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口腔和局部用西罗莫司治疗血管异常:一项多中心研究和综述。

Oral and Topical Sirolimus for Vascular Anomalies: A Multicentre Study and Review.

机构信息

Department of Dermatology, Sheba Medical Center, Sackler School of Medicine, Tel Aviv University, Tel Aviv, Israel.

出版信息

Acta Derm Venereol. 2019 Oct 1;99(11):990-996. doi: 10.2340/00015555-3262.

DOI:10.2340/00015555-3262
PMID:31304557
Abstract

Vascular anomalies (VAs) may be associated with significant morbidity and mortality. The aim of this study was to evaluate the efficacy and safety of sirolimus (rapamycin) in the treatment of children and young adults with complicated VAs. A retrospective chart was created that included 19 patients treated with sirolimus for complicated VAs. Concurrently, a search of the PubMed database for VA cases treated with sirolimus was conducted. Descriptive analysis was performed and the efficiency rate of sirolimus was calculated. This retrospective study included 19 patients, 17 of whom were treated with oral sirolimus and 2 with topical sirolimus. Clinical improvement occurred in 15 patients (79%). One patient experienced near-complete resolution. Only 2 patients showed poor response and discontinued treatment. The literature review analysed 150 cases of VA treated with sirolimus. Sirolimus was efficient in 85% of cases, including 5 cases of complete resolution. Sirolimus appears to be an effective and safe treatment for children and young adults with complicated VAs.

摘要

血管异常(VA)可能与显著的发病率和死亡率相关。本研究旨在评估西罗莫司(雷帕霉素)治疗儿童和青年复杂性 VA 的疗效和安全性。创建了一份回顾性图表,其中包括 19 名接受西罗莫司治疗复杂性 VA 的患者。同时,在 PubMed 数据库中对接受西罗莫司治疗的 VA 病例进行了搜索。进行了描述性分析,并计算了西罗莫司的有效率。这项回顾性研究包括 19 名患者,其中 17 名接受口服西罗莫司治疗,2 名接受局部西罗莫司治疗。15 名患者(79%)出现临床改善。1 名患者接近完全缓解。仅 2 名患者反应不佳并停止治疗。文献复习分析了 150 例接受西罗莫司治疗的 VA 病例。西罗莫司在 85%的病例中有效,包括 5 例完全缓解。西罗莫司似乎是治疗儿童和青年复杂性 VA 的一种有效且安全的治疗方法。

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