RTI INTERNATIONAL

Pomalidomide Shows Promise in Reducing Bleeding for Hereditary Hemorrhagic Telangiectasia

8 months ago

• A Phase II clinical trial (PATH-HHT) demonstrated that pomalidomide significantly reduces the severity of nosebleeds in patients with hereditary hemorrhagic telangiectasia (HHT). • The study, published in _The New England Journal of Medicine_, showed a notable improvement in the HHT-specific quality of life for patients treated with pomalidomide. • Pomalidomide was generally well-tolerated, although some patients experienced side effects such as neutropenia, constipation, and rash, leading to dose adjustments. • These findings suggest pomalidomide may offer a new therapeutic avenue for HHT, a bleeding disorder with limited FDA-approved treatment options.