Efficacy and safety of Tocilizumab in systemic sclerosis patients: the EUSTAR Database analysis

• Conditions: M34; Systemic sclerosis

• Registration Number: DRKS00015537
• Lead Sponsor: F.Hoffmann- La Roche Ltd

Brief Summary

No significant effectiveness of tocilizumab was shown in this broader, multicentre, propensity score matched, controlled observational, heterogeneous, non-enriched, real-life SSc population from the large European Scleroderma Trial and Research registry. - The consistency of direction in all predefined primary and secondary endpoints generates hypothesis for potential effectiveness in a broader SSc population rather than in highly selective RCT.

Detailed Description

Not available

Study Timeline

• Study Completion: 2018-09-01
• Study Start: 2018-10-17
• Last Update Posted: 19 August 2024

Recruitment & Eligibility

• Status: Complete
• Sex: All
• Target Recruitment: 98

Inclusion Criteria

Age greater or equal 17 years
- Observations of patients entering the database after 1.1.2010
- Classification criteria for SSc fulfilled (ACR 1980 criteria or 2013 ACR/EULAR criteria)
- Potential control patients with disease duration less or equal 35 years

Exclusion Criteria

Not signed or withdrawn informed consent for EUSTAR database

Study & Design

• Study Type: observational
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Primary endpoints are mRSS (modified Rodnan Skin Score) and FVC, both at 12 months follow-up time. The time window for the 12 months assessment will be a 9-15 months<br>window. The two different endpoints will be addressed separately, but a single matched set of patient case-control pairs will be used for analysis.

Secondary Outcome Measures

Name	Time	Method
The percentage of progressive patients for skin under therapy, defined as an increase in (mRSS of 5 points AND of 25% compared to baseline). The percentage of progressive patients for lung under therapy, defined as a decrease in either (FVC > 10%) or (FVC >5% AND DLCO > 15%).