Common Treatment for Patients With Progressive Lung Disease Found to Be Ineffective
N-acetylcysteine fails to improve lung function
A drug used to treat patients with mild-to-moderate lung damage from idiopathic pulmonary fibrosis (IPF) is no better than placebo for preserving lung function, according to a study supported by the National Institutes of Health (NIH).
The new data will be published May 23 in the New England Journal of Medicine.
Previous studies suggested that therapies combining N-acetylcysteine (NAC) with immunosuppressive and anti-inflammatory drugs could slow progression of the disease.
IPF, an incurable disease characterized by the buildup of fibrous scar tissue in the lungs, causes progressively worsening shortness of breath, coughing, chest pain, and fatigue. The disease affects approximately 200,000 people in the U.S., and only half of these individuals survive more than 2 to 3 years after their initial diagnosis
“IPF is a devastating disease,” said James Kiley, PhD, director of the Division of Lung Diseases at the NIH’s National Heart, Lung, and Blood Institute (NHLBI). “While it is disappointing that NAC was ineffective in preserving lung function in IPF, these are the kind of high-quality data that patients and their caregivers need to make informed decisions.”
Among other measures, researchers relied on the forced vital capacity (FVC) to assess changes in lung function. FVC is the total amount of air a person can exhale after taking the deepest breath possible. During 60 weeks of follow-up, the study found no statistically significant differences in FVC declines, IPF symptom flare-ups, deaths, or hospitalizations between patients treated with NAC and those given placebo.
The PANTHER-IPF trial was originally designed as a three-arm clinical study to compare a multidrug regimen of azathioprine, NAC, and prednisone with placebo and NAC alone.
The NIH stopped the multidrug therapy arm of the trial in 2011 because of safety concerns. Interim results from that arm showed that patients receiving the multidrug treatment had more deaths and more hospitalizations than had those given placebo, with no difference in lung function.
Source: NIH; May 18, 2014.