[nf2相关神经鞘瘤病的分子发病机制、遗传特征和治疗策略]。

Q4 Medicine
Yu Teranishi, Satoru Miyawaki, Nobuhito Saito
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引用次数: 0

摘要

2型神经纤维瘤病(NF2),现在被重新定义为NF2相关神经鞘瘤病(NF2- swn),是一种以双侧前庭神经鞘瘤、多发性脑膜瘤和其他中枢神经系统肿瘤为特征的遗传性肿瘤综合征。修订后的诊断标准包括基因检测和考虑体细胞嵌合体,使更准确和更早的诊断。基因型与表型之间存在很强的相关性:截短突变与早期发病和严重的临床病程有关,而错义或剪接位点突变通常与较轻的表型和较好的功能结果有关。免疫和分子图谱揭示了前庭神经鞘瘤和脑膜瘤的不同亚型,反映了肿瘤行为和治疗反应的差异。在单侧前庭神经鞘瘤的年轻患者中,临床医生应警惕NF2-SWN的可能性,并考虑基于进展和遗传背景进行监测。靶向治疗——包括贝伐单抗、布加替尼和血管内皮生长因子-A疫苗——已经显示出希望,特别是在难治性病例中。虽然常规的密集监测不是普遍需要的,但早期遗传筛查和长期随访可能有益于选定的个体。全面整合遗传、病理和免疫学数据对于推进NF2-SWN的个性化治疗策略至关重要。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
[Molecular Pathogenesis, Genetic Profiles, and Therapeutic Strategies for NF2-Related Schwannomatosis].

Neurofibromatosis type 2 (NF2), now redefined as NF2-related schwannomatosis (NF2-SWN), is a hereditary tumor syndrome characterized by bilateral vestibular schwannomas, multiple meningiomas, and other central nervous system tumors. The revised diagnostic criteria incorporate genetic testing and consideration of somatic mosaicism, enabling a more accurate and earlier diagnosis. A strong genotype-phenotype correlation has been established: truncating mutations are associated with an earlier onset and severe clinical course, whereas missense or splice-site mutations are often linked to milder phenotypes and better functional outcomes. Immune and molecular profiling have revealed distinct subtypes of vestibular schwannomas and meningiomas, reflecting differences in tumor behavior and therapeutic responses. In young patients presenting with unilateral vestibular schwannoma, clinicians should remain alert to the possibility of NF2-SWN and consider surveillance based on progression and genetic background. Targeted therapies-including bevacizumab, brigatinib, and vascular endothelial growth factor -A vaccines-have shown promise, particularly in refractory cases. Although routine intensive surveillance is not universally required, early genetic screening and long-term follow-ups may benefit select individuals. The comprehensive integration of genetic, pathological, and immunological data is essential for advancing personalized treatment strategies for NF2-SWN.

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来源期刊
Neurological Surgery
Neurological Surgery Medicine-Medicine (all)
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