Health-related Quality Of Life In Children Research Articles

An Investigation of Inter-Rater and Intra-Proxy Agreement in Measuring Quality of Life of Children in the Community Using the EQ-5D-Y-3L.

Self-reporting of health-related quality of life (HRQoL) in children is not always feasible. To date, proxy perspectives (Proxy versions 1 and 2) using the EQ-5D-Y-3L have not been explored for its impact on agreement with child self-report. Proxy version 1 requires the proxy to consider their own view of the child's HRQoL (proxy-proxy), while with Proxy version 2, the proxy is asked to respond as they believe their child would self-report their HRQoL (proxy-child). This study compared the inter-rater and intra-proxy agreement (overall and dimension level) using the EQ-5D-Y-3L self, proxy-proxy, and proxy-child reports. A community-based sample of child (aged 6-12 years) and parent dyads were invited to participate in a semi-structured interview. The child self-completed the EQ-5D-Y-3L independently of the parent who completed the EQ-5D-Y-3L from proxy-proxy and proxy-child perspectives. Agreement was determined using Concordance Correlation Coefficients (CCCs) for the overall (preference-weighted) HRQoL, while agreement at the dimension level was evaluated using Gwet's agreement coefficient (AC1). To assess the differences between the self and the two proxy reports, the Wilcoxon matched-pair signed-rank test was used. This study involved 85 child-parent dyads. The agreement between self and proxy overall HRQoL was low (fair) with both proxy-proxy (CCC=0.28) and proxy-child (CCC=0.26) reports. The largest discrepancy in the child-proxy agreement at dimension level with both the proxy versions was observed for 'feeling worried, sad or unhappy'. Within this dimension, the proxy-child perspective resulted in a stronger agreement (AC1=0.7, good) with child self-report compared with the traditional proxy-proxy perspective (AC1=0.58, moderate). Although the preference-weighted HRQoL was consistent across both the proxy perspectives, a significant difference was observed in the EQ VAS scores (p=0.02). This study demonstrates that choice of proxy perspective may have an impact on the problems reported on HRQoL dimensions and EQ VAS scores. However, in this community-based sample of generally healthy children, no significant difference was observed in the inter-rater agreement for child-self and proxy preference-weighted EQ-5D-Y-3L values based on proxy perspectives. While this suggests that preference-weighted data are not sensitive to the choice of perspective, these findings may differ for different HRQoL instruments and for alternative value sets with different properties.

Healthy diets positively associated with health-related quality of life in children and adolescents from low socioeconomic areas: Findings from the Greek Food Aid Program, DIATROFI

ObjectivesTo assess the relationship of diet with health-related quality of life (HRQoL) in vulnerable children and adolescents. MethodsData included 6583 children and adolescents (aged 3–18 years old) from the Greek Food-Aid DIATROFI Program in the 2015 to 2016 and 2017 to 2018 school years. HRQoL was measured with the Pediatric Quality of Life Inventory questionnaire and diet with food frequency questionnaires. The healthy plant-based diet index (hPDI), animal score, and dietary patterns were investigated. ResultsThe hPDI and animal score were associated with good HRQoL (odds ratio [OR] [95% confidence interval], 10-unit increase: ORhPDI = 1.28 [1.05, 1.57], ORanimal = 1.51 [1.14, 2.00]) and physical (ORanimal = 1.62 [1.23, 2.13]), emotional (ORhPDI = 1.30 [1.07, 1.58], ORanimal = 1.41 [1.08, 1.85]) and school function (ORhPDI = 1.32 [1.09, 1.59], ORanimal = 1.46 [1.12, 1.89]). Dietary patterns of fruits, raw vegetables, and cheese were associated with good HRQoL (OR of 1-unit increase: 1.22 [1.13, 1.32]), and physical OR = 1.18 [1.09, 1.27]) and emotional function (OR = 1.09 [1.02, 1.18]). Starchy foods and sweetened beverages were associated with poor HRQoL (OR = 0.75 [0.63, 0.90]), and emotional (OR = 0.80 [0.68, 0.95]) and school function (OR = 0.72 [0.61, 0.85]). ConclusionHealthy diets and dietary patterns were positively associated with the HRQoL of vulnerable children and adolescents, which may offer opportunities for prevention.

Health-related quality of life of Indonesian children with hemophilia.

