About HHT and the PATH Study
Hereditary Hemorrhagic Telangiectasia (HHT) is a genetic disorder that affects 1 in 5,000 people worldwide. Malformed blood vessels cause excessive bleeding from the nose (epistaxis) and gastrointestinal (GI) tract, requiring medical treatment and impairing quality of life. There are no effective medical therapies for HHT.
The PATH study is exploring the use of an oral medication called pomalidomide for the treatment of nosebleeds in HHT at 10 research centers across the United States. Adults suffering from HHT with moderate to severe nosebleeds (epistaxis) who require iron infusions or blood transfusions are eligible. During the 6-month study, patients might receive either pomalidomide or a matching placebo (sugar pill) in addition to their usual care.
PATH is funded by a grant from the National Heart, Lung and Blood Institute, and is led by researchers at the Cleveland Clinic and RTI International. Pomalidomide is an FDA-approved drug for the treatment of some cancers and is manufactured by Celgene.