Stepped, evidence-based and integrated care service model vs. usual care for mental disorders: A randomized controlled trial (RECOVER).

Stepped, evidence-based and integrated care service models have the potential to be used as a reference for mental health services. RECOVER aimed to evaluate cost savings, effectiveness, and cost-effectiveness of such a model within a two arm, assessor- and data analysist-blinded RCT in Hamburg, Germany. Participants aged 16-79 years with mental disorders were randomly assigned either to RECOVER or treatment as usual (TAU). Primary outcomes comprised costs, effectiveness (combined symptoms, functioning, quality of life), and cost-effectiveness, hierarchically ordered. Outcomes were evaluated according to the ITT principle, group differences regarding costs with adjusted generalized linear models, effectiveness with ANCOVA models, and cost-effectiveness with the incremental cost-effectiveness ratio (ICER) and cost-effectiveness acceptability curves (CEACs). Between 1/1/2018 and 12/31/2020, n = 891 were finally included (n = 477 in RECOVER, n = 444 in TAU). RECOVER was associated with significantly lower annual total costs (-22 %), health and social care costs (-25 %) and hospital costs (-50 %). Effectiveness analyses showed a significantly better outcome for RECOVER with the fully imputed data . The CEACs descriptively demonstrated that RECOVER was cost-effective with a probability of >95 %. Treatment in RECOVER resulted in substantial cost reductions with better cost-effectiveness. RECOVER can be recommended as a reference model for comprehensive and integrated mental health services.

[Assessment of Direct (COVID-19-Related) and Collateral, Psychosocial Pandemic Consequences for Vulnerable Groups by the Example of Serious Mental Illness]. / Erfassung direkter (COVID-19-bezogener) und kollateraler, psychosozialer Folgen für vulnerable Gruppen am Beispiel schwerer psychischer Erkrankungen.

The indirect pandemic consequences could by far exceed the direct effects of SARS-CoV-2 in terms of costs, morbidity, and mortality. This essay includes a proposed method (matrix) to visualize virus-related and psychosocial risks for different populations side by side in a systematic and concise manner. COVID-19-related and psychosocial vulnerability, stressors, direct and indirect consequences are derived on a theoretical and empirical basis. An exemplary quantification of the matrix for the vulnerable group of people with severe mental illness revealed a very high risk for severe COVID-19 consequences, as well as a pronounced risk for psychosocial collateral effects. The proposed approach could be further discussed for a risk-graded pandemic management, crisis recovery, and future preparedness to adequately address psychosocial collateral effects and better identify and protect vulnerable groups in this regard.

Remission with or without comorbid substance use disorders in early psychosis: long-term outcome in integrated care (ACCESS III study).

Introduction: Schizophrenia-Spectrum-Disorders are associated with poor long-term outcome as well as disability and often severely affect the lives of patients and their families often from symptom onset. Up to 70% of first episode psychosis (FEP) patients suffer from comorbid substance use disorders (SUD). We aimed at studying the course of illness in FEP patients within evidence-based care, with and without comorbid SUD, to examine how decreased, remitted or persistent substance use impacted rates of a combined symptomatic and functional long-term recovery compared with patients without SUD. Methods: ACCESS III is an integrated care model for FEP or patients in the early phase of non-affective and affective psychotic disorders. Treatment trajectories of patients, who had been in ACCESS care for 1 year, with and without SUD were compared with regard to the course of illness and quality of life using Mixed Model Repeated Measures (MMRM) and recovery rates were compared using binary logistic regression. Change in substance use was coded as either persistent, decreased/remitted or no use. Results: ACCESS III was a prospective 1-year study (N = 120) in patients aged 12-29 years. Of these, 74 (61.6%) had a comorbid SUD at admission. There were no group differences regarding the course of illness between patients with or without comorbid SUD or between patients with a substance abuse or substance dependence. The only outcome parameter that was affected by SUD was quality of life, with larger improvement found in the group without substance use (p = 0.05) compared to persistent and remitted users. Using LOCF, 44 patients (48.9%) fulfilled recovery criteria at the endpoint; recovery did not differ based on substance use status. Discussion: SUD and especially substance dependence are common in psychotic disorders even in FEP patients. Evidence-based integrated care led to long-term improvement in patients with comorbid SUD and rate of recovery did not differ for patients with substance use.

Service users' perceptions of relevant and helpful components of an integrated care concept (ACCESS) for psychosis.

