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Medical treatment of Marfan syndrome: a time for change

¹ Wales Heart Research Institute, Cardiff University, UK
² University Hospital of Wales, UK
³ The Royal Hospital For Sick Children, Bristol, UK

Correspondence to:
Dr A Williams, Wales Heart Research Institute, Wales College of Medicine, Cardiff CF14 4XN, UK; awilliams97{at}supanet.com

It is accepted practice to prescribe β-blockers in order to retard aortic dilatation and prevent aortic dissection and rupture in patients with Marfan syndrome. A critical review of the published pharmacological studies shows this practice to be based on limited evidence. The data from small clinical and experimental studies with surrogate end points suggest greater potential benefit from alternative drug regimens, and a recent experimental study showed that losartan may interrupt the mechanism of disease as well as deal with its functional consequences. It is now essential to perform large, collaborative, randomised controlled trials with clinical end points of new treatments in Marfan syndrome.