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 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 ( score ≥6) and proportions of patients achieving score stabilization (dilatation rate <0.1 /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 /y (95% confidence interval [CI]: -0.086 to 0.022) and -0.021 /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 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.