Factors for changes in self-care and mobility capabilities in young children with cerebral palsy involved in regular outpatient rehabilitation care

BACKGROUND: Assessing prognosis of self-care and mobility capabilities in children with cerebral palsy (CP) is important for goal setting, treatment guidance and meaningful professional-caregiver conversations. AIMS: Identifying factors associated with changes in self-care and mobility capabilities in regular outpatient multidisciplinary paediatric CP rehabilitation care. METHODS AND PROCEDURES: Routinely monitored longitudinal data, assessed with the Paediatric Evaluation of Disability Inventory (PEDI-Functional-Skills-Scale, FSS 0–100) was retrospectively analysed. We determined contributions of age, gross-motor function, bimanual-arm function, intellectual function, education type, epilepsy, visual function, and psychiatric comorbidity to self-care and mobility capability changes (linear-mixed-models). OUTCOMES AND RESULTS: For 90 children (53 boys), in all Gross-Motor-Function-Classification-System (GMFCS) levels, 272 PEDI's were completed. Mean PEDI–FSS–scores at first measurement (median age: 3,2 years) for self-care and mobility were 46.3 and 42.4, and mean final FSS-scores respectively were 55.1 and 53.1 (median age: 6,5 years). Self-care capability change was significantly associated with age (2.81, p < 0.001), GMFCS levels III-V (-9.12 to -46.66, p < 0.01), and intellectual impairment (-6.39, p < 0.01). Mobility capability change was significantly associated with age (3.25, p < 0.001) and GMFCS levels II-V (-6.58 to -47.12, p < 0.01). CONCLUSIONS AND IMPLICATIONS: Most important prognostic factor for self-care and mobility capabilities is GMFCS level, plus intellectual impairment for self-care. Maximum capability levels are reached at different ages, which is important for individual goal setting and managing expectations

The Role of Botulinum Toxin in Multimodal Treatment of Spasticity in Ambulatory Children with Spastic Cerebral Palsy: Extensive Evaluation of a Cost-effectiveness Trial

Background: A cost-effectiveness trial (the Space Bop study) on the added value of botulinum toxin injections (BoNT-A) in the leg muscles, as part of a multimodal intervention for ambulatory children with spastic cerebral palsy in the context of a single distinct cycle of care was performed recently by our group. For a broad set of effect outcomes, we found that BoNT-A had no added value if children received comprehensive rehabilitation. However, this counterintuitive finding was met with scepticism.Objective: Since several noteworthy facts and experiences were recorded during the course of the trial and the dissemination phase, the aim of this paper was to describe and discuss some crucial aspects of, and barriers to, the Space Bop study, related to context and perspective, design and results, as well as publication and implementation.Methods: This paper discusses 5 issues: (i) the design, interpretation and presentation of previous research; (ii) the role of one's own clinical experience and interpretation; (iii) the aims of (BoNT-A) treatment; (iv) conflict of interest, role of industry, and the role of history; (v) optimal treatment modalities and dose-response relationships.Conclusion: Despite the unambiguous findings from the Space Bop study, several factors hindered acceptance of the results. Awareness of these factors is important when performing rehabilitation research and disseminating and implementing research findings

Prevalence of Fatigue, Pain, and Affective Disorders in Adults With Duchenne Muscular Dystrophy and Their Associations With Quality of Life

Abstract Objectives To assess the prevalence of fatigue, pain, anxiety, and depression in adults with Duchenne muscular dystrophy (DMD), and to analyze their relationship with health-related quality of life. Design Cross-sectional study. Setting Home of participants. Participants Adults (N=80) with DMD. Interventions Not applicable. Main Outcome Measures Fatigue was assessed with the Fatigue Severity Scale; pain with 1 item of the Medical Outcomes Study 36-Item Short-Form Health Survey and by interview; and anxiety and depression by using the Hospital Anxiety and Depression Scale. Health-related quality of life was assessed using the World Health Organization Quality of Life Scale-Brief Version. Associations between these conditions and quality of life were assessed by means of univariate and multivariate logistic regression analyses. Results Symptoms of fatigue (40.5%), pain (73.4%), anxiety (24%), and depression (19%) were frequently found. Individuals often had multiple conditions. Fatigue was related to overall quality of life and to the quality-of-life domains of physical health and environment; anxiety was related to the psychological domain. Conclusions Fatigue, pain, anxiety, and depression, potentially treatable symptoms, occur frequently in adults with DMD and significantly influence health-related quality of life

