Long-term observational studies of pirfenidone from international collaborative experiences

Study namePatients nType of studyPatient characteristicsMedian length of treatmentEfficacy outcomeAdverse eventsTreatment discontinuation due to adverse events
RECAP [25, 28]603Ongoing open-label, long-term, follow-up extension studyThe baseline characteristics of patients were similar to those in the CAPACITY study in terms of FVC % predicted and DLCO % predicted; age: 68.3 years163.3 weeks (provisional)FVC and survival outcome were similar to those in the CAPACITY pirfenidone groupNausea 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]530Ongoing, 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 yearsAge: 69±8.8 years; baseline FVC (% pred): 64.5±16.65.5 months (provisional)The longer term safety profile of pirfenidone appears to be consistent with those seen in the clinical trialsNausea in 15.7% of casesRash in 7.5% of cases and photosensitivity reaction in 4.2%16%
INSIGHTS-IPF [29]502Multicentre, non-interventional study (registry)Age: 68.7±9.4 years; baseline FVC (% pred): 67±18.2Started in November 2012Prospectively 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.