A Follow-up of Children Enrolled in the Management of Myelomeningocele Study

描述（由申请人提供）：MOMS 2是对髓系脑膜膨出管理研究（MOMS）中儿童的随访研究。mom是由Eunice Kennedy Shriver国家儿童健康与人类发展研究所资助的一项多中心随机试验，研究产前与产后手术修复脊髓脊膜膨出的效果。符合条件且同意携带T1-S1期脊髓脊膜膨出病变且有后脑疝证据、胎龄19-24周的单胎胎儿的妇女，随机分为两组，一组通过子宫切开术在子宫内进行胎儿修复，另一组在婴儿出生后不久进行标准产后修复。这些孩子被跟踪到30个月大。然而，在那个年龄，残疾的许多影响还不明显。mom 2的目的是确定产前修复脊髓脊膜膨出是否影响学龄儿童的适应行为、认知功能、运动水平和功能、大脑形态和微观结构、泌尿系统健康等方面的健康。此外，还将评估产前手术对母亲生殖健康和家庭福利的影响。大约180个有5到8岁孩子的家庭参加了mom，将有资格参加mom 2。主要结果将由Vineland适应行为量表II来衡量。认知功能将通过一系列神经心理学测试来测量。运动水平和功能将通过体格检查来评估。使用高分辨率体积MRI，将比较产前和产后修复组的整体和区域大脑结构的体积分析。弥散张量成像（DTI）将用于确定各组白质束的微观结构组织是否存在差异，如胼胝体、投射和关联通路。脑磁图（MEG）将用于定位皮质激活区域并进行功能映射（包括运动，体感，视觉和听觉），然后可以与MRI和DTI数据共同注册。所有儿童将接受尿动力学视频、肾/膀胱超声和尿液培养来评估泌尿系统状况。还将评估生活质量、家庭影响、养育子女的压力和产妇生殖功能。如果这种干预被证明可以减轻与脊柱裂相关的负面后果，它可以改善未来许多儿童和成人的生活，使他们能够更独立、更有成效地生活。相反，如果没有持久的好处，研究结果将阻止女性接受侵入性和昂贵的手术。无论产前手术的效果如何，本研究将深入了解儿童从出生前到学龄的临床和发育过程，从而更深入地了解脊柱裂的不同方面，改善对受影响儿童的临床护理，并为携带脊髓脊膜膨出胎儿的妇女提供更好的产前咨询。

DESCRIPTION (provided by applicant): MOMS 2 is a follow-up study of the children from the Management of Myelomeningocele Study (MOMS). MOMS is a multi-center randomized trial of prenatal versus postnatal surgery for repair of myelomeningocele funded by the Eunice Kennedy Shriver National Institute of Child Health and Human Development. Eligible and consenting women carrying a singleton fetus with a myelomeningocele lesion starting at T1-S1 with evidence of hindbrain herniation, and gestational age 19-24 weeks are randomized to fetal repair in utero via hysterotomy versus standard postnatal repair for their infant shortly after birth. The children are followed until 30 months of age. However, at that age many of the implications of the disability are not yet apparent. The purpose of MOMS 2 is to determine whether prenatal repair of myelomeningocele affects adaptive behavior, cognitive functioning, motor level and function, brain morphology and microstructure, urologic health, and other aspects of health of the child at school age. In addition, the impact of prenatal surgery on the reproductive health of the mother, and on family wellbeing will be assessed. Approximately 180 of the families with children of 5 to 8 years old that participated in MOMS will be eligible for enrollment in MOMS 2. The primary outcome will be measured by the Vineland Adaptive Behavior Scales II. Cognitive function will be measured by a battery of neuropsychological tests. Motor level and function will be assessed by physical examination. Using high resolution volumetric MRI, volumetric analysis of whole and regional brain structures will be compared between the prenatal and postnatal repair groups. Diffusion tensor imaging (DTI) will be performed to determine if there are differences by group in the microstructural organization of white matter tracts, such as the corpus callosum and projection and association pathways. Magnetoencephalography (MEG) will be performed to localize areas of cortical activation and to perform functional mapping (including motor, somatosensory, visual and auditory) which can then be co-registered with the MRI and DTI data. All children will undergo video urodynamics, renal/bladder ultrasound and urine culture to evaluate urologic status. Quality of life, family impact, parenting stress and maternal reproductive functioning also will be assessed. If this intervention is shown to mitigate the negative consequences associated with spina bifida, it could improve the lives of many children and adults in the future, allowing them to live more independently and productively. Conversely, if there is little or no lasting benefit, the study results will prevent women from undergoing an invasive and expensive procedure. Regardless of the efficacy of prenatal surgery, this study will give insight into clinical and developmental course of children from before birth to school age, which can lead to a deeper understanding of the different facets of spina bifida, improved clinical care for affected children and better informed prenatal counseling for women carrying a fetus with myelomeningocele. PUBLIC HEALTH RELEVANCE: The MOMS trial is designed to investigate whether performing prenatal surgery to repair the spinal defect in a fetus diagnosed with spina bifida while still in the womb is better than the usual postnatal surgery when a baby is a few days old. This study is a follow-up of the MOMS children at school age to determine whether children who received the surgery before birth have better health and mental outcomes and live more independently and function more safely and appropriately in daily life than those who received the surgery after birth. If prenatal surgery is shown to be better and the effects to last into childhood and potentially adulthood, this will provide evidence for offering this intervention to pregnant women in the future, however if there is little or no lasting benefit, it will prevent women from undergoing an invasive and expensive procedure.