Neuroimaging and Neurocognitive Markers of Brain Injury in Young Children with Sickle Cell Disease
Neuroimaging and Neurocognitive Markers of Brain Injury in Young Children with Sickle Cell Disease
批准号:
10636709
负责人:
EBONI I LANCE
金额:
$37.33万
依托单位国家:
美国
项目类别:
财政年份:
2023
资助国家:
美国
项目状态:
未结题
起止时间:
2023-04-01 至 2028-03-31
关键词:
3 year old4 year old5 year old6 year oldAcuteAdultAgeAnesthesia proceduresAxonBehavioralBloodBrain InjuriesCase/Control StudiesCephalicCerebral InfarctionCerebrovascular CirculationCerebrumCharacteristicsChildChronic DiseaseClinicalClinical DataCognitionCognitiveCohort StudiesCollectionDataData CollectionDevelopmentDiagnosisDiagnosticDiseaseEarly identificationEarly treatmentEducationEnzyme-Linked Immunosorbent AssayEvaluationFetal HemoglobinFutureGoalsGuidelinesHematological DiseaseHemoglobin concentration resultImpaired cognitionInflammatoryInheritedInjuryIntelligenceIschemic StrokeKnowledgeLanguageLesionLiteratureLongitudinal StudiesMagnetic Resonance ImagingMathematicsMeasuresMedicalMedical HistoryMetabolicMetabolic stressMetabolismMorbidity - disease rateNeurocognitiveNeurodevelopmental DeficitNeurologicNeurologic ExaminationNeurologic SymptomsNeuronsNeuropsychological TestsOutcomeOxygenParticipantPatientsPerformancePhysical FunctionPhysiciansPilot ProjectsPlasmaPlasma ProteinsPopulationProteinsPsychologistReadingRecommendationRecording of previous eventsRecurrenceResearchResearch PersonnelRiskRisk FactorsSample SizeSamplingSchool-Age PopulationSedation procedureSensitivity and SpecificityServicesSickle Cell AnemiaSiteStrokeTest ResultTestingTherapeutic InterventionTimeTrainingVulnerable PopulationsWhite Blood Cell Count procedureWorkbrain magnetic resonance imagingbrain tissuedisabilityexecutive functionexperiencehigh riskhydroxyureaimprovedmaleminimal riskmultidisciplinarymultimodalityneurocognitive testneuroimagingneuroprotectionpredictive markerpreventprospectiverisk predictionscreeningsexskillsstandard of careverbal
中文摘要
项目摘要/摘要
镰状细胞病是一种遗传性血液疾病,具有多种神经系统和发育性疾病。
并发症。特别是2至5岁患有SCD的幼儿,患上
缺血性中风和无症状脑梗塞(SCI)。脊髓损伤是高信号的T2脑磁共振
无急性神经系统症状但伴有相关损害的MRI病变
认知和未来进展性或额外卒中/脊髓损伤的风险增加。神经影像与神经认知
在学龄期和成年SCD人群中,与SCI相关的资料已经很好地建立了。此信息是
在6岁以下患有SCD的幼儿中,由于MRI有镇静/麻醉的风险,这种情况很少发生。
我们的初步研究表明,行为训练可以产生高质量的神经成像数据,而不需要
通过先导性研究对患有SCD的3-4岁儿童进行镇静。
该应用程序的长期目标是开发一种诊断电池,该电池使用
未用镇静剂的神经成像测量、神经认知测试和血浆脑损伤蛋白水平以确定
患有SCD的幼儿有患SCI的风险。通过前瞻性纵向病例对照研究,我们将收集
病史和临床数据,每年进行神经系统检查和抽血
患有SCD的儿童从研究开始到研究结束。参与者还将在3点接受初步的神经成像电池
至4岁,在研究结束时重复进行神经成像,并在研究中进行纵向神经认知测试
入门,进行初始神经成像,以及出口神经成像。这一多学科、多模式的提案
有两个目标。目标1将在3至4年内确定横断面神经成像和神经认知发现-
对SCD和SCI的老年儿童进行初步MRI检查,并与无SCI的SCD儿童比较。目标
2将比较有脊髓损伤和无脊髓损伤的6岁儿童在出口MRI上的纵向数据。探索性目标
将探索不同疾病调整治疗方法下儿童神经影像测量的差异
伴和不伴脊髓损伤儿童的羟基尿素和血浆蛋白水平。拟议的工作将确定
哪种神经成像测量和神经认知测试可以预测脊髓损伤,导致了一项多点研究
在更大的地区异质性SCD人群中验证这些发现。这项研究的PI,a
神经发育内科医生,是一名早期研究员,非常适合扩大她现有的研究队列
在一组合作调查人员的支持下,包括一名高级行为心理学家和神经成像
物理学家,以及现有的合作者和工作人员。
英文摘要
Project Summary/Abstract
Sickle cell disease (SCD) is an inherited blood disorder with several neurological and developmental
complications. Young children with SCD between 2 and 5 years of age, in particular, have an increased risk for
ischemic stroke and silent cerebral infarctions (SCI). SCI are hyperintense T2 brain magnetic resonance
imaging (MRI) lesions with no accompanying acute neurological symptoms but with associated impaired
cognition and increased risk of future progressive or additional stroke/SCI. Neuroimaging and neurocognitive
profiles associated with SCI are well established in school-aged and adult SCD populations. This information is
largely unknown in young children under 6 years of age with SCD due to risk of sedation/anesthesia with MRI.
Our preliminary studies have shown that behavioral training can yield high quality neuroimaging data without
