Brain arteriovenous malformations (bAVMs) are relatively rare, although their potential for secondary intracranial haemorrhage (ICH) makes their diagnosis and management essential to the community. Currently, invasive therapies (surgical resection, stereotactic radiosurgery and endovascular embolisation) are the only interventions that offer a reduction in ICH risk. There is no designated medical therapy for bAVM, although there is growing animal and human evidence supporting a role for bevacizumab to reduce the size of AVMs. In this single-arm pilot study, two patients with large bAVMs (deemed unresectable by an interdisciplinary team) received bevacizumab 5 mg/kg every 2 weeks for 12 weeks. Due to limitations of external funding, the intended sample size of 10 participants was not reached. Primary outcome measure was change in bAVM volume from baseline at 26 and 52 weeks. No change in bAVM volume was observed 26 or 52 weeks after bevacizumab treatment. No clinically important adverse events were observed during the 52-week study period. There were no observed instances of ICH. Sera vascular endothelial growth factor levels were reduced at 26 weeks and returned to baseline at 52 weeks. This pilot study is the first to test bevacizumab for patients with bAVMs. Bevacizumab therapy was well tolerated in both subjects. No radiographic changes were observed over the 52-week study period. Subsequent larger clinical trials are in order to assess for dose-dependent efficacy and rarer adverse drug effects. Trial registration number: [NCT02314377][1]. [1]: /lookup/external-ref?link_type=CLINTRIALGOV&access_num=NCT02314377&atom=%2Fbmjno%2F3%2F1%2Fe000114.atom

中文翻译：

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贝伐单抗治疗脑动静脉畸形的概念验证单臂试验

脑动静脉畸形 (bAVM) 相对罕见，尽管它们可能导致继发性颅内出血 (ICH)，因此其诊断和管理对社区至关重要。目前，侵入性疗法（手术切除、立体定向放射外科和血管内栓塞）是唯一能降低 ICH 风险的干预措施。尽管有越来越多的动物和人类证据支持贝伐单抗在减小 AVM 大小方面的作用，但尚无针对 bAVM 的指定药物疗法。在这项单臂试验研究中，两名患有大 bAVM（跨学科团队认为不可切除）的患者每 2 周接受 5 mg/kg 的贝伐单抗治疗，持续 12 周。由于外部资金的限制，未达到 10 名参与者的预期样本量。主要结果指标是 26 周和 52 周时 bAVM 体积相对于基线的变化。贝伐单抗治疗后 26 或 52 周未观察到 bAVM 体积的变化。在 52 周的研究期间未观察到临床上重要的不良事件。没有观察到 ICH 的情况。血清血管内皮生长因子水平在 26 周时降低，并在 52 周时恢复到基线水平。这项试点研究是首次针对 bAVM 患者测试贝伐单抗。贝伐单抗治疗在两名受试者中均具有良好的耐受性。在 52 周的研究期间没有观察到放射学变化。随后的更大规模的临床试验是为了评估剂量依赖性疗效和罕见的药物不良反应。试验注册号：[NCT02314377][1]。[1]：/lookup/external-ref?link_type=CLINTRIALGOV&access_num=NCT02314377&