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

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
 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)
 Incidence of Secondary autoimmune diseases
 Incidence of secondary malignancies
 Incidence of severe cardiovascular events

: 80 patients
Follow-up duration: 3 years
Follow-up: According to local standard protocol with documentation of disease activity/survival Y3, Y4, Y5.