The Role of Medical Therapy in Management of Bicuspid Aortic Valve–Associated Aortopathy in Children

Background

Patients with bicuspid aortic valve (BAV) are often treated with medication to slow the rate of aortic dilatation, without established efficacy.

Methods

We conducted a retrospective, multicentre study of 558 children (83 treated and 475 not treated) with BAV and ascending aorta (AscAo) dilatation. The median follow-up was 3.6 years for treated patients and 5.6 years for not treated patients. Longitudinal mixed models assessed the rate of AscAo and sinus of Valsalva (SoV) dilatation expressed as a change in Z score units per year for patients treated and not treated with a β-blocker or an afterload-reducing agent. Secondary outcomes included time to significant AscAo dilatation (Z score ≥6) and proportions of patients achieving Z score stabilization (dilatation rate <0.1 Z/y).

Results

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. Patients treated had a small reduction of the time to significant dilatation of AscAo (hazard ratio: 0.83; 95% CI: 0.43-1.61). Patients treated were more likely to achieve Z score stabilization with an increase in the proportion of patients by 4.5% for AscAo (95% CI: −11.3% to 20.2%) and 7% for SoV (95% CI: −9.7% to 22.5%). Overall, the probability of a null effect was high, as the 95% CI for all outcomes between the groups overlapped.

Conclusion

Pharmacologic treatment was not associated with a meaningful reduction of AscAo and SoV dilatation rates in children with BAV.