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PMID: 15249325 Published · ppublish English Evaluation Study Journal Article Multicenter Study Research Support, Non-U.S. Gov't

Autologous stem cell transplantation in the treatment of systemic sclerosis: report from the EBMT/EULAR Registry.

Annals of the rheumatic diseases ·Vol. 63 ·No. 8 ·2004-08-00 ·Pages 974-81

Farge D, Passweg J, van Laar JM, Marjanovic Z, Besenthal C, Finke J, Peter HH, Breedveld FC, Fibbe WE, Black C, Denton C, Koetter I, Locatelli F, Martini A, Schattenberg AV, van den Hoogen F, van de Putte L, Lanza F, Arnold R, Bacon PA, Bingham S, Ciceri F, Didier B, Diez-Martin JL, Emery P, Feremans W, Hertenstein B, Hiepe F, Luosujärvi R, Leon Lara A, Marmont A, Martinez AM, Pascual Cascon H, Bocelli-Tyndall C, Gluckman E, Gratwohl A, Tyndall A, EBMT/EULAR Registry

Abstract

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. 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). 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). 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.

MeSH Terms
Adolescent Adult Aged Child Clinical Trials, Phase I as Topic Clinical Trials, Phase II as Topic Europe/epidemiology Female Hematopoietic Stem Cell Transplantation/methods,mortality Humans Male Middle Aged Registries Scleroderma, Systemic/mortality,physiopathology,therapy Severity of Illness Index Skin/pathology Survival Analysis Treatment Outcome Ventricular Function, Left Vital Capacity
Authors & Affiliations
38 authors, click to expand affiliations / ORCID
Farge D
St Louis Hospital, Paris, France. dominique.farge-bancel@chu-stlouis.fr
Passweg J
van Laar J M
Marjanovic Z
Besenthal C
Finke J
Peter H H
Breedveld F C
Fibbe W E
Black C
Denton C
Koetter I
Locatelli F
Martini A
Schattenberg A V N
van den Hoogen F
van de Putte L
Lanza F
Arnold R
Bacon P A
Bingham S
Ciceri F
Didier B
Diez-Martin J L
Emery P
Feremans W
Hertenstein B
Hiepe F
Luosujärvi R
Leon Lara A
Marmont A
Martinez A M
Pascual Cascon H
Bocelli-Tyndall C
Gluckman E
Gratwohl A
Tyndall A
EBMT/EULAR Registry
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Article Info
Journal
Annals of the rheumatic diseases
Abbr.
Ann Rheum Dis
ISSN
0003-4967
DOI
10.1136/ard.2003.011205
Email
dominique.farge-bancel@chu-stlouis.fr
Published
2004-08-00
Pages
974-81
Language
English
Region
England
NLM ID
0372355
PMCID
PMC1755096
Subset
IM
External Links
PubMed source DOI: 10.1136/ard.2003.011205 PMC Full Text View journal details
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