Allogeneic hematopoietic stem cell transplantation in congenital disorders: A single‐center experience

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%).