Double-blind, placebo-controlled trial of pirfenidone in patients with idiopathic pulmonary fibrosis.
Idiopathic pulmonary fibrosis (IPF) is a fatal disorder without an effective therapy to date. In a double-blind, randomized, placebo-controlled trial, 107 patients were prospectively evaluated for efficacy of a novel compound, pirfenidone. The difference in the change in the lowest oxygen saturation by pulse oximetry (SpO2) during a 6-minute exercise test, the primary endpoint, from baseline to 6 months was not significant between the two groups (p = 0.0722). In a prespecified subset of patients who maintained a SpO2 greater than 80% during a 6-minute exercise test at baseline, the lowest SpO2 improved during a 6-minute exercise test in the pirfenidone group at 6 and 9 months (p = 0.0069 and 0.0305, respectively). Positive treatment effect was demonstrated in secondary endpoints: (1) change in VC measurements at 9 months (p = 0.0366) and (2) episodes of acute exacerbation of IPF occurring exclusively in the placebo group during the 9 months (p = 0.0031). Significant adverse events were associated with pirfenidone; however, adherence to treatment regimen was similar between pirfenidone and placebo groups. In conclusion, treatment with pirfenidone improved VC and prevented acute exacerbation of IPF during the 9 months of follow-up. Future long-term studies are needed to clarify the overall safety and efficacy of pirfenidone in IPF.
Azuma
Arata
A
Fourth Department of Internal Medicine, Nippon Medical School, Tokyo, Japan.
Nukiwa
Toshihiro
T
Tsuboi
Eiyasu
E
Suga
Moritaka
M
Abe
Shosaku
S
Nakata
Koichiro
K
Taguchi
Yoshio
Y
Nagai
Sonoko
S
Itoh
Harumi
H
Ohi
Motoharu
M
Sato
Atsuhiko
A
Kudoh
Shoji
S
D000368:Aged; D000894:Anti-Inflammatory Agents, Non-Steroidal / Q000009:adverse effects / Q000627:therapeutic use*; D004311:Double-Blind Method; D015444:Exercise / Q000502:physiology; D005080:Exercise Test; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D010101:Oxygen Consumption; D011446:Prospective Studies; D011658:Pulmonary Fibrosis / Q000188:drug therapy* / Q000503:physiopathology; D011728:Pyridones / Q000009:adverse effects / Q000627:therapeutic use*; D014109:Total Lung Capacity; D014797:Vital Capacity
D000894:Anti-Inflammatory Agents, Non-Steroidal; D011728:Pyridones; C093844:pirfenidone