Evaluation of Proposed Criteria for Remission and Evidence-Based Development of Criteria for Complete Response in Patients With Chronic Refractory Gout.

Abstract

ObjectiveThe objective of this study is to assess criteria for gout remission and to use the results to inform criteria for a complete response (CR).MethodsA post hoc analysis of two clinical trials was undertaken to determine the frequency with which subjects with chronic refractory gout who were treated with pegloticase met remission criteria. Mixed modeling was then employed to identify the components that best correlated with time to maximum benefit.ResultsOf the 56 subjects treated with biweekly pegloticase for whom adequate data were collected, 48.2% met the remission criteria. When subjects with persistent lowering of urate levels were examined separately, 27 of 32 (84.4%) met the criteria for remission. In contrast, even when the requirement for lowering of serum urate levels was waived, only 2 of 24 (8.3%) subjects without persistent lowering of urate levels and 0 of 43 subjects receiving placebo met criteria. Mixed modeling indicated that in addition to urate levels, assessment of tophi, swollen joints, and tender joints and patient global assessment best correlated with time to maximum benefit. Using these criteria of CR, 23 of the responders (71.9%) met the criteria. All patients who achieved a CR maintained it for a mean duration of 507.4 days. Finally, 64% of persistent responders to monthly pegloticase also met criteria for CR.ConclusionThese results have validated the proposed remission criteria for gout and have helped define criteria for CR in individuals with chronic gout treated with pegloticase. This composite CR index can serve as an evidence‐based target to inform the design and end points of future clinical trials.