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.