PATIENT-REPORTED EXPERIENCES OF TENOSYNOVIAL GIANT CELL TUMOR (TGCT) SYMPTOMS IN THE MANEUVER PHASE 3 TRIAL: INSIGHTS FROM EXIT INTERVIEWS
Author(s)
Vinod Ravi, MD1, Andrea Phillips Beyer, PhD2, Marcis Bajars, MD3, Qingping Zou, PhD4, Amy Clark, PhD5, Savita Bakhshi Anand, PhD5, Carla Dias Barbosa, MSc6, Hans Gelderblom, MD7.
1The University of Texas MD Anderson Cancer Center, Houston, TX, USA, 2Ares Trading S.A., an affiliate of Merck KGaA, Darmstadt, Germany, Eysins, Switzerland, 3Merck Healthcare KGaA, Darmstadt, Germany, 4Abbisko Therapeutics Co. Ltd., Shanghai, China, 5PPD Evidera Patient-Centered Research, Thermo Fisher Scientific, London, United Kingdom, 6PPD Evidera Patient-Centered Research, Thermo Fisher Scientific, Ivry-sur-Seine, France, 7Leiden University Medical Center, Leiden, Netherlands.
1The University of Texas MD Anderson Cancer Center, Houston, TX, USA, 2Ares Trading S.A., an affiliate of Merck KGaA, Darmstadt, Germany, Eysins, Switzerland, 3Merck Healthcare KGaA, Darmstadt, Germany, 4Abbisko Therapeutics Co. Ltd., Shanghai, China, 5PPD Evidera Patient-Centered Research, Thermo Fisher Scientific, London, United Kingdom, 6PPD Evidera Patient-Centered Research, Thermo Fisher Scientific, Ivry-sur-Seine, France, 7Leiden University Medical Center, Leiden, Netherlands.
OBJECTIVES: Patients with TGCT can experience swelling and pain in the affected joint. We characterized baseline symptoms in patients with TGCT in the global phase 3 MANEUVER trial (NCT05804045) and explored changes after receiving placebo or pimicotinib.
METHODS: Semi-structured ~90-minute telephone/web interviews were conducted with patients with TGCT from 6 countries ≤4 weeks after the 24-week double-blind, placebo-controlled phase of the MANEUVER trial. Interview transcripts were analyzed following thematic and content analysis approaches. Saturation of concepts was assessed on symptoms and impacts experienced at baseline; quantitative data were descriptively analyzed. Severity scales were 0=non-existent to 10=extremely severe/as bad as imaginable.
RESULTS: Twenty patients (pimicotinib: n=12; placebo: n=8) were interviewed; 65.0% were female, mean age was 43.0 (SD 13.5) years, and 50.0% from the US or Netherlands. Joints affected by TGCT included the knee (n=13), foot (n=2), hip (n=2), ankle (n=2), and wrist (n=1). Saturation was reached for all symptom and impact categories. At baseline, most patients reported pain (95.0%; n=19), stiffness/tightness (90.0%; n=18), restriction/reduction in movement (90.0%; n=18), swelling (85.0%; n=17), and weakness (65.0%; n=13) of their affected joint. Most symptoms were experienced across all TGCT-affected anatomical locations, excluding restriction/reduction of movement for foot, pain and weakness for wrist, and swelling for hip. Nearly all patients treated with pimicotinib reported improvement in their TGCT symptoms: 90.9% (n=10/11) had improvement in swelling, and severity reduced from 6.5 to 2.6 (placebo, 5.8 to 4.2); 90.9% (n=10/11) had improvement in pain, and severity reduced from 6.8 to 3.9 (placebo, 5.0 to 4.6). Symptom worsening was only reported with placebo.
CONCLUSIONS: This study confirmed substantial symptom burden experienced by patients with TGCT, regardless of affected joint. Swelling and pain were common, which affected patient’s wellbeing and quality of life. These results further support use of pimicotinib for clinically meaningful improvements in symptoms and physical function, key patient-relevant outcomes.
METHODS: Semi-structured ~90-minute telephone/web interviews were conducted with patients with TGCT from 6 countries ≤4 weeks after the 24-week double-blind, placebo-controlled phase of the MANEUVER trial. Interview transcripts were analyzed following thematic and content analysis approaches. Saturation of concepts was assessed on symptoms and impacts experienced at baseline; quantitative data were descriptively analyzed. Severity scales were 0=non-existent to 10=extremely severe/as bad as imaginable.
RESULTS: Twenty patients (pimicotinib: n=12; placebo: n=8) were interviewed; 65.0% were female, mean age was 43.0 (SD 13.5) years, and 50.0% from the US or Netherlands. Joints affected by TGCT included the knee (n=13), foot (n=2), hip (n=2), ankle (n=2), and wrist (n=1). Saturation was reached for all symptom and impact categories. At baseline, most patients reported pain (95.0%; n=19), stiffness/tightness (90.0%; n=18), restriction/reduction in movement (90.0%; n=18), swelling (85.0%; n=17), and weakness (65.0%; n=13) of their affected joint. Most symptoms were experienced across all TGCT-affected anatomical locations, excluding restriction/reduction of movement for foot, pain and weakness for wrist, and swelling for hip. Nearly all patients treated with pimicotinib reported improvement in their TGCT symptoms: 90.9% (n=10/11) had improvement in swelling, and severity reduced from 6.5 to 2.6 (placebo, 5.8 to 4.2); 90.9% (n=10/11) had improvement in pain, and severity reduced from 6.8 to 3.9 (placebo, 5.0 to 4.6). Symptom worsening was only reported with placebo.
CONCLUSIONS: This study confirmed substantial symptom burden experienced by patients with TGCT, regardless of affected joint. Swelling and pain were common, which affected patient’s wellbeing and quality of life. These results further support use of pimicotinib for clinically meaningful improvements in symptoms and physical function, key patient-relevant outcomes.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
PCR75
Topic
Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
Oncology, Rare & Orphan Diseases