Development of an International Standard Set of Outcomes and Measurement Methods for Routine Practice for Infants, Children, and Adolescents with Epilepsy: The International Consortium for Health Outcomes Measurement Consensus Recommendations.

At present, there is no internationally accepted set of core outcomes or measurement methods for epilepsy clinical practice. The International Consortium for Health Outcomes Measurement (ICHOM) convened an international working group of experts in epilepsy, people with epilepsy, and their representatives to develop minimum sets of standardized outcomes and outcome measurement methods for clinical practice. Using modified Delphi consensus methods with consecutive rounds of online voting over 12 months, a core set of outcomes and corresponding measurement tool packages to capture the outcomes were identified for infants, children, and adolescents with epilepsy. Consensus methods identified 20 core outcomes. In addition to the outcomes identified for the ICHOM Epilepsy adult standard set, behavioral, motor, and cognitive/language development outcomes were voted as essential for all infants and children with epilepsy. The proposed set of outcomes and measurement methods will facilitate the implementation of the use of patient-centered outcomes in daily practice.

Development of an International Standard Set of Outcomes and Measurement Methods for Routine Practice for Adults with Epilepsy: The International Consortium for Health Outcomes Measurement Consensus Recommendations.

At present, there is no internationally accepted set of core outcomes or measurement methods for epilepsy clinical practice. Therefore, the International Consortium for Health Outcomes Measurement (ICHOM) convened an international working group of experts in epilepsy, people with epilepsy and their representatives to develop minimum sets of standardized outcomes and outcomes measurement methods for clinical practice that support patient-clinician decision-making and quality improvement. Consensus methods identified 20 core outcomes. Measurement tools were recommended based on their evidence of strong clinical measurement properties, feasibility, and cross-cultural applicability. The essential outcomes included many non-seizure outcomes: anxiety, depression, suicidality, memory and attention, sleep quality, functional status, and the social impact of epilepsy. The proposed set will facilitate the implementation of the use of patient-centered outcomes in daily practice, ensuring holistic care. They also encourage harmonization of outcome measurement, and if widely implemented should reduce the heterogeneity of outcome measurement, accelerate comparative research, and facilitate quality improvement efforts.

Weight Stigma in Latin America, Asia, the Middle East, and Africa: A Scoping Review.

INTRODUCTION: Being stigmatized because of one's weight can pose physical, mental, and social challenges. While weight stigma and its consequences are established throughout Europe, North America, and Australasia, less is known about weight stigma in other regions. The objective of this study was to identify the extent and focus of weight stigma research in Latin America, Asia, the Middle East, and Africa. METHODS: A scoping review of weight stigma research in Latin America, Asia, the Middle East, and Africa was conducted. SCOPUS and PsychINFO databases were searched, and weight stigma experts were contacted to identify relevant literature. Sources were classified based on country/region, population, setting, and category of weight stigma researched. RESULTS: A total of 130 sources were identified from 33 countries and territories. Results indicate that weight stigma has been investigated across populations and settings, mainly focusing on manifestations of weight stigma through experiences, practices, drivers, and personal outcomes of these manifestations. CONCLUSIONS: Weight stigma is a developing global health concern not restricted to Europe, North America, and Australasia. The extent and focus of weight stigma research in Latin America, Asia, the Middle East, and Africa vary between countries and regions leaving several research gaps that require further investigation.

Validation of the Swedish version of PROMIS-29v2 and FACIT-Dyspnea Index in patients with systemic sclerosis.

