University of Tours, CHRU Tours, FR-37044 Tours, France.
Acta Derm Venereol. 2019 Nov 1;99(12):1105-1109. doi: 10.2340/00015555-3273.
Managing extracranial arteriovenous malformations is challenging. Sirolimus (rapamycin) is increasingly being used when surgery and embolization are not advised. Because of its anti-angiogenic properties here we report all extracranial arteriovenous malformation cases treated with sirolimus in 2 French tertiary centers for vascular anomalies. The outcomes were efficacy (complete, partial, no response) based on arteriovenous malformation volume and necrosis/hemorrhage and side effects. We retrospectively included 10 patients (7 children). The sirolimus dose ranged from 0.6 to 3.5 mg/m2. Median (interquartile range [IQR]) treatment time was 24.5 (4.5; 35) months. Five patients showed no response and 5 showed partial response at a median (IQR) of 3 (1; 5) months followed in 2 cases by therapeutic resistance (i.e., progressive disease after 9 and 24 months of treatment). The most frequent side effect was mouth ulcers. This study shows poor efficacy of sirolimus for treating extracranial arteriovenous malformations.
管理颅外动静脉畸形具有挑战性。当手术和栓塞不被建议时，西罗莫司（雷帕霉素）越来越多地被使用。由于其抗血管生成特性，我们在此报告了在 2 家法国三级血管畸形中心用西罗莫司治疗的所有颅外动静脉畸形病例。根据动静脉畸形体积和坏死/出血以及副作用来评估疗效（完全、部分、无反应）。我们回顾性纳入了 10 名患者（7 名儿童）。西罗莫司剂量范围为 0.6 至 3.5mg/m2。中位（四分位间距[IQR]）治疗时间为 24.5（4.5；35）个月。5 名患者无反应，5 名患者部分反应，中位（IQR）为 3（1；5）个月，其中 2 例随后出现治疗抵抗（即治疗 9 个月和 24 个月后疾病进展）。最常见的副作用是口腔溃疡。这项研究表明西罗莫司治疗颅外动静脉畸形的疗效不佳。
