Bone marrow transplantation for sickle cell anemia.

M R Abboud, S M Jackson, J Barredo, J Beatty, J Laver
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Abstract

Purpose: To investigate the role of bone marrow transplantation in patients with severe sickle cell anemia (SCA).

Patients and methods: 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.

Results: 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.

Conclusions: Bone marrow transplantation is an effective therapeutic modality that should be considered in patients with severe SCA.

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骨髓移植治疗镰状细胞性贫血。
目的:探讨骨髓移植在重度镰状细胞性贫血(SCA)患者中的作用。患者和方法:我们设计了一个方案来选择可能受益于骨髓移植(BMT)的严重SCA患者。在此方案的基础上,一名患有严重复发性疼痛危机和脾功能障碍的3 /12岁女孩接受了血红蛋白a纯合子的兄弟的BMT。结果:移植导致迅速植入,随后是供体细胞持久的血液学和免疫学重建。BMT后一年,患者继续表现良好。她没有发生任何移植物抗宿主病,生长速度加快,脾脏功能恢复。自从接受BMT以来,她没有经历过任何痛苦的危机。结论:骨髓移植是严重SCA患者应考虑的有效治疗方式。
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