STEM CELL TRANSPLANTS FROM HLA-IDENTICAL FAMILY MEMBERS

来自 HLA 相同家族成员的干细胞移植

• 批准号: 6202258
• 负责人: Rainer F. Storb
• 金额: $ 17.57万
• 依托单位: FRED HUTCHINSON CANCER RESEARCH CENTER
• 依托单位国家: 美国
• 财政年份: 1999
• 资助国家: 美国
• 起止时间: 1999-08-01 至 2000-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6202258
• 关键词: aplastic anemia; artificial immunosuppression; autoimmune disorder; blood disorder chemotherapy; bone marrow purging; bone marrow transplantation; clinical research; clinical trials; combination chemotherapy; cyclophosphamide; cyclosporines; graft versus host disease; hematopoietic tissue transplantation; homologous transplantation; human subject; human therapy evaluation; longitudinal human study; methotrexate; transplant rejection

We propose to decrease the morbidity and mortality associated with severe aplastic anemia by continuing the evaluation of its treatment by marrow grafting using HLA-identical family members as donors. We hope to define the ultimate role of this approach and compare short-and long-term results to those obtained with unrelated marrow grafts and to results without marrow transplantation. Studies will address the problems of graft rejection, acute and chronic graft-host disease (GVHD), and long-term sequelae. During the next grant period, we anticipate, firstly, to evaluate novel GVHD prevention consisting of a combination of mycophenolate mofetil and cyclosporine, and, secondly, to evaluate the usefulness of novel conditioning regimens consisting of monoclonal antibodies to lymphocyte surface determinants as substitute for the traditionally used cyclophosphamide regimen. We will also continue exploring allogeneic marrow transplantation for the treatment of various genetically hematological disorders, including sickle cell-anemia. Finally, we shall explore a new area of investigation, transplantation of allogeneic stem cells as therapy of patients with severe or refractory autoimmune diseases, e.g. scleroderma.

我们建议降低与严重疾病相关的发病率和死亡率 通过继续评估再生障碍性贫血的骨髓治疗 使用 HLA 相同的家庭成员作为捐赠者进行移植。 我们希望定义 这种方法的最终作用并比较短期和长期结果 对于那些通过不相关的骨髓移植获得的结果以及未经 骨髓移植。 研究将解决贪污问题 排斥反应、急性和慢性移植物宿主病（GVHD）以及长期 后遗症。 在下一个资助期内，我们首先预计 评估由霉酚酸酯组合组成的新型 GVHD 预防措施 莫非替尔和环孢菌素，其次，评估其有用性 由单克隆抗体组成的新型预处理方案 淋巴细胞表面决定簇作为传统使用的替代品 环磷酰胺方案。 我们还将继续探索同种异体骨髓移植 治疗各种遗传性血液疾病，包括镰状细胞病 细胞性贫血。 最后，我们将探索一个新的研究领域， 同种异体干细胞移植治疗重症患者 或难治性自身免疫性疾病，例如硬皮病。