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

描述（由申请人提供）：MOMS 2是对脊髓脊膜膨出治疗研究（MOMS）中儿童的随访研究。MOMS是由Eunice Kennedy Shriver National Institute of Child Health and Human Development资助的一项多中心随机试验，旨在比较产前与产后手术修复脊髓脊膜膨出的效果。在T1-S1时开始具有脊髓脊膜膨出病变的单胎胎儿，具有后脑疝的证据，并且胎龄19-24周的合格且同意的妇女被随机分配至通过子宫切开术在子宫内进行胎儿修复与在出生后不久对其婴儿进行标准产后修复。孩子们被跟踪到30个月大。然而，在这个年龄，残疾的许多影响还不明显。MOMS 2的目的是确定脊髓脊膜膨出的产前修复是否影响学龄儿童的适应行为、认知功能、运动水平和功能、脑形态和显微结构、泌尿系统健康以及其他方面的健康。此外，还将评估产前手术对母亲生殖健康和家庭福祉的影响。大约有180个参加MOMS的5至8岁儿童的家庭将有资格参加MOMS 2。主要结果将通过Vineland适应行为量表II进行测量。认知功能将通过一系列神经心理学测试进行测量。将通过体格检查评估运动水平和功能。使用高分辨率体积MRI，将在产前和产后修复组之间比较全脑和局部脑结构的体积分析。将进行扩散张量成像（DTI），以确定各组在白色物质束（如胼胝体和投射及联合通路）的显微结构组织方面是否存在差异。将进行脑磁图（MEG）以定位皮质激活区域并进行功能映射（包括运动、躯体感觉、视觉和听觉），然后将其与MRI和DTI数据配准。所有儿童将接受视频尿动力学、肾脏/膀胱超声和尿培养，以评估泌尿系统状态。还将评估生活质量、家庭影响、养育压力和产妇生殖功能。如果这种干预措施能够减轻脊柱裂相关的负面影响，它可以改善未来许多儿童和成人的生活，使他们能够更独立和更有成效地生活。相反，如果很少或没有持久的好处，研究结果将阻止妇女接受侵入性和昂贵的程序。无论产前手术的疗效如何，这项研究将深入了解儿童从出生前到学龄的临床和发育过程，这可以加深对脊柱裂不同方面的理解，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.