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Updated: Jan 31, 2026

Isolation and Transplantation of Hematopoietic Stem Cells HSCs
Published on: February 25, 2007
Hematopoietic Stem Cell Transplantation in Primary Immunodeficiencies Beyond Severe Combined Immunodeficiency
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.
Abstract:
Hematopoietic stem cell transplantation (HSCT) has been the standard of care for infants with severe combined immunodeficiency (SCID) for several decades due to the dismal prognosis early in life without immune reconstitution. In recent years, as HSCT conditioning regimens and supportive care have greatly improved, HSCT is gaining in acceptance for more non-SCID primary immunodeficiencies (PIDs) and outside the early childhood period. In addition, potential donor options for non-SCID PIDs are expanding with increasing success for haploidentical donor transplants. In this brief report of a presentation at the PIDS-St. Jude 2018 conference, PIDs for which transplants are increasingly performed outside of early childhood will be discussed.
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