title: Hematopoietic stem cell transplantation in Taiwanese children with primary immunodeficiency.
abstract: OBJECTIVE
  Since 1968 it has been known that hematopoietic stem cell transplantation (HSCT) can ameliorate primary immunodeficiencies, but data on the long-term efficacy of this treatment in Taiwan are limited. This study analyzed the outcome of HSCT and the immune reconstruction in 10 children with primary immunodeficiencies in Taiwan.
  
  
  METHODS
  We retrospectively analyzed the outcome of HSCT in 10 children with primary immunodeficiencies between 1986 and 2002. The primary immunodeficiencies in these children included severe combined immunodeficiency (SCID) in 4, Wiskott-Aldrich syndrome (WAS) in 4, Chediak-Higashi syndrome (CHS) in 1, and leukocyte adhesion deficiency (LAD) in 1. The conditioning protocols included busulfan and cyclophosphamide in 2 patients with SCID, 3 patients with WAS, 1 patient with LAD and 1 patient with CHS. Anti-thymocyte globulin was given to only 1 patient with WAS and no conditioning therapy was given in 2 SCID patients. Graft-versus-host disease (GVHD) prophylaxis with cyclosporine (CsA) and methotrexate was prescribed in 6 children, CsA alone in 1, and CsA and T-cell depletion in 1.
  
  
  RESULTS
  Six patients were cured and 1 improved during a follow-up period from 3.5 years to 13 years after transplantation. Three patients died of severe sepsis. Three patients developed acute GVHD, which was grade 2 in 2 patients, and grade 3 in 1. Veno-occlusive disease developed in 1 patient and chronic GVHD with contracture of joints in 1.
  
  
  CONCLUSIONS
  Our results support the benefits of allo-HSCT in children with primary immunodeficiencies. However, HSCT should be performed as soon as possible before severe infection develops.
authors.affiliation: Department of Pediatrics, College of Medicine, National Taiwan University Hospital,Taipei, Taiwan.
authors.forename: Yung-Li
authors.initials: YL
authors.lastname: Yang
authors.forename: Meng-Yao
authors.initials: MY
authors.lastname: Lu
authors.forename: Shiann-Tarng
authors.initials: ST
authors.lastname: Jou
authors.forename: Kai-Hsin
authors.initials: KH
authors.lastname: Lin
authors.forename: Dong-Tsamn
authors.initials: DT
authors.lastname: Lin
mesh_terms: D005260:Female; D006086:Graft vs Host Disease / Q000209:etiology; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects; D006801:Humans; D007153:Immunologic Deficiency Syndromes / Q000628:therapy*; D007223:Infant; D008297:Male; D012189:Retrospective Studies; D013624:Taiwan; D019172:Transplantation Conditioning / Q000379:methods