First-year predictors of health-related quality of life changes in short-statured children treated with human growth hormone.

PURPOSE: Little attention has been directed towards examining the impact of predictors on change in health-related quality of life (HRQOL) within the course of growth hormone (GH) treatment in pediatric short stature. We aimed to assess changes in HRQOL and its sociodemographic, clinical and psychosocial predictors in children and adolescents diagnosed with growth hormone deficiency (GHD), and born short for gestational age (SGA) before and 12-month after start of GH treatment from the parents' perspective. Results were compared with an untreated group with idiopathic short stature (ISS). In this prospective multicenter study, 152 parents of children/adolescents (aged 4-18 years) provided data on their children's HRQOL at baseline and at 12-month follow-up. METHOD: Repeated-measures multivariate analyses of covariance were performed to examine parent-reported HRQOL changes from baseline to 1-year after treatment and hierarchical linear regressions to identify the predictors of HRQOL changes. RESULTS: Results showed that parents of children that were treated with GH report an increase in their children's HRQOL after 1 year. Changes in HRQOL were mostly explained by psychosocial predictors followed by sociodemographic and clinical variables. Specifically, the diagnosis SGA significantly predicted a greater increase in parent-reported HRQOL. Furthermore, a lower caregiving burden significantly predicted a decrease in parent-reported HRQOL. CONCLUSION: In conclusion, a substantial percentage of explained variance in HRQOL relates to psychosocial and sociodemographic predictors. However, there appears to be other important factors that are predictors of HRQOL, which need to be determined in large, population-based samples.

The psychometric evaluation of the quality of life in short stature youth (QoLISSY) instrument for German children born small for gestational age.

PURPOSE: This study aimed to validate the disease-specific "quality of life in short stature youth (QoLISSY)-instrument" that assessesQuery the health-related quality of life (HrQoL) in German children and adolescents diagnosed as small for gestational age (SGA) in a patient and parent report. METHODS: The psychometric performance of the German version of the QoLISSY questionnaire was examined in terms of reliability and validity in 65 SGA families (17 child reports/64 parent reports) and compared to the psychometric performance of the original European QoLISSY dataset of over 200 children with growth hormone deficiency and idiopathic short stature (ISS). RESULTS: The analysis yielded psychometrically favorable results with excellent reliability and acceptable discriminant validity. The instrument's operating characteristics were comparable to the results of the original European QoLISSY data. In the parent- as well as child report, children with SGA had lower HrQoL scores than children with ISS. Convergent validity was demonstrated by significant correlations between the QoLISSY scales and the generic KIDSCREEN-10 Index. CONCLUSION: Psychometric testing suggests that QoLISSY is a promising instrument to assess the HrQoL of young German people with SGA. Both versions (parent- and child report) appear to detect differences between SGA and other conditions (e.g. ISS). QoLISSY can be used in clinical studies, health service research, as well as in practice in children with SGA and their parents. For a cross-cultural application of the instrument in SGA, the tool needs be validated in sufficiently large SGA samples within respective countries.

Validation of the Italian Quality of Life in Short Stature Youth (QoLISSY) questionnaire.

PURPOSE: The Quality of Life in Short Stature Youth (QoLISSY) questionnaire is a disease-specific instrument developed to assess health-related quality of life (HrQoL) in children with short stature. While the original instrument was simultaneously developed in five European countries, this study describes the results of the Italian QoLISSY translation, cultural adaptation, and validation. METHODS: Focus group discussions and a cognitive debriefing process with children (N = 12) diagnosed with growth hormone deficiency or idiopathic short stature and one parent each, as well as parents of younger children (N = 20) were conducted to examine the linguistic and content validity of the Italian version. Psychometric testing was performed using data from the subsequent field- and re-test (N = 32). RESULTS: The results of the qualitative testing of the Italian sample revealed comparability of content to data of the original five European countries. The following field- and re-test results were psychometrically satisfactory including good item and scale operating characteristics, sufficient evidence of reliability, and acceptable evidence of construct validity. CONCLUSION: In conclusion, the Italian QoLISSY HrQoL-dimensions are comparable to other European countries. The psychometric quality of the Italian QoLISSY version is satisfactory and the instrument is ready for use in Italian patients and their parents.

