Who will benefit from vorasidenib? Review of data from the literature and open questions.

IF 2.4 Q2 CLINICAL NEUROLOGY

Neuro-oncology practice Pub Date : 2024-11-14 eCollection Date: 2025-02-01 DOI:10.1093/nop/npae104

Amélie Darlix, Matthias Preusser, Shawn L Hervey-Jumper, Helen A Shih, Emmanuel Mandonnet, Jennie W Taylor

{"title":"Who will benefit from vorasidenib? Review of data from the literature and open questions.","authors":"Amélie Darlix, Matthias Preusser, Shawn L Hervey-Jumper, Helen A Shih, Emmanuel Mandonnet, Jennie W Taylor","doi":"10.1093/nop/npae104","DOIUrl":null,"url":null,"abstract":"<p><p>The clinical efficacy of isocitrate dehydrogenase (IDH) inhibitors in the treatment of patients with grade 2 IDH-mutant (mIDH) gliomas is a significant therapeutic advancement in neuro-oncology. It expands treatment options beyond traditional radiation therapy and cytotoxic chemotherapy, which may lead to significant long-term neurotoxic effects while extending patient survival. The INDIGO study demonstrated that vorasidenib, a pan-mIDH inhibitor, improved progression-free survival for patients with grade 2 mIDH gliomas following surgical resection or biopsy compared to placebo and was well tolerated. However, these encouraging results leave a wake of unanswered questions: Will higher-grade mIDH glioma patients benefit? When is the appropriate timing to start and stop treatment? Where does this new treatment option fit in with other treatment modalities? In this study, we review the limited data available to start addressing these questions, provide a framework of how to discuss these gaps with current patients, and highlight what is needed from the neuro-oncology community for more definitive answers.</p>","PeriodicalId":19234,"journal":{"name":"Neuro-oncology practice","volume":"12 Suppl 1","pages":"i6-i18"},"PeriodicalIF":2.4000,"publicationDate":"2024-11-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11703370/pdf/","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Neuro-oncology practice","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1093/nop/npae104","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2025/2/1 0:00:00","PubModel":"eCollection","JCR":"Q2","JCRName":"CLINICAL NEUROLOGY","Score":null,"Total":0}

引用次数: 0

Abstract

The clinical efficacy of isocitrate dehydrogenase (IDH) inhibitors in the treatment of patients with grade 2 IDH-mutant (mIDH) gliomas is a significant therapeutic advancement in neuro-oncology. It expands treatment options beyond traditional radiation therapy and cytotoxic chemotherapy, which may lead to significant long-term neurotoxic effects while extending patient survival. The INDIGO study demonstrated that vorasidenib, a pan-mIDH inhibitor, improved progression-free survival for patients with grade 2 mIDH gliomas following surgical resection or biopsy compared to placebo and was well tolerated. However, these encouraging results leave a wake of unanswered questions: Will higher-grade mIDH glioma patients benefit? When is the appropriate timing to start and stop treatment? Where does this new treatment option fit in with other treatment modalities? In this study, we review the limited data available to start addressing these questions, provide a framework of how to discuss these gaps with current patients, and highlight what is needed from the neuro-oncology community for more definitive answers.

查看原文本刊更多论文

谁将受益于vorasidenib？回顾文献资料和开放性问题。

异柠檬酸脱氢酶（IDH）抑制剂治疗2级IDH突变（mIDH）胶质瘤的临床疗效是神经肿瘤学治疗的重大进展。它扩大了传统放射治疗和细胞毒性化疗之外的治疗选择，这可能导致显着的长期神经毒性作用，同时延长患者的生存期。INDIGO研究表明，与安慰剂相比，泛mIDH抑制剂vorasidenib可改善手术切除或活检后2级mIDH胶质瘤患者的无进展生存期，并且耐受性良好。然而，这些令人鼓舞的结果留下了一系列悬而未决的问题：更高级别的mIDH胶质瘤患者会受益吗？何时开始和停止治疗是合适的时机？这种新的治疗方案与其他治疗方式有何不同？在这项研究中，我们回顾了有限的可用数据来开始解决这些问题，提供了一个如何与当前患者讨论这些差距的框架，并强调了神经肿瘤学社区需要什么来获得更明确的答案。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

求助全文

约1分钟内获得全文求助全文

来源期刊

Neuro-oncology practice CLINICAL NEUROLOGY-

CiteScore

5.30

自引率

11.10%

发文量

期刊介绍： Neuro-Oncology Practice focuses on the clinical aspects of the subspecialty for practicing clinicians and healthcare specialists from a variety of disciplines including physicians, nurses, physical/occupational therapists, neuropsychologists, and palliative care specialists, who have focused their careers on clinical patient care and who want to apply the latest treatment advances to their practice. These include: Applying new trial results to improve standards of patient care Translating scientific advances such as tumor molecular profiling and advanced imaging into clinical treatment decision making and personalized brain tumor therapies Raising awareness of basic, translational and clinical research in areas of symptom management, survivorship, neurocognitive function, end of life issues and caregiving