Background In RA, there is increasing interest in “unusual” patient-reported outcomes (PROs), over and above pain, function and fatigue; namely, quality of sleep, well-being and ability to cope . Although these outcomes are important from the patient’s point of view, are they useful as outcome measures and in particular, are they reliable, and responsive, i.e., do they improve when disease improves with treatment?
Objectives To assess the reliability and responsiveness after introduction of etanercept, of different PROs.
Methods Study: open-label 12-week trial of etanercept in RA . PROs of interest: numeric rating scales (NRS) for sleep, physical and emotional well-being, and coping . Comparison outcomes: “standard” PROs, namely, pain NRS, patient global assessment of disease activity (PGA), modified-HAQ (mHAQ) and fatigue NRS. Reliability: assessed between screening and baseline visits by intra-class correlation (ICC). Responsiveness: assessed between baseline and 12 weeks, by standardized response means (SRM). Only patients with at least 1 etanercept injection were analysed.
Results In all, 108 of the 120 screened patients received at least one etanercept injection: mean age, 54 (SD 13) yrs, mean disease duration 8 (SD 7), 75% were women, all were biologic naïve. Disease was active: mean DAS28 5.5 (SD 0.8), mean mHAQ 0.9 (SD 0.5), mean PGA, 6.5 (SD 1.9). At 12 weeks, mean DAS28 was 3.4 (SD 1.2).
Reliability was highest for sleep, and lowest for PGA and pain, whereas responsiveness was highest for pain and PGA, and lowest for mHAQ (Table).
Conclusions NRS assessing sleep, well-being and coping were found to be generally as reliable and as responsive as “usual” PROs in RA. mHAQ showed poor responsiveness possibly due to floor effects. Sleep and coping were the least responsive “unusual” PROs in this trial, indicating these domains of health may be less accessible to biologic treatment. When assessing the patient’s perspective on treatment, it is feasible and valid to measure “unusual” domains of health by NRS.
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Disclosure of Interest L. Gossec Consultant for: Abbott, BMS, Pfizer, Roche, UCB, Y. Brault Employee of: Pfizer, I. Logeart Employee of: Pfizer, M. Dougados Consultant for: Abbott, BMS, Pfizer, Roche, UCB,