Development and pilot-testing of a condition-specific instrument to assess the quality-of-life in children and adolescents born with esophageal atresia.

The survival rate of children with esophageal atresia has today reached 95%. However, children are at risk of chronic morbidity related to esophageal and respiratory dysfunction, and associated anomalies. This study describes the pilot testing of a condition-specific health-related quality-of-life instrument for children with esophageal atresia in Sweden and Germany, using a patient-derived development approach consistent with international guidelines. Following a literature review, standardized focus groups were conducted with 30 Swedish families of children with esophageal atresia aged 2-17 years. The results were used for item generation of two age-specific pilot questionnaire versions. These were then translated from Swedish into German with considerations of linguistic and semantical perspectives. The 30-item pilot questionnaire for children aged 2-7 years was completed by 34 families (parent report), and the 50-item pilot questionnaire for children aged 8-17 years was completed by 52 families (51 child report, 52 parent report), with an overall response rate of 96% in the total sample. Based on predefined psychometric criteria, poorly performing items were removed, resulting in an 18-item version with three domains (Eating, Physical health and treatment, Social isolation and stress,) for children aged 2-7 years and a 26-item version with four domains (Eating, Social relationships, Body perception, and Health and well-being) for children aged 8-17 years. Both versions demonstrated good internal consistency reliability and acceptable convergent and known-groups validity for the total scores. The study identified specific health-related quality-of-life domains for pediatric patients with esophageal atresia, highlighting issues that are important for follow-up care. After field testing in a larger patient sample, this instrument can be used to enhance the evaluation of pediatric surgical care.

[Evaluation of a Self-Help Supported Counseling Concept for Children and Adolescents with Disproportional Short Stature]. / Evaluation eines selbsthilfegestützten psychosozialen Interventionsangebotes für Kinder und Jugendliche mit disproportionalem Kleinwuchs.

BACKGROUND: Disproportionate short stature may impair the quality of life (QoL) of patients and their families. This study aimed to evaluate a self-help supported counseling concept to increase the QoL of the participants. METHODS: QoL data from 58 children/adolescents (8-17 years) with a diagnosis of achondroplasia was collected at 2 measurement points during one year using the the QoLISSY questionnaire (self-/parental report). Differences before and after participation vs. non-participation in the intervention were evaluated using a linear mixed model. RESULTS: The longitudinal results show a greater increase of QoL in the active intervention group compared to a passive control group (p=0,005). The increase in the self-reported QoL of affected patients was significantly higher than for the parent-report (p=0,048). CONCLUSIONS: The study shows that patients with achondroplasia benefit from a self-help supported counseling concept. However, this should be tested in a randomized trial.

Why the (dis)agreement? Family context and child-parent perspectives on health-related quality of life and psychological problems in paediatric asthma.

BACKGROUND: Children's health-related quality of life (HrQoL) and psychological problems are important outcomes to consider in clinical decision making in paediatric asthma. However, children's and parents' reports often differ. The present study aimed to examine the levels of agreement/disagreement between children's and parents' reports of HrQoL and psychological problems and to identify socio-demographic, clinical and family variables associated with the extent and direction of (dis)agreement. METHODS: The sample comprised 279 dyads of Portuguese children with asthma who were between 8 and 18 years of age (M = 12.13; SD = 2.56) and one of their parents. The participants completed self- and proxy-reported questionnaires on paediatric generic HrQoL (KIDSCREEN-10), chronic-generic HrQoL (DISABKIDS-37) and psychological problems (Strengths and Difficulties Questionnaire). Children's and parents' perceptions of family relationships were measured with the Family Environment Scale and the caregiving burden was assessed using the Revised Burden Measure. RESULTS: The child-parent agreement on reported HrQoL and psychological problems was poor to moderate (intraclass correlation coefficients between 0.32 and 0.47). The rates of child-parent discrepancies ranged between 52.7% (psychological problems) and 68.8% (generic HrQoL), with 50.5% and 31.5% of the parents reporting worse generic and chronic-generic HrQoL, respectively, and 33.3% reporting more psychological problems than their children. The extent and direction of disagreement were better explained by family factors than by socio-demographic and clinical variables: a greater caregiving burden was associated with increased discrepancies in both directions and children's and parents' perceptions of less positive family relationships were associated with discrepancies in different directions. CONCLUSIONS: Routine assessment of paediatric HrQoL and psychological problems in healthcare and research contexts should include self- and parent-reported data as complementary sources of information, and also consider the family context. The additional cost of conducting a more in-depth assessment of paediatric adaptation outcomes can be offset through more efficient allocation of health resources.

