Quality of life and mental health in children and adolescents during the first year of the COVID-19 pandemic: results of a two-wave nationwide population-based study.

BACKGROUND: The COVID-19 pandemic has disrupted the lives of children and adolescents worldwide. The German COPSY study is among the first population-based longitudinal studies to examine the mental health impact of the pandemic. The objective of the study was to assess changes in health-related quality of life (HRQoL) and mental health in children and adolescents and to identify the associated risk and resource factors during the pandemic. METHODS: A nationwide longitudinal survey was conducted with two waves during the pandemic (May/June 2020 and December 2020/January 2021). In total, n = 1923 children and adolescents aged 7 to 17 years and their parents participated (retention rate from wave 1 to wave 2: 85%). The self-report and parent-proxy surveys assessed HRQoL (KIDSCREEN-10), mental health problems (SDQ with the subscales emotional problems, conduct problems, hyperactivity, and peer problems), anxiety (SCARED), depressive symptoms (CES-DC, PHQ-2) and psychosomatic complaints (HBSC-SCL). Mixed model panel regression analyses were conducted to examine longitudinal changes in mental health and to identify risk and resource factors. RESULTS: The HRQoL of children and adolescents decreased during the pandemic, and emotional problems, peer-related mental health problems, anxiety, depressive and psychosomatic symptoms increased over time, however the change in global mental health problems from wave 1 to wave 2 was not significant, and some changes were negligible. Socially disadvantaged children and children of mentally burdened parents were at particular risk of impaired mental health, while female gender and older age were associated with fewer mental health problems. A positive family climate and social support supported the mental health of children and adolescents during the pandemic. DISCUSSION: Health promotion, prevention and intervention strategies could support children and adolescents in coping with the pandemic and protect and maintain their mental health.

[Quality of Life and Experienced Distress of Patients Suspected of having a Rare (Chronic) Health Condition - Initial Findings from the ZSE-DUO Study]. / Lebensqualität und erlebte Belastungen von Patient*innen mit Verdacht auf eine Seltene Erkrankung ­ erste Erkenntnisse aus der ZSE-DUO Studie.

OBJECTIVE: Patients suspected of having a rare (chronic) health condition have often gone a long way within the healthcare system. To date, little is known about the health-related quality of life of this group of patients. The study aims to describe the health-related quality of life and the perceived distress of patients suspected of having a rare (chronic) health condition and compare the results with standard values of the German population. METHODS: Eighty patients suspected of having a rare (chronic) health condition were recruited in the nationwide intervention study "ZSE-DUO" and reported their health-related quality of life and perceived distress using the SF-8 and the Distress-Thermometer. RESULTS: The patients rated all eight dimensions of quality of life as well as the physical and mental component scores of the SF-8 significantly lower than the general population. On average, the perceived distress was rated significantly higher. More than 90% of the sample indicated distress in the clinical range. Exhaustion, pain, limited mobility as well as worries and fears were mentioned most frequently as concrete problems, with percentages ranging from 73% to 90% of the total sample. DISCUSSION: In comparison to German reference data, patients suspected of having a rare (chronic) health condition report a massive impairment of their quality of life and a high burden, which is especially characterized by physical and emotional problems. The lack of a diagnosis could explain the high proportion of emotional problems, as it can create a form of legitimation of one's own disease experience. CONCLUSION: The present results underline the need for research on the psychosocial impact of the possible presence of a rare (chronic) health condition. The high distress and the impact on the physical and psychological quality of life domains also highlight the need for care in this patient group.

Quality of Life of Children and Adolescents with Short Stature: The Twofold Contribution of Physical Growth and Adaptive Height-Related Cognitive Beliefs.

