de Keijzer Adine R, Keuning Zoë A, Meccanici Frederike, van Kimmenade Roland R L, van Melle Joost P, Bouma Berto J, Kluin Jolanda, Jongbloed Monique R M, Voskuil Michiel, Roos-Hesselink Jolien W, van den Bosch Annemien E
Department of Cardiology, Erasmus University Medical Centre, Rotterdam, The Netherlands.
Department of Cardiothoracic Surgery, Erasmus University Medical Centre, Rotterdam, The Netherlands.
Open Heart. 2025 May 27;12(1):e003355. doi: 10.1136/openhrt-2025-003355.
Supravalvular aortic stenosis (SVAS) is a rare condition with limited data on patients beyond childhood. This study aims to investigate the clinical course and outcomes of SVAS in adults.
All adult (≥18 years) patients with SVAS, prospectively registered in the Dutch Congenital Cor Vitia database between 2001 and 2019, were included. Survival and event-free survival were assessed. Evolution of peak velocity was analysed using linear mixed models. Differences in previous operated state, sex and Williams-Beuren syndrome were explored.
65 patients were included (age: 23 (IQR: 20, 31) years, 31% female, 46% previous SVAS correction, 47% Williams-Beuren syndrome). The peak velocity was 2.3±1.0 m/s at inclusion. Median follow-up time was 13 (IQR: 10, 17) years. Four patients died (one patient after cardiac surgery, two of non-cardiac causes and in one patient the cause of death was unknown) resulting in a 10-year survival of 95% (95% CI 90% to 100%) and event-free survival of 83% (95% CI 74% to 93%). There were no differences in event-free survival between previous operated state (p=0.2), sex (p=0.48) or Williams-Beuren syndrome (p=0.85). 31 cardiovascular events occurred in 15 patients, with the majority being arrhythmias. All SVAS-related interventions (three surgeries in two patients) occurred in unoperated patients (7 (95% CI 2 to 21)/1000 patient years). Although no patient showed fast progression (≥0.3 m/s/year), the peak velocity evolution over time increased faster in females compared with males (first time spline: 0.8 m/s, p=0.017).
In adulthood, SVAS patients showed a stable clinical course without rapid progression. While cardiovascular events occurred in this young cohort, they were mostly obsereved in those with additional congenital heart defects, suggesting a more optimistic view for SVAS itself. No significant differences in outcomes were observed in patients with/without Williams-Beuren syndrome. Overall, SVAS tends to follow a more benign course in adulthood compared with childhood, potentially allowing for less intensive follow-up- though follow-up should still be individualised based on associated congenital heart defects and cardiovascular risks.
瓣上主动脉狭窄(SVAS)是一种罕见疾病,关于儿童期以后患者的数据有限。本研究旨在调查成人SVAS的临床病程及预后。
纳入2001年至2019年在荷兰先天性心血管疾病数据库中前瞻性登记的所有成年(≥18岁)SVAS患者。评估生存率和无事件生存率。使用线性混合模型分析峰值速度的变化。探讨既往手术状态、性别和威廉姆斯-贝伦综合征的差异。
共纳入65例患者(年龄:23岁(四分位间距:20,31),31%为女性,46%曾接受SVAS矫正,47%患有威廉姆斯-贝伦综合征)。纳入时峰值速度为2.3±1.0米/秒。中位随访时间为13年(四分位间距:10,17)。4例患者死亡(1例死于心脏手术后,2例死于非心脏原因,1例死亡原因不明),10年生存率为95%(95%置信区间90%至100%),无事件生存率为83%(95%置信区间74%至93%)。既往手术状态(p = 0.2)、性别(p = 0.48)或威廉姆斯-贝伦综合征(p = 0.85)之间的无事件生存率无差异。15例患者发生31次心血管事件,多数为心律失常。所有与SVAS相关的干预措施(2例患者接受3次手术)均发生在未手术患者中(7(95%置信区间2至21)/1000患者年)。尽管没有患者表现出快速进展(≥0.3米/秒/年),但女性的峰值速度随时间的变化比男性更快(第一次样条分析:0.8米/秒,p = 0.017)。
在成年期,SVAS患者临床病程稳定,无快速进展。虽然该年轻队列中发生了心血管事件,但大多发生在合并其他先天性心脏病的患者中,提示SVAS本身预后更乐观。患有/未患有威廉姆斯-贝伦综合征的患者在预后方面未观察到显著差异。总体而言,与儿童期相比,SVAS在成年期往往病程更良性,可能允许随访强度降低——尽管仍应根据相关先天性心脏病和心血管风险进行个体化随访。