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

Résumé fourni par la source

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.

Ce résumé expose les affirmations des auteurs. BNTIC ne l’interprète pas comme une validation indépendante des résultats.