Department of Radiation Oncology, Miami Cancer Institute, Baptist Health South Florida, Miami, FL.
Division of Neuro-Oncology, Departments of Neurology, Neurological Surgery, and Medicine, University of Virginia Health System, Charlottesville, VA.
J Clin Oncol. 2024 Jul 20;42(21):2588-2598. doi: 10.1200/JCO.23.02195. Epub 2024 Jun 4.
Mutations in isocitrate dehydrogenase (IDH) genes, an early step in the ontogeny of lower-grade gliomas, induce global epigenetic changes characterized by a hypermethylation phenotype and are critical to tumor classification, treatment decision making, and estimation of patient prognosis. The introduction of IDH inhibitors to block the oncogenic neomorphic function of the mutated protein has resulted in new therapeutic options for these patients. To appreciate the implications of these recent IDH inhibitor results, it is important to juxtapose historical outcomes with chemoradiotherapy. Herein, we rationally evaluate recent IDH inhibitor data within historical precedents to guide contemporary decisions regarding the role of observation, maximal safe resection, adjuvant therapies, and the import of patient and tumor variables. The biological underpinnings of the IDH pathway and the mechanisms, impact, and limitations of IDH inhibitors, the actual magnitude of tumor regression and patient benefit, and emergence of resistance pathways are presented to guide future trial development. Management in the current, molecularly defined era will require careful patient selection and risk factor assessment, followed by an open dialog about the results of studies such as INDIGO, as well as mature data from legacy trials, and a discussion about risk-versus-benefit for the choice of treatment, with multidisciplinary decision making as an absolute prerequisite.
IDH 基因突变(IDH)是低级别神经胶质瘤发生的早期步骤,可诱导全基因组表观遗传改变,表现为高甲基化表型,这对肿瘤分类、治疗决策以及患者预后评估具有重要意义。IDH 抑制剂的引入可阻断突变蛋白的致癌新表型,为这些患者提供了新的治疗选择。为了理解这些最近的 IDH 抑制剂研究结果的意义,将其与放化疗的历史结果进行对比是很重要的。在此,我们基于历史数据对最近的 IDH 抑制剂数据进行了合理评估,以指导关于观察、最大限度安全切除、辅助治疗以及患者和肿瘤变量的作用的当代决策。本文还介绍了 IDH 通路的生物学基础以及 IDH 抑制剂的作用机制、影响和局限性、实际肿瘤消退和患者获益的程度以及耐药途径的出现,以指导未来的试验开发。在当前分子定义的时代,需要对患者进行仔细的选择和危险因素评估,然后对 INDIGO 等研究的结果进行公开讨论,以及对传统试验的成熟数据进行讨论,并根据治疗选择的风险与获益进行讨论,多学科决策是绝对必要的。