AA&MDSIF Second Bone Marrow Failure Disease Scientific Symposium

描述（由申请人提供）： 再生障碍性贫血（AA）、骨髓增生异常综合征（MDS）和阵发性睡眠性血红蛋白尿症（PNH）是罕见的疾病，均会导致骨髓衰竭。当骨髓无法产生血细胞时，贫血、血小板减少和中性粒细胞减少会导致衰弱症状和死亡。一旦被认为是不同的，这三种疾病现在被认为是由相似的病理生理学联系在一起。探索AA，MDS和PNH的当前研究问题将大大受益于基础和临床科学家之间的合作，以及研究个体疾病的科学家之间的合作。需要增加对驱动这些疾病的分子事件以及对治疗的反应的理解，以确定高危人群并改进当前的治疗方法。再生障碍性贫血和MDS国际基金会。(AA和MDSIF）建议在2009年秋季举行第二届骨髓衰竭疾病科学研讨会，理查德M。Stone，医学博士来自丹娜-法伯癌症研究所的首席研究员和Neal S. Young，医学博士来自国家心肺血液研究所的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的免疫学和细胞生物学的实验室研究人员，讨论当前的争议领域，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具体建议。会议形式将包括六场专题介绍会，每场会议结束时有时间提问和讨论，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会，以及建立联系的机会。会议记录，包括研究建议，将在AA&MDSIF网站上在线发布，并免费向公众提供。 (End摘要）

DESCRIPTION (provided by applicant): 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. (End of Abstract)