Introduction: Psychotic disorders have a significant impact on patients' lives and their families, and long-term treatment with individually tailored multimodal combinations of therapies is often required. Integrated care (IC) concepts such as the "Hamburg Model (ACCESS)" with a focus on psychotic disorders, includes different (therapeutic) components with pharmaco- and psychotherapy, family involvement, home treatment and the option of using a 24/7 crisis hotline. All components are offered by a therapeutically-oriented assertive community treatment (TACT) team in a need-adapted manner. So far, however, little is known about which specific components are regarded as especially relevant and helpful by the users of IC. Methods: Patients currently participating in IC completed a questionnaire as part of the continuous quality assurance study (ACCESS II) in which they were asked to rate the different components of treatment according to their relevance and helpfulness, considering the individual's unique experiences with IC and needs in mental health care. Furthermore, they were asked to make suggestions regarding additional helpful components of treatment. Results: Fifty patients participated in this survey (23% of the patients currently participating in the IC concept). For participants, the most helpful and important factors were having the same therapist in the long-term and the 24/7 crisis telephone. Additional components suggested by patients included more addiction-specific therapies and increased focus on vocational rehabilitation and integration. Conclusion: From the perspective of the users of IC, long-term care from a trusted therapist with whom there is a therapeutic relationship and the possibility to reach someone they already know from the TACT team 24/7 serves as the best basis for effective care, fostering trust, understanding, and open communication. In contrast, home treatment remains a relevant aspect of evidence-based care for people with severe mental illness, but perhaps surprisingly, is not viewed as the most important issue.

Mediators of quality of life change in people with severe psychotic disorders treated in integrated care: ACCESS II study.

BACKGROUND: Patients with severe psychotic disorders exhibit a severely reduced quality of life (QoL) at all stages of the disease. Integrated care often led to an improvement in QoL. However, the specific mediators of QoL change are not yet well understood. METHODS: The ACCESS II study is a prospective, long-term study investigating the effectiveness of an integrated care program for people with severe psychotic disorders (IC-TACT) that includes Therapeutic Assertive Community Treatment within a care network of in- and outpatient services at the University Medical Center Hamburg-Eppendorf, Germany. We examined longitudinal associations between QoL and the hypothesized mediators of change (i.e., negative symptoms, depression, and anxiety), using cross-lagged panel models. RESULTS: The sample includes 418 severely ill patients treated in IC-TACT for at least 1 year. QoL increased, whereas symptom severity decreased significantly from baseline to 6-month follow-up (p-values ≤ 0.001), and remained stable until 12-month follow-up. QoL and symptom severity demonstrated significant auto-correlated effects and significant cross-lagged effects from QoL at baseline to negative symptoms (6 months, ß = -0.20, p < 0.001) to QoL (12 months, ß = -0.19, p < 0.01) resulting in a significant indirect, mediated effect. Additionally, negative symptoms after 6 months had a significant effect on the severity of depression after 12 months (ß = 0.13, p < 0.05). CONCLUSIONS: Negative symptoms appear to represent an important mechanism of change in IC-TACT indicating that improvement of QoL could potentially be achieved through optimized intervention on negative symptoms. Moreover, this may lead to a reduction in the severity of depression after 12 months.

Health-related quality of life in severe psychotic disorders during integrated care: 5-year course, prediction and treatment implications (ACCESS II).

PURPOSE: Studies on outcomes mapping Quality of Life (QoL) as patient-reported outcome over a longer period in severe psychotic disorders are scarce. However, such data would be particularly important for structuring, implementing and operating effective and efficient care models and for promoting satisfaction with care, service engagement and adherence. METHODS: The ACCESS II study is a prospective long-term study of an integrated care model for people with severe psychotic disorders. The model includes Therapeutic Assertive Community Treatment within a cross-sectoral and interdisciplinary network. This publication analyses the course of QoL assessed with the Q-LES-Q-18 using a mixed model for repeated measures. RESULTS: Mapping the course of QoL in N = 329 participants, there is a significant increase in the first 6 weeks of treatment (early course). Comparison to a published norm show significant lower QoL for severe psychotic disorders. The variable having a traumatic event before the age of 18 was significantly negatively associated with QoL. A decrease in the severity of depressive as well as in positive symptomatology in the first six weeks after admission was associated with increase of QoL. CONCLUSION: Results indicate that the overall symptom burden at time of inclusion is not decisive for the perceived QoL in the long-term course while the reduction in the severity of depressive and positive symptoms is important. This means focusing even more on the treatment of depressive symptoms and include traumatherapeutic aspects in the long-term treatment of severe psychotic disorders if needed. TRAIL REGISTRATION: ClinicalTrials.gov (identifier: NCT01888627).

