TABLE 1.
Real-life experience with pirfenidone in observational studies
First author [ref.] | Country | Patients n | Patient characteristics | Efficacy outcome | Adverse events | Treatment discontinuation due to adverse events % | |
GI | Skin | ||||||
Bonella [18] | Germany | 45 | Age: 69±7 years Baseline FVC (% pred): 61±15 Baseline DLCO (% pred): 48±14 | 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 [19] | Japan | 76 | Age: 70.5±8.3 years Baseline FVC (% pred): 65.3±16.1 Baseline DLCO (% pred): 55.9±17.8 | Reduction in FVC and DLCO decline | 18 (24)# | 19 (25)¶ | 18 |
Arai [20] | Japan | 41 | Age: 70 (65.5–75.5) years Baseline VC (% pred): 66.7 (54.8–77.8) | Significant reduction in VC decline in patients with severity grades I–II (Japanese Respiratory Society criteria) | 24 (59)+ | 5 (12)§ | 15 |
Oltmanns [21] | Germany | 63 | Age: 68±7 years Baseline FVC (% pred): 70±19 Baseline DLCO (% pred): 40±14 | Stable lung function in 62% of patients | N/A | N/A | 13 |
Chaudhuri [22] | UK | 40 | Age: 65.8 (48–80) years Baseline FVC (% pred): 77.3 (range 46–146) Baseline DLCO (% pred): 42.4 (14–81) | Reduction in FVC and DLCO decline at 9 months | 87 | 10 | 15 |
Loeh [23] | Germany and Italy | 197 | Age: 69.8±0.6 years Baseline FVC (% pred): 65±18.8 Baseline DLCO (% pred): 39.8±17 | Decrease in annual decline in FVC; greater effect was observed in progressive IPF | N/A | N/A | N/A |
Harari [24] | Italy | 128 | Age: 69±7 years Baseline FVC (% pred): 75±18 Baseline DLCO (% pred): 47±15 | Decrease in annual decline in FVCƒ; greater effect in patients with more severe disease (GAP II–III and FVC at baseline ≤75% pred) | N/A | N/A | N/A |
Data are presented as mean±sd, mean (range) or n (%), unless otherwise stated. GI: gastrointestinal; FVC: forced vital capacity; DLCO: diffusing capacity of the lung for carbon monoxide; VC: vital capacity; N/A: not applicable; GAP: GAP severity index (a simple point-scoring system based on gender (G), age (A) and two lung physiology (P) variables (FVC and DLCO). #: gastric distress (n=9) and nausea (n=9); ¶: photosensitivity (n=14) and rash (n=5); +: anorexia and/or nausea; §: photosensitivity; ƒ: relative change in FVC. Reproduced and modified from [25] with permission from the publisher.