Psychometric Properties of the EQ-5D-Y-5L for Children With Intellectual Disability.

OBJECTIVES: The EQ-5D-Y-5L is a generic preference-based measure of health-related quality of life for children. This study aimed to describe the distributional properties, test-retest reliability, and convergent validity of the EQ-5D-Y-5L in children with intellectual disability (ID). METHODS: Caregivers of children with ID (aged 4 to 18 years) completed an online survey, including a proxy-report EQ-5D-Y-5L, the Quality-of-life Inventory-Disability, and disability-appropriate measures corresponding to the EQ-5D dimensions: mobility, self-care (SC), usual activities (UA), pain/discomfort (PD), and worry/sadness/unhappiness. Twenty-one participants repeated the EQ-5D-Y-5L a few weeks later. Test-retest reliability was computed using weighted kappa and intraclass correlation coefficients, and convergent validity using Spearman's and Pearson's correlation coefficients. RESULTS: Caregivers of 234 children completed the survey, with <1% missing values. Only 1.7% reported "no problems" on all dimensions (11111). The dimensions with the lowest percentage of "no problems" were SC and UA (both 8%). Test-retest reliability coefficients were fair to substantial for 4 dimensions (weighted kappa .30 to .79) but low for PD and overall health, as measured by the visual analog scale (EQ-VAS). Convergent validity was strong (Spearman's correlation .65 to .87) for mobility, SC, and PD; moderate to strong for worry/sadness/unhappiness (.47 to .60) and the EQ-VAS (Pearson's correlation .49); and weak to moderate for UA (.21 to .52). CONCLUSIONS: Convergent validity was generally good; test-retest reliability varied. Children with ID had lower scores on SC and UA than other populations, and their EQ-VAS could fluctuate greatly, indicating poorer and less stable health-related quality of life.

Can RESPiratory hospital Admissions in children with cerebral palsy be reduced? A feasibility randomised Controlled Trial pilot study protocol (RESP-ACT).

INTRODUCTION: The most common cause of morbidity and mortality in children with severe cerebral palsy (CP) is respiratory disease. BREATHE-CP (Better REspiratory and Airway Treatment and HEalth in Cerebral Palsy) is a multidisciplinary research team who have conducted research on the risk factors associated with CP respiratory disease, a systematic review on management and a Delphi study on the development of a consensus for the prevention and management of respiratory disease in CP. These strategies have not been investigated; therefore, it is not known if implementation is feasible, if they improve patient outcomes or if they are acceptable for families. METHODS AND ANALYSIS: Mixed-method feasibility pilot randomised controlled trial with economic analysis. Twenty children with CP aged 0-12 years who are at risk of respiratory disease will be followed up for 1 year. All children will receive baseline assessments for comparison. The control group will receive usual care from their treating teams. The intervention group will receive comprehensive assessments from physiotherapy, speech pathology and respiratory medicine. An individualised investigation and treatment plan will then be made. Participants in both groups will complete fortnightly patient-reported outcome surveys to assess symptoms and health service use. Analysis will include assessments of acceptability through qualitative interviews, implementation by ability to recruit, randomise and retain, practicality including costs of intervention and hospitalisation, and explore efficacy through quality-of-life surveys and decreased health service use for respiratory-related symptoms. ETHICS AND DISSEMINATION: Ethics and governance approvals have been obtained through Child and Adolescent Health Service Human Research Ethics Committee. At completion, this study will lead to the design of the definitive protocol to test intervention efficacy that maximises recruitment, retention and adherence to interventions. TRIAL REGISTRATION NUMBER: Australian New Zealand Clinical Trials Registry (ACTRN12620000114943).

The cost of respiratory hospitalizations in children with cerebral palsy.

