TREATMENT OF APLASTIC ANEMIA WITH SUPPORTIVE CARE, HGFS, AND IMMUNOSUPPRESSIVES

使用支持性护理、HGFS 和免疫抑制剂治疗再生障碍性贫血

• 批准号: 3736531
• 负责人: KRISTINE C DONEY
• 金额: --
• 依托单位: FRED HUTCHINSON CANCER RESEARCH CENTER
• 依托单位国家: 美国
• 资助国家: 美国
• 起止时间: 至
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/3736531
• 关键词: aplastic anemia; artificial immunosuppression; biological response modifiers; blood disorder chemotherapy; bone marrow transplantation; cell mediated cytotoxicity; clinical trials; colony stimulating factor; combination chemotherapy; corticosteroids; corticosterone; dosage; drug screening /evaluation; dyserythropoietic anemia; hematopoietic growth factor; human subject; human therapy evaluation; immunosuppressive; interleukin 2; longitudinal human study; medical complication; methylprednisolone; monoclonal antibody; paroxysmal nocturnal hemoglobinuria; relapse /recurrence

This project continues to develop effective therapies for patients with aplastic anemia who are not candidates for genotypically HLA-identical donor bone marrow transplants. Previous studies performed in Seattle have shown that immunosuppressive regimens including anti-human thymocyte globulin (ATG), high or low-dose corticosteroids and androgens, achieves long-term survival in 44% of patients. In an effort to improve these results the role of hematopoietic growth factors given singly or in combination along with immunosuppressive therapy will be evaluated. Long-term follow-up of patients will determine the incidence of late complications such as recurrent aplasia, myelodysplastic syndrome, and paroxysmal nocturnal hemoglobinuria (PNH). Statistical analyses will be used to (1) identify clinical or laboratory parameters associated with response to therapy and (2) compare the results of immunosuppressive therapy to those of marrow transplantation.

该项目继续为患有以下疾病的患者开发有效的疗法 再生障碍性贫血患者不属于 HLA 基因相同的候选者 供体骨髓移植。 先前在西雅图进行的研究 已经表明，包括抗人胸腺细胞在内的免疫抑制方案 球蛋白 (ATG)、高或低剂量皮质类固醇和雄激素，可实现 44%的患者获得长期生存。 为了努力改善这些 结果是单独或联合给予造血生长因子的作用 将评估联合免疫抑制治疗。 对患者的长期随访将确定晚期的发生率 复发性发育不全、骨髓增生异常综合征等并发症 阵发性睡眠性血红蛋白尿（PNH）。 统计分析将 用于（1）识别与以下相关的临床或实验室参数 对治疗的反应和（2）比较免疫抑制的结果 骨髓移植治疗。