Pirfenidone treatment in idiopathic pulmonary fibrosis: too much of a great expectation?

Abstract

To the Editors: Idiopathic pulmonary fibrosis (IPF) is a dreadful, chronic and irreversibly progressive fibrosing disease lacking any effective treatment and leading to death in all affected patients [1, 2]. The scientific community is becoming aware that corticosteroids and immunosuppressors have failed to prove any efficacy concerning both mortality and the prevention of devastating complications, such as IPF acute exacerbations [1, 3]. Not only has this approach proven ineffective, but also harmful. Recently, the National Heart, Lung, and Blood Institute (Bethesda, MD, USA) aborted the continuation of treatment of combined prednisone, azathioprine and N -acetylcysteine (one arm of the three-arm multicentre PANTHER-IPF clinical trial) due to safety concerns [4]. The results of the interim analyses revealed that patients with IPF receiving the conventionally used triple-drug therapy consisting of prednisone, azathioprine and N -acetylcysteine had worse outcomes than those who received matched placebos. Thus, the need for an effective and safe treatment for IPF is still being pursued in several clinical trials. Pirfenidone (5-methyl-1-phenyl-2-[1H]-pyridone), a novel compound with anti-inflammatory, antifibrogenic and antioxidant properties, appears promising in IPF patients [1, 2 …