Hereditary hemorrhagic telangiectasia (HHT) is an inherited vascular dysplasia characterized by mucocutaneous
telangiectasia and visceral
arteriovenous malformations. Hepatic involvement with
vascular malformations may lead to
portal hypertension, biliary
ischemia, and high-output cardiac failure. There is no curative treatment for the disease.
Liver transplantation is indicated for life-threatening complications, but it carries significant risk due to surgery and immunosuppressive treatment. Some case reports or small open studies suggest that
bevacizumab, a recombinant humanized anti-
VEGF monoclonal antibody, should be efficient in limiting
bleeding and in reducing
liver disease in HHT.We report a case of a 63-year-old woman with HHT presenting with ischemic cholangiopathy.
Liver transplant was indicated, but given a previous encouraging report showing a regression of biliary disease with
bevacizumab in 3 patients with HHT this
drug was proposed. No significant efficacy but a severe adverse effect was observed after 3 months: bilateral
pulmonary embolisms,
thrombosis in the right atrial cavity, and
thrombosis of the right hepatic vein were evidenced.
Bevacizumab was stopped;
anticoagulant started. Four months later, the patient received a transplanted liver. She feels well 1 year later.This case report intends to provide the information for clinicians to consider the use of
bevacizumab in HHT. Whereas several uncontrolled series and case reports have suggested the efficacy of this
drug in reducing
bleeding and
liver disease, no severe side effects were mentioned to date. For the first time in HHT we report a life-threatening side effect of this
drug and no efficacy. Moreover, systemic
thrombosis, the observed complication, may preclude
transplantation. To date, caution seems still indispensable when considering the use of
bevacizumab in HHT.