This study aimed to examine the health-related quality of life (HrQoL), coping, height-related beliefs, and social support of children/adolescents with short stature, the sociodemographic, clinical, and psychosocial variables associated with HrQoL, and the moderating role of sociodemographic and clinical variables on the associations between psychosocial variables and HrQoL. 114 Portuguese children/adolescents with short stature, aged 8-18 years old, completed the Quality of Life in Short Stature Youth questionnaire and the Satisfaction with Social Support Scale. Regression analyses explained 54% of the variance of HrQoL, with significant main effects of current height deviation and height-related beliefs, and a significant interaction effect between beliefs and diagnosis. Results suggest that a multidisciplinary therapeutic approach, not only focused on hormone treatment to boost physical growth, but also including psychosocial interventions focused on the modification of height-related beliefs, may contribute to improve the HrQoL of pediatric patients with short stature.

Evaluation of two family-based intervention programs for children affected by rare disease and their families - research network (CARE-FAM-NET): study protocol for a rater-blinded, randomized, controlled, multicenter trial in a 2x2 factorial design.

BACKGROUND: Families of children with rare diseases (i.e., not more than 5 out of 10,000 people are affected) are often highly burdened with fears, insecurities and concerns regarding the affected child and its siblings. Although families caring for children with rare diseases are known to be at risk for mental disorders, the evaluation of special programs under high methodological standards has not been conducted so far. Moreover, the implementation of interventions for this group into regular care has not yet been accomplished in Germany. The efficacy and cost-effectiveness of a family-based intervention will be assessed. METHODS/DESIGN: The study is a 2x2 factorial randomized controlled multicenter trial conducted at 17 study centers throughout Germany. Participants are families with children and adolescents affected by a rare disease aged 0 to 21 years. Families in the face-to-face intervention CARE-FAM, online intervention WEP-CARE or the combination of both will be treated over a period of roughly 6 months. Topics discussed in the interventions include coping, family relations, and social support. Families in the control condition will receive treatment as usual. The primary efficacy outcome is parental mental health, measured by the Structured Clinical Interview for DSM-IV (SCID-I) by blinded external raters. Further outcomes will be assessed from the parents' as well as the children's perspective. Participants are investigated at baseline, 6, 12 and 18 months after randomization. In addition to the assessment of various psychosocial outcomes, a comprehensive health-economic evaluation will be performed. DISCUSSION: This paper describes the implementation and evaluation of two family-based intervention programs for Children Affected by Rare Disease and their Family's Network (CARE-FAM-NET) in German standard care. A methodologically challenging study design is used to reflect the complexity of the actual medical care situation. This trial could be an important contribution to the improvement of care for this highly burdened group. TRIAL REGISTRATION: German Clinical Trials Register: DRKS00015859 (registered 18 December 2018) and ClinicalTrials.gov : NCT04339465 (registered 8 April 2020). Protocol Version: 15 August 2020 (Version 6.1). Trial status: Recruitment started on 1 January 2019 and will be completed on 31 March 2021.

Psychometric properties of the quality of life in short statured youth (QoLISSY) questionnaire within the course of growth hormone treatment.

BACKGROUND: The Quality of Life of Short Stature Youth (QoLISSY) questionnaire is a patient- and parent-reported outcome measure assessing health-related quality of life (HRQOL) in short stature youth. This study evaluates the psychometric properties of the QoLISSY questionnaire within a German prospective trial of short statured children treated with human growth hormone (hGH). METHOD: The instrument was administered to children with idiopathic growth hormone Deficiency (IGHD) and small for gestational age (SGA) before and after 12 month of hGH treatment. Children with idiopathic short stature (ISS) served as a reference group receiving no treatment. Psychometric testing included scale distribution characteristics, reliability (internal consistency), criterion-and convergent validity (correlations with the generic KIDSCREEN-Index, inter-correlations among QOLISSY subscales), known-group validity (treatment status, height SDS), and responsiveness analysis (ability to detect change). RESULTS: One hundred fifty-two parents and 66 children/adolescents completed both HRQOL assessments. The QoLISSY demonstrated good reliability with Cronbach's alpha > .70. Moderate significant correlations between QoLISSY domains and the KIDSCREEN-10 Index supported criterion validity. Statistically significant differences in HRQOL were observed between treatment groups at baseline with children who were about to start treatment reporting a significantly lower HRQOL compared to the children who will not receive treatment. No significant differences were found between the level of short stature based on height SDS scores (≤ - 2 SDS, > - 2 SDS). Furthermore, the instrument detected significant changes in HRQOL between the treated and the untreated group in patient-reports. CONCLUSIONS: In conclusion, the scales showed satisfactory reliability, adequate validity and ability to detect change in self-reported HRQOL within GH treatment. Findings support QoLISSY's further use in clinical trials, offering the opportunity to adequately assess HRQOL from the patients' and caregivers' perspective to improve patient-centered care.