Measuring health-related quality of life (HRQoL) in haemophilia patients provides a comprehensive patient's functional and the impact of the disease and its treatment. This study aimed to assess the HRQoL of haemophilia children and related factors. We conducted a cross-sectional study from 6 April 2019 to 29 April 2019. We recruited children aged 8-16years old who were members of the Yogyakarta Province Branch, Indonesian Haemophilia Society. Children filled in the Indonesian Haemo-QoL short-version questionnaire that consists of nine dimensions, and the score ranges from 0 to 100, in which a higher score shows a higher impartment. Forty-four children participated in this study. The mean age was 11.99+2.49. The median age of the first bleeding episode was 5years, and the median frequency of spontaneous bleeding during the previous year was 13.5. All children received antihemophilic factor (AHF) on-demand; most (81.8%) received AHF in the advanced symptoms. The mean total score of HRQoL was 42.7±15.5, with the highest impairment in the family dimension, with a mean score of 68.0±22.3. The severity of haemophilia was significantly correlated with the total score of HRQoL and dimensions of physical health, feeling, view, and family (effect size ranges from 0.30 to 0.42). The frequency of bleeding was significantly related to the total score of HRQoL and the feeling dimension (effect size of 0.47 and 0.34). The HRQoL of our study was lower than in countries where AHF prophylactic is provided. More severe haemophilia and more frequent bleeding significantly lower HRQoL.

The RETRIEVE Checklist for Studies Reporting the Elicitation of Stated Preferences for Child Health-Related Quality of Life.

Recent systematic reviews show varying methods for eliciting, modelling, and reporting preference-based values for child health-related quality-of-life (HRQoL) outcomes, thus producing valuesets with different characteristics. Reporting in many of the reviewed studies was found to be incomplete and inconsistent, making them difficult to assess. Checklists can help to improve standards of reporting; however, existing checklists do not address methodological issues for valuing child HRQoL.Existing checklists also focus on reporting methods and processes used in developing HRQoL values, with less focus on reporting of the values' key characteristics and properties. We aimed to develop a checklist for studies generating values for child HRQoL, including for disease-specific states and value sets for generic child HRQoL instruments. A conceptual model provided a structure for grouping items into five modules. Potential items were sourced from an adult HRQoL checklist review, with additional items specific to children developed using recent reviews. Checklist items were reduced by eliminating duplication and overlap, then refined for relevance and clarity via an iterative process. Long and short checklist versions were produced for different user needs. The resulting long RETRIEVE contains 83 items, with modules for reporting methods (A-D) and characteristics of values (E), for researchers planning and reporting child health valuation studies. The short RETRIEVE contains 14 items for decision makers or researchers choosing value sets. Applying the RETRIEVE checklists to relevant studies suggests feasibility. RETRIEVE has the potential to improve completeness in the reporting of preference-based values for child HRQOL outcomes and to improve assessment of preference-based value sets.

Long-term clinical outcomes and health-related quality of life in patients with isolated methylmalonic acidemia after liver transplantation: experience from the largest cohort study in China.

Liver transplantation (LT) has been proposed as a viable treatment option for selected methylmalonic acidemia (MMA) patients. However, there are still controversies regarding the therapeutic value of LT for MMA. The systematic assessment of health-related quality of life (HRQoL)-targeted MMA children before and after LT is also undetermined. This study aimed to comprehensively assess the long-term impact of LT on MMA, including multiorgan sequelae and HRQoL in children and families. We retrospectively evaluated 15 isolated MMA patients undergoing LT at our institution between June 2013 and March 2022. Pre- and post-transplant data were compared, including metabolic profiles, neurologic consequences, growth parameters, and HRQoL. To further assess the characteristics of the HRQoL outcomes in MMA, we compared the results with those of children with biliary atresia (BA). All patients had early onset MMA, and underwent LT at a mean age of 4.3years. During 1.3-8.2years of follow-up, the patient and graft survival rates were 100%. Metabolic stability was achieved in all patients with liberalized dietary protein intake. There was a significant overall improvement in height Z scores (P = 0.0047), and some preexisting neurological complications remained stable or even improved after LT. On the Pediatric Quality of Life Inventory (PedsQL™) generic core scales, the mean total, physical health, and psychosocial health scores improved significantly posttransplant (P < 0.05). In the family impact module, higher mean scores were noted for all subscales post-LT, especially family function and daily activities (P < 0.01). However, the total scores on the generic core scales and transplant module were significantly lower (Cohen's d = 0.57-1.17) when compared with BA recipients. In particular, social and school functioning (Cohen's d = 0.86-1.76), treatment anxiety, and communication (Cohen's d = 0.99-1.81) were far behind, with a large effect size. This large single-center study of the mainland of China showed an overall favorable impact of LT on isolated MMA in terms of long-term survival, metabolic control, and HRQoL in children and families. The potential for persistent neurocognitive impairment and inherent metabolic fragility requires long-term special care. Video Abstract (MP4 153780 KB).

