title: Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe: entering a new century, do we do better?
abstract: BACKGROUND
  Hematopoietic stem cell transplantation remains the only treatment for most patients with severe combined immunodeficiencies (SCIDs) or other primary immunodeficiencies (non-SCID PIDs).
  
  
  OBJECTIVE
  To analyze the long-term outcome of patients with SCID and non-SCID PID from European centers treated between 1968 and 2005.
  
  
  METHODS
  The product-limit method estimated cumulative survival; the log-rank test compared survival between groups. A Cox proportional-hazard model evaluated the impact of independent predictors on patient survival.
  
  
  RESULTS
  In patients with SCID, survival with genoidentical donors (n = 25) from 2000 to 2005 was 90%. Survival using a mismatched relative (n = 96) has improved (66%), similar to that using an unrelated donor (n = 46; 69%; P = .005). Transplantation after year 1995, a younger age, B(+) phenotype, genoidentical and phenoidentical donors, absence of respiratory impairment, or viral infection before transplantation were associated with better prognosis on multivariate analysis. For non-SCID PID, in contrast with patients with SCID, we confirm that, in the 2000 to 2005 period, using an unrelated donor (n = 124) gave a 3-year survival rate similar to a genoidentical donor (n = 73), 79% for both. Survival was 76% in phenoidentical transplants (n = 23) and worse in mismatched related donor transplants (n = 47; 46%; P = .016).
  
  
  CONCLUSIONS
  This is the largest cohort study of such patients with the longest follow-up. Specific issues arise for different patient groups. Patients with B-SCID have worse survival than other patients with SCID, despite improvements in each group. For non-SCID PID, survival is worse than SCID, although more conditions are now treated. Individual disease categories now need to be analyzed so that disease-specific prognosis may be better understood and the best treatments planned.
authors.affiliation: Institute of Cellular Medicine, Child Health, University of Newcastle upon Tyne, Newcastle Upon Tyne, United Kingdom. a.r.gennery@ncl.ac.uk
authors.forename: Andrew R
authors.initials: AR
authors.lastname: Gennery
authors.forename: Mary A
authors.initials: MA
authors.lastname: Slatter
authors.forename: Laure
authors.initials: L
authors.lastname: Grandin
authors.forename: Pierre
authors.initials: P
authors.lastname: Taupin
authors.forename: Andrew J
authors.initials: AJ
authors.lastname: Cant
authors.forename: Paul
authors.initials: P
authors.lastname: Veys
authors.forename: Persis J
authors.initials: PJ
authors.lastname: Amrolia
authors.forename: H Bobby
authors.initials: HB
authors.lastname: Gaspar
authors.forename: E Graham
authors.initials: EG
authors.lastname: Davies
authors.forename: Wilhelm
authors.initials: W
authors.lastname: Friedrich
authors.forename: Manfred
authors.initials: M
authors.lastname: Hoenig
authors.forename: Luigi D
authors.initials: LD
authors.lastname: Notarangelo
authors.forename: Evelina
authors.initials: E
authors.lastname: Mazzolari
authors.forename: Fulvio
authors.initials: F
authors.lastname: Porta
authors.forename: Robbert G M
authors.initials: RG
authors.lastname: Bredius
authors.forename: Arjen C
authors.initials: AC
authors.lastname: Lankester
authors.forename: Nico M
authors.initials: NM
authors.lastname: Wulffraat
authors.forename: Reinhard
authors.initials: R
authors.lastname: Seger
authors.forename: Tayfun
authors.initials: T
authors.lastname: Güngör
authors.forename: Anders
authors.initials: A
authors.lastname: Fasth
authors.forename: Petr
authors.initials: P
authors.lastname: Sedlacek
authors.forename: Benedicte
authors.initials: B
authors.lastname: Neven
authors.forename: Stephane
authors.initials: S
authors.lastname: Blanche
authors.forename: Alain
authors.initials: A
authors.lastname: Fischer
authors.forename: Marina
authors.initials: M
authors.lastname: Cavazzana-Calvo
authors.forename: Paul
authors.initials: P
authors.lastname: Landais
mesh_terms: D002648:Child; D002675:Child, Preschool; D005060:Europe; D005500:Follow-Up Studies; D018380:Hematopoietic Stem Cell Transplantation / Q000592:standards* / Q000639:trends; D049673:History, 20th Century; D049674:History, 21st Century; D006801:Humans; D015999:Multivariate Analysis; D011379:Prognosis; D016511:Severe Combined Immunodeficiency / Q000628:therapy*; D015996:Survival Rate; D013997:Time Factors; D016896:Treatment Outcome