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Allogenic hematopoietic stem cell transplantation in sickle cell disease

Dana K Furstenau1, John F Tisdale2

  • 1Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, 9000 Rockville Pike, Building 10, 9N112, Bethesda, MD 20892, United States; Department of Pediatric Oncology, Johns Hopkins University School of Medicine, 1800 Orleans Street, Room 11379, Baltimore, MD, 21287, United States.

Transfusion and Apheresis Science : Official Journal of the World Apheresis Association : Official Journal of the European Society for Haemapheresis
|January 24, 2021
PubMed

Insights

Hematopoietic stem cell transplant (HSCT) is the only cure for sickle cell disease (SCD). This review examines various allogeneic HSCT approaches and their outcomes for SCD patients.

Area of Science:

  • Hematology
  • Genetics
  • Immunology

Background:

  • Sickle cell disease (SCD) is a prevalent monogenic disorder affecting 100,000+ individuals in the US.
  • SCD leads to severe complications like anemia, pain, stroke, and organ failure, often resulting in mortality.
  • Current treatments offer limited disease modification, with allogeneic hematopoietic stem cell transplant (HSCT) as the sole curative option.

Keywords:
Allogeneic transplantationAlternative donorHaploidentical transplantationHematopoietic stem cell transplantationMatched siblingSickle cell disease

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