PURPOSE: To evaluate the reliability, internal consistency, and construct validity of the Swedish versions of PROMIS-29 and Functional Assessment of Chronic Illness Therapy-Dyspnea (FACIT-Dyspnea) instruments in patients with systemic sclerosis (SSc). METHODS: In a cross-sectional study, consecutive SSc patients completed a paper-based survey. Internal consistency was assessed using Cronbach's alpha. Test-retest reliability was tested employing weighted Kappa (Kw) and intra-class correlation coefficient (ICC). Construct validity was evaluated by hypotheses testing using RAND-36, MRC Dyspnea score, Scleroderma Health Assessment Questionnaire (SHAQ) and clinical measurements. RESULTS: Forty-nine patients (86% female; 73% limited cutaneous SSc) completed the survey. The mean disease duration was 11 years and mean SHAQ was 0.5. Internal consistency and test-retest reliability were good with the exception of PROMIS-29 anxiety. PROMIS-29, FACIT-Dyspnea, and Functional limitation showed strong correlations to corresponding RAND-36 domains (|rs|=0.67 to -0.85). Relevant PROMIS-29 domains, FACIT-Dyspnea and Functional limitation correlated strongly to SHAQ and VAS overall disease severity (|rs|=0.60 to -0.75). Ceiling effects (>15%) were found in six PROMIS-29 domains and in both FACIT-Dyspnea and Functional limitations. Four (4/5) hypotheses were confirmed. CONCLUSIONS: PROMIS-29 and FACIT-Dyspnea meet the requirements for reliability and have adequate construct validity in Swedish patients with SSc.Implications for rehabilitationPROMIS-29v2 and Functional Assessment of Chronic Illness Therapy-Dyspnea (FACIT-Dyspnea) Index are patient outcome measures that gain increasing interest for the evaluation of patient with rheumatologic diseases.PROMIS-29v2 and FACIT-Dyspnea Index meet the requirements for reliability and have adequate construct validity compared to legacy measures in Swedish patients with systemic sclerosis.Translation and validation of PROMs is important for studies of rare diseases in multi-center collaborations.

Perceived stigma in adults with epilepsy in Sweden and associations with country of birth, socioeconomic status, and mental health.

BACKGROUND: Stigma contributes to negative health outcomes and amplifies health disparities in epilepsy. This study aimed to investigate associations of perceived stigma with the country of birth and socioeconomic status (SES). METHODS: This is a cross-sectional questionnaire study. Participants were recruited consecutively from three demographically different neurology outpatient clinics in the southwest of Sweden. Participants responded to a questionnaire concerning their epilepsy, country of birth, mother tongue, and different SES-variables. The Neuro-QOL stigma scale and the Jacoby stigma scale were applied to assess stigma and the Hospital Anxiety and Depression Scale (HADS) and PROMIS Mental Health scale were applied to assess mental health. RESULTS: In total 161 adults with epilepsy were included in the cohort. The median Neuro-QOL stigma score was 48.3, and was higher among foreign-born than in native-born participants (foreign-born in non-European country 52.3, in other European country 49.8, and in native-born 47.0, p = 0.003). Other factors associated with Neuro-QOL were seizure frequency last year (≥2 seizures 52.4 compared to 1 seizure 50.9 and no seizures 44.3, p < 0.001), having had seizures in public (yes 50.9 compared to no 44.7, p = 0.035), HADS depression score ≥11 (57.4 compared to 47.8 for score <11 points, p < 0.001), HADS anxiety score ≥11 (53.5 compared to 46.8 for score <11 points, p < 0.001), and lower PROMIS Mental Health score (42.9 for PROMIS < 40 compared to 54.4 for PROMIS > 60, p < 0.01). A stepwise multiple regression analysis indicated that having had seizures the last year increased the average Neuro-QOL stigma score with 5.89 and appeared as the most determining factor for the Neuro-QOL stigma score among the variables investigated. CONCLUSIONS: It is important that the concerns of foreign-born patients are acknowledged and that the focus of seizure control and the detection and treatment of comorbidities are prioritized in the management of epilepsy and perceived stigma.

Factors of family impact in a Swedish-German cohort of children born with esophageal atresia.

