Child Outcome Measures Research Articles

Towards the harmonization of outcome measures in children with mitochondrial disorders

A clinical trial is only as reliable as its outcomes, therefore the careful and systematic selection of outcome measures is extremely important. Currently, the selection of outcome measures for clinical trials designed to evaluate new drugs in patients with mitochondrial disorders is inefficient and has not been addressed systematically. Given that meaningful data can be obtained only from trials in which outcomes are assessed using valid instruments, one should first focus on the validation of a set of selected instruments in the target population. The aim of this review is to systematically select a 'toolbox' of robust outcome measures that are relevant to all patients. Using an extensive search of published literature, we systematically compiled a toolbox with outcome measures based on a primary search for possible instruments Subsequently, we reduced this toolbox using strict criteria that were adapted from the United States Food and Drug Administration. A toolbox with clinically relevant and psychometrically robust instruments for performing clinical research in children with mitochondrial disorders was compiled. In coming years, more experience using these outcome measures in children with various mitochondrial disease phenotypes must be obtained before reliable conclusions regarding the validity of these instruments can be drawn.

Clozapine-Induced Restless Legs Syndrome Treated With Aripiprazole

Clozapine-Induced Restless Legs Syndrome Treated With Aripiprazole

Can balance in children with cerebral palsy improve through use of an activity promoting computer game?

This study aimed to evaluate if use of an activity promoting computer game, used in the home (Nintendo Wii Fit; Nintendo Co Ltd, Japan), could influence balance related outcome measures in children with cerebral palsy. Eighteen children with hemiplegic or diplegic cerebral palsy were recruited for the study. A randomised cross-over design was used with children tested at baseline, after five weeks of playing Wii Fit games and after five weeks without any intervention. Outcome measures of interest included: performance on the modified sensory organisation test, reactive balance test and rhythmic weight shift test. No significant difference was observed between testing occasions for any of the balance measures investigated (p > 0.05). Our results suggest that use of a Nintendo Wii balance board and Wii Fit software for a minimum of thirty minutes per day in the patient's own home, over a five week period, is not effective as a balance training tool in children with cerebral palsy.

Perimetry in Children: Survey of Current Practices in the United Kingdom and Ireland

Purpose: Visual fields are key functional outcome measures in children with a variety of ophthalmologic disorders. However, reliably assessing fields in children is challenging. We report the findings of a survey of current practices of perimetry in children in the United Kingdom and Ireland.Methods: An electronic questionnaire was sent to Orthoptic Service Heads in July 2008.Respondents were asked for comments regarding visual field testing in children as well as details of the volume and type of perimetry performed in their units, over a 1-year period.Results: Of the 98 (62%) completed questionnaires, 16 departments reported not testing visual fields in children. In total 3675 subjects under 16 years of age were reported to have undergone perimetry in 1 year, most in units with a ≥ 50% pediatric caseload for orthoptics. A total of 42% of units used static perimetry alone, 11% kinetic, and 47% used a combination of both.Conclusion: High numbers of visual field tests are carried out in children in the UK and Ireland annually. Automated perimetry is used predominantly, despite the underlying algorithms having been developed for adult populations. Thus there is a clear need for more research, to ensure that evolving management practices are informed by understanding of the diagnostic accuracy and value of perimetry in children.

Climate change and children's health--a call for research on what works to protect children.

Climate change is affecting and will increasingly influence human health and wellbeing. Children are particularly vulnerable to the impact of climate change. An extensive literature review regarding the impact of climate change on children’s health was conducted in April 2012 by searching electronic databases PubMed, Scopus, ProQuest, ScienceDirect, and Web of Science, as well as relevant websites, such as IPCC and WHO. Climate change affects children’s health through increased air pollution, more weather-related disasters, more frequent and intense heat waves, decreased water quality and quantity, food shortage and greater exposure to toxicants. As a result, children experience greater risk of mental disorders, malnutrition, infectious diseases, allergic diseases and respiratory diseases. Mitigation measures like reducing carbon pollution emissions, and adaptation measures such as early warning systems and post-disaster counseling are strongly needed. Future health research directions should focus on: (1) identifying whether climate change impacts on children will be modified by gender, age and socioeconomic status; (2) refining outcome measures of children’s vulnerability to climate change; (3) projecting children’s disease burden under climate change scenarios; (4) exploring children’s disease burden related to climate change in low-income countries; and (5) identifying the most cost-effective mitigation and adaptation actions from a children’s health perspective.

