Marfan Drug Study Argues for Treatment at Younger Age and Earlier Stage of Disease; Suggests Losartan Equally Effective As Atenol

Between 70 and 80 percent of patients with the connective tissue condition Marfan syndrome have aortic-root dilation, which happens when the aorta, the main blood vessel between the heart and body, becomes too large and tears. This condition can result in serious illness and sometimes death. A National Institutes of Health-funded study comparing treatment with widely used blood pressure medications atenolol or losartan in patients with Marfan syndrome who had an enlarged aortic root found no significant difference in the rate of aortic-root dilation between the two treatment groups over three years. The results of the Atenolol versus Losartan in Children and Young Adults with Marfan Syndrome study, supported by NIH’s National Heart, Lung, and Blood Institute (NHLBI), were presented today at the American Heart Association (AHA) Scientific Sessions in Chicago. The study was published simultaneously today (November 18, 2014) in the New England Journal of Medicine. Marfan syndrome is a genetic disorder that affects connective tissue, which helps to hold the body together. Features of the disorder are most often found in the heart, blood vessels, bones, joints, and eyes. People with the syndrome tend to be extremely tall. Standard care includes frequent cardiac imaging, exercise restriction, administration of a beta-blocker such as atenolol or other medications that may decrease the rate of aortic enlargement, and elective aortic-root replacement when the aortic root becomes too large. Although early diagnosis and refined medical and surgical management have improved survival, patients with Marfan syndrome continue to have high rates of complications and death from heart problems, even at a young age.
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