BONE MARROW TRANSPLANTATION IN CHILDREN WITH SICKLE CELL DISEASE COMPLICATIONS

我们建议评估异基因骨髓移植的疗效。 移植治疗患有镰状细胞性贫血的患者 症状性疾病。骨髓移植已经被证明是 对一些由基因决定的疾病的有效治疗。这个 在骨髓移植方面最有经验的是治疗 严重的地中海贫血。研究表明，总体上没有任何事件 使用丁硫丹和环磷酰胺联合用药的存活率为75% 作为一种调理养生法。在未满16岁的患者中 肝肿大或门脉纤维化的3年无事件生存 超过90%。镰状细胞性贫血患者的总存活率 通过采取支持性和预防性护理措施，情况有所改善。然而， 在管理这些项目时会产生严重的发病率和费用 病人。将对患者进行骨髓移植 患有镰状细胞性贫血的人有明显的发病率风险 疾病。患者将接受白花丹、抗胸腺细胞球蛋白治疗 和环磷酰胺，接受异基因骨髓移植 来自一个与人类白细胞抗原完全相同的家庭成员。将进行研究，以 评估手术的并发症，如器官毒性、移植物 排斥反应，移植物抗宿主病，免疫缺陷和长期- 包括生活质量评估在内的长期后遗症。这些研究 应该有助于定义骨髓移植作为一种 这种疾病的治疗方法。

We propose to evaluate the efficacy of allogeneic bone marrow transplantation as therapy for patients with sickle cell anemia who have symptomatic disease. Bone marrow transplantation has proven to be effective therapy for a number of genetically determined diseases. The largest experience with marrow transplantation has been in the treatment of thalassemia major. Studies have demonstrated an overall event-free survival of 75% using the combination of busulfan and cyclophosphamide as a conditioning regimen. In patients under the age of 16 without hepatomegaly or portal fibrosis the 3-year event-free survival has been in excess of 90%. Overall survival for patients with sickle cell anemia has improved with supportive and preventive care measures. However, significant morbidity and expense are incurred in managing these patients. Bone marrow transplantation will be performed in patients with sickle cell anemia at risk for significant morbidity from their disease. Patients will be treated with busulfan, antithymocyte globulin and cyclophosphamide and receive an allogeneic bone marrow transplant from an HLA-identical family member. Studies will be performed to evaluate complications from the procedure such as organ toxicity, graft rejection, graft versus host disease, immunological deficiency and long- term sequelae including quality of life assessments. These studies should help define the role of bone marrow transplantation as a therapeutic modality in this disease.