An Angiotensin-II Receptor Blocker for Marfan Syndrome?

Marfan syndrome, a genetic disorder caused by mutations that encode fibrillin-1, is associated with dilation, dissection, and rupture of the aortic root. Beta-blockers are recommended to reduce the rate of aortic root dilation. Angiotensin-II type 1 receptor blockers (ARBs) might interrupt the pathways leading to dilation, but studies have been inconclusive, with losartan failing to show a benefit. To determine the effect of the ARB irbesartan on the rate of dilation, investigators in the U.K. conducted a randomized, placebo-controlled study of 192 participants with Marfan syndrome and aged 6 to 40 years (52% female; median age, 18).

The anticipated enrollment was 490 participants, but the study was terminated early because of slow recruitme…