Hematopoietic cell transplantation for correction of primary immunodeficiencies
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
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