TY - JOUR
T1 - Adapting a measure of gross motor skills for individuals with CDKL5 deficiency disorder
T2 - A psychometric study
AU - Saldaris, J. M.
AU - Jacoby, P.
AU - Marsh, E. D.
AU - Suter, B.
AU - Leonard, H.
AU - Olson, H. E.
AU - Rajaraman, R.
AU - Pestana-Knight, E.
AU - Weisenberg, J.
AU - Price, D.
AU - Drummond, C.
AU - Benke, T. A.
AU - Demarest, S.
AU - Downs, J.
N1 - Publisher Copyright:
© 2024 Elsevier B.V.
PY - 2024/2
Y1 - 2024/2
N2 - Purpose: Validated 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. Methods: Item 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. Results: The 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. 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.
AB - Purpose: Validated 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. Methods: Item 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. Results: The 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. 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.
KW - CDKL5 deficiency disorder
KW - Developmental epileptic encephalopathy
KW - Gross motor function
KW - Psychometric properties
UR - http://www.scopus.com/inward/record.url?scp=85183555833&partnerID=8YFLogxK
U2 - 10.1016/j.eplepsyres.2024.107287
DO - 10.1016/j.eplepsyres.2024.107287
M3 - Article
C2 - 38237219
AN - SCOPUS:85183555833
SN - 0920-1211
VL - 200
JO - Epilepsy Research
JF - Epilepsy Research
M1 - 107287
ER -