Patient-reported continued benefits of pexidartinib for tenosynovial giant cell tumor based on a real-world study in the United States Journal Article


Authors: Dai, D.; Pan, I.; Freivogel, K.; Ye, X.; Tecson, K.; Tap, W.
Article Title: Patient-reported continued benefits of pexidartinib for tenosynovial giant cell tumor based on a real-world study in the United States
Abstract: Background: Pexidartinib (Turalio) is the only systemic therapy approved by the United States Food and Drug Administration for the treatment of patients with tenosynovial giant cell tumors (TGCT ) based on clinical benefits demonstrated in the Phase III ENLIVEN trial. The present study assessed longitudinal patient-reported outcomes of patients treated with pexidartinib for TGCT. Methods: A longitudinal study was conducted in adult patients who received pexidartinib via the Risk Evaluation and Mitigation Strategy in the United States. Two web-based surveys containing patient-reported outcome questionnaires were administered to eligible patients (at least 18 years of age, had taken at least 1 dose of pexidartinib before baseline survey and still on treatment with pexidartinib at the time of follow-up survey, had not participated in any clinical trials for pexidartinib, and could complete questionnaires in English). The first assessment was initiated in 2021 (baseline), and the second was completed in 2022-2023 (follow-up). Results: Of 83 eligible patients who completed baseline assessment, 45 were eligible for follow-up. Thirty-one of whom consented and 28 completed the assessment. At the time of the follow-up survey, the mean (range) treatment duration was 18.5 (10.6-36.2) months, mean (SD) age was 41.9 (13.70) years, and 67.7% of the patients were female. The most common tumor sites were in the knee (67.7%) and ankle (16.1%). At follow-up, over 85% of patients reported symptom improvement since initiation of treatment with pexidartinib, and nearly 70% of patients reported symptoms to be “Very much improved” or “Much improved.” From baseline to follow-up assessment, changes in patient-reported measures on physical function, pain, and stiffness were not statistically significant (P > .05). Conclusions: Findings from this longitudinal study showed continued benefit of pexidartinib in overall symptom improvement from patients’ perspectives after an additional year of continued use among patients with symptomatic TGCT. © The Author(s) 2025. Published by Oxford University Press.
Keywords: adult; clinical article; treatment response; aged; middle aged; major clinical study; treatment duration; united states; outcome assessment; follow up; pain; health survey; pathology; information processing; questionnaire; longitudinal studies; family history; diabetes mellitus; satisfaction; drug dose; patient reported outcome measures; drug therapy; pyrroles; hypertriglyceridemia; physical performance; gallbladder disease; geography; longitudinal study; rigidity; pyrrole derivative; geographic distribution; demographics; patient-reported outcome; tenosynovial giant cell tumor; humans; human; male; female; article; hepatitis virus; aminopyridines; numeric rating scale; aminopyridine derivative; surveys and questionnaires; primary tumor site; pexidartinib; giant cell tumor of tendon sheath; risk evaluation and mitigation strategy; symptom change; treatment satisfaction questionnaire for medication
Journal Title: The Oncologist
Volume: 30
Issue: 3
ISSN: 1083-7159
Publisher: Oxford University Press  
Date Published: 2025-03-01
Start Page: oyaf028
Language: English
DOI: 10.1093/oncolo/oyaf028
PUBMED: 40163687
PROVIDER: scopus
PMCID: PMC11957242
DOI/URL:
Notes: Source: Scopus
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  1. William Douglas Tap
    375 Tap