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Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy
Published on: January 12, 2019
Outcome measures for children with movement disorders
Emanuela Pagliano1, Giovanni Baranello1, Riccardo Masson1
1Developmental Neurology Unit, C. Besta Neurological Institute Foundation, Milan, Italy.
Insights
Advances in pediatric neurosciences offer new treatment models for childhood dystonia. This review discusses current assessment tools, highlighting the need for standardized, comprehensive measures for effective pediatric dystonia management.
Area of Science:
- Pediatric Neurosciences
- Developmental Neurology
- Rehabilitation Medicine
Background:
- Recent decades show significant advances in pediatric neurosciences, clinical practice, and therapeutics for dystonia.
- Childhood dystonia impacts motor function, affecting perceptive, motor, and cognitive development and adaptive capabilities.
- Understanding treatment effects on plasticity and neuronal reorganization is crucial for early intervention.
Purpose of the Study:
- To review and discuss the strengths and weaknesses of current dystonia assessment tools in pediatric populations.
- To address the need for more comprehensive and generally-accepted outcome measures in pediatric dystonia.
- To inform the development of improved therapeutic strategies and rehabilitation models.
Main Methods:
- Literature review of neurophysiological studies, neuroimaging, and cognitive psychology research.
- Analysis of established assessment tools for childhood dystonia.
- Discussion of the International Classification of Functioning, Disability and Health (ICF) framework and family-centered care models.
Main Results:
- Current literature lacks universally accepted, exhaustive assessment tools for pediatric dystonia.
- Existing tools have limitations that necessitate further development and validation.
- Multidimensional therapeutic approaches are essential for managing childhood motor disorders.
Conclusions:
- There is a critical need for standardized, validated assessment tools for pediatric dystonia.
- An ecological, family-centered approach is vital for effective therapy and quality of life.
- Further research is required to develop and refine outcome measures for pediatric dystonia.
Abstract:
The huge contribution of advances in the pediatric neurosciences, improvements in clinical practice, and new therapeutic options, has led to the development of new models of treatment and rehabilitation for dystonia in the last decade. It is now generally agreed that a multidimensional therapeutic approach is needed for children with motor disorders, whose motor function-conceived as a complex perceptive, motor and cognitive process - is impaired at a crucial time in their development, with a fall out on how their various adaptive functions evolve. Neurophysiological studies, modern neuroimaging techniques, and advances in cognitive psychology have all contributed to improving our understanding of the potential effects of treatments in early age - not only on the symptoms, but also on plasticity processes and neuronal reorganization. The International Classification of Functioning, Disability and Health (ICF) promoted by the WHO, and the diffusion of family-centered models of healthcare have underscored the importance of the ecological perspective with a view to providing effective therapies and a satisfactory quality of life for dystonic children and their families. The advances made in this area have made it necessary to study and develope more appropriate treatment outcome measures. In the light of these aspects, there is still not enough literature on the generally-accepted, exhaustive dystonia assessment tools. Given these limits, it might be useful to discuss the strengths and weaknesses of the main tools currently used in this setting.
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