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, commonly presenting with epistaxis and gastrointestinal (GI) 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 receptor.

Objectives: To report single-center, retrospective real-world use of pazopanib, an orally administered tyrosine kinase inhibitor that blocks vascular endothelial growth factor receptors, in 6 patients with HHT-associated epistaxis and/or GI bleeding.

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 January 1, 2019, and June 14, 2023. The Indiana Hemophilia and Thrombosis institutional electronic medical record 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 review board approval was obtained for data pull as an exempt study.

Results: Our observations on the real-world use of pazopanib in 6 HHT patients with moderate-to-severe bleeding showed improvement in hemoglobin levels, with reduction in iron infusions and red blood cell transfusion requirement.

Conclusion: Pazopanib may be a reasonable option for patients with HHT with epistaxis or gastrointestinal bleeding that are refractory to standard treatment.