{"title":"[nf2相关神经鞘瘤病的分子发病机制、遗传特征和治疗策略]。","authors":"Yu Teranishi, Satoru Miyawaki, Nobuhito Saito","doi":"10.11477/mf.030126030530040802","DOIUrl":null,"url":null,"abstract":"<p><p>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.</p>","PeriodicalId":35984,"journal":{"name":"Neurological Surgery","volume":"53 4","pages":"802-810"},"PeriodicalIF":0.0000,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"[Molecular Pathogenesis, Genetic Profiles, and Therapeutic Strategies for NF2-Related Schwannomatosis].\",\"authors\":\"Yu Teranishi, Satoru Miyawaki, Nobuhito Saito\",\"doi\":\"10.11477/mf.030126030530040802\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>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.</p>\",\"PeriodicalId\":35984,\"journal\":{\"name\":\"Neurological Surgery\",\"volume\":\"53 4\",\"pages\":\"802-810\"},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2025-07-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Neurological Surgery\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.11477/mf.030126030530040802\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q4\",\"JCRName\":\"Medicine\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Neurological Surgery","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.11477/mf.030126030530040802","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q4","JCRName":"Medicine","Score":null,"Total":0}
[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.