Treatment of idiopathic pulmonary fibrosis with a new antifibrotic agent, pirfenidone - Results of a prospective, open-label phase II study

Idiopathic pulmonary fibrosis (IPF) is a progressive clinical syndrome of u nknown etiology and fatal outcome. Currently available therapies are ineffe ctive and associated with significant adverse effects. Pirfenidone, a new, investigational antifibrotic agent, was evaluated for its tolerability and usefulness in terminally ill patients with advanced IPF. Consecutive patien ts with IPF and deterioration despite conventional therapy or who were unab le to tolerate or unwilling to try conventional therapy were treated with o ral pirfenidone. Treatment was administered on a compassionate basis (open- label). Fifty-four patients were followed for mortality, change in lung fun ction, and adverse effects. Their mean age was 62, mean duration of symptom s 4.6 yr, and time since lung biopsy was 3.2 yr. Conventional therapy was d iscontinued in 38 of 46 patients; the other eight were able to decrease the ir prednisone dosage and eight had no previous conventional treatment. One- and 2-yr survival was 78% (95% CI 66%, 89%) and 63% (95% CI 50%, 76%), res pectively. Patients whose lung functions had deteriorated prior to enrollme nt appeared to stabilize after beginning treatment. Adverse effects were re latively minor. The results of this study are encouraging. Pirfenidone is a promising new treatment for IPF that is well tolerated.