Young people's evaluation of a nurse‐led bladder training service: working towards developing a patient reported experience and outcome measure

ABSTRACTThe attainment of urinary continence is usually achieved in early childhood, however, many children and young people can experience challenges to their urinary continence during childhood and into adolescence. Urinary incontinence has been linked to physiological comorbidities such as constipation and urinary tract infections and can also affect the social confidence and quality of life for children and young people. The aim of this study was to develop an experience and outcome measure for children and young people who experience lower urinary tract symptoms. A questionnaire‐based evaluation to directly consult with young people attending a nurse‐led bladder training clinic was used, pre and post treatment session, to evaluate their experiences and reported outcomes. The findings demonstrated that many of the young people reported a good response to bladder training as a resolution or reduction in their urinary symptoms; however, some young people remained reluctant to discuss their continence problems in clinic. The questionnaires highlighted the value they placed on the psychosocial support from the nurses in the clinic and how this in turn influenced their reported self‐confidence and ability to manage their condition. Exploring reported experiences and outcomes can be helpful in determining what is important to young people attending a bladder training clinic. This has also highlighted the specific contribution nurses make in the assessment, intervention and management of this group.

The Brachial Plexus Outcome Measure: Development, Internal Consistency, and Construct Validity

PurposeThe purpose of this article was to report the development of a new assessment tool, the Brachial Plexus Outcome Measure (BPOM) and the evaluation of its internal consistency and construct validity. MethodsA retrospective case series of children aged 4–19 years with obstetrical brachial plexus palsy (OBPP) was conducted. Intraclass coefficients were calculated for the BPOM Activity Scale items. Correlation between the Active Movement Scale (AMS) and BPOM Activity Scale scores were conducted to determine the convergent validity. ResultsThe BPOM Activity Scale items (N=306) had good internal consistency (Cronbach's α=0.87). A strong correlation between the BPOM Activity Scale and AMS (n=284) was found (r=0.71, p=0.001, α=0.05). ConclusionsThe BPOM Activity Scale demonstrates good internal consistency and construct validity as a discriminative functional outcome measure in children with OBPP. Level of EvidenceIV

Outcome measurements in major trauma—Results of a consensus meeting

BackgroundThe NHS Outcomes Framework for England has identified recovery from major injury as an important clinical area. At present, there are no established outcome indicators. As more patients survive major trauma, outcomes will need to be measured in terms of morbidity and not mortality alone. ObjectiveTo make recommendations for a selection of outcome measures that could be integrated into National Clinical Audit data collection and form part of clinical governance requirements for Regional Trauma Networks (RTNs) and measures by which RTNs are held to account by government. Specific focus was given to acute care and rehabilitation for both adults and children. MethodA Multiprofessional, multidisciplinary expert group reviewed the current evidence on outcome measures for major trauma in the adult and children's populations, informed by a systematic review carried out jointly by the Trauma Audit and Research Network (TARN) and the Cochrane Injuries Group. A structured discussion covered functional and quality of life outcome measures as well as patient experience and indicators such as return to work, education and social dependency. ResultsFor the adult population the group agreed with the in-hospital performance and hospital discharge measures recommended in the TARN and Cochrane systematic review. Concerning longer-term outcome indicators, the group suggested the use of the Glasgow Outcome Scale – Extended (GOS-E) and European Quality of Life 5D (EQ-5D) with consideration to be given to the World Health Organisation Quality of Life survey (WHO-QoL). For patients who had ongoing inpatient rehabilitation needs the group thought the measurement of the Rehabilitation Complexity Scale (RCS) and Functional Independence Measure (FIM) were important in total brain injury and, the American Spinal Injury Association Impairment Scale (ASIA) and Spinal Cord Independence Measure (SCIM) in spinal cord injury. For children the group recommended the use of the King's Outcome Scale for Childhood Head Injury (KOSCHI) and Paediatric Quality of Life measure (Peds-QL) preferably at multiple intervals following injury to take into account effects on development. ConclusionSpecific recommendations were made for the use of outcome measures in adults and children with major trauma and those with complex rehabilitation needs following injury. More work on outcome measures in major trauma is needed especially for children. There are currently no robust measures of patient experience for use in major trauma. The importance of data linkage to allow measurement of non-clinical outcomes such as return to work, maintainence of education and societal dependency was emphasised by the group. A system for recording outcomes should be piloted post injury and at 6 and 12 months, with those still requiring inpatient rehabilitation after this time having longer follow up.

