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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. [ABSTRACT FROM AUTHOR]

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.

We report a case study of cognitive linguistic treatment in a teenager with chronic severe Landau Kleffner Syndrome. The effect of speech and language therapy in LKS is rarely examined and our case is unique in that we use an effective approach in adult aphasia to treat language deficits in aphasia in LKS. The results show successful acquisition of a considerable amount of new words as well as improved communication in daily life. However, auditory verbal agnosia, the most prominent feature in LKS, persisted. Cognitive linguistic treatment seems a promising treatment to improve spoken language production in LKS, but more research is needed to optimize speech and language therapy of auditory verbal agnosia and auditory language comprehension in children with LKS., Competing Interests: Declaration of conflicting interests: The authors declared no potential conflict of interests with respect to the research, authorship, and/or publication of this article., (© The Author(s) 2021.)

Background: Different interventions are offered to children with cerebral palsy (CP) to improve the activity domain of the international classification of functioning (ICF). In therapy settings, the focus is mostly on motor capacity, but the ultimate goal is to improve motor performance. We therefore examined if changes in motor capacity outcomes are accompanied by changes in objectively measured motor performance after a 3-month intensive treatment period in ambulatory children with CP., Methods: A secondary analysis on prospective clinical trial data was performed using multivariate linear regression. Sixty-five children (37 boys and 28 girls) with spastic CP, mean age 7 years and 3 months, Gross Motor Function Classification System (GMFCS) levels I-III were involved in a distinct 3-month intensive treatment period. Motor capacity (Gross Motor Function Measure [GMFM], functional muscle strength [FMS], and walking speed [WS]) and motor performance (using three Actigraph-GT3X+-derived outcome measures) were measured at baseline, 12 and 24 weeks., Results: No significant associations were found for any of the change scores (∆ 12 ) between motor capacity and motor performance after a 12-week intensive treatment period. After 24 weeks, ∆ 24 FMS (p = .042) and ∆ 24 WS (p = .036) were significantly associated with changes in motor performance outcome measure percentage of time spent sedentary (∆ 24 %sedentary). In this model, 16% of variance of ∆ 24 %sedentary was explained by changes in motor capacity (p = .030)., Conclusions: Changes in motor capacity are mostly not accompanied by changes in objectively measured motor performance after an intensive treatment period for ambulatory children with CP. These findings should be taken into account during goal setting and are important to manage expectations of both short- and longer term effects of treatment programmes., (© 2019 The Authors. Child: Care, Health and Development published by John Wiley & Sons Ltd.)

Objective: Botulinum toxin (BoNT-A) is widely used in combined treatment for spastic cerebral palsy, but its added value preceding comprehensive rehabilitation for motor impairments, gait, and goal attainment has not been studied., Design: A comparative multi-centre trial, in which two groups underwent comprehensive rehabilitation (i.e. high-intensive functional physiotherapy, and indicated casting/orthoses). One group received intramuscular BoNT-A prior to rehabilitation, and the other group did not receive BoNT-A., Subjects/patients: Children with spastic cerebral palsy, Gross Motor Function Classification System (GMFCS) levels I-III, age range 4-12 years, indicated for BoNT-A treatment regarding mobility problems., Methods: Sixty-five children participated (37 boys), mean age 7.3 years (standard deviation (SD) 2.3, range 4-12 years), equally distributed across GMFCS levels. Forty-one children received BoNT-A+ comprehensive rehabilitation and 24 received comprehensive rehabilitation only. Functional leg muscle strength, passive range of motion, angle of catch, cerebral palsy-related pain, walking speed, kinematic gait parameters, goal attainment, and proxy-reported general functioning were assessed at baseline, primary end-point (12 weeks) and 24-week follow-up. Statistical analyses were performed with linear mixed models., Results: At the primary end-point there were no statistically significant differences in treatment effects between the groups, except for the angle-of-catch of the rectus femoris, which was in favour of comprehensive rehabilitation without BoNT-A (12° difference, 95% confidence interval (95% CI) 2:23, p = 0.025). Results at follow-up were similar., Conclusion: At the group level, treating with BoNT-A prior to comprehensive rehabilitation did not add to the clinical effectiveness of rehabilitation. Thus, BoNT-A prescription and use should be critically reconsidered in this cerebral palsy age- and GMFCS-subgroup.

