title: Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies: report of the European experience 1968-99.
abstract: BACKGROUND
  Transplantation of allogeneic haemopoietic stem cells can cure several primary immunodeficiencies. This European report focuses on the long-term results of such procedures done between 1968 and December, 1999, for primary immunodeficiencies.
  
  
  METHODS
  The report includes data from 37 centres in 18 countries, which participated in a European registry for stem-cell transplantation in severe combined immuno deficiencies (SCID) and in other immunodeficiency disorders (non-SCID). 1082 transplants in 919 patients were studied (566 in 475 SCID patients, 512 in 444 non-SCID patients; four procedures excluded owing to insufficient data). Minimum follow-up of 6 months was required.
  
  
  RESULTS
  In SCID, 3-year survival with sustained engraftment was significantly better after HLA-identical than after mismatched transplantation (77% vs 54%; p=0.002) and survival improved over time. In HLA-mismatched stem-cell transplantation, B(-) SCID had poorer prognosis than B(+) SCID. However, improvement with time occurred in both SCID phenotypes. In non-SCID, 3-year survival after genotypically HLA-matched, phenotypically HLA-matched, HLA-mismatched related, and unrelated-donor transplantation was 71%, 42%, 42%, and 59%, respectively (p=0.0006). Acute graft versus host disease predicted poor prognosis whatever the donor origin except in related HLA-identical transplantation in SCID.
  
  
  CONCLUSIONS
  The improvement in survival over time indicates more effective prevention and treatment of disease-related and procedure-related complications--eg, infections and graft versus host disease. An important factor is better prevention of graft versus host disease in the HLA-non-identical setting by use of more efficient methods of T-cell depletion. For non-SCID, stem-cell transplantation can provide a cure, and grafts from unrelated donors are almost as beneficial as those from genetically HLA-identical relatives.
authors.affiliation: Service de Biostatistique et Service d'Immunologie et d'Hématologie Pédiatrique, Hôpital Necker Enfants Malades, Paris, France.
authors.forename: Corinne
authors.initials: C
authors.lastname: Antoine
authors.forename: Susanna
authors.initials: S
authors.lastname: Müller
authors.forename: Andrew
authors.initials: A
authors.lastname: Cant
authors.forename: Marina
authors.initials: M
authors.lastname: Cavazzana-Calvo
authors.forename: Paul
authors.initials: P
authors.lastname: Veys
authors.forename: Jaak
authors.initials: J
authors.lastname: Vossen
authors.forename: Anders
authors.initials: A
authors.lastname: Fasth
authors.forename: Carsten
authors.initials: C
authors.lastname: Heilmann
authors.forename: Nicolas
authors.initials: N
authors.lastname: Wulffraat
authors.forename: Reinhard
authors.initials: R
authors.lastname: Seger
authors.forename: Stéphane
authors.initials: S
authors.lastname: Blanche
authors.forename: Wilhelm
authors.initials: W
authors.lastname: Friedrich
authors.forename: Mario
authors.initials: M
authors.lastname: Abinun
authors.forename: Graham
authors.initials: G
authors.lastname: Davies
authors.forename: Robert
authors.initials: R
authors.lastname: Bredius
authors.forename: Ansgar
authors.initials: A
authors.lastname: Schulz
authors.forename: Paul
authors.initials: P
authors.lastname: Landais
authors.forename: Alain
authors.initials: A
authors.lastname: Fischer
mesh_terms: D002675:Child, Preschool; D005060:Europe; D018380:Hematopoietic Stem Cell Transplantation; D006648:Histocompatibility / Q000235:genetics; D006801:Humans; D007223:Infant; D012042:Registries; D016511:Severe Combined Immunodeficiency / Q000235:genetics / Q000401:mortality / Q000628:therapy*; D015996:Survival Rate; D013997:Time Factors