Real-world experience with pirfenidone reported in single-centre observational studies
| First author [ref.] | Country | Patients | Patient characteristics | Efficacy outcome | Adverse events | Treatment discontinuation due to adverse events % | |
| GI | Skin | ||||||
| Wijsenbeek [13] | Netherlands | 52 | Age: 63.4±7.7 years Baseline FVC (% pred): 68.3±18.4 | Stable lung function in 17 out of 20 patients treated for >6 months (three out of 20 declined). In 11 out of 19 patients cough score decreased (unchanged: n=7; increased: n=1) | NA | NA | 19 |
| Ravaglia [14] | Italy | 81# | Age: 69 (41–81) years Baseline FVC (% pred): 70.8 | Stable or significantly improved lung function in 40 (59%) out of 68 patients | NA | NA | 16 |
| Nieto Barbero [15] | Spain | 86 | Age: NA Baseline FVC (% pred): 70±19 | Stable FVC and DLCO in those who had pulmonary function testing (n=20) | 35 (41) | 11 (13)+ | 14 |
| Bonella [16] | Germany | 45 | Age: 69±7 years Baseline FVC (% pred): 61±15 | Stable lung function in 28 (70%) out of 40 patients; subjective improvement in cough in 12 (33%) out of 36 patients | 17 (38) | 10 (22) | 13 |
| Okuda [17] | Japan | 76 | Age: 70.5±8.3 years Baseline FVC (% pred): 65.3±16.1 | Reduction in FVC and DLCO decline | 18 (24)§ | 19 (25)ƒ | 18 |
| Arai [18] | Japan | 41 | Age: 70 (65.5–75.5) years Baseline FVC (% pred): 66.7 (54.8–77.8)¶ | Significant reduction in vital capacity decline in patients with severity grades I–II (Japanese Respiratory Society criteria) | 24 (59)## | 5 (12)+ | 15 |
| Oltmanns [19] | Germany | 63 | Age: 68±7 years Baseline FVC (% pred): 70±19 | Stable lung function in 62% of patients | NA | NA | 13 |
| Chaudhuri [20] | UK | 40 | Age: 65.8 (48–80) years Baseline FVC (% pred): 77.3 (46–146) | Reduction in FVC and DLCO decline at 9 months | 87 | 10+ | 15 |
Data are presented as n, mean±sd, mean (range) or n (%), unless otherwise stated. GI: gastrointestinal; FVC: forced vital capacity; NA: not applicable; DLCO: diffusing capacity of the lung for carbon monoxide. #: 81 patients were treated with pirfenidone but only 68 were included in the study as 13 patients discontinued the drug in the first 3 months because of adverse events; ¶: % predicted vital capacity; +: photosensitivity; §: gastric distress (n=9) and nausea (n=9); ƒ: photosensitivity (n=14) and rash (n=5); ##: anorexia and/or nausea.