Characterizing Improvements in Physical Functioning with PROMIS-PF and In-Trial Exit Interviews: A Mixed-Methods Analysis to Understand the Meaningful Changes Experienced by TGCT Patients on Vimseltinib in the MOTION Trial
Author(s)
Heather Gelhorn, PhD1, Katelyn N. Cutts, MS1, Tsion Fikre, MPH2, Yipin Han, MHS2, Christopher Tait, PhD3, Nicholas Zeringo, PhD3, Amanda Saunders, DO3, Brooke Harrow, PhD3.
1PPD Evidera Patient-Centered Research, Thermo Fisher Scientific, Wilmington, NC, USA, 2Thermo Fisher Scientific, Wilmington, NC, USA, 3Deciphera Pharmaceuticals, LLC, Waltham, MA, USA.
1PPD Evidera Patient-Centered Research, Thermo Fisher Scientific, Wilmington, NC, USA, 2Thermo Fisher Scientific, Wilmington, NC, USA, 3Deciphera Pharmaceuticals, LLC, Waltham, MA, USA.
Presentation Documents
OBJECTIVES: Tenosynovial giant cell tumor (TGCT) is associated with joint destruction, pain, stiffness, limited range of motion and impact on physical functioning (PF). In clinical trials, mean change scores on many clinical outcome assessments (COAs) often establish statistically significant changes; however, it is challenging to characterize improvements based on change scores from multi-item measures. This study explicitly characterized the improvements in physical functioning that responding TGCT patients treated with vimseltinib experienced in the MOTION trial.
METHODS: Mixed-methods were used to characterize the improvements in physical functioning among patients on vimseltinib who met/exceeded the meaningful change threshold (≥3-points) on PROMIS-PF. Prior to unblinding, qualitative in-trial exit interviews were conducted in the MOTION Phase 3 trial (NCT05059262) of vimseltinib in TGCT. Patients described the changes they experienced in PF during the trial. Within the trial, patients completed a custom Patient-Reported Outcomes Measurement Information System - Physical Function (PROMIS-PF) form and Patient Global Impression of Change (PGIC) items.
RESULTS: Data were analyzed for the 36 PROMIS-PF responders out of n=83 in the vimseltinib treatment group. During the exit interviews, many patients reported improvements in walking, climbing stairs, squatting, kneeling, and housework. Change scores for each individual PROMIS-PF item are presented including the frequency and magnitude of the observed improvements for each.
CONCLUSIONS: Many TGCT patients on vimseltinib experienced clinically meaningful changes in physical functioning, and through the approach presented here, the nature and extent of these improvements can now be more clearly communicated with physicians and patients. It is often challenging to characterize the specific improvements contributing to total scores that patients have experienced using multi-item measures such as the PROMIS-PF. The method presented here to characterize changes on multi-item and/or concept COA measures may be applicable across disease areas and provides clinically useful information for physicians and patients considering effective treatment options.
METHODS: Mixed-methods were used to characterize the improvements in physical functioning among patients on vimseltinib who met/exceeded the meaningful change threshold (≥3-points) on PROMIS-PF. Prior to unblinding, qualitative in-trial exit interviews were conducted in the MOTION Phase 3 trial (NCT05059262) of vimseltinib in TGCT. Patients described the changes they experienced in PF during the trial. Within the trial, patients completed a custom Patient-Reported Outcomes Measurement Information System - Physical Function (PROMIS-PF) form and Patient Global Impression of Change (PGIC) items.
RESULTS: Data were analyzed for the 36 PROMIS-PF responders out of n=83 in the vimseltinib treatment group. During the exit interviews, many patients reported improvements in walking, climbing stairs, squatting, kneeling, and housework. Change scores for each individual PROMIS-PF item are presented including the frequency and magnitude of the observed improvements for each.
CONCLUSIONS: Many TGCT patients on vimseltinib experienced clinically meaningful changes in physical functioning, and through the approach presented here, the nature and extent of these improvements can now be more clearly communicated with physicians and patients. It is often challenging to characterize the specific improvements contributing to total scores that patients have experienced using multi-item measures such as the PROMIS-PF. The method presented here to characterize changes on multi-item and/or concept COA measures may be applicable across disease areas and provides clinically useful information for physicians and patients considering effective treatment options.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
PCR222
Topic
Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
SDC: Oncology, SDC: Rare & Orphan Diseases