Competing Application for Blood and Marrow Transplant Network

血液和骨髓移植网络竞争应用

• 批准号: 7125219
• 负责人: JOANNE KURTZBERG
• 金额: $ 16.49万
• 依托单位: DUKE UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2001
• 资助国家: 美国
• 起止时间: 2001-09-30 至 2011-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7125219
• 关键词: bone marrow transplantation; clinical research; clinical trials; cooperative study; cord blood; disease /disorder prevention /control; hematopoietic tissue transplantation; human subject; human therapy evaluation; infection; patient oriented research; stem cell transplantation

DESCRIPTION (provided by applicant): Hematopoietic stem cell (HCT) transplantation offers curative therapy for a variety of malignant and nonmalignant disorders. It is limited by donor availability, transplant related toxicity, graft vs. host disease (GVHD), malignant relapse, infections, and for some patients, reduction in their post-transplant quality of life. Continuing progress from the previous funding period, the Blood and Marrow Transplant Clinical Trials Network (BMT CTN) proposes to develop and execute scientifically meritorious, prospective clinical trials addressing key issues in HCT. We will conduct multicenter Phase II and more importantly, prospective Phase III trials in six key areas including: alternative donors and graft sources; regimen related toxicity; GVHD; disease recurrence; infection and immune reconstitution; and late effects and quality of life. Due to the complexity of pediatric transplantation, especially in those with rare inherited disorders, which are complex and difficult to study, we will devote special attention and focused scientific expertise to using the coordinated strength of the Network to improve BMT outcomes for this unique population. The BMT CTN will also amplify and leverage Network resources through collaborative research and ancillary studies of biologic endpoints that will be integrated with and complement clinical endpoints. The Network will also seek active collaboration with other scientific bodies including NCI- funded Cancer Cooperative Groups to improve the efficiency of clinical transplant studies for the large number of patients who could benefit from BMT and to maximize our successful completion of high quality and high priority clinical trials. (End of abstract.)

描述（由申请人提供）： 造血干细胞（HCT）移植为多种恶性和非恶性疾病提供了治疗方法。它受到供体可用性、移植相关毒性、移植物抗宿主病（GVHD）、恶性复发、感染以及某些患者移植后生活质量下降的限制。血液和骨髓移植临床试验网络 (BMT CTN) 在上一个资助期的基础上继续取得进展，提议开发和执行具有科学价值的前瞻性临床试验，解决 HCT 的关键问题。我们将在六个关键领域进行多中心二期试验，更重要的是，前瞻性三期试验，包括：替代供体和移植来源；与治疗方案相关的毒性；移植物抗宿主病；疾病复发；感染和免疫重建；以及后期影响和生活质量。由于儿科移植的复杂性，特别是那些患有罕见遗传性疾病的移植，这些疾病复杂且难以研究，我们将给予特别关注和集中的科学专业知识，利用网络的协调力量来改善这一独特人群的 BMT 结果。 BMT CTN 还将通过生物终点的合作研究和辅助研究来扩大和利用网络资源，这些研究将与临床终点相整合并补充。该网络还将寻求与包括 NCI 资助的癌症合作组织在内的其他科学机构的积极合作，以提高大量可以从 BMT 中受益的患者的临床移植研究的效率，并最大限度地成功完成高质量和高优先级的临床工作。试验。 （摘要结束。）