A DELPHI study on aspects of study design to overcome knowledge gaps on the burden of disease caused by serogroup B invasive meningococcal disease.

BACKGROUND: Value assessment of vaccination programs against serogroup B invasive meningococcal disease (IMD) is on the agenda of public health authorities. Current evidence on the burden due to IMD is unfit for pinning down the nature and magnitude of the full social and economic costs of IMD for two reasons. First, the concepts and components that need to be studied are not agreed, and second, measures of the concepts that have been studied are weak and inconsistent. Thus, the economic evaluation of the available serogroup B meningococcal (MenB) vaccines is difficult. The aims of this DELPHI study are to: (1) agree on the concepts and components determining the burden of MenB diseases that need to be studied; and (2) seek consensus on appropriate methods and study designs to measure quality of life (QoL) associated with MenB induced long-term sequelae in future studies. METHODS: We designed a DELPHI questionnaire based on the findings of a recent systematic review on the QoL associated with IMD-induced long-term sequelae, and iteratively interviewed a panel of international experts, including physicians, health economists, and patient representatives. Experts were provided with a controlled feedback based on the results of the previous round. RESULTS: Experts reached consensus on all questions after two DELPHI rounds. Major gaps in the literature relate (i) to the classification of sequelae, which allows differentiation of severity levels, (ii) to the choice of QoL measures, and (iii) to appropriate data sources to examine long-term changes and deficits in patients' QoL. CONCLUSIONS: Better conceptualisation of the structure of IMD-specific sequelae and of how their diverse forms of severity might impact the QoL of survivors of IMD as well as their family network and care-providers is needed to generate relevant, reliable and generalisable data on QoL in the future. The results of this DELPHI panel provide useful guidance on how to choose the study design, target population and appropriate QoL measures for future research and hence, help promote the appropriateness and consistency in study methodology and sample characteristics.

Do 8- to 18-year-old children/adolescents with chronic physical health conditions have worse health-related quality of life than their healthy peers? a meta-analysis of studies using the KIDSCREEN questionnaires.

PURPOSE: This meta-analytic review aimed to estimate the magnitude of health-related quality of life (HrQoL) impairments, as assessed by the KIDSCREEN questionnaires, both self- and parent-reported, in 8- to 18-years-old children/adolescents with chronic health conditions. METHODS: To identify studies using the KIDSCREEN questionnaires, three electronic databases (PubMed, PsycINFO, EBSCOhost Psychology & Behavioral Sciences) were searched. The final search (February 14-15, 2018) revealed 528 non-duplicated articles, of which 23 papers (21 studies) directly compared the HrQoL of pediatric patients to community/healthy controls and were included in the meta-analysis. Pooled mean differences (MD) with 95% CIs were estimated using the inverse-variance random-effects method. RESULTS: Of the 21 studies, 16 used self-reports, one used parent-reports and four adopted a multi-informant approach. Self-reported data were retrieved from 20 studies (4852 cases/28,578 controls), and parent-reported data were retrieved from four studies (511 cases/433 controls). Pediatric patients presented significant HrQoL impairments in the domains of physical well-being (MD = - 4.84, 95% CI - 6.44/- 3.24 for self-reports; MD = - 6.86, 95% CI - 10.42/- 3.29 for parent-reports) and peers and social support (MD = - 1.29, 95% CI - 2.25/- 0.34 for self-reports; MD = - 3.90, 95% CI - 5.28/- 2.52 for parent-reports), compared to community/healthy peers. Between-studies heterogeneity was explained by diagnostic categories, instrument version and informants. CONCLUSIONS: The identification of significant HrQoL impairments among pediatric patients, specifically in the physical and social domains, highlights the importance of routine psychosocial assessment and intervention in primary pediatric healthcare services. Specific recommendations include the use of profile measures, both self- and parent-reports, and the prioritization of oncology, endocrinology and neurology services.

