Systemic bevacizumab for the treatment of chronic bleeding in hereditary haemorrhagic telangiectasia

Background Hereditary haemorrhagic telangiectasia (HHT) is a rare hereditary multisystem vascular disorder causing visceral arteriovenous malformations and mucocutaneous bleeding. Chronic gastrointestinal bleeding and epistaxis often produce profound anaemia refractory to conventional treatment. Bevacizumab, an anti‐vascular endothelial growth factor monoclonal antibody, may be effective in treatment of bleeding in HHT. Methods All HHT patients treated with systemic bevacizumab for chronic bleeding were selected for retrospective analysis. Data collected included demographics, baseline HHT characteristics, epistaxis grade, surgical interventions, bevacizumab dosing, adverse events, haemoglobin, red cell transfusions, intravenous iron infusions, and other anaemia and/or bleeding‐directed therapies. Results Thirteen HHT patients were treated with bevacizumab for a median of 13.9 (range 4.9–30.1) months. Compared with pretreatment values, bevacizumab treatment increased the mean haemoglobin by 4.0 g dL−1 (95% CI, 2.6–5.3 g dL−1) [mean (95% CI) haemoglobin 8.5 (7.8, 9.9) g dL−1 vs. 12.5 (11.2, 13.7) g dL−1, P < 0.001)], reduced red cell units transfused by 92% [median of 6 (range 0–59) units vs. 0 (range 0–15) units, P = 0.004] and reduced quantity of iron infused by 73% [mean (95% CI) 462 (257, 668) mg month−1 vs. 126 (75, 178) mg month−1, P = 0.002]. Epistaxis control was achieved in 85% with bevacizumab versus 0% before treatment (P < 0.001). No patient required nasal or GI procedures during the maintenance period. Two patients (15%) developed grade 3 hypertension requiring medical management. Conclusion Systemic bevacizumab was highly effective to treat chronic bleeding in HHT. Further study is needed to confirm the magnitude of benefit and further define optimal dosing, treatment duration and long‐term safety.