BACKGROUND: After repair of esophageal atresia (EA), childhood survivors commonly present with digestive and respiratory morbidity, and around 55% have associated anomalies. Although it is known that these problems can reduce health-related quality of life in children with EA, less is understood about the impact on the family. We aimed to identify factors related to family impact in children with EA. METHODS: One parent each of a child with EA (2-18 years) in 180 families from Sweden and Germany answered the PedsQL™ Family Impact Module as the dependent variable. The independent variables were the child's parent-reported health-related quality of life as measured by PedsQL™ 4.0, current symptoms, school situation, and parent/family characteristics together with child clinical data from the medical records. RESULTS: Stepwise multivariable regression analysis showed a multifactorial model of the total family impact scores (R2 = 0.60), with independent factors being the child's overall generic health-related quality of life, school-absence ≥ 1/month, severe tracheomalacia, a family receiving carer's allowance, and a parent with no university/college education, p < 0.05. Logistic regression analysis showed that an increased number of symptoms in the child the preceding 4 weeks lowered the family impact scores; however, the child's feeding (R2 = 0.35) and digestive symptoms (R2 = 0.25) explained more in the variation of scores than the child's respiratory symptoms (R2 = 0.09), p < 0.0001. CONCLUSIONS: Family functioning may be a contributing factor to the maintenance of child health. The study findings suggest multifactorial explanations to family impact in children with EA, which are essential when optimizing the support to these families in clinical and psychosocial practice. Future research should explore experiences of family impact from all family members' perspectives and multicenter studies are warranted to understand better the effectiveness of psychosocial-educational interventions to families of children with EA.

Features of Childhood Growth, Lifestyle, and Environment Associated with a Cardiometabolic Risk Score in Young Adults.

INTRODUCTION: In young adults, metabolic syndrome (MS) is rare. To better assess the risks for future cardiovascular disease (CVD), a cardiometabolic score can be used, ranking the disease risk in each subject. The score is a continuous variable, summarizing the individual z-scores for waist circumference, blood pressure, blood levels of glucose, triglycerides, and high-density-lipoprotein cholesterol. Our main aim was to assess the association between early childhood growth and the cardiometabolic score in young adults. METHODS: Study participants were recruited among subjects in the longitudinal population-based GrowUp 1990 Gothenburg study. Those with information on weight and length at birth, as well as weight, height, waist circumference, and parental body mass index (BMI) at 10 years of age were invited to participate in a health survey at 18-20 years of age. Five hundred and thirteen young adults (female 51%) were included. Multivariable linear stepwise regression analysis was applied. RESULTS: The mean (standard deviation) BMI was 22.2 (3.26) in males and 21.3 (2.69) kg/m2 in females; the cardiometabolic score was 0.24 (3.12) and -0.22 (3.18), respectively. A statistically significantly higher score (p < 0.001) was seen in individuals with MS, as defined by IDF. After controlling for adult lifestyle features, the BMI z-score at 10 years of age was a significant risk factor in both sexes for an elevated cardiometabolic score in early adulthood with mean [standard error] beta 0.47 [0.19], p = 0.014 in males, and 0.82 [017], p < 0.0001 in females. In males, a high maternal BMI and low age at adiposity rebound and in females, high birth weight were also associated with a statistically significant risk. Additionally, contraceptive use in females was a risk factor for an elevated cardiometabolic score and in males, a high lifestyle-related index score showed a protective association with the cardiometabolic score. CONCLUSION: A high BMI z-score at 10 years of age is a risk factor for the cardiometabolic state in young adults, an outcome pointing to the preventive potential of monitoring BMI in 10-year-old schoolchildren. This finding must however be validated in a new large cohort. Moreover, in young adults in whom MS is rare, the cardiometabolic score seems to be a promising and more powerful tool to detect risks for CVD later in life than using MS categorization.

Development of an international standard set of patient-centred outcome measures for overall paediatric health: a consensus process.

