Bone marrow transplantation for sickle cell disease.
Bone marrow transplantation for sickle cell disease.
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DOI：
10.1136/adc.69.2.176
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发表时间：
1993-08
影响因子：
5.2
通讯作者：
S. Davies
中科院分区：
文献类型：
--
作者：
S. Davies
背景：我们研究了镰状细胞病并发症患儿异基因骨髓移植的风险和获益。方法1991年9月至1995年4月，22例16岁以下有症状的镰状细胞病患儿接受了HLA相合同胞的骨髓移植。移植指征包括卒中史（n = 12）、复发性急性胸部综合征（n = 5）和复发性疼痛危象（n = 5）。患者准备移植白消安，环磷酰胺，抗胸腺细胞球蛋白。结果22例患者中有20例存活，中位随访时间为23.9个月（10.1 ~ 51.0个月），16例患者的供者造血细胞稳定植入。在三名患者中，移植物被排斥，镰状细胞病复发;在第四名患者中，移植物排斥伴随着骨髓再生障碍性贫血。在16例植入患者中，有1例存在稳定的混合嵌合体。2例患者死于中枢神经系统疾病。
Background We investigated the risks and benefits of allogeneic bone marrow transplantation in children with complications of sickle cell disease. Methods Twenty-two children less than 16 years of age who had symptomatic sickle cell disease received marrow allografts from HLA-identical siblings between September 1991 and April 1995. The indications for transplantation included a history of stroke (n = 12), recurrent acute chest syndrome (n = 5), and recurrent painful crises (n = 5). Patients were prepared for transplantation with busulfan, cyclophosphamide, and antithymocyte globulin. Results Twenty of the 22 patients survived, with a median follow-up of 23.9 months (range, 10.1 to 51.0), and 16 patients had stable engraftment of donor hematopoietic cells. In three patients the graft was rejected and sickle cell disease recurred; in a fourth patient graft rejection was accompanied by marrow aplasia. In 1 of the 16 patients with engraftment, there was stable mixed chimerism. Two patients died of central ne...