Pegloticase may reduce or eradicate the urate crystal mass in painful gout tophi, according to phase 3 trial results.
Baraf HSB, Becker MA, Gutierrez-Urena SR, et al., Tophus burden reduction with pegloticase: results from phase 3 randomized trials and open-label extension in patients with chronic gout refractory to conventional therapy. Arthritis Research & Therapy (2013) doi:10.1186/ar4318. First published online September 26, 2013 (open access).
Biweekly or monthly intravenous pegloticase in refractory chronic gout patients with multiple tophi reduce the burden and size of painful tophi, particularly for those who achieve sustained urate lowering, multinational clinical trials show.
The studies -- two randomized, placebo-controlled, 6-month trials and a subsequent open-label extension among pegloticase-responders -- used a standard IV dose (8 mg) every 2 weeks, monthly, or alternating with placebo at each infusion.
In the randomized trials, after six months 45% of biweekly patients (n=85), and 26% of the monthly group (n=84), showed complete resolution of at least one tophus with no new or enlarging tophi, versus 8% of placebo patients (n=43). Complete response was more likely in those sustaining plasma uric acid (PUA) below 6 mg/dL.
Other results include:
• Complete response in more than 80% of patients maintaining a PUA below 6 mg/dL for 1 year.
• Complete resolution of at least one tophus in 45% of the 212 patients in the randomized trials after 25 weeks of pegloticase therapy.
• Complete resolution of more than one tophus in 60% of the 113 patients in the extension study at the 101-week final visit.