Successful therapy with bevacizumab in a case of hereditary hemorrhagic telangiectasia.

Hereditary hemorrhagic telangiectasia (HHT), genetic disorder manifested by uncontrolled multisystem angiogenesis with epistaxis, gastrointestinal bleeding, iron-deficiency anaemia, and arteriovenous malformations (AVM) is often related with increased levels of vascular endothelial growth factor (VEGF). Bevacizumab, a VEGF inhibitor, reduces epistaxis, telangiectasias, and iron-deficiency anaemia. A case of a female patient with HHT and chronic gastrointestinal bleeding is presented. The patient required iron supplementation and multiple blood transfusions. Bevacizumab brought marked symptomatic improvement and allowed for transfusion-independence. It is intended to apply for approval of the indications for bevacizumab administration in HHT as the 'orphan drug'.