Hematopoietic cell transplantation for correction of primary immunodeficiencies

Bone Marrow Transplantation
Ah Filipovich

Abstract

The first hematopoietic cell transplants in humans with durable success were reported in 1968, in three patients with primary immunodeficiencies who received grafts from HLA-matched siblings (two with SCID and one with Wiskott-Aldrich syndrome). Significant progress has been made in correcting lethal primary immunodeficiencies (PIDs) with hematopoietic transplantation in the ensuing 40 years due to several factors: (1) ability to phenotype and quantitate (CD34+) hematopoietic stem cells, (2) advent of high-resolution tissue typing, (3) availability of closely matched unrelated donor bone marrow, peripheral blood stem cells, and cord blood, and (4) the application of reduced intensity conditioning regimens pre-transplant. Furthermore, the genetic basis of the majority of lethal PIDs has been defined, allowing more accurate studies of the natural history of the disorders without HCT intervention, and providing a compelling rationale for early transplantation in disorders with median survivals of 15-20 years. In the current era, we can identify several factors, which influence the ultimate success of HCT for PID. These include the age at transplant and general health of the patient. Young age is associated with fewer comorbidities...Continue Reading

References

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Feb 13, 2007·Bone Marrow Transplantation·M EapenA H Filipovich

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Citations

Mar 13, 2010·International Journal of Hematology·Tang-Her JaingYung-Kuei Soong
Mar 23, 2010·Bone Marrow Transplantation·P A RamirezC G Brunstein
Jun 15, 2011·Expert Review of Hematology·Jacob Rozmus, Kirk R Schultz
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Sep 4, 2015·Journal of Molecular Biology·Viktor Haellman, Martin Fussenegger
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Apr 18, 2014·The Journal of Immunology : Official Journal of the American Association of Immunologists·Gustavo SalgueroRenata Stripecke
Jul 2, 2019·NeoReviews·Amy E O'Connell

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