• American heart journal · May 2013

    Randomized Controlled Trial Multicenter Study Comparative Study Clinical Trial

    Characteristics of children and young adults with Marfan syndrome and aortic root dilation in a randomized trial comparing atenolol and losartan therapy.

    • Ronald V Lacro, Lin T Guey, Harry C Dietz, Gail D Pearson, Anji T Yetman, Bruce D Gelb, Bart L Loeys, D Woodrow Benson, Timothy J Bradley, Julie De Backer, Geoffrey A Forbus, Gloria L Klein, Wyman W Lai, Jami C Levine, Mark B Lewin, Larry W Markham, Stephen M Paridon, Mary Ella Pierpont, Elizabeth Radojewski, Elif Seda Selamet Tierney, Angela M Sharkey, Stephanie Burns Wechsler, Lynn Mahony, and Pediatric Heart Network Investigators.
    • Children's Hospital Boston, Boston, MA 02115, USA. ron.lacro@cardio.chboston.org
    • Am. Heart J. 2013 May 1;165(5):828-835.e3.

    BackgroundThe Pediatric Heart Network designed a clinical trial to compare aortic root growth and other short-term cardiovascular outcomes in children and young adults with Marfan syndrome randomized to receive atenolol or losartan. We report here the characteristics of the screened population and enrolled subjects.Methods And ResultsBetween 2007 and 2011, 21 clinical sites randomized 608 subjects, aged 6 months to 25 years who met the original Ghent criteria and had a body surface area-adjusted aortic root diameter z-score >3.0. The mean age at study entry was 11.2 years, 60% were male, and 25% were older teenagers and young adults. The median aortic root diameter z-score was 4.0. Aortic root diameter z-score did not vary with age. Mitral valve prolapse and mitral regurgitation were more common in females. Among those with a positive family history, 56% had a family member with aortic surgery, and 32% had a family member with a history of aortic dissection.ConclusionsBaseline demographic, clinical, and anthropometric characteristics of the randomized cohort are representative of patients in this population with moderate to severe aortic root dilation. The high percentage of young subjects with relatives who have had aortic dissection or surgery illustrates the need for more definitive therapy; we expect that the results of the study and the wealth of systematic data collected will make an important contribution to the management of individuals with Marfan syndrome.Copyright © 2013 Mosby, Inc. All rights reserved.

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