EBMT ADWP Prospective Non Interventional Study : AutoHSCT in SSc Patients

Study Overview
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Brief Summary
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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.

Detailed Description
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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.

Official Title
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Autologous Stem Cell Transplantation for Progressive Systemic Sclerosis: a Prospective Non-Interventional Approach Across Europe (NISSC) for the Autoimmune Diseases Working Party of the EBMT

Conditions
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Autoimmune Diseases

Intervention / Treatment
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* Procedure: Autologous HSCT


Participation Criteria
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Eligibility Criteria
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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: Pregnancy or inadequate contraception Severe concomitant disease Reduced lung function Previously damaged bone marrow Uncontrolled severe infection Severe concomitant psychiatric illness

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 65 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| NISSC<br>Autologous HSCT | Procedure: Autologous HSCT<br>* 1st AHSCT<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Progression free survival | Progression free survival (PFS), defined as survival since Baseline (the 1st day of mobilisation) without evidence of progression of SSc. | 2 year post transplant | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | 2 year post transplant | 
| Overall Survival | Overall Survival | 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 | at 1 year post transplant | 
| Improvement in Quality of life | Assessed by SHAQ (Scleroderma Health Assessment Questionnaire) evolution | 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 | 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 | 100 days post transplant | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Autoimmune diseases, Systemic sclerosis, Autologous Hematopoietic stem cell transplant