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

Research output: Contribution to journalComment/debatepeer-review

2 Scopus citations

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.

Original languageEnglish (US)
Pages (from-to)201-209
Number of pages9
JournalMolecular Genetics and Metabolism
Volume137
Issue number1-2
DOIs
StatePublished - Sep 1 2022

Bibliographical note

Funding Information:
The Webinar launched from the Robert J. Gorlin symposium, and Open Access fees, were supported by the National MPS Society.Work on CLN3 outcomes is supported by NIH/NINDS: 5U01NS101946-03

Funding Information:
Work on CLN3 outcomes is supported by NIH/NINDS : 5U01NS101946-03

Funding Information:
The Webinar launched from the Robert J. Gorlin symposium, and Open Access fees, were supported by the National MPS Society .

Publisher Copyright:
© 2022 Elsevier Inc.

Keywords

  • Ability score
  • Clinical outcomes
  • Clinical trials
  • Growth scale value
  • Measurement, neurocognitive decline

PubMed: MeSH publication types

  • Journal Article
  • Research Support, N.I.H., Extramural

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