BMT Clincal Trial Network at Stanford

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。它受到供者可获得性、移植相关毒性、移植物抗宿主病(GVHD)、恶性复发、感染以及对一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)继续在前一个资助期取得进展，建议开发和执行科学上有价值的、前瞻性的临床试验，以解决HCT的关键问题。我们将在六个关键领域进行多中心第二阶段试验，更重要的是，预期第三阶段试验，包括：替代供体和移植物来源；与方案相关的毒性；移植物抗宿主病；疾病复发；感染和免疫重建；以及后遗症和生活质量。由于儿科移植的复杂性，特别是罕见遗传性疾病的复杂性和研究难度，我们将特别关注和集中科学知识来使用协调的 网络的力量，以改善这一独特人群的BMT结果。BMT CTN还将通过合作研究和对生物终点的辅助研究来扩大和利用网络资源，这些生物终点将与临床终点整合并补充。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，为可能从骨髓移植中受益的大量患者提供帮助，并最大限度地成功完成高质量和高优先级的临床试验。 (摘要结束。)

DESCRIPTION (provided by applicant): Hematopoietic stem cell (HCT) transplantation offers curative therapy for a variety of malignant and non-malignant 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 posttransplant 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.)