Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe: entering a new century, do we do better?
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.
Gennery
Andrew R
AR
Institute of Cellular Medicine, Child Health, University of Newcastle upon Tyne, Newcastle Upon Tyne, United Kingdom. a.r.gennery@ncl.ac.uk
Slatter
Mary A
MA
Grandin
Laure
L
Taupin
Pierre
P
Cant
Andrew J
AJ
Veys
Paul
P
Amrolia
Persis J
PJ
Gaspar
H Bobby
HB
Davies
E Graham
EG
Friedrich
Wilhelm
W
Hoenig
Manfred
M
Notarangelo
Luigi D
LD
Mazzolari
Evelina
E
Porta
Fulvio
F
Bredius
Robbert G M
RG
Lankester
Arjen C
AC
Wulffraat
Nico M
NM
Seger
Reinhard
R
Güngör
Tayfun
T
Fasth
Anders
A
Sedlacek
Petr
P
Neven
Benedicte
B
Blanche
Stephane
S
Fischer
Alain
A
Cavazzana-Calvo
Marina
M
Landais
Paul
P
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