Blood Marrow Transplant Clinical Trials Research Network

描述（由申请人提供）： 造血干细胞（HCT）移植为各种恶性和非恶性疾病提供了治愈性治疗。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）从上一个资助期继续取得进展，建议开发和执行科学上有价值的前瞻性临床试验，解决HCT中的关键问题。我们将在六个关键领域进行多中心II期和更重要的前瞻性III期试验，包括：替代供体和移植物来源;方案相关毒性; GVHD;疾病复发;感染和免疫重建;以及晚期效应和生活质量。由于儿科移植的复杂性，特别是在那些罕见的遗传性疾病，这是复杂和难以研究，我们将特别关注和集中的科学专业知识，利用网络的协调力量，以改善BMT结果为这一独特的人群。BMT CTN 还将通过生物学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结合并补充临床终点。该网络还将寻求与其他科学机构的积极合作，包括NCI资助的癌症合作小组，以提高临床移植研究的效率，为大量可能从BMT中受益的患者，并最大限度地成功完成高质量和高优先级的临床试验。 (End抽象）。

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.)