SCLÉRODERMIE SYSTÉMIQUE: NISSc-1 LTE (EBMT) – Greffe de cellules souches hématopoïétiques autologues

Greffe de cellules souches hématopoïétiques autologues

Protocol Title: Long term evaluation of efficacy after autologous stem cell transplantation for systemic sclerosis – NISSc 1 LTE
Short name: NISSc 1 LTE
Sponsor: EBMT/ADWP
Chief investigator: Joerg Henes Internal Medicine II (Haematology, oncology, immunology and rheumatology) , Germany

Representatives
FFrance: 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:Prospective, open, non-interventional long term extension evaluation of our NISSc 1 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
Autologous HSCT
Autologous HSCT for progressive SSc within the NISSc-1 trial

Exclusion criteria
AHSCT outside the NISSC 1 trial

Primary Endpoint: Progression free survival (PFS), defined as survival since Baseline without evidence of progression of
SSc.
Secondary Endpoints
1. Overall Survival
2. Relapse incidence (RI)
3. Long term follow up of mRSS
4. Long term follow up of lung function test (FVC and DLCO SB)
5. Occurrence of secondary auto-Immune diseases, secondary malignancies, Major adverse cardiovascular events (MACE)

Recruitment: 80 patients
Follow-up duration: 5 years after stem cell transplantation