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Allogeneic hematopoietic stem cell transplantation in congenital disorders: A single‐center experience
Author(s) -
Faraci Maura,
Giardino Stefano,
Bagnasco Francesca,
Morreale Giuseppe,
Terranova M. Paola,
Di Martino Daniela,
Lanino Edoardo
Publication year - 2017
Publication title -
pediatric transplantation
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.457
H-Index - 69
eISSN - 1399-3046
pISSN - 1397-3142
DOI - 10.1111/petr.12997
Subject(s) - medicine , hematopoietic stem cell transplantation , cord blood , pediatrics , stem cell , transplantation , bone marrow , haematopoiesis , bone marrow transplantation , genetics , biology
Allogeneic hematopoietic stem cell transplantation (allo‐ HSCT ) is the treatment of choice for a variety of congenital disorders. We report the experience of children affected by congenital diseases other than bone marrow failure syndromes who received allo‐ HSCT over a period of 25 years at G. Gaslini Paediatric Research Institute. HSCT s were performed in 57 children with congenital diseases (25 with congenital immunodeficiencies, 10 with severe combined immunodeficiencies, and 22 with metabolic diseases). Overall survival rate at 3 years in the whole group of patients was 76.9%, with a trend in favor of better outcome in children with metabolic diseases and in those who received cord blood cells (85.9%) vs bone marrow cells (72.4%).