Parent-reported Outcome Measures Research Articles

Parent-Reported Outcome Measures for Individuals with Fragile X Syndrome: Clinically Meaningful Change Thresholds.

Estimating meaningful change thresholds (MCT) on clinical outcome assessments is an important consideration when evaluating treatments. In fragile X syndrome (FXS) research, there has been no consensus on how to define MCT's on several commonly used outcome measures. The purpose of the current study was to determine clinically relevant MCT's of caregiver-rated assessments using data from a phase 3 clinical trials of arbaclofen (Berry-Kravis et al., 2017). Data were collected as a part of previous phase 3, double-blind, placebo-controlled studies of arbaclofen in individuals with FXS (Berry-Kravis et al., 2017). The two studies enrolled age groups of 5-11-years (n = 159) and 12-50-years (n = 119). The current study examines meaningful within-patient change thresholds from baseline to treatment week 8 across several measures: ABC-CFXS; PSI; Vineland-II; and a Visual Analog Scale (VAS) of Anxiety and Disruptive Behaviors. MCT's were established by using anchor-based methods, using the CGI-S and CGI-I as anchors. Examining the results of the anchor-based analyses and visual CDF plots, MCT's were observed for the pediatric study for the ABC-CFXS subscales (with a range depending on use of CGI-S or CGI-I as anchor): Irritability: 11.1-14.8 points; Hyperactivity: 6.7-8.9 points; and Socially Unresponsive/Lethargic: 6.6-8.1 points; as well both VAS subscales: Anxiety: 28.3-36.2mm; and Disruptive Behavior: 22.4-27.4mm. Such thresholds were not observed for the Vineland-II and PSI subscales. Our analysis of MCT's helps set the stage for interpreting clinical trial results in FXS. This may include use of relevant subscales of the ABC-CFXS and VAS as primary outcomes using the MCT's for response definition. This work may help define future study inclusion criteria and enable future interpretation of treatment outcome results in the field.

Parent-reported outcome measures evaluating communication in individuals with rare neurodevelopmental disorders: A systematic review.

Communication impairments are a leading concern for parent caregivers of individuals with rare neurodevelopmental disorders (RNDDs). Clinical trials of disease modifying therapies require valid and responsive outcome measures that are relevant to individuals with RNDDs. Identifying and evaluating current psychometric properties for communication measures is a critical step towards the selection and use of appropriate instruments. This systematic review offers (1) a description of parent-reported communication measures and (2) evidence for their psychometric properties, in RNDDs. The systematic review protocol was registered in the International Prospective Register of Systematic Reviews (PROSPERO; CRD42022334649). MEDLINE (Ovid), Embase, PsychINFO, Web of Science, CINAHL Plus, Cochrane Library, ClinicalTrials.gov, the Australian New Zealand Clinical Trials Registry were searched from inception to August 2023. Methodological assessment of quality was completed using the COnsensus-based Standards for the selection of health status Measurement INstruments (COSMIN) checklist. Parent-reported measures used in observational studies and clinical trials were identified. Data on utility, reliability and validity for RNDDs were extracted. Sixteen parent-reported communication measures were used in RNDD research, the Vineland Adaptive Behavior Scales being most commonly used. Validation data in RNDDs were identified for six of these measures. Limitations related to sample size or the scope of psychometric testing. Many communication measures have been used for RNDDs but there are few data validating their use. Valid and reliable methods of measuring communication in persons with RNDDs is a priority for future high-quality clinical trials. What is already known on the subject Communication is a critical domain for families with a child with a rare neurodevelopmental disorder (RNDD). Validated outcome measures are essential for accurate evaluation and interpretation of responses to treatments in clinical trials. What this paper adds to existing knowledge We identified 16 parent-reported communication measures that have been used with RNDDs, but only six measures had validation data for at least one RNDD. High quality evidence is accumulating, with all validation studies in this review published between 2020 to 2023. Modifications of existing measures may be required to assess communication for RNDDs. What are the clinical implications of this work? This systematic review catalogues the available psychometric data for communication measures and indicates an ongoing need for new validation studies to ensure they are fit-for-purpose for upcoming clinical trials in RNDDs. This review will inform the selection of communication measures for clinical trials and research studies.

Pediatric Outcomes Data Collection Instrument Scores Within Gross Motor Function Classification Scale Levels and Functional Mobility Scale Ratings in Individuals With Cerebral Palsy.

