Disease Scientific Symposium in the fall of 2009, with Richard M. Stone, M.D. from Dana-Farber Cancer Institute as Principal Investigator 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 and laboratory researchers studying the immunology and cell biology of bone marrow failure to discuss current areas of controversy, share recent research results, and propose specific recommendations for the highest priority directions for basic and clinical research needed to advance the field. The format will consist of six presentation sessions, with time at the end of each session for questions and discussions, as well as for capture of recommendations for future research. The two days of the conference will also include poster and discussion sessions, and networking opportunities. The proceedings, including research recommendations, will be published online on the AA&MDSIF website and made freely available to the public.

Public Health Relevance

Second Aplastic Anemia &MDS International Foundation, Inc. Bone Marrow Failure Disease Scientific Symposium Although rare diseases, aplastic anemia (AA), the myelodysplastic syndromes (MDS), and paroxysmal nocturnal hemoglobinuria (PNH) cause significant morbidity and mortality in those afflicted. The incidence of MDS is expected to rise significantly with the aging of the United States'population. Collaborative efforts between physicians treating these diseases and laboratory scientists studying the pathophysiology of bone-marrow failure are needed to speed the development of effective new therapies.

Agency
National Institute of Health (NIH)
Institute
National Heart, Lung, and Blood Institute (NHLBI)
Type
Conference (R13)
Project #
1R13HL096252-01
Application #
7674176
Study Section
Special Emphasis Panel (ZHL1-CSR-N (F1))
Program Officer
Di Fronzo, Nancy L
Project Start
2009-09-01
Project End
2010-08-31
Budget Start
2009-09-01
Budget End
2010-08-31
Support Year
1
Fiscal Year
2009
Total Cost
$27,000
Indirect Cost
Name
Aplastic Anemia & Mds International Fdn
Department
Type
DUNS #
964688993
City
Rockville
State
MD
Country
United States
Zip Code
20850