What is the role of allogenic hematopoietic stem cell transplantation (HSCT) in the treatment of sickle cell anemia (SCA)?

Updated: May 12, 2021
  • Author: Joseph E Maakaron, MD; Chief Editor: Emmanuel C Besa, MD  more...
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Answer

Allogeneic hematopoietic stem cell transplantation (HSCT) can cure SCD, but it has many risks, so the risk-to-benefit ratio must be assessed carefully. In addition, while HSCT from an HLA-matched sibling donor has a high success rate, especially in young recipients, only a minority of those with SCD have an HLA-matched sibling who does not have SCD, or a fully matched unrelated donor in the national pool. [64] Expansion of the donor pool to include partial-matched unrelated donors, half-matched (ie, haploidentical) donors, and partial-matched cord blood has ameliorated this shortage, however, and use of reduced-intensity or nonmyeloablative conditioning regimens in these cases has reduced the increased risk of graft-versus-host disease and graft failure otherwise seen with non–fully matched donors. [64, 65]


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