title: Long-term immune reconstitution and clinical outcome after stem cell transplantation for severe T-cell immunodeficiency.
abstract: BACKGROUND
  Currently, hematopoietic stem cell transplantation allows long-term survival in a high proportion of infants with congenital severe T-cell immunodeficiency. However, relatively little is known of their long-term quality of life.
  
  
  OBJECTIVE
  We sought to assess the long-term immune reconstitution and clinical status in children treated with stem cell transplantation for severe T-cell immunodeficiency.
  
  
  METHODS
  Immune function and clinical status have been analyzed in a cohort of 40 patients with severe T-cell immunodeficiency who are alive at a follow-up of at least 5 years after transplantation.
  
  
  RESULTS
  Most patients have attained normal T- and B-cell function. Weight and height were normal at last follow-up in most patients. Endocrine and severe neurologic abnormalities have been observed in 17.5% and 10% of the patients, respectively.
  
  
  CONCLUSIONS
  These data indicate that with current management strategies, stem cell transplantation can lead to long-term survival and good quality of life in the majority of patients with severe T-cell immunodeficiency.
  
  
  CONCLUSIONS
  Prompt recognition of congenital severe T-cell immunodeficiency, followed by stem cell transplantation, allows excellent perspectives of long-term survival and good quality of life for these otherwise fatal disorders.
authors.affiliation: Divisione di Oncoematologia Pediatrica, Ospedale dei Bambini, Spedali Civili.
authors.forename: Evelina
authors.initials: E
authors.lastname: Mazzolari
authors.forename: Concetta
authors.initials: C
authors.lastname: Forino
authors.forename: Sara
authors.initials: S
authors.lastname: Guerci
authors.forename: Luisa
authors.initials: L
authors.lastname: Imberti
authors.forename: Arnalda
authors.initials: A
authors.lastname: Lanfranchi
authors.forename: Fulvio
authors.initials: F
authors.lastname: Porta
authors.forename: Luigi D
authors.initials: LD
authors.lastname: Notarangelo
mesh_terms: D001327:Autoimmune Diseases / Q000209:etiology; D002657:Child Development; D002675:Child, Preschool; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects; D006801:Humans; D007223:Infant; D012677:Sensation; D016511:Severe Combined Immunodeficiency / Q000276:immunology / Q000401:mortality / Q000628:therapy*