Sirolimus for Venous Malformations: A Systematic Review of Efficacy and Safety.

IF 1.9 4区 医学 Q3 MEDICINE, RESEARCH & EXPERIMENTAL
Joyce Teng, Jeff Martini, Michael Kelly, Megha Tollefson, Alexander Greer
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引用次数: 0

Abstract

Background: Venous malformations can cause substantial morbidity and long-term complications. There are no Food and Drug Administration (FDA)-approved therapies for the treatment of venous malformations. However, off-label use of sirolimus has demonstrated clinical benefit in these patients. This systematic review evaluates the efficacy and safety of sirolimus in the management of venous malformations. Methods and Results: A systematic literature search identified 26 studies comprising 98 patients treated with sirolimus for venous malformations. Most studies were case reports or case series (77%), with 15% prospective trials and 8% retrospective analyses. Sirolimus, primarily administered orally, yielded some level of clinical improvement in 72% of reported patients, with consistent symptomatic relief observed in coagulopathy, bleeding, anemia, pain, and improved function. However, reduction in the size of the malformation varied and treatment-limiting side effects were observed. Conclusions: Oral sirolimus is a promising treatment option for venous malformations, especially for those with deep venous malformations. A direct, targeted topical treatment could provide clinical benefit for the cutaneous manifestation without the risk of off-target effects as occur with oral sirolimus. Further prospective, controlled studies are warranted to elucidate sirolimus's role in managing venous malformations.

西罗莫司治疗静脉畸形:有效性和安全性的系统评价。
背景:静脉畸形可引起严重的发病率和长期并发症。目前还没有食品和药物管理局(FDA)批准的治疗静脉畸形的疗法。然而,在这些患者中,未经核准使用西罗莫司已证明有临床益处。本系统综述评价了西罗莫司治疗静脉畸形的有效性和安全性。方法和结果:系统的文献检索确定了26项研究,包括98例使用西罗莫司治疗静脉畸形的患者。大多数研究为病例报告或病例系列(77%),其中15%为前瞻性试验,8%为回顾性分析。西罗莫司主要口服给药,在72%的报告患者中产生了一定程度的临床改善,在凝血病、出血、贫血、疼痛和功能改善方面观察到一致的症状缓解。然而,在缩小的大小不一的畸形和治疗限制的副作用观察。结论:口服西罗莫司是治疗静脉畸形,尤其是深静脉畸形的理想选择。直接,有针对性的局部治疗可以为皮肤表现提供临床益处,而不会像口服西罗莫司那样出现脱靶效应的风险。进一步的前瞻性对照研究需要阐明西罗莫司在静脉畸形治疗中的作用。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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来源期刊
Lymphatic research and biology
Lymphatic research and biology Medicine-Cardiology and Cardiovascular Medicine
CiteScore
3.10
自引率
7.10%
发文量
85
审稿时长
>12 weeks
期刊介绍: Lymphatic Research and Biology delivers the most current peer-reviewed advances and developments in lymphatic biology and pathology from the world’s leading biomedical investigators. The Journal provides original research from a broad range of investigative disciplines, including genetics, biochemistry and biophysics, cellular and molecular biology, physiology and pharmacology, anatomy, developmental biology, and pathology. Lymphatic Research and Biology coverage includes: -Vasculogenesis and angiogenesis -Genetics of lymphatic disorders -Human lymphatic disease, including lymphatic insufficiency and associated vascular anomalies -Physiology of intestinal fluid and protein balance -Immunosurveillance and immune cell trafficking -Tumor biology and metastasis -Pharmacology -Lymphatic imaging -Endothelial and smooth muscle cell biology -Inflammation, infection, and autoimmune disease
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