Cross-cultural selection and validation of instruments to assess patient-reported outcomes in children and adolescents with achondroplasia.

PURPOSE: Achondroplasia, as the most common form of disproportionate short stature, potentially impacts the health-related quality of life (HRQOL) and functioning of people with this condition. Because there are no psychometrically validated patient-reported outcome (PRO) condition-specific instruments for achondroplasia, this study selected and tested available generic, disease-specific and under development questionnaires for possible use in multinational clinical research. METHODS: A three-step approach was applied. First, a literature review and clinician/expert opinions were used to select relevant PRO questionnaires. Second, focus group discussions, including a group cognitive debriefing for piloting of the questionnaires with children/adolescents with achondroplasia and their parents, were performed in Spain and Germany. Third, a field-test study was conducted to test the psychometric properties of these instruments. RESULTS: Six questionnaires were identified as potentially relevant in children with achondroplasia. In each country, five focus groups including a cognitive debriefing were conducted, and the results narrowed the possibilities to three instruments as most appropriate to assess HRQOL (the generic PedsQL, the height-specific QoLISSY, and the achondroplasia-specific APLES). Results of the field study indicate the QoLISSY and the PedsQL questionnaires to be most appropriate for use in clinical research at this time. CONCLUSION: This selection study is a step forward in assessing the impact of achondroplasia on HRQOL. Of the instruments examined, the QoLISSY and the PedsQL both capture items relevant to children with achondroplasia and have met the psychometric validation criteria needed for use in research. The APLES instrument is a promising tool that should be revisited upon psychometric validation.

Agreement between mothers', fathers', and children's' ratings on health-related quality of life in children born with esophageal atresia - a German cross-sectional study.

BACKGROUND: Esophageal atresia (EA) is a rare congenital malformation, which is characterized by the discontinuity of the esophagus. We investigated the agreement between mothers', fathers', and children's' ratings on health-related quality of life (HRQOL) in children born with EA. We aimed to broaden the understanding of subjective experiences of HRQOL from different perspectives. We hypothesized that the agreement between mother and father ratings would be high, whereas the agreement between child and mother ratings as well as child and father ratings would show more substantial differences. METHODS: We obtained data from 40 families (23 mother-father dyads of children aged 2-7 years and 17 mother-father-child triads of children and adolescents aged 8-18 years) with children born with EA, who were treated in two German hospitals. HRQOL was measured using the generic PedsQL™ questionnaires and the condition-specific EA-QOL© questionnaires. We calculated intraclass coefficients and performed one-way repeated measures ANOVAs to analyze differences for each domain as well as for the total scores. RESULTS: Intraclass correlation coefficients (ICCs) indicated a strong agreement (≥.80) between mother and father reports of children's HRQOL for both generic and condition-specific measurements. The ICCs for the generic HRQOL for mother/father-child-dyads revealed only fair to good agreement, whereas ICCs for condition-specific HRQOL showed high agreement for mother-child and father-child-agreement. Analyses of Covariance revealed differences in mother/father-child agreement in the generic domain School, both parents reporting lower HRQOL scores than the children themselves. Fathers reported significantly higher scores in the condition-specific domain Social than their children. CONCLUSIONS: Results showed that mothers' and fathers' reports corresponded to each other. Nonetheless, these reports might not be interchangeably used because mother-child and father-child agreement showed differences. Children might know the best on how they feel, and parent proxy-report is recommended when reasons such as young age, illness, or cognitive impairments do not allow to ask the child. But parent-report - no matter if reported by mother or father - should only be an additional source to broaden the view on the child's health status and well-being. The current study contributes to a better understanding of the complex family relationships involved when parenting a child born with EA.

Subjective integration and quality of life of Sub-Saharan African migrants in Germany.

