神经纤维瘤病 2 型（NF2）相关前庭神经鞘瘤的靶向治疗进展。
Advances in Targeted Therapy for Neurofibromatosis Type 2 (NF2)-Associated Vestibular Schwannomas.
机构信息
Department of Otolaryngology-Head & Neck Surgery, Indiana University School of Medicine, Fesler Hall Suite 400 1130 W. Michigan Street, Indianapolis, IN, 46202, USA.
Department of Pediatrics, Indiana University School of Medicine, Indianapolis, IN, USA.
出版信息
Curr Oncol Rep. 2023 May;25(5):531-537. doi: 10.1007/s11912-023-01388-3. Epub 2023 Mar 18.
PURPOSE OF REVIEW
Neurofibromatosis 2 (NF2) is an autosomal-dominant genetic disorder characterized by bilateral vestibular schwannomas (VS), meningiomas, ependymomas, spinal and peripheral schwannomas, optic gliomas, and juvenile cataracts. Ongoing studies provide new insight into the role of the NF2 gene and merlin in VS tumorigenesis.
RECENT FINDINGS
As NF2 tumor biology becomes increasingly understood, therapeutics targeting specific molecular pathways have been developed and evaluated in preclinical and clinical studies. NF2-associated VS are a source of significant morbidity with current treatments including surgery, radiation, and observation. Currently, there are no FDA-approved medical therapies for VS, and the development of selective therapeutics is a high priority. This manuscript reviews NF2 tumor biology and current therapeutics undergoing investigation for treatment of patients with VS.
目的综述
神经纤维瘤病 2 型（NF2）是一种常染色体显性遗传疾病，其特征是双侧前庭神经鞘瘤（VS）、脑膜瘤、室管膜瘤、脊髓和周围神经鞘瘤、视神经胶质瘤和少年性白内障。目前的研究为 NF2 基因和 Merlin 在 VS 肿瘤发生中的作用提供了新的见解。
最近的发现
随着 NF2 肿瘤生物学的不断深入了解，针对特定分子途径的治疗方法已经在临床前和临床研究中得到了开发和评估。NF2 相关 VS 是一种严重的致残性疾病，目前的治疗方法包括手术、放疗和观察。目前，还没有 FDA 批准的 VS 治疗药物，因此选择性治疗方法的开发是当务之急。本文综述了 NF2 肿瘤生物学和目前正在研究的治疗 VS 患者的治疗方法。
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