LAUSR

BACKGROUND Enrichment strategies from clinical trials for progressive systemic sclerosis-associated interstitial lung disease (SSc-ILD) have not been tested in a real-life cohort. RESEARCH QUESTION Do enrichment strategies for progressive ILD impact efficacy, representativeness and feasibility in SSc-ILD patients from the European Scleroderma Trials and Research (EUSTAR) database? STUDY DESIGN AND METHODS We applied the inclusion criteria of major recent SSc-ILD trials (focuSSced, SLS II and SENSCIS) and assessed progressive ILD, defined as absolute change in forced vital capacity (FVC) and as significant progression (FVC decline ≥10%). Data were compared to all patients and patients not fulfilling any inclusion criteria. RESULTS In total, 2258 SSc-ILD patients were included, with 31.2% meeting SENSCIS, 5.8% SLS II, 1.6% focuSSced and 1529 (67.7%) not meeting any criteria. In the first 12+/-3 months, the absolute FVC decline in all and in patients fulfilling criteria from SENSCIS was -0.1%, from focuSSced -3.7%, and from SLS II 2.3%, with accompanying more progressors in focuSSced. The patient populations fulfilling the different study inclusion criteria significantly differed in various clinical parameters. In the second 12 months period, SENSCIS enriched patients had a further absolute FVC% decline as described for the total cohort. In contrast, patients fulfilling the focuSSced and SLS II criteria showed numerical improvement of lung function. There were no significant associations of enrichment criteria and ILD progression. INTERPRETATION The application of enrichment criteria from previous clinical trials showed enrichment for progression with variable success, leading to selected patient populations reducing feasibility of recruitment. These findings are important for future clinical trial design and interpretation of the results of published trials. CLINICAL TRIAL REGISTRATION None.