Patient- versus physician-reported outcomes in rheumatoid arthritis patients treated with recombinant interleukin-1 receptor antagonist (anakinra) therapy

Abstract

Objectives: To determine whether patient-reported outcomes may differentiate treatment response better than physician-reported outcomes for rheumatoid arthritis (RA) patients being treated with anakinra.

Methods: A meta-analysis was conducted using data obtained from three separate randomized controlled clinical trials (RCTs) (n = 1007). Outcomes from 6-month assessments were grouped into four categories: American College of Rheumatology (ACR) response criteria, patient-reported measures (patient-reported pain, patient global assessment, and assessment of physical function using the Health Assessment Questionnaire), physician-reported measures (tender and swollen joint counts and physician global assessment), and laboratory tests (C-reactive protein and erythrocyte sedimentation rate). Effect sizes were calculated using changes from baseline and pooled standard deviations for each of these types of outcome.

Results: Active treatment with anakinra was superior to placebo by ACR(20) responses in all three RCTs. Effect sizes for patient-reported outcomes were greater than for physician-reported outcomes, and also greater than ACR(20) in three of five anakinra cohorts. Across the RCTs, placebo responses were greater with physician-reported than with patient-reported outcomes. In the two studies evaluating patients with longer-standing disease, differences between pooled effect sizes for patient-reported and physician-reported outcomes were even more pronounced.

Conclusions: In three pivotal RCTs, active treatment with anakinra resulted in greater improvements in patient-reported than physician-reported outcomes compared with placebo. These observations confirm those previously reported from RCTs evaluating conventional DMARDs, demonstrating better discrimination of treatment effect with patient-reported outcomes.