AIM: To establish the burden of respiratory illness in cerebral palsy (CP) on the Western Australian health care system by quantifying the costs of respiratory hospitalizations in children with CP, compared with non-respiratory hospitalizations. METHOD: A 2-year (2014-2015) retrospective study using linked hospital data (excluding emergency department visits), in a population of children with CP in Western Australia aged 18 years and under (median age at hospitalization 7 years; interquartile range 5-12 years). RESULTS: In 671 individuals (57% male) there were 726 emergency hospitalizations, and 1631 elective hospitalizations. Although there were more elective hospitalizations, emergency hospitalizations were associated with longer stays in hospital, and more days in an intensive care unit, resulting in a higher total cost of emergency hospitalizations than elective hospitalizations (total costs: emergency AU$7 748 718 vs elective AU$6 738 187). 'Respiratory' was the leading cause of emergency hospitalizations, contributing to 36% of all emergency admission costs. For a group of high-cost inpatient users (top 5% of individuals with the highest total inpatient costs) the most common reason for hospitalization was 'respiratory'. Where non-respiratory admissions were complicated by an additional respiratory diagnosis, length of stay was greater. INTERPRETATION: Respiratory hospitalizations in CP are a significant driver of health care costs. In the paediatric group, they are a burden for a subgroup of children with CP. WHAT THIS PAPER ADDS: Respiratory illness is the most costly area for unplanned, emergency hospitalizations for children and young people with cerebral palsy. The top 5% of individuals with the highest total inpatient costs account for a disproportionate amount of health care costs.

How Well Does the EQ-5D-Y-5L Describe Children With Intellectual Disability?: "There's a Lot More to My Child Than That She Can't Wash or Dress Herself."

OBJECTIVES: The EQ-5D-5L is a generic health utility instrument for measuring health-related quality of life (HRQoL), with self-report and proxy report versions for children (EQ-5D-Y-5L). Children with intellectual disability (ID) are a heterogeneous population whose impairments and comorbidities place them at risk of poor HRQoL. This study aimed to describe the content validity and suitability for children with ID of a proxy report version of the EQ-5D-Y-5L as seen by their caregivers. METHODS: A proxy report EQ-5D-Y-5L was administered to caregivers of children with ID. Using cognitive think-aloud interviewing, participants were encouraged to provide the reasoning for their choices, assess the questions' relevance, comprehensibility, and comprehensiveness, and comment on the tool's strengths and weaknesses. Qualitative content analysis used both directed (deductive) and conventional (inductive) methods. RESULTS: There were 28 interviews with 30 caregivers of children with ID (aged 8-22 years, 17 boys, with autism spectrum disorder, cerebral palsy, Down syndrome, and rare genetic disorders). The EQ-5D-Y-5L was considered clear, concise, and largely relevant, but insufficiently comprehensive for this population. Interviewees sought clarification of the definition of HRQoL, whether it included unchanging impairments (vs fluctuating health states), and what basis of comparison to use (child or peer). Many interviewees suggested inclusion of questions for other domains, including communication and social engagement, equipment and human supports required, and a wider range of mental health questions. CONCLUSIONS: The study suggests that further work is required to ensure accurate responses to the EQ-5D-Y-5L from caregivers of children with ID and to describe these children adequately.

Can telehealth increase physical activity in individuals with Rett syndrome? A multicentre randomized controlled trial.

AIM: To evaluate the effects of a physical activity programme on sedentary behaviour and physical activity in ambulant individuals with Rett syndrome (RTT). METHOD: In this multicentre randomized waitlist-controlled trial, we recruited 43 ambulatory individuals with RTT in Australia and Denmark. Adequate baseline data were obtained from 38 participants (mean age 20 years, range 6-41, SD 10 years 6 months, one male). All completed the trial. Participants received 12 weeks of usual care (n = 19) or a goal-based, telehealth-supported programme in which activities occurred in their familiar environments (n = 19). Sedentary time and daily steps were assessed at baseline, post-test, and 12-week follow-up. The data analyst was blinded to group allocation. RESULTS: Sedentary time decreased in the intervention group by 2.7% (95% confidence interval [CI] -6.0 to 0.6) and increased in the control group by 1.3% (95% CI -4.8 to 7.4). Intervention and control groups increased the number of their steps per day by 264.7 (95% CI -72.2 to 601.5) and 104.8 (95% CI -178.1 to 387.7) respectively. No significant differences were found on any outcomes at post-test. There were three minor adverse events. INTERPRETATION: A goal-based telehealth intervention seemed to produce small improvements in physical activity for individuals with RTT. Families require more support to increase these individuals' extremely low physical activity levels. WHAT THIS PAPER ADDS: A telehealth-supported intervention may produce small changes in physical activity in Rett syndrome (RTT). Increasing physical activity in individuals with RTT is challenging for caregivers. Families require substantial out-of-home support to increase their children's activity levels.

Chronic Pain in Young People With Cerebral Palsy: Activity Limitations and Coping Strategies.

