Department of Pediatric Surgery, Asan Medical Center Children's Hospital, University of Ulsan College of Medicine, Seoul, Korea.
Department of Pediatric Surgery, Asan Medical Center Children's Hospital, University of Ulsan College of Medicine, Seoul, Korea.
J Vasc Surg Venous Lymphat Disord. 2021 Nov;9(6):1488-1494. doi: 10.1016/j.jvsv.2021.03.014. Epub 2021 Apr 6.
Recently, sirolimus has emerged as a safe and effective treatment modality for unresectable vascular lesions. In the present study, we investigated the effectiveness and safety of sirolimus from our early experience with patients with unresectable vascular anomalies.
The medical records and radiologic images of all patients with unresectable vascular anomalies treated with sirolimus at our center from January 2018 to November 2019 were retrospectively reviewed. All patients were administered oral doses of sirolimus 0.8 mg/m every 12 hours as the initial dose, followed by maintenance of a target serum concentration (5-10 ng/mL) with therapeutic drug monitoring.
Six patients with unresectable vascular anomalies were treated with sirolimus for ≥10 months. Their median age at the initiation of sirolimus treatment was 17 months (range, 8-67 months). The median duration of treatment was 13 months (range, 10-16 months). One patient had a good response, four had an intermediate response, and one had no response to sirolimus therapy. None of the patients had discontinued sirolimus therapy because of adverse effects.
Sirolimus can be used effectively and safely for patients with unresectable vascular anomalies. However, further prospective studies are warranted to evaluate the long-term effects of sirolimus and clarify the indications for early intervention.
最近，西罗莫司已成为一种安全有效的治疗不可切除血管病变的方法。本研究旨在从我们对不可切除血管畸形患者的早期经验中，探讨西罗莫司的有效性和安全性。
回顾性分析了 2018 年 1 月至 2019 年 11 月期间在我院接受西罗莫司治疗的所有不可切除血管畸形患者的病历和影像学图像。所有患者初始剂量均为口服西罗莫司 0.8mg/m2，每 12 小时 1 次，随后通过治疗药物监测维持目标血清浓度（5-10ng/mL）。
6 例不可切除血管畸形患者接受西罗莫司治疗≥10 个月。起始治疗时患者的中位年龄为 17 个月（8-67 个月）。中位治疗时间为 13 个月（10-16 个月）。1 例患者对西罗莫司治疗反应良好，4 例患者反应中等，1 例患者无反应。无患者因不良反应而停止西罗莫司治疗。
西罗莫司可有效且安全地用于治疗不可切除的血管畸形。然而，需要进一步的前瞻性研究来评估西罗莫司的长期效果，并阐明早期干预的适应证。
