ArticleThe oncologist2025
Patient-reported continued benefits of pexidartinib for tenosynovial giant cell tumor based on a real-world study in the United States.
Article in The oncologist, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers, 1 of them a synthesis that pooled it.
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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
2 citing papers in PubMed, 1 synthesis or guideline pooled it.
- The impact of CSF1R inhibitor-mediated microglial depletion in rodent models of Alzheimer's and Parkinson's disease: a systematic review and meta-analysis.Frontiers in aging neuroscience · 2026Pooled it
- Assessing the effectiveness of the TURALIO (pexidartinib) risk evaluation and mitigation strategy program: patient knowledge, attitudes, and behavior survey (2020-2024).Patient related outcome measures · 2026Article
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Authors and funding
6 authors.
Funding
Abstract
backgroundPexidartinib (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.
methodsA 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).
resultsOf 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).
conclusionsFindings 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.
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