Artner Anna, Diler Irem, Hankó Balázs, Sebők Szilvia, Zelkó Romána
Center of Pharmacology and Drug Research & Development, 1085 Budapest, Hungary.
University Pharmacy Department of Pharmacy Administration, Semmelweis University, 1085 Budapest, Hungary.
J Clin Med. 2025 Aug 1;14(15):5436. doi: 10.3390/jcm14155436.
Statins exhibit pleiotropic anti-inflammatory, antioxidant, and immunomodulatory effects, suggesting their potential in non-cardiovascular conditions. However, evidence supporting their repurposing remains limited, and off-label prescribing policies vary globally. To systematically review evidence on statin repurposing in oncology and infectious diseases, and to assess Hungarian regulatory practices regarding off-label statin use. A systematic literature search (PubMed, Web of Science, Scopus, ScienceDirect; 2010-May 2025) was conducted using the terms "drug repositioning" OR "off-label prescription" AND "statin" NOT "cardiovascular," following PRISMA guidelines. Hungarian off-label usage data from the NNGYK (2008-2025) were also analyzed. Out of 205 publications, 12 met the inclusion criteria-75% were oncology-focused, and 25% focused on infectious diseases. Most were preclinical (58%); only 25% offered strong clinical evidence. Applications included hematologic malignancies, solid tumors, Cryptococcus neoformans, SARS-CoV-2, and dengue virus. Mechanisms involved mevalonate pathway inhibition and modulation of host immune responses. Hungarian data revealed five approved off-label statin uses-three dermatologic and two pediatric metabolic-supported by the literature and requiring post-treatment reporting. While preclinical findings are promising, clinical validation of off-label statin use remains limited. Statins should be continued in cancer patients with cardiovascular indications, but initiation for other purposes should be trial-based. Future directions include biomarker-based personalization, regulatory harmonization, and cost-effectiveness studies.
他汀类药物具有多效性的抗炎、抗氧化和免疫调节作用，表明它们在非心血管疾病方面具有潜力。然而，支持其重新用途的证据仍然有限，且全球各地的非标签处方政策各不相同。本研究旨在系统回顾他汀类药物在肿瘤学和传染病领域重新用途的证据，并评估匈牙利关于他汀类药物非标签使用的监管实践。按照PRISMA指南，使用“药物重新定位”或“非标签处方”以及“他汀类药物”而非“心血管”等术语，对（PubMed、Web of Science、Scopus、ScienceDirect；2010年至2025年5月）进行了系统的文献检索。还分析了来自NNGYK（2008年至2025年）的匈牙利非标签使用数据。在205篇出版物中，12篇符合纳入标准，其中75%聚焦于肿瘤学，25%聚焦于传染病。大多数是临床前研究（58%）；只有25%提供了有力的临床证据。应用包括血液系统恶性肿瘤、实体瘤、新型隐球菌、严重急性呼吸综合征冠状病毒2和登革热病毒。作用机制涉及甲羟戊酸途径抑制和宿主免疫反应调节。匈牙利的数据显示有五种批准的他汀类药物非标签用途，其中三种用于皮肤科，两种用于儿科代谢疾病，这些用途得到了文献支持且需要治疗后报告。虽然临床前研究结果很有前景，但他汀类药物非标签使用的临床验证仍然有限。对于有心血管适应症的癌症患者，应继续使用他汀类药物，但出于其他目的开始使用时应以试验为基础。未来的方向包括基于生物标志物的个性化、监管协调和成本效益研究。
