Ute Thyen

Self-reported quality of life of 8–12-year-old children with cerebral palsy: a cross-sectional European study

BACKGROUND Little is known about the quality of life (QoL) of disabled children. We describe self-reported QoL of children with cerebral palsy, factors that influence it, and how it compares with QoL of the general population. METHODS 1174 children aged 8-12 years were randomly selected from eight population-based registers of children with cerebral palsy in six European countries and 743 (63%) agreed to participate; one further region recruited 75 children from multiple sources. Researchers visited these 818 children. 318 (39%) with severe intellectual impairment could not self-report; 500 (61%) reported their QoL using KIDSCREEN, an instrument with scores in ten domains, each with SD=10. Multivariable regression was used to relate QoL to impairments, pain, and sociodemographic characteristics. Comparisons were made with QoL data from the general population. FINDINGS Impairments were not significantly associated with six KIDSCREEN domains. Comparison of least and most able groups showed that severely limited self-mobility was significantly associated with reduced mean score for physical wellbeing (7.6, 95% CI 2.7-12.4); intellectual impairment with reduced mean for moods and emotions (3.7, 1.5-5.9) and autonomy (3.3, 0.9-5.7); and speech difficulties with reduced mean for relationships with parents (4.5, 1.9-7.1). Pain was common and associated with lower QoL on all domains. Impairments and pain explained up to 3% and 7%, respectively, of variation in QoL. Children with cerebral palsy had similar QoL to children in the general population in all domains except schooling, in which evidence was equivocal, and physical wellbeing, in which comparison was not possible. INTERPRETATION Parents can be reassured that most children aged 8-12 years with cerebral palsy will have similar QoL to other children. This finding should guide social and educational policy to ensure that disabled children participate fully in society. Because of its association with QoL, childrens pain should be carefully assessed.

Determinants of child-parent agreement in quality-of-life reports: a European study of children with cerebral palsy.

OBJECTIVES. The differences between child self-reports and parent proxy reports of quality of life in a large population of children with cerebral palsy were studied. We examined whether child characteristics, severity of impairment, socioeconomic factors, and parental stress were associated with parent proxy reports being respectively higher or lower than child self-reports of quality of life. METHODS. This study was conducted in 2004–2005 and assessed child quality of life (using the Kidscreen questionnaire, 10 domains, each scored 0–100) through self-reports and parent proxy reports of 500 children aged 8 to 12 years who had cerebral palsy and were living in 7 countries in Europe. RESULTS. The mean child-reported scores of quality of life were significantly higher than the parent proxy reports in 8 domains, significantly lower for the finances domain, and similar for the emotions domain. The average frequency of disagreement (child-parent difference greater than half an SD of child scores) over all domains was 64%, with parents rating their childs quality of life lower than the children themselves in 29% to 57% of child-parent pairs. We found that high levels of stress in parenting negatively influenced parents’ perception of their childs quality of life, whereas the main factor explaining parents’ ratings of childrens quality of life higher than the children themselves is self-reported severe child pain. CONCLUSIONS. This study shows that the factors associated with disagreement are different according to the direction of disagreement. In particular, parental well-being and child pain should be taken into account in the interpretation of parent proxy reports, especially when no child self-report of quality of life is available. In the latter cases, it may be advisable to obtain additional proxy reports (from caregivers, teachers, or clinicians) to obtain complementary information on the childs quality of life.

Participation in life situations of 8-12 year old children with cerebral palsy: cross sectional European study

Objectives To evaluate how involvement in life situations (participation) in children with cerebral palsy varies with type and severity of impairment and to investigate geographical variation in participation. Design Cross sectional study. Trained interviewers visited parents of children with cerebral palsy; multilevel multivariable regression related participation to impairments, pain, and sociodemographic characteristics. Setting Eight European regions with population registers of children with cerebral palsy; one further region recruited children from multiple sources. Participants 1174 children aged 8-12 with cerebral palsy randomly selected from the population registers, 743 (63%) joined in the study; the further region recruited 75 children. Main outcome measure Children’s participation assessed by the Life-H questionnaire covering 10 main areas of daily life. Scoring ignored adaptations or assistance required for participation. Results Children with pain and those with more severely impaired walking, fine motor skills, communication, and intellectual abilities had lower participation across most domains. Type of cerebral palsy and problems with feeding and vision were associated with lower participation for specific domains, but the sociodemographic factors examined were not. Impairment and pain accounted for up to a sixth of the variation in participation. Participation on all domains varied substantially between regions: children in east Denmark had consistently higher participation than children in other regions. For most participation domains, about a third of the unexplained variation could be ascribed to variation between regions and about two thirds to variation between individuals. Conclusions Participation in children with cerebral palsy should be assessed in clinical practice to guide intervention and assess its effect. Pain should be carefully assessed. Some European countries facilitate participation better than others, implying some countries could make better provision. Legislation and regulation should be directed to ensuring this happens.

