IDENTIFICATION AND VALIDATION OF OUTCOMES IMPORTANT TO BOTH PATIENTS WITH TENOSYNOVIAL GIANT CELL TUMOR (TGCT) AND SPECIALIST CLINICIANS: A THREE-ROUND DELPHI METHODOLOGY STUDY
Author(s)
Andrea Phillips Beyer, PhD1, João Bana e Costa, BSc2, Louise Young, PhD3, Marcis Bajars, MD4, Vishal Ghori, MD1.
1Ares Trading S.A., an affiliate of Merck KGaA, Darmstadt, Germany, Eysins, Switzerland, 2Decision Eyes, Lisbon, Portugal, 3Merck Healthcare KGaA, London, United Kingdom, 4Merck Healthcare KGaA, Darmstadt, Germany.
1Ares Trading S.A., an affiliate of Merck KGaA, Darmstadt, Germany, Eysins, Switzerland, 2Decision Eyes, Lisbon, Portugal, 3Merck Healthcare KGaA, London, United Kingdom, 4Merck Healthcare KGaA, Darmstadt, Germany.
OBJECTIVES: TGCT is a rare, locally aggressive soft-tissue tumor that can significantly impact quality of life and physical function. Using a three-round Delphi methodology, we identified and validated TGCT outcomes important to patients and clinicians for research standardization.
METHODS: Round 1 was a qualitative open-ended instrument completed by 13 participants (9 patients, 4 clinicians), generating 26 outcomes for rating. Round 2 asked 12 continuing participants (8 patients, 4 clinicians) to rate each outcome on a 5-point Likert scale. Round 3 was a structured confirmation round: participants reviewed their Round 2 ratings alongside the group distribution and anonymous peer comments and were asked to confirm or revise. Consensus required qualified majority agreement (SA+A [strongly agree + agree]≥75%), a positive central tendency (median≥4), and low spread (IQR≤1).
RESULTS: Round 3 confirmed high stability: 92.3% (288/312) of individual ratings were unchanged after group review; all 26 items were stable (Wilcoxon Signed-Rank test). Nine items achieved very high consensus (SA+A=92-100%, IQR=0): pain, stiffness, swelling, reduced range of motion, fatigue, activities of daily living, mobility, symptom progression, and emotional and psychosocial impact. Eight items achieved high consensus (SA+A≥75%, median≥4, IQR≤1): secondary osteoarthritis, insomnia/sleep disruption, liver blood tests elevated, peripheral edema, falls leading to injury, weight gain, neuropathic pain, and clinician-measured joint movement. Nine items did not meet the full consensus criteria: nausea, facial edema, hypertension, pruritus, skin rash, hair and skin lightening, joint popping/clicking, photosensitivity, and blood clotting problems. Blood clotting problems had the lowest mean (Round 3: 2.92), reflecting no reported thrombosis linked to TGCT CSF-1R inhibitors.
CONCLUSIONS: This study validated 26 patient-relevant and clinically meaningful TGCT outcomes, with nine achieving very high consensus, representing core disease burden and treatment impact attributes endorsed by patients and clinicians. The structural divergence between patient and clinician perspectives is a consistent and meaningful feature to guide future outcome prioritization processes.
METHODS: Round 1 was a qualitative open-ended instrument completed by 13 participants (9 patients, 4 clinicians), generating 26 outcomes for rating. Round 2 asked 12 continuing participants (8 patients, 4 clinicians) to rate each outcome on a 5-point Likert scale. Round 3 was a structured confirmation round: participants reviewed their Round 2 ratings alongside the group distribution and anonymous peer comments and were asked to confirm or revise. Consensus required qualified majority agreement (SA+A [strongly agree + agree]≥75%), a positive central tendency (median≥4), and low spread (IQR≤1).
RESULTS: Round 3 confirmed high stability: 92.3% (288/312) of individual ratings were unchanged after group review; all 26 items were stable (Wilcoxon Signed-Rank test). Nine items achieved very high consensus (SA+A=92-100%, IQR=0): pain, stiffness, swelling, reduced range of motion, fatigue, activities of daily living, mobility, symptom progression, and emotional and psychosocial impact. Eight items achieved high consensus (SA+A≥75%, median≥4, IQR≤1): secondary osteoarthritis, insomnia/sleep disruption, liver blood tests elevated, peripheral edema, falls leading to injury, weight gain, neuropathic pain, and clinician-measured joint movement. Nine items did not meet the full consensus criteria: nausea, facial edema, hypertension, pruritus, skin rash, hair and skin lightening, joint popping/clicking, photosensitivity, and blood clotting problems. Blood clotting problems had the lowest mean (Round 3: 2.92), reflecting no reported thrombosis linked to TGCT CSF-1R inhibitors.
CONCLUSIONS: This study validated 26 patient-relevant and clinically meaningful TGCT outcomes, with nine achieving very high consensus, representing core disease burden and treatment impact attributes endorsed by patients and clinicians. The structural divergence between patient and clinician perspectives is a consistent and meaningful feature to guide future outcome prioritization processes.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
PCR127
Topic
Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
Oncology, Rare & Orphan Diseases