Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies: report of the European experience 1968-99.
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.
Antoine
Corinne
C
Service de Biostatistique et Service d'Immunologie et d'Hématologie Pédiatrique, Hôpital Necker Enfants Malades, Paris, France.
Müller
Susanna
S
Cant
Andrew
A
Cavazzana-Calvo
Marina
M
Veys
Paul
P
Vossen
Jaak
J
Fasth
Anders
A
Heilmann
Carsten
C
Wulffraat
Nicolas
N
Seger
Reinhard
R
Blanche
Stéphane
S
Friedrich
Wilhelm
W
Abinun
Mario
M
Davies
Graham
G
Bredius
Robert
R
Schulz
Ansgar
A
Landais
Paul
P
Fischer
Alain
A
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