Providing meaningful interpretation of performance outcome measures by co-calibration with patient-reported outcomes through the Rasch model: illustration with multiple sclerosis measures

JOURNAL OF BIOPHARMACEUTICAL STATISTICS(2023)

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摘要
Performance outcome (PerfO) measures are based on tasks performed by patients in a controlled environment, making their meaningful interpretation challenging to establish. Co-calibrating PerfO and patient-reported outcome (PRO) measures of the same target concept allow for interpretation of the PerfO with the item content of the PRO. The Rasch model applied to the discretized PerfO measure together with the PRO items allows expressing parameters related to the PerfO measure in the PRO metric for it to be linked to the PRO responses. We applied this approach to two PerfO measures used in multiple sclerosis (MS) for walking and manual ability: the Timed 25-Foot Walk (T25FW) and the 9-Hole Peg Test (9HPT). To determine meaningful interpretation of these two PerfO measures, they were co-calibrated with two PRO measures of closely related concepts, the MS walking scale - 12 items (MSWS-12) and the ABILHAND, using the data of 2,043 subjects from five global clinical trials in MS. The probabilistic relationships between the PerfO measures and the PRO metrics were used to express the response pattern to the PRO items as a function of the unit of the PerfOs. This example illustrates the promises of the co-calibration approach for the interpretation of PerfO measures but also highlights the challenges associated with it, mostly related to the quality of the PRO metric in terms of coverage of the targeted concept. Co-calibration with PRO measures could also be an adequate solution for interpretation of digital sensor measures whose meaningfulness is also often questioned.
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关键词
Clinical outcome assessments,performance outcome measure,meaningfulness,Rasch model,multiple sclerosis
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