title: [Survival analysis of hematopoietic stem cell transplantation in children with primary immunodeficiency in Spain].
abstract: BACKGROUND
  Children with primary immunodeficiency have severe life-threatening infections and a higher prevalence of autoimmune problems, allergy and lymphoproliferative disorders. Allogenic hematopoietic stem cell transplantation has been the only potentially curative option.
  
  
  METHODS
  Patients with primary immunodeficiency underwent allogenic stem cell transplantation in the period 1985-2011, and registered in the Spanish Working Party for Bone Marrow Transplantation in Children.
  
  
  RESULTS
  One hundred and fifty nine patients underwent 173 allogenic stem cell transplantations, of whom 97 had severe combined immunodeficiency, 30 with immune dysregulation disorders, 25 Wiskott-Aldrich syndrome, and 21 phagocyte disorders. The median patient age at diagnosis was 6 months (range: 17 days - 168 months) and the median patient age at transplant was 12 months (range: 1 month - 189 months). The donors were 30 (19%) identical siblings, 40 (25%) alternative family donors, and 89 (56%) unrelated donors. The source of stem cells was bone marrow in 68 (43%), cord blood in 52 (33%), and peripheral blood in 39 (24%). Ninety eight (61.6%) are alive, 57 (35.9%) died. Event-free survival at 10 years was 63%, with 90% for children transplanted from identical siblings, 36% for those transplanted from alternative family donors, and 66% for those transplanted from unrelated donors.
  
  
  CONCLUSIONS
  The best results have been obtained with identical siblings, but other options may be considered.
authors.affiliation: Servicio de Oncología, Hematología y Trasplante de Progenitores Hematopoyéticos Pediátrica, Hospital Vall d'Hebron, Barcelona, España. Electronic address: rhladun@vhebron.net.
authors.forename: R
authors.initials: R
authors.lastname: Hladun
authors.affiliation: Servicio de Oncología y Hematología Pediátrica, Hospital Sant Pau i Santa Creu, Barcelona, España.
authors.forename: I
authors.initials: I
authors.lastname: Badell
authors.affiliation: Servicio de Oncología y Hematología Pediátrica, Hospital del Niño Jesús, Madrid, España.
authors.forename: M
authors.initials: M
authors.lastname: González
authors.affiliation: Servicio de Oncología y Hematología Pediátrica, Hospital La Paz, Madrid, España.
authors.forename: A M
authors.initials: AM
authors.lastname: Martínez
authors.affiliation: Servicio de Oncología, Hematología y Trasplante de Progenitores Hematopoyéticos Pediátrica, Hospital Vall d'Hebron, Barcelona, España.
authors.forename: J
authors.initials: J
authors.lastname: Sánchez de Toledo
authors.affiliation: Servicio de Oncología, Hematología y Trasplante de Progenitores Hematopoyéticos Pediátrica, Hospital Vall d'Hebron, Barcelona, España.
authors.forename: M T
authors.initials: MT
authors.lastname: Olivé
authors.affiliation: Servicio de Oncología y Hematología Pediátrica, Hospital Regional Universitario Carlos Haya, Málaga, España.
authors.forename: M E
authors.initials: ME
authors.lastname: González
authors.affiliation: Servicio de Oncología, Hematología y Trasplante de Progenitores Hematopoyéticos Pediátrica, Hospital Vall d'Hebron, Barcelona, España.
authors.forename: I
authors.initials: I
authors.lastname: Elorza
authors.affiliation: Servicio de Oncología, Hematología y Trasplante de Progenitores Hematopoyéticos Pediátrica, Hospital Vall d'Hebron, Barcelona, España.
authors.forename: C
authors.initials: C
authors.lastname: Díaz de Heredia
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007153:Immunologic Deficiency Syndromes / Q000401:mortality* / Q000601:surgery*; D007223:Infant; D008297:Male; D012189:Retrospective Studies; D013030:Spain; D016019:Survival Analysis
keywords: Hematopoietic stem cell transplantation; Immunosuppression; Inmunodeficiencias primarias; Inmunosupresión; Primary immunodeficiencies; Trasplante alogénico de progenitores hematopoyéticos