US Pharm. 2011;36(9):HS-39-HS-40. 

Patients with severe, chronic gout who received pegloticase for 6 months exhibited greater improvement in uric acid (UA) level measurements as well as physical function and quality of life than with conventional treatments, according to a study published in JAMA. Although available oral urate-lowering agents can achieve target UA in most patients, urate-lowering therapy fails in approximately 3% of patients due to treatment resistance, contraindication, or intolerance. Many of these patients may progress to severe chronic gout and suffer from frequent arthritic flares and chronic arthropathy, often accompanied by deformity, chronic pain, functional disability, and impaired health-related quality of life. Pegloticase, which was recently approved, was developed for patients who do not respond to conventional urate-lowering agents. The drug is intravenously administered and degrades urate.

John S. Sundy, MD, PhD, of Duke University Medical Center, Durham, North Carolina, and colleagues report results of two randomized, placebo-controlled, 6-month trials of the urate-lowering and clinical efficacy and tolerability of pegloticase in patients with refractory gout. The researchers found that patients treated with biweekly doses of pegloticase experienced response rates of 47% and 38% in the two trials. Patients treated with monthly pegloticase reported response rates of 20% and 49%. Response rates were 0% in both placebo groups. The researchers also found that both pegloticase dosing groups reported significant improvements in physical function and quality of life compared with those taking placebo. Patient-reported pain was significantly reduced with biweekly pegloticase versus placebo.