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. 2020 Nov;26(6):1038-1045.
doi: 10.1111/hae.14034. Epub 2020 May 20.

An international survey to evaluate systemic bevacizumab for chronic bleeding in hereditary haemorrhagic telangiectasia

Hanny Al-Samkari  1 , Hasan A Albitar  2 , Scott E Olitsky  3 , Marianne S Clancy  3 , Vivek N Iyer  4
Affiliations

An international survey to evaluate systemic bevacizumab for chronic bleeding in hereditary haemorrhagic telangiectasia

Hanny Al-Samkari et al. Haemophilia. 2020 Nov.

Abstract

Introduction: Systemic bevacizumab is a novel targeted therapy for severe epistaxis and chronic gastrointestinal bleeding in hereditary haemorrhagic telangiectasia (HHT), but published data are very limited.

Aim: We conducted a survey-based study to characterize current treatment practices and physician-reported safety and effectiveness of systemic bevacizumab for bleeding in (HHT).

Methods: A 27-item survey was sent to physician centre directors of 31 International HHT Centers of Excellence.

Results: Response rate was 84%. Approximately half of centres had treated >10 HHT patients with systemic bevacizumab for chronic bleeding for a total of 291 patients treated. All centres utilize a 5 mg/kg dose for induction treatment and most administer six doses (range, 4-8) every 2 weeks. However, maintenance regimens varied considerably between centres. Bevacizumab was highly effective, with 86% reporting significant (>50%) improvement in GI bleeding and/or epistaxis and haemoglobin rise in most patients treated with bevacizumab; 52% reported haemoglobin normalization in most patients. All centres reported adverse event rates <30% and two-thirds of centres reported adverse event rates <10%. Discontinuation for adverse events or inefficacy was rare. Bleeding severity thresholds for initiation of bevacizumab were highly variable, and it is typically administered by haematologists (76% of centres). Two-thirds of centres reported obtaining insurance approval for bevacizumab for most or all patients but 48% reported difficulty in obtaining coverage.

Conclusion: Systemic bevacizumab is widely used to treat bleeding in HHT with excellent physician-reported effectiveness and safety. There is considerable variation in maintenance treatment practices and thresholds for initiation of bevacizumab among HHT centres.

Keywords: Hereditary haemorrhagic telangiectasia; Osler-Weber-Rendu; bevacizumab; bleeding; epistaxis.

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References

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