Interstitial lung disease · Antifibrotics in IPF
Assessment of Pirfenidone to Confirm Efficacy and Safety in IPF
| Design | Phase 3, double-blind, placebo-controlled RCT |
|---|---|
| Treatment | Oral pirfenidone 2403 mg per day |
| Control | Placebo |
| Population | 555 patients with idiopathic pulmonary fibrosis |
| Follow-up | 52 weeks |
| Primary endpoint | Change in FVC or death at week 52 |
| Result | A 47.9% relative reduction in the proportion of patients with an absolute decline of 10 percentage points or more in predicted FVC or death, and a 132.5% relative increase in the proportion with no FVC decline (p<0.001). Six-minute walk distance decline was reduced (p=0.04) and progression-free survival improved (p<0.001). No significant difference in dyspnoea (p=0.16) or death from any cause (p=0.10). |
| Secondary endpoints | Six-minute walk distance, progression-free survival, dyspnoea, mortality |
Pirfenidone, as compared with placebo, reduced disease progression, as reflected by lung function, exercise tolerance, and progression-free survival, in patients with idiopathic pulmonary fibrosis. Treatment was associated with an acceptable side-effect profile and fewer deaths.
The trial authors, in the published abstract
Published back to back with INPULSIS, and together they ended the era in which IPF had no treatment. Neither drug reverses fibrosis or clearly prolongs life on its own trial; both slow the slope. That distinction is the substance of every antifibrotic conversation with a patient.
King TE Jr, Bradford WZ, Castro-Bernardini S, et al. A phase 3 trial of pirfenidone in patients with idiopathic pulmonary fibrosis. N Engl J Med 2014 May 29;370(22):2083-92.