[Quality of life in children, adolescents, and young adults with achondroplasia]. / Lebensqualität bei Kindern, Jugendlichen und jungen Erwachsenen mit Achondroplasie.

BACKGROUND: Compared to research on short-statured adults, quality of life (QoL) of children has been rarely studied. One reason for this might be the lack of appropriate disease-specific questionnaires. THE AIM OF THE WORK: The aim of this study was to analyse the quality of life in a sample of short-statured children with achondroplasia, using generic and disease-specific instruments. In addition, a comparison of patient and population norms is presented. MATERIALS AND METHODS: The sample included children (8-28 years) with achondroplasia and parents of participating children (8-17 years). Quality of life was analyzed with the KIDSCREEN, the DISABKIDS and the disease-specific Quality of Life in Short Stature Youth (QoLISSY) questionnaire. In addition group differences according to clinical and sociodemographic data were analyzed within the sample and compared to available KIDSCREEN representative population data. RESULTS: The physical QoL was rated poorly in this sample of short-statured patients, while the emotional QoL was rated more favorably. Compared to the KIDSCREEN population norm, parents of children with achondroplasia rate the QoL lower. DISCUSSION: The QoLISSY questionnaire is a reliable tool to assess the subjective wellbeing of patients with skeletal dysplasia. The instrument can now be used clinically as a screening for patient wellbeing, as an outcome criterion in clinical research and as a psychosocial indicator in orthopedic cohort studies.

[Health-related quality of life: a pivotal endpoint in benefit assessment of medical procedures]. / Gesundheitsbezogene Lebensqualität - ein zentraler patientenrelevanter Endpunkt in der Nutzenbewertung medizinischer Maßnahmen.

BACKGROUND: According to German law, health-related quality of life is a relevant criterion in the early assessment of the benefits of drug treatment. In spite of its regulatory importance, the role of quality of life assessment in quantifying the additional benefits of novel vs standard treatments is not yet clear. OBJECTIVES: The paper is aimed at analyzing the theoretical foundation, the methodological foundation, and the empirical evaluation of the criterion "quality of life" in recent benefit dossiers, as published by the Federal Joint Committee (G-BA) and the German Institute for Quality and Efficiency in Health Care (IQWiG). MATERIALS AND METHODS: Benefit dossiers assessed by the end of 2013 were quantitatively analyzed regarding the choice, frequency of use, and consequences of quality of life assessment. RESULTS: In 59 of the 66 dossiers quality of life assessment was performed, frequently using the EQ-5D (n = 16 procedures) and the SF-36 (n = 8 procedures). Of the 107 evaluations of the subpopulation within these 59 dossiers, in only 2 cases was an additional benefit due to the quality of life declared by the IQWiG and G-BA. CONCLUSIONS: In spite of the acceptance of quality of life measures and their inclusion in the dossiers, they have rarely been the basis for the declaration of additional benefits. This reluctance does not stem from the availability of instruments, but results from problems in their selection, the design and conduct of the study, data interpretation, together with shortcomings regarding the statistical and clinical significance of quality of life data.

Why should we care about quality of life in persons with haemophilia?

