title: Stem cell transplantation for congenital immunodeficiencies using reduced-intensity conditioning.
abstract: The optimal preparation for stem cell transplantation (SCT) in children with congenital immunodeficiencies is currently unknown. In all, 81 children with immunodeficiency underwent 82 SCTs using reduced-intensity conditioning (RIC). The incidence of significant GVHD was low; viral reactivation was prominent with an unexpected increase in EBV reactivation; immune reconstitution was similar between different donor groups and comparable to conventional SCT. Overall, 68/81 (84%) survive with no significant difference between donor types or between severe combined immunodeficiency (SCID) and non-SCID diseases. Findings suggest a significant survival advantage in the unrelated donor setting for RIC compared to conventional SCT.
authors.affiliation: Great Ormond Street Hospital (GOSH) for Children NHS Trust, London, UK. VEYSP@gosh.nhs.uk
authors.forename: P
authors.initials: P
authors.lastname: Veys
authors.forename: K
authors.initials: K
authors.lastname: Rao
authors.forename: P
authors.initials: P
authors.lastname: Amrolia
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D005260:Female; D006086:Graft vs Host Disease / Q000401:mortality / Q000517:prevention & control; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D016511:Severe Combined Immunodeficiency / Q000151:congenital / Q000401:mortality / Q000628:therapy*; D033581:Stem Cell Transplantation / Q000401:mortality; D019172:Transplantation Conditioning / Q000379:methods* / Q000401:mortality; D014184:Transplantation, Homologous