The Pediatric Outcomes Data Collection Instrument (PODCI) is a patient/parent-reported outcome measure used in children with cerebral palsy (CP). PODCI score variability has not been widely examined in patients of Gross Motor Function Classification System (GMFCS) level IV or using the Functional Mobility Scale (FMS). The purpose of this study is to examine the distribution of PODCI scores within patients with CP GMFCS levels I-IV and FMS levels 1-6. Retrospectively identified patients with CP whose parent/caregiver had completed the PODCI at their visit were grouped based on GMFCS and FMS level. One-way ANOVA with pairwise Bonferroni-adjusted post hoc tests was performed to compare the effect of GMFCS and FMS levels (1, 2-4, 5, or 6) on PODCI scores. Three hundred sixty-seven patients were included (128 female, 11.7 years, SD 3.6). Global, Sports, Transfer, and Upper Extremity scores differed among all GMFCS levels ( P ≤0.056) and were significantly lower for GMFCS IV compared with all other levels. Happiness, Expectations, and Pain scores did not differ significantly among GMFCS levels including level IV ( P >0.06). Similar trends were seen at all FMS distances (5, 50, and 500âm). At 50âm, Global, Sports, Transfer, and Upper Extremity scores differed significantly among all FMS levels ( P <0.001) except that Upper Extremity Scores were similar between levels 2-4 and level 5 ( P =1.00). Happiness and Pain scores were not different between FMS levels ( P >0.27). Expectations scores differed only between FMS 1 and FMS 6 with FMS 6 being higher at the 50-m distance only ( P =0.03). Parent-reported outcome measures are important for providing patient-centered care. Providers can examine these measures alongside functional classification systems to create a more complete clinical picture of the patient. Providers should be aware of the score trends seen in our results when evaluating the PODCI for individuals with CP to improve shared decision-making and better monitor their need for future care. Level III-retrospective study.

Sepsis epidemiology in Australian and New Zealand children (SENTINEL): protocol for a multicountry prospective observational study

IntroductionSepsis affects 25.2 million children per year globally and causes 3.4 million deaths, with an annual cost of hospitalisation in the USA of US$7.3 billion. Despite being common, severe and...

84 The Association between Family Relations and School Outcomes in School-Aged Children 5-12 years old

Abstract Introduction School-aged children between the ages of 5 and 12 years face new environments and experiences as they transition through grade school. They are still developing and learning as they engage within their home and carry these experiences into their school environment. This study is an assessment of the association between family relations and school outcomes in children 5 to 12 years of age as measured by the field-tested SA-LIBRE5-12 (School-Aged Life Impact Burn Recovery Evaluation), a new parent-reported outcome measure. Methods Within the social and family functioning domain of the SA-LIBRE5-12, 10 family relations and 13 school items were scored on a scale from 0 (Never) to 4 (Always). Scores were re-coded so that higher scores denote better functioning. An exploratory factor analysis (EFA) was conducted to confirm model fit. A regression model assessed the relationship between the school and family factors, controlling for demographic and clinical characteristics (child sex, race, ethnicity, age at survey completion, burn size, burns to critical areas, and pain severity). Results A total of 356 parents completed these items from the SA-LIBRE5-12. EFA resulted in four sub domains of family engagement, family stress impact, school success, and school difficulties. A univariate analysis of the demographic characteristics showed that the child being female (p=0.008) and non-white (p=0.0216) had a better mean score in school success (3.33 vs. 3.19 + 0.78 (SD) and 3.38 vs. 3.21 + 0.75 (SD), respectively). In addition, children with a burn size &amp;lt; 1% had better school difficulties score (3.76 vs 3.61 + 0.38, p=0.0018). Regression analysis showed positive correlations between family engagement and school difficulties (r=0.26, p&amp;lt; .0001), family engagement and school success (r = 0.56, p&amp;lt; .0001), family stress impact and school success (r=0.21, p&amp;lt; .0001) and family stress impact and school difficulties (r=0.49, p&amp;lt; .0001). Conclusions This study indicates that a cohesive family relationship and home life may promote productive school outcomes in children between the ages of 5 and 12 based on parent reported outcomes. Applicability of Research to Practice This research will provide burn care providers, parents, and teachers with greater insight into the relationship between a child’s current family relations and their success in school. Results will guide caregivers in fostering a productive and healthy family environment.