PURPOSE: To describe the effect of chronic pain on the activities of children and adolescents with cerebral palsy, to describe coping strategies, and to examine associations between effect of pain on activities, coping strategies, and level of pain. METHODS: Using an online survey, 27, 8- to 18-year-olds with cerebral palsy and chronic pain or their parents reported pain intensity, distribution, activity limitations (using the Child Activity Limitations Interview-21), and coping strategies (using the Pediatric Pain Coping Inventory). RESULTS: Pain intensity ranged from 3 to 10, and 89% reported pain in multiple areas, principally the lower limbs. Individuals reporting higher pain intensity had more activity limitations and were more likely to use catastrophizing coping strategies. CONCLUSIONS: Coping strategies may be an important mediator between pain and its effect on activities in children with cerebral palsy. Individualized pain management should be based on routine pain assessment.

Daytime sleepiness and emotional and behavioral disturbances in Prader-Willi syndrome.

Individuals with Prader-Willi syndrome (PWS) often have excessive daytime sleepiness and emotional/behavioral disturbances. The objective of this study was to examine whether daytime sleepiness was associated with these emotional/behavioral problems, independent of nighttime sleep-disordered breathing, or the duration of sleep. Caregivers of individuals with PWS (aged 3 to 25 years) completed the Pediatric Sleep Questionnaire (PSQ), Epworth Sleepiness Scale for Children and Adolescents (ESS-CHAD), and the parent version of the Developmental Behavior Checklist (DBC-P). Sleep adequacy was adjusted for age by computing sleep duration against age-specific recommendations. The associations between ESS-CHAD and the total DBC and its subscale scores were evaluated by linear regression, adjusted for sleep-related breathing difficulties, sleep adequacy, and body mass index (BMI). There were 54 responses for individuals with PWS (including 22 males) aged 4.4-24.0 (mean 12.5) years. Daytime sleepiness predicted a substantial proportion of the variance in total DBC-P scores in the unadjusted model (28%; ß = 0.028; p < 0.001) and when adjusted for sleep adequacy, BMI, and sleep-related breathing difficulties (29%; ß = 0.023; p = 0.007). This relationship was not moderated by BMI Z-scores, but the relationship was more prominent for children younger than 12 years than for children older than 12 years.Conclusions: These findings provide preliminary novel evidence that daytime sleepiness may drive the expression of emotional/behavioral disturbances, and should be explored as a potential modifiable risk factor for these disturbances in PWS, particularly pre-adolescent children.

Strengths and challenging behaviors in children and adolescents with Prader-Willi syndrome: Two sides to the coin.

Prader-Willi Syndrome (PWS) is a rare genetic disorder associated with emotional/behavioral disturbances. These difficulties are well documented in the literature, but the positive attributes of these individuals are not described. Taking a strengths-based approach, the aim of this study was to describe the emotional/behavioral strengths and difficulties in children and young people with PWS from their parent caregivers' perspectives. Parent caregivers of 52 individuals with PWS aged 4-24 years (median = 12.1 years; including 22 males) completed the parent form of the Developmental Behavior Checklist (DBC-P), including its original two open-ended questions regarding positive traits. Prevalences of emotional/behavioral disturbances were comparable to those reported in previous literature: common behaviors of concern across studies being skin-picking (75%), impulsivity (69%), poor sense of danger (67%), lying (67%), and tantrums (54%). Total DBC-P scores showed that just over half (n = 28, 54%) had scores indicative of clinically significant behavior problems. However, thematic analysis of caregivers' written comments regarding their children's strengths resolved into three themes: warmth (94%), persistence (41%), and skills (41%). Warmth encompassed friendliness, happiness, and empathy. A strength-based approach to behavioral difficulties in PWS provides a more balanced view of the children and a more holistic foundation for interventions.

Evaluation and Management of Respiratory Illness in Children With Cerebral Palsy.

