BACKGROUND: There is no clear evidence whether pirfenidone has a benefit in patients with probable or possible UIP, i.e. when idiopathic pulmonary fibrosis (IPF) is diagnosed with a lower degree of diagnostic certainty. We report on outcomes of treatment with pirfenidone in IPF patients diagnosed with various degrees of certainty. METHODS AND FINDINGS: We followed patients in the multi-national European MultiPartner IPF Registry (EMPIRE) first seen between 2015 and 2018. Patients were assessed with HRCT, histopathology and received a multi-disciplinary team (MDT) IPF diagnosis. Endpoints of interest were overall survival (OS), progression-free survival (PFS) and lung function decline. RESULTS: A total of 1626 patients were analysed, treated with either pirfenidone (N = 808) or receiving no antifibrotic treatment (N = 818). When patients treated with pirfenidone were compared to patients not receiving antifibrotic treatment, OS (one-, two- and three-year probability of survival 0.871 vs 0.798; 0.728 vs 0.632; 0.579 vs 0.556, P = 0.002), and PFS (one-, two- and three-year probability of survival 0.597 vs 0.536; 0.309 vs 0.281; 0.158 vs 0.148, P = 0.043) was higher, and FVC decline smaller (-0.073 l/yr vs -0.169 l/yr, P = 0.017). The benefit of pirfenidone on OS and PFS was also seen in patients with probable or possible IPF. CONCLUSIONS: This EMPIRE analysis confirms the favourable outcomes observed for pirfenidone treatment in patients with definitive IPF and indicates benefits also for patients with probable or possible IPF.

1st Department of Pulmonary Diseases Institute of Tuberculosis and Lung Diseases Warsaw Poland

Clinic of Pneumology and Phthisiology University Hospital Bratislava Bratislava Slovakia

Department of Pulmonary Medicine Ege University Medical School Izmir Turkey

Department of Pulmonology Semmelweis University Budapest Hungary

Department of Respiratory Diseases of the 1st Faculty of Medicine Charles University Thomayer University Hospital Prague Czech Republic

F Hofmann La Roche Ltd Basel Switzerland

Institute of Biostatistics and Analyses Faculty of Medicine Masaryk University Brno Czech Republic

Institute of Pulmonary Medicine Rabin Medical Center Petah Tikva Israel

Internal Medicine Clinic Akta Medica Belgrade Serbia

Paracelsus Medical University SALK Salzburg Austria

Pulmonary Department Acibadem City Clinic Tokuda Hospital Sofia Bulgaria

Pulmonary Department University Hospital Dubrava Zagreb Croatia

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