112 Computer Adaptive Test Development of Preschool Life Impact Burn Recovery Evaluation

Abstract Introduction Measuring physical, psychological, and social outcomes following childhood burn injury is important to understand the impact of the burn. Condition-specific outcomes are highly relevant in assessments for purposes of gauging early growth and development through parent-reported outcome measures (PROMs). Item content in existing PROMs for preschool children is limited and often covers only one construct. Computer adaptive tests (CATs) with advanced psychometric artificial intelligence technologies minimizes respondent burden and enables the creation of a comprehensive condition-specific instrument. This project developed a parent-reported CAT (Preschool1-5 Life Impact Burn Recovery Evaluation (PS-LIBRE1-5) Profile) to measure relevant outcomes after burn injuries in children aged one to under six years. Methods Responses to the field-tested PS-LIBRE1-5 Profile (188 items) were measured on a scale of frequency or ability levels. Scores were coded from 0 to 4 where higher scores reflected better functioning. Factor analysis and Partial Credit Models identified the items retained in the final item bank of each scale. Differential item functioning (DIF) determined if subgroups of burn survivors at the same ability level demonstrated different probabilities of responses. CAT simulations were conducted to estimate the mean score of each scale. The simulated CAT score and full item bank scores were compared based upon the score range, ceiling and floor effects, and marginal reliabilities. Results The child mean age was 3.0 + 1.5 years (n = 498), with primarily mothers (83%) responding. Average burn size and time since burn injury were 4.2% TBSA and 1.1 years, respectively. Analysis resulted in eight scales: Physical (27 items), Communication and language (20 items), Emotional wellbeing (16 items), Mood (11 items), Anxiety (13 items), Peer acceptance (7 items), Play (8 items), and Peer relations (9 items). Severe DIF by ‘age at survey completion’ was shown in 13 items. Ceiling effects were acceptable at &amp;lt; 14%. A CAT was created with the exception of four of the scales (Peer acceptance, Play, Mood, and Anxiety) due to limited item content in these scales’ full item bank. Marginal reliabilities were credible (0.83 to 0.90). CAT simulations revealed that administering the CAT would save the respondent about 14 minutes compared to administering the full item bank. Conclusions Final scales of the PS-LIBRE1-5 Profile contain 111 items and is a comprehensive measure that captures physical, communication and language, psychological, and social functioning of preschool burn survivors in the community. Applicability of Research to Practice This work lays the groundwork for developing a set of age-based CATs, adding to prior adult LIBRE project. The CAT aims to detect the progress of a child during burn recovery for use in clinical practice.

Effect of Race and Geography on Patient- and Parent-Reported Quality of Life for Children With Congenital Upper Limb Differences

Patient beliefs about health and disability are shaped by many social factors and are a key determinant in their ultimate outcome. We hypothesized that pediatric and parent-reported outcome measures regarding a child's congenital upper limb difference will be affected by geographic location, parent education, sex, ethnicity, race, age, and presence of additional medical comorbidities. Patients enrolled in the multicenter Congenital Upper Limb Difference registry were included. Age, sex, race, ethnicity, medical comorbidities, highest level of parental education, area deprivation index, and geographic region were recorded. Patient-Reported Outcomes Measurement Information System (PROMIS) in the pediatric and parent-reported domains of upper extremity, anxiety, pain interference, peer relationships, and depressive symptoms were collected. The only difference between geographic regions in the United States in pediatric and parent-reported PROMIS was that parents in the Midwest reported higher upper extremity function scores in children with upper limb differences than the West. Black patients demonstrated higher scores in parent and child-reported domains of depression, pain, and anxiety, and lower scores in upper extremity function than White and Asian peers. Additionally, children with medical comorbidities also demonstrated worse outcomes in multiple PROMIS domains. There was no difference in scores based on sex, parent education, and ethnicity. In children with congenital upper limb differences, race and additional medical comorbidities have an impact on patient- and parent-reported PROMIS outcome measures in multiple domains, with Black children and those with additional medical comorbidities scoring lower than their peers. As we strive to develop a health care system that provides equitable care to all patients, providers who care for children with upper limb differences should be aware that race and additional medical comorbidities can negatively affect patient- and parent-reported PROMIS outcome measures.

Wound closure with a mesh and liquid tissue adhesive (Dermabond Prineo) system in pediatric spine surgery: a prospective single-center cohort study incorporating parent-reported outcome measures.

