Advances in hematopoietic stem cell transplantation for primary immunodeficiency.
The molecular bases of most primary immunodeficiencies (PID) have been discovered. Long-term follow-up of patient cohorts treated with antimicrobial prophylaxis has demonstrated good short-term prognosis but with increasing morbidity and mortality over time. The results of hematopoietic stem cell transplantation (HSCT) for PID have improved incrementally over time, with survival and cure of 90% for some defined diseases. This article examines the advances in HSCT for PID and argues that HSCT should be considered earlier for most patients.
Gennery
Andrew R
AR
Institute of Cellular Medicine, University of Newcastle upon Tyne, Newcastle upon Tyne, NE1 7RU, UK; Children's Bone Marrow Transplant Unit, Ward 23, Newcastle General Hospital, Westgate Road, Newcastle upon Tyne, NE4 6BE, UK.
Cant
Andrew J
AJ
D023201:CD40 Ligand / Q000172:deficiency / Q000276:immunology; D036101:Cord Blood Stem Cell Transplantation; D006085:Graft Survival; D006086:Graft vs Host Disease / Q000276:immunology* / Q000628:therapy; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects; D006648:Histocompatibility / Q000276:immunology*; D006801:Humans; D007153:Immunologic Deficiency Syndromes / Q000150:complications / Q000276:immunology / Q000628:therapy*; D016511:Severe Combined Immunodeficiency / Q000276:immunology / Q000628:therapy; D019172:Transplantation Conditioning; D016896:Treatment Outcome; D014923:Wiskott-Aldrich Syndrome / Q000276:immunology / Q000628:therapy
D023201:CD40 Ligand