[Characteristics of psychoses in adolescence-Longitudinal data of integrated care]. / Charakteristika von Psychosen in der Adoleszenz ­ longitudinale Daten der Integrierten Versorgung.

BACKGROUND: Integrated care according to the Hamburg model combines therapeutic assertive community treatment (TACT) with initiatives for early detection and early treatment of schizophrenia and affective psychoses. The aim of this study was to identify the clinical characteristics of adolescents in comparison to adult patients and to derive knowledge for transition-specific treatment approaches. METHODOLOGY: Sociodemographic and clinical variables as well as treatment performance and clinical outcome were investigated over a period of 12 months in 167 patients with psychoses (16-25 years, n = 88; and >25 years, n = 79). RESULTS: Patients with psychosis in adolescence had significantly more outpatient treatment contacts (3.5/week vs. 1.6/week; p < 0.001), while adults were hospitalized for twice as long (10 days vs. 21 days; p = 0.003). The duration of untreated psychoses was significantly shorter in the adolescent group than in adults (122 weeks vs. 208 weeks; p = 0.002). The proportion of comorbid mental disorders was significantly higher in the adolescent group (87% vs. 63%; p < 0.001). In addition, the adolescence patients already showed greater impairment of daily functions and a higher severity of illness at the start of treatment. DISCUSSION: The treatment of psychoses in adolescence was characterized by a particularly high need for flexibility across all sectors and support systems, taking comorbid problem areas into account. Care models for adolescents and young adults with psychoses should therefore combine treatment approaches for severely ill patients with transition psychiatric interventions to avoid breaks in care and to meet the complex requirements of young patients with severe mental illnesses.

Course, remission and recovery in patients with severe psychotic disorders with or without comorbid substance use disorders: Long-term outcome in evidence-based integrated care (ACCESS II study).

OBJECTIVES: People with psychotic disorders have a high prevalence of comorbid mental disorders, especially if severe mental illness (SMI) criteria are fulfilled. Substance Use Disorders (SUD) are the most common comorbidity. The aim of the study is to investigate whether SMI patients with and without comorbid SUD have a comparable course, remission and recovery rates within evidence-based care. METHODS: ACCESS is an integrated care model for patients with severe nonaffective and affective psychotic disorders. Treatment trajectories of patients, who have been in ACCESS care for at least 4 years, with and without SUD were compared with regard to the course of illness using Mixed Model Repeated Measures (MMRM) as well as recovery rates and its predictors. RESULTS: 187 of 312 patients (60%) were at least 4 years in ACCESS. Of these, 126 (67.4%) had a comorbid SUD at admission. Patients had on average 2.96 SUD, 87 (69%) had a dependence. Both groups improved significantly over 4 years in all outcome parameters. However, patients with substance dependence showed significantly worse outcomes in psychopathology (p < 0.001), functioning (p = 0.006) and quality of life (p = 0.026). Using LOCF, 44 patients (23.5%) fulfilled recovery criteria at endpoint. Comorbid substance use dependence was the only significant predictor for non-recovery (OR = 0.462, p = 0.048). CONCLUSION: SUD and especially substance dependence are common in psychotic disorders with SMI. Evidence-based integrated care also leads to long-term improvement in these patients, but to a lesser extent than in patients without SUD. In particular, the "optimal" outcome recovery is made more difficult by SUD dependence.

Study protocol for a randomised controlled trial evaluating an evidence-based, stepped and coordinated care service model for mental disorders (RECOVER).