OBJECTIVE: This article presents empirical evidence on the quality of life (QoL) of Sub-Saharan African (SSA) migrants in Germany, exploring its association with subjective integration and the influence of some socio-economic and sociodemographic characteristics. STUDY DESIGN: This is a cross-sectional study design using quantitative data from 518 SSA migrants collected across the 16 federal states of Germany, and these data were analysed in this study. METHODS: Association between participants' QoL, measured by the four domains of the Bref version of the World Health Organisation QoL measure, subjective integration and sociodemographic characteristics were evaluated using Pearson product-moment correlations. Stepwise multiple linear regressions were performed to explore the contribution of predictor variables on the QoL domain. RESULTS: Participants' age averaged 32.5 years (standard deviation [SD] 7.93). The sample reported a low QoL score with a mean score of 64.3 (SD 14.4, range 70.2). Multiple linear regression analyses revealed that subjective integration, age, education and gender had significant associations and explained up to 27% of the variance in the QoL domain scores. CONCLUSION: The findings of this study support the conclusion that subjective integration positively and significantly associates with the physical health, psychological health, social relationships and environmental domains of SSA migrants' QoL in Germany.

Health-related quality of life experiences in children and adolescents born with esophageal atresia: A Swedish-German focus group study.

BACKGROUND: Esophageal atresia (EA) is a rare malformation of the esophagus, which needs surgical treatment. Survival rates have reached 95%, but esophageal and respiratory morbidity during childhood is frequent. Child and parent perspectives and cultural and age-specific approaches are fundamental in understanding children's health-related quality of life (HRQoL) and when developing a pediatric HRQoL questionnaire. We aimed to increase the conceptual and cross-cultural understanding of condition-specific HRQoL experiences among EA children from Sweden and Germany and investigate content validity for an EA-specific HRQoL questionnaire. METHODS: Eighteen standardized focus groups (FGs) with 51 families of EA children aged 2-17 years in Sweden (n = 30 families) and Germany (n = 21 families) were used to explore HRQoL experiences, which were content analyzed into HRQoL domains. The Swedish HRQoL domains were analyzed first and used as framework to evaluate HRQoL content reported in the German FGs. HRQoL experiences were then categorized as physical, social, and emotional HRQoL burden or resource. RESULTS: One thousand nine hundred eight HRQoL statements were recorded. All nine EA-specific HRQoL domains identified in the Swedish FGs (eating, social relationships, general life issues, communication, body issues, bothersome symptoms, confidence, impact of medical treatment, and additional difficulties due to concomitant anomalies) were recognized in the FGs held in Germany, and no additional EA-specific HRQoL domain was found. The HRQoL dimensions referenced physical burden (n = 655, 34.5%), social burden (n = 497, 26.0%), social resources (n = 303, 15.9%), emotional burden (n = 210, 11.0%), physical resources (n = 158, 8.3%), and emotional resources (n = 85, 4.5%). CONCLUSION: This first international FG study to obtain the EA child and his or her parents' perspective on HRQoL suggests Swedish-German qualitative comparability of the HRQoL domains and content validity for a cross-cultural EA-specific HRQoL questionnaire. EA children make positive and negative HRQoL experiences, but prominently related to physical and social burden, which underlines appropriate follow-up care and future research.

Exploring the Potential of a Pretend Play Intervention in Young Patients With Leukemia.

PURPOSE: The aims of the study are 1) to gain knowledge of parents' and professionals' perceptions about cancer stricken children's resources, burdens, and ability to pretend play, and 2) to prepare the initiation of a pretend play intervention based on children's needs and included parents' and professionals' feedback. DESIGN AND METHODS: Qualitative design using semi-structured interviews with 13 parents of children diagnosed with leukemia and 15 professionals in the field of pediatric oncology. Themes were derived with content analysis via deductive and inductive coding. RESULTS: Analysis resulted in five topics. (1) Ability to play in the context of leukemia (2) ways of coping with leukemia (3) difficulty in transition to normality (4) parental quality of life and parents' needs (5) perceptions of the potential of pretend play. CONCLUSION: Study results indicate the potential of pretend play interventions for young cancer patients and the need for additional professional support of parents. PRACTICE IMPLICATIONS: Pretend play is a tool children carry with them regardless of their circumstances. If we can enhance their ability to play, doing so should give them an advantage in creative problem solving and creative expression as they deal with a life threatening disease.

