This Phase II investigator-initiated trial evaluated the safety and efficacy of tofacitinib 5 mg BID over 48 weeks in 39 patients with rheumatoid arthritis-associated interstitial lung disease (RA-ILD), with KL-6 as a serum biomarker of lung disease activity.
92% of patients experienced at least one adverse event (most commonly upper airway infections); 3 patients died; 15 patients (38.5/100 patient-years) met the combined endpoint of mortality and/or progressive pulmonary fibrosis (PPF). FVC declined by a mean of -72 mL at 48 weeks. KL-6 fell from a baseline LSM of 34,358 mU/mL to 32,191 mU/mL (LSM decline of -2,167 mU/mL). At 48 weeks, 51% of patients achieved low disease activity by SDAI.
- Small sample (n=39) limits power to detect efficacy signals beyond biomarker trends. - No control arm; observed changes in FVC and KL-6 cannot be attributed solely to tofacitinib. - Nine patients also received nintedanib during the trial, introducing potential confounding despite subgroup analysis.
Tofacitinib carries a high adverse-event burden in RA-ILD (92%), and nearly 40% of patients still progressed or died at 48 weeks — clinicians should weigh this risk carefully. The decline in KL-6 levels may support its use as a monitoring biomarker in this population, but efficacy conclusions require confirmation in a controlled trial.
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