Safety and efficacy of pirfenidone in severe idiopathic pulmonary fibrosis

Introduction: Pirfenidone is the only, so far, drug approved for the treatment of mild or moderate IPF. Aim: To study the safety and efficacy of pirfenidone in a group of patients with severe disease (FVCl50% and/or TLco l 35%). Methods: Pirfenidone was prescribed to 41 patients with severe IPF. Lung function tests, GAP score and 6 minute walk test (6MWT) were recorded prior and 6 months after treatment initiation. Additionally, patients were strictly monitored with blood tests and liver biochemistry, while adverse events (AE) were recorded and compared to data of previous studies. Results: Mean age was 66.25 ± 12.25. 19.5% were female and 80.5% male. 14.6% had type I respiratory failure. 4.8% had dyspnea MRC I, 22% MRC 2, 39% MRC III, and 34%MRC IV. During the study period 3 patients died from irrelevant to drug causes (1 septic shock, 1 from myocardial infarction and 1 from disease progression) and 7 stopped treatment due to mild-moderate AE. 1 patient stopped for reasons that he did not wish to share. AE included gastro-intestinal discomfort (n=8, 19.5%), photosensitivity (n=5, 12.2%), rash (n=5, 12.2%), fatigue (n=4, 9.75%), diarrhea (n=3, 7.3%), community-acquired pneumonia (n=3, 7.3%), anorexia (n=2, 5%), hematuria (n=2, 4.8%), septic shock (n=2, 4.8%), insomnia, myocardial infarction and irritability one each. No significant differences were observed regarding FVC, TLco, 6MWT, GAP score, SGOT and SGPT pre and 6 months post treatment. Conclusion: Pirfenidone is safe when administered to patients with severe IPF, since AE were comparable to the AZUMA and CAPACITY trials results. Although patients remained stable, longer treatment periods are needed for definite conclusions regarding efficacy.