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.
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.
Study Type
OBSERVATIONAL
Enrollment
82
1st AHSCT
Badoglio Manuela- EBMT Paris Office
Paris, France
Progression free survival
Progression free survival (PFS), defined as survival since Baseline (the 1st day of mobilisation) without evidence of progression of SSc.
Time frame: 2 year post transplant
Safety assessed by Treatment related toxicity throughout the study period using WHO toxicity parameters (expressed as maximum grade toxicity per organ system, see appendix)
Incidence of Adverse Events (AE) and Serious Adverse Events (SE) Neutrophil and platelet engraftment, defined as first day after transplantation with absolute neutrophil count \> 500 cells/μL and \>20.000 platelets/μL without platelet transfusion, respectively
Time frame: 2 year post transplant
Overall Survival
Overall Survival
Time frame: 2 year post transplant
Response to treatment
Response to treatment within 1 year following autologous HSCT, defined as * 25% improvement in mRSS (modified Rodnan Skin Score) and/or * ≥10% improvement in Diffuse Capacity for carbon monoxide (DLCO) or Forced Vital Capacity (FVC) as compared to baseline without need of further immunosuppression
Time frame: at 1 year post transplant
Improvement in Quality of life
Assessed by SHAQ (Scleroderma Health Assessment Questionnaire) evolution
Time frame: 2 year post transplant
Relapse incidence
Defined as any of the following changes after prior response to treatment on quarterly follow up as defined below: * Worsening of mRSS \> 25% * New/Worsening of organ manifestation: lungs, heart or kidney
Time frame: 2 year post transplant
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
100-day Treatment related mortality
any death during 100 day following transplant that cannot be attributed to progression or relapse of the disease
Time frame: 100 days post transplant