Bone marrow transplantation for severe combined immune deficiency

E Grunebaum, E Mazzolari, F Porta, D Dallera… - Jama, 2006 - jamanetwork.com
ContextBone marrow transplantation (BMT) using stem cells obtained from a family-related,
HLA-identical donor (RID) is the optimal treatment for patients with severe combined
immune deficiency (SCID). In the absence of an RID, HLA-mismatched related donors
(MMRDs) are often used. However, compared with RIDs, use of MMRDs for BMT is
associated with reduced survival and inferior long-term immune reconstitution. Use of HLA-
matched unrelated donors (MUDs) represents another potential alternative for BMT …