ASCEND (pirfenidone)
Trial question
What is the role of pirfenidone in patients with idiopathic pulmonary fibrosis?
Study design
Multi-center
Open label
RCT
Population
Characteristics of study participants
22.0% female
78.0% male
N = 555
555 patients (120 female, 435 male)
Inclusion criteria: patients with idiopathic pulmonary fibrosis
Key exclusion criteria: history of malignancy, severe hepatic impairment, ESRD, unstable deteriorating cardiac or pulmonary disease, pregnancy or lactation, or history of alcohol or substance abuse in the past 2 years
Interventions
N=278 pirfenidone (2403 mg PO per day for 52 weeks)
N=277 placebo (matching placebo PO for 52 weeks)
Primary outcome
Death or a ≥ 10% decline in forced vital capacity at 52 weeks
16.5
31.8
31.8 %
23.9 %
15.9 %
8.0 %
0.0 %
Pirfenidone
Placebo
Significant decrease ▼
NNT = 6
Significant decrease in death or a ≥ 10% decline in FVC at 52 weeks (16.5% vs. 31.8%; RR 0.52, 95% CI 0.21 to 0.83)
Secondary outcomes
Significant decrease in change in 6-minute walk distance or death at 52 weeks (25.9% vs. 35.7%; RR 0.73, 95% CI 0.03 to 1.43)
No significant difference in death from any cause (4% vs. 7.2%; HR 0.55, 95% CI 0.26 to 1.15)
Safety outcomes
Significant difference in grade 3 GI adverse events (5.4% vs. 1.4%) and grade 3 skin-related adverse events (1.8% vs. 0.4%).
Conclusion
In patients with idiopathic pulmonary fibrosis, pirfenidone was superior to placebo with respect to death or a ≥ 10% decline in FVC at 52 weeks.
Reference
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.
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