RT Journal Article
T1 Review of Pharmacological Strategies with Repurposed Drugs for Hereditary Hemorrhagic Telangiectasia Related Bleeding
A1 Albiñana, Virginia
A1 Cuesta Martínez, Ángel
A1 Rojas-P., Isabel de
A1 Gallardo-Vara, Eunate
A1 Recio-Poveda, Lucía
A1 Bernabéu, Carmelo
A1 Botella, Luisa María
AB The 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.
PB MDPI
SN 2077-0383
YR 2020
FD 2020-06-06
LK https://hdl.handle.net/20.500.14352/114718
UL https://hdl.handle.net/20.500.14352/114718
LA eng
NO Albiñana 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/2077-0383/9/6/1766
NO Ministerio de Economía y Competitividad (España)
DS Docta Complutense
RD 5 abr 2025