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.
Hoppe
C C
CC
Department of Hematology/Oncology, Children's Hospital Oakland, Oakland, California 96409, USA.
Walters
M C
MC
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*