The 2013 American Thoracic Society Guidelines for the Diagnosis and Treatment of Idiopathic Pulmonary Fibrosis (available at www.nxtbook.com/nxtbooks/md_conference_express/ATSIPF/index.php?startid=24#/0) essentially recommends that we SHOULD NOT TREAT patients with IPF given the lack of efficacy of most treatments coupled with the potential toxicity of available agents. However, since that publication was drafted three major new studies have been published in the New England Journal of Medicine that may require us to revisit this conclusion.
The first study appears to be the final nail in the coffin for N-acetyl cysteine in IPF as patients given NAC fared no better than their placebo-treated comparators (The Idiopathic Pulmonary Fibrosis Clinical Research Network. N Engl J Med 2014; 370:2093-2101). The second study (Richeldi L., du Bois R.M., Raghu G., et al. N Engl J Med 2014; 370:2071-2082) showed that the tyrosine kinase inhibitor nintedanib led to a reduced rate of decline in the forced vital capacity (FVC) over time. While promising, this molecule is relatively early-on in the prolonged FDA-approval process. The third study (King T.E., Bradford W.Z., Castro-Bernardini S., et al.N Engl J Med 2014; 370:2083-2092) found that pirfenidone – already approved for use in IPF in most of the rest of the world – significantly reduces the rate of decline in FVC and DLCO.
Given the paucity of effective therapies for IPF here in the U.S. and the consequences of untreated disease, it is anticipated that the FDA will approve pirfenidone making it the first/only agent indicated for this disease state. However, the FDA’s action is not anticipated to happen quickly for a variety of reasons. In an effort to get this apparently effective therapy into the hands of anxious IPF patients as quickly as possible, the manufacturer of pirfenidone (Intermune) has allowed sites who participated in prior pirfenidone trials to apply to become Early Access Providers. Given the involvement of Creighton Pulmonary with this agent for the past several years, we are one of a handful of centers authorized to provide patients with pirfenidone. For those patients meeting appropriate criteria, we can provide them with pirfenidone free of charge in return for their agreement to allow us to do safety monitoring (blood work). We have been swamped with patients coming to Omaha from Wyoming, Minnesota, Wiconsin, Missouri, Oklahoma, Kansas, etc. in the hopes they can be started on therapy.
If you have interested IPF patients – send them our way. If not – keep your eyes open for more regarding the evolution of therapy in this area.