The study, which appeared online May 20 in the New England Journal of Medicine to coincide with a presentation at the annual meeting of the American Thoracic Society (ATS), was conducted by IPF Clinical Research Network, funded by the National Heart, Lung, and Blood Institute of the National Institutes of Health.
Fernando Martinez, MD, University of Michigan Health System lung specialist reports that the findings demonstrate how important it is to test all treatments, even those treatments that doctors give routinely for any type of condition.
Martinez, who presented the findings at this week’s ATS meeting, and his colleagues report that patients in the mild to moderate stages of the progressive lung-scarring disease had a far higher chance of dying or being hospitalized if they were taking a three-drug combination used worldwide, compared with those taking a placebo.
More disturbing, the three-drug combo yielded no improvement in lung function or even slowing of loss of lung function, compared with placebo. This evidence is from a randomized, placebo-controlled, double-blind, federally-funded trial called PANTHER-IPF, for Prednisone, Azathioprine, and N-Acetylcysteine: a Study That Evaluates Response in Idiopathic Pulmonary Fibrosis. PANTHER-IPF included patients with a definitive diagnosis of IPF who were treated at 25 centers taking part in the IPF Clinical Research Network or IPFNet.
The study was stopped early when an interim analysis showed signs of harm from the three-drug combination of prednisone, azathioprine, and NAC.
The dramatic finding of harm from a standard treatment should cause physicians worldwide to stop using this combination to treat IPF patients similar to those in the trial and to apply rigorous testing methods to other types of treatment, the researchers say.
The authors salute the volunteer IPF patients who agreed to be randomly assigned to a treatment or placebo for 60 weeks.
In the results presented this week, the authors report that eight patients in the group of 77 assigned to the three-drug combination died, compared with one in the placebo group. A total of 23 of the three-drug patients were hospitalized during the trial, compared with 7 in the placebo group. There was no sign that the three-drug combination slowed the progression of IPF or improved lung function, as measured by forced vital capacity.
Because lung transplants are such a dramatic and rarely available therapy, researchers at U-M and other centers are working to find new treatments while also studying the underlying biological factors in the disease. The PANTHER-IPF trial was designed to test a standard therapy in a rigorous way.
Source: University of Michigan Healthcare System