Bone marrow transplantation for hemoglobinopathies

Abstract

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.