[Quality of life as a target criterion in schizophrenia therapy]. / Lebensqualität als Zielkriterium in der Schizophrenietherapie.

Consideration of quality of life has developed into an important concept of a patient-oriented medicine in the last 50 years. With it, it should be possible to capture the subjective experience of the patients and describe its different dimensions. Today, the quality of life as a patient-reported endpoint is also an integral part of the early benefit assessment under the German Drug Market Reorganization Act. In the treatment of schizophrenic patients, however, improvement in quality of life as an accepted target criterion has only been established with delay.

Measuring Spiritual Well-Being in Brazilian Adolescents with Chronic Illness Using the FACIT-Sp-12: Age Adaptation of the Self-Report Version, Development of the Parental-Report Version, and Validation.

Spiritual well-being is a major issue in health care, but instruments for measuring this construct in adolescents are lacking. This study adapted the 12-item Functional Assessment of Chronic Illness Therapy-Spiritual Well-being Scale (FACIT-Sp-12) for use with Brazilian adolescents with chronic diseases and developed a parental observer-rated version, using an expert panel, back-translation, and cognitive interviews with 72 participants. The psychometric properties of both versions were verified with two- and three-factor models by testing with 212 participants. The self- and parental-reported versions showed face validity, content validity, and acceptable levels of internal consistency for the overall scale and the two-factor model. The convergent validity was satisfactory for most items in both two- and three-factor models, but there was a lack of discrimination in the three-factor model using multitrait-multimethod analysis. This study presents the first instrument to assess the spiritual well-being of adolescents from their point of view and to allow their parents to serve as evaluators. However, we recommend further psychometric testing of the self- and parental-report scales to assess spiritual well-being in adolescents with chronic diseases in Brazil.

Assessing health-related quality of life in Japanese children with a chronic condition: validation of the DISABKIDS chronic generic module.

BACKGROUND: This study examined the reliability and validity of the Japanese versions of the DISABKIDS-37 generic modules, a tool for assessing the health-related quality of life (HRQOL) of children and adolescents with a chronic condition. METHODS: The study was conducted using a sample of 123 children/adolescents with a chronic medical condition, aged 8-18 years, and their parents. Focus interviews were performed to ensure content validity after translation. The classical psychometric tests were used to assess reliability and scale intercorrelations. The factor structure was examined with confirmatory factor analysis (CFA). Convergent validity was assessed by the correlation between the total score and the sub-scales of DISABKIDS-37 as well as the total score of KIDSCREEN-10. RESULTS: Both the children/adolescent and parent versions of the score showed good to high internal consistency, and the test-retest reliability correlations were r = 0.91 or above. The CFA revealed that the modified models for all domains were better fit than the original 37 item scale model for both self-report and proxy-report. Moderate to high positive correlations were found for the associations within DISABKIDS-37 sub-scales and between the subscales and total score, except for the treatment sub-scale, which correlated weakly with the remaining sub-scales. The total score of the child-reported version of KIDSCREEN-10 correlated significantly and positively with the total score and all the sub-scales of the child-reported version of DISABKIDS-37 except the Treatment sub-scale in adolescents. CONCLUSIONS: The modified models of Japanese version of DISABKIDS generic module were psychometrically robust enough to assess the HRQOL of children with a chronic condition.

The Esophageal-Atresia-Quality-of-life Questionnaires: Feasibility, Validity and Reliability in Sweden and Germany.

