Patients with hereditary haemorrhagic telangiectasia (HHT) who suffer from severe epistaxis (nosebleeds) or gastrointestinal bleeding may benefit from intravenous bevacizumab, according to a new study.
In severe HHT, malformed blood vessels in the nasal cavity and gastrointestinal tract are prone to spontaneous and recurrent bleeding, and patients require frequent red blood cell transfusions and iron infusions.
Bevacizumab is an inhibitor of vascular endothelial growth factor A (VEGF), a factor that promotes blood vessel growth, and has been shown reduce HHT bleeding in a small number of studies.
Now, in a larger-scale study, a US team demonstrates that the drug could be an effective treatment for even the most severe cases.
Dr Vivek Iyer of the Mayo Clinic, Rochester, Minnesota, USA, and colleagues recruited 34 patients with severe HHT. Over the course of three-and-a-half years, all were given at least 4 doses of intravenous bevacizumab spaced at two-week to monthly intervals.
After bevacizumab therapy, there was a significant reduction in epistaxis severity scores and number of red blood cell transfusions. New onset or worsened hypertension was noted in 4 patients.
Writing in Mayo Clinic Proceedings the authors state: "Intravenous bevacizumab is an effective treatment option for patients with severe anaemia-related to epistaxis and/or gastrointestinal bleeding. Further studies are needed to establish a dose-response relationship as well as clinical, genetic, and biomarker predictors of response."
Dr Iyer said: "Some hereditary haemorrhagic telangiectasia patients suffer from severe epistaxis and gastrointestinal bleeding, which can result in severe anaemia and years of blood transfusions. Both problems also appear to sometimes worsen with age. Quality of life is very poor in these cases.
"Currently, there are no Food and Drug Administration-approved pharmacological treatments available for these patients with severe bleeding.
"This study provides good quality evidence for the excellent efficacy and safety of intravenous bevacizumab in the treatment of these patients. Intravenous bevacizumab should be considered as a standard, first-line treatment option."
Source: Iyer, V. N. et al. Intravenous Bevacizumab for Refractory Hereditary Hemorrhagic Telangiectasia-Related Epistaxis and Gastrointestinal Bleeding. Mayo Clinic Proceedings 29 January 2018 doi: 10.1016/j.mayocp.2017.11.013
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