Autologous stem cell transplantation in the treatment of systemic sclerosis: report from the EBMT/EULAR Registry.
OBJECTIVE
To analyse the durability of the responses after haematopoietic stem cell transplantation (HSCT) for severe systemic sclerosis (SSc) and determine whether the high transplant related mortality (TRM) improved with experience. This EBMT/EULAR report describes the longer outcome of patients originally described in addition to newly recruited cases.


METHODS
Only patients with SSc, treated by HSCT in European phase I-II studies from 1996 up to 2002, with more than 6 months of follow up were included. Transplant regimens were according to the international consensus statements. Repeated evaluations analysed complete, partial, or non-response and the probability of disease progression and survival after HSCT (Kaplan-Meier).


RESULTS
Given as median (range). Among 57 patients aged 40 (9.1-68.7) years the skin scores improved at 6 (n = 37 patients), 12 (n = 30), 24 (n = 19), and 36 (n = 10) months after HSCT (p<0.005). After 22.9 (4.5-81.1) months, partial (n = 32) or complete response (n = 14) was seen in 92% and non-response in 8% (n = 4) of 50 observed cases. 35% of the patients with initial partial (n = 13/32) or complete response (n = 3/14) relapsed within 10 (2.2-48.7) months after HSCT. The TRM was 8.7% (n = 5/57). Deaths related to progression accounted for 14% (n = 8/57) of the 23% (n = 13/57) total mortality rate. At 5 years, progression probability was 48% (95% CI 28 to 68) and the projected survival was 72% (95% CI 59 to 75).


CONCLUSIONS
This EBMT/EULAR report showed that response in two thirds of the patients after HSCT was durable with an acceptable TRM. Based on these results prospective, randomised trials are proceeding.
Farge
D
D
St Louis Hospital, Paris, France. dominique.farge-bancel@chu-stlouis.fr
Passweg
J
J
van Laar
J M
JM
Marjanovic
Z
Z
Besenthal
C
C
Finke
J
J
Peter
H H
HH
Breedveld
F C
FC
Fibbe
W E
WE
Black
C
C
Denton
C
C
Koetter
I
I
Locatelli
F
F
Martini
A
A
Schattenberg
A V N
AV
van den Hoogen
F
F
van de Putte
L
L
Lanza
F
F
Arnold
R
R
Bacon
P A
PA
Bingham
S
S
Ciceri
F
F
Didier
B
B
Diez-Martin
J L
JL
Emery
P
P
Feremans
W
W
Hertenstein
B
B
Hiepe
F
F
Luosujärvi
R
R
Leon Lara
A
A
Marmont
A
A
Martinez
A M
AM
Pascual Cascon
H
H
Bocelli-Tyndall
C
C
Gluckman
E
E
Gratwohl
A
A
Tyndall
A
A
D000293:Adolescent; D000328:Adult; D000368:Aged; D002648:Child; D017321:Clinical Trials, Phase I as Topic; D017322:Clinical Trials, Phase II as Topic; D005060:Europe / Q000453:epidemiology; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods* / Q000401:mortality; D006801:Humans; D008297:Male; D008875:Middle Aged; D012042:Registries; D012595:Scleroderma, Systemic / Q000401:mortality / Q000503:physiopathology / Q000628:therapy*; D012720:Severity of Illness Index; D012867:Skin / Q000473:pathology; D016019:Survival Analysis; D016896:Treatment Outcome; D016277:Ventricular Function, Left; D014797:Vital Capacity