In an international study, researchers at the LMU Munich Hospital have shown for the first time that the inhaled drug treprostinil can significantly slow down the progression of idiopathic pulmonary fibrosis (IPF) and improve the quality of life of those affected. The drug, which has already been approved for pulmonary hypertension, proved to be effective and well tolerated overall in the phase III study. The results were published in the New England Journal of Medicine.

In idiopathic pulmonary fibrosis, the lung tissue scarred progressively without a known cause. Those affected suffer from increasing shortness of breath, dry dry cough and decreasing performance. The median survival time after diagnosis is three to five years. In Germany, about 25 to 30 people per 100,000 inhabitants fall ill every year, especially from the age of 65. So far, there are only two approved drugs that slow down the progression of the disease, but are sometimes associated with significant side effects. A lung transplant is the only option for a significant improvement, but it is only open to a few patients.

In the double-blind, randomized and placebo-controlled study, 593 patients with an average age of 72 years were observed for more than one year. The primary endpoint was the change in forced vital capacity (FVC), a measure of usable lung volume. In the treprostinil group, the FVC decreased by an average of 50 milliliters, in the placebo group by 136 milliliters. Clinically relevant deterioration occurred in 27 percent of participants in the verum group and 39 percent in the control group. Event-free survival without disease progression was also more favorable in the treatment group.

The quality of life developed clinically better with treprostinil than with placebo. The tolerability was rated as good overall. The most common adverse reaction was inhalation-induced cough (about 50 percent in the treprostinil group compared to 25 percent with placebo). One third of the patients in the verum group discontinued therapy due to side effects, compared to a quarter in the placebo group. According to the study authors, coughing could be controlled by appropriate measures in most of those affected.

The study was coordinated under the leadership of the Medical Clinic and Polyclinic V of the LMU Munich Hospital. Treprostinil was originally developed for pulmonary hypertension and had already shown positive effects on fibrotic processes in a previous study. The current results are considered an important advance for a disease that was previously difficult to treat. Whether the inhaled therapy concept will be approved depends on the results of two ongoing studies.

Original Paper:

Inhaled Treprostinil for Idiopathic Pulmonary Fibrosis | New England Journal of Medicine

Editor: X-Press Journalistenbüro GbR

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