Effect of Executive Function on Health-Related Quality of Life in Children with Chronic Kidney Disease.

As the highest function in the brain that regulates our daily activity, executive dysfunction might affect someone's health-related quality of life (HRQoL), especially in those with chronic diseases, including chronic kidney disease (CKD). Neurocognitive functions, including intelligence quotient (IQ) and executive function can be affected through various mechanisms in CKD. However, there was still no specific study regarding how IQ and executive function might affect HRQoL in children with CKD. To assess Executive Function's impact on HRQoL and to find association between treatment modalities and CKD stages with HRQoL in children with CKD. A cross sectional study was conducted at Pediatric Nephrology Clinic at Hasan Sadikin General Hospital, Bandung, Indonesia from September 2022 to April 2023. We included 38 children whose age range were 6-16 years 11 months old with CKD stage III - V. Assessment tools used were: BRIEF questionnaire for executive function; WISC III tool for IQ; PedsQLTM questionnaire generic module for HRQoL. Data was analyzed using SPSS ver. 26.0. Total number of samples was 38. Complete examinations were done on 30 patients. Eight other patients did not undergo the IQ test. There was a negative correlation between executive function components scores (GEC, BRI, MI) with HRQoL scores on parents' proxy in all domains. We found no correlation between HRQoL and IQ scores, but we found a correlation between IQ and CKD stage. There was a significant difference in HRQoL from the children's perspective among the three modalities; children who underwent conservative treatment were having the best HRQoL scores. Interventions to improve executive function of children with CKD should be done to improve their HRQoL in the future. Early diagnosis and treatment of CKD should be done at the earliest to improve neurocognitive function and HRQoL.

A153: The Relationships Between Physical Activity and Health-Related Quality of Life in Parent-Child Dyads with ASD

Purpose: Researchers have found that the core and combined symptoms of autism spectrum disorder (ASD) negatively influence the health-related quality of life (HRQoL) of autistic children and their parents. Although it is supported that participation in physical activity (PA) can enhance the HRQoL and well-being in general, few studies have addressed the relationship between HRQoL with PA in the population with ASD. This study examines how much leisure-time physical activity participation is correlated to HRQoL in parent-child dyads with ASD. Method: Participants were recruited from two Rehabilitation Centers for neurodevelopmental disability children. Eighty-five ASD children and their parents participated in this study. HRQoL for ASD children and their parents was measured with the World Health Organization Quality of Life short form (WHOQoL-26) and the Pediatric Quality of Life Inventory 4.0 (PedsQL 4.0, parent-report version) respectively, while leisure-time PA participation of both children and parents was assessed using the Godin Leisure-Time Exercise Questionnaire (GLTEQ). We first confirmed the psychometrics of the WHOQoL-26 and PedQL 4.0 and then analyzed the relationships between different levels of PA and HRQoL in dyads. Results: Descriptive statistics showed that 30.6% of ASD children (Mage=5.25±1.48; 85.9% boys) and 42.4% of their parents (75.3% between 31-40 years; 73 mothers and 12 fathers) were insufficiently active. For parents of children with ASD, the physical domain of HRQoL had a small positive correlation with light PA (r=0.27, p=0.013) while the psychology domain had a small to moderate positive correlation with moderate (r= 0.23, p=0.04), light (r=0.3, p=0.006), and total (r=0.23, p=0.032) PA. Also, the environment domain of HRQoL had a small positive correlation with moderate (r=0.28, p=0.011) and light (r=0.32, p=0.003) PA. Concerning ASD children, the physical domain of HRQoL had a small to moderate positive correlation with moderate (r= 0.24, p= 0.027) and total (r= 0.32, p= 0.004) PA. Furthermore, there were several interrelationships between parent-child dyads’ PA and parent-child dyads’ HRQoL. ASD children’s social domain in HRQoL was correlated to their parents’ light PA (r=0.25, p=0.021) whereas parents’ environment domain was correlated to their children’s mild PA (r=0.24, p=0.03). Conclusion: There are significant relationships between PA and HRQoL for both children with ASD and their parents. Particularly, the parent-child dyads’ HRQoL may benefit from each other’s leisure-time PA involvement. Due to ASD children’s restricted daily PA norm and their parent’s challenging caregiving environment, the designation of effective PA enhancement strategies may enhance the quality of life in both children with ASD and their parents.