Wound healing can be challenging in children undergoing spine surgery for neurological conditions due to a high risk of cerebrospinal fluid (CSF) leakage and wound infection. In adults, use of the Dermabond Prineo (DP) skin closure system, which consists of both tissue adhesive glue and a self-adhesive mesh, for wound closure of medium-length surgical incisions has been reported. The aim of this study was to investigate the efficiency and cosmetic outcome of DP for wound closure in extra- and intradural pediatric neurological spine surgery. In this prospective cohort study, 47 children underwent 50 spine procedures using DP for wound closure between 2018 and 2022 at a single institution. Patient demographic and surgical data were collected. The primary outcome was revision surgery for wound healing disorders, while secondary outcomes were infections, minor wound healing disorders, and both physician and parental satisfaction (parent-reported outcome measures [PROMs]) at last follow-up. Among 50 spinal (45 intra- and 5 extradural) interventions, 1 patient (2%) underwent revision surgery for a cutaneous CSF fistula and pseudomeningocele. Minor wound healing disorders occurred after 16 surgeries, which did not require surgical wound revision and resolved completely. No allergic reactions to DP or surgical site infections within 30 days were observed. The parents and the medical team described wound care as significantly facilitated since wound dressing changes were not needed. Three families (6.4%) encountered difficulties in wound care, and 46 (97.9%) were satisfied with DP. The cosmetic outcome based on PROMs was excellent, with a mean score of 8 (IQR 2) on a scale from 1 to 10. At long-term follow-up, a mean of 11.3 ± 10.7 months after surgery, physicians rated the cosmetic outcome on the visual analog scale (median score 9, IQR 1) and Hollander scale (median score 6, IQR 1). The outcomes were similar among the different pathologies and age groups and did not differ in patients with and without syndromic malformations. The application of DP is simple, enables good patient comfort, facilitates both professional and parental wound care, and leads to excellent cosmetic results. DP possibly aids in the reduction of postoperative CSF leakage and infections after pediatric neurological spine surgery.

Effects of the Big Brothers Big Sisters of America Community-Based Mentoring Program on social-emotional, behavioral, and academic outcomes of participating youth: A randomized controlled trial

This research is a randomized controlled trial of effects of the Big Brothers Big Sisters of America (BBBSA) Community-Based Mentoring Program on the social-emotional, behavioral, and academic outcomes of participating youth over a 13-month period. Seven hundred sixty-four youth between 9 and 14 years old were enrolled in the study through two BBBSA agencies that served predominantly urban areas on the West Coast. Each participating youth was randomly assigned either to be immediately eligible for being matched with a mentor (treatment group; n = 379) or to remain on the program waitlist for 13 months (control group; n = 385). Analyses for the present study are based on 654 youth for whom outcome data were able to be obtained at a 13-month follow-up (87.0% of the treatment group and 84.2% of the control group). Outcome measures were completed by youth (depressive symptoms, prosocial behavior, social acceptance, parent–child relationship quality, misconduct, self-perception of academic ability, skipping school, and academic performance) and primary caregivers (Strengths and Difficulties Questionnaire [SDQ]) at baseline and at the 13-month follow-up. Composite indices reflecting the average of youth- and/or parent-reported outcome measures were also examined. Findings indicated effects of mentoring (i.e., being offered a mentor) that reached statistical significance, all favoring the treatment group, for youth-reported depressive symptoms, parent-reported emotional symptoms, peer problems, conduct problems and the total difficulties score on the SDQ, and the parent-report and combined youth- and parent-report composite indices at the 13-month assessment (Cohen’s d ranging from 0.138 to 0.253).

Patient- and parent-reported outcome measures of developmental adaptive abilities in visually impaired children: The Visual Impairment Developmental Autonomy (VIDA) scale

In the pediatric context, parents’ and patients’ engagement in the care process is strongly recommended and could be pursued using patient-reported outcome measures (PROMs), which therefore become useful for planning and monitoring treatments. Nevertheless, few data are available from families of children with neurodevelopmental disorders such as visual impairment (VI). The Visual Impairment Developmental Autonomy (VIDA) project aims to develop and validate a patient- and parent-reported tool to measure the most relevant aspects concerning everyday adaptive abilities in children and adolescents with visual impairment: the VIDA scale. The present paper illustrates the Delphi process of item generation engaging parents and patients and presents a protocol for the validation of this new co-designed tool in an Italian visually impaired pediatric population. Twenty-three families and five adolescents provided a list of 192 items and assessed their relevance. Items were categorized in 5 areas of adaptive abilities (i.e., table manners, clothing, personal hygiene, orientation and mobility, and socio-affectivity) and into three age ranges based on the patient’s age. The final 102-item Vida Scale will be administered to a minimum of 300 visually impaired children together with measures of quality of life and child adjustment to investigate its psychometric properties.

