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Area of Science:

  • Hematology
  • Transplantation Immunology
  • Genetic Medicine

Background:

  • Sickle cell disease (SCD) is a severe inherited blood disorder causing significant morbidity and mortality.
  • Hematopoietic stem cell transplantation (HSCT) is the only current cure for SCD.
  • Limited availability of human leukocyte antigen (HLA)-identical sibling donors restricts HSCT access.

Purpose of the Study:

  • To review current literature on HSCT for SCD.
  • To explore alternative donor sources and emerging gene therapies.
  • To assess the potential of these approaches to expand treatment options.

Main Methods:

  • Literature review of PubMed and Google Scholar.
  • Search terms included SCD, HSCT, bone marrow transplantation, donor sources, gene therapy, and HLA matching.
  • Analysis of retrieved articles for information on indications, donors, and gene therapy.

Main Results:

  • HSCT can establish normal blood cell production, improve symptoms, and stabilize organ damage in SCD patients.
  • High survival rates are achieved with HLA-identical sibling donors, primarily in children.
  • HLA-matched unrelated and haploidentical donors, along with gene therapy, show promise for broader HSCT application.

Conclusions:

  • Alternative donor sources and gene therapy can expand HSCT eligibility for sickle cell disease.
  • Further comparative clinical trials are needed to evaluate long-term benefits and late effects of curative therapies.