Pirfenidone treatment in idiopathic pulmonary fibrosis: nationwide Danish results

BACKGROUND: Pirfenidone was approved by the European Medicines Agency and introduced in most European countries in 2011 for treatment of idiopathic pulmonary fibrosis (IPF). OBJECTIVE: To describe the national Danish experiences of pirfenidone treatment for IPF during 30 months with respect to target population, safety, adherence to the treatment and effect analysis in a well-characterised IPF population in a real-life setting. METHODS: Retrospective data collection from medical records of all patients in Denmark with IPF from 2011 to 2014. Data included baseline demographics, high-resolution computed tomography (HRCT), histopathology, forced vital capacity (FVC) and 6-min walk test (6MWT). Longitudinal data on FVC, walk test, adherence to the treatment and vital status were also collected. RESULTS: Pirfenidone treatment was initiated in 113 patients. Mean age was 69.6±8.1 years (±SD), and 71% were male. Definite IPF diagnosis required thoracoscopic lung biopsy in 45 patients (39.8%). The remaining 68 cases had a definite (64 patients) or possible usual interstitial pneumonia (four patients) pattern on HRCT. Patients were followed for 0.1–33.8 months (median 9.4 months). Fifty-one patients (45.2%) needed dose adjustment, 18 (16%) patients discontinued therapy and 13 patients (11.5%) died. The annual mean decline in FVC was 164 ml (SE 33.2). The decline in 6MWT was 18.2 m (SE 11.2). Nausea (44.2%), fatigue (38.9%) and skin reactions (32.7%) were frequent adverse events. CONCLUSION: Patients with IPF treated with pirfenidone experienced tolerable adverse events. Patients were maintained on treatment due to a careful follow-up and dose adjustment programme. The annual decline in physiological parameters and mortality rate was comparable to previous randomised controlled trials.