多中心、前瞻性、II 期研究：贝伐珠单抗维持治疗 NF2 相关听神经鞘瘤和进行性前庭神经鞘瘤患儿及成人。
Multicenter, prospective, phase II study of maintenance bevacizumab for children and adults with NF2-related schwannomatosis and progressive vestibular schwannoma.
机构信息
Massachusetts General Hospital and Harvard Medical School, Boston, Massachusetts, USA.
NYU Langone Health, New York, New York, USA.
出版信息
Neuro Oncol. 2023 Aug 3;25(8):1498-1506. doi: 10.1093/neuonc/noad066.
BACKGROUND
Prospective data on maintenance therapy with bevacizumab for persons with NF2-related schwannomatosis (NF2-SWN) is lacking. In this prospective multicenter phase II study, we evaluated the efficacy, safety, and tolerability of bevacizumab for maintenance therapy in children and adults with NF2-SWN and hearing loss due to vestibular schwannomas (VS).
METHODS
Following induction therapy, participants received bevacizumab 5 mg/kg every 3 weeks for 18 months. Participants were monitored for changes in hearing, tumor size, and quality of life (QOL), and for adverse events. Hearing loss was defined as a statistically significant decline in word recognition score (WRS) or pure-tone average compared to the study baseline; tumor growth was defined as >20% increase in volume compared to baseline.
RESULTS
Twenty participants with NF2-SWN (median age 23.5 years; range, 12.5-62.5 years) with hearing loss in the target ear (median WRS 70%, range 2%-94%) received maintenance bevacizumab. Freedom from hearing loss in the target ear was 95% after 48 weeks, 89% after 72 weeks, and 70% after 98 weeks. Freedom from tumor growth in the target VS was 94% after 48 weeks, 89% after 72 weeks, and 89% after 98 weeks. NF2-related QOL remained stable for 98 weeks whereas tinnitus-related distress decreased. Maintenance bevacizumab was well tolerated, with 3 participants (15%) discontinuing treatment due to adverse events.
CONCLUSIONS
Maintenance bevacizumab (5 mg/kg every 3 weeks) is associated with high rates of hearing and tumor stability during 18 months of follow-up. No new unexpected adverse events related to bevacizumab were identified in this population.
背景
关于贝伐珠单抗用于神经纤维瘤病 2 型相关 schwannomatosis（NF2-SWN）患者维持治疗的前瞻性数据尚缺乏。在这项前瞻性多中心 II 期研究中，我们评估了贝伐珠单抗用于 NF2-SWN 伴听神经瘤（VS）所致听力损失儿童和成人维持治疗的疗效、安全性和耐受性。
方法
在诱导治疗后，参与者接受贝伐珠单抗 5mg/kg，每 3 周一次，共 18 个月。监测参与者的听力、肿瘤大小和生活质量（QOL）变化以及不良反应。听力损失定义为与研究基线相比，言语识别得分（WRS）或纯音平均听力下降具有统计学意义；肿瘤生长定义为与基线相比体积增加>20%。
结果
20 名 NF2-SWN 患者（中位年龄 23.5 岁；范围 12.5-62.5 岁）目标耳存在听力损失（中位 WRS 70%，范围 2%-94%）接受维持贝伐珠单抗治疗。目标耳听力损失无进展率在 48 周时为 95%，72 周时为 89%，98 周时为 70%。目标 VS 肿瘤生长无进展率在 48 周时为 94%，72 周时为 89%，98 周时为 89%。NF2 相关 QOL 在 98 周时保持稳定，而耳鸣相关困扰减轻。维持贝伐珠单抗治疗耐受性良好，3 名患者（15%）因不良反应停药。
结论
在 18 个月的随访中，贝伐珠单抗（5mg/kg，每 3 周一次）维持治疗与较高的听力和肿瘤稳定性相关。在该人群中未发现与贝伐珠单抗相关的新的意外不良事件。
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