Treating subependymal giant cell astrocytoma in patients with tuberous sclerosis complex: an update of the literature.

IF 3.4 2区 医学 Q2 CLINICAL NEUROLOGY Expert Review of Neurotherapeutics Pub Date : 2025-04-01 Epub Date: 2025-03-07 DOI:10.1080/14737175.2025.2472922
David M Ritter, Sara Twardowski, David N Franz
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Abstract

Introduction: Subependymal giant cell astrocytomas (SEGAs) are one of the predominant features of the tuberous sclerosis complex (TSC). Before the use of mTOR inhibitors (mTORi; everolimus and sirolimus) in TSC, many patients had to undergo surgical operations (both preemptively and emergently). However, with mTORis gaining increased use, the role of medical and surgical therapy in SEGA is unclear.

Areas covered: The authors have based this review on publications listed in PubMed that delve into the role of surgery and mTORi in the treatment of SEGAs.

Expert opinion: There is no sizable head-to-head comparison of surgery and medical therapy in treating SEGA. Factors that reduce the ability to do these types of studies are the lack of uniform diagnosis of SEGA, provider preference for treatment, and variability in each treatment group (dosing of mTORis and various surgical providers). However, with the safety of mTORi, the authors recommend starting mTORi therapy for any growth in a nodule on serial scans and relying on surgery only for failed mTORi therapy.

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治疗结节性硬化症患者的室管膜下巨细胞星形细胞瘤:文献更新。
导语:室管膜下巨细胞星形细胞瘤(SEGAs)是结节性硬化症(TSC)的主要特征之一。在使用mTOR抑制剂(mTORi;依维莫司和西罗莫司)在TSC中,许多患者不得不接受外科手术(先发制人和紧急)。然而,随着mTORis的使用增加,药物和手术治疗在SEGA中的作用尚不清楚。涵盖领域:作者基于PubMed上列出的研究手术和mTORi在SEGAs治疗中的作用的出版物进行综述。专家意见:在治疗SEGA方面,没有相当大的手术和药物治疗的正面比较。降低此类研究能力的因素是SEGA缺乏统一的诊断,提供者对治疗的偏好,以及每个治疗组的可变性(mTORis的剂量和不同的手术提供者)。然而,考虑到mTORi的安全性,作者建议在连续扫描的任何结节生长中开始mTORi治疗,只有在mTORi治疗失败时才依赖手术。
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来源期刊
Expert Review of Neurotherapeutics
Expert Review of Neurotherapeutics Medicine-Neurology (clinical)
CiteScore
7.00
自引率
2.30%
发文量
61
审稿时长
4-8 weeks
期刊介绍: Expert Review of Neurotherapeutics (ISSN 1473-7175) provides expert reviews on the use of drugs and medicines in clinical neurology and neuropsychiatry. Coverage includes disease management, new medicines and drugs in neurology, therapeutic indications, diagnostics, medical treatment guidelines and neurological diseases such as stroke, epilepsy, Alzheimer''s and Parkinson''s. Comprehensive coverage in each review is complemented by the unique Expert Review format and includes the following sections: Expert Opinion - a personal view of the data presented in the article, a discussion on the developments that are likely to be important in the future, and the avenues of research likely to become exciting as further studies yield more detailed results Article Highlights – an executive summary of the author’s most critical points
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