The very high cost of haemophilia care, including the increase in use of factor prophylaxis in both children and adults requires that funders of clotting factor concentrates require objective measures of health, such as joint status and quality of life (QOL). Many clinical trials, especially those for licensing of new products, are including QOL instruments in their protocols to evaluate the patients' perspective of wellbeing before and during therapy. This article gives a perspective on QOL the importance of QOL measurement in the field of haemophilia and its impact on patient outcome.

Haemophilia care in Europe: the ESCHQoL study.

The aim of this study was to determine the clinical conditions of patients with haemophilia within Europe as recommended by the European Commission. In this multicentre, cross-sectional, ambispective study, conducted within 21 European countries patients' clinical data were collected, amongst others haemophilia type, severity, treatment pattern, use of factor products, bleeding, orthopaedic joint scores and infections. A total of 1400 patients, 84.3% with haemophilia A and 15.7% with haemophilia B were enrolled by 42 centres between 2004 and 2006. Thereof, 417 were children (30.0%) and 983 were adults (70.0%). About 70% of patients had severe factor deficiency (<1%). More than half of the adults were carriers of chronic infections (12.6% HIV, 55.8% HCV), compared to only 3.8% children (no HIV, 2.9% HCV). Patients were grouped according to per capita amount of clotting factor used in patients' region of residence in 2005: region 1: >5 IU; region 2: 2-5 IU; region 3: <2 IU. Paediatric and adult patients in region 3 had median numbers of three and eight joint bleeds, respectively, with worse joint scores compared to region 1 with zero and one bleed. Prophylactic therapy was used in only 31.3% children and 8.9% adults with severe haemophilia in region 3 compared to 93.7% and 54.1%, respectively, in region 1. Statistical analysis revealed that residence in areas with low factor consumption/availability is the most prominent risk factor for joint disease. Access of European patients with haemophilia to optimal care with safe factor VIII concentrates is limited and depends on the region of residence.

[Hypertension, dyslipoproteinemia and BMI-category characterise the cardiovascular risk in overweight or obese children and adolescents: data of the BZgA-observational study (EvAKu-J-project) and the KiGGS-study]. / Hypertonie, Dyslipoproteinämie und BMI-Kategorie charakterisieren das kardiovaskuläre Risiko bei übergewichtigen oder adipösen Kindern und Jugendlichen: Daten der BZgA-Beobachtungsstudie (EvAKuJ-Projekt) und der KiGGS-Studie.

BACKGROUND: The considerable increase of obesity in children and adolescents poses a major challenge to the health care system. METHODS: In an observation study of the Bundeszentrale für gesundheitliche Aufklärung (BZgA) somatic data of 1916 overweight and obese children and adolescents aged 8-17 years were compared to data of 7 451 normal weight children and adolescents (KiGGS). Age, sex, body weight, height, BMI-SDS, blood pressure, and lipids were analyzed. Body weight was assessed using the BMI categories of the Arbeitsgemeinschaft Adipositas im Kindes- und Jugendalter (AGA) guidelines. Blood pressure measurements were given as above 95 (th) percentile and categorized according to the classification of the European Society of Hypertension (ESH). In addition blood pressure in BZgA-patients were estimated as above 95 (th) percentile by age, sex and height in German normal weight children and adolescents. Lipid values were evaluated according to American Heart Association specifications. RESULTS: Out of the participants of BZgA-study 14% were overweight, 48% obese, and 38% extremely obese. Blood pressure values were above the 95 (th) percentile (ESH) in 35%. The blood pressure in normal weight participants of the KiGGS-study were elevated in 5%. Total cholesterol of BZgA-patients was elevated in 13%, LDL-cholesterol was elevated in 13%, HDL-cholesterol was low in 7%, and triglycerides in the fasting state were elevated in 12%. CONCLUSIONS: The rising prevalence of cardiovascular risk factors in children and adolescents with increasing BMI category requires effective strategies for prevention and treatment of obesity.

[Psychological comorbidity in children and adolescents with chronic somatic diseases]. / Psychische Komorbidität bei chronisch somatischen Erkrankungen im Kindes- und Jugendalter.

