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. 2025 Apr 14:16:20406207241300828.
doi: 10.1177/20406207241300828. eCollection 2025.

BEST study: one-year descriptive follow-up of bevacizumab treatment in hereditary haemorrhagic telangiectasia post-BABH interventional study

Sophie Dupuis-Girod  1   2 , Evelyne Decullier  3   4 , Sophie Rivière  5 , Christian Lavigne  6 , Vincent Grobost  7 , Vanessa Leguy-Seguin  8 , Hélène Maillard  9 , Thierry Chinet  10 , Anne-Emmanuelle Fargeton  11 , Alexandre Guilhem  12 , Ruben Hermann  13
Affiliations

BEST study: one-year descriptive follow-up of bevacizumab treatment in hereditary haemorrhagic telangiectasia post-BABH interventional study

Sophie Dupuis-Girod et al. Ther Adv Hematol. .

Abstract

Background: Hereditary haemorrhagic telangiectasia (HHT) is a genetic vascular disorder characterised by telangiectases, which cause nasal and gastrointestinal (GI) bleeding, and visceral arteriovenous malformations. Since 2012 bevacizumab, a monoclonal antibody targeting vascular endothelial growth factor, has been a promising treatment for HHT-related bleeding and was evaluated in the phase II BABH study.

Objective: To follow and describe evolution and treatments of patients with HHT post-BABH study.

Design: This study is a 1-year, multi-centre descriptive study.

Methods: We collected clinical (nose and GI bleeding, red blood cell transfusions) and biological (haemoglobin and ferritin levels) data and treatment information.

Results: Of 22 patients included across 4 centers, 15 received bevacizumab. Among them, 12 (86%) had a >50% decrease in the number of RBC units transfused 3 months post-treatment. Mean haemoglobin levels increased from 83.08 to 105.98 g/L.

Conclusion: Bevacizumab effectively reduces RBC transfusions and is efficient for treating severe bleeding in patients with HHT.

Trial registration: This trial was registered with the ClinicalTrials.gov Identifier #NCT06039124.

Keywords: bevacizumab; hereditary haemorrhagic telangiectasia; rare vascular disease; red blood cell transfusion.

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Conflict of interest statement

The authors declare that there is no conflict of interest.

Figures

Figure 1.
Figure 1.
Flow chart.
Figure 2.
Figure 2.
Haemoglobin levels in the 3-month periods before treatment, during treatment and after treatment. As a reminder, we provided the data for the BABH study (in grey) for these patients. There might be some overlap between the BABH-after period and the BEST-before period for patients being retreated right after the end of the study. For haemoglobin, during the BABH study, there was only one measure per timepoint.
Figure 3.
Figure 3.
Number of RBC units transfused in the 3-month periods before treatment, during treatment and after treatment. As a reminder, we provided the data for the BABH study (in grey) for these patients. There might be some overlap between the BABH-after period and the BEST-before period for patients being retreated right after the end of the study.
See this image and copyright information in PMC

References

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    1. Dupuis-Girod S, Rivière S, Lavigne C, et al.. Efficacy and safety of intravenous bevacizumab on severe bleeding associated with hemorrhagic hereditary telangiectasia: a national, randomized multicenter trial. J Intern Med. Epub ahead of print 17 August 2023. DOI: 10.1111/joim.13714. - DOI - PubMed
    1. Al-Samkari H. Even effective drugs require adequately powered trials: systemic bevacizumab in hereditary hemorrhagic telangiectasia. J Intern Med. Epub ahead of print 22 August 2023. DOI: 10.1111/joim.13713. - DOI - PMC - PubMed
    1. Dupuis-Girod S, Ginon I, Saurin J-C, et al.. Bevacizumab in patients with hereditary hemorrhagic telangiectasia and severe hepatic vascular malformations and high cardiac output. JAMA 2012; 307: 948–955. - PubMed
    1. Chavan A, Schumann-Binarsch S, Luthe L, et al.. Systemic therapy with bevacizumab in patients with hereditary hemorrhagic telangiectasia (HHT). Vasa 2013; 42: 106–110. - PubMed

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