Blood and marrow transplantation for sickle cell disease: overcoming barriers to success

Sickle cell disease (SCD) is a common health problem in the United States; yet, the only curative therapy, a bone marrow transplant (BMT), is seldom applied. The objective of this report is to review the most recent clinical trials involving blood and BMT for SCD and to discuss novel approaches to overcome the many barriers to successful use of BMT for SCD.

In select patients, disease-free and overall survival is greater than 80% following matched sibling BMT for SCD. Unfortunately, most patients with SCD do not have a suitable human lymphocyte antigen-matched sibling donor. In an attempt to expand the donor pool, several groups are beginning to explore the use of alternative sources of stem cells such as haploidentical donors and umbilical cord cell blood.

The curative potential of BMT in SCD is irrefutable, with outstanding results in children following a myeloablative conditioning regimen and a matched sibling donor transplant. Well tolerated and effective application of alternative sources of stem cells for BMT in SCD could greatly increase the cure rate for this devastating disease.