Subjective caregiver burden of parents of adults with Duchenne muscular dystrophy

Purpose: To describe subjective caregiver burden of parents of adults with Duchenne muscular dystrophy (DMD) and to identify factors associated with the level of subjective burden. Methods: In a cross-sectional study in 80 parents of 57 adult, severely disabled DMD patients' level of subjective caregiver burden was measured with the Caregiver Strain Index (CSI) and the Self Rated Burden Scale. A visual analogue scale was used to measure happiness. The expanded CSI version, the CarerQoL and open questions were used to analyse caregiving in more depth. In uni and multivariate analyses, associations of objective care characteristics, patient characteristics and caregiver characteristics with burden were explored. Results: Parents indicated substantial burden, but valued giving care as being important and rewarding. Subjective burden was associated with received support, tracheotomy, active coping by the patient and anxiety in patient and parents, together explaining 34%-36% of variance. Living situation was not associated with experienced burden. Conclusions: Caring for an adult son with DMD is burdensome, but rewarding. Subjective caregiver burden of parents may be alleviated by adequate support and respite care, by avoiding tracheotomy and by intervention programs targeting anxiety and promoting active coping by the patient from childhood

Subjective caregiver burden of parents of adults with Duchenne muscular dystrophy

Purpose: To describe subjective caregiver burden of parents of adults with Duchenne muscular dystrophy (DMD) and to identify factors associated with the level of subjective burden. Methods: In a cross-sectional study in 80 parents of 57 adult, severely disabled DMD patients' level of subjective caregiver burden was measured with the Caregiver Strain Index (CSI) and the Self Rated Burden Scale. A visual analogue scale was used to measure happiness. The expanded CSI version, the CarerQoL and open questions were used to analyse caregiving in more depth. In uni and multivariate analyses, associations of objective care characteristics, patient characteristics and caregiver characteristics with burden were explored. Results: Parents indicated substantial burden, but valued giving care as being important and rewarding. Subjective burden was associated with received support, tracheotomy, active coping by the patient and anxiety in patient and parents, together explaining 34%-36% of variance. Living situation was not associated with experienced burden. Conclusions: Caring for an adult son with DMD is burdensome, but rewarding. Subjective caregiver burden of parents may be alleviated by adequate support and respite care, by avoiding tracheotomy and by intervention programs targeting anxiety and promoting active coping by the patient from childhoo

Quantification of the development of trunk control in healthy infants using inertial measurement units

Trunk motor control is essential for the proper functioning of the upper extremities and is an important predictor of gait capacity in children with delayed development. Early diagnosis and intervention could increase the trunk motor capabilities in later life, but current tools used to assess the level of trunk motor control are largely subjective and many lack the sensitivity to accurately monitor development and the effects of therapy. Inertial measurement units could yield an objective quantitative assessment that is inexpensive and easy-to-implement. We hypothesized that root mean square of jerk, a proxy for movement smoothness, could be used to distinguish age and thereby presumed motor development. We attached a sensor to the trunks of six young children with no known developmental deficits. Root mean square of jerk decreases with age, up to 24 months, and is correlated to a more established method, i.e., center-of-pressure velocity, as well as other standard inertial measurement unit outputs. This metric therefore shows potential as a method to differentiate trunk motor control levels

Quantification of the development of trunk control in healthy infants using inertial measurement units

Trunk motor control is essential for the proper functioning of the upper extremities and is an important predictor of gait capacity in children with delayed development. Early diagnosis and intervention could increase the trunk motor capabilities in later life, but current tools used to assess the level of trunk motor control are largely subjective and many lack the sensitivity to accurately monitor development and the effects of therapy. Inertial measurement units could yield an objective quantitative assessment that is inexpensive and easy-to-implement. We hypothesized that root mean square of jerk, a proxy for movement smoothness, could be used to distinguish age and thereby presumed motor development. We attached a sensor to the trunks of six young children with no known developmental deficits. Root mean square of jerk decreases with age, up to 24 months, and is correlated to a more established method, i.e., center-of-pressure velocity, as well as other standard inertial measurement unit outputs. This metric therefore shows potential as a method to differentiate trunk motor control levels.</p