151. [Survival analysis of hematopoietic stem cell transplantation in children with primary immunodeficiency in Spain].
- Author
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Hladun R, Badell I, González M, Martínez AM, Sánchez de Toledo J, Olivé MT, González ME, Elorza I, and Díaz de Heredia C
- Subjects
- Adolescent, Child, Child, Preschool, Female, Humans, Infant, Male, Retrospective Studies, Spain, Survival Analysis, Hematopoietic Stem Cell Transplantation, Immunologic Deficiency Syndromes mortality, Immunologic Deficiency Syndromes surgery
- Abstract
Introduction: 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., Patients and 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., (Copyright © 2014 Asociación Española de Pediatría. Published by Elsevier España, S.L.U. All rights reserved.)
- Published
- 2015
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