Pegloticase ( Krystexxa ) is a potential new treatment option for patients with chronic gout intolerant to other urate-lowering therapies.

An analisys has been performed to investigate safety ( adverse events, death ) and efficacy ( pain, function, frequency of flares, quality of life, uric acid level, radiographic damage ) of Pegloticase in various doses or as compared to placebo or other interventions for treatment of hyperuricemia in patients with chronic gout.

Only one open-label, phase-II randomized controlled trial ( RCT ) ( n=41 ) met the selection criteria that compared various doses of Pegloticase without comparison to placebo or another treatment.
Patients were randomized to one of the four doses of Pegloticase for 12 to 14 weeks - 4mg every 2 weeks, 8mg every 2 weeks, 8mg every 4 weeks and 12mg every 4 weeks.
Percent responders ( uric acid below 6 mg/dl 80% or more time ) in the four dose groups were 56%, 88%, 52% and 62%. Percent time without hyperuricemia ( uric acid below 6 mg/dl ) was 78%, 92%, 76% and 76% respectively. No between dose differences were noted.
Most common adverse events ( 10% or more patients ) included nephrolithiasis, arthralgia, anemia, dyspnea, headache, muscle spasms, nausea and pyrexia; 89% reported one or more gout flares during the study.
Pain, patient/physician global, function, quality of life, tophus size/regression and radiographic progression were not reported in this study.

In conclusion, there are no published double-blind, placebo-controlled RCTs of Pegloticase. More evidence is needed to assess risks/benefits of Pegloticase in patients with chronic gout.

Source: Cochrane Database Syst Rev, 2010

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