title: B-cell function in severe combined immunodeficiency after stem cell or gene therapy: a review.
abstract: Although bone marrow transplantation has resulted in life-saving T-cell reconstitution in infants with severe combined immunodeficiency (SCID), correction of B-cell function has been more problematic. This review examines B-cell reconstitution results presented in 19 reports from the United States and Europe on posttransplantation immune reconstitution in patients with SCID over the past 2 decades. The analysis considered whether pretransplantation conditioning regimens were used, the overall survival rate, the percentage with donor B-cell chimerism, the percentage with B-cell function, and the percentage of survivors requiring immunoglobulin replacement. The survival rates were higher at those centers that did not use pretransplantation conditioning or posttransplantation graft-versus-host disease prophylaxis. The percentage of survivors with B-cell chimerism, function, or both was higher and the percentage requiring immunoglobulin replacement was lower at those centers that used pretransplantation conditioning. However, there were substantial numbers of patients requiring immunoglobulin replacement at all centers. Thus pretransplantation conditioning does not guarantee that B-cell function will develop. Because most infants with SCID either present with serious infections or are given diagnoses as newborns, one must decide whether there is justification for using agents that compromise innate immunity and have intrinsic toxicities to gain B-cell immune reconstitution.
authors.affiliation: Departments of Pediatrics and Immunology, Duke University Medical Center, Durham, NC, USA. buckL003@mc.duke.edu
authors.forename: Rebecca H
authors.initials: RH
authors.lastname: Buckley
mesh_terms: D001402:B-Lymphocytes / Q000166:cytology / Q000276:immunology*; D002986:Clinical Trials as Topic; D005060:Europe; D005260:Female; D015316:Genetic Therapy / Q000706:statistics & numerical data; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D015337:Multicenter Studies as Topic; D016511:Severe Combined Immunodeficiency / Q000276:immunology* / Q000401:mortality / Q000628:therapy*; D033581:Stem Cell Transplantation / Q000706:statistics & numerical data; D015996:Survival Rate; D016896:Treatment Outcome; D014481:United States