Relationship between body mass index, fundamental movement skills, and quality of life in primary school children

Background: Obesity in children is a serious public health problem, affecting their physical health and quality of life. Fundamental movement skills (FMS) have been suggested to play an important role in reducing the risk of obesity in primary school children and improving their health-related quality of life (HRQoL). Objectives: This study aims to investigate the relationship between body mass index (BMI), FMS, and HRQoL in primary school children. Methods: 193 primary school children (81 boys, 112 girls) aged 6 to 8 years were included in this cross-sectional study. Weight and height, FMS, and HRQoL were measured using BMI percentiles, the Test of Gross Motor Development, Second Edition (TGMD-2) including the object control, locomotor subtests and the total gross motor quotient, and the Pediatric Quality of Life Inventory (PedsQLTM) respectively. Results: There was a negative significant correlation between BMI and gross motor quotient, and between BMI and the locomotor subscale of the TGMD. There was also a negative significant correlation between BMI and the PedsQL scale. There was a positive significant correlation between TGMD and PedsQoL scales. According to linear regression analysis, BMI significantly predicted TGMD gross motor quotient and PedsQL total score. In addition, the TGMD gross motor quotient significantly predicted the total PedsQL score. Conclusion: Mastery of FMS might be an important factor in avoiding obesity and improving HRQoL in primary school children.

Pictures versus words: can we use a pictorial scale to measure child health-related quality of life?

Measuring health-related quality of life (HRQoL) is important because it can serve as an indicator or a predictor of subsequent mortality or morbidity. HRQoL has been shown to be directly related to child growth and development and indirectly related to the healthcare costs of young children. Existing measures of HRQoL in children have heavily relied on traditional questionnaires that use age-suited versions or parent proxy questionnaires. However, both of these methods may present with different types of biases and may misrepresent underlying HRQoL. The current mini reivew will first illustrate these methodological limitations and highlight the potential use of pictorial scales in addition to discussing their suitability for specifically measuring HRQoL as an alternative. We will also synthesize existing recommendations on the development of pictorial scales to provide a protocol as a recommendation to researchers who are aiming to develop an overall HRQoL pictorial scale that is suited for children.

DEVELOPMENT AND VALIDATION OF QUALITY OF LIFE QUESTIONNAIRE FOR PARENT BURDEN OF CHILDREN WITH FOOD ALLERGY

Quality of life (QOL) questionnaires for parents of children with food allergies have been developed in the United States and Europe. However, no original Japanese QOL questionnaire has been developed till date. We aimed to develop an original questionnaire to evaluate the QOL in parents of children with food allergies in Japan. We collected QOL-related questions from parents of children with food allergies aged 0-15 years, and created a primary questionnaire. Responses to the primary questionnaire were obtained from the parents again, and question items were reduced using factor analysis to create a secondary questionnaire comprising eight items. In addition to the secondary questionnaire, responses to the Food Allergy QOL Questionnaire-Parent Form (FAQLQ-PF) Japanese version, Parent reported Health-Related QOL in children and adolescents (KINDL) and Health-related QOL (SF-8) were obtained from parents to assess the validity of the secondary questionnaire. A total of 407 parents completed all questionnaires. The secondary questionnaire scores were positively correlated with those of FAQLQ-PF and weakly negatively correlated with the KINDL and SF-8 mental component summary scores. Parents of children with food allergies with ≥3 culprit foods or severe reactions to daily foods, a history of anaphylaxis, and those carrying adrenaline autoinjectors scored higher and had lower QOL. The developed original questionnaire is a valid QOL questionnaire for Parents of children with food allergies.

Health-Related Quality of Life in Kindergarten Children with Developmental Language Disorder: Child-Mother Agreement.

Language disorders are associated with difficulties in various aspects of life, such as academic and social functioning, resulting in impaired health-related quality of life (HRQoL). Most studies use a parent proxy method to assess HRQoL. Since HRQoL refers to the subjective experience of an individual, it is necessary to assess children's perspectives along with their mothers'. The aim of the current study is to explore HRQoL rating agreement between children and their mothers, since the literature on other conditions suggests that discrepancies seem to reflect their different perspectives. Thus, 53 Greek-speaking children diagnosed with DLD attending kindergarten and their mothers completed, respectively, self-report and parent proxy PedsQLTM questionnaires. Mothers reported significantly better HRQoL than their children with developmental language disorder (DLD) in all HRQoL domains (p < 0.001). Poor agreement was revealed after comparing the scores from both responders, both in abstract domains, such as emotional functioning, as well as in more observable ones, such as physical health (ICC ranged from -0.05 to 0.07). Bland-Altman plots also showed poor agreement on HRQoL. Our results expand on the already known, from other conditions, importance of evaluating children's subjective experience of their HRQoL in kindergarten children with DLD. A multi-informant approach is ideal, and clinicians should prioritize children's view about their lives even when they are kindergarten-age. This approach could inform interventions focusing not only on language skills but also on other areas where it is necessary, depending on the child's subjective experience combined with the maternal perspective.

