Background Idiopathic pulmonary fibrosis (IPF) is a fatal pulmonary disease with few therapeutic alternatives. Pirfenidone is the first drug that has shown clinical benefit in mild to moderate IPF in clinical trials. Due to a high economic impact, it is essential that we assess patient clinical outcomes in a real world practice.

Purpose The aim of this study was to assess the effectiveness and safety of pirfenidone in patients with mild to moderate IPF over a 12 month follow-up period.

Material and methods A retrospective, observational and descriptive study including patients with IPF who initiated therapy with pirfenidone from March 2013 to February 2014 was conducted. Clinical data were collected from the electronic clinical history including: demographic parameters (age, sex), forced vital capacity (FVC,%), diffusion capacity of the lung for carbon monoxide (DLCO,%) date of start of the treatment, dosage (mg/day), toxicity experienced during treatment and dispensing records. The main outcome evaluated was clinical response at 12 months, being considered positive when FVC and DLCO were increased from baseline, and stable disease when FVC and/or DLCO did not decrease more than 10% and 15% from baseline, respectively.

Results 10 patients (9 men) with a mean age of 69.5 ± 5.0 years were included. Mean baseline FVC and DLCO were 85.3 ± 15.4% and 55.6 ± 16.7%, respectively. Mean change in FVC at 12 months was -2.4 ± 6.9% (in pivotal clinical trials FVC decreased by 5.2% in the pirfenidone arm and by 8.3% in the placebo group). 1/10 patient died due to an unrelated lung disease cause, 1/10 stopped treatment due to poor tolerance (dizziness, fatigue, tremors and respiratory infection) and 8/10 continued treatment for 12 months, with 7 obtaining stable disease.

All patients showed some mild or moderate adverse effects. When needed, pirfenidone dose was reduced due to gastrointestinal intolerance (3/10) and phototoxicity (1/10) to 66% of the standard dose.

Conclusion In this clinical practice cohort, pirfenidone showed effectiveness and safety profiles consistent with those seen in previous clinical trials, showing that it is a well tolerated and effective drug in patients with mild-moderate IPF after 12 months of treatment. Dose adjustment was necessary in 3/10 patients due to gastrointestinal toxicity.

No conflict of interest.