Results from a recent study published in the journal Advance Therapy, showed that pirfenidone (known by the brand name Esbriet from Roche) is effective and has a safety profile consistent with those seen in the Phase III clinical study ASCEND for the treatment of IPF. According to the researchers, these results highlight the benefits and challenges related to treatment with pirfenidone.
Idiopathic pulmonary fibrosis (IPF) is a rare, progressive, irreversible and ultimately fatal chronic lung disease, with a median survival time of 2–5 years.
Pirfenidone is an oral anti-fibrotic drug with anti-inflammatory properties and was the first drug approved for the treatment of adult patients with IPF by the European Medicines Agency (EMA) in 2011, followed by approval by the Food and Drug Administration (FDA) in 2014. Nintedanib, a tyrosine kinase inhibitor, has been approved for IPF by the FDA and the EMA.
In the study, researchers investigated early clinical experiences of the management of IPF treated with pirfenidone at three centers in the Netherlands and Belgium. The team of researchers then compared the data with clinica results from a recent Phase III clinical study (ASCEND). Using the clinical records the researchers examined the effectiveness and safety profile of pirfenidone for the treatment of patients with IPF.
The assessment of pulmonary function includes % predicted forced vital capacity (%FVC) and % predicted diffusing capacity of the lungs for carbon monoxide (%DLCO). These parameters were analyzed from 6 months prior to pirfenidone treatment up to 12 months of treatment.
Lung function decline was defined as an absolute C10% decline in FVC from baseline or death at 12 months.
Results revealed that in 63 IPF patients, the average baseline %FVC and %DLCO were 75.0% and 47.9%, respectively. Results also showed that 46% of patients had a surgical lung biopsy and 69.8% had a high-resolution computed tomography scan with a definite usual interstitial pneumonia pattern.
The mean decline in %FVC for 32 patients with available data was 4.8% from -6 months to baseline and 0.8% from baseline to 6 months. Across these time interims, a minor decline in DLCO was similarly observed during therapy.
At 12 month follow-up, 10 patients died or had an %FVC decline C10%. Nausea (11.1%) and loss of appetite (25.3%) were the most frequent gastrointestinal observed adverse events. Nausea was the most reported reason for treatment discontinuation (7.9%).
The researchers concluded that these real-world results might help to further educate physicians who lack experience in managing a patient with IPF receiving pirfenidone treatment and, in turn, support better patient outcomes.