Bone marrow transplantation for sickle cell anemia.

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.