Long‐term outcomes of pexidartinib in tenosynovial giant cell tumors

作者：Hans Gelderblom, Andrew J. Wagner, William D. Tap, Emanuela Palmerini, Zev A. Wainberg, Jayesh Desai, John H. Healey, Michiel A. J. van de Sande, Nicholas M. Bernthal, Eric L. Staals, Charles Peterfy, Anna Maria Frezza, Henry H. Hsu, Qiang Wang, Dale E. Shuster, Silvia Stacchiotti · 发表于：Cancer · 年份：2020 · DOI：10.1002/cncr.33312 · 被引用次数：72 · 研究领域：Musculoskeletal synovial abnormalities and treatments、Tendon Structure and Treatment、Shoulder Injury and Treatment

BACKGROUND: The objective of this study was to report on the long-term effects of pexidartinib on tenosynovial giant cell tumor (TGCT). METHODS: This was a pooled analysis encompassing 3 pexidartinib-treated TGCT cohorts: 1) a phase 1 extension study (NCT01004861; 1000 mg/d; n = 39), 2) ENLIVEN patients randomized to pexidartinib (1000 mg/d for 2 weeks and then 800 mg/d; n = 61), and 3) ENLIVEN crossover patients (NCT02371369; 800 mg/d; n = 30). Eligible patients were 18 years old or older and had a histologically confirmed TGCT that was unresectable and symptomatic. Efficacy endpoints included the best overall response (complete or partial response) and the duration of response (DOR) by the Response Evaluation Criteria in Solid Tumors (RECIST) and the tumor volume score (TVS). The safety assessment included the frequency of treatment-emergent adverse events (TEAEs) and hepatic laboratory abnormalities (aminotransferase elevations and mixed/cholestatic hepatotoxicity). The data cutoff was May 31, 2019. RESULTS: One hundred thirty patients with TGCT received pexidartinib (median treatment duration, 19 months; range, 1 to 76+ months); 54 (42%) remained on treatment at the end of the analysis (26 months after initial data cut of March 2017). The RECIST overall response rate (ORR) was 60%; the TVS ORR was 65%. The median times to response were 3.4 (RECIST) and 2.8 months (TVS), with 48 of the responding patients (62%) achieving a RECIST partial response by 6 months and with 72 (9...