biostudies-literatureKroon STopZorg by ZonMwKees Westermann Fonds, Stichting Wetenschappelijk Onderzoek Rendu OslerZonMw379-386https://www.ebi.ac.uk/biostudies/studies/S-EPMC7615075biostudies-literatureUnknown24(2)The inhibiting effects of itraconazole, an antifungal drug on vascular endothelial growth factor (VEGF) have recently been discovered. By inhibiting VEGF, itraconazole has shown potential in clinical trials as anti-cancer treatment. In hereditary hemorrhagic telangiectasia (HHT) patients, VEGF levels are elevated and inhibition of VEGF can decrease bleeding. Itraconazole could potentially serve as anti-angiogenic therapy for HHT-related bleeding. We report a proof of concept study with HHT patients and severe epistaxis. Patients were treated with daily 200 mg orally administered itraconazole for sixteen weeks. Twenty-one HHT patients, 8 females (38%), 13 males (62%), median age of 59 years (interquartile range (IQR) 55-69) were enrolled. Of these patients, 13 (62%) were diagnosed with HHT AngiogenesisOral itraconazole for epistaxis in hereditary hemorrhagic telangiectasia: a proof of concept study.PMC7615075446002501Post MCHosman AEDisch FJMVorselaars VMMKroon SSnijder RJMager JJfalseOral itraconazole for epistaxis in hereditary hemorrhagic telangiectasia: a proof of concept study.The inhibiting effects of itraconazole, an antifungal drug on vascular endothelial growth factor (VEGF) have recently been discovered. By inhibiting VEGF, itraconazole has shown potential in clinical trials as anti-cancer treatment. In hereditary hemorrhagic telangiectasia (HHT) patients, VEGF levels are elevated and inhibition of VEGF can decrease bleeding. Itraconazole could potentially serve as anti-angiogenic therapy for HHT-related bleeding. We report a proof of concept study with HHT patients and severe epistaxis. Patients were treated with daily 200 mg orally administered itraconazole for sixteen weeks. Twenty-one HHT patients, 8 females (38%), 13 males (62%), median age of 59 years (interquartile range (IQR) 55-69) were enrolled. Of these patients, 13 (62%) were diagnosed with HHT 2021-01-01T00:00:00Z2021 May2026-05-28T12:15:27.263Z2025-04-07T10:14:33.252ZS-EPMC76150753321121610.1007/s10456-020-09758-2