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

血液和骨髓移植临床试验网络 (BMT CTN) - 核心临床 C*

• 批准号: 8316236
• 负责人: CLAUDIO ANASETTI
• 金额: $ 17.28万
• 依托单位: H. LEE MOFFITT CANCER CTR & RES INST
• 依托单位国家: 美国
• 财政年份: 2011
• 资助国家: 美国
• 起止时间: 2011-08-08 至 2017-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8316236
• 关键词: Acute Graft Versus Host Disease; Acute leukemia; Address; Allogenic; Antithymoglobulin; Area; Autologous; Behavior Therapy; Blood; Bone Marrow Transplantation; Cancer Center; Cancer Patient; Caring; Cells; Cessation of life; Clinical; Clinical Protocols; Clinical Research; Clinical Trials; Clinical Trials Design; Clinical Trials Network; Conceptions; Cyclophosphamide; Data; Data Analyses; Development; Diagnosis; Disease; Dose; Enrollment; Environment; Future; Goals; Haplotypes; Hematologic Neoplasms; Hematological Disease; Hematopoietic; Hematopoietic Stem Cell Transplantation; Hematopoietic stem cells; Human Herpesvirus 4; Immunobiology; Immunotherapy; Incidence; Individual; Institution; Instruction; Knowledge; Large-Cell Lymphomas; Literature; Lymphoproliferative Disorders; Malignant Neoplasms; Marrow; Measures; Medical; Multicenter Studies; Multicenter Trials; Multiple Myeloma; Patients; Phase; Prevention; Principal Investigator; Prophylactic treatment; Protocols documentation; Quality of life; Radiation therapy; Recurrence; Regimen; Relapse; Research; Research Infrastructure; Research Personnel; Resistance; Risk; Science; Secure; Severities; Siblings; Sirolimus; Source; Stem cells; Supportive care; Tacrolimus; Technology; Testing; Therapeutic; Toxic effect; Translational Research; Transplantation; Umbilical Cord Blood; base; cancer cell; chemotherapeutic agent; chronic graft versus host disease; conditioning; design; disability; disorder prevention; efficacy testing; evidence base; experience; graft vs host disease; illness length; improved; mortality; novel; novel strategies; peripheral blood; pilot trial; prevent; programs; randomized trial; reconstitution; rituximab; success

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.

描述（由申请人提供）：BMT CTN 的目标是进行 II 期和 III 期多中心试验，以推进造血干细胞移植（HSCT）技术并衡量相对于非移植疗法治疗造血疾病的治疗优势。莫菲特 BMT 计划的科学环境将为多中心试验的原创概念的开发提供肥沃的土壤，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（提出的概念）：在 BMT CTN 实施的 III 期多中心试验中，测试 ATG 在无关供体 HSCT 之前预防 GVHD 的功效。在标准药物预防中添加 ATG 将更有效地预防无关供体 HSCT 后的 III-IV 级急性 GVHD。 ATG 还将减少慢性 GVHD 并改善患者的生活质量。在开发出有效的方法来降低 Epstein-Barr 病毒 (EBV) 再激活的发生率并预防移植后淋巴细胞增殖性疾病 (PTLD)（ATG 最严重的并发症）之前，ATG 对于急性 GVHD 预防的功效才能完全实现治疗。在 ATG 治疗的患者中，我们将检验以下假设：与利妥昔单抗先发性治疗相比，利妥昔单抗预防可以降低 EBV 再激活的发生率。具体目标 2：与 BMT CTN 合作设计和开展多中心研究以及传播结果。尽管新颖的想法通常来自单个中心的个人或研究团队，但通过多中心 III 期随机试验，医疗保健方面取得了重大进展。莫菲特癌症中心拥有完善的 HSCT 临床和转化研究计划，其基础设施每年可促进 400 例 HSCT。我们的团队带来 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。我们现在每年进行超过 350 例 HSCT，其中包括 130 例同种异体 HSCT，并承诺为在我们机构接受自体和同种异体 HSCT 的患者提供 BMT CTN 试验。相关性（参见说明）：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推进移植技术。血液病治愈人数将会增加。莫菲特癌症中心将为多中心临床试验、患者入组、数据分析和知识传播的构想做出贡献。