Pickard Sarah S, Wong John B, Bucholz Emily M, Newburger Jane W, Tworetzky Wayne, Lafranchi Terra, Benson Carol B, Wilkins-Haug Louise E, Porras Diego, Callahan Ryan, Friedman Kevin G
Department of Cardiology, Boston Children's Hospital, MA (S.S.P., E.M.B., J.W.N., W.T., T.L., D.P., R.C., K.G.F.).
Departments of Pediatrics (S.S.P., E.B., J.W.N., W.T., D.P., R.C., K.G.F.), Harvard Medical School, Boston, MA.
Circ Cardiovasc Qual Outcomes. 2020 Apr;13(4):e006127. doi: 10.1161/CIRCOUTCOMES.119.006127. Epub 2020 Apr 7.
Fetal aortic valvuloplasty (FAV) may prevent progression of midgestation aortic stenosis to hypoplastic left heart syndrome. However, FAV has well-established risks, and its survival benefit remains unknown. Our primary aim was to determine whether FAV for midgestation aortic stenosis increases survival from fetal diagnosis to age 6 years.
We performed a retrospective analysis of 143 fetuses who underwent FAV from 2000 to 2017 and a secondary analysis of the Pediatric Heart Network Single Ventricle Reconstruction trial. Using these results, we developed a decision model to estimate probability of transplant-free survival from fetal diagnosis to age 6 years and postnatal restricted mean transplant-free survival time. FAV was technically successful in 84% of 143 fetuses with fetal demise in 8%. Biventricular circulation was achieved in 50% of 111 live-born infants with successful FAV but in only 16% of the 19 patients with unsuccessful FAV. The model projected overlapping probabilities of transplant-free survival to age 6 years at 75% (95% CI, 67%-82%) with FAV versus 72% (95% CI, 61%-82%) with expectant fetal management, resulting in a restricted mean transplant-free survival time benefit of 1.2 months. When limiting analyses to the improved FAV experience since 2009 to reflect current practice, (probability of technical success [94%], fetal demise [4%], and biventricular circulation [66%]), the model projected that FAV increased the probability of survival to age 6 years to 82% (95% CI, 73%-89%). Expectant management is favored if risk of fetal demise exceeded 12% or probability of biventricular circulation fell below 26%, but FAV remained favored over plausible recent range of technical success.
Our model suggests that FAV provides a modest, medium-term survival benefit over expectant fetal management. Appropriate patient selection and low risk of fetal demise with FAV are critical factors for obtaining a survival benefit.
胎儿主动脉瓣成形术（FAV）可能预防妊娠中期主动脉瓣狭窄进展为左心发育不全综合征。然而，FAV存在公认的风险，其生存获益仍不明确。我们的主要目的是确定针对妊娠中期主动脉瓣狭窄的FAV是否能提高从胎儿诊断至6岁时的生存率。
我们对2000年至2017年接受FAV的143例胎儿进行了回顾性分析，并对儿科心脏网络单心室重建试验进行了二次分析。利用这些结果，我们建立了一个决策模型，以估计从胎儿诊断至6岁时无移植生存的概率以及出生后无移植生存的受限平均时间。在143例胎儿中，84%的FAV技术成功，8%的胎儿死亡。在111例FAV成功的活产婴儿中，50%实现了双心室循环，但在19例FAV不成功的患者中，只有16%实现了双心室循环。该模型预测，FAV组至6岁时无移植生存的概率为75%（95%CI，67%-82%），与胎儿期待管理组的72%（95%CI，61%-82%）重叠，无移植生存的受限平均时间获益为1.2个月。当将分析限制在2009年以来改进的FAV经验以反映当前实践时（技术成功概率[94%]、胎儿死亡概率[4%]和双心室循环概率[66%]），该模型预测FAV将6岁时的生存概率提高到82%（95%CI，73%-89%）。如果胎儿死亡风险超过12%或双心室循环概率低于26%，则倾向于期待管理，但在近期合理的技术成功范围内，FAV仍更受青睐。
我们的模型表明，与胎儿期待管理相比，FAV提供了适度的中期生存获益。合适的患者选择以及FAV时较低的胎儿死亡风险是获得生存获益的关键因素。
