Albiñana, VirginiaCuesta Martínez, ÁngelRojas-P., Isabel deGallardo-Vara, EunateRecio-Poveda, LucíaBernabéu, CarmeloBotella, Luisa María2025-01-162025-01-162020-06-06Albiñana V, Cuesta AM, De Rojas-P I, Gallardo-Vara E, Recio-Poveda L, Bernabéu C, et al. Review of pharmacological strategies with repurposed drugs for hereditary hemorrhagic telangiectasia related bleeding. JCM [Internet]. 6 de junio de 2020 [citado 16 de enero de 2025];9(6):1766. Disponible en: https://www.mdpi.com/2077-0383/9/6/17662077-038310.3390/jcm9061766https://hdl.handle.net/20.500.14352/114718The diagnosis of hereditary hemorrhagic telangiectasia (HHT) is based on the Curaçao criteria: epistaxis, telangiectases, arteriovenous malformations in internal organs, and family history. Genetically speaking, more than 90% of HHT patients show mutations in ENG or ACVRL1/ALK1 genes, both belonging to the TGF-β/BMP9 signaling pathway. Despite clear knowledge of the symptoms and genes of the disease, we still lack a definite cure for HHT, having just palliative measures and pharmacological trials. Among the former, two strategies are: intervention at "ground zero" to minimize by iron and blood transfusions in order to counteract anemia. Among the later, along the last 15 years, three different strategies have been tested: (1) To favor coagulation with antifibrinolytic agents (tranexamic acid); (2) to increase transcription of ENG and ALK1 with specific estrogen-receptor modulators (bazedoxifene or raloxifene), antioxidants (N-acetylcysteine, resveratrol), or immunosuppressants (tacrolimus); and (3) to impair the abnormal angiogenic process with antibodies (bevacizumab) or blocking drugs like etamsylate, and propranolol. This manuscript reviews the main strategies and sums up the clinical trials developed with drugs alleviating HHT.engAttribution-NonCommercial-NoDerivatives 4.0 Internationalhttp://creativecommons.org/licenses/by-nc-nd/4.0/journal articlehttps://doi.org/10.3390/jcm9061766open access577.1577.2HHTN-acetylcysteineetamsylateFK506propranololtranexamic acidbazedoxifeneraloxifeneendoglinALK1Biología molecular (Farmacia)Bioquímica (Farmacia)24 Ciencias de la Vida