An observational study in Autoimmune Diseases, sponsored by European Society for Blood and Marrow Transplantation. Completed at 1 site in France. Open to participants aged 18 Years to 65 Years. Per ClinicalTrials.gov, last updated 2018-05-01.
Sponsored by European Society for Blood and Marrow Transplantation · Observational
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.
655 studies on the registry are indexed under Autoimmune Diseases; 275 are open to participants now.
This study's enrollment of 82 is below the median of 300 across 208 observational studies indexed under Autoimmune Diseases.
Browse Autoimmune Diseases studies →European Society for Blood and Marrow Transplantation is the lead sponsor of 28 studies on the registry; 1 is open to participants now.
Counted across the registry records on this site, refreshed daily.
Patients treated with autologous HSCT for severe systemic sclerosis
Exclusion Criteria:
Autologous HSCT
Procedure: Autologous HSCT
1st AHSCT
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
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
This study is completed, as verified in Jul 2017. You cannot join it, but the record below documents what was studied.
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European Society for Blood and Marrow Transplantation