Clinical trial NCT02516124: EBMT ADWP Prospective Non Interventional Study : AutoHSCT in SSc Patients
Official title: Autologous Stem Cell Transplantation for Progressive Systemic Sclerosis: a Prospective Non-Interventional Approach Across Europe (NISSC) for the Autoimmune Diseases Working Party of the EBMT
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Autoimmune Diseases
Keywords: Autoimmune diseases
Systemic sclerosis
Autologous Hematopoietic stem cell transplant
Lead sponsor: European Society for Blood and Marrow Transplantation
Sponsor class: NETWORK
First posted: 2015-08-05
Enrollment: 82
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients treated with autologous HSCT for severe systemic sclerosis

Brief summary: The purpose of this study is to assess the effectiveness of Autologous Hematopoietic Stem Cell transplantation (AHSCT) for early severe or rapidly progressive Systemic Sclerosis (SSc) as currently performed by different study protocols used across Europe in various EBMT centres through the careful recording and analysis of routinely collected clinical and biological data.

Detailed description: Different protocols are used in the different centres, it is not yet clear which approach will be the most efficient and the safest. Every centre will follow its own local protocol for AHSCT which usually refers to the recent update of the EBMT Guidelines for HSCT in autoimmune disease. Patient selection for AHSCT treatment technique with regard to the risk/benefit balance has to be carefully addressed by standard patient pretransplant evaluation, whereas treatment local regimen, follow-ups evaluation, supportive medication and prophylaxis will be recorded and analysed.

Eligibility criteria: Inclusion Criteria:

* Autologous HSCT
* Age between 18 and 65 years at time of transplant.
* Established diagnosis of progressive systemic sclerosis according to ARA-criteria

Exclusion Criteria:

* Pregnancy or inadequate contraception
* Severe concomitant disease
* Reduced lung function
* Previously damaged bone marrow
* Uncontrolled severe infection
* Severe concomitant psychiatric illness

Primary outcomes:
measure: Progression free survival
timeFrame: 2 year post transplant
Secondary outcomes:
measure: Safety assessed by Treatment related toxicity throughout the study period using WHO toxicity parameters (expressed as maximum grade toxicity per organ system, see appendix)
timeFrame: 2 year post transplant
measure: Overall Survival
timeFrame: 2 year post transplant
measure: Response to treatment
timeFrame: at 1 year post transplant
measure: Improvement in Quality of life
timeFrame: 2 year post transplant
measure: Relapse incidence
timeFrame: 2 year post transplant
measure: 100-day Treatment related mortality
timeFrame: 100 days post transplant
Other outcomes:
Locations:
city: Paris
country: France
facility: Badoglio Manuela- EBMT Paris Office
Study officials:
affiliation: EBMT ADWP
name: Dominique Farge, PhD
role: STUDY_CHAIR
References:
citation: Henes J, Oliveira MC, Labopin M, Badoglio M, Scherer HU, Del Papa N, Daikeler T, Schmalzing M, Schroers R, Martin T, Pugnet G, Simoes B, Michonneau D, Marijt EWA, Lioure B, Olivier Bay J, Snowden JA, Rovira M, Huynh A, Onida F, Kanz L, Marjanovic Z, Farge D. Autologous stem cell transplantation for progressive systemic sclerosis: a prospective non-interventional study from the European Society for Blood and Marrow Transplantation Autoimmune Disease Working Party. Haematologica. 2021 Feb 1;106(2):375-383. doi: 10.3324/haematol.2019.230128.