Abboud M R, Jackson S M, Barredo J, Beatty J, Laver J
Department of Pediatrics, Medical University of South Carolina, Charleston 29425.
Am J Pediatr Hematol Oncol. 1994 Feb;16(1):86-9.
To investigate the role of bone marrow transplantation in patients with severe sickle cell anemia (SCA).
We have designed a protocol for selecting patients with severe SCA who may benefit from bone marrow transplantation (BMT). On the basis of this protocol, a girl 3 9/12 years of age who had severe recurrent pain crises and splenic dysfunction received a BMT from her brother, who is homozygous for hemoglobin A.
Transplantation resulted in prompt engraftment, followed by durable hematologic and immunologic reconstitution with donor cells. One year after BMT, the patient continued to do well. She did not experience any graft versus host disease, her growth velocity increased, and recovery of splenic function was demonstrated. Since undergoing BMT, she has not experienced any painful crises.
Bone marrow transplantation is an effective therapeutic modality that should be considered in patients with severe SCA.
探讨骨髓移植在重症镰状细胞贫血（SCA）患者中的作用。
我们设计了一个方案来筛选可能从骨髓移植（BMT）中获益的重症SCA患者。基于此方案，一名3岁9个月大、患有严重复发性疼痛危机和脾功能不全的女孩接受了来自她哥哥（血红蛋白A纯合子）的BMT。
移植导致迅速植入，随后供体细胞实现持久的血液学和免疫重建。BMT后一年，患者情况持续良好。她未经历任何移植物抗宿主病，生长速度加快，脾功能得到恢复。自接受BMT以来，她未经历任何疼痛危机。
骨髓移植是一种有效的治疗方式，重症SCA患者应考虑采用。
