HLA单倍型相合外周血造血干细胞移植治疗重型地中海贫血

目的 探索单倍型相合外周血造血干细胞移植(PBHSCT)治疗重型地中海贫血的可行性.方法 以氟达拉滨+白舒非+环磷酰胺+ATG+低剂量TBI作为预处理方案,以环孢素A+骁悉+甲氨蝶呤预防GVHD,对5例重型地贫患儿进行单倍型相合PBHSCT.结果中位随访24月,5例患者全部存活,其中4例无病存活.结论 单倍型相合PBHSCT是治疗重型地中海贫血的有效方法.

Observations on After-Effect Duration of Kidney-Nourishing and Marrow-Replenishing Therapy on 58 Cases of Mediterranean Anemia

To observe the after-effect duration of kidney-nourishing and marrow-replenishing therapy on Mediterranean anemia. Methods： To observe the kidney-nourishing and marrow-replenishing therapy on 58 cases of Mediterranean anemia and the influence of various relative factors on the after-effect duration. Results： The after-effect duration on 58 cases varied from 3-6 months, about 4 months on average, and was not influenced by sex, clinical types, genetic types, types of Mediterranean anemia and other factors. Conclusion： Kidney-nourishing and marrow-replenishing therapy used to treat Mediterranean anemia can not only produce good therapeutic effect during treatment but also keep after effect lasting for about 4 months, indicating that the therapy used to treat Mediterranean anemia has good clinical after effect.