2014 Bone Marrow Failure Disease Scientific Symposium

描述(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(PNH)是导致骨髓衰竭的罕见疾病。当骨髓不能产生血细胞时，会导致贫血、血小板减少(血液中的血小板减少)和中性粒细胞减少(中性粒细胞减少)，导致虚弱的症状和死亡。这三种疾病曾经被认为是不同的，现在被认为是由相似的病理生理联系在一起的。基础和临床科学家以及研究个别疾病的科学家之间加强合作，将极大地有助于探索目前在AA、MDS和PNH方面的研究。需要更多地了解导致这些疾病的分子事件和对治疗的反应，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国际基金会公司(AA&MDSIF)计划于2014年3月27-28日在马里兰州罗克维尔举行第四次骨髓衰竭疾病科学研讨会，达纳-法伯癌症研究所的理查德·M·斯通医学博士担任首席研究员和研讨会的联合主席，国家心肺和血液研究所的尼尔·S·杨医学博士担任联合主席。研讨会将把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聚集在一起，讨论当前达成共识和争议的领域，分享最新的研究成果，并就推进该领域所需的基础和临床研究的最优先方向提出建议。论坛将包括六个会议：(1)骨髓衰竭的遗传学和基因组学，(2)骨髓衰竭的病理生理学和新的分子靶点，(3)再生障碍性贫血和PNH的免疫生物学，(4)骨髓衰竭的移植治疗，(5)再生障碍性贫血的非移植治疗，以及(6)骨髓衰竭的非移植治疗。两个特别会议将讨论发展中国家的再生障碍性贫血以及临床和实验室中骨髓衰竭研究的未来。每一次会议都将提供提问和讨论的时间，并为未来的研究收集建议。为期两天的会议还将包括海报和讨论以及建立联系的机会。会议策划者将扩大以往吸引年轻研究人员参加研讨会(以及骨髓衰竭疾病研究领域)的努力，提供海报会议、旅行支持、与专家的更多讨论机会，以及在资金允许的情况下设立青年调查人员奖。AA&MDSIF将在其网站上发布包括研究建议在内的会议记录的通俗版本，会议领导人将在同行评议的期刊上为研究人员和临床医生发布会议记录摘要。

DESCRIPTION (provided by applicant): 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 (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 believed to be linked by similar pathophysiologies. Exploration of current research 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 fourth Bone Marrow Failure Disease Scientific Symposium on March 27-28, 2014, 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 and laboratory researchers studying the immunology and cell biology of bone marrow failure to discuss current areas of consensus and 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 sessions: (1) Genetics and Genomics of Bone Marrow Failure, (2) Pathophysiology and New Molecular Targets in MDS, (3) Immunobiology of AA and PNH, (4) Transplant Treatments for Bone Marrow Failure, (5) Non-Transplant Therapy for AA, and (6) Non-Transplant Therapy of MDS. Two special sessions will address AA in the developing world and the future of bone marrow failure 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 poster and discussion sessions and networking opportunities. The conference planners will expand on previous efforts to attract young investigators to the Symposium (and to the bone marrow failure 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.