CHANGES IN PAIN AND STIFFNESS EXPERIENCED BY PATIENTS WITH TENOSYNOVIAL GIANT CELL TUMOR (TGCT) IN THE MANEUVER PHASE 3 TRIAL: INSIGHTS FROM EXIT INTERVIEWS
Author(s)
Vinod Ravi, MD1, Paul Kamudoni, PhD2, Andrea Phillips Beyer, PhD2, Niki Karachaliou, MD, PhD2, Amy Clark, PhD3, Agkreta Leventi, MSc3, Savita Bakhshi Anand, PhD3, Carla Dias Barbosa, MSc4, Hans Gelderblom, MD5.
1The University of Texas MD Anderson Cancer Center, Houston, TX, USA, 2Merck Healthcare KGaA, Darmstadt, Germany, 3PPD Evidera Patient-Centered Research, Thermo Fisher Scientific, London, United Kingdom, 4PPD Evidera Patient-Centered Research, Thermo Fisher Scientific, Ivry-sur-Seine, France, 5Leiden University Medical Center, Leiden, Netherlands.
1The University of Texas MD Anderson Cancer Center, Houston, TX, USA, 2Merck Healthcare KGaA, Darmstadt, Germany, 3PPD Evidera Patient-Centered Research, Thermo Fisher Scientific, London, United Kingdom, 4PPD Evidera Patient-Centered Research, Thermo Fisher Scientific, Ivry-sur-Seine, France, 5Leiden University Medical Center, Leiden, Netherlands.
OBJECTIVES: TGCT causes joint destruction, pain, stiffness, and reduced physical functioning. We characterized changes in patient-reported pain and stiffness in pimicotinib- or placebo-treated patients with TGCT in the global Phase 3 MANEUVER trial (NCT05804045) and established clinically meaningful thresholds for Worst Pain and Stiffness numerical rating scale (NRS) scores.
METHODS: Semi-structured interviews (~90 minutes, telephone/web assisted) were conducted across 6 countries ≤4 weeks after the 24-week, double-blind, placebo-controlled phase of MANEUVER. Qualitative data from verbatim transcripts were analyzed thematically and quantitative data were analyzed descriptively.
RESULTS: Overall, 20 patients were interviewed (pimicotinib: n=12; placebo: n=8), 65.0% were female, most resided in the US (25.0%) or Netherlands (25.0%); mean (standard deviation) age was 43.0 (13.5) years. Most patients (95.0%; n=19) had a lower extremity affected by TGCT. At baseline, most patients reported pain (95.0%; n=19) or stiffness (90.0%; n=18). At interview, 10/11 pimicotinib-treated patients and 2/6 placebo-treated patients reported reduced pain. Worst Pain NRS was relevant to 75.0% of patients. Of 19 patients responding to meaningful improvement thresholds for Worst Pain NRS, most found a one- (42.1%) or two-point (36.8%) improvement meaningful. Of 14 patients reporting on worsening, 71.4% found a one-point worsening meaningful. At interview, 10/11 pimicotinib-treated patients and 3/5 placebo-treated patients reported reduced stiffness. Most patients found Worst Stiffness NRS relevant (75.0%) to them. Of 19 patients reporting on Worst Stiffness NRS improvements, 47.4% found a two-point improvement meaningful, followed by a one-point improvement (31.6%). Of 13 patients reporting on worsening, 58.3% found a one-point worsening meaningful.
CONCLUSIONS: This study highlights the baseline impact of pain and stiffness on patients with TGCT, with most patients treated with pimicotinib reporting improvements at exit interview. Results support the content validity of Worst Pain and Stiffness NRS and suggest a two-point change in both scales as clinically meaningful for improvement and worsening.
METHODS: Semi-structured interviews (~90 minutes, telephone/web assisted) were conducted across 6 countries ≤4 weeks after the 24-week, double-blind, placebo-controlled phase of MANEUVER. Qualitative data from verbatim transcripts were analyzed thematically and quantitative data were analyzed descriptively.
RESULTS: Overall, 20 patients were interviewed (pimicotinib: n=12; placebo: n=8), 65.0% were female, most resided in the US (25.0%) or Netherlands (25.0%); mean (standard deviation) age was 43.0 (13.5) years. Most patients (95.0%; n=19) had a lower extremity affected by TGCT. At baseline, most patients reported pain (95.0%; n=19) or stiffness (90.0%; n=18). At interview, 10/11 pimicotinib-treated patients and 2/6 placebo-treated patients reported reduced pain. Worst Pain NRS was relevant to 75.0% of patients. Of 19 patients responding to meaningful improvement thresholds for Worst Pain NRS, most found a one- (42.1%) or two-point (36.8%) improvement meaningful. Of 14 patients reporting on worsening, 71.4% found a one-point worsening meaningful. At interview, 10/11 pimicotinib-treated patients and 3/5 placebo-treated patients reported reduced stiffness. Most patients found Worst Stiffness NRS relevant (75.0%) to them. Of 19 patients reporting on Worst Stiffness NRS improvements, 47.4% found a two-point improvement meaningful, followed by a one-point improvement (31.6%). Of 13 patients reporting on worsening, 58.3% found a one-point worsening meaningful.
CONCLUSIONS: This study highlights the baseline impact of pain and stiffness on patients with TGCT, with most patients treated with pimicotinib reporting improvements at exit interview. Results support the content validity of Worst Pain and Stiffness NRS and suggest a two-point change in both scales as clinically meaningful for improvement and worsening.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
PCR18
Topic
Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
SDC: Oncology, SDC: Rare & Orphan Diseases