INTRODUCTION: Healthcare systems around the world are looking for solutions to the growing problem of mental disorders. RECOVER is the synonym for an evidence-based, stepped and cross-sectoral coordinated care service model for mental disorders. RECOVER implements a cross-sectoral network with managed care, comprehensive psychological, somatic and social diagnostics, crisis resolution and a general structure of four severity levels, each with assigned evidence-based therapy models (eg, assertive community treatment) and therapies (eg, psychotherapy). The study rationale is the investigation of the effectiveness and efficiency of stepped and integrated care in comparison to standard care. METHODS AND ANALYSIS: The trial is conducted in accordance to the Standard Protocol Items: Recommendations for Interventional Trials Statement. The study aims to compare the RECOVER model with treatment as usual (TAU). The following questions are examined: Does RECOVER reduce healthcare costs compared with TAU? Does RECOVER improve patient-relevant outcomes? Is RECOVER cost-effective compared with TAU? A total sample of 890 patients with mental disorders will be assessed at baseline and individually randomised into RECOVER or TAU. Follow-up assessments are conducted after 6 and 12 months. As primary outcomes, cost reduction, improvement in symptoms, daily functioning and quality of life as well as cost-effectiveness ratios will be measured. In addition, several secondary outcomes will be assessed. Primary and secondary outcomes are evaluated according to the intention-to-treat principle. Mixed linear or logistic regression models are used with the direct maximum likelihood estimation procedure which results in unbiassed estimators under the missing-at-random assumption. Costs due to healthcare utilisation and productivity losses are evaluated using difference-in-difference regressions. ETHICS AND DISSEMINATION: Ethical approval from the ethics committee of the Hamburg Medical Association has been obtained (PV5672). The results will be disseminated to service users and their families via the media, to healthcare professionals via professional training and meetings and to researchers via conferences and publications. TRIAL REGISTRATION NUMBER AND REGISTRY NAME: ClinicalTrials.gov (NCT03459664), RECOVER PROTOCOL VERSION: 19 March 2020 (V.3.0).

Reduction of Involuntary Admissions in Patients With Severe Psychotic Disorders Treated in the ACCESS Integrated Care Model Including Therapeutic Assertive Community Treatment.

Objective: The ACCESS treatment model offers assertive community treatment (ACT) embedded in an integrated care program to patients with severe psychotic disorders. Compared to standard care, it proved to be more effective in terms of service disengagement and other outcomes in patients with psychotic disorders over 12, 24, and 48 months. Many patients with severe mental disorders experience involuntary admissions which can be potentially traumatic. In this study, we assessed the effect of ACT on reducing involuntary admissions over an observation period of 4 years. Method: One hundred seventy-one patients treated in ACCESS were included in this study. The primary outcome was rate of involuntary admissions during 48 months. Secondary outcomes were differences between those with and without involuntary admissions in the 2 years prior to ACCESS regarding change of psychopathology, severity of illness, psychosocial functioning, quality of life, satisfaction with care, medication non-adherence, and service-disengagement. Results: Of 171 patients, 58 patients (33.9%) were involuntarily admitted to hospital in the past 2 years before entry. During the 4 years of treatment, 16 patients (9.4%) were involuntarily admitted to hospital which was a significantly lower rate compared to the 2 years before inclusion in ACCESS (p < .001). Comparing the two groups, larger improvements in severity of illness (p = .004) and functional status (p = .043) were detected in the group with no history of involuntary admissions. At 4-year follow-up, of the remaining patients, 69.2% (n = 81) were full adherent (p < .001), compared to 18.9% (n = 31) at baseline with no differences between the two groups over the study period (p = .25). Over 4 years, only 13 patients (13.2%) were service-disengaged due to non-practical reasons. Conclusions: In this long-term study, we were able to demonstrate a reduction in involuntary admissions in four treatment years compared to the 2 years prior to admission to the ACCESS model in patients with severe and mostly multiphase schizophrenia spectrum disorders and affective disorders with psychotic features. This may help prevent patients from suffering from a potentially traumatic experience during treatment in the psychiatric system. Clinical Trial Registration:www.ClinicalTrials.gov, identifier NCT01888627.

[Innovative care models for people with schizophrenia]. / Innovative Versorgungsmodelle für Menschen mit schizophrenen Erkrankungen.

Schizophrenia is a complex mental disorder. Hence, the mental health system faces enormous structural, therapeutic, and health-economic challenges. Innovative models of healthcare can facilitate making treatment more efficient and effective.This paper aims to give an overview of evidence-based and innovative models of care and treatment for schizophrenia and severe mental illnesses. For this purpose, we performed a literature search covering the last 10 years of publications regarding "care or treatment models" and "schizophrenia," "psychosis," or "severe mental illness."Many publications do not provide details about the tested care model. Innovative care models for schizophrenia comprise early psychosis services (EPSs) or models for severe mental illnesses (SMI) integrating, combining and/or developing the treatment models crisis resolution team (CRT), assertive community treatment (ACT), and/or (intensive) case management (ICM). For illustration, the innovative models Early Psychosis Prevention and Intervention Centre (EPPIC) in Australia, the Flexible Assertive Community Treatment model in the Netherlands, and the Therapeutic Assertive Community Treatment model in Hamburg are explained. All three models regularly offer integrated, specialized, and assertive care.In Germany, innovative models of care have not been implemented sufficiently: neither EPS nor ACT are part of standard care; CRT can now be funded by health insurances via "ward-equivalent treatment" as defined by a new German law (§§ 39 und 115d SGB V). Regarding the implementation of CRT and ACT in Germany, a clinical study evaluating the RECOVER model is underway. This is a stepped-care, trans-sectorally coordinated and evidence-based treatment model, the evaluation of which is supported by the innovation fund of the Joint Federal Committee (Gemeinsamen Bundesausschuss, G­BA) from 2017 to 2020.