Epidemiology and Initial Management of Ambiguous Genitalia at Birth in Germany

Background: In this epidemiological study, we sought to capture the incidence of ambiguous genitalia in neonates and to describe initial management strategies. Methods: We used the registry for rare diseases in pediatrics in Germany to ascertain cases and asked reporting institutions for information on phenotype, laboratory tests, imaging results, diagnosis, initial management and sex assignment. Results: We identified 80 cases within a 2-year study period and calculated an incidence of 2 per 10,000 births with ambiguous genitalia per year in Germany. Prevalence was higher in infants from non-German family background. In more than 50% of all infants a definite diagnosis was lacking even at the age of 6 months. In those cases where the etiology was confirmed, the most common diagnosis was congenital adrenal hyperplasia, followed by androgen insensitivity syndrome and mixed gonadal dysgenesis. Associated malformations were very common, affecting 37.5% of all infants. Sex assignment was female in 46,XY infants with predominately female phenotype and all 46,XX infants. Early surgery was performed in many cases irrespective whether a definite diagnosis had been established or not. Integrated psychosocial care was the exception rather than the rule. Conclusions: Classification and management of ambiguous genitalia at birth remain a challenge for all professionals involved. National and international registries may help to provide a better understanding of the incidence and clinical course of such disorders.

Summary of Consensus Statement on Intersex Disorders and Their Management

Advances in understanding of genetic control of sexual determination and differentiation, improvements in diagnostic testing and surgical genital repair, and the persistent controversies inherent to clinical management were all compelling factors that led to the organization of an international consensus conference. The goals were to acknowledge and discuss the more controversial issues in intersex management, provide management guidelines for intersex patients, and identify and prioritize questions that need additional investigation. This is a summary statement. Advances in molecular genetic causes of abnormal sexual development and heightened awareness of the ethical and patient-advocacy issues mandate reexamination of existing nomenclature for patients with intersex.1 Terminology such as “pseudohermaphroditism” is controversial, potentially pejorative to patients,2 and inherently confusing. Therefore, the term “disorders of sex development” (DSD) is proposed to indicate congenital conditions with atypical development of chromosomal, gonadal, or anatomic sex. Additional rationale for new classification is the need for modern categorization to integrate the modern molecular genetic aspects, to maximize precision when applying definitions and diagnostic labels,3 and to meet the need for psychologically sensitive yet descriptive medical terminology. Nomenclature should be flexible enough to incorporate new information, robust enough to maintain a consistent framework, use descriptive terms, reflect genetic etiology, accommodate phenotypic variation spectrum, and be useful for clinicians, scientists, patients, and families. Hence, we propose a new classification (see “Consensus Statement on Management of Intersex Disorders”4 in this months issue of Pediatrics Electronic Edition ). Three traditionally conceptualized domains of psychosexual development are gender identity (ones self-representation [ie, male or female]), gender role (sexually dimorphic behaviors within the general population, such as toy preferences, aggression, and spatial ability), and sexual orientation (direction[s] of erotic interest). Gender dissatisfaction denotes unhappiness with assigned sex and may result in gender self-reassignment. Psychosexual developmental factors relate to parental psychopathology, parent-child … Address correspondence to Peter A. Lee, MD, PhD, Department of Pediatrics, MC-H085, Penn State College of Medicine, Milton S. Hershey Medical Center, Box 850, 500 University Dr, Hershey, PA 17033-0850. E-mail: plee{at}psu.edu

Parent-Reported Quality of Life of Children With Cerebral Palsy in Europe

OBJECTIVE. The goal was to determine whether the type and severity of the childs impairments and the familys psychosocial, social, and economic characteristics influence parent-reported child quality of life across the spectrum of severity of cerebral palsy. METHODS. Our population-based, cross-sectional survey conducted in 2004 to 2005 involved 818 children with cerebral palsy, 8 to 12 years of age, from 7 countries (9 regions) in Europe. Child quality of life was assessed through parent reports by using the Kidscreen questionnaire, and data were analyzed separately for each of its 10 domains. RESULTS. The parental response rates were >93% for all domains except one. Gross motor function and IQ level were found to be associated independently with quality of life in most domains. However, greater severity of impairment was not always associated with poorer quality of life; in the moods and emotions, self-perception, social acceptance, and school environment domains, less severely impaired children were more likely to have poor quality of life. Pain was associated with poor quality of life in the physical and psychological well-being and self-perception domains. Parents with higher levels of stress were more likely to report poor quality of life in all domains, which suggests that factors other than the severity of the childs impairment may influence the way in which parents report quality of life. CONCLUSIONS. The parent-reported quality of life for children with cerebral palsy is associated strongly with impairment. However, depending on the areas of life, the most severely impaired children (in terms of motor functioning or intellectual ability) do not always have the poorest quality of life.

