Bone Marrow Transplantation for Severe Combined Immune Deficiency

Abstract
Severe combined immune deficiency (SCID) is a heterogeneous group of inherited diseases with an estimated frequency of 1 in 50 000 to 100 000 live births.1 It is characterized by significant impaired immunity leading to death in infancy unless treated by hematopoietic stem cell transplantation. The optimal treatment for most patients with SCID is bone marrow transplantation (BMT) from a related, HLA-identical donor (RID).2 Unfortunately, such donors are found for only a minority of patients with SCID.3,4