IPF: pirfenidone linked to better outcomes in advanced disease

Takeaway

• Patients with idiopathic pulmonary fibrosis (IPF) appear to benefit from pirfenidone (e.g., Esbriet) even amid advanced lung-function impairment.

Why this matters

• Most enrollees in pivotal pirfenidone trials ASCEND and CAPACITY had less advanced lung-function impairment.
• Diagnosis of IPF is often delayed, with some patients experiencing advanced impairment at diagnosis.

Key results

• Pirfenidone vs placebo groups (HRs; 95% CIs):
  • All-cause mortality (ACM): 0.28 (0.09-0.86; P=.0180);
    • Number needed to treat: 10.
  • ≥10% absolute %FVC decline or ACM: 0.40 (0.23-0.69; P=.0006).
  • Respiratory hospitalization (RH) or ACM: 0.45 (0.22-0.91; P=.0219).
  • ≥10% absolute %FVC decline or RH or ACM: 0.46 (0.28-0.76; P=.0018).
  • Pirfenidone group also had greater improvement from baseline in 6-minute walk distance and University of California-San Diego Shortness of Breath Questionnaire total score.
• No new safety signal.

Study design

• Post-hoc analysis of subgroup of ASCEND and CAPACITY trial participants (n=170). 
• Participants had %FVC <50% and/or percentage predicted carbon monoxide diffusing capacity <35%.
• As with other participants, they were randomly assigned to pirfenidone 2403 mg/day vs placebo over 52 weeks.
• Outcomes: all-cause mortality, composite outcomes.
• Funding: Genentech, Inc.; F. Hoffmann-La Roche, Ltd.

Limitations

• Small study population.