OBJECTIVE: To develop an Overall Pediatric Health Standard Set (OPH-SS) of outcome measures that captures what matters to young people and their families and recognising the biopsychosocial aspects of health for all children and adolescents regardless of health condition. DESIGN: A modified Delphi process. SETTING: The International Consortium for Health Outcomes Measurement convened an international Working Group (WG) comprised of 23 international experts from 12 countries in the field of paediatrics, family medicine, psychometrics as well as patient advisors. The WG participated in 11 video-conferences, through a modified Delphi process and 9 surveys between March 2018 and January 2020 consensus was reached on a final recommended health outcome standard set. By a literature review conducted in March 2018, 1136 articles were screened for clinician and patient-reported or proxy-reported outcomes. Further, 4315 clinical trials and 12 paediatric health surveys were scanned. Between November 2019 and January 2020, the final standard set was endorsed by a patient validation (n=270) and a health professional (n=51) survey. RESULTS: From a total of 63 identified outcomes, consensus was formed on a standard set of outcome measures that comprises 10 patient-reported outcomes, 5 clinician-reported measures, and 6 case-mix variables. The four developmental age-specific packages (ie, 0-5, 6-12, 13-17, 18-24 years) include either five or six measures with an average time for completion of 20 min. CONCLUSIONS: The OPH-SS is a starting point to drive value-based paediatric healthcare delivery from a global perspective for enhancing child and adolescent physical health and psychosocial well-being.

Microbial invasion of the amniotic cavity is associated with impaired cognitive and motor function at school age in preterm children.

BACKGROUND: Chorioamnionitis is an important cause of preterm delivery. Data on neurodevelopmental outcome in exposed infants are inconsistent due to difficulties in diagnosing intrauterine infection/inflammation and lack of detailed long-term follow-up. We investigate cognitive and motor function in preterm infants at early school age and relate the findings to bacteria in amniotic fluid obtained by amniocentesis (microbial invasion of the amniotic cavity (MIAC)) or placenta findings of histological chorioamnionitis (HCA) or fetal inflammatory response syndrome (FIRS). METHOD: Sixty-six infants with gestational age <34 weeks at birth and without major disabilities were assessed using WISC-III and the Bruininks-Oseretsky Test of Motor Proficiency. Results were corrected for gestational age and sex. RESULTS: Children exposed to MIAC had significantly lower scores for full-scale IQ and verbal IQ compared to the non-MIAC group and the difference in full-scale IQ remained after correction for confounding factors. The MIAC group had also significantly lower motor scores after correction. In contrast, motor function was not affected in infants exposed to HCA or FIRS and differences between groups for cognitive scores were lost after corrections. CONCLUSION: Exposure to bacteria in amniotic fluid is associated with lower motor and cognitive scores in school age preterm infants without major disabilities.

Generic Health-Related Quality of Life after Repair of Esophageal Atresia and Its Determinants within a German-Swedish Cohort.

AIM: Despite advances of outcomes of esophageal atresia (EA), knowledge on patients' health-related quality of life (HRQoL) is sparse. Due to the heterogeneity of EA, larger cohorts need to be investigated to ensure reliability of data. Aim was to determine generic HRQoL after EA repair in a Swedish-German cohort. PATIENTS AND METHODS: Ethical approval was obtained. A total of 192 patients (2-18 years; 134 Swedish; 58 German) were included. Clinical data were reviewed. EA was classified in "severe" and "mild/moderate." Pediatric Quality of Life Inventory 4.0 Generic Core Scale (PedsQL 4.0) was used in appropriate versions (2-7 years; 8-18 years; self- [SR] and proxy report [PR]) to determine generic HRQoL. RESULTS: Swedish and German samples were clinically and demographically comparable. HRQoL was lower in "severe EA" versus "mild/moderate" (2-18 years; total score; PR 85.6 vs. 73.6; p < 0.001) and Gross A versus Gross C type EA (2-7 years; total score; PR 61.0 vs. 79.3; p = 0.035). Total HRQoL was higher in the Swedish versus German sample (2-18 years; total score; PR 82.3 vs. 72.7; p = 0.002). HRQoL was impaired in the German sample versus healthy population (2-18 years; total score; PR 72.7 vs. 82.7; p = 0.001). In German patients (8-18 years), HRQoL was higher in SR versus PR (80.7 vs. 74.7; p = 0.044). Patients' age and presence of VACTERL association or isolated anorectal malformations did not affect HRQoL. Various differences were detected regarding different dimensions of PedsQL 4.0. CONCLUSION: In this first international study, we found several differences in perception of generic HRQoL. HRQoL appears to be determined by the type of EA and severity rather than patients' age or the presence of typical associated malformations. Country-specific differences may be culturally dependent, but further investigations are suggested. A condition-specific instrument validated for EA may provide additional insights.

