Bone marrow transplantation for primary immunodeficiency diseases

Advances in immunology have led to a breathtaking expansion of recognized primary immunodeficiency diseases (PID) with over 120 disease-related genes identified. In North America alone more than 1000 children have received allogeneic blood or marrow transplant over the past 30 years, with the majority surviving long term. This review presents results and highlights challenges and notable advances, including novel less toxic conditioning regimens, to transplant the more common and severe forms of PID. HLA-matched sibling donors remain the ideal option, however, advances in living donor unrelated HSCT and banked umbilical cord blood grafts provide hope for all children with severe PID.