SYSEMIC SCLEROSIS: NISSc-1 LTE (EBMT)– Autologous Hematopoietic Stem Cell transplantation
Greffe de cellules souches hématopoïétiques autologues
Long term evaluation of efficacy after autologous stem cell transplantation for systemic sclerosis – NISSc-1 long term extension
Short name: NISSc 1 LTE
Sponsor: EBMT/ADWP
Chief investigator: Joerg Henes; University Hospital Tuebingen, Germany
Representatives
France: Dominique Farge-Bancel
Germany: Joerg Henes, Tuebingen
Netherlands: Hans Ulrich Scherer, Leiden
Spain: Montserrat Rovira; Barcelona
Italy: Nicoletta del Papa; Milano
Brazil: Marina Carolina Oliveira Ribeirao Preto
Statistician: Myriam Labopin; Hôpital Saint Antoine, EBMT office, Paris, France
Protocol Design: Retrospective data analysis of the patients transplanted within the NISSc 1 noninterventional study
Aim of the study:To assess the long term effectiveness of Autologous Hematopoietic Stem Cell transplantation (AHSCT) for early severe or rapidly progressive Systemic Sclerosis (SSc) of patient transplanted within the non-interventional NISSc-1 trial.
Inclusion criteria
Patient transplanted for systemic sclerosis within the NISSc-1 study
Exclusion criteria
SSc-Patient being transplanted in another setting
Primary Endpoint: Progression free survival (PFS), defined as survival since aHSCT without evidence of progression of SSc
Secondary Endpoints
Efficacy:
Overall Survival (OS)
Progression free survival
Relapse incident: number of patients experiencing a relapse after initial response to treatment
Long term follow up of mRSS
Long term follow up of lung function test (FVC and DLCO SB)
Safety
Incidence of Secondary autoimmune diseases
Incidence of secondary malignancies
Incidence of severe cardiovascular events
Recruitment: 80 patients
Follow-up duration: 3 years
Follow-up: According to local standard protocol with documentation of disease activity/survival Y3, Y4, Y5.
