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An international survey to evaluate systemic bevacizumab for chronic bleeding in hereditary haemorrhagic telangiectasia
Author(s) -
AlSamkari Hanny,
Albitar Hasan A.,
Olitsky Scott E.,
Clancy Marianne S.,
Iyer Vivek N.
Publication year - 2020
Publication title -
haemophilia
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.213
H-Index - 92
eISSN - 1365-2516
pISSN - 1351-8216
DOI - 10.1111/hae.14034
Subject(s) - medicine , bevacizumab , discontinuation , adverse effect , telangiectasia , surgery , pediatrics , chemotherapy
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.