Author Insights: New Treatment Offers Hope for Patients With Severe Gout

Michael A. Becker, MD, of the University of Chicago and colleagues, say pegloticase can be an effective treatment for patients with severe, chronic gout, who do not respond to usual care. (Image: David Christopher for the University of Chicago Medical Center)

For patients with severe, chronic gout, who do not respond to conventional treatment, pegloticase (Krystexxa) appears to reduce plasma uric acid and pain levels while improving physical function and quality of life. Pegloticase, estimated to cost $2500 per dose, is an enzymatic alternative to conventional uric acid–lowering agents.

An article appearing today in JAMA finds pegloticase, given through biweekly intravenous infusion for 6 months, reduced plasma uric acid levels to the recommended 6.0 mg/dL² or less in 36 of 85 patients (42%) receiving pegloticase in the study. None of the 43 study patients receiving placebo met the uric acid reduction goal.

Michael A. Becker, MD, professor emeritus of medicine at the University of Chicago and coauthor, discussed some of his team’s findings.

news@JAMA: Which patients should consider taking this drug?

Dr Becker: Our best estimates are that pegloticase is best reserved for the small percentage of patients [approximately 3%] with gout who have progressed to serious disease—significant ongoing pain, disability, poor quality of life, and deformity—because they were not responding to conventional therapy.

news@JAMA: While the drug is considered efficacious, why did more than half of the patients not achieve the recommended uric acid level?

Dr Becker: Many had infusion reactions deemed significant enough to withdraw from the study; some had recurrence of gout flares and did not wish to continue; while others had adverse events that they, or their physicians, determined were serious enough to not go through with the trial. But you should know that while 58% did not achieve the primary end point at 6 months, we showed that everyone on the drug achieved benefits regarding the secondary end points such as pain relief and improved quality of life.

news@JAMA: How do you see physicians using this drug?

Dr Becker: I prefer to look at gout as a potentially progressive disease, and instead of curing the disease, what we did with pegloticase is move patients back in time to a point where the progression to more severe disease was relieved. In patients who have reached the maximum benefit from pegloticase, I’d be content to discontinue the drug.

Categories: Rheumatology