Examining the concurrent validity of assessing preschool language and literacy skills

The purpose of this study was to examine the concurrent validity of the assessment for an early language and literacy project. The goal of the project was to promote a successful transition to kindergarten for preschoolers who were at risk for reading difficulties by equipping them with the necessary language and literacy skills needed for early reading success. Both child outcome data and classroom/teacher level data were collected at the beginning and the end of the academic semester. Child-level pre- and post-tests indicated that participating children's language and literacy skills were improved in name writing, letter recognition, beginning sound awareness, rhyming awareness, and print knowledge. Findings from the validity study suggested a significant relationship between- and within-child outcome measures. A significant relationship was also found between norm-referenced child outcome measures and classroom-level measures; however, no significant relationship was identified between criterion-referenced child outcome measures and classroom-level measures. Implications of this study were discussed.

Parent goals as outcome measures for children receiving treadmill training: A series of case reports

Background: This series of case reports examined attainment of parents’ goals for children receiving treadmill training and parents’ satisfaction with goal attainment.Methods: Four children with cerebral palsy completed 5–8 weeks of standardized training while wearing a body weight support harness. Outcome measures were family goals using goal attainment scaling (GAS): a score of 0 equaled goal attainment, scores of −2 and −1 represented lower-than-expected performance and +1 and +2 exceeded expected performance. Parent satisfaction with goal attainment was assessed on a Likert scale from 1 (very satisfied) to 5 (very unsatisfied).Results: All participants achieved GAS levels of ≥0, indicating all goals were met. All parents rated their satisfaction with goal attainment as ‘very satisfied’.Conclusion: This is the first report using family goals as outcome measures to assess effects of treadmill training. Future studies of treadmill training should include family- and child-centred goals as potential outcomes.

Finding out what matters: validity of quality of life measurement in young people with ASD

Compared with other conditions there has been a lack of focus on quality of life (QoL) as an outcome measure for children and young people with Autism Spectrum Disorder (ASD). This pilot study aimed to evaluate the validity of existing QoL questionnaires for use with children with ASD aged 8-12 years. A literature review (1990-2011) identified the PedsQL (Pediatric Quality of Life Inventory) and Kidscreen as robust measures used with children with neurodevelopmental disorders. These measures were completed by 10 children and 11 parents. In addition semi-structured interviews were conducted with 10 parents and four children to explore their experience of completing the QoL questionnaires. Young people with ASD, and their parents, report lower child QoL compared with a normative sample. Framework analysis of the data highlighted six key themes which may affect the validity of generic QoL measures when administered within an ASD sample and which warrant further investigation. Our results indicate that a new condition-specific measure of QoL, grounded in ASD children's own perspectives of their lives, is needed and that such a measure should assess experiences of anxiety and access to special interests when measuring QoL of children with ASD. Active involvement of young people and their families is critical for the development of a theoretical framework for QoL within ASD, and any future development of an ASD-specific measure.

Validity of a Pediatric Version of the Glasgow Outcome Scale–Extended

The Glasgow Outcome Scale (GOS) and its most recent revision, the GOS-Extended (GOS-E), provide the gold standard for measuring traumatic brain injury (TBI) outcome. The GOS-E exhibits validity when used with adults and some adolescents, but validity with younger children is not established. Because the GOS-E lacks the developmental specificity necessary to evaluate children, toddlers, and infants, we modified the original version to create the GOS-E Pediatric Revision (GOS-E Peds), a developmentally appropriate structured interview, to classify younger patients. The criterion, predictive, and discriminant validity of the GOS-E Peds was measured in 159 subjects following TBI (mild: 36%; moderate: 12%; severe: 50%) at 3 and 6 months after injury. Participants were included from two studies completed at the Pediatric Neurotrauma Center at Children's Hospital of Pittsburgh. We assessed the relationship among GOS-E Peds, the GOS, and the Vineland Adaptive Behavior Scales as well as other standardized measures of functional, behavioral, intellectual, and neuropsychological outcome. Premorbid function was assessed 24-36 h after injury. The GOS-E Peds showed a strong correlation with the GOS at 3 and 6 month time points. Criterion-related validity was also indicated by GOS-E Peds' association with most measures at both time points and at injury severity levels. The 3 month GOS-E Peds was associated with the 6 month GOS-E Peds, everyday function, behavior, and most cognitive abilities. Discriminant validity is suggested by weak correlations between both 3 and 6 month GOS-E Peds and premorbid measures. The GOS-E Peds is sensitive to severity of injury and is associated with changes in TBI sequelae over time. This pediatric revision provides a valid outcome measure in infants, toddlers, children, and adolescents through age 16. Findings support using the GOS-E Peds as the primary outcome variable in pediatric clinical trials.

