title: Long-term outcome after hematopoietic stem cell transplantation of a single-center cohort of 90 patients with severe combined immunodeficiency.
abstract: Allogeneic hematopoietic stem cell transplantation (HSCT) is a curative treatment for severe combined immunodeficiency (SCID). Detailed assessment of the long-term outcome of HSCT, ie, the occurrence of clinical events and the quality and stability of immune reconstitution, is now required. We performed a single-center retrospective analysis of the long-term outcome of HSCT in 90-patient cohort followed for between 2 and 34 years (median, 14 years). Clinical events and immune reconstitution data were collected. Almost half the patients have experienced one or more significant clinical events, including persistent chronic graft-versus-host disease (GVHD), autoimmune and inflammatory manifestations, opportunistic and nonopportunistic infections, chronic human papilloma virus (HPV) infections, and a requirement for nutritional support. With the notable exception of severe HPV infection, these complications tend to become less common 15 years later after HSCT. A multivariate analysis showed that the occurrence of these events correlated with non-genoidentical donors, diagnosis of Artemis SCID, and quality of immune reconstitution. In most cases, HSCT enables long-term survival with infrequent sequelae. However, the occurrence of relatively late-onset complications is a concern that requires specific means of prevention and justifies careful patient follow-up.
authors.affiliation: Unité d'Immuno-Hématologie et Rhumatologie Pédiatrique, Hôpital Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris, Paris, France.
authors.forename: Bénédicte
authors.initials: B
authors.lastname: Neven
authors.forename: Sandrine
authors.initials: S
authors.lastname: Leroy
authors.forename: Hélène
authors.initials: H
authors.lastname: Decaluwe
authors.forename: Francoise
authors.initials: F
authors.lastname: Le Deist
authors.forename: Capucine
authors.initials: C
authors.lastname: Picard
authors.forename: Despina
authors.initials: D
authors.lastname: Moshous
authors.forename: Nizar
authors.initials: N
authors.lastname: Mahlaoui
authors.forename: Marianne
authors.initials: M
authors.lastname: Debré
authors.forename: Jean-Laurent
authors.initials: JL
authors.lastname: Casanova
authors.forename: Liliane
authors.initials: L
authors.lastname: Dal Cortivo
authors.forename: Yoann
authors.initials: Y
authors.lastname: Madec
authors.forename: Salima
authors.initials: S
authors.lastname: Hacein-Bey-Abina
authors.forename: Geneviève
authors.initials: G
authors.lastname: de Saint Basile
authors.forename: Jean-Pierre
authors.initials: JP
authors.lastname: de Villartay
authors.forename: Stéphane
authors.initials: S
authors.lastname: Blanche
authors.forename: Marina
authors.initials: M
authors.lastname: Cavazzana-Calvo
authors.forename: Alain
authors.initials: A
authors.lastname: Fischer
mesh_terms: D015331:Cohort Studies; D005260:Female; D005500:Follow-Up Studies; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007223:Infant; D008297:Male; D016511:Severe Combined Immunodeficiency / Q000453:epidemiology* / Q000276:immunology / Q000523:psychology / Q000601:surgery*; D015996:Survival Rate; D013997:Time Factors; D016896:Treatment Outcome