Understanding UK public views on normative decisions made to value health-related quality of life in children: A qualitative study

Developing methodology for measuring and valuing child health-related quality of life (HRQoL) is a priority for health technology agencies. One aspect of this is normative decisions that are made in child HRQoL valuation. This qualitative study aimed to better understand adult public opinion on the normative questions of whose preferences to elicit (adults, children, or both) and from which perspective (who should be imagined living in impaired health), when valuing child HRQoL. Opinions of the adult UK public (N = 32) were solicited using online semi-structured focus groups, featuring a breadth of age, sex, ethnicities, and responsibility for children under 18 years. Participants were provided with bespoke informational material on health state valuation and were probed for their views. Arguments for and against different positions were discussed. Data was analysed using framework analysis. Participants demonstrated near-to-universal agreement that children should be involved in valuation in some form, yet this should differ depending on age or maturity. There was strong support for approaches combining involvement from children and adults (e.g., their parents), especially for younger children. There was little intuitive support for the ‘taxpayer argument’ for asking taxpaying adults. In the context of greater involvement of children in valuation, most participants supported using an ‘own’ perspective. Most participants thought that valuation study participants should know the exercise is about valuing child health states for ethical reasons. Informed views from the UK public on who should be asked and with what perspective when valuing child HRQoL appear to differ from normative positions previously advocated by some health economists, such as prioritising the preferences of taxpaying adults. In contrast, the results suggest including adults and children in valuation, with the proviso that the children are of an appropriate age and level of maturity, and that an own perspective is used wherever possible.

Parent-Reported Health-Related Quality of Life Outcomes and Impact on Family of Congenital Diaphragmatic Hernia Survivors After Repair: A Cross-Sectional Comparison to Healthy Controls

BackgroundCongenital diaphragmatic hernia (CDH) survivors often experience long-term CDH-associated morbidities, including musculoskeletal, gastrointestinal and respiratory issues. This study evaluates parent-reported health-related quality of life (HRQOL) and family impact of the disease. MethodsElectronic medical records (EMR) were reviewed and phone surveys performed with parents of CDH survivors who underwent repair at our institution from 2010 to 2019. They completed the following Pediatric Quality of Life Inventory™ (PedsQL™) questionnaires: Generic Core Scales 4.0 (parent-proxy report) and Family Impact (FI) Module 2.0. Age-matched and gender-matched healthy controls from an existing database were used for comparison. Subgroup analysis of CDH patients alone was also performed. Appropriate statistical analysis was used with p < 0.05 significance. Data are reported as median (range). Ethical approval was obtained. ResultsOf 76 CDH survivors, 45 parents (59 %) consented for study participation. Patients and controls were aged 6 (3–12) years; 51 % were male. In Core Scales, there were significant differences between the groups in the overall scores (p = 0.003) and the psychosocial health sub-scores (p = 0.004), but no difference in the physical health domain scores. In the Family Impact Module, there were no significant differences between the groups in overall scores, in parent HRQOL and in family functioning sub-scores. However, CDH patients with learning disabilities had significantly worse Family Impact Module overall scores compared to CDH patients without learning disabilities (p = 0.04). ConclusionThe overall HRQOL in children with CDH survivors is worse compared to controls, but impact on family appears similar. We highlight the need for long-term follow-up of CDH patients, especially those with learning disabilities. Healthcare providers should formulate tailored care plans to address patient and caregiver needs. Type of studyProspective Study. Level of evidenceLevel III.

Health-Related Quality of Life after Pediatric Traumatic Brain Injury: A Quantitative Comparison between Children’s and Parents’ Perspectives of the QOLIBRI-KID/ADO Questionnaire

Pediatric health-related quality of life (HRQoL) as a measure of subjective wellbeing and functioning has received increasing attention over the past decade. HRQoL in children and adolescents following pediatric traumatic brain injury (pTBI) has been poorly studied, and performing adequate measurements in this population is challenging. This study compares child/adolescent and parent reports of HRQoL following pTBI using the newly developed Quality of Life after Brain Injury in Children and Adolescents (QOLIBRI-KID/ADO) questionnaire. Three hundred dyads of 8–17-year-old children/adolescents and their parents were included in the study. The parent–child agreement, estimated using intraclass correlation coefficients and Cohen’s κ, displayed poor to moderate concordance. Approximately two-fifths of parents (39.3%) tended to report lower HRQoL for their children/adolescents on the total QOLIBRI-KID/ADO score. At the same time, about one-fifth (21.3%) reported higher HRQoL Total scores for their children/adolescents. The best agreement for parents rating adolescents (aged 13–17 years) was found in terms of the Total score and the Cognition and Self scale scores. To date, parent-reported HRQoL has been the preferred choice in pediatric research after TBI. However, with a parent–child disagreement of approximately 60%, our results highlight the importance of considering self-reports for children/adolescents capable of answering or completing the HRQoL measures.

