神经纤维瘤病 1 型和 2 型患者中枢及周围神经系统肿瘤治疗进展。
Updates in the Management of Central and Peripheral Nervous System Tumors among Patients with Neurofibromatosis Type 1 and Neurofibromatosis Type 2.
机构信息
Division of Oncology, Department of Pediatrics, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.
University of Pennsylvania Perelman School of Medicine, Philadelphia, Pennsylvania, USA.
出版信息
Pediatr Neurosurg. 2023;58(5):267-280. doi: 10.1159/000529507. Epub 2023 Feb 6.
BACKGROUND
Neurofibromatosis type 1 and neurofibromatosis type 2 are unrelated, distinct genetic disorders characterized by the development of central and peripheral nervous system tumors.
SUMMARY
Neurofibromatosis type 1 is the most common inherited tumor predisposition syndrome with a lifelong increased risk of benign and malignant tumor development, such as glioma and nerve sheath tumors. Neurofibromatosis type 2 classically presents with bilateral vestibular schwannoma, yet it is also associated with non-vestibular schwannoma, meningioma, and ependymoma. Historically, the number of effective therapies for neurofibromatosis-related neoplasms has been limited.
KEY MESSAGE
In the past decade, there have been significant advances in the development of precision-based therapies for NF-associated tumors with an increased emphasis on functional outcomes in addition to tumor response. Continued scientific discovery and advancement of targeted therapies for NF-associated neoplasms are necessary to continue to improve outcomes for patients with NF.
背景
神经纤维瘤病 1 型和神经纤维瘤病 2 型是两种互不相关的、独特的遗传性疾病，其特征是中枢和周围神经系统肿瘤的发展。
摘要
神经纤维瘤病 1 型是最常见的遗传性肿瘤易感性综合征，一生中良性和恶性肿瘤发展的风险增加，如神经胶质瘤和神经鞘瘤。神经纤维瘤病 2 型经典表现为双侧前庭神经鞘瘤，但也与非前庭神经鞘瘤、脑膜瘤和室管膜瘤有关。历史上，神经纤维瘤相关肿瘤的有效治疗方法数量有限。
关键信息
在过去的十年中，针对 NF 相关肿瘤的基于精准医学的治疗方法取得了重大进展，除了肿瘤反应外，对功能结果的重视程度也有所提高。为了继续改善 NF 患者的预后，有必要继续进行针对 NF 相关肿瘤的靶向治疗的科学发现和进展。
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