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Assessment and Evaluation of the High Risk Neonate: The NICU Network Neurobehavioral Scale
Published on: August 25, 2014
Methods of neurodevelopmental assessment in children with neurodegenerative disease: Sanfilippo syndrome
Kathleen A Delaney1, Kyle R Rudser, Brianna D Yund
1Department of Pediatrics, University of Minnesota, Minneapolis, MN, USA, delan011@umn.edu.
Insights
This study developed a reliable method to assess cognitive and developmental data in children with neurodegenerative diseases, validating age-equivalent scores for monitoring disease progression and treatment outcomes.
Area of Science:
- Pediatric Neurology
- Neurodevelopmental Disorders
- Clinical Assessment
Background:
- Children with neurodegenerative diseases often present with cognitive impairment and behavioral challenges, complicating accurate assessment.
- Reliable data collection is crucial for monitoring disease progression and evaluating treatment efficacy in this population.
Purpose of the Study:
- To develop and validate a methodology for obtaining reliable cognitive and developmental data in children with neurodegenerative disease and cognitive impairment.
- To establish the validity of age-equivalent scores for monitoring disease state and treatment outcomes.
Main Methods:
- Guidelines were established for accurate testing in low-functioning and behaviorally disruptive pediatric patients.
- A validation study used disease-specific protocols, appropriate tests, and behavioral management techniques.
- Age-equivalent scores from standardized cognitive tools (Bayley Scales-III, KABC-II) were compared with parent-reported scores (Vineland Adaptive Behavior Scales-II) in 25 children with mucopolysaccharidosis type IIIA.
Main Results:
- Cognitive age-equivalents were successfully obtained for 25 patients with MPS IIIA, including those with severe behavioral disruption.
- A high correlation (0.95) was found between parent-reported scores and cognitive age-equivalents, validating the age-equivalent approach.
Conclusions:
- The implemented approach enables assessment of severely impaired children, including those with behavioral disruption.
- This methodology is applicable to children with other severe neurological diseases.
- The approach enhances disease progression assessment and treatment outcome monitoring in clinical trials.
Objectives:
(1) Develop a methodology for obtaining reliable cognitive and developmental data in children with neurodegenerative disease and cognitive impairment and in turn monitor disease state and treatment outcomes. (2) Demonstrate validity of age-equivalent scores.
Methods:
We present guidelines for obtaining accurate test scores in low-functioning and behaviorally disruptive pediatric patients, followed by a method validation study: (1) using disease-specific protocols to assess salient aspects of the known phenotype, (2) selecting appropriate tests, (3) managing behavior, and (4) using age-equivalent scores on standardized tools. We used the Bayley Scales of Infant Development-III or Kaufman Assessment Battery for Children-II with a group of 25 children with mucopolysaccharidosis type IIIA (MPS IIIA or Sanfilippo syndrome type A) with dementia. To demonstrate concurrent validity, we used the Vineland Adaptive Behavior Scales-II, comparing parent-reported age-equivalent scores (AEs) with those of the cognitive measures.
Results:
We were successful in obtaining cognitive age-equivalents for 25 patients with MPS IIIA including those with severe behavioral disruption and a correlation of 0.95 was obtained comparing scores on the parent measure with cognitive age-equivalents validating the age-equivalent approach.
Conclusion:
An approach to the assessment of severely impaired children including those with behavioral disruption was implemented and is applicable to children with other severe neurological diseases. This approach will enhance the assessment of disease progression and monitoring of treatment outcome in clinical trials.