Post-traumatic stress and health-related quality of life after admission to paediatric intensive care: Longitudinal associations in mother–child dyads

ObjectiveElevated post-traumatic stress symptoms (PTSS) and reduced health-related quality of life (HRQoL) are commonly experienced in both children and their parent's following admission to the paediatric intensive care unit (PICU). Previous research has demonstrated a conflict in the directionality between PTSS and HRQoL in children and their parents. Our study sought to explore the interconnection and transmission between PTSS and HRQoL in children and their mothers following an admission to the PICU. DesignProspective longitudinal design. SettingTwo tertiary care PICUs in Brisbane, Australia. PatientsTwo hundred eighty-two mother–child dyads of children aged 2–16 years admitted to the PICU for >8 h. InterventionsNone. Measurement and main resultsFour waves of dyadic data (n = 282 mother–child dyads) over a 12-month period from the PICU post-traumatic stress study were used. Cross-lagged panel modelling was used to examine the link between maternal-rated PTSS and HRQoL across children and their mothers. Actor–partner interdependence modelling was then used to examine the interconnections between mothers and children over time. In the dyadic model, partner effects were only present from mother to their child (i.e., higher maternal PTSS was predictive of higher child PTSS at subsequent time points). Higher maternal PTSS predicted lower maternal mental HRQoL but not lower child psychosocial HRQoL. Actor effects were also present with lower child psychosocial HRQoL, predicting higher child PTSS at subsequent time points. Findings indicated that a unidirectional transmission process from mother to child may be present after a child's life-threatening illness. ConclusionsThis study provides evidence for a long-term negative influence of maternal PTSS on child PTSS in families who have experienced a paediatric critical illness or injury. This highlights the important role of maternal wellbeing in children's mental health outcomes following PICU admission. Further research needs to explore the temporal and dyadic relationships of PTSS and HRQoL.

Quality of Life in Childhood Immune Thrombocytopenia: Revision of the Kids' ITP Tools (KIT)

Introduction New treatment approaches have emerged for pediatric ITP over the years, potentially impacting the health-related quality of life (HRQoL) of affected individuals. The Kids' ITP Tools (KIT) (Child self-report/Parent proxy-report and Parent impact-report) have gained international attention as valid tools for monitoring HRQoL in children with ITP and the impact on their parents. The questionnaires were recently updated to reflect changes in clinical management. However, it has also become common practice to evaluate the impact of new therapies using domain scales of QOL (i.e., sub-categories), which are not yet available for the KIT. Thus, the primary objective of this study was to establish domain scales for the KIT 2.0 that accurately quantify and identify specific aspects of HRQoL that are impacted in children with ITP and their parents. Methods The study was conducted in two sequential phases. Phase 1 involved ITP experts identifying key conceptual domains for the KIT questionnaires based on their clinical experiences. An online survey (“card sort survey”) was distributed, and experts sorted the current KIT 2.0 questions into domains, with domain headings suggested as well. Phase 2 involved a statistical approach (i.e., exploratory factor analysis (EFA)) to yield models with different number of domains. Data from children with ITP and their parents was utilized to develop the models, and the data was specifically from three previous international multicentre studies: ITP Consortium of North America (ICON1 study), UK Children's Hospital (UK ITP Paediatric Registry), and the Children's Hospital of Eastern Ontario (KIT international validation). Results In Phase 1, 24 experts completed the Child self-report card-sort survey, while 12 experts completed the Parent impact survey. There were two models that were derived for the Child Self-Report, with one having three domain scales and the other with four domain scales. The Parent Impact resulted in only one model that had three domain scales. In Phase 2, exploratory factor analysis (EFA) resulted in three models for each of the three questionnaires (Table 1), with the final models selected from the EFA and based on clinical interpretability. The four-domain model was selected for the Child Self-Report. The model had a higher Tucker Lewis Index (TLI) score and cumulative percentage of variance compared to the three-domain model. Though the cumulative percentage of variance was higher for the six-domain model, the model had two domains that each contained only two questions, which is not ideal for face validity. The final domain headings for the Child self-report were derived from the expert card sort survey: ITP Uncertainty; General symptoms; ITP challenges/disruptions; and Loss of control. The four-domain model was also selected for the Parent proxy-report on the basis of the same factors as the Child self-report. Likewise, the four-factor model had higher TLI score and relatively lower root mean square error of approximation (RMSEA) compared to the three-factor. The six-factor model had an ideal RMSEA, but contained one domain that had only two questions. The final domain headings are the same as the Child self-report as the context of the questions are the same. The four-domain model was selected for the Parent impact-report. Though both the four- and six-factor models contained two domains with only two questions each, the two-factor model was not ideal clinically as the questions within each of the two domains were not all similar. The headings were also derived from the expert card-sort: ITP Uncertainty; Treatment; Functional well-being; and Activities. Conclusion The revised KIT 2.0 aims to comprehensively summarize health-related quality of life in pediatric patients with ITP and their parents. The introduction of a domain structure derived from expert- and patient-data to the KIT will enable identification of specific aspects of QOL that are affected, which is imperative given the introduction of novel pharmaceutical agents in ITP treatment.

