PDF(Pubmed)
Abstract：
UNASSIGNED: Vascular anomalies (VAs) constitute a heterogeneous group of tumors and malformations capable of inducing significant clinical events in specific patients, such as the compression of vital organs, pain, functional impairment, or acquired coagulopathy. Molecular investigations into the underlying mechanisms of VAs have unveiled the frequent involvement of the PI3 K/AKT/mTOR pathway. Sirolimus, a specific mTOR inhibitor, has emerged as a potential therapeutic agent; however, its routine clinical application in complex VAs is currently restricted by a lack of extensive clinical experience.
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.
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.
摘要：
■血管异常（VA）构成了一组异质性的肿瘤和畸形，能够在特定患者中引起重大临床事件，比如重要器官的压迫，疼痛,功能损害，或获得性凝血病。对VAs潜在机制的分子研究揭示了PI3K/AKT/mTOR通路的频繁参与。西罗莫司,一种特定的mTOR抑制剂，已经成为一种潜在的治疗剂；然而，目前，由于缺乏丰富的临床经验，其在复杂血管腔中的常规临床应用受到限制。
■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。
■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。
