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 type 1, seven (33%) with HHT type 2 and in one patient (5%), no pathognomonic HHT mutation was found. Four patients (19%) prematurely terminated the study (3 due to mild or moderate side-effects) resulting in 17 patients included in the analyses. The median epistaxis severity score significantly decreased during treatment from 6.0 (IQR 5.1–7.2) to 3.8 (IQR 3.1–5.2) (p = 0.006). The monthly epistaxis frequency decreased from 56 to 38 epistaxis episodes (p = 0.004) and the monthly duration from 407 to 278 minutes (p = 0.005). Hemoglobin levels did not significantly change. The quality of life showed a small but significant improvement. In conclusion, oral itraconazole significantly improved epistaxis in HHT patients. The potential benefit of itraconazole in HHT should be further investigated.

最近发现了抗真菌药物伊曲康唑对血管内皮生长因子（VEGF）的抑制作用。通过抑制 VEGF，伊曲康唑在临床试验中显示出抗癌治疗的潜力。在遗传性出血性毛细血管扩张症 (HHT) 患者中，VEGF 水平升高，抑制 VEGF 可以减少出血。伊曲康唑有可能作为 HHT 相关出血的抗血管生成疗法。我们报告了一项针对 HHT 患者和严重鼻出血的概念验证研究。患者每天口服 200 mg 伊曲康唑治疗 16 周。纳入 21 名 HHT 患者，其中 8 名女性 (38%)、13 名男性 (62%)，中位年龄 59 岁（四分位距 (IQR) 55-69）。在这些患者中，13 名 (62%) 被诊断为 HHT 1 型，7 名 (33%) 被诊断为 HHT 2 型，1 名患者 (5%) 未发现特征性 HHT 突变。4 名患者 (19%) 提前终止了研究（其中 3 名患者因轻度或中度副作用），导致 17 名患者纳入分析。治疗期间中位鼻出血严重程度评分从 6.0 (IQR 5.1–7.2) 显着下降至 3.8 (IQR 3.1–5.2) ( p  = 0.006)。每月鼻出血频率从 56 次减少至 38 次（p  = 0.004），每月持续时间从 407 分钟减少至 278 分钟（p  = 0.005）。血红蛋白水平没有显着变化。生活质量出现了微小但显着的改善。总之，口服伊曲康唑显着改善 HHT 患者的鼻出血。伊曲康唑在 HHT 中的潜在益处应进一步研究。