Résumé :
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Background : The natural history of psychological dysfunction and quality of life in children with neuromuscular disease has not been defined. Given the expected clinical deterioration in these conditions and the significant improvements that may be seen with interventions such as non-invasive ventilation, it is important to establish a baseline in this population. Methods : This prospective study enrolled a cohort of 34 children 6-17 years, with static or progressive congenital or genetically-confirmed neuromuscular disease, recruited from two tertiary care pediatric hospital clinics. Exclusion criteria were known developmental disability and/or lack of comprehension of English or French. All participants underwent testing at baseline and 1 year later, consisting of overnight polysomnography, pulmonary function testing, manual muscle strength assessment (Kendall Scale), rating scales of behavioural and cognitive functioning (Connor's Rating Scale, Behavior Assessment System for Children and Behavior Rating Inventory of Executive Function) and quality of life (Child Health Questionnaire, Quality of Life for Families of Children with a Neuromuscular Condition). A pre-post design was used to compare differences from baseline in variables measured 1 year later. Results : The only significant change over 1 year seen in this cohort was a subtle improvement in manual muscle strength testing3 (median 0.6; p=0.015). There were no significant changes in pulmonary function, psychological measures of behavior, executive functioning, or any significant changes in physical, social or emotional well-being in children without nocturnal hypoventilation (NH) (N=30). Their parents however, reported greater disturbances in their own sleep, necessitated by attending to their child's physical needs during the night, 1 year later (median -2; p=0.005). The 4 participants identified with NH at baseline had significant differences over time in pulmonary function (median RV/TLC ratio 7.5 vs. -3, p=0.031) and muscle strength (median -0.7 vs. 0.6, p=0.007). Conclusions : Children with neuromuscular disease showed no evidence of deterioration in muscle strength, pulmonary function, psychological function and quality of life over one year. Those with hypoventilation may have a more rapid progression of changes in pulmonary function and/or muscle strength. Changes in these parameters in an individual would therefore be significant and may require further investigation as to underlying cause, so that intervention strategies can be implemented.
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