A phase 2 clinical trial has demonstrated that pomalidomide, used to treat bone marrow cancer, is safe and effective for managing hereditary hemorrhagic telangiectasia, a rare genetic bleeding disorder. The results were published Sept. 18 in the New England Journal of Medicine.
The trial, led by Keith McCrae, MD, of Cleveland Clinic and supported by the National Institutes of Health, was stopped early because of positive results, according to a Sept. 18 news release from the health system.
The randomized, placebo-controlled study enrolled 144 adults with moderate to severe HHT at 11 U.S. medical centers between 2019 and 2023. The participants were then assigned either pomalidomide or a placebo for 24 weeks.
The primary outcome measured was nosebleed severity, while the secondary outcome focused on quality of life specific to HHT. The results showed that pomalidomide treatment improved both nosebleeds and quality of life for patients.
HHT affects more than 1 in 5,000 people worldwide, causing fragile tangled blood vessels that can lead to life-threatening complications, the release said. There are no FDA-approved treatments to treat the disorder long term.