sedation in 3 to 4 years old children with SCD through a pilot study.
The long term goal of this application is to develop a diagnostic battery using a combination of
unsedated neuroimaging measures, neurocognitive testing, and plasma brain injury protein levels to identify
young children with SCD at risk for SCI. Through a prospective longitudinal case-control study, we will collect
medical history and clinical data and perform neurological examination and blood draws annually in 100
children with SCD from study entry to study exit. Participants will also undergo initial neuroimaging battery at 3
to 4 years of age with repeat neuroimaging at study exit as well as longitudinal neurocognitive testing at study
entry, with initial neuroimaging, and with exit neuroimaging. This multi-disciplinary and multi-modality proposal
has two Aims. Aim 1 will identify the cross-sectional neuroimaging and neurocognitive findings in 3 to 4 year-
old children with SCD and SCI on initial MRI by comparing their results to children with SCD without SCI. Aim
2 will compare longitudinal data from 6 year-old children with and without SCI on exit MRI. Exploratory aims
will explore differences in the neuroimaging measures in children on different disease-modifying treatments
(hydroxyurea) and plasma protein levels in children with and without SCI. The proposed work will determine
which neuroimaging measures and neurocognitive testing may predict SCI, leading to a multi-site study to
validate these findings in a larger regionally heterogenous SCD population. The study PI, a
neurodevelopmental physician, is an early stage investigator well suited to expand her existing study cohort
with support from a team of co-investigators, including a senior behavioral psychologist and neuroimaging
physicist, as well as existing collaborators and staff.
期刊论文(0)
专著(0)
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会议论文
Neuroimaging, Neurocognitive, and Plasma Protein Markers of Brain Injury in Young Children with Sickle Cell Disease
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批准号:10281597
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项目类别:
-
资助金额:$8.1万
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财政年份:2021
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负责人:EBONI I LANCE
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依托单位:
Neuroimaging, Neurocognitive, and Plasma Protein Markers of Brain Injury in Young Children with Sickle Cell Disease
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批准号:10470928
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项目类别:
-
资助金额:$8.1万
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财政年份:2021
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负责人:EBONI I LANCE
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依托单位:
Clinical and Neuroimaging Phenotypes of Neurodevelopmental Disorders inPediatric Sickle Cell Disease
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批准号:9385561
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项目类别:
-
资助金额:$18.14万
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财政年份:2017
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负责人:EBONI I LANCE
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依托单位:
Clinical and Neuroimaging Phenotypes of Neurodevelopmental Disorders inPediatric Sickle Cell Disease
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批准号:9539716
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项目类别:
-
资助金额:$19.59万
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财政年份:2017
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负责人:EBONI I LANCE
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依托单位:
海外基金