39-OR: Improved Psychosocial and Quality of Life Measures in Children and Youth with Type 1 Diabetes Initiating Control-IQ Technology

Objective: To determine perceptions of the impact of Control-IQ™ technology (CIQ) on psychosocial functioning and quality of life (QoL) in children/ youth with T1D and their parents, using patient and parent reported outcome measures (PROMs) . Research Design and Methods: Single-center prospective real-world study of 6-18 year olds with T1D using the t:slim X2™ insulin pump, who were initiating CIQ. Youth (≥ 8years) and parents completed validated PROMs at baseline and end of study (16 weeks) . INSPIRE (INsulin delivery Systems: Perceptions, Ideas, Reflections and Expectations) end of study scores were the primary outcome with a score &amp;gt; 60 considered to indicate a perceived benefit of CIQ on psychosocial functioning and QoL. Glycemic control measures were recorded at baseline and every 4 weeks. Results: In total, 65 children/youth with T1D participated; 50.8% female, median (IQR) age 13.9 (11.1, 15.7) years and T1D duration 6.2 (3.1, 8.4) years. INSPIRE end of study mean (SD) scores were 73.0 (11.6) for youth (n=49) and 70.7 (12.2) for parents (n=45) , both significantly &amp;gt; 60 (p &amp;lt; 0.001) . Diabetes Impact and Devices Satisfaction - Device Satisfaction subscale score increased by a mean (95% CI) of 0.43 (0.11, 0.74) in youth (p = 0.01) and 0.58 (0.31, 0.85) in parents (p&amp;lt;0.001) . WHO-5 well-being index median (IQR) score increased from 60 (48, 76) to 72 (60, 80) (p=0.25) in youth, and from 68 (55, 76) to 76 (68, 80) (p=0.002) in parents. Hypoglycemia Fear Survey mean (SD) score improved from 1.7 (0.6) to 1.4 (0.5) (p=0.01) in youth and from 2.0 (0.7) to 1.7 (0.6) (p&amp;lt;0.001) in parents. Time in range improved from 50.8% (baseline) to 62.2% (4 weeks) , with mean difference (95% CI) 9.62 (7.01, 12.23) , p&amp;lt;0.001; this was sustained to 16 weeks. Conclusions: Initiation of CIQ in a real-world setting significantly improved measures of psychosocial functioning and QoL in children/youth with T1D and their parents. The change tended to be greater for parents than youth. Disclosure C.S.Zuijdwijk: Other Relationship; Medtronic, Research Support; Tandem Diabetes Care, Inc. J.Courtney: None. N.Mitsakakis: None. L.Hayawi: None. S.E.Lawrence: Advisory Panel; Pfizer Inc., Research Support; Tandem Diabetes Care, Inc. Funding Tandem Diabetes Care

Development of a Parent-Reported Outcome Measure for Febrile Infants ≤60 Days Old.

We aimed to develop a parent-reported outcome measure for febrile infants 60 days or younger evaluated in the emergency department. We conducted a 3-part study: (1) individual, semistructured interviews with parents of febrile infants 60 days or younger to generate potential items for the measure; (2) expert review with pediatric emergency medicine physicians and member checking with parents, who rated each item's clarity and relevance using 4-point scales; and (3) cognitive interviews with a new sample of parents, who gave feedback and rated the measure's ease of use on a 4-point scale. The measure was iteratively revised during each part of the development process. In part 1, we interviewed 24 parents of 21 infants. Interviews revealed several themes: parents' experiences with medical care, communication, and decision making; parents' emotions, particularly worry, fear, and stress; the infant's outcomes valued by parents; and the impact of the infant's illness on the family. From these themes, we identified 22 potential items for inclusion in the measure. In part 2, 10 items were revised for clarity based on feedback from physicians and parents, primarily under the domains of parents' emotions and the infant's outcomes. In part 3, we further revised the measure for clarity and added an item. The final measure included 23 items and was rated as excellent in its ease of use. The 23-item parent-reported outcome measure includes the experiences and outcomes important to parents. Further studies are needed to evaluate the measure's psychometric properties.

