EBMT ADWP Prospective Non Interventional Study : AutoHSCT in SSc Patients

Completed

• Conditions: Autoimmune Diseases
• Interventions: Procedure: Autologous HSCT

• Registration Number: NCT02516124
• Lead Sponsor: European Society for Blood and Marrow Transplantation

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.

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 Timeline

• Study Start: 2012-12
• Study First Submitted: 2015-08-03
• Study First Submitted QC: 2015-08-04
• Study First Posted: 2015-08-05
• Status Verified: 2017-07
• Primary Completion: 2018-01
• Study Completion: 2018-03
• Last Update Submitted: 2018-04-30
• Last Update Posted: 2018-05-01

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 82

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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
NISSC	Autologous HSCT	Autologous HSCT

Primary Outcome Measures

Name	Time	Method
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

Name	Time	Method
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

Trial Locations

Locations (1)

Badoglio Manuela- EBMT Paris Office; 🇫🇷 Paris, France