Walters M C, Patience M, Leisenring W, Eckman J R, Scott J P, Mentzer W C, Davies S C, Ohene-Frempong K, Bernaudin F, Matthews D C, Storb R, Sullivan K M
Division of Clinical Research, Fred Hutchinson Cancer Research Center, Seattle, WA 98104, USA.
N Engl J Med. 1996 Aug 8;335(6):369-76. doi: 10.1056/NEJM199608083350601.
We investigated the risks and benefits of allogeneic bone marrow transplantation in children with complications of sickle cell disease.
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.
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 nervous system hemorrhage or graft-versus-host disease. Kaplan-Meier estimates of survival and event-free survival at four years were 91 percent and 73 percent, respectively. Among patients with a history of acute chest syndrome, lung function stabilized; among patients with prior central nervous system vasculopathy who had engraftment, stabilization of cerebrovascular disease was documented by magnetic resonance imaging.
Allogeneic stem-cell transplantation can be curative in young patients with symptomatic sickle cell disease.
我们研究了异基因骨髓移植治疗镰状细胞病并发症患儿的风险和益处。
1991年9月至1995年4月期间，22名年龄小于16岁的有症状镰状细胞病患儿接受了来自 HLA 相同同胞的骨髓移植。移植指征包括中风病史（n = 12）、复发性急性胸综合征（n = 5）和复发性疼痛危象（n = 5）。患者接受白消安、环磷酰胺和抗胸腺细胞球蛋白预处理以进行移植。
22名患者中有20名存活，中位随访时间为23.9个月（范围10.1至51.0个月），16名患者供体造血细胞稳定植入。3名患者移植被排斥，镰状细胞病复发；第四名患者移植排斥伴有骨髓再生障碍。16名植入患者中有1名出现稳定的混合嵌合体。2名患者死于中枢神经系统出血或移植物抗宿主病。四年时的Kaplan-Meier生存估计和无事件生存估计分别为91%和73%。在有急性胸综合征病史的患者中，肺功能稳定；在先前有中枢神经系统血管病变且植入的患者中，磁共振成像显示脑血管疾病稳定。
异基因干细胞移植可治愈有症状的镰状细胞病年轻患者。