OBJECTIVES: Esophageal atresia (EA) is a rare malformation characterized of discontinuity of the esophagus, concurrent with or without a tracheoesophageal fistula (TEF). We report the feasibility validity and reliability of a condition-specific quality-of-life (QOL) tool for EA/TEF children, the age-adapted EA-QOL-questionnaires, when used in Sweden and Germany. METHODS: A total of 124 families of children with EA/TEF participated in the study; 53 parents completed the EA-QOL-questionnaire for children aged 2 to 7 years; 62 children/71 parents the EA-QOL-questionnaire for children 8 to 17 years. Feasibility was determined from the percentage of missing item responses. Based on clinical data and previously validated generic QOL-instruments (PedsQL 4.0, DISABKIDS-12), the final EA-QOL scores were evaluated against hypotheses of validity (known-groups/concurrent/convergent) and reliability (internal consistency/retest reliability of scores for 3 weeks). Significant level was P < 0.05. RESULTS: In the questionnaire for EA/TEF children aged 2 to 7 years, 16/18 items were completed with missing values <6% (range 0%-7.5%), and in the questionnaire for 8 to 17-year-olds, 24/24 child-reported items (range 0%-4.8%) and 21/24 parent-reported items (range 0%-7.0%). In both age-specific EA-QOL-questionnaires, desirable standards for known-groups and concurrent validity were fulfilled; digestive symptoms and feeding difficulties negatively impacted EA-QOL-Total-scores (P < 0.001), and as hypothesized, in 2 to 7-year-olds, respiratory symptoms decreased EA-QOL-Total-scores (P = 0.002). Correlations between the EA-QOL and generic QOL questionnaires supported convergent validity. Internal consistency reliability was satisfactory. The level of agreements of EA-QOL-scores between the field- and retest study were good to excellent. CONCLUSIONS: The overall psychometric performance of the EA-QOL-questionnaires for EA/TEF children is satisfactory and can enhance outcome evaluations in future research and clinical practice.

Children's psychosocial functioning and parents' quality of life in paediatric short stature: The mediating role of caregiving stress.

OBJECTIVES: On the basis of the multidimensional model of the caregiving process, this study aimed (a) to compare the levels of quality of life (QoL) and psychological problems of children with short stature and the levels of caregiving stress and QoL of their parents, between diagnostic, treatment, and current height deviation groups, and (b) to examine the direct and indirect links, via caregiving stress, between children's psychosocial functioning and their parents' QoL. METHOD: The sample was collected in 5 European countries and comprised 238 dyads of 8- to 18-year-old children and adolescents with a clinical diagnosis of growth hormone deficiency or idiopathic short stature and one of their parents. The children completed self-report measures of height-related QoL (Quality of Life in Short Stature Youth Core Module) and psychological problems (Strengths and Difficulties Questionnaire); the parents reported on their own QoL (EUROHIS-QOL-8 Index) and caregiving stress (Quality of Life in Short Stature Youth Effects on Parents subscale). RESULTS: Children who were treated and who achieved normal height reported better QoL compared to those untreated and with current short stature. Parents of children with idiopathic short stature and current short stature presented greater caregiving stress than parents of children with growth hormone deficiency and achieved normal height. Children's better psychosocial functioning was indirectly associated with parents' better QoL, via less caregiving stress, and these links were invariant across diagnoses, treatment status, and current height deviation. CONCLUSIONS: These results suggest that, along with growth hormone treatments, multidisciplinary interventions in paediatric endocrinology should be family-centred, by targeting both the children's psychosocial functioning and the parents' stress, in order to improve individual and family adaptation.

Assessing health-related quality of life in young Japanese children with chronic conditions: Preliminary validation of the DISABKIDS smiley measure.

