Long-term observational studies of pirfenidone from international collaborative experiences
| Study name | Patients n | Type of study | Patient characteristics | Median length of treatment | Efficacy outcome | Adverse events | Treatment discontinuation due to adverse events | |
| GI | Skin | |||||||
| RECAP [25, 28] | 603 | Ongoing open-label, long-term, follow-up extension study | The baseline characteristics of patients were similar to those in the CAPACITY study in terms of FVC % predicted and DLCO % predicted; age: 68.3 years | 163.3 weeks (provisional) | FVC and survival outcome were similar to those in the CAPACITY pirfenidone group | Nausea in 30% of cases; diarrhoea in 22% | Rash in 13.3% of cases; photosensitivity in 8.8% | 3.3% (due to nausea, diarrhoea, photosensitivity and rash) |
| PASSPORT [27] | 530 | Ongoing, post-authorisation safety registry mandated by the EMA following the approval of pirfenidone; prospective, observational, long-term registry with a follow-up period of 2 years | Age: 69±8.8 years; baseline FVC (% pred): 64.5±16.6 | 5.5 months (provisional) | The longer term safety profile of pirfenidone appears to be consistent with those seen in the clinical trials | Nausea in 15.7% of cases | Rash in 7.5% of cases and photosensitivity reaction in 4.2% | 16% |
| INSIGHTS-IPF [29] | 502 | Multicentre, non-interventional study (registry) | Age: 68.7±9.4 years; baseline FVC (% pred): 67±18.2 | Started in November 2012 | Prospectively assessed the characteristics, diagnostic procedures, treatment patterns, quality of life and long-term outcome; 44.2% of patients were treated with pirfenidone | |||
Data are presented as mean±sd, unless otherwise stated. GI: gastrointestinal; FVC: forced vital capacity; DLCO: diffusing capacity of the lung for carbon monoxide; EMA: European Medicines Agency.