Immunological aspects of immunodeficiency diseases part 58

Immune 3




Untreated SCID is invariably fatal in early infancy. Once diagnosis of SCID is confirmed, irrespective of the molecular diagnosis, HSCT from a human leucocyte antigen (HLA) identical or haplo-identical family donor is treatment of choice. Treatment of SCID with HSCT before 3.5 months of age results in good immune reconstitution and 95% survive long term. Delay in treatment, or the occurence of infection, impairs outcome. Infection and GVHD are the main complications following HSCT. North American and European data indicate that long-term survival after transplants from HLA-matched unrelated donors was close to 60%. Review of European data between 1968 and 1999 indicates the progressive improvement of outcome, which is mainly because of better prevention of GVHD and the treatment of infection.  Analysis of the outcome of European and U.S.  bone marrow transplant programs for the treatment of SCID is ongoing and will be regularly  reported.

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