Current and emerging pharmacotherapies for treating vascular malformations.

IF 3.6 3区医学 Q2 PHARMACOLOGY & PHARMACY

Expert Review of Clinical Pharmacology Pub Date : 2026-04-01 Epub Date: 2026-03-17 DOI:10.1080/17512433.2026.2641807

Emmanuel Seront, An Van Damme, Julien Coulie, Laurence M Boon, Miikka Vikkula

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引用次数: 0

Abstract

Introduction: Vascular malformations are chronic, often progressive disorders for which conventional procedures frequently provide incomplete control. Advances in molecular genetics have revealed recurrent pathway alterations that now enable mechanism-based pharmacologic treatment.

Areas covered: This narrative review summarizes current and emerging targeted therapies for vascular malformations, including mTOR, PI3K, MEK, anti-angiogenic, and genotype-specific inhibitors. Vascular malformations - capillary, lymphatic, venous, and arteriovenous - are increasingly recognized as disorders driven by dysregulation of the PI3K - AKT - mTOR and RAS - MAPK - ERK pathways, supporting rational repurposing of targeted anticancer drugs. We discuss clinical evidence, limitations, and practical considerations for sirolimus, PI3K inhibitors, thalidomide, MEK inhibitors, and KRAS-directed agents, as well as emerging combination and intermittent strategies to balance efficacy and toxicity. The review is based on a structured PubMed/MEDLINE search up to December 2025, prioritizing original translational studies, prospective trials, and large clinical series.

Expert opinion: Targeted therapies are shifting management from procedure-centered to biology-guided care. Future progress depends on standardized molecular testing, patient-centered outcomes, and optimized treatment duration to achieve durable disease control with acceptable long-term toxicity.

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当前和新兴的治疗血管畸形的药物疗法。

简介：血管畸形是慢性的，通常是进行性疾病，常规手术往往不能完全控制。分子遗传学的进展揭示了复发性通路改变，现在使基于机制的药物治疗成为可能。涵盖领域：本文综述了当前和新兴的血管畸形靶向治疗，包括mTOR、PI3K、MEK、抗血管生成和基因型特异性抑制剂。血管畸形-毛细血管、淋巴管、静脉和动静脉-越来越多地被认为是由PI3K - AKT - mTOR和RAS - MAPK - ERK通路失调引起的疾病，支持靶向抗癌药物的合理重新利用。我们讨论了西罗莫司、PI3K抑制剂、沙利度胺、MEK抑制剂和kras定向药物的临床证据、局限性和实际考虑，以及平衡疗效和毒性的新组合和间歇性策略。该综述基于到2025年12月的结构化PubMed/MEDLINE检索，优先考虑原始转化研究、前瞻性试验和大型临床系列。专家意见：靶向治疗正在将管理从以手术为中心转向以生物学为指导的护理。未来的进展取决于标准化的分子检测、以患者为中心的结果和优化的治疗时间，以实现持久的疾病控制和可接受的长期毒性。

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

Expert Review of Clinical Pharmacology PHARMACOLOGY & PHARMACY-

CiteScore

7.30

自引率

2.30%

发文量

127

期刊介绍： Advances in drug development technologies are yielding innovative new therapies, from potentially lifesaving medicines to lifestyle products. In recent years, however, the cost of developing new drugs has soared, and concerns over drug resistance and pharmacoeconomics have come to the fore. Adverse reactions experienced at the clinical trial level serve as a constant reminder of the importance of rigorous safety and toxicity testing. Furthermore the advent of pharmacogenomics and ‘individualized’ approaches to therapy will demand a fresh approach to drug evaluation and healthcare delivery. Clinical Pharmacology provides an essential role in integrating the expertise of all of the specialists and players who are active in meeting such challenges in modern biomedical practice.