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- Title
Stem cell transplantation for primary immunodeficiencies.
- Authors
Porta, F; Forino, C; De Martiis, D; Soncini, E; Notarangelo, L; Tettoni, K; D'Ippolito, C; Soresina, R; Shiha, K; Berta, S; Baffelli, R; Bolda, F; Bosi, A; Schumacher, F R; Lanfranchi, A; Mazzolari, E
- Abstract
BMT is curative in almost 75% of children affected by severe primary immunodeficiencies (PIDs). Recently, the chance of cure has increased thanks to the availability of matched unrelated donors (MUDs). Nevertheless, besides the conventional indications to BMT (profound or absent T-cell function, profound or absent natural killer function, known syndromes with T-cell deficiencies), indications to BMT for PIDs affecting the quality of life or having an expectation of life that does not exceed the third-fourth decade remain unclear. Infact, if it is evident that the survival rate in an infant grafted for a PID with a MUD is expected to be more than 80%, alternative treatments such as gene therapy are now available.
- Publication
Bone marrow transplantation, 2008, Vol 41 Suppl 2, pS83
- ISSN
0268-3369
- Publication type
Journal Article
- DOI
10.1038/bmt.2008.61