Evidence map›Paper›PMID 38216926›Full record

ArticleBMC pediatrics2024

Assessing the quality and value of metabolic chart data for capturing core outcomes for pediatric medium-chain acyl-CoA dehydrogenase (MCAD) deficiency.

Ryan Iverson, Monica Taljaard, Michael T Geraghty, Michael Pugliese, Kylie Tingley, Doug Coyle, Jonathan B Kronick, Kumanan Wilson, Valerie Austin, Catherine Brunel-Guitton and 40 more

Open access · goldAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in BMC pediatrics, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
0.4field-weighted citation impact, top 43% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

1 citing paper in PubMed, 1 citations in OpenAlex.

  1. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

50 authors at 20 institutions in 2 countries.

Ryan IversonSchool of Epidemiology and Public Health, University of Ottawa, Ottawa, Canada.
Monica TaljaardSchool of Epidemiology and Public Health, University of Ottawa, Ottawa, Canada.
Michael T GeraghtyDepartment of Pediatrics, Children's Hospital of Eastern Ontario and University of Ottawa, 401 Smyth Road, Ottawa, ON, K1H 8L1, Canada.
Michael PuglieseSchool of Epidemiology and Public Health, University of Ottawa, Ottawa, Canada.
Kylie TingleySchool of Epidemiology and Public Health, University of Ottawa, Ottawa, Canada.
Doug CoyleSchool of Epidemiology and Public Health, University of Ottawa, Ottawa, Canada.
Jonathan B KronickThe Hospital for Sick Children/University of Toronto, Toronto, Canada.
Kumanan WilsonSchool of Epidemiology and Public Health, University of Ottawa, Ottawa, Canada.
Valerie AustinThe Hospital for Sick Children/University of Toronto, Toronto, Canada.
Catherine Brunel-GuittonLe Centre Hospitalier Universitaire Ste-Justine, Montreal, Canada.
Daniela BuhasMcGill University Health Centre, Montreal, Canada.
Nancy J ButcherThe Hospital for Sick Children Research Institute/University of Toronto, Toronto, Canada.
Alicia K J ChanDepartment of Medical Genetics, University of Alberta/Stollery Children's Hospital, Edmonton, Canada.
Sarah DyackIWK Health Centre/Dalhousie University, Halifax, Canada.
Sharan GoobieIWK Health Centre/Dalhousie University, Halifax, Canada.
Cheryl R GreenbergHealth Sciences Centre Winnipeg/University of Manitoba, Winnipeg, Canada.
Shailly Jain-GhaiDepartment of Medical Genetics, University of Alberta/Stollery Children's Hospital, Edmonton, Canada.
Michal Inbar-FeigenbergThe Hospital for Sick Children/University of Toronto, Toronto, Canada.
Natalya KarpLondon Health Sciences Centre/Western University, London, Canada.
Mariya KozenkoMcMaster Children's Hospital, Hamilton, Canada.
Erica LangleyDepartment of Pediatrics, Children's Hospital of Eastern Ontario and University of Ottawa, 401 Smyth Road, Ottawa, ON, K1H 8L1, Canada.
Matthew LinesHamilton Health Sciences Centre/McMaster University, Hamilton, Canada.
Julian LittleSchool of Epidemiology and Public Health, University of Ottawa, Ottawa, Canada.
Jennifer MacKenzieMcMaster Children's Hospital, Hamilton, Canada.
Bruno MarandaCIUSSSE-CHUS, Université de Sherbrooke, Sherbrooke, Canada, Sherbrooke, Canada.
Saadet Mercimek-AndrewsThe Hospital for Sick Children/University of Toronto, Toronto, Canada.
Aizeddin MhanniHealth Sciences Centre Winnipeg/University of Manitoba, Winnipeg, Canada.
John J MitchellMcGill University Health Centre, Montreal, Canada.
Laura NagyThe Hospital for Sick Children/University of Toronto, Toronto, Canada.
Martin OffringaThe Hospital for Sick Children Research Institute/University of Toronto, Toronto, Canada.
Amy PenderMcMaster Children's Hospital, Hamilton, Canada.
Murray PotterMcMaster Children's Hospital, Hamilton, Canada.
Chitra PrasadLondon Health Sciences Centre/Western University, London, Canada.
Suzanne RatkoLondon Health Sciences Centre/Western University, London, Canada.
Ramona SalvarinovaBC Children's Hospital/University of British Columbia, Vancouver, Canada.
Andreas SchulzeThe Hospital for Sick Children/University of Toronto, Toronto, Canada.
Komudi SiriwardenaDepartment of Medical Genetics, University of Alberta/Stollery Children's Hospital, Edmonton, Canada.
Neal SondheimerThe Hospital for Sick Children/University of Toronto, Toronto, Canada.
Rebecca SparkesAlberta Children's Hospital/University of Calgary, Calgary, Canada.
Sylvia Stockler-IpsirogluBC Children's Hospital/University of British Columbia, Vancouver, Canada.
Kendra TapscottBC Children's Hospital/University of British Columbia, Vancouver, Canada.
Yannis TrakadisMcGill University Health Centre, Montreal, Canada.
Lesley TurnerJaneway Children's Hospital/Memorial University, St John's, Canada.
Clara Van KarnebeekBC Children's Hospital/University of British Columbia, Vancouver, Canada.
Anthony VandersteenIWK Health Centre/Dalhousie University, Halifax, Canada.
Jagdeep S WaliaKingston Health Sciences/Queen's University, Kingston, Canada.
Brenda J WilsonJaneway Children's Hospital/Memorial University, St John's, Canada.
Andrea C YuDepartment of Pediatrics, Children's Hospital of Eastern Ontario and University of Ottawa, 401 Smyth Road, Ottawa, ON, K1H 8L1, Canada.
Beth K PotterSchool of Epidemiology and Public Health, University of Ottawa, Ottawa, Canada.
Pranesh ChakrabortyDepartment of Pediatrics, Children's Hospital of Eastern Ontario and University of Ottawa, 401 Smyth Road, Ottawa, ON, K1H 8L1, Canada. pchakraborty@cheo.on.ca.
University of Ottawa · CAHospital for Sick Children · CAMcMaster Children's Hospital · CALondon Health Sciences Centre · CAMcGill University Health Centre · CASickKids Foundation · CADalhousie University · CAMemorial University of Newfoundland · CAUniversity of Alberta · CAUniversity of British Columbia · CAUniversity of Manitoba · CAUniversity of Toronto · CAAlberta Children's Hospital · CAAmsterdam University Medical Centers · NLBC Children's Hospital · CACentre Hospitalier Universitaire Sainte-Justine · CAChildren's Hospital of Eastern Ontario · CAHamilton Health Sciences · CAIzaak Walton Killam Health Centre · CANewborn Screening Ontario · CA

