Walters M C, Patience M, Leisenring W, Rogers Z R, Dinndorf P, Davies S C, Roberts I A, Yeager A, Kurtzberg J, Bunin N, Scott J P, Wall D A, Wayne A S, Wiley J, Darbyshire P J, Mentzer W C, Smith F O, Sullivan K M
Fred Hutchinson Cancer Research Center and University of Washington, Seattle 98104, USA.
Biol Blood Marrow Transplant. 1997 Dec;3(6):310-5.
We present updated results of a multicenter collaborative investigation of bone marrow transplantation for sickle cell disease. Between September 1991 and April 1997, thirty-four children less than 16 years of age with severe sickle cell disease received marrow allografts from HLA-identical siblings. Indications for transplantation included a history of stroke (n = 17), recurrent acute chest syndrome or sickle pulmonary disease (n = 10), and recurrent vaso-occlusive crises (n = 7). Twenty-one patients received regular red blood cell (RBC) transfusions to prevent complications of sickle cell disease. Patients were prepared for transplantation with busulfan, cyclophosphamide, and antithymocyte globulin or CAMPATH (Cambridge Pathology) antibody. Thirty-two of the 34 patients survived, with a median follow-up of 26.5 months (range, 0.2-66.9 months); and 28 patients demonstrated stable engraftment of donor hematopoietic cells. Graft rejection or recurrence of sickle cell disease occurred in four patients, and two patients died of intracranial hemorrhage or graft-vs.-host disease. In the group of 34 children with symptoms of advanced sickle cell disease, current Kaplan-Meier estimates of survival and event-free survival are 93% and 79%, respectively.
我们展示了一项关于镰状细胞病骨髓移植的多中心协作研究的最新结果。在1991年9月至1997年4月期间，34名16岁以下患有严重镰状细胞病的儿童接受了来自 HLA 匹配同胞的骨髓同种异体移植。移植指征包括有中风病史（n = 17）、复发性急性胸部综合征或镰状肺疾病（n = 10）以及复发性血管闭塞性危机（n = 7）。21名患者接受了定期红细胞（RBC）输血以预防镰状细胞病的并发症。患者接受白消安、环磷酰胺以及抗胸腺细胞球蛋白或 CAMPATH（剑桥病理学）抗体进行移植预处理。34名患者中有32名存活，中位随访时间为26.5个月（范围为0.2 - 66.9个月）；28名患者显示供体造血细胞稳定植入。4名患者发生移植物排斥或镰状细胞病复发，2名患者死于颅内出血或移植物抗宿主病。在这34名患有晚期镰状细胞病症状的儿童组中，目前采用 Kaplan - Meier 法估计的生存率和无事件生存率分别为93%和79%。
