Endoscopic versus Shunt Treatment of Hydrocephalus in Infants

脑积水主要影响一岁以下的婴儿。脑积水的传统治疗方法是 脑脊液分流术。脑脊液分流术的终生并发症高而昂贵，每年 住院费用近20亿美元，占所有儿科住院费用的3.1%。AS分流 并发症也会对生活质量造成不利影响，因此对受疾病影响的家庭进行调查并不奇怪 脑积水表明，他们迫切需要无分流的治疗方案。最有希望的--无分流 内窥镜下第三脑室吻合术联合脉络丛烧灼术治疗婴幼儿脑积水 (ETV+CPC)，我们的脑积水临床研究网络(HCRN)和其他人已经证明是安全的 而且可行。然而，与分流术相比，ETV+CPC的认知结果尚不清楚。我们的数据显示 大多数家庭会接受ETV+CPC的风险，以获得无分流的机会，但前提是最初 与分流术相比，ETV+CPC治疗对认知功能的影响不明显。使用 这份U01建议，我们的目标是：1.确定婴儿≤12个月的校正年龄，并要求 在北美三级儿科神经外科中心接受治疗，ETV+CPC与分流术比较 结果在手术后12个月时，根据贝利婴儿和 幼儿发展(贝利-III)认知量表，非自卑边际为1.5。2A。要确定，请在 相同的队列，如果ETV+CPC与分流术相比在非劣质Bayley-III运动/语言量表或 维尼兰-3级。2B。为了比较治疗效果的其他指标(脑/脑室体积， 治疗失败、住院天数、重复手术、使用成像)和治疗臂之间的并发症。 3A.弥散磁共振确定ETV+CPC和分流术对脑结构连通性的影响 手术后12个月，胼胝体的分数各向异性(FA)。3B.定义关系的步骤 术前脑/脑室体积和脑脊液NCAM-1水平与手术后的关系 术后12个月，采用FA和Bayley-III认知量表进行评定。3C。定义关系的步骤 脑/脑室容量的变化(术前至术后12个月)与FA之间 术后12个月，分别进行胼胝体、皮质脊髓束及Bayley-III认知和运动功能量表评定。 为此，我们计划了一项随机对照试验，比较ETV+CPC和分流术在婴儿脑积水中的作用，在HCRN内， 由14个北美领先的儿科神经外科中心组成的承诺集团，长期记录 成功合作临床研究和随机对照试验治疗脑积水。最优认知结果，首要的 因此，家庭的关切将是我们的主要结果。经验证的非侵入性磁共振成像的评估 白质微结构完整性和结构连通性的测量方法 大脑，将进一步洞察这两种治疗方法的发育后果。这项RCT将 帮助家庭确定儿童脑积水的最佳治疗方案。

Hydrocephalus primarily affects infants under one year of age. The traditional treatment for hydrocephalus is cerebrospinal fluid (CSF) shunt. Life-time complications of CSF shunts are high and expensive, with annual hospital charges of nearly $2.0 billion, accounting for 3.1% of all pediatric hospital charges. As shunt complications also adversely impact quality of life, it is not surprising that surveys of families affected by hydrocephalus show that they desperately desire shunt-free treatment options. The most promising shunt-free treatment for infant hydrocephalus is endoscopic third ventriculostomy with choroid plexus cauterization (ETV+CPC), which our Hydrocephalus Clinical Research Network (HCRN) and others have shown to be safe and viable. However, the cognitive outcome of ETV+CPC compared to shunt is not known. Our data suggest that most families would accept the risks of ETV+CPC for the chance to be free of shunt, but only if initial treatment with ETV+CPC will not lead to noticeable sacrifice in cognitive outcome compared to shunting. With this U01 proposal, we aim: 1. To determine, in infants ≤12 months corrected age, with hydrocephalus requiring treatment at tertiary care pediatric neurosurgery centers in North America, does ETV+CPC compared to shunt result in non-inferior cognitive outcome at 12 months from surgery, as measured by Bayley Scales of Infant and Toddler Development (Bayley-III) Cognitive Scale with a non-inferiority margin of 1.5. 2a. To determine, in the same cohort, if ETV+CPC compared to shunt results in non-inferior Bayley-III Motor/Language Scales or Vineland-3 Scales. 2b. To compare other measures of treatment performance (brain/ventricle volume, treatment failure, hospital days, repeat surgery, use of imaging) and complications between treatment arms. 3a. To determine the effect of ETV+CPC and shunt on cerebral structural connectivity, with diffusion MR fractional anisotropy (FA) in the corpus callosum 12 months after surgery. 3b. To define the relationships between pre-operative brain/ventricular volume and cerebrospinal fluid (CSF) NCAM-1 level to post-operative corpus callosum FA and Bayley-III Cognitive Scale 12 months after surgery. 3c. To define the relationships between change in brain/ventricular volume (pre-operatively to 12 months post-operatively) to FA in the corpus callosum and corticospinal tract and Bayley-III Cognitive and Motor Scales 12 months post-operatively. To do this, we plan an RCT comparing ETV+CPC and shunt in infants with hydrocephalus, within the HCRN, a committed group of 14 leading North American pediatric neurosurgical centers with a long track-record of successful collaborative clinical research and RCTs in hydrocephalus. Optimal cognitive outcome, the primary concern of families, will therefore, be our primary outcome. Assessment of dMRI, a validated, non-invasive method of measuring white matter microstructural integrity and structural connectivity in the developing brain, will provide further insight into the developmental consequences of these 2 treatments. This RCT will help families determine the optimal treatment of hydrocephalus for their child.