A Psychometric Evaluation of the Motor-Behavioral Assessment Scale for Use as an Outcome Measure in Rett Syndrome Clinical Trials

Melissa Raspa, Carla M. Bann, Angela Gwaltney, Timothy A. Benke, Cary Fu, Daniel G. Glaze, Richard Haas, Peter Heydemann, Mary Jones, Walter E. Kaufmann, David Lieberman, Eric Marsh, Sarika Peters, Robin Ryther, Shannon Standridge, Steven A. Skinner, Alan K. Percy, Jeffrey L. Neul

Research output: Contribution to journalArticlepeer-review

1 Scopus citations

Abstract

Rett syndrome (RTT) is a neurodevelopmental disorder that primarily affects females. Recent work indicates the potential for disease modifying therapies. However, there remains a need to develop outcome measures for use in clinical trials. Using data from a natural history study (n ¼ 1,075), we examined the factor structure, internal consistency, and validity of the clinician-reported Motor Behavior Assessment scale (MBA). The analysis resulted in a five-factor model: (1) motor dysfunction, (2) functional skills, (3) social skills, (4) aberrant behavior, and (5) respiratory behaviors. Item Response Theory (IRT) analyses demonstrated that all items had acceptable discrimination. The revised MBA subscales showed a positive relationship with parent reported items, age, and a commonly used measure of clinical severity in RTT, and mutation type. Further work is needed to evaluate this measure longitudinally and to add items related to the RTT phenotype.

Original languageEnglish
Pages (from-to)493-509
Number of pages17
JournalAmerican Journal on Intellectual and Developmental Disabilities
Volume125
Issue number6
DOIs
StatePublished - Nov 2020

Keywords

  • Clinical trial
  • Outcome measure
  • Psychometrics
  • Rett syndrome

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