Health-Related Quality of Life Measured by EQ-5D Utility Among Patients With TGCT: A Trial-Based Analysis of EQ-5D Data From Patients in the MOTION Trial
Author(s)
Ágnes Benedict, MA, MSc1, Balazs Dobi, PhD2, Nicholas Zeringo, PhD3, Paul Edward Hewitt, MSc4, Ulrike Beckert, PharmD, PhD5, Venediktos Kapetanakis, PhD6, Brooke Harrow, PhD3.
1Senior Research Leader, PPD Evidera, Vienna, Austria, 2PPD Evidera Health Economics & Market Access, Budapest, Hungary, 3Deciphera Pharmaceuticals, Waltham, MA, USA, 4Deciphera Pharmaceuticals, London, United Kingdom, 5Deciphera Pharmaceuticals, München, Germany, 6Thermo Fisher Scientific, London, United Kingdom.
1Senior Research Leader, PPD Evidera, Vienna, Austria, 2PPD Evidera Health Economics & Market Access, Budapest, Hungary, 3Deciphera Pharmaceuticals, Waltham, MA, USA, 4Deciphera Pharmaceuticals, London, United Kingdom, 5Deciphera Pharmaceuticals, München, Germany, 6Thermo Fisher Scientific, London, United Kingdom.
OBJECTIVES: Tenosynovial giant cell tumor (TGCT) is a locally aggressive neoplasm that involves the synovium, bursae, or tendon sheath. Tumor location varies and is often associated with joint destruction, pain, stiffness, and limited range of motion. This study characterizes HRQoL among TGCT patients not amenable to surgery, in terms of EQ-5D utility values based on the MOTION Phase 3 trial (NCT05059262) of vimseltinib.
METHODS: EQ-5D-5L values collected from 123 patients during a year of follow-up were mapped to age- and sex-adjusted EQ-5D-3L utility index values using published mapping algorithm. EQ-5D utility scores using UK tariffs were analysed using a mixed model for repeated measures (MMRM). Predictors tested included time-varying symptom measures (mean Brief Pain Inventory (BPI) worst pain numeric rating scale (NRS), stiffness, range of motion and PROMIS-PF) used in clinical practice to characterize disease activity, age, gender and disease type utilities.
RESULTS: Mean baseline utility score was 0.53 (SD=0.25) versus 0.88 for age- and gender-adjusted UK general population utility. At the end of the double-blind period (25 weeks), the mean EQ-5D utility scores were 0.72 (SD=0.17) on vimseltinib versus 0.59 (SD=0.24) on placebo. The MMRM, including treatment as the only covariate, estimated 0.089 (SE=0.035) improvement for vimseltinib. The final multivariable model included baseline EQ-5D was linear in Worst Pain NRS and non-linear in PROMIS-PF.
CONCLUSIONS: TGCT patients not amenable for surgery in the MOTION trial had poor HRQoL at baseline (0.53) comparable to other pain-ridden diseases, such as osteoarthritis or multiple sclerosis. Based on MOTION, vimseltinib significantly improves PF and worst pain NRS over time, leading to large gains in HRQoL. These results fill a gap in describing HRQoL for TGCT patients not amenable for surgery and will support modeling the changes expected in EQ-5D due to changes in key symptoms of TGCT for health technology assessment.
METHODS: EQ-5D-5L values collected from 123 patients during a year of follow-up were mapped to age- and sex-adjusted EQ-5D-3L utility index values using published mapping algorithm. EQ-5D utility scores using UK tariffs were analysed using a mixed model for repeated measures (MMRM). Predictors tested included time-varying symptom measures (mean Brief Pain Inventory (BPI) worst pain numeric rating scale (NRS), stiffness, range of motion and PROMIS-PF) used in clinical practice to characterize disease activity, age, gender and disease type utilities.
RESULTS: Mean baseline utility score was 0.53 (SD=0.25) versus 0.88 for age- and gender-adjusted UK general population utility. At the end of the double-blind period (25 weeks), the mean EQ-5D utility scores were 0.72 (SD=0.17) on vimseltinib versus 0.59 (SD=0.24) on placebo. The MMRM, including treatment as the only covariate, estimated 0.089 (SE=0.035) improvement for vimseltinib. The final multivariable model included baseline EQ-5D was linear in Worst Pain NRS and non-linear in PROMIS-PF.
CONCLUSIONS: TGCT patients not amenable for surgery in the MOTION trial had poor HRQoL at baseline (0.53) comparable to other pain-ridden diseases, such as osteoarthritis or multiple sclerosis. Based on MOTION, vimseltinib significantly improves PF and worst pain NRS over time, leading to large gains in HRQoL. These results fill a gap in describing HRQoL for TGCT patients not amenable for surgery and will support modeling the changes expected in EQ-5D due to changes in key symptoms of TGCT for health technology assessment.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
PCR107
Topic
Clinical Outcomes, Health Technology Assessment, Patient-Centered Research
Topic Subcategory
Health State Utilities
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Oncology