Empowering aspects for healthy food and physical activity habits: adolescents' experiences of a school-based intervention in a disadvantaged urban community.

PURPOSE: This study aimed to describe adolescents' experiences of participating in a health-promoting school-based intervention regarding food and physical activity, with a focus on empowering aspects. Method: The school was located in a urban disadvantaged community in Sweden, characterized by poorer self-reported health and lower life expectancy than the municipality average. Focus group interviews with adolescents (29 girls, 20 boys, 14-15 years) and their teachers (n = 4) were conducted two years after intervention. Data were categorized using qualitative content analysis. RESULTS: A theme was generated, intersecting with all the categories: Gaining control over one's health: deciding, trying, and practicing together, in new ways, using reflective tools. The adolescents appreciated influencing the components of the intervention and collaborating with peers in active learning activities such as practicing sports and preparing meals. They also reported acquiring new health information, that trying new activities was inspiring, and the use of pedometers and photo-food diaries helped them reflect on their health behaviours. The adolescents' experiences were also echoed by their teachers. Conclusions: To facilitate empowerment and stimulate learning, health-promotion interventions targeting adolescents could enable active learning activities in groups, by using visualizing tools to facilitate self-reflection, and allowing adolescents to influence intervention activities.

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.

The development of a health-related quality-of-life instrument for young people with narcolepsy: NARQoL-21.

BACKGROUND: Narcolepsy is a lifelong sleep disorder with a prevalence of between 0.03% and 0.06% and onset at around puberty. It is associated with psychiatric comorbidities and cognitive difficulties. No valid and reliable condition-specific health-related quality-of-life (HrQoL) instrument has been developed for this population. METHODS: A questionnaire based on four mixed-gender age-defined focus group discussions and a patient panel analysis was administered to young people with narcolepsy and a control group. External reliability was measured by a test-retest procedure and internal reliability was measured using Cronbach's alpha. Convergent validity with the KIDSCREEN-10 index was assessed using with intraclass correlation coefficients (ICC) and receiver operating characteristic (ROC) curves. Factor analysis techniques were used to identify suitable items and confirm the factor structure. Baseline values were assessed for convergent validity, ceiling effects, agreement and sensitivity. Comparison with KIDSCREEN-10 was made on the basis of area under the curve (AUC). RESULTS: One hundred young people with narcolepsy and 95 control subjects returned questionnaires. The factor structure revealed two main factors with five domains and 21 questions, which was confirmed with confirmatory factor analysis. The domains of the NARQoL-21 showed good independence while the floor and ceiling effects were acceptable. The external reliability (0.928), convergent validity (rs = 0.769) and internal consistency (Cronbach's alpha = 0.886) were excellent. A Bland-Altman plot revealed some proportional bias. Good discriminant validity was detected for control/patient (Cohen's d = 2.114). ROC analysis showed significantly better AUC for NARQoL-21 (0.939) than KIDSCREEN (0.877). A cut-off score equivalent to KIDSCREEN-10 for suboptimal HrQoL which maximized sensitivity (84%) and specificity (92%) was found at NARQoL-21 score below 42. CONCLUSIONS: Establishing the validity of a disease-specific HrQoL instrument in a population of people with a rare condition poses significant challenges. The mixed-methods approach adopted here has resulted in a questionnaire of 21 items with good discrimination and convergent validity, and excellent internal and external reliability, allowing precise and stable measurements. The cut-off score can be useful to identify patients with very poor HrQoL and thus improve the design of treatment options. Further testing in a longitudinal cohort is recommended in order to establish responsiveness.

