UNASSIGNED: Between 2015 and 2024, we administered sirolimus to 14 pediatric patients with various types of vascular anomalies in two Italian centers, subjecting them to clinical and instrumental follow-up to investigate its efficacy and the possible occurrence of adverse events.
UNASSIGNED: An overall improvement in or stability of their vascular anomalies was reported by 86% of patients. We also assessed toxicity, noting a low prevalence of life-threatening adverse events: only one case of sepsis was reported in a patient with a severe clinical condition, and four cases of recurrent aphthosis (28%) were reported. The most common side effect was dyslipidemia, with 43% of patients developing hypercholesterolemia (21%) or hypertriglyceridemia (21%), although these patients generally did not reach severe levels.
UNASSIGNED: In line with data in the literature, according to our experience, medical therapy with sirolimus should be considered in pediatric patients affected by vascular anomalies.
■在2015年至2024年之间,我们在两个意大利中心对14名患有各种类型血管异常的儿科患者进行了西罗莫司治疗,对其进行临床和仪器随访,以调查其疗效和不良事件的可能发生情况。
■86%的患者报告其血管异常总体改善或稳定。我们还评估了毒性,注意到危及生命的不良事件的发生率较低:只有一例败血症在严重临床疾病的患者中报告,报告了4例复发性口疮(28%)。最常见的副作用是血脂异常,43%的患者出现高胆固醇血症(21%)或高甘油三酯血症(21%),尽管这些患者通常没有达到严重水平。
■与文献中的数据一致,根据我们的经验,对于受血管异常影响的儿科患者,应考虑使用西罗莫司进行药物治疗。