Eur Respir J. 2006 Sep;28(3):463-5.

Idiopathic pulmonary fibrosis: treatment options in pursuit of evidence-based approaches

Raghu G.

Summary

The long-standing need to determine an effective medical treatment regimen with clinically significant and relevant end-points to improve outcome for patients with idiopathic pulmonary fibrosis (IPF) is being pursued by investigators in several phase I, II and III clinical trials. Patients confronted with IPF are understandably frustrated and vulnerable to hearsay and are desperate to try any medication in the hope of easing their suffering and increasing the likelihood of enhanced survival. This is particularly so when such medicines are available for clinical use either by prescription and/or over the counter. Caring physicians, also frustrated with the lack of evidence for an effective IPF treatment regimen, may find themselves reaching out for anecdotal reports and reacting to signals generated by post hoc exploratory and subgroup analyses derived from clinical studies, wanting to try some treatment regimen for their patients. In this regard, the joint American Thoracic Society/European Respiratory Society international consensus statement on IPF provided a guideline pending documentation of an effective treatment regimen 1. Acknowledging the limitations of the then-existing skeletal data, low-dose prednisone plus azathioprine or cyclophosphamide was suggested as a treatment regimen for patients with IPF, and an apparent "standard of care" for treatment of IPF with prednisone and azathioprine has evolved worldwide. The statement contained the disclaimer that its recommendation was based on the consensus of opinion of the expert panel, and the approach needs to be revisited as there was no grade-A evidence to support the treatment guidelines.

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