Randomized trial of acetylcysteine in idiopathic pulmonary fibrosis.

Abstract

BACKGROUND:

Acetylcysteine has been suggested as a beneficial treatment for idiopathic pulmonary fibrosis, although data from placebo-controlled studies are lacking.

METHODS:

In our initial double-blind, placebo-controlled trial, we randomly assigned patients who had idiopathic pulmonary fibrosis with mild-to-moderate impairment in pulmonary function to receive a three-drug regimen of prednisone, azathioprine, and acetylcysteine; acetylcysteine alone; or placebo. The study was interrupted owing to safety concerns associated with the three-drug regimen. The trial continued as a two-group study (acetylcysteine vs. placebo) without other changes; 133 and 131 patients were enrolled in the acetylcysteine and placebo groups, respectively. The primary outcome was the change in forced vital capacity (FVC) over a 60-week period.

RESULTS:

At 60 weeks, there was no significant difference in the change in FVC between the acetylcysteine group and the placebo group (-0.18 liters and -0.19 liters, respectively; P=0.77). In addition, there were no significant differences between the acetylcysteine group and the placebo group in the rates of death (4.9% vs. 2.5%, P=0.30 by the log-rank test) or acute exacerbation (2.3% in each group, P>0.99).

CONCLUSIONS:

As compared with placebo, acetylcysteine offered no significant benefit with respect to the preservation of FVC in patients with idiopathic pulmonary fibrosis with mild-to-moderate impairment in lung function. (Funded by the National Heart, Lung, and Blood Institute and others; ClinicalTrials.gov number, NCT00650091.).

• Authors: Idiopathic Pulmonary Fibrosis Clinical Research Network, Fernando J Martinez, Joao A de Andrade, Kevin J Anstrom, Talmadge E King Jr, Ganesh Raghu
• Journal: The New England journal of medicine (N Engl J Med) Vol. 370 Issue 22 Pg. 2093-101 (May 29 2014) ISSN: 1533-4406 [Electronic] United States
• PMID: 24836309 (Publication Type: Journal Article, Multicenter Study, Randomized Controlled Trial, Research Support, N.I.H., Extramural, Research Support, Non-U.S. Gov't)

Chemical References

• Free Radical Scavengers
• Azathioprine
• Prednisone
• Acetylcysteine

Topics

• Acetylcysteine (adverse effects, therapeutic use)
• Aged
• Azathioprine (adverse effects, therapeutic use)
• Disease Progression
• Double-Blind Method
• Drug Therapy, Combination
• Female
• Free Radical Scavengers (adverse effects, therapeutic use)
• Humans
• Idiopathic Pulmonary Fibrosis (drug therapy, mortality, physiopathology)
• Kaplan-Meier Estimate
• Male
• Middle Aged
• Prednisone (adverse effects, therapeutic use)
• Treatment Failure
• Vital Capacity (drug effects)