Declining Well-Being in Young Swedes Born in 1990 Versus 1974.

PURPOSE: Well-being is affected by the environment, including societal changes. In this study, specific dimensions of well-being were compared in two cohorts of Swedish adolescents born 16 years apart. METHODS: Two groups of 18-year-olds, "Grow up Gothenburg" 1974 and 1990 birth cohorts, completed a self-reported questionnaire including the Gothenburg Well-Being in adolescence scale (GWBa). In addition, height and weight were measured, resulting in 4,362 participants (1974 birth cohort) and 5,151 participants (1990 birth cohort) with age, height, weight, and well-being data. The GWBa consists of a total score and five dimensions: mood, physical condition, energy, self-esteem, and stress balance. RESULTS: Total well-being was significantly lower in the later-born cohort, and the greatest difference was seen for the dimension stress balance (feeling calm, unconcerned, unstressed, and relaxed), although effect sizes were modest. In both boys and girls, well-being was lower for all dimensions in the later-born cohort, with the exception of Self-esteem in girls, which was higher in the later-born cohort. In both cohorts, boys reported higher well-being than girls for all dimensions. The mean body mass index z-score was higher in boys from the later-born cohort, but after adjusting for weight status, the differences in well-being between the cohorts persisted. CONCLUSIONS: Well-being was lower in the later-born cohort, particularly for the dimension stress balance. Differences were not explained by the shift in weight status indicating that other societal changes have had an impact on well-being levels. Managing high levels of stress might be an area of intervention in adolescents for improved well-being.

Evaluating the predictive ability of childhood body mass index classification systems for overweight and obesity at 18 years.

AIM: To evaluate the performance of three childhood body mass index classification systems defining weight status at age 10, for predicting overweight and obesity at 18 years, according to the World Health Organization adult body mass index classification. METHODS: Weight and height of 4235 Swedish girls and boys were measured both at around ages 10 and 18 years. Predictive ability of the extended International Obesity Task Force body mass index cut-offs (2012), the World Health Organization body mass index-for-age (2007) and a Swedish body mass index reference (2001) were assessed for sensitivity and specificity. RESULTS: For predicting overweight including obesity at 18 years, the World Health Organization 2007 and the Swedish body mass index reference 2001 had similar sensitivity, 68% and 71%. The International Obesity Task Force 2012 had a significantly lower sensitivity, 53%. Specificity was 82-91% and highest for International Obesity Task Force 2012. For predicting obesity, the sensitivity for International Obesity Task Force 2012 was 29%, significantly lower than for the other two, 63% and 70%. Specificity was 94-100%, and highest for International Obesity Task Force 2012. CONCLUSIONS: In situations when optimal screening sensitivity is required for identifying as many high-risk children as possible, the World Health Organization 2007 and the Swedish body mass index reference 2001 performed better than the International Obesity Task Force 2012. However, it is important to keep in mind that the International Obesity Task Force 2012 will identify the fewest false positives.

Cross-cultural equivalence of the patient- and parent-reported quality of life in short stature youth (QoLISSY) questionnaire.

