Idiopathic pulmonary fibrosis is a rare disease whose cause is unknown, characterised by gradual, progressive and irreversible damage to the lung tissue. It affects men more often than women, and those over the age of 50 in particular. Its symptoms are increasing difficulty in breathing, with a dry cough, progressing in stages towards respiratory failure and death within 2 to 5 years.
There is no known drug treatment that is able to halt or slow down the progress of the disease or to increase life expectancy. Treatments attempt to maintain quality of life as best they can, in particular through pulmonary rehabilitation and continuous oxygen therapy if necessary. The patient’s condition sometimes requires a lung transplant.
Pirfenidone, an immunosuppressant commercialised for the treatment of pulmonary fibrosis, raised hopes. However, its harm-benefit balance is unfavourable. According to the two published clinical trials, it is ineffective against the aggravation of the fibrosis, and has no effect on mortality. Patients would probably be unaware of the minimal decrease in the forced vital capacity under treatment observed in a single trial. Nearly 15% of patients stopped the treatment because of its adverse effects. Severe adverse effects were reported, especially skin, cardiac and digestive, and there might be carcinogenic effects. Other adverse effects are to be expected given the immunosuppressant properties of this substance.
It is better to concentrate on respiratory physiotherapy and oxygen therapy if necessary, while pursuing research into treatments offering real therapeutic benefits.
©Prescrire 1 May 2013
"Pirfenidone. First, do no harm" Prescrire Int 2013; 22 (138): 117-119. (Pdf, subscribers only).