Efficacy of Nintedanib Per os as a Treatment for Epistaxis in HHT Disease.
Status:
Recruiting
Trial end date:
2022-12-01
Target enrollment:
Participant gender:
Summary
The recognized manifestations of HHT are all due to abnormalities in vascular structure.
Epistaxis are spontaneous, very variable, may occur as often as several times every day, and
are recurrent in 90% of patients and associated with chronic and severe anemia in 2-10%. They
also significantly reduce quality of life.
Blood transfusions are sometimes required in 10-30% of patients. Previous studies showed that
antiangiogenic treatments such as anti-VEGF treatment (bevacizumab) administered
intravenously was efficient on epistaxis and dramatically reduced nosebleeds.
Tyrosine kinase inhibitors are anti-angiogenic molecules which are available orally and could
therefore overcome the difficulties encountered with bevacizumab. The investigator
hypothesized that nintedanib, acting by indirect inhibition of the VEGF receptor should allow
a reduction of epistaxis in HHT patient.
Nintedanib has been used in one HHT patient following the diagnosis of Insterstitial
Pulmonary Fibrosis (published case report in 2017, Kovacs et al) with encouraging results.
The aim is to evaluate efficacy of nintedanib for the treatment of epistaxis in HHT patients