Abstract
BACKGROUND: Tenosynovial giant-cell tumor (TGCT) is a rare condition characterized by pain, stiffness, and limited range of motion. Pexidartinib is the first regulatory-approved systemic therapy for adult patients with symptomatic TGCT not amenable for surgery. This study evaluated symptom improvement in patients who newly initiated pexidartinib. METHODS: A longitudinal, observational study collected data on patient-reported outcomes, demographics, disease history, pexidartinib dosing, and clinicians' perspective. RESULTS: Identified from the TURALIO Risk Evaluation and Mitigation Strategy (REMS) program, 80 patients who newly initiated pexidartinib between April and October 2022 received a study invitation, and 14 completed the baseline assessment; mean (SD) age was 39.8 (13.60) years, 50.0% were female, and most common tumor sites were in the lower extremities (71.4%). By the end of study follow-up (May 2023), 11 patients completed some PRO assessments at follow-up, and median time on pexidartinib was 7.5 months. Results showed a trend of reduction in pain and stiffness and improvement in physical functioning. Overall, 80% (8/10) endorsed moderately high treatment satisfaction with pexidartinib treatment. CONCLUSIONS: Findings from this study provided real-world evidence of an association between pexidartinib initialization and symptom improvements among patients with symptomatic TGCTs from the patients' and clinicians' perspective.