Cerebral palsy (CP) is the most common cause of disability in childhood. Respiratory illness is the most common cause of mortality, morbidity, and poor quality of life in the most severely affected children. Respiratory illness is caused by multiple and combined factors. This review describes these factors and discusses assessments and treatments. Oropharyngeal dysphagia causes pulmonary aspiration of food, drink, and saliva. Speech pathology assessments evaluate safety and adequacy of nutritional intake. Management is holistic and may include dental care, and interventions to improve nutritional intake, and ease, and efficiency of feeding. Behavioral, medical, and surgical approaches to drooling aim to reduce salivary aspiration. Gastrointestinal dysfunction, leading to aspiration from reflux, should be assessed objectively, and may be managed by lifestyle changes, medications, or surgical interventions. The motor disorder that defines cerebral palsy may impair fitness, breathing mechanics, effective coughing, and cause scoliosis in individuals with severe impairments; therefore, interventions should maximize physical, musculoskeletal functions. Airway clearance techniques help to clear secretions. Upper airway obstruction may be treated with medications and/or surgery. Malnutrition leads to poor general health and susceptibility to infection, and improved nutritional intake may improve not only respiratory health but also constipation, gastroesophageal reflux, and participation in activities. There is some evidence that children with CP carry pathogenic bacteria. Prophylactic antibiotics may be considered for children with recurrent exacerbations. Uncontrolled seizures place children with CP at risk of respiratory illness by increasing their risk of salivary aspiration; therefore optimal control of epilepsy may reduce respiratory illness. Respiratory illnesses in children with CP are sometimes diagnosed as asthma; a short trial of asthma medications may be considered, but should be discontinued if ineffective. Overall, management of respiratory illness in children with CP is complex and needs well-coordinated multidisciplinary teams who communicate clearly with families. Regular immunizations, including annual influenza vaccination, should be encouraged, as well as good oral hygiene. Treatments should aim to improve quality of life for children and families and reduce burden of care for carers.

Systematic Review and Meta-analysis: Mental Health in Children With Neurogenetic Disorders Associated With Intellectual Disability.

OBJECTIVE: The behavioral phenotype of neurogenetic disorders associated with intellectual disability often includes psychiatric comorbidity. The objectives of this systematic review and meta-analysis were to systematically review the prevalence of psychiatric disorders and symptoms in children and adolescents with these disorders and compare phenotypic signatures between syndromes. METHOD: MEDLINE and PsycINFO databases were searched for articles from study inception to December 2018. Eligible articles were peer reviewed, were published in English, and reported prevalence data for psychiatric disorders and symptoms in children and adolescents aged 4 to 21 years using a formal psychiatric assessment or a standardized assessment of mental health symptoms. Pooled prevalence was determined using a random-effects meta-analysis in studies with sufficient data. Prevalence estimates were compared with general population data using a test of binomial proportions. RESULTS: Of 2,301 studies identified for review, 39 articles were included in the final pool, which provided data on 4,039 children and adolescents. Ten syndromes were represented, and five were predominant: Down syndrome, 22q11.2 deletion syndrome, fragile X syndrome, Williams syndrome, and Prader-Willi syndrome. The Child Behavior Checklist was the most commonly used assessment tool for psychiatric symptoms. The pooled prevalence with total scores above the clinical threshold was lowest for Down syndrome (32% [95% confidence interval, 19%-44%]) and highest for Prader-Willi syndrome (74% [95% CI, 65%-82%]) with each syndrome associated with significantly higher prevalence than in the general population. Parallel trends were observed for the internalizing and externalizing domains and social subscale scores. CONCLUSION: Differential vulnerability for psychiatric phenotype expression across the disorders was observed. Syndromes with higher levels of social ability or competence appear to offer relative protection against developing psychopathology. This preliminary finding merits further exploration.

Effects of Adaptive Bungee Trampolining for Children With Cerebral Palsy: A Single-Subject Study.

PURPOSE: To assess effects of adaptive bungee trampoline training for children with cerebral palsy. METHODS: This was a single-subject intervention study, A-B-A, with 4 children aged 6 to 11 years. Measurements included muscle strength, balance, functional muscle strength, functional mobility, selected Gross Motor Function Measure items, heart rate, enjoyment, and for adverse effects-range of motion and spasticity. Goals were measured using the Canadian Occupational Performance Measure. RESULTS: Lower limb muscle strength improved in 3 children, and balance and functional strength in 2 children. The child who was not walking increased sitting and supported standing times. All participants had clinically significant increases on the Canadian Occupational Performance Measure. Adherence and enjoyment were high, with no adverse effects. CONCLUSION: Adaptive bungee trampoline training can improve strength, balance, and functional mobility in children with cerebral palsy.

Sleep concerns in children and young people with cerebral palsy in their home setting.

