Anja Rohenkohl

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

BackgroundWhen 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.MethodsThe 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.ResultsFollowing 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.ConclusionsThe 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.

Psychometric evaluation of the German version of the Patient Activation Measure (PAM13).

BackgroundThe Patient Activation Measure (PAM) consists of 13 items and assesses patient (or consumer) self-reported knowledge, skills, and confidence for self-management of one’s health or chronic condition. The aim of this study was to translate the original American version of the PAM13 into German and to test the psychometric properties of the German version in an elderly, multimorbid population with various chronic conditions.MethodsTranslation was performed by a standardized forward-backward translation process. The PAM13 was sent to 9.075 participants enrolled in a randomized controlled study. 4.306 participants responded to the questionnaire. Descriptive and reliability analyses were carried out. To examine scale properties, Andrich’s Rasch Rating Scale Model was fitted.ResultsThe internal consistency is good (α = 0.88) and the item-rest-correlations were found as strong to moderate. The unidimensionality of the construct was confirmed, with a variance explanation of 40.9% and good model-fits for the Rasch model. However, the lowest response options were very rarely used across all items (below 5%) and ranking order of items according to their difficulty was substantially different from that of the American version. Differential item functioning (DIF) was found in subgroups (sex, age, health status), but differences were small.ConclusionThe German version of the PAM13 showed acceptable reliability and the model-fit statistics confirmed the Rasch model. The different ranking order of the items and the unfair distribution of the response options suggest further research on validation and revision of the construct.

Gesundheitsbezogene Lebensqualität und psychische Gesundheit von kleinwüchsigen Kindern und Jugendlichen

Three percent of children and adolescents in Germany have short stature (SS) which also includes patients with growth hormone deficiency (GHD) and idiopathic short stature (ISS). Growth hormone substitution in these patients is expected to increase height, health-related quality of life (HrQoL) and mental health. The present cross-sectional study investigated healthrelated quality of life and mental health of children with SS in comparison to a representative German population sample and examined differences within patients with respect to treatment status, diagnosis and actual height. A total of 143 patients (4 ‐ 18 years) and their parents completed questionnaires for mental health (SDQ) and health-related quality of life (KIDSCREEN-52). Results showed that children and adolescents with short stature report significant HrQoL impairments in comparison to children with normal height, while differences in mental health were apparent at specific subscale levels. Within the clinical sample, no differences regarding age and gender were detected. Also, no significant differences in HrQoL and mental health were found according to diagnosis (GHD/ISS), treatment status and actual height. Parents rated their childrens’ HrQoL higher than the children themselves, whereas they considered their children’ psychological health problems more serious than the children themselves did. Results suggest that SS is associated with impairments in HrQol and mental health as compared to a population reference group, suggesting that these impairments should be addressed with condition-specific interventions. Generic tools such as KIDSCREEN did not yield differences within the patient group regarding clinical and socio-demographic characteristics. Clinical studies therefore should consider making use of condition specific measures.

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 QoLISSYs cross-cultural equivalence and is recommended for future research.

Evaluation of the American-English Quality of Life in Short Stature Youth (QoLISSY) questionnaire in the United States

BackgroundThe European Quality of Life in Short Stature Youth (QoLISSY) is a novel condition-specific instrument developed to assess health related quality of life (HrQoL) in children/adolescents with short stature from patient and parent perspectives. Study objective was to linguistically validate and psychometrically test the American-English version of the QoLISSY instrument.MethodsUpon conversion of the British-English version to American-English, content validity and acceptance of the questionnaire were examined through focus group discussions with cognitive debriefing in 28 children/adolescents with growth hormone deficiency (GHD) or idiopathic short stature (ISS) and their parents. In the subsequent field test with 51 families and a re-test with 25 families the psychometric performance of the American-English version was examined and compared with the original European dataset.ResultsPilot test results supported the suitability of the American-English version. Good internal consistency with Cronbach’s Alpha ranging from 0.84 to 0.97 and high test-re-test reliabilities were observed in the field test. The QoLISSY was able to detect significant differences according to the degree of short stature with higher HrQoL for taller children. Correlations with a generic HrQoL tool support the QoLISSY’s concurrent validity. The scale’s operating characteristics were comparable to the original European data.ConclusionResults support that the QoLISSY American-English version is a psychometrically sound short stature-specific instrument to assess the patient- and parent- perceived impact of short stature. The QoLISSY instrument is fit for use in clinical studies and health services research in the American-English speaking population.

