Tops Annemijn L, Schopman Josefine E, Koot Radboud W, Gelderblom Hans, Putri Nabila A, Rahmi Latifah N A, Jansen Jeroen C, Hensen Erik F
Department of Otorhinolaryngology-Head and Neck Surgery, Leiden University Medical Center, 2333 ZA Leiden, The Netherlands.
Department of Medical Oncology, Leiden University Medical Center, 2333 ZA Leiden, The Netherlands.
Cancers (Basel). 2025 Feb 4;17(3):519. doi: 10.3390/cancers17030519.
BACKGROUND/OBJECTIVES: NF2-related schwannomatosis (NF2) is a tumor predisposition syndrome that typically presents with bilateral vestibular schwannomas, together with other intracranial and spinal schwannomas, meningiomas, and/or ependymomas. Bevacizumab, a VEGF inhibitor, has the potential to decrease schwannoma volume and improve hearing in adults, but the literature on the effects in children is sparse. This narrative review aims to evaluate the use of bevacizumab in pediatric NF2 patients, focusing on hearing, tumor progression, and toxicity.
A literature review was conducted following PRISMA guidelines. Articles were searched in PubMed, Embase, Web of Science, Cochrane Library, Emcare, and Academic Search Premier on 18 July 2024. Inclusion criteria were patients ≤ 18 years, diagnosed with NF2 and treated with bevacizumab. Two authors independently assessed the quality of the evidence and extracted relevant data from the included articles.
Seventeen articles including 62 pediatric NF2 patients met the inclusion criteria. Studies varied widely in treatment regimens and outcome parameters. Tumor regression was reported in 6/56 patients (11%) and 38/56 (68%) remained stable. Hearing improved in 15/45 patients (33%) and did not further deteriorate in 27/45 (60%). An improvement in other symptoms was seen in 6/29 patients (28%). Toxicity was reported in five studies, documenting 13 adverse events in 28 patients ranging from grade 1 to grade 3. Treatment was discontinued in both patients who experienced grade 3 toxicity.
Bevacizumab seems to be a viable treatment option for pediatric NF2 patients. Tumor regression or stabilization is achieved in the majority of patients (77%). Moreover, a considerable number of pediatric patients experience hearing stabilization or improvement (93%). Bevacizumab appears to be relatively well tolerated, offering a non-invasive therapeutic option for children with NF2 suffering from progressive vestibular schwannomas and hearing loss.
背景/目的：与神经纤维瘤病2型相关的神经鞘瘤病（NF2）是一种肿瘤易感综合征，通常表现为双侧前庭神经鞘瘤，以及其他颅内和脊髓神经鞘瘤、脑膜瘤和/或室管膜瘤。贝伐单抗是一种血管内皮生长因子（VEGF）抑制剂，有可能减小成人神经鞘瘤的体积并改善听力，但关于其对儿童影响的文献较少。本叙述性综述旨在评估贝伐单抗在儿科NF2患者中的应用，重点关注听力、肿瘤进展和毒性。
按照系统评价和Meta分析的首选报告项目（PRISMA）指南进行文献综述。于2024年7月18日在PubMed、Embase、科学网、Cochrane图书馆、Emcare和学术搜索高级版中检索文章。纳入标准为年龄≤18岁、诊断为NF2并接受贝伐单抗治疗的患者。两名作者独立评估证据质量，并从纳入的文章中提取相关数据。
17篇文章纳入了62例儿科NF2患者，符合纳入标准。研究的治疗方案和结局参数差异很大。6/56例患者（11%）报告肿瘤消退，38/56例（68%）病情稳定。15/45例患者（33%）听力改善，27/45例（60%）听力未进一步恶化。6/29例患者（28%）的其他症状有所改善。五项研究报告了毒性反应，记录了28例患者的13起不良事件，分级从1级到3级。发生3级毒性反应的两名患者均停止了治疗。
贝伐单抗似乎是儿科NF2患者的一种可行治疗选择。大多数患者（77%）实现了肿瘤消退或病情稳定。此外，相当数量的儿科患者听力稳定或改善（93%）。贝伐单抗的耐受性似乎相对良好，为患有进行性前庭神经鞘瘤和听力损失的NF2儿童提供了一种非侵入性治疗选择。
