Background and Aims: This all-case post marketing surveillance study was conducted to evaluate the safety and effectiveness of nintedanib in patients with idiopathic pulmonary fibrosis (IPF) in a Japanese real-world setting.

Methods: The study included Japanese patients with IPF who started nintedanib between 31 August 2015 and 25 December 2018. The primary outcome was the frequency of adverse drug reactions (ADRs), defined as adverse events (AEs) for which a causal relationship with nintedanib could not be excluded. The secondary outcome was change from baseline in forced vital capacity (FVC).

Results: A total of 5,717 patients were included in the safety analysis set; 78.1% were male, mean age was 71.7 years, 68.9% were ex-smokers, mean %FVC at baseline was 69.5%, mean exposure duration was 370.7 days, and initial nintedanib doses were 150 mg twice daily and 100 mg twice daily for 83.9% and 13.4% of patients, respectively. The nintedanib discontinuation rate was 66.8%, and the main reason for discontinuation (44.5%) was AEs. ADRs were noted in a total of 3,839 patients (67.2%), and the most frequently reported AEs were diarrhoea (36.2%) and hepatic function disorder (28.7%). Serious AEs were reported in 40.5% of patients. In the FVC effective set (N=3,124), the adjusted absolute mean changes in FVC from baseline were ?129.8 ± 9.4 mL at 52 weeks (N=1,032) and ?212.3 ± 11.7 mL at 104 weeks (N=481), and 744 patients (13.1%) experienced ?1 acute exacerbation over 104 weeks.

Conclusion: The results from this real-world clinical setting support findings from previously conducted clinical trials.