2014 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）是导致骨髓衰竭的罕见疾病。当骨髓不能产生血细胞时，由此产生的贫血、血小板减少症（血液中血小板数量少）和中性粒细胞减少症（中性粒细胞数量少）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。探索目前在AA，MDS和PNH的研究将大大受益于基础和临床科学家和研究个别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&MDSIF）提议于2014年3月27日至28日在马里兰州的罗克维尔举行第四届骨髓衰竭疾病科学研讨会，理查德M。斯通，医学博士，来自丹娜-法伯癌症研究所，作为研讨会的首席研究员和联合主席，Neal S.杨博士，来自国家心脏，肺和血液研究所，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的免疫学和细胞生物学的实验室研究人员，讨论当前的共识和争议领域，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建议。该格式将包括六个会议：（1）骨髓衰竭的遗传学和基因组学，（2）MDS的病理生理学和新分子靶点，（3）AA和PNH的免疫生物学，（4）骨髓衰竭的移植治疗，（5）AA的非移植治疗，和（6）MDS的非移植治疗。两个特别会议将讨论AA在发展中国家和骨髓衰竭的研究在临床和实验室的未来。每次会议将提供时间进行问题和讨论，以及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会以及建立联系的机会。会议策划者将扩大以往的努力，以吸引年轻的研究人员参加研讨会（和骨髓衰竭疾病研究领域），提供海报会议，旅行支持，与专家进行广泛讨论的机会，如果资金允许，还将设立青年研究人员奖。AA&MDSIF将在其网站上发布会议记录的非专业版本，包括研究建议，会议领导人将在同行评审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.