AIMS: The aims were to identify in-home concerns about sleep in children and young people with cerebral palsy (CP) across age and Gross Motor Function Classification Scale (GMFCS) levels. METHODS: This was a retrospective review of clinical notes of 154 children and young people with CP, aged 1-18 years (M = 7.8; standard deviation = 5.4) who received a home-based sleep service. Reported concerns were synthesised, for analysis according to age groups (1-5, 6-13, 14-18) and GMFCS levels. RESULTS: Sixteen factors of concern were derived from the home-based assessment reports. Most children and young people had multiple factors of concern. These varied across age groups and GMFCS levels. Body position was of concern across all age groups, for over 90% at GMFCS levels IV and V, and for 10% at GMFCS level I. Settling routines were of concern for more than 90% at GMFCS levels I and II, but for less than 50% at GMFCS levels IV and V. Settling routines were of concern to over 65% of those under 6 years but less than 25% of those over 14 years. Conversely, pain and pressure care concerned less than 10% of children under 6, and more than 35% of those over 14 years. CONCLUSIONS: Concerns about sleep vary across ages and GMFCS levels of children and young people with CP. Concerns relate to impairment of body structure and function, activity, environment, and personal supports. Multi-disciplinary, home-based assessment and interventions are recommended to address these concerns.

Daily functional electrical stimulation during everyday walking activities improves performance and satisfaction in children with unilateral spastic cerebral palsy: a randomized controlled trial.

BACKGROUND: The aim of this paper is to determine whether daily functional electrical stimulation (FES) is effective in improving self-perceptions of individually identified mobility performance problems in children with unilateral spastic cerebral palsy (USCP). We hypothesized that children receiving 8 weeks of FES treatment would have higher scores for self-perceived performance and satisfaction on the Canadian Occupational Performance Measure (COPM) for individually identified priorities than children not receiving FES. METHODS: Thirty-two children (mean age 10 y 8 mo SD 3y 3mo) with USCP and a Gross Motor Function Classification System I or II were randomly assigned to the FES treatment group (8 weeks of daily FES) and control group (usual treatments). Participants were assessed at baseline (week 0), post treatment (week 8) and 6 weeks follow-up (week 14). The primary outcome measures were self-perceived scores for performance and satisfaction of child- and parent-identified priorities assessed using the COPM post treatment and at follow-up. The secondary outcome measures were the categorization of the performance problems from the COPM and self-report responses according to the International Classification of Functioning Child and Youth version (ICF-CY). This was clinically important because an understanding of mobility performance problems for children with USCP is needed for family-centred service planning. RESULTS: Performance scores (mean difference 1.6, 95 % CI 0.1 to 3.2, p = 0.034) and satisfaction scores post treatment (mean difference 2.4, 95 % CI 0.5 to 4.2, p = 0.004) were significantly higher in the treatment group than in the control group. There were no significant differences between the groups for performance scores at follow up, however there was a significant difference between the groups for satisfaction (mean difference 1.9, 95 % CI 0.1 to 3.8, p = 0.03) in favour of the treatment group. Priorities were identified across all levels of the ICF-CY but were most commonly identified in the activity and participation domains of the ICF-CY (79.5 %). CONCLUSIONS: Daily FES applied during everyday walking is effective in addressing self-perceptions of individually identified priorities by improving the performance and satisfaction of functional skills after treatment. TRIAL REGISTRATION: Australian New Zealand Clinical Trials Register ACTRN12614000949684. Registered 4 September 2014.

Effects of short-term daily community walk aide use on children with unilateral spastic cerebral palsy.

PURPOSE: To determine the effects of functional electrical stimulation (FES) on the main impairments affecting gait in children with unilateral spastic cerebral palsy. METHODS: A 20-week, multiple single-subject A-B-A design included a 6-week pre-FES phase, an 8-week FES phase, and a 6-week post-FES phase. Twelve children, aged 5 to 16 years, wore an FES device (the Walk Aide) daily for 8 weeks. Weekly measures included ankle range of motion, selective motor control, dorsiflexion and plantar flexion strength, gastrocnemius spasticity, single-limb balance, Observational Gait Scale (OGS) score, and self-reported toe drag and falls in the community. RESULTS: Compared with the pre-FES phase, the FES phase showed significant improvements in ankle range of motion, selective motor control and strength, and reductions in spasticity, toe drag, and falls, but no change in OGS score. These improvements were maintained during the post-FES phase. CONCLUSIONS: Intermittent, short-term use of FES is potentially effective for reducing impairments affecting gait in children with unilateral spastic cerebral palsy.