Effectiveness of integrated care including therapeutic assertive community treatment in severe schizophrenia-spectrum and bipolar I disorders: Four-year follow-up of the ACCESS II study.

The ACCESS-model offers integrated care including assertive community treatment to patients with psychotic disorders. ACCESS proved more effective compared to standard care (ACCESS-I study) and was successfully implemented into clinical routine (ACCESS-II study). In this article, we report the 4-year outcomes of the ACCESS-II study. Between May 2007 and December 2013, 115 patients received continuous ACCESS-care. We hypothesized that the low 2-year disengagement and hospitalization rates and significant improvements in psychopathology, functioning, and quality of life could be sustained over 4 years. Over 4 years, only 10 patients disengaged from ACCESS. Another 23 left for practical reasons and were successfully transferred to other services. Hospitalization rates remained low (13.0% in year 3; 9.1% in year 4). Involuntary admissions decreased from 35% in the 2 years prior to ACCESS to 8% over 4 years in ACCESS. Outpatient contacts remained stably high at 2.0-2.4 per week. We detected significant improvements in psychopathology (effect size d = 0.79), illness severity (d = 1.29), level of functioning (d = 0.77), quality of life (d = 0.47) and stably high client satisfaction (d = 0.02) over 4 years. Most positive effects were observed within the first 2 years with the exception of illness severity, which further improved from year 2 to 4. Within continuous intensive 4-year ACCESS-care, sustained improvements in psychopathology, functioning, quality of life, low service disengagement and re-hospitalization rates, as well as low rates of involuntary treatment, were observed in contrast to other studies, which reported a decline in these parameters once a specific treatment model was stopped. Yet, stronger evidence to prove these results is required. TRIAL REGISTRATION: Clinical Trial Registration Number: NCT01888627.

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.

Comorbidity of chronic somatic diseases in patients with psychotic disorders and their influence on 4-year outcomes of integrated care treatment (ACCESS II study).

BACKGROUND: People with psychotic disorders fulfilling criteria of a severe and persistent mental illness (SPMI) display a high risk of somatic comorbidity (SC). METHODS: ACCESS II is a prospective, long-term study examining the effectiveness of Integrated Care for people with psychotic disorders fulfilling SPMI criteria. Chronic comorbid somatic disorders were systematically assessed according to ICD-10-GM criteria. Patients treated for ≥4years in ACCESS were categorized as early psychosis (treatment: ≤2years) or non-early psychosis (treatment: >2years) patients. RESULTS: Of 187 patients treated in ACCESS for ≥4years (mean age=41.8years, males=44.4%), 145 (77.5%) had SC, (mean=2.1±2.1). Overall, 55 different diseases from 15 different ICD-10-GM disease areas were identified. Prevalence of ≥1 SC (p=0.09) and specific types of SC (p=0.08-1.00) did not differ between early and non-early psychosis patients, but non-early psychosis patients had a higher mean number of SC (2.3±2.2 vs. 1.3±1.3, p=0.002). SC patients had higher rates of comorbid mental disorders (93% vs. 81%, p=0.002), specifically posttraumatic stress disorder (23% vs. 7%, p=0.002), and suicide attempts (43% vs. 19%, p<0.001). At the 4-year endpoint, both patients with and without comorbidity displayed major improvements in psychopathology, severity of illness, functioning, quality of life and satisfaction with care. CONCLUSIONS: SC is frequent in patients with severe psychotic disorders, even in the early psychosis phase. The magnitude of the problem underlines the need for regular screening, comprehensive assessment, preventive pharmacotherapy, and targeted SC management.

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).

