National Healthcare Quality and Disparities Report
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Search All Research Studies
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- Adverse Drug Events (ADE) (1)
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AHRQ Research Studies
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Research Studies is a compilation of published research articles funded by AHRQ or authored by AHRQ researchers.
Results
1 to 5 of 5 Research Studies DisplayedTreadwell JR, Kessler SK, Wu M
Pharmacologic and dietary treatments for epilepsies in children aged 1-36 months: a systematic review.
The purpose of this systematic review study was to assess the effectiveness and harms of pharmacologic and dietary treatments for epilepsy in children aged 1-36 months without infantile spasms. The researchers searched EMBASE, MEDLINE, PubMed, and the Cochrane Library for studies published from 1/1/1999 to 8/19/21 to identify studies reporting data on children aged 1-36 months receiving pharmacologic or dietary treatments for epilepsy. Twenty-three studies met the inclusion criteria. The study found that Levetiracetam leads to seizure freedom in some infants but the data on 6 other medications (lamotrigine, phenytoin, rufinamide, stiripentol, topiramate, and vigabatrin) were too limited to allow conclusions about their effectiveness. Three medications (lamotrigine, levetiracetam, and topiramate) were rarely discontinued due to adverse effects, and severe events were also rare. In the category of diets, the ketogenic diet lead to seizure freedom in some infants and both the ketogenic diet and modified Atkins diet reduced average seizure frequency.
AHRQ-funded; 75Q80120D00002.
Citation: Treadwell JR, Kessler SK, Wu M .
Pharmacologic and dietary treatments for epilepsies in children aged 1-36 months: a systematic review.
Neurology 2023 Jan 3;100(1):e16-e27. doi: 10.1212/wnl.0000000000201026..
Keywords: Children/Adolescents, Neurological Disorders, Newborns/Infants, Medication, Nutrition, Treatments, Evidence-Based Practice
Chen HF, Rose AM, Waisbren S
Newborn screening and treatment of phenylketonuria: projected health outcomes and cost-effectiveness.
This study’s objective was to evaluate the cost-effectiveness of newborn screening and treatment for phenylketonuria (PKU) in the context of new data on adherence to a recommended diet treatment and a newly available drug (sapropterin dihydrochloride). A computer simulation model was developed to project outcomes for a hypothetical cohort of newborns which compared four strategies. The strategies compared were: 1) clinical identification (CI) with diet treatment; 2) newborn screening (NBS) with diet treatment; 3) CI with diet and medication; and 4) NBS with diet and medication. The range of incremental cost-effectiveness ratio went from $6400/QALY for newborn screening with diet treatment compared to clinical identification with diet treatment up to $16,000,000/QALY for adding medication to NBS with diet treatment. Future research is needed to consider conditions under which sapropterin dihydrochloride would be more economically attractive.
AHRQ-funded; HS020644.
Citation: Chen HF, Rose AM, Waisbren S .
Newborn screening and treatment of phenylketonuria: projected health outcomes and cost-effectiveness.
Children 2021 May 12;8(5). doi: 10.3390/children8050381..
Keywords: Newborns/Infants, Neurological Disorders, Screening, Healthcare Costs
Feldman AG, Parsons JA, Dutmer CM
Subacute liver failure following gene replacement therapy for spinal muscular atrophy type 1.
This paper reports on two cases of transient, drug-induced liver failure after gene replacement therapy using an adeno-associated virus vector containing the survival motor neuron 1 gene.
AHRQ-funded; HS026510.
Citation: Feldman AG, Parsons JA, Dutmer CM .
Subacute liver failure following gene replacement therapy for spinal muscular atrophy type 1.
J Pediatr 2020 Oct;225:252-58.e1. doi: 10.1016/j.jpeds.2020.05.044..
Keywords: Newborns/Infants, Neurological Disorders, Genetics, Treatments, Adverse Drug Events (ADE), Adverse Events, Medication, Medication: Safety, Patient Safety, Case Study
Williams CN, Eriksson CO, Kirby A
Hospital mortality and functional outcomes in pediatric neurocritical care.
Pediatric neurocritical care (PNCC) outcomes research is scarce. In this study, the investigators aimed to expand knowledge about outcomes in PNCC by evaluating death and changes in Functional Status Scale (FSS) from baseline among PNCC diagnoses. The investigators concluded that PNCC patients had high rates of death and new disability at discharge, varying significantly between PNCC diagnoses. Multiple domains of disability were affected, underscoring the ongoing multidisciplinary health care needs of survivors.
AHRQ-funded; HS022981.
Citation: Williams CN, Eriksson CO, Kirby A .
Hospital mortality and functional outcomes in pediatric neurocritical care.
Hosp Pediatr 2019 Dec;9(12):958-66. doi: 10.1542/hpeds.2019-0173..
Keywords: Children/Adolescents, Newborns/Infants, Intensive Care Unit (ICU), Critical Care, Neurological Disorders, Mortality, Hospitals, Inpatient Care, Outcomes, Patient-Centered Outcomes Research
Carmody D, Pastore AN, Landmeier KA
Patients with KCNJ11-related diabetes frequently have neuropsychological impairments compared with sibling controls.
This study compared neurodevelopmental outcomes in patients with KCNJ11 mutations and their sibling controls. It found that patients with KCNJ11-related diabetes without global developmental delay had significant differences compared with sibling controls on a range of assessments including IQ, measures of academic achievement and executive function.
AHRQ-funded; HS023007.
Citation: Carmody D, Pastore AN, Landmeier KA .
Patients with KCNJ11-related diabetes frequently have neuropsychological impairments compared with sibling controls.
Diabet Med 2016 Oct;33(10):1380-6. doi: 10.1111/dme.13159.
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Keywords: Children/Adolescents, Diabetes, Disabilities, Neurological Disorders, Newborns/Infants