Objective: Despite the widespread use of botulinum toxin in ambulatory children with spastic cerebral palsy, its value prior to intensive physiotherapy with adjunctive casting/orthoses remains unclear., Design: A pragmatically designed, multi-centre trial, comparing the effectiveness of botulinum toxin + intensive physiotherapy with intensive physiotherapy alone, including economic evaluation., Subjects/patients: Children with spastic cerebral palsy, age range 4-12 years, cerebral palsy-severity Gross Motor Function Classification System levels I-III, received either botulinum toxin type A + intensive physiotherapy or intensive physiotherapy alone and, if necessary, ankle-foot orthoses and/or casting., Methods: Primary outcomes were gross motor func-tion, physical activity levels, and health-related quality-of-life, assessed at baseline, 12 (primary end-point) and 24 weeks (follow-up). Economic outcomes included healthcare and patient costs. Intention-to-treat analyses were performed with linear mixed models., Results: There were 65 participants (37 males), with a mean age of 7.3 years (standard deviation 2.3 years), equally distributed across Gross Motor Function Classification System levels. Forty-one children received botulinum toxin type A plus intensive physio-therapy and 24 received intensive physiotherapy treatment only. At primary end-point, one statistically significant difference was found in favour of intensive physiotherapy alone: objectively measured percentage of sedentary behaviour (-3.42, 95% confidence interval 0.20-6.64, p=0.038). Treatment costs were significantly higher for botulinum toxin type A plus intensive physiotherapy (8,963 vs 6,182 euro, p=0.001). No statistically significant differences were found between groups at follow-up., Conclusion: The addition of botulinum toxin type A to intensive physiotherapy did not improve the effectiveness of rehabilitation for ambulatory children with spastic cerebral palsy and was also not cost-effective. Thus botulinum toxin is not recommended for use in improving gross motor function, activity levels or health-related quality-of-life in this cerebral palsy age- and severity-subgroup.

Objective: To assess quality of life of adults with Duchenne muscular dystrophy in the Netherlands and to identify domains and major problems influencing quality of life., Design: Cross-sectional., Subjects: Seventy-nine men aged ≥ 20 years with Duchenne muscular dystrophy., Methods: The Medical Outcome Study Short Form-36 (SF-36), World Health Organization Quality of Life - BREF (WHOQOL-BREF) and an interview were used to assess quality of life and problems., Results: Compared with Dutch general population reference values, the SF-36 domains scores were lower on all domains except mental health and role limitations due to emotional problems. On the WHOQOL-BREF the social relationships domain score was lower. Main problems were intimate relationships, work, leisure, transport and meaningfulness of life. Seventy-three percent stated overall quality of life as "(very) good". The SF-36 domains mental health (rs 0.53, p < 0.001) and vitality (rs 0.49, p < 0.001) had the strongest associations with overall quality of life., Conclusion: Adult men with Duchenne muscular dystrophy assess their health status as low in the physical, but not in the mental, domains. Experienced problems are mainly in the area of participation. They are generally satisfied with their overall quality of life.

Objective: To determine upper limb function and associated factors in adults with Duchenne muscular dystrophy., Design: Cross-sectional study., Subjects: A sample of 70 men with Duchenne muscular dystrophy (age range 20-43 years)., Methods: General motor function and, in particular, upper limb distal motor function, were assessed with the Motor Function Measure. Muscle strength and range of motion of the upper limb were evaluated using hand-held dynamo-metry, manual muscle-testing and goniometry. Associations were studied using Spearman's correlation coefficients and multiple linear regression analysis., Results: General motor function was severely impaired. Wide variability was found in distal motor function, muscle strength and range of motion of the upper limb, especially in early adulthood. Muscle strength and range of motion explained 76% of the variance in upper limb distal motor function., Conclusion: This study illustrates a large variability in upper limb function in adult patients with Duchenne muscular dystrophy, and identifies muscle strength and range of motion as factors strongly associated with upper limb function. These results suggest that preserving muscle strength and range of motion in Duchenne patients might be relevant for a better outcome of distal motor function of the upper limb when adult.