This article analyzes emotional and behavioral problems in children and adolescents with chronic somatic disorders. Within the German Health Interview and Examination Survey for Children and Adolescents (Kinder- und Jugendgesundheitssurvey, KIGGS), chronic somatic conditions and obesity were assessed in 11,529 children and adolescents aged 7-17 years old. Special health care needs (CSHCN), emotional and behavioral problems (SDQ), as well as personal, familial, and social resources were surveyed. About 10.8% of the respondents displayed special health care needs and declared a chronic somatic disorder. Of these cases, 20.6% were classified as abnormal in the SDQ (non-somatic conditions: 6.4%). In a logistic regression analysis, male gender (OR=2.0), low socioeconomic status (Winkler index; OR=2.6), family structure (OR>1), and deficits in familial (OR=2.4) and personal (OR=2.1) resources were found to be significantly associated with psychological comorbidity in chronic somatic conditions. The results confirmed previous findings. Especially socioeconomic, structural, and functional aspects of a family have to be considered in the development and prevention of psychological comorbidity in chronic somatic conditions in childhood and adolescence.

[Overweight and obesity in childhood and adolescence. Evaluation of inpatient and outpatient care in Germany: the EvAKuJ study]. / Übergewicht und Adipositas in Kindheit und Jugend. Evaluation der ambulanten und stationären Versorgung in Deutschland in der "EvAKuJ-Studie".

Numerous forms of therapy exist for the increasing number of obese children and adolescents in Germany, but these are heterogeneous and have not been evaluated. Access to health care, long- and short-term treatment outcome, as well as factors determining success of therapy were examined for the first time using standardized instruments to measure somatic and psychosocial variables. A total of 1,916 children aged 8-16 years from 48 (5 rehabilitation, 43 outpatient) institutions were examined. Data were collected for height, weight, blood pressure, and lipid status before treatment started (t0), at the end of treatment (t1), and 1 year after completion of treatment (t2). Furthermore, psychosocial variables were documented using questionnaires for parents and children. The mean BMI-SDS (body mass index standard deviation score) reduction at t1 was -0.27 and 1 year later at t2 was -0.23 (per protocol analysis; intention to treat: t1=-0.24; t2=-0.06). Psychological health and quality of life, which were markedly impaired at the beginning, improved. However, physical activity, media consumption, and nutrition remained basically unchanged. A reduction in weight is associated with an improvement in cardiovascular risk profiles, and long-term behavior changes are possible. However, the institutions differed considerably in the percentage of follow-up examinations and in the weight reduction accomplished.

[Evaluation of obesity treatment in children and adolescents (EvAKuJ Study). Role of therapeutic concept, certification, and quality indicators]. / Evaluation von Therapieangeboten für adipöse Kinder und Jugendliche (EvAKuJ-Projekt). Welche Rolle spielen Behandlungskonzept, AGA-Zertifizierung und initiale Qualitätsangaben?

AIM: Different providers of obesity treatment in children and adolescents in Germany were compared using the following criteria: outpatient/inpatient; with/without AGA certification; good/less good quality. METHODS: A total of 1,916 patients (8-16.9 years) from 48 study centers were examined before (t0), after (t1), and at least 1 year after therapy (t2/3). Body mass index (BMI), blood pressure, blood lipids, and psychosocial data were measured. RESULTS: Patients from inpatient rehabilitation centers were older and more obese. Patients from AGA-certified centers were more obese, and the completeness of comorbidity screening was higher. There were no differences in short- or long-term BMI reduction. "Good" treatment centers (classified after the UKE study 2004) did not differ from those centers not rated as "good" in weight reduction. Patients treated in "good" centers were more obese, and screening for comorbidity was better. No differences in drop out and loss to follow-up were found. CONCLUSION: There were only small differences between the different groups. Pronounced differences were found between the individual treatment centers. In order to improve therapy processes and outcomes, benchmarking and quality management have to be extended.

[Treatment of obesity in pediatric patients in Germany: anthropometry, comorbidity and socioeconomic gradients based on the BZgA Observational Study]. / Beobachtungsstudie der BZgA zur Adipositastherapie bei Kindern und Jugendlichen in Deutschland: Anthropometrie, Komorbidität und Sozialstatus.

