title: European experience of bone-marrow transplantation for severe combined immunodeficiency.
abstract: The outcome of bone-marrow transplantations (BMT) carried out between 1968 and March 1, 1989, in 183 patients with severe combined immunodeficiency (SCID) was analysed. Recipients of HLA-identical BMTs (70) had a 76% probability of survival (median follow-up 73 months). Of the 32 treated since 1983, 97% have been cured (median follow-up 41 months). This good prognosis was associated with rapid development of T and B cell function. HLA-non-identical, T-cell-depleted, BMT (n = 100) gave significantly lower survival (52%; median follow-up 47 months). Factors associated with poor prognosis were the presence of a lung infection before BMT, absence of a protected environment, and use of female donors for male recipients. Use of a conditioning regimen significantly increased the frequency of sustained engraftment (86% vs 50% for non-conditioned BMT) and resulted in more frequent engraftment of donor B lymphocytes and myeloid cells. Donor B-cell chimerism was strongly associated with the development of normal B-cell function.
authors.affiliation: Immunology and Haematology Unit, Hôpital Necker-Enfants Malades, Paris, France.
authors.forename: A
authors.initials: A
authors.lastname: Fischer
authors.forename: P
authors.initials: P
authors.lastname: Landais
authors.forename: W
authors.initials: W
authors.lastname: Friedrich
authors.forename: G
authors.initials: G
authors.lastname: Morgan
authors.forename: B
authors.initials: B
authors.lastname: Gerritsen
authors.forename: A
authors.initials: A
authors.lastname: Fasth
authors.forename: F
authors.initials: F
authors.lastname: Porta
authors.forename: C
authors.initials: C
authors.lastname: Griscelli
authors.forename: S F
authors.initials: SF
authors.lastname: Goldman
authors.forename: R
authors.initials: R
authors.lastname: Levinsky
mesh_terms: D000206:Actuarial Analysis; D001402:B-Lymphocytes / Q000276:immunology; D016026:Bone Marrow Transplantation / Q000276:immunology / Q000401:mortality*; D005060:Europe; D005260:Female; D005500:Follow-Up Studies; D006085:Graft Survival / Q000187:drug effects; D006086:Graft vs Host Disease / Q000209:etiology / Q000276:immunology; D006648:Histocompatibility / Q000276:immunology; D006801:Humans; D007153:Immunologic Deficiency Syndromes / Q000145:classification / Q000276:immunology / Q000401:mortality / Q000601:surgery*; D007223:Infant; D007621:Karyotyping; D008297:Male; D011292:Premedication; D011379:Prognosis; D012189:Retrospective Studies; D012737:Sex Factors; D013601:T-Lymphocytes / Q000276:immunology; D013997:Time Factors