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Published on: January 12, 2019
Measurement of Motor Function in Children With Canavan Disease: Concordance Between Remote and In-Person Assessments
Elise L Townsend1, Michael Kiefer2, Beth Leiro3
1BridgeBio Gene Therapy LLC, d/b/a Aspa Therapeutics, Palo Alto, California; PhD in Rehabilitation Sciences Program, School of Health and Rehabilitation Sciences, MGH Institute of Health Professions, Boston, Massachusetts.
Background:
The increasing use of decentralized and hybrid clinical trials generates a critical need for validation of remote virtual assessments in pediatric rare diseases. This study evaluates the concordance between standardized motor assessments obtained remotely and in-person over 2 years in children with Canavan disease.
Methods:
Thirteen participants completed paired remote/in-person motor assessment visits with one of 5 trained physical therapist raters. Paired Hammersmith Infant Neurological Exam Section 2 (HINE-2) Motor Milestones and Gross Motor Function Measure (GMFM-88) scores were compared using Lin's Concordance correlation coefficients (CCCs) and Bland-Altman plots.
Results:
HINE-2 total scores from 85 remote/ in-person visit pairs were highly correlated (Lin's CCC = 0.98; P < 0.001) with low systematic assessment method bias and high precision. GMFM-88 remote/ in-person total raw scores were also highly correlated (Lin's CCC = 0.98; P < 0.001) with low systematic assessment method bias and modest difference score variability. Estimated mean paired differences between remote and in-person total scores was 0.01 for HINE-2 and 0.54 for GMFM-88.
Conclusions:
Findings from this study support the utility and validity of two remote motor clinical outcome assessments in children with Canavan disease and highlight the potential value of combining a motor milestone assessment (HINE-2) with a granular evaluation of motor function (GMFM-88) for a more comprehensive evaluation in medically high-risk children.
