What is the efficacy of stem cell transplantation (SCT) for the treatment of sickle cell disease (SCD)?

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. Most transplants are performed in younger patients, and results are better in that population: an international survey of human leukocyte antigen (HLA)–identical sibling HSCT found that median age at transplantation was 9 years, and the 5-year overall survival rate was 95% for children under age 16 and 81% for those age 16 and older; the 5-year graft-vs-host disease (GVHD)–free survival rate in those age groups was 86% and 77%, respectively. [64]


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