Real-world experience with pirfenidone reported in single-centre observational studies

First author [ref.]CountryPatientsPatient characteristicsEfficacy outcomeAdverse eventsTreatment discontinuation due to adverse events %
Wijsenbeek [13]Netherlands52Age: 63.4±7.7 years Baseline FVC (% pred): 68.3±18.4Stable 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)NANA19
Ravaglia [14]Italy81#Age: 69 (41–81) years Baseline FVC (% pred): 70.8Stable or significantly improved lung function in 40 (59%) out of 68 patientsNANA16
Nieto Barbero [15]Spain86Age: NA Baseline FVC (% pred): 70±19Stable FVC and DLCO in those who had pulmonary function testing (n=20)35 (41)11 (13)+14
Bonella [16]Germany45Age: 69±7 years Baseline FVC (% pred): 61±15Stable lung function in 28 (70%) out of 40 patients; subjective improvement in cough in 12 (33%) out of 36 patients17 (38)10 (22)13
Okuda [17]Japan76Age: 70.5±8.3 years Baseline FVC (% pred): 65.3±16.1Reduction in FVC and DLCO decline18 (24)§19 (25)ƒ18
Arai [18]Japan41Age: 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]Germany63Age: 68±7 years Baseline FVC (% pred): 70±19Stable lung function in 62% of patientsNANA13
Chaudhuri [20]UK40Age: 65.8 (48–80) years Baseline FVC (% pred): 77.3 (46–146)Reduction in FVC and DLCO decline at 9 months8710+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.