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: 8165426
• 负责人: CLAUDIO ANASETTI
• 金额: $ 16.53万
• 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/8165426
• 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的目的是进行II和III阶段多中心试验，以推进血肿干细胞移植（HSCT）技术，并测量与非移植疗法的造血疾病的治疗优势。 Moffitt BMT计划的科学环境将通过为开发用于多中心试验的原始概念，招募患者并为HSCT领域传播新知识的原始概念提供肥沃的基础，从而有助于BMT CTN的成功。具体目标1（提出的概念）：在BMT CTN实施的III期多中心试验中，在无关的供体HSCT之前测试ATG对GVHD预防的功效。在不相关的供体HSCT之后，在标准药理预防中添加ATG将更有效地预防LLL-IV急性GVHD。 ATG还将减少慢性GVHD并提高患者的生活质量。在开发出有效的方法以降低Epstein-Barr病毒（EBV）重新激活的发生率并防止移植后淋巴细胞增生性疾病（PTLD）（PTLD）之前，ATG对急性GVHD预防的疗效将无法完全实现。在ATG治疗的患者中，我们将检验以下假设：与利妥昔单抗先发制体治疗相比，可利妥昔单抗预防可以降低EBV重新激活的发生率。具体目的2：与BMT CTN合作在多中心研究的设计和行为和结果传播中。尽管新颖的想法始于通常在一个中心的个人或研究人员团队，但医疗保健的重大进展是通过多中心III期随机试验而发生的。莫菲特癌症中心（Moffitt Cancer Center）在HSCT中拥有完全建立的临床和转化研究计划，并具有基础设施，每年促进400 HSCT。我们的团队在GVHD免疫生物学，骨髓瘤治疗，癌症患者的行为疗法和循证研究方面具有专业知识。现在，我们每年执行超过350个HSCT，其中包括130个同种异体HSCT，并保证为在我们机构中接受自体和同种异体HSCT的患者提供了BMT CTN试验。相关性（请参阅说明）：血液和骨髓移植临床试验网络将通过提高疗效和降低造血干细胞移植的毒性来提高移植技术。从血液疾病中治愈的患者人数将增加。莫菲特癌症中心将有助于多中心临床试验，患者入学，数据分析和知识传播的概念。