Blood and marrow transplantation for sickle cell disease: Is less more?

Blood and marrow transplantation is a curative therapy for patients with sickle cell disease yet this option is seldom used. Clinical studies have shown however that children transplanted for this condition can achieve excellent results. In children with sickle cell disease transplanted following conditioning with busulfan, cyclophosphamide, and anti-thymocyte globulin, cure rates in excess of 80% can be obtained when an HLA-matched sibling is used as the donor. However, the large majority of patients with sickle cell disease will not have such a donor, or will not be able to tolerate high dose conditioning regimens.
Source: Blood Reviews - Category: Hematology Authors: Source Type: research