About HHT


Hereditary Hemorrhagic Telangiectasia (HHT) is a genetic disorder of the blood vessels that affects approximately 1 in 5,000 people worldwide. Also known as Osler-Weber-Rendu Syndrome, HHT is an inherited disease that leads to malformed blood vessels in multiple organs of the body and typically begins with nosebleeds during childhood. More than 80% of those with HHT are affected by mucosal (referring to the thin skin that covers the inside surface of parts of the body such as the nose and mouth) bleeding, resulting in recurrent, excessive bleeding from the nose (epistaxis) and gastrointestinal (GI) tract—both major causes of impaired quality of life in these individuals.. Few randomized trials establish evidence-based guidelines for management of HHT, and thus treatment is not standardized. Interventional procedures have formed the mainstay of HHT therapy and although initially effective, are usually associated with recurrence of bleeding. Given the current absence of proven effective medical therapies, there continues to be a pressing need for developing more efficacious agents for the management of HHT.