Bone marrow transplantation for hemoglobinopathies.
In patients with hemoglobinopathy who are treated by allogeneic matched sibling bone marrow transplantation before the onset of disease-associated organ damage, long term, disease-free survival currently stands at approximately 90%, and transplant-associated mortality is 5% or less. Less toxic nonmyeloablative conditioning regimens that have the potential to reduce procedure-related mortality to even lower levels are under active investigation. Expansion of the donor pool by use of unrelated matched donors awaits improvement in HLA-typing methodology.
Mentzer
W C
WC
University of California at San Francisco, USA.
D000755:Anemia, Sickle Cell / Q000628:therapy; D001521:Behavior Therapy; D016026:Bone Marrow Transplantation / Q000009:adverse effects* / Q000401:mortality; D002986:Clinical Trials as Topic; D006453:Hemoglobinopathies / Q000628:therapy*; D006801:Humans; D013789:Thalassemia / Q000628:therapy; D014184:Transplantation, Homologous / Q000009:adverse effects / Q000401:mortality