Editorial : Improved Understanding of Marfan Syndrome

So what should we do with this information clinically? There are strong mouse data and preliminary human clinical data that losartan can slow or even reverse aortic aneurysm in Marfan syndrome. Several well-designed randomized clinical trials are now underway to evaluate the clinical utility of ARBs in this situation, and results are expected in the next 3 years. Until we have detailed clinical safety and efficacy data, we should stick with the standard of care. Beta-blockers are still indicated as first-line therapy in Marfan syndrome patients with a dilated aortic root. Those who cannot tolerate beta-blockade, or whose aortic diameter continues to enlarge despite maximal beta-blockade, should be considered for referral to a clinical trial. However, if participation in clinical research is not an option, it seems reasonable at this point to use losartan clinically as a second-line medication.

Dr. Howard P. Levy is an assistant professor in the division of general internal medicine and McKusick-Nathans Institute of Genetic Medicine, Johns Hopkins University, Baltimore.