A new study recently published in the journal Advances in Therapy provided evidence of the therapeutic benefit of pirfenidone (known by the brand name Esbriet) for IPF patients in a real-world clinical practice setting. The study is entitled “Early Experience of Pirfenidone in Daily Clinical Practice in Belgium and The Netherlands: a Retrospective Cohort Analysis,” and was conducted by researchers at Erasmus MC, University Hospital Rotterdam and University Medical Center Utrecht in The Netherlands, and the University Hospitals Leuven in Belgium.

IPF is a rare, progressive fatal lung disease of unknown origin in which the alveoli and the lung tissue are damaged, becoming thick and scarred (fibrosis), leading to severe breathing difficulties and compromising oxygen transfer between the lungs and the bloodstream. The disorder is characterized by a shortness of breath that gradually worsens, with respiratory failure being the main cause of death. There is no cure for IPF and it is estimated that almost 130,000 individuals in the United States and 5 million worldwide suffer from the disease. IPF has a poor prognosis and around two-thirds of the patients succumb to the disease within five years after being diagnosed.

Pirfenidone is an oral drug developed for the treatment of pulmonary fibrosis. It is an anti-fibrotic drug thought to act by decreasing the production of collagen, growth and inflammation factors, ultimately leading to a reduction in lung fibrosis. Pirfenidone was the first drug approved for the treatment of IPF in adult patients by the European Medicines Agency (EMA) in 2011, and the U.S. Food and Drug Administration (FDA) in 2014. In a recent Phase 3 clinical trial (ASCEND, NCT01366209), pirfenidone has been shown to slow disease progression, and to reduce lung function decline and mortality rates in IPF patients.

The goal of the study was to evaluate the effectiveness and safety profile of pirfenidone for IPF treatment in real-world clinical practice and compare the results to the ones obtained in controlled clinical trials.

The team analyzed clinical records of 63 patients with mild-to-moderate IPF under pirfenidone therapy in three different centers in Belgium and the Netherlands in the period between April 2011 and October 2013. Special attention was paid to pulmonary function measurements before and after pirfenidone treatment, including the forced vital capacity (FVC; the amount of air that can be exhaled from the lungs after maximum inhalation). The decline in lung function was defined as an absolute 10% or more of decline in FVC after 12 months of treatment.

Researchers found that the mean decline in FVC was 4.8% for 32 patients with available data 6 months prior to therapy, with an improvement to 0.8% at 6 months after starting pirfenidone treatment. After 12 months of treatment, ten patients had an absolute decline of ≥10% in FVC or have died. Concerning adverse events, loss of appetite (25.3%) and nausea (11.1%) were the most frequently reported, with nausea being the underlying reason for treatment discontinuation in 7.9% of the cases.

The research team concluded that their clinical practice study in a cohort of IPF patients resulted in a favorable effectiveness profile and an acceptable safety profile (with fewer deaths) comparable to the Phase 3 clinical trial study ASCEND. The authors believe that their findings in the context of a real-world IPF patient cohort support the clinical benefit offered by pirfenidone therapy in clinical practice. The authors, however, emphasize that physicians prescribing pirfenidone should wisely and constantly evaluate the balance between adverse effects and the efficacy of pirfenidone in slowing lung function decline for each patient.