Non-invasive risk assessment tool for pediatric heart failure
Non-invasive risk assessment tool for pediatric heart failure
批准号:
9410210
负责人:
CARMEN C SUCHAROV
金额:
$15.35万
依托单位:
依托单位国家:
美国
项目类别:
财政年份:
2017
资助国家:
美国
项目状态:
已结题
起止时间:
2017-09-01 至 2019-02-28
关键词:
AdultAlgorithmsAssessment toolBiological MarkersBiotechnologyBloodBusinessesCardiacCaringCategoriesCathetersCessation of lifeChildChildhoodClinicalColoradoDevelopmentDiagnosisDilated CardiomyopathyDiseaseDisease OutcomeFacultyFamilyGene Expression ProfileGoalsHealthHealth ExpendituresHeart TransplantationHeart failureHospitalsHuman ResourcesIncidenceIndustryInstitutionLeadLifeLongevityMeasurementMeasuresMethodsMicroRNAsMolecular ProfilingNewly DiagnosedOperative Surgical ProceduresOutcomePatient riskPatient-Focused OutcomesPatientsPhasePhysiciansPopulationProceduresProspective StudiesProtocols documentationQuantitative Reverse Transcriptase PCRReadinessRecoveryRelative RisksResearchResearch DesignRiskRisk AssessmentRisk stratificationSamplingSerumStatistical Data InterpretationTechniquesTestingTransplantationTreatment outcomeUniversitiesVentricular FunctionWorkagedbasecirculating microRNAcomparison groupdesigndisease phenotypeexperimental studyfollow-upheart functionimprovedmicroRNA biomarkersoutcome forecastpatient populationpediatric cardiologistpediatric heart failurepediatric patientsphase II trialpreventprospectivetool
中文摘要
摘要
英文摘要
ABSTRACT
Heart Failure (HF) in pediatric patients is a devastating disease that is difficult to evaluate, even with
invasive hospital procedures. Importantly, and unique to the pediatric population, approximately 25% of
pediatric HF patients spontaneously recover normal heart function within 1 year of diagnosis. Unfortunately,
there is no reliable clinical parameter or biomarker that allows pediatric cardiologists to correctly predict which
of their HF patients will recover normal heart function without surgical intervention, forcing clinicians to place
their these children on transplant lists even though surgical intervention would be unnecessary in some cases.
Offering a potential solution to this problem, a recent study performed at the University of Colorado Denver has
identified unique expression signatures of microRNAs found in circulating blood that correlate with a
subpopulation of pediatric HF patients who recover ventricular function without surgical intervention. It is the
objective of this Phase I project to complete initial proof-of-concept experiments related to the development of
a commercial test based on the measurement of these circulating miRNAs. The work proposed to complete
this project is designed to provide the groundwork for a prospective Phase II trial. By establishing and
validating the test protocols with control samples, this project will create the basis for a test that can then be
evaluated in a subsequent prospective study designed to tests newly diagnosed pediatric HF patients and
follow their treatment outcomes. The successful development of a non-invasive test for pediatric HF patients
would have significant patient and commercial value. By providing clinicians with a non-invasive risk
stratification tool for pediatric patients with HF, a test of this kind would (1) help clinicians, patients and families
understand health related prognosis for appropriate long-term planning, (2) reduce the number of invasive
procedures performed on these compromised patients, (3) prevent unnecessary transplantations, improving
survival for decades beyond the current 10-20 year lifespan of a transplanted heart, and (4) decrease health
care expenditures associated with cardiac transplantation.
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海外基金