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 镰状细胞性贫血患者的移植治疗 症状性疾病 骨髓移植已被证明是 对许多遗传决定的疾病的有效治疗。 的 骨髓移植的最大经验是在治疗方面， 重型地中海贫血 研究表明， 使用白消安和环磷酰胺的组合的存活率为75% 作为一种调理方案。 16岁以下的患者， 肝肿大或门静脉纤维化，3年无事件生存率 超过90%。 镰状细胞性贫血患者的总生存期 通过支持性和预防性护理措施得到改善。 然而，在这方面， 在管理这些疾病时会产生显著发病率和费用 患者 将在患者中进行骨髓移植 镰状细胞性贫血的患者， 疾病 患者将接受白消安，抗胸腺细胞球蛋白 和环磷酰胺，并接受异基因骨髓移植 来自一个HLA相同的家族成员 将进行研究， 评估手术并发症，如器官毒性、移植物 排斥、移植物抗宿主病、免疫缺陷和长期- 长期后遗症，包括生活质量评估。 这些研究 应该有助于确定骨髓移植的作用， 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.