AIM: 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. METHODS: This is a prospective, single-centre, 1-year cohort study comparing an intervention condition (early detection plus integrated care, n = 120) to the historical control condition (standard care, SC, n = 105) for adolescents and young adults aged 12-29 years suffering from a severe, early-phase psychotic disorder (i.e. within 2 years of treatment). RESULTS: Primary outcome is the rate of combined symptomatic (i.e. Positive and Negative Syndrome Scale (PANSS) criteria) and functional (i.e. Global Assessment of Functioning scale (GAF) ≥ 60 points criterion) remission over at least 6 months at study endpoint. Secondary outcome comprises the comparison of the reduction in the duration of untreated psychosis within the 4-year study duration between integrated care and SC, course of psychopathology, functioning, quality of life, satisfaction with care, cost and quality-adjusted life years (QALYs) in comparison to a historical control group. CONCLUSION: To the authors' knowledge, this is the first study assessing the efficacy and cost-effectiveness of a combined intervention consisting of early detection strategies and strategies to improve quality of care in both adolescents and young adults with early-phase psychosis. The results will be published in 2016.

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.

Explaining parent-child (dis)agreement in generic and short stature-specific health-related quality of life reports: do family and social relationships matter?

BACKGROUND: In the context of health-related quality of life (HrQoL) assessment in pediatric short stature, the present study aimed to examine the levels of agreement/disagreement between parents' and children's reports of generic and condition-specific HrQoL, and to identify socio-demographic, clinical and psychosocial variables associated with the extent and direction of parent-child discrepancies. METHODS: This study was part of the retest phase of the QoLISSY project, which was a multicenter study conducted simultaneously in France, Germany, Spain, Sweden and UK. The sample comprised 137 dyads of children/adolescents between 8 and 18 years of age, diagnosed with growth hormone deficiency (GHD) or idiopathic short stature (ISS), and one of their parents. The participants completed child- and parent-reported questionnaires on generic (KIDSCREEN-10 Index) and condition-specific HrQoL (QoLISSY Core Module). Children/adolescents also reported on social support (Oslo 3-items Social Support Scale) and parents assessed the parent-child relationships (Parental Role subscale of the Social Adjustment Scale) and burden of short stature on parents (QoLISSY- additional module). RESULTS: The parent-child agreement on reported HrQoL was strong (intraclass correlation coefficients between .59 and .80). The rates of parent-child discrepancies were 61.5 % for generic and 35.2 % for condition-specific HrQoL, with the parents being more prone to report lower generic (42.3 %) and condition-specific HrQoL (23.7 %) than their children. The extent of discrepancies was better explained by family and social relationships than by clinical and socio-demographic variables: poorer parent-child relationships and better children's social support were associated with larger discrepancies in generic HrQoL, while more parental burden was associated with larger discrepancies in condition-specific HrQoL reports. Regarding the direction of discrepancies, higher parental burden was significantly associated with parents' underrating, and better children's social support was significantly associated with parents' overrating of condition-specific HrQoL. CONCLUSIONS: Routine assessment of pediatric HrQoL in healthcare and research contexts should include child- and parent-reported data as complementary sources of information, and also consider the family and social context.

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.

Psychometric performance of the Quality of Life in Short Stature Youth (QoLISSY) questionnaire in the Netherlands.

UNLABELLED: The European Quality of Life in Short Stature Youth (QoLISSY) questionnaire is a disease-specific instrument assessing quality of life (QoL) in children with short stature from the child and parent perspectives. In order to use the QoLISSY in Dutch samples, a translation process and psychometric testing is needed. Children diagnosed with short stature (8 to 18 years) and their parents were recruited from a Dutch growth clinic. Reliability was assessed using Cronbach's α and intraclass correlation coefficients (ICCs). Pearsons' correlations with the generic KIDSCREEN and a confirmatory factor analysis (CFA) were performed to test validity. Scales showed good internal consistency with α ranging from 0.80 to 0.94 (child report) and from 0.85 to 0.95 (parent report). Test-retest reliability (ICC) ranged from 0.15 to 0.91 (child report) and from 0.14 to 0.83 (parent report). Correlations with the KIDSCREEN in the mean range indicated criterion validity. The models' goodness of fit was confirmed by CFA results in the Dutch and in comparison with the European sample. CONCLUSION: The Dutch QoLISSY is a psychometrically reliable and valid short stature-specific QoL measure. It is now available for use in clinical research and practice to evaluate well-being and possible effects of growth hormone treatment and psychological interventions in the Netherlands.

[Living with achondroplasia- how do young persons with disproportional short stature rate their quality of life and which factors are associated with quality of life?]. / 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.