title: Characteristics of children and young adults with Marfan syndrome and aortic root dilation in a randomized trial comparing atenolol and losartan therapy.
abstract: BACKGROUND
  The 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.
  
  
  RESULTS
  Between 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.
  
  
  CONCLUSIONS
  Baseline 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.
authors.affiliation: Children's Hospital Boston, Boston, MA 02115, USA. ron.lacro@cardio.chboston.org
authors.forename: Ronald V
authors.initials: RV
authors.lastname: Lacro
authors.forename: Lin T
authors.initials: LT
authors.lastname: Guey
authors.forename: Harry C
authors.initials: HC
authors.lastname: Dietz
authors.forename: Gail D
authors.initials: GD
authors.lastname: Pearson
authors.forename: Anji T
authors.initials: AT
authors.lastname: Yetman
authors.forename: Bruce D
authors.initials: BD
authors.lastname: Gelb
authors.forename: Bart L
authors.initials: BL
authors.lastname: Loeys
authors.forename: D Woodrow
authors.initials: DW
authors.lastname: Benson
authors.forename: Timothy J
authors.initials: TJ
authors.lastname: Bradley
authors.forename: Julie
authors.initials: J
authors.lastname: De Backer
authors.forename: Geoffrey A
authors.initials: GA
authors.lastname: Forbus
authors.forename: Gloria L
authors.initials: GL
authors.lastname: Klein
authors.forename: Wyman W
authors.initials: WW
authors.lastname: Lai
authors.forename: Jami C
authors.initials: JC
authors.lastname: Levine
authors.forename: Mark B
authors.initials: MB
authors.lastname: Lewin
authors.forename: Larry W
authors.initials: LW
authors.lastname: Markham
authors.forename: Stephen M
authors.initials: SM
authors.lastname: Paridon
authors.forename: Mary Ella
authors.initials: ME
authors.lastname: Pierpont
authors.forename: Elizabeth
authors.initials: E
authors.lastname: Radojewski
authors.forename: Elif Seda
authors.initials: ES
authors.lastname: Selamet Tierney
authors.forename: Angela M
authors.initials: AM
authors.lastname: Sharkey
authors.forename: Stephanie Burns
authors.initials: SB
authors.lastname: Wechsler
authors.forename: Lynn
authors.initials: L
authors.lastname: Mahony
mesh_terms: D000293:Adolescent; D058671:Adrenergic beta-1 Receptor Antagonists / Q000627:therapeutic use; D000328:Adult; D047228:Angiotensin II Type 1 Receptor Blockers / Q000627:therapeutic use; D017545:Aortic Aneurysm, Thoracic / Q000150:complications / Q000188:drug therapy*; D001262:Atenolol / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007223:Infant; D019808:Losartan / Q000627:therapeutic use*; D008297:Male; D008382:Marfan Syndrome / Q000150:complications / Q000188:drug therapy*; D012189:Retrospective Studies; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D058671:Adrenergic beta-1 Receptor Antagonists; D047228:Angiotensin II Type 1 Receptor Blockers; D001262:Atenolol; D019808:Losartan