BACKGROUND: Although there is an increasing need to investigate the health-related quality of life (HRQOL) of children and adolescents with chronic conditions in Japan, there is currently no standardized measure in which young children can directly answer questions about their HRQOL. The DISABKIDS Smiley measure uses face emoticons to measure HRQOL and distress caused by illness and related treatments among young children. We tested the reliability and validity of the DISABKIDS Smiley measure in a sample of young Japanese children. METHODS: After translating the child and parent questionnaires into Japanese, a pre-test was performed to test the content validity in accordance with guidelines from the DISABKIDS Group. In total, 60 child-parent pairs were recruited to participate in the survey. We measured internal consistency of the scales using Cronbach's alpha as well as Guttman split-half, test-retest reliability using intraclass correlation coefficients (ICCs) at a two-week interval, and ICCs between child- and parent-reported scores. Convergent validity of the scale was also examined against the Kiddy-KINDL scale. RESULTS: Both child-reported and parent-reported scales showed good internal consistency and split-half reliability. Test-retest reliability of the child-reported version (ICC = 0.53, p = 0.004) was lower than that of the parent-reported version (ICC = 0.80, p < 0.001). Moderate to good agreement between child- and parent- reported scales was observed in both the first (ICC = 0.75, p < 0.001) and second administration (ICC = 0.71, p < 0.001). Moderate to very strong positive correlations were observed with the total score of the Kiddy-KINDL child-reported version (r = 0.51, p < 0.001), and facets of the Kiddy-KINDL parent-reported version (ranging from r = 0.364 to r = 0.60, p < 0.001) and total score (r = 0.71, p < 0.001). CONCLUSIONS: The psychometric property of the instrument showed that the Japanese version of the DISABKIDS Smiley can be applied to assess the HRQOL of Japanese children with chronic conditions. Further investigation will be needed to explore the reliability and validity for repeated use of the instrument in clinical practice as an indicator of clinical significance.

Perceived stress in first year medical students - associations with personal resources and emotional distress.

BACKGROUND: Medical students have been found to report high levels of perceived stress, yet there is a lack of theoretical frameworks examining possible reasons. This cross-sectional study examines correlates of perceived stress in medical students on the basis of a conceptual stress model originally developed for and applied to the general population. The aim was to identify via structural equation modeling the associations between perceived stress and emotional distress (anxiety and depression), taking into account the activation of personal resources (optimism, self-efficacy and resilient coping). METHODS: Within this cross-sectional study, 321 first year medical students (age 22 ± 4 years, 39.3% men) completed the Perceived Stress Questionnaire (PSQ-20), the Self-Efficacy Optimism Scale (SWOP) and the Brief Resilient Coping Scale (BRCS) as well as the Patient Health Questionnaire (PHQ-4). The statistical analyses used t-tests, ANOVA, Spearman Rho correlation and multiple regression analysis as well as structural equation modeling. RESULTS: Medical students reported higher levels of perceived stress and higher levels of anxiety and depression than reference samples. No statistically significant differences in stress levels were found within the sample according to gender, migration background or employment status. Students reported more self-efficacy, optimism, and resilient coping and higher emotional distress compared to validation samples and results in other studies. Structural equation analysis revealed a satisfactory fit between empirical data and the proposed stress model indicating that personal resources modulated perceived stress, which in turn had an impact on emotional distress. CONCLUSIONS: Medical students' perceived stress and emotional distress levels are generally high, with personal resources acting as a buffer, thus supporting the population-based general stress model. Results suggest providing individual interventions for those students, who need support in dealing with the challenges of the medical curriculum as well as addressing structural determinants of student stress such as course load and timing of exams.

Understanding, Assessing and Improving Health-Related Quality of Life of Young People with Achondroplasia- A Collaboration between a Patient Organization and Academic Medicine.

Achondroplasia (ACH) is a rare, genetically determined health condition. Patients suffer from disproportional short stature and multiple physical and functional impairments as well as socioemotional problems. Despite the burden of disease, only few studies focus on health-related quality of life (HrQoL) of young ACH patients. In a series of studies, the BKMF e.V. in cooperation with the UKE studied ACH patients' and parents' experience of HrQoL, their responses to HrQoL questionnaires and their evaluation of a HrQoL based intervention. Both qualitative and quantitative approaches were used. Psychometrically appropriate instruments were identified and the wellbeing of young patients with ACH was analyzed showing no difference from a healthy norm sample using generic instruments. However, disease-specific instruments showed discrepancies between patients with proportional and disproportional short stature. Still, results show a significant effect by age and the evaluation of the counselling concept reveals that young ACH-patients especially benefit from such intervention.