AB1039 PATIENT REPORTED OUTCOMES AMONG PATIENTS RECEIVING TREATMENT FROM THE PEGLOTICASE PHASE 3 CLINICAL TRIALS FOR UNCONTROLLED GOUT

Abstract

BackgroundGout affects more than 9 million Americans,1 with some suffering so severely that everyday life becomes painful and difficult.2 Pegloticase is an infused uricase-based medicine that significantly reduces serum uric acid (sUA) levels in patients suffering from treatment-resistant gout,3 but the direct effect of treatment on quality of life (QoL) by response status has not yet been reported.4ObjectivesTo determine if the effect of pegloticase treatment on QoL and other patient-reported outcomes is significant compared to placebo, regardless of treatment response status.MethodsThis analysis utilized results from two parallel 6-month randomized phase 3 registrational clinical trials in which subjects were treated with 8 mg of pegloticase or placebo biweekly. Responders were those who maintained sUA levels <6 mg/dL for ≥80% of the time during Months 3 and 6 combined. Multiple QoL measurements were taken throughout the 6-month treatment period, including the Health Assessment Questionnaire (HAQ; Pain, Disability Index [DI], and Health), the 36-Item Short Form Health Survey Questionnaire (SF-36), and the number of swollen and/or tender joints. This analysis assessed the differences between three groups: treatment responders, treatment non-responders, and placebo-treated patients. The change from baseline to Week 25 in each assessment was analyzed using a mixed model for repeated measures adjusting for age, visit, presence of tophi at baseline, and the interaction between visit and group.ResultsA total of 36, 49, and 43 patients were included in the treatment responder, treatment non-responder, and placebo-treated groups, respectively. Significant differences in the change from baseline to Week 25 in HAQ-Pain, HAQ-DI, HAQ-Health, and SF-36 Physical Component Score (PCS) between treatment responders and placebo-treated patients were observed (all P ≤ 0.006, Figure 1). The reduction in the number of swollen or tender joints was also significantly greater in treatment responders compared to placebo-treated patients (P < 0.001). Between treatment responders and treatment non-responders, there were no significant differences in the change from baseline in HAQ-Pain, HAQ-DI, HAQ-Health, and SF-36 PCS. Significant differences were observed between treatment non-responders and placebo-treated patients in the change in HAQ-Pain, HAQ-DI, HAQ-Health, and SF-36 PCS (all P ≤ 0.003), as well as the number of swollen or tender joints (P = 0.049, Figure 1). The change in SF-36 Mental Component Score was not significantly different between any of the 3 groups examined.ConclusionIn the Phase 3 registration trials of pegloticase used as monotherapy for uncontrolled gout, QoL parameters improved among pegloticase-treated patients regardless of response status.