Pazopanib in treatment of Hereditary Hemorrhagic Telangiectasia-related epistaxis and gastrointestinal bleeding.

Background: Hereditary hemorrhagic telangiectasia (HHT) is a bleeding disorder characterized by arteriovenous malformations (AVMs), commonly presenting with epistaxis and gastrointestinal bleeding. Bleeding symptoms may be difficult to manage and may become life-threatening, with many patients developing dependence on parenteral iron and/or blood transfusion. There is a growing body of evidence that antiangiogenic therapies may be effective in management of bleeding symptoms, presumably targeting pathogenic HHT pathways such as vascular endothelial growth factor (VEGF) receptor.

Objectives: To report single-center, retrospective real-world use of pazopanib, an orally administered tyrosine kinase inhibitor that blocks VEGF receptors, in six patients with HHT-associated epistaxis and/or gastrointestinal (GI) bleeding.

Patient/methods: A retrospective observational analysis was performed to assess the safety/efficacy of pazopanib use in patients with confirmed HHT-associated epistaxis and/or GI bleeding between 01 January 2019 and 14 June 2023 The Indiana Hemophilia and Thrombosis (IHTC) institutional EMR was queried for HHT patients who were treated with pazopanib for >3 months. Patient data were obtained from patient documentation, physician/nursing notes, and on-call documentation Institutional IRB approval was obtained for data pull as an exempt study RESULTS AND CONCLUSIONS: Our observations on the real-world use of pazopanib in six HHT patients with moderate to severe bleeding showed improvement in hemoglobin levels, with reduction in iron infusions and red blood cell transfusion requirement. Pazopanib may be a reasonable option for patients with HHT with epistaxis or gastrointestinal bleeding that are refractory to standard treatment.