Gelderblom Hans, de Sande Michiel van
Leiden University Medical Center, Leiden, Netherlands.
Future Oncol. 2020 Oct;16(29):2345-2356. doi: 10.2217/fon-2020-0542. Epub 2020 Jul 23.
Pexidartinib is an orally administered small molecule tyrosine kinase inhibitor. Phase III ENLIVEN study results provided clinical evidence for US FDA approval for treatment of adult patients with symptomatic tenosynovial giant cell tumor associated with severe morbidity or functional limitations and not amenable to improvement with surgery. Recommended dosage is 400 mg orally twice daily on an empty stomach. Long-term follow-up in pooled analyses showed increased response rates compared with those observed in ENLIVEN. Patients on pexidartinib also experience meaningful improvements in range of motion. Side effects associated with pexidartinib are generally manageable; however, there is a risk of potentially life-threatening mixed or cholestatic hepatotoxicity and pexidartinib has a Risk Evaluation and Mitigation Strategy (REMS) program to ensure appropriate monitoring.
培西达替尼是一种口服小分子酪氨酸激酶抑制剂。III期ENLIVEN研究结果为美国食品药品监督管理局（US FDA）批准其用于治疗患有症状性腱鞘巨细胞瘤且伴有严重发病率或功能受限、无法通过手术改善的成年患者提供了临床证据。推荐剂量为空腹口服400 mg，每日两次。汇总分析中的长期随访显示，与ENLIVEN研究中观察到的反应率相比有所提高。接受培西达替尼治疗的患者在活动范围方面也有显著改善。与培西达替尼相关的副作用通常是可控的；然而，存在潜在危及生命的混合性或胆汁淤积性肝毒性风险，并且培西达替尼有一个风险评估与缓解策略（REMS）计划以确保进行适当监测。
