Trajectories of Health-Related Quality of Life and HbA1c Values of Children and Adolescents With Diabetes Mellitus Type 1 Over 6 Months: A Longitudinal Observational Study

Introduction: To achieve optimized blood glucose concentrations (assessed by HbA1c) and high health-related quality of life (HRQL), children and adolescents with diabetes mellitus type 1 (T1DM) must follow strict disease management strategies. This study aims to investigate HRQL of children and adolescents with T1DM and its association with HbA1c values over the course of 6 months. Methods: Patients aged 7-17 years (n = 203) with T1DM provided HRQL data on a monthly basis. HRQL was measured using the Kids-CAT, a computer-adaptive test (CAT) comprising five generic HRQL domains. HbA1c concentrations were assessed at baseline, at 3 and 6 months. We explored the trajectory of HRQL at the domain level using linear mixed effects models. Further, we investigated the association between HRQL and HbA1c concentrations over time using path analysis models. Results: Children and adolescents with T1DM reported high scores across all HRQL domains over time. However, those with an HbA1c concentrations of \u3e 9.0% reported significantly lower scores in physical well-being and parent relations compared with those with an HbA1c concentration of \u3c 7.5%. Path analysis models revealed a minimal temporal relationship between HbA1c and HRQL, with a small negative impact of HbA1c on physical well-being, psychological well-being and parent relations. Conclusion: Although observed HRQL of young patients with T1DM was comparable to age-related German-speaking reference population over the course of 6 months, those with an HbA1c concentration \u3e 9.0% reported lower scores in selected HRQL domains. Thus, special attention should be drawn to HRQL of children and adolescents with higher HbA1c concentrations. The minimal relationship between HbA1c and HRQL indicates that the two therapy goals, i.e., achievement and maintenance of glycemic targets and high HRQL, should be considered and evaluated independently in clinical routine. Trial Registration: DRKS00006326 (German Clinical Trial Register), date of registration: August 1st, 2014

The European DISABKIDS project: development of seven condition-specific modules to measure health related quality of life in children and adolescents

BACKGROUND: The European DISABKIDS project aims to enhance the Health Related Quality of Life (HRQoL) of children and adolescents with chronic medical conditions and their families. We describe the development of the seven cross-nationally tested condition-specific modules of the European DISABKIDS HRQoL instrument in a population of children and adolescents. The condition-specific modules are intended for use in conjunction with the DISABKIDS chronic generic module. METHODS: Focus groups were used to construct the pilot version of the DISABKIDS condition-specific HRQoL modules for asthma, juvenile idiopathic arthritis, atopic dermatitis, cerebral palsy, cystic fibrosis, diabetes and epilepsy. Analyses were conducted on pilot test data in order to construct field test versions of the modules. A series of factor analyses were run, first, to determine potential structures for each condition-specific module, and, secondly, to select a reduced number of items from the pilot test to be included in the field test. Post-field test analyses were conducted to retest the domain structure for the final DISABKIDS condition-specific modules. RESULTS: The DISABKIDS condition-specific modules were tested in a pilot study of 360 respondents, and subsequently in a field test of 1152 respondents in 7 European countries. The final condition-specific modules consist of an 'Impact' domain and an additional domain (e.g. worry, stigma, treatment) with between 10 to 12 items in total. The Cronbach's alpha of the final domains was found to vary from 0.71 to 0.90. CONCLUSION: The condition-specific modules of the DISABKIDS instrument were developed through a step-by-step process including cognitive interview, clinical expertise, factor analysis, correlations and internal consistency. A cross-national pilot and field test were necessary to collect these data. In general, the internal consistency of the domains was satisfactory to high. In future, the DISABKIDS instrument may serve as a useful tool with which to assess HRQoL in children and adolescents with a chronic condition. The condition-specific modules can be used in conjunction with the DISABKIDS chronic generic module

