Neurocognitive outcomes and changes in brain and CSF volume after treatment of post-infectious hydrocephalus in Ugandan infants by shunting or ETV/CPC: a randomized prospective trial

通过分流或 ETV/CPC 治疗乌干达婴儿感染后脑积水后的神经认知结果以及大脑和脑脊液体积的变化：一项随机前瞻性试验

• 批准号: 10733168
• 负责人: Abhaya V Kulkarni
• 金额: $ 50.23万
• 依托单位: YALE UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2022
• 资助国家: 美国
• 起止时间: 2022-11-01 至 2026-06-30
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10733168
• 关键词: Neurocognitive; outcomes; changes; brain; CSF

The goals of this R01 Renewal are driven by the results of our successfully conducted randomized controlled clinical trial comparing endoscopic third ventriculostomy combined with choroid plexus cauterization (ETV/CPC) to shunt treatment for post-infectious hydrocephalus (PIH, the most common hydrocephalus etiology in Uganda) in infants < 6 months of age (ClinicalTrials.gov NCT01936272, R01HD085853, R21HD068213) at the CURE Children’s Hospital of Uganda (CCHU). The interim outcomes of this study are becoming a landmark in children’s medicine, finding that: a) at up to 24 months, there was no significant difference between ETV/CPC and shunt in regard to failure rate, developmental outcome, and brain growth; b) brain volume, not CSF volume, correlated strongly with developmental outcome; c) successful treatment of the hydrocephalus led to improved brain growth in the first year, which stagnated during the second year. Because almost all of the failures of endoscopic treatment occurred early in the first year of life, we anticipate that the longer term outcomes of hydrocephalus treatment may favor endoscopy if we follow these children through primary school age from 5 to 10 years. Furthermore, our ability to perform much more detailed neurocognitive assessments during this further followup will be much more accurate than during our pre-school assessments. Lastly, we can gather all of our data and perform a much more definitive cost-effectiveness analysis if we can follow this unique cohort for a full 10 years. We have engaged in substantial capacity building during the previous 5 years of support during this clinical trial. We have developed a thriving research unit at the CURE Children’s Hospital of Uganda, enabling high-quality research to be conducted in multiple other NIH funded studies unrelated to this clinical trial (DP1HD086071, R01AI145057, and R01HD096693). Furthermore, since all hydrocephalus treatment requires brain imaging, we have developed a thriving center for sustainable low-field MRI engineering at the Mbarara University of Science and Technology. This present project seeks to follow this unique cohort to 10 years of age, seeking to definitively establish whether endoscopic treatment is more sustainable for infants in the developing world, establish low field MRI as a sustainable imaging technology, and establish the capacity of both the medical and engineering sites in Uganda as independent research and technology development centers for Africa.

本次R 01更新的目标是由我们成功进行的随机 比较内镜下第三脑室底造瘘术联合脉络丛的对照临床试验 感染后脑积水（PIH，最常见的 乌干达脑积水病因学）在小于6个月大的婴儿中的应用（ClinicalTrials.govNCT01936272， R 01 HD 085853，R21 HD 068213）在乌干达CURE儿童医院（CCHU）。临时 这项研究的结果正在成为儿童医学的一个里程碑，发现：a）在高达24 3个月时，ETV/CPC和分流术在失败率方面无显著差异， 发育结果和脑生长; B）脑体积，而不是CSF体积，与 发育结果; c）脑积水的成功治疗导致脑发育改善， 第一年，第二年停滞不前。因为几乎所有内窥镜检查的失败 治疗发生在生命的第一年早期，我们预计， 如果我们对这些孩子进行小学阶段的随访，脑积水治疗可能更有利于内窥镜检查 5到10年。此外，我们进行更详细的神经认知评估的能力 在此期间，进一步的后续行动将比我们的学前评估更加准确。最后， 我们可以收集所有的数据，并尽可能地进行更明确的成本效益分析 跟随这个独特的群体整整10年。 我们在过去5年的支持中进行了大量的能力建设， 临床试验我们在乌干达的CURE儿童医院建立了一个蓬勃发展的研究单位， 使高质量的研究能够在与此无关的多个其他NIH资助的研究中进行 临床试验（DP 1HD 086071、R 01 AI 145057和R 01 HD 096693）。此外，由于所有脑积水 治疗需要大脑成像，我们已经开发了一个蓬勃发展的中心，可持续的低场MRI 他是姆巴拉拉科技大学的工程师。 本项目旨在跟踪这一独特的队列到10岁， 确定内窥镜治疗是否对发展中国家的婴儿更可持续， 低场MRI作为一种可持续的成像技术，并建立医疗和 作为非洲独立的研究和技术开发中心。