The sensory-reactivity PROM set: identification of a parent reported outcome measure set for autism spectrum disorder

BackgroundMost children with autism spectrum disorder (ASD) suffer from aberrant responses to sensory stimuli that significantly impact the quality of life. To develop sensory interventions, individually tailored outcome measures are crucially needed for the domain of sensory reactivity problems. Here, we describe the identification of relevant sensory themes according to caregivers of children with ASD according to the guidelines for developing a (parent proxy) patient-reported outcome measure set. Subsequently, we identify parallels between these themes and a well-validated and supported PROMIS® portal to facilitate implementation. Interviews with clinicians and focus groups and interviews with parents of children with ASD were used in the initial phase for concept elicitation. Codes and themes were generated by qualitative thematic data analysis on the transcripts and cognitive interviews with different parents were used for revisions. The resulting themes were compared to existing generic PROMIS-item banks and other existing questionnaires.ResultsA total of 11 parent-reported outcomes were identified that could be either classified as directly or indirectly related to sensory reactivity. Directly related themes comprised of: (1) sensory stimulation tolerance and (2) sensitivity to sensory stimuli. Indirectly related themes were: (3) irritable behavior (4) anxiety problems (5) mood problems (6) sleep problems (7) fatigue (8) physical complaints (9) daily functioning and participation (10) routines, structure and dealing with change and (11) problems in social interaction and communication. Seven out of 11 themes could be measured with generic PROMIS item banks. The four remaining outcomes (sensory stimulation tolerance; irritable behaviour; routines, structure and dealing with change; and sensitivity to sensory stimuli) were found suitable to be inventoried by existing PROMs.ConclusionThe majority of parent-reported problems seemed related to indirect consequences of sensory reactivity, which are suitable to be measured with generic item banks. In sum, we identified a sensory-reactivity PROM (parent-proxy) set consisting of PROMIS® item banks and additional domains that together form a comprehensive and readily available outcome set for sensory reactivity problems in children with ASD.

A randomized controlled trial of intranasal oxytocin in Phelan-McDermid syndrome

BackgroundPhelan-McDermid syndrome (PMS) is a rare neurodevelopmental disorder caused by haploinsufficiency of the SHANK3 gene and characterized by global developmental delays, deficits in speech and motor function, and autism spectrum disorder (ASD). Monogenic causes of ASD such as PMS are well suited to investigations with novel therapeutics, as interventions can be targeted based on established genetic etiology. While preclinical studies have demonstrated that the neuropeptide oxytocin can reverse electrophysiological, attentional, and social recognition memory deficits in Shank3-deficient rats, there have been no trials in individuals with PMS. The purpose of this study is to assess the efficacy and safety of intranasal oxytocin as a treatment for the core symptoms of ASD in a cohort of children with PMS.MethodsEighteen children aged 5–17 with PMS were enrolled. Participants were randomized to receive intranasal oxytocin or placebo (intranasal saline) and underwent treatment during a 12-week double-blind, parallel group phase, followed by a 12-week open-label extension phase during which all participants received oxytocin. Efficacy was assessed using the primary outcome of the Aberrant Behavior Checklist-Social Withdrawal (ABC-SW) subscale as well as a number of secondary outcome measures related to the core symptoms of ASD. Safety was monitored throughout the study period.ResultsThere was no statistically significant improvement with oxytocin as compared to placebo on the ABC-SW (Mann–Whitney U = 50, p = 0.055), or on any secondary outcome measures, during either the double-blind or open-label phases. Oxytocin was generally well tolerated, and there were no serious adverse events.LimitationsThe small sample size, potential challenges with drug administration, and expectancy bias due to relying on parent reported outcome measures may all contribute to limitations in interpreting results.ConclusionOur results suggest that intranasal oxytocin is not efficacious in improving the core symptoms of ASD in children with PMS.Trial registration NCT02710084.

Backyard benefits? A cross-sectional study of yard size and greenness and children\u2019s physical activity and outdoor play

BackgroundThe home environment is the most important location in young children’s lives, yet few studies have examined the relationship between the outdoor home environment and child physical activity levels, and even fewer have used objectively measured exposures and outcomes. This study examined relationships between objectively assessed home yard size and greenness, and child physical activity and outdoor play.MethodsData were drawn from the HealthNuts study, a longitudinal study of 5276 children in Melbourne, Australia. We used cross-sectional data from a sample at Wave 3 (2013–2016) when participants were aged 6 years (n = 1648). A sub-sample of 391 children had valid accelerometer data collected from Tri-axial GENEActive accelerometers worn on their non-dominant wrist for 8 consecutive days. Yard area and greenness were calculated using geographic information systems. Objective outcome measures were minutes/day in sedentary, light, and moderate-vigorous physical activity (weekday and weekend separately). Parent-reported outcome measures were minutes/day playing outdoors (weekend and weekday combined). Multi-level regression models (adjusted for child’s sex, mother’s age at the birth of child, neighbourhood socioeconomic index, maternal education, and maternal ethnicity) estimated effects of yard size and greenness on physical activity.ResultsData were available on outdoor play for 1648 children and usable accelerometer data for 391. Associations between yard size/greenness and components of physical activity were minimal. For example, during weekdays, yard size was not associated with daily minutes in sedentary behaviour (β: 2.4, 95% CI: − 6.2, 11.0), light physical activity (β: 1.4, 95% CI: − 5.7, 8.5) or MVPA (β: -2.4, 95% CI: − 6.5, 1.7), with similar patterns at weekends. There was no relationship between median annual yard greenness and physical activity or play.ConclusionIn our study of young children residing in higher socio-economic areas of Melbourne yard characteristics did not appear to have a major impact on children’s physical activity. Larger studies with greater variation in yard characteristics and identification of activity location are needed to better understand the importance of home outdoor spaces and guide sustainable city planning.

