EBMT ADWP Prospective Non Interventional Study : AutoHSCT in SSc Patients (NISSC)

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

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.

Study Overview

• Status: Completed
• Conditions: Autoimmune Diseases
• Intervention / Treatment: Procedure: Autologous HSCT

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 Type: Observational
• Enrollment (Actual): 82

Contacts and Locations

Study Locations

• France
  • Paris, France, 75010
    • Badoglio Manuela- EBMT Paris Office

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 65 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Patients treated with autologous HSCT for severe systemic sclerosis

Description

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 Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
NISSC; Autologous HSCT	Procedure: Autologous HSCT; 1st AHSCT

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: European Society for Blood and Marrow Transplantation
• Investigators: Study Chair: Dominique Farge, PhD, EBMT ADWP

Publications and helpful links

General Publications

• Henes J, Oliveira MC, Labopin M, Badoglio M, Scherer HU, Del Papa N, Daikeler T, Schmalzing M, Schroers R, Martin T, Pugnet G, Simoes B, Michonneau D, Marijt EWA, Lioure B, Olivier Bay J, Snowden JA, Rovira M, Huynh A, Onida F, Kanz L, Marjanovic Z, Farge D. Autologous stem cell transplantation for progressive systemic sclerosis: a prospective non-interventional study from the European Society for Blood and Marrow Transplantation Autoimmune Disease Working Party. Haematologica. 2021 Feb 1;106(2):375-383. doi: 10.3324/haematol.2019.230128.

Helpful Links

• Organisation website

Study record dates

Study Major Dates

• Study Start: December 1, 2012
• Primary Completion (Actual): January 1, 2018
• Study Completion (Actual): March 1, 2018

Study Registration Dates

• First Submitted: August 3, 2015
• First Submitted That Met QC Criteria: August 4, 2015
• First Posted (Estimate): August 5, 2015

Study Record Updates

• Last Update Posted (Actual): May 1, 2018
• Last Update Submitted That Met QC Criteria: April 30, 2018
• Last Verified: July 1, 2017

More Information

Terms related to this study

• Keywords: Systemic sclerosis; Autoimmune diseases; Autologous Hematopoietic stem cell transplant
• Additional Relevant MeSH Terms: Immune System Diseases; Autoimmune Diseases
• Other Study ID Numbers: NCT02516124