title: Single-center analysis of long-term outcome after hematopoietic cell transplantation in children with congenital severe T cell immunodeficiency.
abstract: We review clinical outcome and immune reconstitution in a consecutive series of 74 infants with severe T cell immunodeficiency who received hematopoietic cell transplantation (HCT) from January 1991 to May 2003. Fifty-three patients (71.6%) are alive. Results were significantly better for recipients of HCT from HLA-matched related donors (100% survival) and unrelated donors (86.4%) than from mismatched related donors (51.6%). A detailed analysis of immune reconstitution and clinical status was performed in 49 surviving patients, most of which have attained robust T and B cell reconstitution and are in very good clinical conditions. No cases of late deaths or of chronic graft-versus-host disease (GvHD) have been observed. However, infections and autoimmunity at >1 year after HCT have been observed in a significant number of patients. Persistence of a low number of circulating naive T cells and long-term requirement for intravenous immunoglobulin were associated with a higher incidence of clinical events.
authors.affiliation: Divisione di Emato-Oncologia Pediatrica, Ospedale dei Bambini, Spedali Civili, Brescia, Italy.
authors.forename: Evelina
authors.initials: E
authors.lastname: Mazzolari
authors.forename: Donatella
authors.initials: D
authors.lastname: de Martiis
authors.forename: Concetta
authors.initials: C
authors.lastname: Forino
authors.forename: Arnalda
authors.initials: A
authors.lastname: Lanfranchi
authors.forename: Silvia
authors.initials: S
authors.lastname: Giliani
authors.forename: Roberto
authors.initials: R
authors.lastname: Marzollo
authors.forename: Paolo
authors.initials: P
authors.lastname: Airò
authors.forename: Luisa
authors.initials: L
authors.lastname: Imberti
authors.forename: Fulvio
authors.initials: F
authors.lastname: Porta
authors.forename: Luigi D
authors.initials: LD
authors.lastname: Notarangelo
mesh_terms: D015551:Autoimmunity / Q000276:immunology; D002648:Child; D002675:Child, Preschool; D005260:Female; D006086:Graft vs Host Disease / Q000276:immunology / Q000401:mortality / Q000628:therapy; D018380:Hematopoietic Stem Cell Transplantation; D006412:Hematopoietic Stem Cells / Q000276:immunology; D006648:Histocompatibility / Q000276:immunology; D006801:Humans; D007223:Infant; D008297:Male; D009894:Opportunistic Infections / Q000276:immunology / Q000628:therapy; D011183:Postoperative Complications / Q000276:immunology / Q000401:mortality / Q000628:therapy; D016511:Severe Combined Immunodeficiency / Q000235:genetics / Q000276:immunology / Q000401:mortality* / Q000601:surgery; D013601:T-Lymphocytes / Q000276:immunology* / Q000473:pathology; D019172:Transplantation Conditioning