Child characteristics associated with child quality of life and parenting stress in Angelman syndrome.

Angelman syndrome (AS) is a rare neurodevelopmental disorder characterised by severe intellectual disability, movement disorder, epilepsy, sleeping problems, and behavioural issues. Little is known on child health-related quality of life (HRQoL) in AS. AS family studies have reported elevated parenting stress and a high impact of the child's syndrome on the parent. It is unclear which factors influence child HRQoL and parenting stress/impact in AS. We collected data prospectively through standardised clinical assessments of children with AS at the ENCORE Expertise centre for Angelman Syndrome at the Erasmus MC Sophia Children's Hospital. A linear regression analysis was conducted for the following outcome variables: (1) child HRQoL (Infant and Toddler Quality of Life Questionnaire); (2) the impact of the child's syndrome on the parent (Infant and Toddler Quality of Life Questionnaire); and (3) parenting stress (Parenting Stress Index). Predictor variables were child genotype, epilepsy, sleeping problems (Sleep Disturbance Scale for Children), cognitive developmental level (Bayley Cognition Scale), autistic features (Autism Diagnostic Observation Schedule) and emotional/behavioural problems (Child Behaviour Checklist). Covariates were sex, age and socio-economic status. The study sample consisted of 73 children with AS, mean age=9.1years, range=2-18years. Emotional/behavioural problems were the strongest significant predictor of lowered child HRQoL. Internalising problems were driving this effect. In addition, having the deletion genotype and higher age was related to lower child HRQoL. Sleeping problems were related to a higher impact of the child's syndrome on the parent. Finally, emotional/behavioural problems were associated with higher parenting stress. Cognitive developmental level, autistic features and epilepsy were not a significant predictor of child HRQoL and parenting stress/impact. These results suggest that interventions aimed at increasing child HRQoL and decreasing parenting stress/impact in AS should focus on child emotional/behavioural problems and sleeping problems, using a family-centred approach.

Fighting to Breathe and Fighting for Health-Related Quality of Life: Measuring the Impact of Being Dependent on Technology for Breathing on the Child and Their Caregiver.

Medical advancement has enabled children to survive congenital airway anomalies, rare diseases and critical illnesses with medical technology including tracheostomies and long-term ventilation to support breathing. This study aimed to assess (1) the validity of the EQ-TIPS and EQ-5D-Y-3L in children dependent on technology and (2) the impact of caring for these children on the EQ-5D-5L and CarerQoL. Caregivers of children aged 1 month to 18 years completed the EQ-TIPS or EQ-5D-Y-3L, Pediatric Quality of Life Inventory (PedsQL) and Paediatric Tracheostomy Health Status Instrument(PTHSI) to reflect the child's health. In addition, caregivers self-completed the EQ-5D-5L and CarerQoL. Reports of problems on EQ dimensions were compared across age groups with the Fisher's exact test. Spearman and Pearson's correlation coefficients and Kruskal-Wallis H-test were used to explore the association between caregiver and child scores, concurrent validity, and known-group validity of the EQ-TIPS and EQ-5D-Y-3L. Responses from 144 caregivers were collected, 66 for children aged 1 month to 4 years completing EQ-TIPS and 78 for children aged 5-18 years completing EQ-5D-Y-3L. The EQ-TIPS showed a higher report of no problems for social interaction for children aged 1-12 months (p = 0.040) than the older age groups, there were however no differences in the level sum score (LSS) or EQ Visual Analogue Scale scores between the age groups. The EQ-5D-Y-3L showed a significantly less report of problems for mobility (p = 0.013) and usual activities (p = 0.006) for children aged 5-7 years compared with children aged 8-12 and children aged 13-18 years. Similarly, the 5-7 years of age group had a significantly lower EQ-5D-Y-3L LSS compared with the older groups (H = 12.08, p = 0.002). The EQ-TIPS and EQ-5D-Y-3L showed moderate-to-strong associations with the PedsQL. EQ-TIPS median LSS was able to differentiate between groups on the clinical prognosis with a better health-related quality of life (HRQoL) in those where weaning from technology is possible compared with those where weaning is not possible (H = 18.98, p = 0.011). The EQ-5D-Y-3L can discriminate between breathing technology, where those with only a tracheostomy reported better HRQoL (H = 8.92, p = 0.012), and between mild and moderate clinical severity (H = 19.42, p < 0.001). Neither the PedsQL nor the PTHSI was able to discriminate between these groups across the age range. Caregiver and child HRQoL scores showed moderate-to-strong associations. The EQ-TIPS and EQ-5D-Y-3L showed good validity in children dependent on the technology for breathing. The EQ-TIPS and EQ-5D-Y-3L LSS were all able to differentiate between children with known clinical variables and outperformed both the PedsQL and PTHSI, making them preferable for intervention research. The caregiver scores are associated with the child HRQoL scores and thus a spill-over should be accounted for in any interventions targeting this cohort. It is recommended that future studies investigate the reliability and responsiveness of these measures in children dependent on technology for breathing.

