Bone marrow transplantation in sickle cell anemia.
Hematopoietic cell transplantation (HCT) is a treatment with curative potential for sickle cell disease (SCD). The experience of HCT for persons with beta-thalassemia major has been successfully extended to SCD. Currently, the event-free survival rate after allogeneic-matched sibling HCT for SCD is 82%. However, short-term and long-term transplant-related complications remain substantial barriers to HCT, particularly in older patients with life-long complications of SCD. Novel conditioning regimens that minimize transplant-associated toxicity have been developed and show promise for wider application of HCT. Alternative stem cell sources may also expand the availability of HCT for selected patients with SCD.
Department of Hematology/Oncology, Children's Hospital Oakland, Oakland, California 96409, USA.
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Hoppe
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Walters
D000755:Anemia, Sickle Cell / Q000628:therapy*; D016026:Bone Marrow Transplantation; D005312:Fetal Blood; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D014019:Tissue Donors / Q000600:supply & distribution*