BACKGROUND: In recent years, the prevalence of overweight and obesity in children and adolescents has markedly increased. Even though numerous treatment options are available for patients, these are heterogeneously structured and insufficiently evaluated. PATIENTS: In this initial report of specific therapy under routine care circumstances, data from 1916 patients aged 8-16.9 years were analyzed. Patients were characterized based on cluster of therapy, sociodemographic, psychosocial and medical features. Furthermore, the quality of care procedures with regard to the diagnosis of comorbidities was analyzed. RESULTS: At initial medical examination mean patient BMI-SDS was +2.43 and mean age was 12.6 years. 43.3% of patients were male. The mean duration of treatment was 6.1 months (55% outpatient, 45% inpatient care setting). 14% of patients were overweight, 48% obese and 37% were extremely obese. In 51% of the patients we could find already comorbidities. Parental BMI was related to patient's BMI. Socioeconomic status was reduced compared to general population. CONCLUSION: In this nationwide, pioneer multicentre observational study initiated by Federal Centre for Health Education (BZgA) in Germany, different therapeutic approaches were compared by examining somatic and psychosocial variables. The short- and long-term effects of different weight reduction programs on BMI-SDS, nutritional- and physical activity habits as well as quality of life and comorbidity will be examined at the end of therapy. Two follow-up assessments are planned for one and two years after the intervention ended.

Medical care of overweight children under real-life conditions: the German BZgA observation study.

OBJECTIVE: Current care for overweight children is controversial, and only few data are available concerning the process of care, as well as the outcome under real-life conditions. METHODS: A nationwide survey of treatment programs for overweight children and adolescents in Germany identified 480 treatment centers. From 135 institutions that had agreed to participate in this study of process of care and outcome, 48 randomly chosen institutions were included in the study. All 1916 overweight children (mean age 12.6 years, 57% female, mean body mass index 30.0 kg/m(2)), who presented at these institutions for lifestyle interventions, were included in this study. Diagnostic procedures according to guidelines and effect of lifestyle interventions on weight status at end of treatment were analyzed. RESULTS: Children treated <3 months were older and more obese, whereas children with >3 months treatment duration demonstrated more cardiovascular risk factors at baseline. On the basis of an intention-to-treat analysis, 75% of the children reduced their overweight. The reduction of overweight varied widely between the treatment institutions (intracluster correlation coefficient 0.15 in the multiple regression model reflecting the intracenter correlation). Screening for hypertension, disturbed glucose metabolism and dyslipidemia was performed in 52% of the children at baseline and in 10% at the end of intervention. CONCLUSION: Overweight reduction is achievable with lifestyle intervention in clinical practice. However, because the clientele, treatment approach and outcome varied widely between different institutions, and screening for comorbidities was seldomly performed as recommended, quality criteria for institutions have to be implemented to improve medical care of overweight children under real-life conditions.

Measuring patient-reported outcomes in haemophilia clinical research.

Patient-reported outcome (PRO) measures have been used to assess quality of life and health state preferences from the patient's perspective. However, they have not been fully utilized in haemophilia clinical practice and research. A series of meetings were convened to review and document the state of the art in PROs relevant to haemophilia. Experts developed a process for selection of measures and identified published measures of health-related quality of life (HRQoL) relevant to patients with haemophilia. These were synthesized and reviewed. Patient preference measures were also identified and reviewed. Although the majority of measures were developed for and validated in adults, several measures were identified for use in paediatric populations. This paper recommends an approach to the selection of PROs for application in haemophilia clinical research and practice and identifies several potential measures relevant for application in haemophilia clinical research and practice.

Influence of educational programs on attitudes of medical students towards psychiatry: Effects of psychiatric experience, gender, and personality dimensions.

