title: Rationale and design of a randomized clinical trial (Marfan Sartan) of angiotensin II receptor blocker therapy versus placebo in individuals with Marfan syndrome.
abstract: BACKGROUND
  Recent studies have demonstrated that blockade of the angiotensin II type 1 receptor with losartan decreases aortic damage in an animal model of Marfan syndrome (a KI mouse model with a pathogenic mutation in the gene coding for fibrillin-1).
  
  
  OBJECTIVE
  To demonstrate a beneficial effect of losartan on aortic dilatation when added to optimal therapy in patients with Marfan syndrome.
  
  
  METHODS
  This is a multicentre, randomized, placebo-controlled, double-blind, clinical trial with a 2-year inclusion period and a 3-year follow-up period. Aortic root diameter will be measured using two-dimensional echocardiography. Secondary endpoints will include incidence of aortic dissection, aortic root surgery, death, quality of life, tolerance and compliance with treatments. We aim to enroll a total of 300 patients aged > or =10 years who fulfil the Ghent criteria for Marfan syndrome. Analyses will be based on intention to treat.
  
  
  CONCLUSIONS
  The results of this clinical trial could lead to profound modification of the management of aortic risk and complications in patients with Marfan syndrome and possibly in patients with thoracic aortic aneurysms of other aetiologies.
authors.affiliation: Centre de référence pour le syndrome de Marfan et apparentés, hôpital Bichat, Paris, France.
authors.forename: Delphine
authors.initials: D
authors.lastname: Detaint
authors.forename: Philippe
authors.initials: P
authors.lastname: Aegerter
authors.forename: Florence
authors.initials: F
authors.lastname: Tubach
authors.forename: Isabelle
authors.initials: I
authors.lastname: Hoffman
authors.forename: Henri
authors.initials: H
authors.lastname: Plauchu
authors.forename: Yves
authors.initials: Y
authors.lastname: Dulac
authors.forename: Laurence Olivier
authors.initials: LO
authors.lastname: Faivre
authors.forename: Marie-Ange
authors.initials: MA
authors.lastname: Delrue
authors.forename: Patrick
authors.initials: P
authors.lastname: Collignon
authors.forename: Sylvie
authors.initials: S
authors.lastname: Odent
authors.forename: Maria
authors.initials: M
authors.lastname: Tchitchinadze
authors.forename: Catherine
authors.initials: C
authors.lastname: Bouffard
authors.forename: Florence
authors.initials: F
authors.lastname: Arnoult
authors.forename: Mathieu
authors.initials: M
authors.lastname: Gautier
authors.forename: Catherine
authors.initials: C
authors.lastname: Boileau
authors.forename: Guillaume
authors.initials: G
authors.lastname: Jondeau
mesh_terms: D047228:Angiotensin II Type 1 Receptor Blockers / Q000627:therapeutic use*; D000818:Animals; D001014:Aortic Aneurysm / Q000000981:diagnostic imaging / Q000209:etiology / Q000517:prevention & control*; D004195:Disease Models, Animal; D004311:Double-Blind Method; D005602:France; D006801:Humans; D019808:Losartan / Q000627:therapeutic use*; D008382:Marfan Syndrome / Q000150:complications / Q000000981:diagnostic imaging / Q000188:drug therapy*; D051379:Mice; D015990:Placebo Effect; D012107:Research Design; D016896:Treatment Outcome; D014463:Ultrasonography
chemical_list: D047228:Angiotensin II Type 1 Receptor Blockers; D019808:Losartan