Висока важливість
19 серп. 2026 р.
To evaluate the use and effectiveness of bevacizumab as a treatment option for patients with hereditary hemorrhagic telangiectasia (HHT) in France.
This retrospective analysis included cohort data from patients with HHT or Rendu-Osler disease treated with intravenous bevacizumab from 2009 to 2024 in French expert centres. The analysis focused on the treatment outcomes related to epistaxis management, anaemia, transfusion dependency, and overall quality of life.
The analysis indicated a significant reduction in the frequency and severity of epistaxis among patients treated with bevacizumab, alongside improvements in haemoglobin levels and a decrease in the need for blood transfusions. Enhanced quality of life metrics were also reported by patients following treatment.
The retrospective design limits the ability to draw definitive causal relationships and to standardise treatment protocols across different centres. Additionally, the variances in local regulations regarding treatment access could affect the generalisability of the findings across different populations.
This research is crucial as it provides evidence supporting the off-label use of bevacizumab in managing HHT, highlighting its potential to significantly improve patient outcomes in a condition that often leads to severe complications and diminished quality of life. It contributes to the ongoing discourse about treatment guidelines and regulatory measures for HHT.
Показано англійською
Rendu-Osler disease (RO), also known as Hereditary Haemorrhagic Telangiectasia (HHT), is an autosomal dominant genetic disorder. Its pathophysiology is associated with an imbalance in angiogenic balance. The resulting epistaxis and digestive bleeding can be severe, requiring repeated transfusions in 5% of patients. In addition, some patients with HHT may present with liver damage, leading to high-flow heart failure. Vascular Endothelial Growth Factor (VEGF) plays a role in the pathophysiology of Rendu-Osler disease, leading to the use of a recombinant anti-VEGF monoclonal antibody, bevacizumab. Studies conducted and promoted by HCL have demonstrated the efficacy and safety of bevacizumab in patients with HHT. In 2014, bevacizumab was granted orphan drug designation for this disease. The 2020 international guidelines for the diagnosis and management of HHT suggest the use of systemic antiangiogenic agents in cases of epistaxis resistant to other treatments. The use of bevacizumab has been widely published in uncontrolled studies, demonstrating its efficacy in reducing epistaxis, improving anaemia, reducing transfusion dependence and improving quality of life. In France, bevacizumab is currently prescribed outside the scope of its marketing authorization. Treatment indications are validated within the French network of expert centres and the FAVAmulti rare disease network. In other countries, access to this treatment varies widely according to local regulations, and is currently being evaluated in the European VASCERN network. The aim of this study is to describe the use of bevacizumab in patients treated for HHT in France.