Parent-Reported PEDI-CAT Mobility and Gross Motor Function in Infants With Cerebral Palsy.

The purpose of this study is to determine the relationship between the Pediatric Evaluation of Disability Index-Computer Adapted Test (PEDI-CAT), a parent-reported outcome measure, and therapist-administered measures of motor function for infants with cerebral palsy (CP) with moderate to severe motor impairments. A prospective, cohort study included 54 infants, ages 6 to 24 months, with CP or high risk of CP, Gross Motor Function Classification System (GMFCS) levels III to V. Measures included the Gross Motor Function Measure (GMFM) and the mobility domain of the PEDI-CAT (PEDI-mob). A significant correlation was found between PEDI-mob and GMFM scores. Significant differences were found in PEDI-mob scores as a function of GMFCS level. The PEDI-mob adds value to motor evaluations of infants with CP. Parents can accurately contribute information about daily motor performance for goal setting and treatment planning. The PEDI-mob offers a practical solution when longer assessments cannot be completed.

The Gait Outcomes Assessment List for Children With Lower Limb Difference (GOAL-LD): Assessment of Reliability and Validity.

The Gait Outcomes Assessment List for children with Lower Limb Difference (GOAL-LD) is a patient and parent-reported outcome measure that incorporates the framework of the International Classification of Functioning, Disability, and Health. This prospective multicenter cohort study evaluates the validity and reliability of the GOAL-LD and the differences between parent and adolescent report. One hundred thirty-seven pediatric patients aged over 5 years attending limb reconstruction clinics at the participating sites were assessed at baseline, and a self-selected cohort also completed an assessment 2 to 6 weeks later. Construct and criterion validity were assessed by comparing GOAL-LD scores with a measure of limb deformity complexity (LLRS-AIM) and the Pediatric Outcomes Data Collection Instrument, using Spearman correlation coefficients. Face and content validity were determined through ratings of item importance. Test-retest reliability was reported as an intraclass correlation coefficient and internal consistency using Cronbach α. Adolescent reports were compared with their parents using paired t tests. The GOAL-LD demonstrated a moderate negative correlation with the LLRS-AIM (r=-0.40, P<0.001) and was able to discriminate between deformity complexity groups as defined by the LLRS-AIM (χ2=11.43, P=0.022). Internal consistency was high across all domains (α≥0.68 to 0.97). Like domains of the Pediatric Outcomes Data Collection Instrument and the GOAL-LD were well correlated. Parents reported a lower total GOAL-LD score when compared with adolescents (mean difference 3.04; SE 1.06; 95% confidence interval, 0.92-5.16; P<0.01); however this difference was only significant for body image and self-esteem (Domain F) and gait appearance (Domain D). Test-retest reliability remained high over the study period (intraclass correlation coefficient 0.85; SE 0.03; 95% confidence interval, 0.77-0.91). The GOAL-LD is a valid and reliable self and parent-reported outcome measure for children with lower limb difference. Parents report a lower level of function and attribute a higher importance to items when compared with their children. The GOAL-LD helps to communicate parent and child perspectives on their function and priorities and therefore has the capacity to facilitate family centered treatment planning and care. Level II-diagnostic. Prospective cross-sectional and a longitudinal cohort design.

