Communicating Change on Multi-item Patient-Reported Outcome Measures: What Were the Meaningful Changes in Physical Functioning Experienced by Patients on Vimseltinib in the MOTION Trial.

BACKGROUND AND OBJECTIVE: Patients with tenosynovial giant cell tumor experience many impacts on their physical functioning associated with pain, stiffness, and limited range of motion. Clinical trials often use clinical outcome assessments to establish meaningful change in treatment; although, conceptualizing improvement on a multi-item measure can be challenging. The goal of this analysis was to explicitly characterize what the significant and meaningful improvements in physical functioning were among those who were considered physical functioning responders. METHODS: A mixed-method analysis was conducted using data from the MOTION phase III trial (NCT05059262) and in-trial exit interviews conducted prior to unblinding. A heat map was developed to characterize individual patient- and item-level data improvements in physical functioning among those on vimseltinib considered responders (change of ≥ 3 points on total score) on the Patient-Reported Outcomes Measurement Information System-Physical Functioning short form (PROMIS-PF®). RESULTS: A total of 36 participants (n = 36/83, 43%) in the vimseltinib treatment group were PROMIS-PF responders. Patients in this group had more than a one-category improvement in 12 of the 15 items and 64-100% of the participants improved on each PROMIS-PF item. Exit interview participants described meaningful improvements in physical functioning from the patient perspective, including better mobility, greater independence, and the ability to resume work, daily activities, and recreational activities. CONCLUSIONS: Patients with tenosynovial giant cell tumor showed meaningful improvement in physical functioning on vimseltinib. Item- and individual patient-level characterization of these improvements can be utilized in communication for treatment decision making between clinicians and patients. This method may be utilized in other measures and indications to generate information when considering treatment options.