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Table 1 Endpoints of the PREDMETH study listed as primary endpoint, secondary endpoints and explorative endpoints

From: Design of a randomized controlled trial to evaluate effectiveness of methotrexate versus prednisone as first-line treatment for pulmonary sarcoidosis: the PREDMETH study

Primary endpoint
 - Between-group difference in change in hospital-measured Forced Vital Capacity (FVC) % predicted between baseline and 24 weeks
Secondary endpoints
 - Difference in change in FVC % predicted at 4, 16 weeks, 1 year and 2 years between prednisone and methotrexate group
 - Time to major pulmonary improvement measured by home-measured FVC, whereby major pulmonary improvement is defined as 80% of the maximum percent predicted FVC reached anywhere during the first 24 weeks of treatment
 - The percentage of patients with ≥5 and ≥ 10% improvement or decline in FVC at 4, 16 and 24 weeks
 - The percentage of patients with ≥10% improvement or decline in DLCO at 4, 16 and 24 weeks
 - Change over time in (Health-related) quality of life measured by King’s Sarcoidosis Questionnaire (KSQ), Global Rating of Change scale (GRoC), Chronic Respiratory Questionnaire (CRQ) and Euroqol-5D-5L questionnaire (EQ-5D-5L) at every clinical visit
 - Time to symptom improvement. Symptom scores measured by Visual Analogue Scales (VAS), Medical Research Council dyspnea scale (MRC) and Fatigue Assessment Scale (FAS)
 - Expectations with medication at baseline
 - Experiences and satisfaction with medication measured by the Patient Experience and Satisfaction with Medication Questionnaire (PESaM)
 - Number, severity and impact of side-effects compared between methotrexate and prednisone
 - Number of patients who discontinue/switch medication
 - Adherence to treatment schedule (% of patients that received at least 90% of the total cumulative dose after 24 weeks).
 - Correlation between angiotensin coverting enzyme (ACE), serum soluble interleukin-2 receptor (sIL-2R) and clinical parameters
Explorative endpoints
 - Asses predictors of disease progression and response to therapy
 - Correlations between biomarker characteristics and clinical parameters
 - Change in biomarkers over time
 - Explore potential differences in distribution/phenotypes of monocytes, Th-cells, dendritic cells and new biomarkers identified using proteomics between patients during treatment with prednisone or methotrexate at all points