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Effect of oral nintedanib vs placebo on epistaxis in hereditary hemorrhagic telangiectasia: the EPICURE multicenter randomized double-blind trial

Effect of Oral Nintedanib vs Placebo on Epistaxis in Hereditary Hemorrhagic Telangiectasia

Study Overview

Epistaxis, or nosebleeds, significantly impacts patients with hereditary hemorrhagic telangiectasia (HHT). Nintedanib, known for its ability to inhibit blood vessel growth, was tested for its effectiveness in reducing the duration of these nosebleeds.

Trial Details

This study was a multicenter, phase 2 trial conducted from June 2020 to February 2023. It involved:

  • Participants aged 18 and older with a confirmed HHT diagnosis.
  • Patients experiencing moderate to severe nosebleeds (epistaxis severity score > 4).

Results

Sixty patients were randomly assigned to receive either nintedanib or a placebo for 12 weeks, followed by an additional 12 weeks of monitoring. Key findings include:

  • 56 patients completed the trial.
  • 43% of the nintedanib group achieved a significant reduction in nosebleed duration compared to 27% in the placebo group.
  • Median epistaxis duration decreased by 57% in the nintedanib group versus 27% in the placebo group.
  • Hemoglobin levels increased significantly in the nintedanib group (+18 g/L) compared to a decrease in the placebo group (-1 g/L).

Conclusion

While the primary goal of reducing epistaxis duration by 50% was not met, the results indicate that nintedanib can significantly reduce the duration of nosebleeds and improve hemoglobin levels in HHT patients. Further research is necessary to confirm these findings.

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