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: 10677041
• 负责人: Abhaya V Kulkarni
• 金额: $ 49.77万
• 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/10677041
• 关键词: 10 year old; Africa; African; Aftercare; Age Months; Anatomy; Brain; Brain imaging; Cauterize; Child; Clinical; Clinical Trials; Cohort Studies; Cost Effectiveness Analysis; Coupled; Data; Development; Diagnosis; Disadvantaged; Endoscopy; Engineering; Etiology; Failure; Funding; Goals; Growth; Health; Hydrocephalus; Image; Imaging technology; Infant; Intracranial Pressure; Lead; Life; Longterm Follow-up; Magnetic Resonance Imaging; Measures; Medical; Medicine; Methods; Neurocognitive; Neurosciences Research; Nursery Schools; Operative Surgical Procedures; Outcome; Outcome Study; Patients; Pediatric Hospitals; Primary Schools; Quality of life; Quality-Adjusted Life Years; Randomized; Randomized Controlled Clinical Trials; Research; Resource-limited setting; School-Age Population; Science; Shunt Device; Site; Structure of choroid plexus; Technology; Treatment Effectiveness; Uganda; United States National Institutes of Health; Universities; Ventriculostomy; X-Ray Computed Tomography; brain volume; cohort; cost; cost effectiveness; empowerment; follow-up; improved; neurodevelopment; prospective; randomized, clinical trials; research and development; success; technology development; trial comparing; 10 year old; Africa; African; Aftercare; Age Months; Anatomy; Brain; Brain imaging; Cauterize; Child; Clinical; Clinical Trials; Cohort Studies; Cost Effectiveness Analysis; Coupled; Data; Development; Diagnosis; Disadvantaged; Endoscopy; Engineering; Etiology; Failure; Funding; Goals; Growth; Health; Hydrocephalus; Image; Imaging technology; Infant; Intracranial Pressure; Lead; Life; Longterm Follow-up; Magnetic Resonance Imaging; Measures; Medical; Medicine; Methods; Neurocognitive; Neurosciences Research; Nursery Schools; Operative Surgical Procedures; Outcome; Outcome Study; Patients; Pediatric Hospitals; Primary Schools; Quality of life; Quality-Adjusted Life Years; Randomized; Randomized Controlled Clinical Trials; Research; Resource-limited setting; School-Age Population; Science; Shunt Device; Site; Structure of choroid plexus; Technology; Treatment Effectiveness; Uganda; United States National Institutes of Health; Universities; Ventriculostomy; X-Ray Computed Tomography; brain volume; cohort; cost; cost effectiveness; empowerment; follow-up; improved; neurodevelopment; prospective; randomized, clinical trials; research and development; success; technology development; trial comparing

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.

R01更新的目标是由我们成功进行随机进行的结果驱动的 比较内窥镜第三室造口术与脉络丛结合的对照临床试验 烧灼（ETV/CPC）分流治疗后感染后脑积水（PIH，最常见的 乌干达的脑积水病因<6个月大（Clinicaltrials.gov NCT01936272， R01HD085853，R21HD068213）在乌干达治疗儿童医院（CCHU）。临时 这项研究的结果已成为儿童医学的里程碑，发现：a）最多24 几个月，就故障率而言，ETV/CPC和分流之间没有显着差异， 发育结果和大脑生长； b）大脑体积，而不是CSF的体积，与 发展结果； c）成功治疗脑积水，从而改善了大脑的生长 第一年，第二年停滞不前。因为几乎所有内窥镜失败 治疗发生在生命的第一年，我们预计 如果我们在小学时代关注这些孩子，脑积水治疗可能有利于内窥镜检查 从5年到10年。此外，我们执行更详细的神经认知评估的能力 在此期间，进一步的随访将比我们的学前评估更准确。最后， 如果可以的话，我们可以收集所有数据并执行更确定的成本效益分析 遵循这个独特的队列整整10年。 在此期间，我们在过去的5年中进行了大量能力建设 临床试验。我们已经在乌干达的治疗儿童医院开发了一个繁荣的研究部门 在与此无关的其他多项NIH资助的研究中，可以进行高质量的研究 临床试验（DP1HD086071，R01AI145057和R01HD096693）。此外，由于所有氢气头 治疗需要大脑成像，我们开发了一个可持续低场MRI的繁荣中心 姆巴拉拉科学技术大学的工程。 本项目旨在遵循这个独特的队列至10岁，试图确定 确定内窥镜治疗对发展中国家的婴儿是否更可持续，建立 低场MRI作为一种可持续成像技术，并确定医疗和 乌干达的工程地点是非洲的独立研究和技术发展中心。