Department of Dermatology, University Hospital Virgen del Rocío, Seville, Spain.
Clin Exp Dermatol. 2022 Jan;47(1):57-62. doi: 10.1111/ced.14841. Epub 2021 Aug 19.
Vascular malformations are a complex pathology with few treatment options. In previously published studies, oral sirolimus (rapamycin) has shown promising results in the treatment of low-flow vascular malformations, but its usefulness in high-flow vascular malformations is controversial.
To evaluate the efficacy and safety of sirolimus for the treatment of high-flow vascular malformations in real-life practice.
In a unit specializing in vascular anomalies, patients treated with oral sirolimus for high-flow vascular malformations were located by consulting the drug dispensations. Reviewing the electronic medical records, data on patient demographics, vascular malformation characteristics, treatments, toxicity and clinical course were collected and statistically analysed.
Nine patients with vascular malformations were included: eight had arteriovenous malformation and one had arteriovenous fistula. Six of these malformations were isolated while three were part of a syndrome. Sirolimus was initiated at a dosage of 1-4 mg/day to be taken as a single dose. Partial response was observed in eight of the nine patients (88.9%) with high-flow vascular malformation, while worsening was observed in the remaining patient. The treatment was well tolerated and at the most recent follow-up, five patients remained on treatment with oral sirolimus.
Our results show that oral sirolimus is a well-tolerated therapeutic option, with an excellent safety profile, which can be useful in the long-term stabilization of patients with high-flow vascular malformations. Single-daily dosage may improve long-term adherence to treatment without worsening its effectiveness.
血管畸形是一种复杂的病理，治疗选择有限。在之前发表的研究中，口服西罗莫司（雷帕霉素）在治疗低流量血管畸形方面显示出良好的效果，但在治疗高流量血管畸形方面的效果仍存在争议。
评估西罗莫司治疗真实世界中高流量血管畸形的疗效和安全性。
在一个专门研究血管异常的单位，通过查阅药物配给记录，定位接受口服西罗莫司治疗高流量血管畸形的患者。通过回顾电子病历，收集患者人口统计学、血管畸形特征、治疗、毒性和临床病程等数据，并进行统计学分析。
共纳入 9 例血管畸形患者：8 例为动静脉畸形，1 例为动静脉瘘。其中 6 例为孤立性病变，3 例为综合征的一部分。西罗莫司起始剂量为 1-4mg/天，每日一次服用。9 例高流量血管畸形患者中，有 8 例（88.9%）观察到部分缓解，1 例病情恶化。治疗耐受性良好，截至最近一次随访，5 例患者仍在接受口服西罗莫司治疗。
我们的结果表明，口服西罗莫司是一种耐受性良好的治疗选择，具有良好的安全性，可长期稳定高流量血管畸形患者的病情。每日一次的剂量可能会提高长期治疗的依从性，而不会降低其疗效。
