[Survival analysis of hematopoietic stem cell transplantation in children with primary immunodeficiency in Spain.]

[severe combined immunodeficiency]

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 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 .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 .The best results have been obtained with identical siblings , but other options may be considered .

Diseases
Validation

Diseases presenting "lymphoproliferative disorders" symptom

liposarcoma

severe combined immunodeficiency

wiskott-aldrich syndrome

This symptom has already been validated