Development of the gait outcomes assessment list for lower-limb differences (GOAL-LD) questionnaire: a child and parent reported outcome measure

BackgroundTo develop a priority-based patient/parent reported outcome measure for children with lower-limb differences (LD) by adapting the Gait Outcomes Assessment List (GOAL) questionnaire.MethodsGuided by a conceptual framework of patient priorities, the GOAL questionnaire was iteratively modified and its sensibility evaluated by field-testing it on children with LD, and their parents. Cognitive interviews were conducted with a subgroup of these children, and an e-survey administered to a multidisciplinary group of health care professionals with expertise in paediatric LD. Findings were integrated to create the final version of the GOAL-LD.ResultsTwenty-five children (9–18 years), 20 parents, and 31 healthcare professionals evaluated the content and sensibility of the GOAL, with an emphasis on the relevance and importance of the items to patients’ health related quality of life (HRQL). This resulted in the retention of 26 of the original 50 items, elimination of 12, modification of 12, and addition of seven new items. The new 45-item GOAL-LD questionnaire was shown to be sensible, and its content deemed important.ConclusionsThe GOAL-LD questionnaire has a high level of face and content validity, and sensibility. It comprehensively captures the HRQL goals and outcomes that matter to children with LD and their parents. Following further psychometric evaluation, the GOAL-LD may serve as a much needed patient and parent reported outcome measure for this population.

Implementation of the norwegian \u2018Starting right\u2019 child health service innovation: implementation adjustments, adoption, and acceptability

BackgroundAn increased and/or stable proportion of the child and adolescent population reports symptoms of impaired health, and the symptoms can be identified early. Therefore, structured child- and parent-reported outcome measures need to be implemented in child and school health services for decision support and identification of children at risk. We aimed to (a) qualitatively examine adjustments of active implementation from the pilot implementation of the Norwegian ‘Starting Right’ health service innovation including an online child health assessment tool and practical routines, and (b) measure practitioners´ adoption and parental acceptability.MethodsWe used a mixed-methods design to qualitatively examine adjustments from working notes and meeting memoranda, and quantitatively assess adoption and acceptability from user rates provided by the systems log. Twenty-one child and school health nurses (CSHNs) from two child health centers participated in the implementation pilot of online health assessments in children aged 2-, 4- and 6-year. We used a deductive and narrative analysis approach using Fixsen et al.´s core implementation components to code and sort adjustments.ResultsCore implementation components were adjusted throughout the pilot implementation. Researchers´ increased their availability in reciprocity with staff evaluation to integrate active implementation adjustments. We launched a project for improved data systems integration. The overall CSHNs adoption rate was satisfactory and higher in center A, where a medical secretary supported the nurses through the entire pilot phase, than in center B (96 vs. 55 %). Parental acceptability rate was overall high (77 %) with increased rates among parents of 6-year-old children (98 %) compared with younger ones (78–85 %), and in cases where both parents received the questionnaires.ConclusionsThe ‘Starting Right’ health service innovation implementation was actively adjusted by integration of core implementation components mainly based on staff evaluation. The CSHNs adopted the innovation which was also acceptable to parents.

A study protocol for a randomised controlled trial to evaluate the effectiveness of a dog-facilitated physical activity minimal intervention on young children\u2019s physical activity, health and development: the PLAYCE PAWS trial

BackgroundPet ownership brings many health benefits to individuals. In children developmental benefits can extend to improved self-esteem, better social competence and decreased loneliness. The majority of households with children own a dog, however only a small proportion of children gain the benefits of dog ownership through dog walking and play. There are few intervention studies investigating the impact of dog-facilitated physical activity in children. The PLAYCE PAWS study aims to test a minimal-contact intervention through the use of mobile health (“mhealth”) strategies, i.e. text (SMS) messages, to parents to encourage their children to walk and play with their dog more, and evaluate the impact on children’s overall physical activity and development.Methods/designThe PLAYCE PAWS intervention study will target parents in dog-owning families with children aged 5 to 8 years in Perth, Western Australia. Approximately 150 dog-owning parents and children will be randomly allocated into either one of two intervention groups or a ‘usual care’ control group. The first intervention group will receive SMS messages over 4 weeks to encourage and prompt parents to undertake dog walking and dog play with their child. The second intervention group will receive the same text messages, plus a dog pedometer and personalised ‘dog steps’ diary for their child to complete. Parent-reported outcome measures include changes in children’s dog walking and play, overall physical activity, socio-emotional development, self-regulation, self-esteem, empathy, and level of attachment to their dog.DiscussionThe PLAYCE PAWS study appears to be the first to examine the effectiveness of a low-cost, mhealth intervention for increasing young children’s physical activity through dog walking and play. Given the high prevalence of dogs as family pets, this study presents a valuable opportunity to investigate if mHealth interventions encourage children to walk and play with their dog more, and if there are any associated impact on children’s overall physical activity and socio-emotional well-being. If effective, a larger trial or program could be implemented at low-cost and with wide reach in the community.Trial registrationANZCTR, ACTRN12620000288921. Registered 4th March 2020 - Retrospectively registered.