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. Methods: 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. Results: 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. Conclusions: 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：NF2相关神经鞘瘤病是一种肿瘤易感综合征，典型表现为双侧前庭神经鞘瘤，并伴有其他颅内及脊髓神经鞘瘤、脑膜瘤和/或室管膜瘤。血管内皮生长因子抑制剂贝伐珠单抗在成人中具有缩小神经鞘瘤体积和改善听力的潜力，但关于其对儿童疗效的文献较少。本综述旨在评估贝伐珠单抗在儿童NF2患者中的应用效果，重点关注听力、肿瘤进展及毒性反应。方法：按照PRISMA指南进行文献综述，于2024年7月18日检索PubMed、Embase、Web of Science、Cochrane Library、Emcare及Academic Search Premier数据库。纳入标准为年龄≤18岁、确诊NF2并接受贝伐珠单抗治疗的患者。由两位研究者独立评估证据质量并提取相关数据。结果：共纳入17篇文献，涉及62例儿童NF2患者。各研究的治疗方案和结局参数差异显著。56例患者中6例（11%）报告肿瘤退缩，38例（68%）保持稳定；45例患者中15例（33%）听力改善，27例（60%）未进一步恶化；29例患者中6例（28%）其他症状得到改善。5项研究报告了毒性反应，28例患者中共发生13起1-3级不良事件，其中2例3级毒性反应患者终止治疗。结论：贝伐珠单抗似乎是儿童NF2患者可行的治疗选择。大多数患者（77%）可实现肿瘤退缩或稳定，相当比例的儿童患者（93%）听力保持稳定或得到改善。该药物耐受性相对良好，为伴有进行性前庭神经鞘瘤和听力损失的NF2儿童提供了非侵入性治疗选择。
肿瘤(癌症)患者之家