Artículo traducido] Mejora de la calidad de vida relacionada con la salud en niños de mediana edad con parálisis cerebral después de un alargamiento miofascial percutáneo selectivo y fisioterapia funcional

Introduction and objectivesChildren with cerebral palsy (CP) experience decreased health-related quality of life (HRQOL). This study aimed to assess the HRQOL of children with CP before versus after a combined program of minimally invasive selective percutaneous myofascial lengthening (SPML) and functional physiotherapy. Material and methodsA single-group pre–posttest design was used. Twenty-six middle childhood children with spastic CP, aged 5–7 years, with Gross Motor Function Classification System levels II–IV underwent SPML surgery and 9 months of postoperative functional strength training therapy. The proxy version of the DISABKIDS-Smiley questionnaire was completed by one parent of each child. Dependent t-tests were used to compare mean pre- and post-measurement scores. ResultsAfter the 9-month intervention, the children with CP had significantly higher quality of life scores (mean difference, 11.06 ± 9.05; 95% confidence interval [CI], 7.40–14.71; p < 0.001). ConclusionsThis study demonstrated that children with CP had better HRQOL after a combined program of minimally invasive SPML surgery and functional physiotherapy (ACTRN12618001535268).

Potential Mediating Roles of Children's Health-Related Quality of Life and Weight-Related Behaviors in the Relationship Between Socio-Educational Advantage and Weight Status.

Background: Socioeconomic status is inversely associated with weight status in developed countries. Underlying mechanisms are still to be clarified. Our aim was to determine if weight-related behaviors and health-related quality of life (HRQoL) mediate the relationship between socio-educational advantage and weight status in Australian children 9 to 13 years of age. Methods: Secondary analysis of data collected by two cluster randomized trials (2019 wave). We measured children's (n = 3978) height, weight, diet, physical activity, sedentary behavior, and HRQoL. Socio-.educational advantage was assessed at school level using the Index of Community Socio-Educational Advantage (ICSEA). A counterfactual framework was used to explore potential mediators in the relationship between socio-educational disadvantage and (1) overweight/obesity and (2) BMI z-score (BMIz). Results: Low socio-educational advantage was associated with increased overweight/obesity and BMIz. The overweight/obesity association was mediated by sedentary behavior (natural indirect effect as a proportion of total, 7.5%) sugar-sweetened beverage (SSB) consumption (12.7%), physical functioning (11.9%), psychosocial health (10.9%), school (6.8%) and social functioning (15.6%), and total HRQoL score (13.8%). The ICSEA-BMIz relationship was mediated by sedentary behavior (5.7%), sleep duration (4.1%), SSB (10.6%), physical functioning (9.9%), psychosocial health (9.1%), school (5.5%) and social (13.7%) functioning, and total HRQoL score (11.7%). Conclusion: Victorian Children with low socio-educational advantage were more likely to be living with overweight or obesity. This relationship was mediated by children's sedentary behavior, SSB consumption, and HRQoL. Trial Registration: Australian New Zealand Clinical Trials Registry (ANZCTR): ACTRN12616000980437 (registered July 26, 2016, retrospectively registered) and ACTRN12618001986268 (registered December 11, 2018, prospectively registered). https://www.anzctr.org.au/Trial/Registration.