Third Bone Marrow Failure Disease Scientific Symposium

再生障碍性贫血与MDS国际基金会2012骨髓衰竭疾病科学研讨会项目摘要再生障碍性贫血（AA）、骨髓增生异常综合征（MDS）和阵发性夜间血红蛋白尿（PNH）是导致骨髓衰竭的罕见疾病。当骨髓不能产生血细胞时，由此引起的贫血、血小板减少症（血液中血小板不足）和中性粒细胞减少症（中性粒细胞不足）可导致衰弱症状和死亡。这三种疾病曾经被认为是不同的，但现在人们认为它们有相似的病理生理学联系。对AA、MDS和PNH当前研究问题的探索将极大地受益于基础和临床科学家与研究个体疾病的科学家之间加强合作。需要进一步了解导致这些疾病的分子事件以及对治疗的反应，以确定高危人群并改进目前的治疗方法。再生障碍性贫血与MDS国际基金会（AA&MDSIF）提议在2012年3月举行第三届骨髓衰竭疾病科学研讨会，由丹娜-法伯癌症研究所的Richard M. Stone医学博士担任首席研究员和研讨会联合主席，国家心脏、肺和血液研究所的Neal S. Young医学博士担任联合主席。研讨会将汇集治疗这些疾病的医生和研究骨髓衰竭免疫学和细胞生物学的实验室研究人员，讨论当前有争议的领域，分享最近的研究成果，并为推进该领域所需的基础和临床研究的最高优先方向提出建议。该形式将包括六次报告会，每一次报告会结束时都有时间提问和讨论，并为今后的研究提出建议。为期两天的会议还将包括海报和讨论环节以及交流机会。AA&MDSIF将在其网站上发布会议纪要的外行版本，包括研究建议，会议领导人将在同行评审的期刊上为研究人员和临床医生发布会议纪要摘要。

DESCRIPTION (provided by applicant): Aplastic Anemia & MDS International Foundation 2012 Bone Marrow Failure Disease Scientific Symposium Project Abstract Aplastic anemia (AA), myelodysplastic syndromes (MDS), and paroxysmal nocturnal hemoglobinuria (PNH) are rare diseases that result in bone marrow failure. When the marrow fails to produce blood cells, the resulting anemia, thrombocytopenia (shortage of platelets in the bloodstream), and neutropenia (neutrophil shortage) 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 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 third Bone Marrow Failure Disease Scientific Symposium in March 2012, 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 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 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. 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. PUBLIC HEALTH RELEVANCE: Aplastic Anemia & MDS International Foundation 2012 Bone Marrow Failure Disease Scientific Symposium Project Narrative Although rare, aplastic anemia, myelodysplastic syndromes, and paroxysmal nocturnal hemoglobinuria cause significant morbidity and mortality. 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. The Aplastic Anemia & MDS International Foundation, Inc. proposes to hold its third Bone Marrow Failure Disease Scientific Symposium in March 2012 to bring together physicians and laboratory researchers to discuss current controversies, share recent research results, and propose recommendations to advance the field.