BACKGROUND: Attitudes of medical students form the basis for medical actions. Because of the specific characteristics of psychiatric patients, positive attitudes of medical students towards psychiatry should be a higher goal in medical education. AIM: We hypothesize that medical students in different educational programs develop different attitudes towards psychiatry. METHODS: In a cross-sectional study, students enrolled in different educational programs completed the 'attitudes towards psychiatry' questionnaire (ATP-30). Data concerning experiences in psychiatry, personality traits and socio-demographic variables including gender were also analyzed. RESULTS: The response rate of students in the PBL-curriculum (n = 61) was >90%, in the traditional curriculum (n = 280) >75%. Attitudes towards psychiatry of male students in the Problem-Based Learning program were equal to the female students' attitudes in both programs. Female students' attitudes in the traditional curriculum reached comparably good results while male students' displayed the worst attitudes. The personality factors 'openness to experience' and 'agreeableness' correlated significantly with positive attitudes towards psychiatry. PBL-students showed significantly more 'openness to experience'. CONCLUSION: Educational programs might play a role for the development of attitudes towards psychiatry, especially in male students. Factors influencing enrollment into special educational programs should also have been taken into account. An independent study with a larger number of participants will be required to support these findings.

Cross-cultural development and psychometric evaluation of a patient-reported health-related quality of life questionnaire for adults with haemophilia.

Co-morbidities of haemophilia, such as arthropathy and blood-borne infections, can adversely affect the quality of life of adult patients with haemophilia. The purpose of this study was to develop and validate a haemophilia-specific health-related quality of life questionnaire for adults (HAEMO-QoL-A). Subjects with varying severities of haemophilia completed the HAEMO-QoL-A at baseline and 4 weeks. Other assessments included the SF-36 and Health Assessment Questionnaire - Functional Disability Index (HAQ-FDI). Two-hundred and twenty-one participants completed the 41-item HAEMO-QoL-A covering six domains (Physical Functioning, Role Functioning, Worry, Consequences of Bleeding, Emotional Impact and Treatment Concerns) and four independent items. Internal consistency was good-to-excellent (Cronbach's alpha-range: 0.75-0.95). Test-retest reproducibility was good, with intraclass correlation coefficients >0.80 except for the Emotional Impact domain (0.79). Concurrent validity between the HAEMO-QoL-A total and subscale scores and all SF-36 subscale scores were generally good (correlations range: 0.13-0.87). Significant correlations between the HAEMO-QoL-A and the HAQ-FDI ranged from -0.14 to -0.69. There were non-significant correlations with the Treatment Concerns subscale and with the Worry subscale. The HAEMO-QoL-A discriminated significantly between adults with haemophilia by severity and HIV status. The Physical Functioning subscale discriminated between patients receiving prophylactic or on-demand therapy. The HAEMO-QoL-A is a valid and reliable instrument for assessing quality of life in haemophilia patients.

Healthcare needs and healthcare satisfaction from the perspective of parents of children with chronic conditions: the DISABKIDS approach towards instrument development.

AIM: Increasingly, families' perspectives are taken into account in the appraisal of health services. The objective of this study was to cross-culturally analyse concepts related to healthcare needs, healthcare utilization and the appraisal and satisfaction with care of parents of children with chronic conditions with the aim of developing a cross-cultural measure. METHODS: Several approaches were employed in the study: (i) a deductive approach integrating existing measurements; and (ii) an inductive approach based on focus groups. Focus groups were conducted in seven countries with mothers and fathers as well as their children with seven different chronic conditions, and qualitatively analysed. RESULTS: As a result of an evaluation of the different existing methodological approaches, the basic structural components were identified: healthcare needs, the receipt of services, problems with receiving services as well as the appraisal of and satisfaction with the quality of care. While items referring to existing healthcare services were primarily derived by the work of an expert group, items related to quality of care and satisfaction with services mainly evolved from the focus group work. From the focus groups, 367 statements were extracted, which were further processed in a Q-sort rating by a multinational expert group in order to identify domains and salient items. The draft questionnaire to be pilot tested cross-nationally consisted of 101 items which were reduced on the basis of psychometric findings. CONCLUSION: On the basis of results of focus groups and existing evidence, a comprehensive measure should be employed in paediatric health services research including structural, process and outcome parameters of care from the perspective of parents.