Stem Cell Transplantation

干细胞移植

• 批准号: 7294710
• 负责人: Rainer F. Storb
• 金额: $ 16.15万
• 依托单位: FRED HUTCHINSON CANCER RESEARCH CENTER
• 依托单位国家: 美国
• 财政年份: 2006
• 资助国家: 美国
• 起止时间: 2006-08-15 至 2011-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7294710
• 关键词: FK506; aplastic anemia; artificial immunosuppression; blood donor; bone marrow transplantation; clinical trial phase II; congenital aplastic anemia; cord blood; cyclophosphamide; fludarabine; genetic disorder; graft versus host disease; hematopoietic stem cells; hematopoietic tissue transplantation; homologous transplantation; human subject; human therapy evaluation; monoclonal antibody; mycophenolate mofetil; patient oriented research; stem cell transplantation; transplant rejection

Two overall objectives will be pursued in our continued efforts to treat patients with otherwise fatal nonmalignant hematologic diseases by allogeneic hematopoietic cell transplantation. One is to reduce regimen-related toxicities and transplant-related complications, including graft rejection and graft-vs-host disease. The other is to broaden the choice of hematopoietic cell donors beyond HLA-matched relatives and unrelated volunteers by including grafts of unrelated cord blood and HLA-haploidentical marrow cells. Nonmalignant disorders include three distinct disease entities, aplastic anemia. Fanconi anemia, and inherited diseases of the hematopoietic and immune systems. In order to assure accrual of patients with these relatively infrequent diseases to the proposed protocols, we have taken two steps. The first has been to include other academic centers as participants in the studies. The second, important for protocols involving patients with immunodeficiency diseases, has been to establish close collaborations with key members of the Pediatric Immunology group at the University of Washington/Children's Hospital and Regional Medical Center, who see and study many of these patients. The studies proposed under this Project have relevance for hematopoietic cell transplantation in patients with hemoglobinopathies, such as sickle cell disease and thalassemia major, autoimmune diseases, and those with malignant diseases, including myelodysplastic syndromes studied under Project 4 of this grant. Also, hematopoietic cell transplantation protocols, that are found to be both effective and safe for patients with hematologic diseases, might eventually be of interest in the treatment of recipients of solid organ grafts, e.g. kidney or lung. In that setting, a concurrent or preceding hematopoietic graft from the kidney or lung donor would provide an immunologic "platform" for the solid organ graft, which would be indefinitely accepted without the need for lifelong immunosuppression.

在我们继续努力治疗其他致命的患者时，将实现两个总体目标 同种异体造血细胞移植的非恶性血液学疾病。一个是减少 方案相关的毒性和与移植相关的并发症，包括移植物排斥和移植VS-HOST 疾病。另一个是将造血细胞供体的选择扩大到HLA匹配的亲戚之外 不相关的志愿者包括无关的脐带血和HLA - 帕克洛尼斯骨髓细胞的移植物。 非恶性疾病包括三个不同的疾病实体，共生性贫血。 Fanconi贫血和 造血和免疫系统的遗传疾病。 为了确保这些相对疾病的患者应对所提出方案的患者的应计， 我们已经采取了两个步骤。首先是将其他学术中心作为参与者 研究。第二个，对于涉及免疫缺陷疾病患者的方案很重要的是 与大学儿科免疫学小组的主要成员建立密切合作 华盛顿/儿童医院和地区医疗中心，他们看到并研究了许多患者。 该项目下提出的研究与患者的造血细胞移植有关 与镰状细胞疾病和丘脑贫血之类的血红蛋白病，自身免疫性疾病和 那些患有恶性疾病的人，包括根据本赠款项目4研究的骨髓增生综合症。 同样，造血细胞移植方案，发现对患者既有效又安全 对于血液学疾病，最终可能会引起固体器官移植的接受者的治疗， 例如肾脏或肺。在这种情况下，来自肾脏或肺的同时或先前的造血移植物 供体将为实体器官移植提供免疫学“平台”，这将是无限期的 接受而无需终身免疫抑制。