Feasibility, reliability and validity of the Thai version of the Pediatric Quality of Life Inventory 3.0 cerebral palsy module

Quality of Life (QOL) and Health-related Quality of Life (HRQOL) are now considered as necessary outcome measures for children with cerebral palsy (CP). Various reliable and valid condition-specific HRQOL tools are available for these children. One of these is Pediatric Quality of Life Inventory (PedsQL) 3.0 CP module which has been widely used and was translated to many languages. As no Thai version is available, the authors have completed this translation. This study then aimed to investigate psychometric properties of the newly translated Thai PedsQL 3.0 CP module and to establish parent confidence in their ratings in the translated tool. Translation of the PedsQL 3.0 CP module was performed based on linguistic translation guidelines. Then, the psychometric properties of the Thai version were established. PedsQL 3.0 CP module was completed by children with CP and their parents or caregivers twice with 2-4 weeks. Respondents were 97 parents or caregivers and 54 children. Minimal missing data were found. Acceptable internal consistency was supported except for Movement and Balance Scale (self-report). Intraclass correlation coefficients for parent proxy and self-report were good to excellent (0.684-0.950). The feasibility, reliability and validity of the translated tool were supported.

Parent needs and family support service outcomes in a Canadian sample

• Summary: Parent support needs and family support service (FSS) outcomes were investigated. A total of 923 parent participants were recruited through 20 community-based FSS providers in Alberta, Canada. Participants completed a survey, incorporating well validated child, parent and family outcome measures, a minimum of 8–12 weeks after utilizing their FSS. • Findings: Overall, parent need satisfaction was high. However, low socio-economic status, English as a second language, parental disability or chronic health condition, and child disability or chronic health condition were associated with lower levels of parent need satisfaction. Higher levels of need satisfaction were linked to lower levels of parenting stress and more positive parent–child interactions. These, in turn, were linked to more positive family functioning and fewer child difficulties. • Applications: Mediation analysis and qualitative findings suggest that family support services are making a positive difference by creating points of ‘connection’, including opportunities for informal learning and peer-support. We argue that the informal, ‘social networking’ role of family support services should be valued alongside evidence-based parenting training programs.

Commentary on “Are Spatiotemporal Gait Characteristics Reliable Outcome Measures in Children With Developmental Coordination Disorder?”

Commentary on “Are Spatiotemporal Gait Characteristics Reliable Outcome Measures in Children With Developmental Coordination Disorder?”

Recent advances in quantitative assessment of juvenile idiopathic arthritis

No single measure can serve as “gold standard” for the assessment of children with rheumatic diseases. Therefore, measurement of the disease status is based on the simultaneous assessment of multiple disease domains. Quantitative measures in pediatric rheumatology have been used primarily in clinical research and therapeutic trials, but have been seldom incorporated in standard clinical care. It is increasingly recognized that regular quantitative assessment in routine practice may help improve the quality of patient care. In the past decade, several new outcome measures for children with juvenile idiopathic arthritis have been developed and validated. Some of these instruments have been designed to be simple and feasible enough to enable their regular use in daily practice. The new measures include criteria for inactive disease, clinical remission and minimal disease activity, definitions of parent- and child-acceptable symptom state, composite disease activity scores, a damage index, questionnaires for the assessment of physical function and health-related quality of life, 21-numbered circle visual analog scales, and a multidimensional assessment questionnaire. The present review provides a brief description of these clinical tools.

P79 Do children describe the benefits of inhaled asthma therapy in the same way as adults?

IntroductionAlthough it is well reported in adults, there is relatively little data on how children with asthma and their parents describe their attitudes to the disease, expectations of therapy and...

Do Sex and Atopy Influence Cough Outcome Measurements in Children?

