TABLE 1

Clinical and real-world safety experience with pirfenidone

Clinical trial experienceReal-world experience
Pooled phase III trials# [12]Integrated trials [20]EAP [21]PASSPORT [22]
Patients n623129916201009
Duration of treatment years+1.0 (0–2.3)1.7 (0–9.9)0.45 (0–0.91)1.0 (0–2.4)
Cumulative total exposure PEY735.73159.9707.9415
AE typeTEAETEAEADRADR
TEAEs or ADRs per 100 PEY§
 Total418.8790.2419.667.0
 Nausea38.626.062.120.4
 Rash33.316.219.312.7
 Diarrhoea26.020.127.610.6
 Fatigue21.216.052.417.9
 Dyspepsia16.39.516.44.6
 Anorexia10.27.116.8NA
 Dizziness9.912.714.46.5
 Gastro-oesophageal reflux disease7.16.612.71.9
 Decreased appetite6.85.113.715.8
 Decreased weight3.47.36.615.6
 ADR/TEAE resulting in death2.218.5ƒNA1.4
 ADR/TEAE leading to discontinuation14.629.768.0

EAP: expanded access programme; PEY: patient-exposure years; AE: adverse event; TEAE: treatment-emergent adverse event; ADR: adverse drug reaction; NA: not available. #: CAPACITY (004/006) and ASCEND (016); : phase III trials+RECAP+PIPF-002, including two patients in the PIPF-002 study with a diagnosis of “pulmonary fibrosis”; +: data presented as median (range); §: one PEY is the equivalent of one patient exposed to a study drug for 1 year, total PEY is the sum of the PEY for each patient, adjusted rate per 100 PEY=(total number of events/total years of exposure)×100; ƒ: combined data for death and discontinuation.