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先天性心脏缺陷在生命第一年内死亡的主要原因。传统上，在生命的前2 - 3年进行三个阶段的手术姑息，需要深低温停循环和心肺转流。该患者队列的神经发育结局仍不理想。混合I期手术为第一期姑息治疗提供了一种替代方法。Hybrid手术不需要心肺转流;因此新生儿不会暴露于循环停止和心肺转流的所有潜在神经损伤风险。混合手术后，脑灌注取决于通过支架未闭动脉导管的血流和逆行主动脉弓流向颈动脉，类似于胎儿生命，大约6个月大时进行下一阶段修复。目前尚不清楚从主动脉弓到大脑的逆行血流是否足以正常生长和发育。本研究的长期目标是验证以下假设：在接受混合I期姑息治疗的左心发育不良综合征婴儿中，脑血流速度与神经发育结局之间存在相关性。本研究将招募HLHS患者以及正常年龄匹配的对照组。将在出生后以及2、4和6月龄时对两组进行经颅多普勒测量大脑中动脉和前动脉血流。此外，将在相同的时间间隔使用出生时、2个月和4个月时的婴儿运动能力测试以及6个月时的Bayley婴幼儿发育量表（第3版）进行婴儿发育评估。这将与正常参考年龄匹配对照进行比较。虽然HLHS患者的样本预计会很低，但本研究将包括两种性别的婴儿以及少数民族。本研究的经颅多普勒结果可能对神经认知发育结局产生深远影响，这可能对患者、家庭和社会产生巨大的长期影响。本研究与NIH在生物医学和行为研究中的使命一致，结合经颅多普勒技术的创新，以评估混合I期姑息治疗后HLHS患者的脑灌注。

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.