Real-world Esbriet Results Are Comparable to Those of Clinical Trials, Study Says

Esbriet (pirfenidone) shows similar benefits for people with idiopathic pulmonary fibrosis (IPF) in real-world settings as it does in clinical trials, a new study shows.

“Pirfenidone in real life: A retrospective observational multicentre study in Italian patients with idiopathic pulmonary fibrosis” was published in the journal Respiratory Medicine, and was funded by Genentech, the company that markets Esbriet.

Esbriet is an anti-fibrotic medication that has been approved for IPF treatment in the United States since 2014, and in Europe since 2011.

Data from clinical trials showed that Esbriet treatment can slow the rate of decline in forced vital capacity (FVC, a measurement of lung function), and that the safety profile of the therapy is reasonable.

However, clinical trials are tightly controlled scientific experiments, and these might not accurately reflect the treatment response of patients in the real world. Therefore, it is important to have data from patients treated outside the context of trials, too.

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In the new study, called IRENE (Italian Real-world Esbriet National Experience), researchers analyzed data from 379 IPF patients who had been treated with Esbriet at centers in Italy. All analyzed patients were at least 40 (mean age 67.6 years), most were male (78.1%), and former smokers (68.9%). All participants had at least one year’s worth of data while on Esbriet treatment, with FVC measured every six months in that time.

Results showed that overall at both six months and one-year timepoints, there was no statistically significant change from baseline in percent predicted FVC — 0.20% at month 6, and minus 0.83% at month 12. These results suggested that the treatment was indeed slowing lung function decline.

Regarding safety, among the patients analyzed, 95 (25.1%) experienced an adverse event that was deemed by the investigators to be related to Esbriet treatment. The most common side effects were gastrointestinal symptoms such as nausea (3.7% of patients), diarrhea (2.1%), and indigestion (2.1%), as well as skin symptoms such as photosensitivity reaction (5.0%) and rash (3.7%).

There were nine serious adverse events deemed related to Esbriet treatment. These included abnormal liver activity, decreased appetite, abdominal pain, gastroesophageal reflux, headache, and rashes. None of the 15 recorded deaths during the study were deemed related to Esbriet treatment.

“This retrospective analysis of a large observational cohort of Italian patients with IPF supported the previously known profile of pirfenidone in effectively delaying disease progression,” the researchers said, adding that “adverse events were consistent with the known safety profile of pirfenidone.”