Evidence map›Paper›PMID 36115283›Full record

ArticleMolecular genetics and metabolism

Increasing precision in the measurement of change in pediatric neurodegenerative disease.

J B Eisengart, M H Daniel, H R Adams, P Williams, B Kuca, E Shapiro

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular genetics and metabolism. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
15citing papers in PubMed, 1 pooled it
–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

15 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Caregiver Meaningful Score Differences and Meaningful Score Regions for the Observer-Reported Communication Ability Measure for Individuals with Angelman Syndrome.Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research · 2026
    Article
  3. Article
  4. In-depth profile analysis and developmental trends in Wechsler performance among individuals with Williams syndrome.Child neuropsychology : a journal on normal and abnormal development in childhood and adolescence · 2026
    Article
  5. Observational
  6. Article
  7. Review
  8. Article
  9. Article
  10. Article
  11. Article
  12. A tutorial on person ability scores for the IDD clinician.International journal of developmental disabilities · 2025
    Article
  13. Article
  14. Article
  15. 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

6 authors.

J B EisengartDepartment of Pediatrics, University of Minnesota, Minneapolis, MN, USA. Electronic address: eisen139@umn.edu.
M H DanielMark Daniel Services LLC, MN, USA.
H R AdamsDepartment of Neurology, University of Rochester, Rochester, NY, USA.
P WilliamsPearson, Minneapolis, MN, USA.
B KucaAllievex Corporation, Marblehead, MA, USA.
E ShapiroDepartment of Pediatrics, University of Minnesota, Minneapolis, MN, USA; Partner, Shapiro Neuropsychology Consulting, LLC, Portland, OR, USA.

Funding

Meaningful Outcomes and Multi-Site Readiness for Clinical Trials in Juvenile Neuronal Ceroid LipofuscinosisU01NS101946 · NINDS · UNIVERSITY OF ROCHESTER · PI AUGUSTINE, ERIKA · 2019 to 2024
$3.7M
NINDS NIH HHS U01 NS101946
6 · The paper itself

Abstract

Due to the surge in new brain-directed treatments, metrics to detect the alteration in developmental trajectories in cognition and adaptive behavior have become increasingly important. We propose Growth Scale Values (GSVs) as a solution to monitoring children with severe neurologic/neurodegenerative conditions. This report stems from a panel of experts presenting at the Gorlin symposium (WORLD Symposium) and a subsequent open Webinar sponsored by the National MPS Society. Because norm-referenced scores (Standard Scores or Intelligence Quotient, i.e., IQ) do not yield information about gain, stability, or loss of skills, they are not suitable for natural history studies or clinical trials. Age-equivalent (AE) scores have been the standard metric used in natural history studies. While AEs are familiar and interpretable to clinicians and parents, they are imprecise due to lack of standard deviations, standard errors of measurement, and equal intervals between scores. Raw scores also have unequal intervals and are not comparable between ages or ability levels. The GSV, a nonlinear transformation of raw scores using item calibration to make an interval scale score, can be used for accurate measures of within-person change. GSVs have been identified as a useful metric for longitudinal measurement of other conditions involving neurodiversity. These growth scores circumvent inaccurate AEs in infants, are not limited by age and can be used for impaired patients who are chronologically above the normative age range. GSVs have interval properties (a given difference between GSV values represents the same difference in ability at all score levels) and each GSV value has a known standard error of measurement (SEM). GSVs are recommended to measure change in cognitive and adaptive behavior in natural history studies and in clinical trials for children with neurologic disease.

Indexed as

Neurodegenerative DiseasesChildCognitionHumansInfantIntelligence TestsAbility scoreClinical outcomesClinical trialsGrowth scale valueMeasurement, neurocognitive decline

Identifiers

PMID36115283
PMCPMC9879307

What OpenQuestion holds

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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.