Evidence map›Paper›PMID 31298737›Full record

ArticleMuscle & nerve2019

Patient-reported study of the impact of pediatric-onset myotonic dystrophy.

Michael Hunter, Anne-Berit Ekstrom, Craig Campbell, Man Hung, Jerry Bounsanga, Kameron Bates, Heather R Adams, Elizabeth Luebbe, Richard T Moxley, Chad Heatwole and 1 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Muscle & nerve, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed
–field-weighted citation impact
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

2 citing papers in PubMed.

  1. Review
  2. 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

11 authors.

Michael HunterDepartment of Neurology, University of Utah School of Medicine, Salt Lake City, Utah.
Anne-Berit EkstromRegional Pediatric Rehabilitation Center, Queen Silvia Children's Hospital, Gothenburg, Sweden.
Craig CampbellDepartment of Pediatrics, Epidemiology and Clinical Neurology, University of Western Ontario, London Health Science Centre, London, Ontario, Canada.
Man HungCollege of Dental Medicine, Roseman University of Health Sciences, Henderson, Nevada.
Jerry BounsangaDepartment of Public Health, University of Utah, Salt Lake City, Utah.
Kameron BatesDepartment of Neurology, Virginia Commonwealth University, Richmond, Virginia.
Heather R AdamsDepartment of Neurology, University of Rochester, Rochester, New York.
Elizabeth LuebbeDepartment of Neurology, University of Rochester, Rochester, New York.
Richard T MoxleyDepartment of Neurology, University of Rochester, Rochester, New York.
Chad HeatwoleDepartment of Neurology, University of Rochester, Rochester, New York.
Nicholas E JohnsonDepartment of Neurology, Virginia Commonwealth University, Richmond, Virginia.ORCID 0000-0002-3917-4257

Funding

Health Endpoints and Longitudinal Progression in Congenital Myotonic Dystrophy (HELP-CDM)K23NS091511 · NINDS · VIRGINIA COMMONWEALTH UNIVERSITY · PI JOHNSON, NICHOLAS ELWOOD · 2015 to 2019
$997k
The Development and Use of a Disease-Specific Instrument for Myotonic DystophyK23AR055947 · NIAMS · UNIVERSITY OF ROCHESTER · PI HEATWOLE, CHAD RYDEL · 2008 to 2012
$648k
NIAMS NIH HHS K23 AR055947NINDS NIH HHS K23 NS091511
6 · The paper itself

Abstract

introductionThe prevalence and impact of symptoms affecting individuals with pediatric forms of myotonic dystrophy type-1 (DM1) are not well understood.

methodsPatients from the United States, Canada, and Sweden completed a survey that investigated 20 themes associated with pediatric-onset DM1. Participants reported the prevalence and importance of each theme affecting their lives. Surveys from participants were matched with surveys from their caregivers for additional analysis.

resultsThe most prevalent symptomatic themes included problems with hands or fingers (79%) and gastrointestinal issues (75%). Problems with urinary/bowel control and gastrointestinal issues were reported to have the greatest impact on patients' lives. Responses from participants and their caregivers had varying levels of agreement among symptomatic themes. DISCUSSION: Many symptoms have meaningful impact on disease burden. The highest levels of agreement between caregivers and individuals with pediatric forms of myotonic dystrophy were found for physical activity themes.

Indexed as

Activities of Daily LivingAdolescentAdultCaregiversChildChild, PreschoolCommunicationCost of IllnessDeglutition DisordersFemaleFingersGastrointestinal DiseasesHandHumansMaleMobility Limitationcaregivershealth status indicatorsmyotonic dystrophyneuromuscular diseasepediatric onsetsymptom assessment

Identifiers

PMID31298737
PMCPMC10970714

What OpenQuestion holds

Textmetadata
LicenceTDM
Read underepoch 390

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.