Evidence map›Paper›PMID 38237219›Full record

ArticleEpilepsy research2024

Adapting a measure of gross motor skills for individuals with CDKL5 deficiency disorder: A psychometric study.

J M Saldaris, P Jacoby, E D Marsh, B Suter, H Leonard, H E Olson, R Rajaraman, E Pestana-Knight, J Weisenberg, D Price and 4 more

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

Article in Epilepsy research, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing papers in PubMed
3.9field-weighted citation impact, top 7% 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

4 citing papers in PubMed, 7 citations in OpenAlex.

  1. Article
  2. Observational
  3. Article
  4. 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

14 authors at 10 institutions in 2 countries.

J M SaldarisTelethon Kids Institute, Centre for Child Health Research, The University of Western Australia, Perth, WA, Australia.
P JacobyTelethon Kids Institute, Centre for Child Health Research, The University of Western Australia, Perth, WA, Australia.
E D MarshDivision of Child Neurology, Children's Hospital of Philadelphia and Departments of Neurology and Pediatrics, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.
B SuterDepartment of Pediatrics & Neurology, Baylor College of Medicine, Houston, TX, USA.
H LeonardTelethon Kids Institute, Centre for Child Health Research, The University of Western Australia, Perth, WA, Australia.
H E OlsonDivision of Epilepsy and Clinical Neurophysiology and Epilepsy Genetics Program, Department of Neurology, Boston Children's Hospital, Boston, MA, USA.
R RajaramanUCLA Mattel Children's Hospital, Los Angeles, CA, USA.
E Pestana-KnightCleveland Clinic, Neurological Institute, Cleveland, OH, USA.
J WeisenbergSt. Louis Children's Hospital and Washington University School of Medicine, St Louis, MO, USA.
D PriceNYU Langone Health and Department of Neurology, New York University, New York, NY, USA.
C DrummondTelethon Kids Institute, Centre for Child Health Research, The University of Western Australia, Perth, WA, Australia.
T A BenkeUniversity of Colorado, School of Medicine, Depts Pediatrics, Neurology and Pharmacology and Children's Hospital Colorado, Aurora, CO, USA.
S DemarestUniversity of Colorado, School of Medicine, Depts Pediatrics, Neurology and Pharmacology and Children's Hospital Colorado, Aurora, CO, USA. Electronic address: Scott.Demarest@childrenscolorado.org.
J DownsTelethon Kids Institute, Centre for Child Health Research, The University of Western Australia, Perth, WA, Australia; Curtin School of Allied Health, Curtin University, Perth, WA, Australia. Electronic address: Jenny.Downs@telethonkids.org.au.
University of Western Australia · AUUniversity of Colorado Denver · USBaylor College of Medicine · USBoston Children's Hospital · USCleveland Clinic · USCurtin University · AUMattel Children's Hospital · USNYU Langone Health · USSt. Louis Children's Hospital · USUniversity of Pennsylvania · US

Funding

Multi-Site Validation of Biomarkers and Core Clinical Outcome Measures for Clinical Trials Readiness in CDKL5 Deficiency DisorderU01NS114312 · NINDS · UNIVERSITY OF COLORADO DENVER · PI BENKE, TIMOTHY A, DOWNS, JENNY · 2021 to 2025
$4.7M
NINDS NIH HHS U01 NS114312
6 · The paper itself

Abstract

purposeValidated measures capable of demonstrating meaningful interventional change in the CDKL5 deficiency disorder (CDD) are lacking. The study objective was to modify the Rett Syndrome Gross Motor Scale (RSGMS) and evaluate its psychometric properties for individuals with CDD.

methodsItem and scoring categories of the RSGMS were modified. Caregivers registered with the International CDKL5 Clinical Research Network uploaded motor videos filmed at home to a protected server and completed a feedback questionnaire (n = 70). Rasch (n = 137), known groups (n = 109), and intra- and inter-rater reliability analyses (n = 50) were conducted.

resultsThe age of individuals with CDD ranged from 1.5 to 34.1 years. The modified scale, Gross Motor-Complex Disability (GM-CD), comprised 17 items. There were no floor or ceiling effects and inter- and intra-rater reliability were good. Rasch analysis demonstrated that the items encompassed a large range of performance difficulty, although there was some item redundancy and some disordered categories. One item, Prone Head Position, was a poor fit. Caregiver-reported acceptability was positive. Scores differed by age and functional abilities. SUMMARY: GM-CD appears to be a suitable remotely administered measure and psychometrically sound for individuals with CDD. This study provides the foundation to propose the use of GM-CD in CDD clinical trials. Longitudinal evaluation is planned.

Indexed as

Epileptic SyndromesRett SyndromeSpasms, InfantileAdolescentAdultChildChild, PreschoolHumansInfantMotor SkillsProtein Serine-Threonine KinasesPsychometricsReproducibility of ResultsYoung AdultCDKL5 protein, humanProtein Serine-Threonine KinasesCDKL5 deficiency disorderDevelopmental epileptic encephalopathyGross motor functionPsychometric properties

Identifiers

PMID38237219
PMCPMC11725349
OpenAlexW4390999885

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.