Hematopoietic Stem Cell Transplantation in Primary Immunodeficiencies Beyond Severe Combined Immunodeficiency

Alexandra F Freeman1

  • 1Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland.

Insights

Hematopoietic stem cell transplantation (HSCT) is now used for more than severe combined immunodeficiency (SCID) in infants. Advances allow HSCT for other primary immunodeficiencies (PIDs) in older children and adults.

Area of Science:

  • Immunology
  • Pediatric Hematology
  • Stem Cell Transplantation

Background:

  • Hematopoietic stem cell transplantation (HSCT) is the established treatment for infants with severe combined immunodeficiency (SCID).
  • Improvements in HSCT conditioning and supportive care have expanded its application.
  • Expanding donor options, including haploidentical transplants, enhance HSCT accessibility for primary immunodeficiencies (PIDs).

Purpose of the Study:

  • To discuss primary immunodeficiencies (PIDs) for which HSCT is increasingly performed beyond early childhood.
  • To highlight the evolving landscape of HSCT for non-SCID PIDs.

Main Methods:

  • Review of current practices and trends in HSCT for PIDs.
  • Discussion based on a presentation at the 2018 PIDS-St. Jude conference.

Main Results:

  • HSCT is increasingly utilized for a broader range of PIDs, not limited to early childhood SCID.
  • Haploidentical donor transplants show increasing success, broadening donor availability.

Conclusions:

  • HSCT is becoming a more versatile treatment for various PIDs.
  • The indications for HSCT are expanding beyond SCID and early childhood, supported by improved techniques and donor options.

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