Neurodevelopmental Outcomes in Infants with HLHS after Hybrid Stage I Palliation

描述（由申请人提供）：左心发育不良综合征仍然是所有出生后第一年先天性心脏缺陷死亡的主要原因。传统上，在生命的最初 2-3 年内进行三阶段姑息手术，需要深度低温停循环和体外循环。这组患者的神经发育结果仍然不理想。混合第一阶段手术为第一阶段姑息治疗提供了另一种方法。混合手术不需要体外循环；因此，新生儿不会面临循环骤停和心肺转流的所有风险，并可能造成神经损伤。混合手术后，脑灌注取决于通过支架动脉导管未闭的血流和逆行主动脉弓流向颈动脉的血流，类似于胎儿的生命，大约 6 个月大时进行下一阶段修复。从主动脉弓到大脑的逆行血流是否足以维持正常生长和发育仍不清楚。本研究计划的长期目标是检验以下假设：接受混合 I 期姑息治疗的左心发育不全综合征婴儿的脑血流速度与神经发育结果之间存在相关性。本研究将招募 HLHS 患者以及正常年龄匹配的对照。两组出生后、2、4、6月龄时均进行经颅多普勒测量大脑中动脉和大脑前动脉血流量。此外，将使用出生时以及 2 个月和 4 个月时的婴儿运动表现测试以及 6 个月时进行的贝利婴幼儿发育量表（第三版）以相同的时间间隔进行婴儿发育评估。这将与正常参考的年龄匹配对照进行比较。尽管 HLHS 患者的样本预计较低，但本研究将包括男女婴儿以及少数族裔。这项研究的经颅多普勒结果可能对神经认知发育结果产生深远的影响，这可能对患者、家庭和社会产生巨大的长期影响。这项研究符合 NIH 在神经发育结果生物医学和行为研究方面的使命，结合当前经颅多普勒技术的创新，评估这一独特的 HLHS 患者在混合 I 期姑息治疗后的脑灌注。

DESCRIPTION (provided by applicant): Hypoplastic Left Heart Syndrome remains the leading cause of death of all congenital heart defects in the first year of life. Traditionally, three staged surgical palliations are performed during the first 2-3 years of life, requiring profound hypothermic circulatory arrest and cardiopulmonary bypass. Neurodevelopmental outcomes in this cohort of patients remain suboptimal. The Hybrid Stage I procedure offers an alternative approach for the first staged palliation. The Hybrid procedure does not require cardiopulmonary bypass; therefore the neonate is not exposed to all the risks of circulatory arrest and cardiopulmonary bypass with potential for neurologic injury. After the Hybrid procedure, cerebral perfusion is dependent upon the blood flow through the stented patent ductus arteriosus and retrograde aortic arch flow to the carotid arteries, similar to fetal life, for approximately 6 months of age when the next staged repair is performed. It remains unknown if the retrograde blood flow from the aortic arch to the brain is adequate for normal growth and development. The long-term objective of this research proposal is to test the hypothesis that a correlation exists between cerebral blood flow velocity and neurodevelopmental outcomes in hypoplastic left heart syndrome babies who have undergone Hybrid Stage I palliation. HLHS patients, along with normal age- matched controls, will be recruited for this study. Transcranial Doppler will be performed to measure the middle and anterior cerebral artery blood flow after birth, and at 2, 4, and 6 months of age in both groups. Additionally, infant developmental assessment will be performed at the same time intervals using the Test of Infant Motor Performance at birth and 2 and 4 months, as well as the Bayley Scale of Infant and Toddler Development, 3rd edition, performed at 6 months of age. This will be compared to norm-referenced age- matched controls. Although the sample of HLHS patients is expected to be low, this study will include infants of both gender, as well as minorities. The transcranial Doppler findings of this study may have a profound impact on neurocognitive developmental outcomes which may have a tremendous long-term impact on patients, families, and society. This study is aligned with the mission of the NIH in biomedical and behavioral research with neurodevelopmental outcomes, coupled with innovation of current technology with transcranial Doppler, to assess cerebral perfusion in this unique cohort of HLHS patients after Hybrid Stage I palliation.