Efficacy and Safety of Pirfenidone in Advanced Idiopathic Pulmonary Fibrosis

Abstract

Background: Although phase 3 trials showed significant efficacy and acceptable safety profiles for pirfenidone in mild-to-moderate idiopathic pulmonary fibrosis (IPF), data on advanced IPF are limited. Objectives: The study aimed to evaluate the efficacy and safety of pirfenidone in advanced IPF patients. Methods: The clinical data of 138 IPF patients (advanced group: 27%) treated with pirfenidone were retrospectively reviewed and compared between advanced and non-advanced groups. Advanced IPF was defined as (1) forced vital capacity (FVC) < 50% predicted or (2) diffusing capacity for carbon monoxide < 30% predicted. Results: The mean treatment duration was 51.3 weeks, and lung function analysis was performed in 81 patients (17 in the advanced group). Changes in FVC and total lung capacity (TLC) were significantly reduced at 6 months after treatment in both the advanced (ΔFVC [6 months]: –6.3 [before] vs. 0.7% predicted [after]; ΔTLC: –5.3 vs. 0.8) and non-advanced (ΔFVC: –3.4 vs. 0.5; ΔTLC: –3.1 vs. –0.9) groups. The rate of decline in FVC and TLC was significant before treatment, but not after treatment in the advanced (FVC: –1.27 [before] vs. 0.21% predicted/month [after]; TLC: –0.89 vs. –0.15) and non-advanced (FVC: –0.60 vs. –0.20; TLC: –0.54 vs. –0.17) groups. The advanced group showed a similar rate of adverse events (AEs) (78.4 vs. 88.1%, p = 0.270), but more serious AEs (40.5 vs. 10.9%, p < 0.001) including death (24.3 vs. 5.0%, p = 0.002). Conclusions: In advanced IPF, pirfenidone showed similar efficacy and safety to non-advanced IPF except for serious AEs, which may be due to the advanced status itself.

Efficacy of lower dose Pirfenidone for idiopathic pulmonary fibrosis in real practice: a retrospective cohort study

“…Although the proportion of patients who maintained the standard dose was low, the rate of complete discontinuation due to adverse events was 24.6%, which is similar to the rate found in other studies (4,7,10,11,19).…”

“…Although there have been several real-world studies of pirfenidone use in IPF (4,10,11,16,17), those studies did not focus on the dose of pirfenidone. Our study comprehensively investigated the effect of the dose of pirfenidone on the pulmonary function of IPF patients from three tertiary referral hospitals in real-world conditions.…”

“…However, the efficacy of pirfenidone according to disease severity varied. It was similar in advanced IPF and non-advanced IPF in one study, but another study showed that it was more beneficial for advanced IPF patients (10,11).…”

Guidelines of the Polish Respiratory Society for Diagnosis and Treatment of Idiopathic Pulmonary Fibrosis

“…Data on the efficacy and safety of antifibrotic agents in IPF patients with severe lung function impairment (FVC < 50% of predicted, T L,CO < 30% of predicted) are indeed limited. Nevertheless, an increasing body of evidence from observational studies using antifibrotic agents in this patient population indicates they have similar efficacy and adverse reaction profiles as those observed in pre-marketing randomised clinical trials [253,[258][259][260][261][262][263]. This data supports antifibrotic treatment in IPF patients outside the range of pulmonary function parameters that was used in the inclusion criteria of randomised studies assessing the use of pirfenidone and nintedanib in IPF.…”

Real-world experiences: Efficacy and tolerability of pirfenidone in clinical practice

“…Therefore, the relationship between IPF prognosis and DLco needs to be further confirmed in the future studies considering the factor of PH. In terms of pirfenidone therapy in advanced IPF, pirfenidone demonstrated clinically relevant benefits and well tolerability in several recent studies [33][34][35]. The former research had demonstrated that smoking was a potential risk factor for the development of IPF (OR: 1.6-2.9) [36].…”