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骨髓移植(BMT)为遗传缺陷病提供了血液学治愈的机会。早期对5岁以下的地中海贫血患者采用定期输血及去铁胺治疗后,在造血器官损伤极小时作 BMT,获得了最好的效果。一组1~15岁的222例地中海贫血患者,作了 HLA 相同供者的BMT 后,其生存率为82%,无病生存率为75%。并发现,移植成功与年龄和输血量无关,而与移植时病人的状况(危险因素)如肝脏肿大的程度(>2cm),任何程度的门静脉纤维化,是否进行了适当的鳌合
Bone marrow transplantation (BMT) provides a hematologic cure for genetic defects. Early use of transfusion and deferoxamine in patients with thalassemia less than 5 years of age achieved the best results with minimal hematopoietic organ damage. A group of 222 patients with thalassemia 1-15 years old who underwent BMT with HLA identical donors had a survival rate of 82% and a disease-free survival rate of 75%. And found that the success of transplantation and age and blood transfusion has nothing to do with the status of transplant patients (risk factors) such as liver enlargement (> 2cm), any degree of portal vein fibrosis, whether the appropriate chelation