Funding

Canadian Institutes of Health Research (CIHR) TR3-119195
6 · The paper itself

Abstract

backgroundGenerating rigorous evidence to inform care for rare diseases requires reliable, sustainable, and longitudinal measurement of priority outcomes. Having developed a core outcome set for pediatric medium-chain acyl-CoA dehydrogenase (MCAD) deficiency, we aimed to assess the feasibility of prospective measurement of these core outcomes during routine metabolic clinic visits.

methodsWe used existing cohort data abstracted from charts of 124 children diagnosed with MCAD deficiency who participated in a Canadian study which collected data from birth to a maximum of 11 years of age to investigate the frequency of clinic visits and quality of metabolic chart data for selected outcomes. We recorded all opportunities to collect outcomes from the medical chart as a function of visit rate to the metabolic clinic, by treatment centre and by child age. We applied a data quality framework to evaluate data based on completeness, conformance, and plausibility for four core MCAD outcomes: emergency department use, fasting time, metabolic decompensation, and death.

resultsThe frequency of metabolic clinic visits decreased with increasing age, from a rate of 2.8 visits per child per year (95% confidence interval, 2.3-3.3) among infants 2 to 6 months, to 1.0 visit per child per year (95% confidence interval, 0.9-1.2) among those ≥ 5 years of age. Rates of emergency department visits followed anticipated trends by child age. Supplemental findings suggested that some emergency visits occur outside of the metabolic care treatment centre but are not captured. Recommended fasting times were updated relatively infrequently in patients' metabolic charts. Episodes of metabolic decompensation were identifiable but required an operational definition based on acute manifestations most commonly recorded in the metabolic chart. Deaths occurred rarely in these patients and quality of mortality data was not evaluated.

conclusionsOpportunities to record core outcomes at the metabolic clinic occur at least annually for children with MCAD deficiency. Methods to comprehensively capture emergency care received at outside institutions are needed. To reduce substantial heterogeneous recording of core outcome across treatment centres, improved documentation standards are required for recording of recommended fasting times and a consensus definition for metabolic decompensations needs to be developed and implemented.

Indexed as

Lipid Metabolism, Inborn ErrorsOutcome Assessment, Health CareAcyl-CoA DehydrogenaseCanadaChildChild, PreschoolHumansProspective StudiesAcyl-CoA DehydrogenaseCore outcome setData qualityMCAD deficiency

Identifiers

PMID38216926
PMCPMC10787451
OpenAlexW4390840255

What OpenQuestion holds

Textmetadata
LicenceCC BY
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Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.