<p><strong>BACKGROUND: </strong>Leukodystrophies are a rare class of disorders characterized by severe neuromotor disability. There is a strong need for research regarding the functional status of people with leukodystrophy which is limited by the need for in-person assessments of mobility. The purpose of this study is to assess the reliability of the Gross Motor Function Measure-88 (GMFM-88) using telemedicine compared with standard in-person assessments in patients with leukodystrophy.</p>

<p><strong>METHODS: </strong>A total of 21 subjects with a diagnosis of leukodystrophy (age range&nbsp;= 1.79-52.82&nbsp;years) were evaluated by in-person and by telemedicine evaluations with the GMFM-88 by physical therapists. Inter-rater reliability was assessed through evaluation of the same subject by two independent raters within a three-week period (n&nbsp;=&nbsp;10 encounters), and intrarater reliability was assessed through blinded rescoring of video-recorded assessments after a one-week time interval (n&nbsp;=&nbsp;6 encounters).</p>

<p><strong>RESULTS: </strong>Remote assessments were performed by caregivers in all 21 subjects using resources found in the home with remote guidance. There was agreement between all paired in-person and remote measurements (Lin's concordance correlation ≥0.995). The Bland-Altman analysis indicated that the paired differences were within ±5%. Intrarater and inter-rater reliability demonstrated an intraclass correlation coefficient of &gt;0.90.</p>

<p><strong>CONCLUSIONS: </strong>These results support that remote application of the GMFM-88 is a feasible and reliable approach to assess individuals with leukodystrophy. Telemedicine application of outcome measures may be of particular value in rare diseases and those with severe neurologic disability that impacts the ability to travel.</p>

<p><strong>Background: </strong>It is unknown what role specific tasks associated with personal care, positioning, communication and social interaction, and comfort and emotions play in predicting the overall health and quality of life of individuals with non-ambulatory cerebral palsy (CP). In this study, we prospectively evaluated which of these factors were significant predictors of overall health and quality of life.</p>

<p><strong>Methods: </strong>Parents and guardians of non-ambulatory children, adolescents and young adults with CP were prospectively recruited from the Cerebral Palsy Clinic of a large pediatric academic hospital. Caregivers completed the CP Child Questionnaire®. Univariate analyses were used to identify relationships between overall health, overall quality of life (QOL), and responses in the following categories: personal care and activities of daily living, positioning and transfer mobility, comfort and emotions, and communication and social interaction. Significant predictors of overall health and QOL were then determined via logistic regression.</p>

<p><strong>Results: </strong>64 patients ages 0-20 years and Gross Motor Function Classification System levels IV and V were included in our study (mean age 9.16 ±4.96 years). Overall QOL (OR 194.2, 95% CI, 9.5-3964.9) and comfort while sitting (OR 15.9, 95% CI, 1.2-205.3) were significant predictors of overall health. Feelings of unhappiness or sadness (OR 59.9, 95% CI, 1.6-2209.8), difficulty understanding the parent or guardian (OR 29.8, 95% CI, 1.6-543.7), and not attending school (OR 57.2, 95% CI, 2.6-1274.4) were significant predictors of lower overall quality of life.</p>

<p><strong>Conclusions: </strong>Overall QOL appears to predict overall health. Factors associated with comfort and emotions and communication and social interaction appear to predict overall QOL to a greater extent than personal care and transfer mobility. Level of Evidence: Prognostic II.</p>