BACKGROUND: Testing cross-cultural equivalence of patient-reported outcomes requires sufficiently large samples per country, which is difficult to achieve in rare endocrine paediatric conditions. We describe a novel approach to cross-cultural testing of the Quality of Life in Short Stature Youth (QoLISSY) questionnaire in five countries by sequentially taking one country out (TOCO) from the total sample and iteratively comparing the resulting psychometric performance. METHODS: Development of the QoLISSY proceeded from focus group discussions through pilot testing to field testing in 268 short-statured patients and their parents. To explore cross-cultural equivalence, the iterative TOCO technique was used to examine and compare the validity, reliability, and convergence of patient and parent responses on QoLISSY in the field test dataset, and to predict QoLISSY scores from clinical, socio-demographic and psychosocial variables. RESULTS: Validity and reliability indicators were satisfactory for each sample after iteratively omitting one country. Comparisons with the total sample revealed cross-cultural equivalence in internal consistency and construct validity for patients and parents, high inter-rater agreement and a substantial proportion of QoLISSY variance explained by predictors. CONCLUSION: The TOCO technique is a powerful method to overcome problems of country-specific testing of patient-reported outcome instruments. It provides an empirical support to QoLISSY's cross-cultural equivalence and is recommended for future research.

The case for an international patient-reported outcomes measurement information system (PROMIS®) initiative.

Patient-reported outcomes (PROs) play an increasingly important role in clinical practice and research. Modern psychometric methods such as item response theory (IRT) enable the creation of item banks that support fixed-length forms as well as computerized adaptive testing (CAT), often resulting in improved measurement precision and responsiveness. Here we describe and discuss the case for developing an international core set of PROs building from the US PROMIS® network.PROMIS is a U.S.-based cooperative group of research sites and centers of excellence convened to develop and standardize PRO measures across studies and settings. If extended to a global collaboration, PROMIS has the potential to transform PRO measurement by creating a shared, unifying terminology and metric for reporting of common symptoms and functional life domains. Extending a common set of standardized PRO measures to the international community offers great potential for improving patient-centered research, clinical trials reporting, population monitoring, and health care worldwide. Benefits of such standardization include the possibility of: international syntheses (such as meta-analyses) of research findings; international population monitoring and policy development; health services administrators and planners access to relevant information on the populations they serve; better assessment and monitoring of patients by providers; and improved shared decision making.The goal of the current PROMIS International initiative is to ensure that item banks are translated and culturally adapted for use in adults and children in as many countries as possible. The process includes 3 key steps: translation/cultural adaptation, calibration, and validation. A universal translation, an approach focusing on commonalities, rather than differences across versions developed in regions or countries speaking the same language, is proposed to ensure conceptual equivalence for all items. International item calibration using nationally representative samples of adults and children within countries is essential to demonstrate that all items possess expected strong measurement properties. Finally, it is important to demonstrate that the PROMIS measures are valid, reliable and responsive to change when used in an international context.IRT item banking will allow for tailoring within countries and facilitate growth and evolution of PROs through contributions from the international measurement community. A number of opportunities and challenges of international development of PROs item banks are discussed.

Assessing the quality of life of health-referred children and adolescents with short stature: development and psychometric testing of the QoLISSY instrument.

BACKGROUND: When evaluating the outcomes of treatment in paediatric endocrinology, the health-related quality of life (HrQoL) of the child is to be taken into consideration. Since few self-reported HrQoL instruments exist for children with diagnosed short stature (dSS), the objective of this study was to develop and psychometrically test a targeted HrQoL instrument for use in multinational clinical research. METHODS: The target population were short stature (height<-2 SDS) children and adolescents (age 8-12 and 13-18 years) with a diagnosis of growth hormone deficiency (GHD) or idiopathic short stature (ISS), differing in growth hormone treatment status. Focus group discussions for concept and item generation, piloting of the questionnaire with cognitive debriefing, and instrument field testing with a retest were conducted simultaneously in five countries. After qualitative and preliminary quantitative analyses, psychometric testing of field test data in terms of reliability and validity including confirmatory factor analyses (CFA) was performed. RESULTS: Following item generation from focus group discussions, 124 items were included in a pilot test with a cognitive debriefing exercise providing preliminary feedback on item and domain operating characteristics. A field test with 268 participants showed high internal consistency reliabilities (alpha 0.82-0.95), good correlations with generic measures (up to r=.58), significant known group differences (e.g. in height: F=32, df 244, p<0.001) and an acceptable CFA model fit suggesting construct validity of the three-domain core structure with 22 items, supplemented by three mediator domains with 28 items. CONCLUSIONS: The QoLISSY questionnaire is a promising step forward in assessing the impact of dSS on HrQoL. It is based on items generated from the subjective experience of short stature children referred for endocrine investigation, is validated for use in five languages and it is easy to administer in clinical and research settings.

