Pan Zhenjiang, Bao Jing, Wei Shepeng
Department of Neurosurgery, Shidong Hospital, Shanghai, China.
Front Oncol. 2025 Jun 27;15:1628195. doi: 10.3389/fonc.2025.1628195. eCollection 2025.
Vorasidenib, a brain-penetrant dual inhibitor of mutant isocitrate dehydrogenase 1 and 2 (IDH1/2), represents a significant advancement in the management of IDH-mutant gliomas. This review explores the clinical implications of its recent FDA approval for grade 2 IDH-mutant astrocytomas and oligodendrogliomas. We delve into the pivotal INDIGO trial, which demonstrated substantial improvements in progression-free survival, and discuss vorasidenib's pharmacokinetics, safety profile, and dosing guidelines. Additionally, we analyze its role within evolving treatment paradigms, including watchful waiting, IDH-targeted therapy, and integration with radiotherapy and chemotherapy. Comparative insights into traditional and novel approaches highlight the potential of vorasidenib to delay invasive therapies while preserving quality of life. Challenges such as adverse effects, long-term safety, and its application to higher-grade gliomas are also addressed. This comprehensive review underscores the transformative impact of vorasidenib and emphasizes the necessity of multidisciplinary care and patient-centered decision-making in glioma management.
伏立西尼是一种可穿透血脑屏障的突变型异柠檬酸脱氢酶1和2(IDH1/2)双重抑制剂,代表了IDH突变型胶质瘤治疗方面的重大进展。本综述探讨了其近期获美国食品药品监督管理局(FDA)批准用于治疗2级IDH突变型星形细胞瘤和少突胶质细胞瘤的临床意义。我们深入研究了关键的INDIGO试验,该试验显示无进展生存期有显著改善,并讨论了伏立西尼的药代动力学、安全性概况和给药指南。此外,我们分析了其在不断演变的治疗模式中的作用,包括观察等待、IDH靶向治疗以及与放疗和化疗的联合应用。对传统方法和新方法的比较见解突出了伏立西尼在延迟侵入性治疗同时保持生活质量方面的潜力。还讨论了诸如不良反应、长期安全性及其在高级别胶质瘤中的应用等挑战。这篇全面的综述强调了伏立西尼的变革性影响,并强调了在胶质瘤管理中多学科护理和以患者为中心的决策的必要性。