Blood and Marrow Transplant Clinical Trials Network (BMT CTN) - Core Clinical C*

描述(由申请人提供)：骨髓移植CTN的目标是进行II期和III期多中心试验，以促进造血干细胞移植(HSCT)技术的发展，并衡量与非移植疗法相比对造血疾病的治疗优势。Moffitt BMT计划的科学环境将为多中心试验的原始概念的发展提供肥沃的土壤，招募患者，并有助于传播HSCT领域的新知识，从而为BMT CTN的成功做出贡献。具体目标1(建议的概念)：在BMT CTN实施的III期多中心试验中，在无关供者HSCT之前测试ATG预防GVHD的有效性。在标准药物预防中加入ATG将更有效地预防无关供者HSCT后11L-IV级急性移植物抗宿主病(GVHD)。ATG还可以减少慢性移植物抗宿主病，提高患者的生活质量。ATG预防急性GVHD的有效性只有在开发出有效的方法来减少EB病毒(EBV)再激活的发生率和预防移植后淋巴增生性疾病(PTLD)才能完全实现，而PTLD是ATG治疗最严重的并发症。在接受ATG治疗的患者中，我们将检验这样一个假设，即与利妥昔单抗先发制人治疗相比，利妥昔单抗预防可以降低EBV再激活的发生率。具体目标2：在设计和开展多中心研究以及传播研究结果方面与BMT CTN合作。虽然新的想法通常是从单个中心的个人或研究团队开始的，但医疗保健方面的重大进步是通过多中心III期随机试验实现的。莫菲特癌症中心在HSCT方面拥有完善的临床和转化性研究计划，其基础设施可为每年400例HSCT提供便利。我们的团队带来了GVHD免疫生物学、骨髓瘤治疗、癌症患者行为治疗和循证研究方面的专业知识。我们现在每年进行350多例HSCT，其中包括130例异基因HSCT，并承诺向在我们机构接受自体和异基因HSCT的患者提供BMT CTN试验。相关性(见说明)：血液和骨髓移植临床试验网络将通过提高造血干细胞移植的疗效和降低毒性来促进移植技术的发展。从血液疾病中治愈的患者数量将会增加。莫菲特癌症中心将为多中心临床试验、患者登记、数据分析和知识传播的概念做出贡献。

DESCRIPTION (provided by applicant): The goal of the BMT CTN is to conduct phase II and III multicenter trials to advance the hematopoietic stem cell transplantation (HSCT) technology and measure therapeutic advantages over non transplant therapies for hematopoietic disorders. The scientific environment of the Moffitt BMT Program will contribute to the success of the BMT CTN by providing fertile ground for the development of original concepts for multicenter trials, enrolling patients, and contributing to disseminate new knowledge in the field of HSCT. Specific Aim 1 (proposed concept): Test the efficacy of ATG for GVHD prophylaxis before unrelated donor HSCT in a phase III multicenter trial implemented by the BMT CTN. The addition of ATG to standard pharmacologic prophylaxis will provide more effective prevention of grade lll-IV acute GVHD following unrelated donor HSCT. ATG will also reduce chronic GVHD and improve patient quality of life. The efficacy of ATG for acute GVHD prophylaxis will not be fully realized until effective approaches are developed to reduce the incidence of Epstein-Barr Virus (EBV) reactivation and prevent post-transplant lymphoproliferative disorders (PTLD), that are the most serious complications of ATG therapy. In ATG- treated patients, we will test the hypothesis that rituximab prophylaxis can reduce the incidence of EBV reactivation compared to rituximab preemptive therapy. Specific Aim 2: Collaborate with the BMT CTN in the design and conduct of multicenter studies and the dissemination of the results. Although novel ideas start from individuals or teams of investigators commonly at a single center, significant advances in medical care occur through multicenter phase III randomized trials. The Moffitt Cancer Center has a fully established clinical and translational research program in HSCT with infrastructure to facilitate 400 HSCT per year. Our team brings expertise in GVHD immunobiology, myeloma therapy, behavioral therapy in cancer patients and evidence-based research. We perform now more than 350 HSCT including 130 allogeneic HSCT per year and pledge to make available BMT CTN trials available to patients receiving autologous and allogeneic HSCT in our institution. RELEVANCE (See instructions): The Blood and Marrow Transplantation Clinical Trial Network will advance transplantation technology by improving efficacy and decreasing toxicity of hematopoietic stem cell transplantation. The number of patients cured from blood disorders will increase. The Moffitt Cancer Center will contribute to the conception of multicenter clinical trials, patient enrollment, data analysis and knowledge dissemination.