Parental perception of health-related quality of life in children and adolescents with short stature: literature review and introduction of the parent-reported QoLISSY instrument.

BACKGROUND: Health-related quality of life (HrQoL) of the child diagnosed with short stature is an important outcome to be assessed both from the patient as well as from the parental perspective. The objective of this study was to review the literature on parent-reported HrQoL and to subsequently develop and psychometrically test the parent-reported version of the Quality of Life in Short Stature Youth (QoLISSY) instrument for use in clinical and epidemiologic research. METHODS: A review of the literature on parental assessment of child HrQoL via PUBMED was followed by a psychometric analysis of data collected within the European QoLISSY study, in which 686 eligible parents of short statured children/adolescents (aged 4-18 years) meeting inclusion criteria participated. Patient inclusion criteria were a height below -2 SD, a diagnosis of growth hormone deficiency (GHD) or idiopathic short stature (ISS), and treatment status in terms of receiving or not receiving recombinant human growth hormone therapy. Focus groups eliciting parental HrQoL statements, pilot testing with cognitive debriefing, and a field test in 317 parents with a retest in 148 parents were conducted simultaneously in France, Germany, Spain, Sweden and the UK. The psychometric performance of the parent-reported instrument, developed in parallel to the child/ adolescent self-report version, was assessed using standard tests of reliability and validity. RESULTS: Literature search failed to identify a cross-culturally developed height specific instrument available for both patient self-report and parental observer report. Analysis of the QoLISSY focus group phase conducted separately in children, adolescents and parents yielded 169 items generated from parent focus groups. A cognitive debriefing exercise followed by a pilot test of preliminary psychometric characteristics resulted in deleting poorly performing items. Field testing of the parent-reported version suggested a three-domain core HrQoL structure with 22 items, additional 44 items assessing three mediator domains and two parent specific domains. The parent report version demonstrated good criterion and construct validity as well as internal consistency and test retest reliability. CONCLUSIONS: The QoLISSY parent report questionnaire closes a gap in the simultaneous assessment of parent and child perception of HrQoL in an international context. It is based on items generated from the experience of short statured children, adolescents and their parents and is validated for use in five European languages. It is feasible, relevant for this population, psychometrically sound and is easy to administer in research and clinical settings.

When do short children realize they are short? Prepubertal short children's perception of height during 24 months of catch-up growth hormone treatment.

AIM: To examine perceived height during the first 24 months of growth hormone (GH) treatment in short prepubertal children. METHODS: Ninety-nine 3- to 11-year-old short prepubertal children with either isolated GH deficiency (n = 32) or idiopathic short stature (n = 67) participated in a 24-month randomized trial of individualized or fixed-dose GH treatment. Children's and parents' responses to three perceived height measures: relative height (Silhouette Apperception Test), sense of height (VAS short/tall), and judgment of appropriate height (yes/no) were compared to measured height. RESULTS: Children and parents overestimated height at start (72%, 54%) and at 24 months (52%, 30%). Short children described themselves as tall until 8.2 years (girls) and 9 years (boys). Prior to treatment, 38% of children described their height as appropriate and at 3 months, 63%. Mother's height, parental sense of the child's tallness and age explained more variance in children's sense of tallness (34%) than measured height (0%). CONCLUSION: Short children and parents overestimate height; a pivotal age exists for comparative height judgments. Even a small gain in height may be enough for the child to feel an appropriate age-related height has been reached and to no longer feel short.