BACKGROUND Aortic-root dissection is the leading cause of death in Marfan's syndrome. Studies suggest that with regard to slowing aortic-root enlargement, losartan may be more effective than beta-blockers, the current standard therapy in most centers. METHODS We conducted a randomized trial comparing losartan with atenolol in children and young adults with Marfan's syndrome. The primary outcome was the rate of aortic-root enlargement, expressed as the change in the maximum aortic-root-diameter z score indexed to body-surface area (hereafter, aortic-root z score) over a 3-year period. Secondary outcomes included the rate of change in the absolute diameter of the aortic root; the rate of change in aortic regurgitation; the time to aortic dissection, aortic-root surgery, or death; somatic growth; and the incidence of adverse events. RESULTS From January 2007 through February 2011, a total of 21 clinical centers enrolled 608 participants, 6 months to 25 years of age (mean [±SD] age, 11.5±6.5 years in the atenolol group and 11.0±6.2 years in the losartan group), who had an aortic-root z score greater than 3.0. The baseline-adjusted rate of change (±SE) in the aortic-root z score did not differ significantly between the atenolol group and the losartan group (−0.139±0.013 and −0.107±0.013 standard-deviation units per year, respectively; P = 0.08). Both slopes were significantly less than zero, indicating a decrease in the degree of aortic-root dilatation relative to body-surface area with either treatment. The 3-year rates of aortic-root surgery, aortic dissection, death, and a composite of these events did not differ significantly between the two treatment groups. CONCLUSIONS Among children and young adults with Marfan's syndrome who were randomly assigned to losartan or atenolol, we found no significant difference in the rate of aortic-root dilatation between the two treatment groups over a 3-year period. (Funded by the National Heart, Lung, and Blood Institute and others; ClinicalTrials.gov number, NCT00429364.)

Objective To assess health-related quality of life (HRQOL) in a large multicenter cohort of children and young adults with Marfan syndrome (MFS) participating in the Pediatric Heart Network Marfan Trial. Study design The Pediatric Quality of Life Inventory (PedsQL) 4.0 Generic Core Scales were administered to 321 subjects with MFS (5–25 years). PedsQL scores were compared with healthy population norms. The impact of treatment arm (atenolol versus losartan), severity of clinical features, and number of patient-reported symptoms (PRS) on HRQOL was assessed by general linear models. Results Mean PedsQL scores in children (5–18 years) with MFS were lower than healthy population norms for physical (P ≤ .003) and psychosocial (P<0.001) domains; mean psychosocial scores for adults (19–25 years) were higher than healthy norms (P<0.001). HRQOL across multiple domains correlated inversely with frequency of PRS (r=0.30–0.38, P<0.0001). Those <18 years of age with neurodevelopmental disorders (ND, mainly learning disability, attention deficit disorder and/or hyperactivity) had lower mean PedsQL scores (5.5–7.4 lower, P<0.04). A multivariable model found age, sex, PRS, and ND to be independent predictors of HRQOL. There were no differences in HRQOL scores by treatment arm, aortic root z-score, number of skeletal features, or presence of ectopia lentis. Conclusions Children and adolescents with MFS were at high risk for impaired HRQOL. PRS and ND, but not treatment arm or severity of MFS-related physical findings, were associated with lower HRQOL.

Surgical PVR is safe with low in-hospital and midterm follow-up mortality and reoperation rates. These outcomes provide a useful benchmark for treatment strategy comparisons.

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