NISSC: EBMT ADWP Prospective Non Interventional Study : AutoHSCT in SSc Patients
Study Details
Study Description
Brief Summary
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Detailed Description
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 Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| NISSC Autologous HSCT |
Procedure: Autologous HSCT
1st AHSCT
|
Outcome Measures
Primary Outcome Measures
- Progression free survival [2 year post transplant]
Progression free survival (PFS), defined as survival since Baseline (the 1st day of mobilisation) without evidence of progression of SSc.
Secondary Outcome Measures
- Safety assessed by Treatment related toxicity throughout the study period using WHO toxicity parameters (expressed as maximum grade toxicity per organ system, see appendix) [2 year post transplant]
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
- Overall Survival [2 year post transplant]
Overall Survival
- Response to treatment [at 1 year post transplant]
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
- Improvement in Quality of life [2 year post transplant]
Assessed by SHAQ (Scleroderma Health Assessment Questionnaire) evolution
- Relapse incidence [2 year post transplant]
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
- 100-day Treatment related mortality [100 days post transplant]
any death during 100 day following transplant that cannot be attributed to progression or relapse of the disease
Eligibility Criteria
Criteria
Inclusion Criteria:
-
Autologous HSCT
-
Age between 18 and 65 years at time of transplant.
-
Established diagnosis of progressive systemic sclerosis according to ARA-criteria
Exclusion Criteria:
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Pregnancy or inadequate contraception
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Severe concomitant disease
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Reduced lung function
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Previously damaged bone marrow
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Uncontrolled severe infection
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Severe concomitant psychiatric illness
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Badoglio Manuela- EBMT Paris Office | Paris | France | 75010 |
Sponsors and Collaborators
- European Society for Blood and Marrow Transplantation
Investigators
- Study Chair: Dominique Farge, PhD, EBMT ADWP
Study Documents (Full-Text)
None provided.More Information
Additional Information:
Publications
None provided.- NCT02516124
- NCT02264405