2014 Bone Marrow Failure Disease Scientific Symposium

2014年骨髓衰竭疾病科学研讨会

• 批准号: 8723629
• 负责人: Richard M Stone
• 金额: $ 0.5万
• 依托单位: APLASTIC ANEMIA & MDS INTERNATIONAL FDN
• 依托单位国家: 美国
• 财政年份: 2014
• 资助国家: 美国
• 起止时间: 2014-03-15 至 2015-02-28
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8723629
• 关键词: Address; Anemia; Aplastic Anemia; Area; Automobile Driving; Basic Science; Blood Cells; Blood Circulation; Bone Marrow Transplantation; Calculi; Cellular biology; Cessation of life; Clinic; Clinical; Clinical Research; Collaborations; Consensus; Dana-Farber Cancer Institute; Development; Disease; Doctor of Medicine; Dysmyelopoietic Syndromes; Event; Foundations; Functional disorder; Funding; Future; Genetic; Genomics; Hemorrhage; Immunobiology; Immunology; Individual; Infection; International; Journals; Laboratories; Laboratory Scientists; Lead; Link; Marrow; Maryland; Molecular; Molecular Target; Morbidity - disease rate; National Heart, Lung, and Blood Institute; Neutropenia; Pancytopenia; Participant; Pathogenesis; Peer Review; Pharmaceutical Preparations; Physicians; Platelet Count measurement; Populations at Risk; Principal Investigator; Prize; Publishing; Rare Diseases; Recommendation; Research; Research Personnel; Scholarship; Science; Scientist; Specialist; Speed; Symptoms; Thrombocytopenia; Time; Training; Translational Research; Transplantation; Travel; United States National Institutes of Health; career; experience; improved; insight; meetings; mortality; neutrophil; novel strategies; posters; public health relevance; rat Piga protein; response; symposium; web site; Address; Anemia; Aplastic Anemia; Area; Automobile Driving; Basic Science; Blood Cells; Blood Circulation; Bone Marrow Transplantation; Calculi; Cellular biology; Cessation of life; Clinic; Clinical; Clinical Research; Collaborations; Consensus; Dana-Farber Cancer Institute; Development; Disease; Doctor of Medicine; Dysmyelopoietic Syndromes; Event; Foundations; Functional disorder; Funding; Future; Genetic; Genomics; Hemorrhage; Immunobiology; Immunology; Individual; Infection; International; Journals; Laboratories; Laboratory Scientists; Lead; Link; Marrow; Maryland; Molecular; Molecular Target; Morbidity - disease rate; National Heart, Lung, and Blood Institute; Neutropenia; Pancytopenia; Participant; Pathogenesis; Peer Review; Pharmaceutical Preparations; Physicians; Platelet Count measurement; Populations at Risk; Principal Investigator; Prize; Publishing; Rare Diseases; Recommendation; Research; Research Personnel; Scholarship; Science; Scientist; Specialist; Speed; Symptoms; Thrombocytopenia; Time; Training; Translational Research; Transplantation; Travel; United States National Institutes of Health; career; experience; improved; insight; meetings; mortality; neutrophil; novel strategies; posters; public health relevance; rat Piga protein; response; symposium; web site

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.

描述（由申请人提供）：性障碍性贫血（AA），骨髓增生综合征（MDS）和阵发性夜间夜间血红蛋白尿（PNH）是罕见的疾病，导致骨髓衰竭。当骨髓无法产生血细胞时，由此产生的贫血，血小板减少症（血液中的血小板数量少）和中性粒细胞减少症（中性粒细胞数量低）会导致症状和死亡。一旦被认为是不同的，这三种疾病现在被认为与类似的病理生理联系在一起。对AA，MDS和PNH当前研究的探索将大大受益于基本和临床科学家与研究各个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了解，以定义高危人群并改善当前疗法。 AA和MDS International Foundation，Inc。（AA＆MDSIF）于2014年3月27日至28日在马里兰州罗克维尔与Dana-Farber Cancer Cancer Institute，作为首席研究员和校长Internity of Flool S. Younger，M。Younger，M。Younger，M。Youngh s。作为联合主席。该研讨会将汇集治疗这些疾病和实验室研究人员的医生，研究骨髓的免疫学和细胞生物学，未能讨论当前共识和争议的领域，分享最新的研究结果，并提出建议，提出建议，以提出有关进步该领域所需的基本和临床研究的最高优先级方向。该格式将由六个会话组成：（1）骨髓衰竭的遗传学和基因组学，（2）MDS中的病理生理学和新分子靶标，（3）AA和PNH的免疫生物学，（4）骨髓衰竭的移植治疗，（4）骨骨髓衰竭，（5）非移植治疗AA和（6）NON-NON-NON-NON-NON-STRANS PRENSPLANT FRANSANS FRINSANS FRANSANS FRANSANS FRANSANS FRANSANS FRANSANS FRANSANS FRANSANS FRASTANT。在诊所和实验室中，有两个特别的会议将解决发展中国家的AA和骨髓衰竭研究的未来。每个会议将为您提供问题和讨论的时间，以及捕获未来研究的建议。会议的两天还将包括海报和讨论会议和网络机会。会议策划者将通过提供海报会议，旅行支持，与专家的扩大讨论机会以及年轻调查人员奖，以吸引年轻调查人员吸引年轻调查人员参加研讨会（以及骨髓衰竭疾病研究领域）的努力。 AA＆MDSIF将在其网站上发布诉讼程序（包括研究建议）的外行版本，会议领导者将在经过同行评审的期刊上发布有关研究人员和临床医生的诉讼程序的摘要。