Abstract

Objectives

The management of vascular malformations is complex and challenging. This study aimed to explore efficacy, plasma trough concentrations of sirolimus, post-withdrawal conditions, and adverse reactions of sirolimus in treating complex vascular malformations.

Methods

In our center, we analyzed vascular malformations treated with sirolimus (and corticosteroid) from August 2017 to June 2021. Meanwhile, we reviewed the medical records, the efficacy, side effects, and laboratory tests. Patients who had stopped taking sirolimus were followed up by telephone.

Results

A total of 25 patients with complicated vascular malformations in our center, including 7 females and 18 males aged 4 months to 15 years, were enrolled. In all, 19 patients (76.0%) responded to sirolimus, and the plasma concentration of sirolimus fluctuated between 0.97 and 27.15 ng/ml. In all, 24 patients (96.0%) were in follow-up. A total of 15 patients (62.5%) stopped taking sirolimus during follow-up, and 2 patients (13.3%) discontinued the sirolimus due to side effects. A total of 3 patients (20.0%) restarted sirolimus treatment.

Conclusion

Starting dose of 1.5–2 mg/m² sirolimus is effective and safe in vascular malformation treatment. The best treatment regimen and discontinuation indications needed more investigation. Most should be done about targeted therapy to improve effectiveness and reduce side effects.

中文翻译：

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亚洲血管畸形儿童使用西罗莫司的真实世界数据

摘要

目标

血管畸形的治疗是复杂且具有挑战性的。本研究旨在探讨西罗莫司治疗复杂血管畸形的疗效、血浆谷浓度、停药后情况和不良反应。

方法

我们中心对2017年8月至2021年6月期间使用西罗莫司（和皮质类固醇）治疗的血管畸形进行了分析。同时，我们回顾了病历、疗效、副作用和实验室检查。通过电话对停止服用西罗莫司的患者进行随访。

结果

我中心共纳入25例复杂血管畸形患者，其中女7例，男18例，年龄4个月～15岁。总共有 19 名患者（76.0%）对西罗莫司有反应，西罗莫司的血浆浓度在 0.97 至 27.15 ng/ml 之间波动。总共有 24 名患者 (96.0%) 接受了随访。随访期间共有15名患者（62.5%）停止服用西罗莫司，2名患者（13.3%）因副作用停用西罗莫司。共有 3 名患者（20.0%）重新开始西罗莫司治疗。

结论

1.5–2 mg/m ²西罗莫司的起始剂量在血管畸形治疗中是有效且安全的。最佳治疗方案和停药指征需要更多研究。最应该做的是靶向治疗，以提高疗效并减少副作用。