Screening of MAMLD1 Mutations in 70 Children with 46,XY DSD: Identification and Functional Analysis of Two New Mutations

More than 50% of children with severe 46,XY disorders of sex development (DSD) do not have a definitive etiological diagnosis. Besides gonadal dysgenesis, defects in androgen biosynthesis, and abnormalities in androgen sensitivity, the Mastermind-like domain containing 1 (MAMLD1) gene, which was identified as critical for the development of male genitalia, may be implicated. The present study investigated whether MAMLD1 is implicated in cases of severe 46,XY DSD and whether routine sequencing of MAMLD1 should be performed in these patients

Assessment of data quality in a multi-centre cross-sectional study of participation and quality of life of children with cerebral palsy

BACKGROUND: SPARCLE is a cross-sectional survey in nine European regions, examining the relationship of the environment of children with cerebral palsy to their participation and quality of life. The objective of this report is to assess data quality, in particular heterogeneity between regions, family and item non-response and potential for bias. METHODS: 1,174 children aged 8–12 years were selected from eight population-based registers of children with cerebral palsy; one further centre recruited 75 children from multiple sources. Families were visited by trained researchers who administered psychometric questionnaires. Logistic regression was used to assess factors related to family non-response and self-completion of questionnaires by children. RESULTS: 431/1,174 (37%) families identified from registers did not respond: 146 (12%) were not traced; of the 1,028 traced families, 250 (24%) declined to participate and 35 (3%) were not approached. Families whose disabled children could walk unaided were more likely to decline to participate. 818 children entered the study of which 500 (61%) self-reported their quality of life; children with low IQ, seizures or inability to walk were less likely to self-report. There was substantial heterogeneity between regions in response rates and socio-demographic characteristics of families but not in age or gender of children. Item non-response was 2% for children and ranged from 0.4% to 5% for questionnaires completed by parents. CONCLUSION: While the proportion of untraced families was higher than in similar surveys, the refusal rate was comparable. To reduce bias, all analyses should allow for region, walking ability, age and socio-demographic characteristics. The 75 children in the region without a population based register are unlikely to introduce bias

Generic health literacy measurement instruments for children and adolescents:a systematic review of the literature

Background Health literacy is an important health promotion concern and recently children and adolescents have been the focus of increased academic attention. To assess the health literacy of this population, researchers have been focussing on developing instruments to measure their health literacy. Compared to the wider availability of instruments for adults, only a few tools are known for younger age groups. The objective of this study is to systematically review the field of generic child and adolescent health literacy measurement instruments that are currently available. Method A systematic literature search was undertaken in five databases (PubMed, CINAHL, PsycNET, ERIC, and FIS) on articles published between January 1990 and July 2015, addressing children and adolescents ?18 years old. Eligible articles were analysed, data was extracted, and synthesised according to review objectives. Results Fifteen generic health literacy measurement instruments for children and adolescents were identified. All, except two, are self-administered instruments. Seven are objective measures (performance-based tests), seven are subjective measures (self-reporting), and one uses a mixed-method measurement. Most instruments applied a broad and multidimensional understanding of health literacy. The instruments were developed in eight different countries, with most tools originating in the United States (n =?6). Among the instruments, 31 different components related to health literacy were identified. Accordingly, the studies exhibit a variety of implicit or explicit conceptual and operational definitions, and most instruments have been used in schools and other educational contexts. While the youngest age group studied was 7-year-old children within a parent-child study, there is only one instrument specifically designed for primary school children and none for early years. Conclusions Despite the reported paucity of health literacy research involving children and adolescents, an unexpected number of health literacy measurement studies in children?s populations was found. Most instruments tend to measure their own specific understanding of health literacy and not all provide sufficient conceptual information. To advance health literacy instruments, a much more standardised approach is necessary including improved reporting on the development and validation processes. Further research is required to improve health literacy instruments for children and adolescents and to provide knowledge to inform effective interventionspublishersversionPeer reviewe