Avastin for the treatment of hereditary hemorrhagic telangiectasia (HHT) – pro
There are unfortunately only case reports and series to support the use fo Avastin for HHT.there is a great of interest in Avastin for this condition (1, 2, 4, 5). In a phase II study, bevacizumab (5 mg/kg given every 14 days for a total of six injections) was given to 25 patients with severe liver involvement and a high cardiac index related to HHT, in whom liver transplantation was not feasible. At six months, the cardiac index normalized in 5 of the 23 evaluable patients and was reduced in another 15, for an overall response rate of 87 percent (. 2) The mean duration of epistaxis decreased from 221 minutes/month at the start of treatment to 134 minutes/month at six months, along with a significant improvement in quality of life.
Reviews, such as Up-to-date and Ref. 3 state that the long term efficacy and safety of Avastin for the treatment of HHT has not been established due to the lack of data from randomized placebo-controlled clinical trials.
1. Shovlin C, Ganesan V. Hereditary hemorrhagic telangiectasia (Osler-Weber-Rendu syndrome). In: UpToDate, Basow, DS (Ed), UpToDate, Waltham, MA, 2014. Literature review current through October 2014
2. Dupuis-Girod S, Ginon I, Saurin JC, et al. Bevacizumab in patients with hereditary hemorrhagic telangiectasia and severe hepatic vascular malformations and high cardiac output. JAMA 2012; 307:948.
3. Shovlin CL. Hereditary haemorrhagic telangiectasia: pathophysiology, diagnosis and treatment. Blood Rev 2010; 24:203.
4. Simonds J, Miller F, Mandel J, Davidson TM. The effect of bevacizumab (Avastin) treatment on epistaxis in hereditary hemorrhagic telangiectasia. Laryngoscope 2009; 119:988.
5. Bose P, Holter JL, Selby GB. Bevacizumab in hereditary hemorrhagic telangiectasia. N Engl J Med 2009; 360:2143.