Role of medical therapy in reducing the rate of aortic dilatation in children with bicuspid aortic valve

Aortic dilatation occurs commonly in patients with bicuspid aortic valve (BAV) and begins to manifest during childhood. Progressive dilatation results in the development of an aortic aneurysm in about 25% of young adults. Long-term medical treatment to reduce the rate of aortic dilatation is often prescribed to these patients. It is still unclear whether the use of oral medications prevents or slows this dilatation.

This was a retrospective cohort study on the value of medication in children with bicuspid aortic valve to prevent aortic dilatation. The objectives were to determine whether pharmacological treatment with either beta blockers or ACEI/ARB decreases the annual rate of aortic dilatation, and to determine the time to severe dilatation and the proportion participants achieving Z-score stabilization in treated compared to untreated patients. We leveraged the fact that some clinicians and institutions treat these patients, while others do not. This enabled to create treated and untreated groups with children with similar risk profiles.

We found that the use of pharmacological treatment did not lead to a clinically or statistically significant reduction in dilatation rate compared to children without treatment. Compared with untreated patients, those treated had a small reduction of AscAo and SoV dilatation rates with an absolute treatment difference of −0.032 Z/y (95% confidence interval [CI]: −0.086 to 0.022) and −0.021 Z/y (95% CI: −0.078 to 0.035), respectively. The probability of a null effect was high, as the 95% CI for all outcomes between the groups overlapped.

The complete results of this study are available in open access in CJC Pediatric and Congenital Heart Disease (https://doi.org/10.1016/j.cjcpc.2024.11.003)

Data from this study is available for secondary analysis or to augment data for other projects on BAV in children. See the data bank section.

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