Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2009 Mar;21(2):158-61.
doi: 10.1097/CCO.0b013e328324ba04.

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

Javier Bolaños-Meade  1 , Robert A Brodsky
Affiliations
Review

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

Javier Bolaños-Meade et al. Curr Opin Oncol. 2009 Mar.

Abstract

Purpose of review: 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.

Recent findings: 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.

Summary: 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.

PubMed Disclaimer

References

    1. Walters MC, Patience M, Leisenring W, et al. Bone marrow transplantation for sickle cell disease. N Engl J Med. 1996;335(6):369–376. - PubMed
    1. Walters MC, Storb R, Patience M, et al. Impact of bone marrow transplantation for symptomatic sickle cell disease: an interim report. Multicenter investigation of bone marrow transplantation for sickle cell disease. Blood. 2000;95(6):1918–1924. - PubMed
    1. Vermylen C, Cornu G, Ferster A, et al. Haematopoietic stem cell transplantation for sickle cell anaemia: the first 50 patients transplanted in Belgium. Bone Marrow Transplant. 1998;22(1):1–6. - PubMed
    1. Bernaudin F, Socie G, Kuentz M, et al. Long-term results of related myeloablative stem-cell transplantation to cure sickle cell disease. Blood. 2007;110(7):2749–2756. - PubMed

    2. **Results of a very large prospective trial on the use of high dose chemotherapy and bone marrow transplant in patients with sickle cell disease.

    1. Chakrabarti S, Bareford D. A survey on patient perception of reduced-intensity transplantation in adults with sickle cell disease. Bone Marrow Transplant. 2007;39(8):447–451. - PubMed

    2. *Brief discussion on the perception of patients with sickle cell disease about transplantation. It gives an idea of the toxicities that this group of patients may be willing to tolerate.