Incidence of cardiovascular events and risk markers in a prospective study of children diagnosed with Marfan syndrome

Background. - Little is known about the incidence of cardiovascular events (CVEs) and their associated risk markers in children with Marfan syndrome (MFS). Aims. - To assess the incidence of CVEs and determine risk markers in a cohort diagnosed with Marfan syndrome during childhood and followed for several years. Methods. - From a French multicentre nationwide database, 462 patients with MES diagnosed during childhood were included prospectively. Patients' files were screened for a period of 20 years (1993-2013). CVEs (e.g. death, aortic dissection, cardiac valve or aortic root surgery) were assessed during the prospective follow-up. Results. - Median (interquartile range) age at the end of follow-up was 17.2 (11.1-21.3) years. CVEs were reported for 35 participants (7.6%; 95% confidence interval [CI] 5.3-10.4%). First CVEs were prophylactic aortic root surgery (n=29), aortic dissection (n=4; two aged <18 years) and death (n=2). Kaplan-Meier cumulative incidence of CVEs was 5.3% (95% Cl 3.3-8.7%) during childhood (aged <= 18 years) and 19.4% (95% CI 13.3-27.9%) at 25 years of age. The cumulative rate of CVEs was higher in case of Valsalva sinus 7-score increase of >= 0.1 per year (P=0.0003), maximal Valsalva sinus diameter growth speed >= 5 mm per year (P=0.03), aortic regurgitation >=, 2 (P=0.0005) and maximal Valsalva sinus Z-score >= 3 before 16 years of age (P<0.0001). In a multivariable Cox proportional analysis, the Valsalva sinus Z-score remained significantly related to outcome. Considering aortic root evolution, aortic regurgitation, age at diagnosis and beta-blocker therapy were related to Valsalva sinus Z-score evolution during follow-up. Conclusions. - CVEs in children with MFS are mainly related to prophylactic aortic root surgery. Aortic dissections are rarely observed in children. The Valsalva sinus Z-score is a strong indicator of subsequent CVEs in children with MFS. Attention to follow-up and beta-blocker observance may be warranted in high-risk children. (C) 2019 Published by Elsevier Masson SAS.