Despite the commonality of cough and its burden, there are no published data on the relationship between atopy or sex on objectively measured cough frequency or subjective cough scores in children. In 202 children with and without cough, we determined the effect of sex and atopy on validated cough outcome measurements (cough receptor sensitivity [CRS], objective cough counts, and cough scores). We hypothesized that in contrast to adult data, sex does not influence cough outcome measures, and atopy is not a determinant of these cough measurements. We combined data from four previous studies. Atopy (skin prick test), the concentration of capsaicin causing two and five or more coughs (C2 and C5, respectively), objectively measured cough frequency, and cough scores were determined and their relationship explored. The children's (93 girls, 109 boys) mean age was 10.6 years (SD 2.9), and 56% had atopy. In multivariate analysis, CRS was influenced by age (C2 coefficient, 5.9; P = .034; C5 coefficient, 29.1; P = .0001). Atopy and sex did not significantly influence any of the cough outcomes (cough counts, C2, C5, cough score) in control subjects and children with cough. Atopy does not influence important cough outcome measures in children with and without chronic cough. However, age, but not sex, influences CRS in children. Unlike adult data, sex does not affect objective counts or cough score in children with and without chronic cough. Studies on cough in children should be age matched, but matching for atopic status and sex is less important.

Healthy & Ready to Learn: Examining the Efficacy of an Early Approach to Obesity Prevention and School Readiness

The collision of the childhood obesity epidemic with pressure to achieve high academic standards is of serious concern in the United States. Growing numbers of low-income, minority children face double jeopardy as alarming obesity rates further widen existing achievement gaps. Health and education disparities persist when children enter kindergarten lacking fundamental school readiness skills and are also at risk of obesity. The goal of this study was to address serious gaps in research by examining the efficacy of an innovative program, Healthy & Ready to Learn, an early approach to obesity prevention and promotion of school readiness. The study targeted low-income, predominantly Latino preschoolers who are particularly at risk of health and educational disparities. The pretest–posttest, quasi-experimental study involved 405 children, ages 3 to 5 years, enrolled in four matched Head Start centers. To ensure rigorous assessment, the study used a battery of objective and validated instruments as direct measures of child outcomes. Using multilevel modeling, several linear growth models were conducted with participant's growth at Level-1 (i.e., time) and subject-level variables at Level-2 (i.e., treatment, gender, age, & body mass index classification) for each outcome variable of interest. Results revealed statistically significant improvements in growth (i.e., height), gross motor skills, physical activity levels, and receptive language development when comparing the treatment and control conditions. These promising results suggest that the Healthy & Ready to Learn program has potential as an early approach to improve children's health and, simultaneously, enhance their trajectory toward better academic performance.

Upper limb children action-observation training (UP-CAT): a randomised controlled trial in Hemiplegic Cerebral Palsy

BackgroundRehabilitation for children with hemiplegic cerebral palsy (HCP) aimed to improve function of the impaired upper limb (UL) uses a wide range of intervention programs. A new rehabilitative approach, called Action-Observation Therapy, based on the recent discovery of mirror neurons, has been used in adult stroke but not in children. The purpose of the present study is to design a randomised controlled trial (RCT) for evaluating the efficacy of Action-Observation Therapy in improving UL activity in children with HCP.Methods/DesignThe trial is designed according to CONSORT Statement. It is a randomised, evaluator-blinded, match-pair group trial. Children with HCP will be randomised within pairs to either experimental or control group. The experimental group will perform an Action-Observation Therapy, called UP-CAT (Upper Limb-Children Action-Observation Training) in which they will watch video sequences showing goal-directed actions, chosen according to children UL functional level, combined with motor training with their hemiplegic UL. The control group will perform the same tailored actions after watching computer games. A careful revision of psychometric properties of UL outcome measures for children with hemiplegia was performed. Assisting Hand Assessment was chosen as primary measure and, based on its calculation power, a sample size of 12 matched pairs was established. Moreover, Melbourne and ABILHAND-Kids were included as secondary measures. The time line of assessments will be T0 (in the week preceding the onset of the treatment), T1 and T2 (in the week after the end of the treatment and 8 weeks later, respectively). A further assessment will be performed at T3 (24 weeks after T1), to evaluate the retention of effects. In a subgroup of children enrolled in both groups functional Magnetic Resonance Imaging, exploring the mirror system and sensory-motor function, will be performed at T0, T1 and T2.DiscussionThe paper aims to describe the methodology of a RCT for evaluating the efficacy of Action-Observation Therapy in improving UL activity in children with hemiplegia. This study will be the first to test this new type of treatment in childhood. The paper presents the theoretical background, study hypotheses, outcome measures and trial methodology.Trial RegistrationNCT01016496