Efficacy of pirfenidone for idiopathic pulmonary fibrosis: An Italian real life study

Abstract

In this national experience, pirfenidone reduced the rate of annual FVC decline (p = 0.065). Since pirfenidone provided significant treatment benefit for patients with moderate-severe disease, our results suggest that the drug may also be effective in patients with more advanced disease.

Idiopathic pulmonary fibrosis: from clinical trials to real-life experiences

“…With regard to attenuation of decline in FVC, the results of the analysis according to the GAP index staging system confirmed that, compared with patients with less advanced disease (GAP stage I), those with more advanced disease (GAP stages II and III) could benefit the most from the administration of pirfenidone ( p-value for homogeneity of difference between strata of 0.041). Ultimately, in this trial, as already reported by LOEH et al [23], 16 (12.5%) out of 128 patients improved their FVC by >10% during therapy with pirfenidone [24]. Only 22 (17.1%) patients out of 128 were treated with lower than the standard dose of the drug (2403 mg·day −1 ); however, such dose reduction did not influence the outcomes.…”

“…Mean±SD FVC was 72±20% predicted, VC was 72±20% predicted and DLCO was 35±15% predicted. Multidisciplinary discussion was the diagnostic basis in 108 (21.8%) patients and, in order to obtain adequate lung specimens, histology was performed in 171 (34.1%) patients (as opposed to 25% of the Italian study [24]). The authors compared the data from their national registry to those of recent randomised clinical trials: patients in INSIGHTS-IPF were the same age as patients in ASCEND [8] and PANTHER [32], but were older than those in the two INPULSIS studies [9].…”

“…Some suggestions can be gathered from these real-life studies, such as: 1) pirfenidone could also be effective in patients who are more severe than those randomised in clinical trials (the Italian experience [24] and the German experience of BONELLA et al [18] are significant in that direction); 2) pirfenidone is well tolerated, and the most common adverse events are gastrointestinal, skin-related events and weight loss; 3) to improve compliance it is crucial to manage any adverse event or ADR with the patients [33]; and 4) some studies report that the association of concomitant medications such as NAC and/or corticosteroids could negatively affect tolerability. However, data regarding this last issue will soon be available thanks to the PANORAMA studies evaluating the combination of pirfenidone with NAC [34].…”

“…During the treatment with the new antifibrotic drug pirfenidone, the difference in percentage changes of FVC compared with the pre-treatment observational time (1 year) was 4.9% ( p=0.065) [24]. No significant variation in DLCO or in 6-min walking distance (6MWD) before and after starting therapy was detected.…”

“…A unique national study describing the experience of all Italian IPF referral centres (n=12), looking at 128 patients, was recently published [24]. This unsponsored experience considered patients who entered straight onto the Named Patient Programme and also patients who had been previously enrolled in the CAPACITY trials and subsequently entered this programme.…”