Early detection and integrated care for adolescents and young adults with severe psychotic disorders: rationales and design of the Integrated Care in Early Psychosis Study (ACCESS III)

The Integrated Care in Early Psychosis (ACCESS III) Study examined the efficacy and cost‐effectiveness of a combined intervention consisting of strategies to improve early detection and quality of care (integrated care including therapeutic assertive community treatment) in adolescents and young adults in the early phase of a severe psychotic disorder from 2011 to 2014.

Leben mit Achondroplasie

OBJECTIVE Presently, little is known aqout the quality of life (QoL) as well as the strengths and difficulties of young people with achondroplasia. This study describes these patient-reported indicators and identifies possible correlates. METHOD At the invitation of a patient organization, a total of 89 short-statured patients aged 8 to 28 years and their parents participated in this study. QoL was assessed cross-sectionally with both generic and disease-specific instruments and the Strengths and Difficulties Questionnaire (SDQ) as a brief behavioral screening. In addition to descriptive analyses, patient data were compared with a reference population. Hierarchical regression analyses reflecting sociodemographic, clinical, and psychological variables were conducted to identify correlates of QoL. RESULTS QoL and the strengths and difficulties of young patients with achondroplasia did not differ substantially from a healthy norm sample. However, the participants reported more behavioral problems and limitations in their physical and social QoL compared to patients with another short stature diagnosis. Strengths and difficulties, height-related beliefs, and social support correlated significantly with QoL. Adding psychological variables to the regression model increased the proportion of variance explained in QoL. CONCLUSIONS Young persons with achondroplasia did not differ in their QoL and strengths and difficulties from healthy controls. Characteristics such as height appear less important for the self-perceived QoL than are strengths and difficulties and protective psychosocia~factors.

Associations between Psychological Problems and Quality of Life in Pediatric Short Stature from Patients’ and Parents’ Perspectives

Short stature has been associated with psychosocial impairments, but whether treatments and achieved height impact on health-related quality of life (HrQoL) and psychological functioning of children/adolescents is still controversial. This study aimed to examine the effects of height deviation and treatment status on psychosocial adaptation outcomes and to identify clinical and psychosocial determinants of internalizing/externalizing problems in a large cohort of short statured children/adolescents from seven European countries. Participants were 345 children aged 8–18 years with a clinical diagnosis of short stature and 421 parents of 4–18 year-old patients. Children and parents reported on psychological problems (Strengths and Difficulties Questionnaire), generic (KIDSCREEN) and condition-specific HrQoL (QoLISSY). According to analyses of covariance, children/adolescents with current short stature presented more parent-reported internalizing problems and lower self- and parent-reported condition-specific HrQoL, compared to patients with an achieved height above -2SD. Treated children self-reported better HrQoL than the untreated group. Hierarchical regression analysis showed that, rather than height–related clinical variables, children’s sex, younger age and poorer HrQoL were the best predictors of psychological problems, explaining 39% of the variance in patient- and 42% in parent-reported internalizing problems, and 22% of the variance in patient- and 24% in parent-reported externalizing problems. Treatment status also moderated the negative links between patient-reported HrQoL and internalizing problems, explaining 2% of additional variance. These results suggest that children with current short stature are at greater risk for internalizing problems. Routine assessment of HrQoL in pediatric healthcare may help identify children for referral to specialized psychological assessment and intervention.

Early detection and integrated care for adolescents and young adults with psychotic disorders: the ACCESS III study

The objective of the study was to investigate whether a combined intervention composed of early detection plus integrated care (EDIC) enhances outcomes in patients with early psychosis compared to standard care (SC).

HRQoL of European children and adolescents with short stature as assessed with generic (KIDSCREEN) and chronic-generic (DISABKIDS) instruments

Short stature may be associated with impairments in health-related quality of life (HRQoL). This study compared the HRQoL of children/adolescents diagnosed with short stature to population norms and examined the effects of height deviation and treatment status on HRQoL. Method: We cross-sectionally assessed 110 children/adolescents aged 8–18, with current short stature (height deviation ≤-2 standard-deviation (SD)) or normal height achieved since diagnosis, and 98 parents, using the generic KIDSCREEN and the chronic-generic DISABKIDS instruments. Results: Generic HRQoL of patients was similar to population norms. Patients with achieved normal height reported better chronic-generic HRQoL when untreated, while patients with current short stature reported better HRQoL upon receiving treatment. Parents reported better HRQoL for treated patients, especially for girls. Conclusion: Although their HRQoL is not significantly compromised, patients diagnosed with short stature may profit from growth-hormone treatment. Specific instruments are needed to adequately assess the effectiveness of treatment.