Pomalidomide for Epistaxis in Hereditary Hemorrhagic Telangiectasia.
David J KuterRaj S KasthuriVivek N IyerAllyson M PishkoJake E DeckerClifford R WeissKevin J WhiteheadMiles B ConradMarc S ZumbergJenny Y ZhouJoseph ParambilDerek MarshMarianne ClancyLauren BradleyLisa WisniewskiBenjamin A CarperSonia M ThomasKeith R McCraePublished in: The New England journal of medicine (2024)
Among patients with HHT, pomalidomide treatment resulted in a significant, clinically relevant reduction in epistaxis severity. No unexpected safety signals were identified. (Funded by the National Heart, Lung, and Blood Institute; PATH-HHT Clinicaltrials.gov number, NCT03910244).