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People with hereditary hemorrhagic telangiectasia (HHT) often experience recurrent nosebleeds and hemorrhages into the lungs, gastrointestinal tract, and brain. The lesions, usually identifiable on physical examination, consist of arteriovenous malformations with direct connections between arterioles and venules. Current treatment involves cautery or surgical excision, but systemic therapy to address the proliferation of the telangiectasias might yield more-durable benefits. One such therapy under consideration is thalidomide, which has antiangiogenic properties and has been shown to strengthen the walls of endothelial cells in a mouse model.
Investigators in France and the Netherlands treated seven HHT patients (age range, 48–75) who had se…