Real-world utilization pattern of pexidartinib in patients with tenosynovial giant cell tumor (TGCT).

• DOI: 10.1200/jco.2023.41.16_suppl.e18792
• Publication Date: 2023-06-04T16:03:39Z
• Authors: Feng Lin; Jenny Tse; Nazneen Fatima Sh...; Elizabeth J Wang; Aimee Near; Jackie Kwong; William D. Tap
• Keywords: Discontinuation

e18792 Background: In August 2019, pexidartinib became the first and only systemic therapy approved in US for treatment of symptomatic TGCT associated with severe morbidity or functional limitations not amenable to improvement surgery. As is a rare condition, real-world utilization data are limited greatly needed inform clinical use. Methods: Adults (≥18 years) ≥1 claim from 8/2/2019 - 1/1/2022 identified IQVIA Longitudinal Prescription Claims (LRx) linked Medical (Dx) databases were analyzed. Patient characteristics 6 months before (index) summarized. Pexidartinib dosing, interruption, duration assessed post-index. Discontinuation was defined as gap ≥90 days. Results: Of 167 patients treated (median age, 45 years; 59.9% female), most common comorbidities osteoarthritis (10.8%), hypertension (8.4%), obesity (7.8%); 3 (1.8%) had mild liver disease none moderate-to-severe pre-index. Median starting dose 800 mg/day (Table). Over median follow-up 17.9 months, (IQR) 622 (442-750) mg/day. Dose reduction observed 57 (34.1%) increase 14 (8.4%) patients. Probability discontinuation 24.0%, 38.3% 53.7% at 3-, 6- 12-month follow-up, respectively. (95% confidence interval [CI]) time 10.1 (7.1, 13.0) months. Fourteen restarted after discontinuation. Overall, CI) on 14.2 (10.1, 18.4) when restart considered. Rate joint surgery low (7.2%, n = 12) index 5.4 (4.2-12.5) last 2.7 (1.0-8.2) Three Conclusions: Compared ENLIVEN, similar demographic characteristics. Real-world average daily lower than full recommended prescribing information. rate higher but 12 Findings could strategy practice future research TGCT. [Table: see text]