Tolerability and Safety of Nintedanib and Immunosuppression in Autoimmune Inflammatory Myopathy and Progressive Pulmonary Fibrosis

Patients with progressive, fibrotic AIM-ILD on background immunosuppression enrolled in this trial were given nintedanib for 24 weeks. Primary endpoint was the percentage of subjects who took 90% or more of the study drug doses. Secondary endpoints included safety (adverse events) and efficacy (lung function) outcomes.

Bulk RNA sequencing of peripheral blood was performed at baseline and each subsequent visits to examine gene expression. Results: Eleven participants were enrolled, of which nine completed the study visits as per protocol at 24 weeks. The trial was discontinued due to slow recruitment.

Only 3 (27%) participants took 90% or more doses of nintedanib. Drug compliance ranged from 27% to 95%. The most common adverse events were diarrhea, nausea / vomiting, and abdominal pain.

There were no significant changes in lung function over time. No difference in gene expression over time was identified. Conclusions: Our trial was limited by small sample size and challenging recruitment, making clear conclusions on safety and tolerability difficult.

The nintedanib treatment did not induce measurable gene expression changes in peripheral blood. Based on this small population, tolerability of nintedanib with immunosuppression in AIM-ILD appears challenging.