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

D Farge; J Passweg; J M van Laar; Z Marjanovic; C Besenthal; J Finke; H H Peter; F C Breedveld; W E Fibbe; C Black; C Denton; I Koetter; F Locatelli; A Martini; A V N Schattenberg; F van den Hoogen; L van de Putte; F Lanza; R Arnold; P A Bacon; S Bingham; F Ciceri; B Didier; J L Diez-Martin; P Emery; W Feremans; B Hertenstein; F Hiepe; R Luosujärvi; A Leon Lara; A Marmont; A M Martinez; H Pascual Cascon; C Bocelli-Tyndall; E Gluckman; A Gratwohl; A Tyndall

doi:10.1136/ard.2003.011205

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

D Farge, J Passweg, J M van Laar, Z Marjanovic, C Besenthal, J Finke, H H Peter, F C Breedveld, W E Fibbe, C Black, C Denton, I Koetter, F Locatelli, A Martini, A V N Schattenberg, F van den Hoogen, L van de Putte, F Lanza, R Arnold, P A BaconS Bingham, F Ciceri, B Didier, J L Diez-Martin, P Emery, W Feremans, B Hertenstein, F Hiepe, R Luosujärvi, A Leon Lara, A Marmont, A M Martinez, H Pascual Cascon, C Bocelli-Tyndall, E Gluckman, A Gratwohl, A Tyndall,

Research output: Contribution to journal › Article › Academic › peer-review

Abstract

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).

CONCLUSION: 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.

Original language	English
Pages (from-to)	974-81
Number of pages	8
Journal	Annals of the Rheumatic Diseases
Volume	63
Issue number	8
DOIs	https://doi.org/10.1136/ard.2003.011205
Publication status	Published - Aug 2004
Externally published	Yes

Keywords

Adolescent
Adult
Aged
Child
Clinical Trials, Phase I as Topic
Clinical Trials, Phase II as Topic
Europe
Female
Hematopoietic Stem Cell Transplantation
Humans
Male
Middle Aged
Registries
Scleroderma, Systemic
Severity of Illness Index
Skin
Survival Analysis
Treatment Outcome
Ventricular Function, Left
Vital Capacity
Evaluation Studies
Journal Article
Multicenter Study
Research Support, Non-U.S. Gov't

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10.1136/ard.2003.011205

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Farge, D., 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. (2004). Autologous stem cell transplantation in the treatment of systemic sclerosis: report from the EBMT/EULAR Registry. Annals of the Rheumatic Diseases, 63(8), 974-81. https://doi.org/10.1136/ard.2003.011205

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title = "Autologous stem cell transplantation in the treatment of systemic sclerosis: report from the EBMT/EULAR Registry",

abstract = "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).CONCLUSION: 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.",

keywords = "Adolescent, Adult, Aged, Child, Clinical Trials, Phase I as Topic, Clinical Trials, Phase II as Topic, Europe, Female, Hematopoietic Stem Cell Transplantation, Humans, Male, Middle Aged, Registries, Scleroderma, Systemic, Severity of Illness Index, Skin, Survival Analysis, Treatment Outcome, Ventricular Function, Left, Vital Capacity, Evaluation Studies, Journal Article, Multicenter Study, Research Support, Non-U.S. Gov't",

author = "D Farge and J Passweg and {van Laar}, {J M} and Z Marjanovic and C Besenthal and J Finke and Peter, {H H} and Breedveld, {F C} and Fibbe, {W E} and C Black and C Denton and I Koetter and F Locatelli and A Martini and Schattenberg, {A V N} and {van den Hoogen}, F and {van de Putte}, L and F Lanza and R Arnold and Bacon, {P A} and S Bingham and F Ciceri and B Didier and Diez-Martin, {J L} and P Emery and W Feremans and B Hertenstein and F Hiepe and R Luosuj{\"a}rvi and {Leon Lara}, A and A Marmont and Martinez, {A M} and {Pascual Cascon}, H and C Bocelli-Tyndall and E Gluckman and A Gratwohl and A Tyndall",

year = "2004",

month = aug,

doi = "10.1136/ard.2003.011205",

language = "English",

volume = "63",

pages = "974--81",

journal = "Annals of the Rheumatic Diseases",

issn = "0003-4967",

publisher = "BMJ Publishing Group",

number = "8",

}

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, AVN, 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 2004, '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, pp. 974-81. https://doi.org/10.1136/ard.2003.011205

TY - JOUR

T1 - Autologous stem cell transplantation in the treatment of systemic sclerosis

T2 - report from the EBMT/EULAR Registry

AU - Farge, D

AU - Passweg, J

AU - van Laar, J M

AU - Marjanovic, Z

AU - Besenthal, C

AU - Finke, J

AU - Peter, H H

AU - Breedveld, F C

AU - Fibbe, W E

AU - Black, C

AU - Denton, C

AU - Koetter, I

AU - Locatelli, F

AU - Martini, A

AU - Schattenberg, A V N

AU - van den Hoogen, F

AU - van de Putte, L

AU - Lanza, F

AU - Arnold, R

AU - Bacon, P A

AU - Bingham, S

AU - Ciceri, F

AU - Didier, B

AU - Diez-Martin, J L

AU - Emery, P

AU - Feremans, W

AU - Hertenstein, B

AU - Hiepe, F

AU - Luosujärvi, R

AU - Leon Lara, A

AU - Marmont, A

AU - Martinez, A M

AU - Pascual Cascon, H

AU - Bocelli-Tyndall, C

AU - Gluckman, E

AU - Gratwohl, A

AU - Tyndall, A

PY - 2004/8

Y1 - 2004/8

N2 - 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).CONCLUSION: 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.

AB - 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).CONCLUSION: 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.

KW - Adolescent

KW - Adult

KW - Aged

KW - Child

KW - Clinical Trials, Phase I as Topic

KW - Clinical Trials, Phase II as Topic

KW - Europe

KW - Female

KW - Hematopoietic Stem Cell Transplantation

KW - Humans

KW - Male

KW - Middle Aged

KW - Registries

KW - Scleroderma, Systemic

KW - Severity of Illness Index

KW - Skin

KW - Survival Analysis

KW - Treatment Outcome

KW - Ventricular Function, Left

KW - Vital Capacity

KW - Evaluation Studies

KW - Journal Article

KW - Multicenter Study

KW - Research Support, Non-U.S. Gov't

U2 - 10.1136/ard.2003.011205

DO - 10.1136/ard.2003.011205

M3 - Article

C2 - 15249325

SN - 0003-4967

VL - 63

SP - 974

EP - 981

JO - Annals of the Rheumatic Diseases

JF - Annals of the Rheumatic Diseases

IS - 8

ER -

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

Abstract

Keywords

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