Blood Reviews
Volume 17, Issue 3 , Pages 163-166, September 2003

Hematopoietic stem cell transplantation in sickle cell disease

  • Christiane Vermylen

      Affiliations

    • Corresponding Author InformationCorrespondence to: Christiane Vermylen MD, PhD, Department of Pediatric Hematology, Cliniques Universitaires Saint-Luc, Université Catholique de Louvain, Avenue Hippocrate 10, B - 1200 Brussels, Belgium. Tel.: +32-2-764-25-50; Fax: +32-2-764-89-64

Department of Pediatric Hematology, Cliniques Universitaires Saint-Luc, Université Catholique de Louvain, Avenue Hippocrate 10, B – 1200 Brussels, Belgium

Abstract 

Since the first report of a young girl affected by sickle cell anemia, treated successfully by bone marrow transplantation (BMT) for acute myeloid leukemia, more than 200 patients have been transplanted worldwide for sickle cell anemia.

The disease-free survival (DFS) is good (80–85% in several series), eventhough many children who received allografts had already significant sickle-related complications.

The best results are obtained in young children who have HLA-identical sibling donors and are transplanted early in the course of the disease (DFS: 93%).

Future directions in the field of stem cell transplantation of sickle cell anemia include (1) the establishment of new protocols with less toxicity, but still effective, (2) adapted conditioning regimen for adult patients, and (3) new sources of stem cells for broader application: umbilical cord blood and volunteer unrelated donors.

Keywords:  sickle cell anemia, stem cell transplantation

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PII: S0268-960X(03)00006-7

doi:10.1016/S0268-960X(03)00006-7

Blood Reviews
Volume 17, Issue 3 , Pages 163-166, September 2003