Ethical principles and recommendations for the medical management of differences of sex development (DSD)/intersex in children and adolescents

The medical management of differences of sex development (DSD)/intersex in early childhood has been criticized by patients’ advocates as well as bioethicists from an ethical point of view. Some call for a moratorium of any feminizing or masculinizing operations before the age of consent except for medical emergencies. No exhaustive ethical guidelines have been published until now. In particular, the role of the parents as legal representatives of the child is controversial. In the article, we develop, discuss, and present ethical principles and recommendations for the medical management of intersex/DSD in children and adolescents. We specify three basic ethical principles that have to be respected and substantiate them. The article includes a critical discussion of the best interest of the child and of family privacy. The argumentation draws upon recommendations by the working group “Bioethics and Intersex” within the German Network DSD/Intersex, which are presented in detail. Unlike other recommendations with regard to intersex, these guidelines represent a comprehensive view of the perspectives of clinicians, patients, and their families.ConclusionThe working group identified three leading ethical principles that apply to DSD management: (1) to foster the well-being of the child and the future adult, (2) to uphold the rights of children and adolescents to participate in and/or self-determine decisions that affect them now or later, and (3) to respect the family and parent–child relationships. Nine recommendations for the management of DSD indicate how these ethical principles can spelled out and balanced against each other in the clinical setting.

Frequency of participation of 8-12-year-old children with cerebral palsy: A multi-centre cross-sectional European study

Participation in home, school and community is important for all children; and little is known about the frequency of participation of disabled children. Frequency of participation is a valuable outcome measure for evaluating habilitation programmes for disabled children and for planning social and health services. We investigated how frequency of participation varied between children with cerebral palsy and the general population; and examined variation across countries to understand better how the environmental factors such as legislation, public attitudes and regulation in different countries might influence participation. We undertook a multi-centre, population-based study in children with and without cerebral palsy. Working from the Life-H instrument, we developed a questionnaire to capture frequency of participation in 8-12-year-old children. In nine regions of seven European countries, parents of 813 children with cerebral palsy and 2939 children from the general populations completed the questionnaire. Frequency of participation for each question was dichotomised about the median; multivariable logistic regressions were carried out. In the general population, frequency of participation varied between countries. Children with cerebral palsy participated less frequently in many but not all areas of everyday life, compared with children from the general population. There was regional variation in the domains with reduced participation and in the magnitude of the differences. We discuss how this regional variation might be explained by the different environments in which children live. Attending a special school or class was not associated with further reduction in participation in most areas of everyday life.

Age, metabolic control and type of insulin regime influences health-related quality of life in children and adolescents with type 1 diabetes mellitus

The effects of illness and treatment of diabetes mellitus extend beyond medical outcomes. We therefore evaluated health-related quality of life (HRQOL) in children (aged 8–12 years) and adolescents (aged 13–16 years) with type 1 diabetes to compare their results with healthy peers and to identify HRQOL determinants. A total of 68 children and adolescents from a tertiary care clinic which specialises in the management of diabetes, completed the generic KINDL-R questionnaire. This instrument for children and adolescents has six dimensions and an additional module assessing condition-related HRQOL. Overall, the HRQOL was not different between patients with type 1 diabetes and healthy controls. In some areas, children and adolescents with diabetes reported a better HRQOL compared to healthy peers: adolescents reported better psychological well-being ( P <0.05) and children higher levels of well-being in the school domain ( P <0.05). In general, children reported a better HRQOL ( P <0.05) than adolescents with type 1 diabetes confirming age-related differences in HRQOL in the general population. Lower HbA1c (<8%) and intensified insulin therapy (>3 injections/day) were associated with a better HRQOL in different domains ( P <0.05). The subscale “chronic illness” showed a better HRQOL ( P <0.001) in children and adolescents with diabetes compared to age-matched controls with other chronic conditions. Conclusion:Children and adolescents from a paediatric department specialising in diabetes management report good health-related quality of life. Younger age, good metabolic control and intensified insulin therapy are associated with a better health-related quality of life. Dimensions of health-related quality of life appear to play different roles at different ages, emphasising the importance of the multidimensional health-related quality of life concept and the value of age-appropriate self-reports.

Association between participation in life situations of children with cerebral palsy and their physical, social, and attitudinal environment: A cross-sectional multicenter European study

OBJECTIVE To evaluate how participation of children with cerebral palsy (CP) varied with their environment. DESIGN Home visits to children. Administration of Assessment of Life Habits and European Child Environment Questionnaires. Structural equation modeling of putative associations between specific domains of participation and environment, while allowing for severity of childs impairments and pain. SETTING European regions with population-based registries of children with CP. PARTICIPANTS Children (n=1174) aged 8 to 12 years were randomly selected from 8 population-based registries of children with CP in 6 European countries. Of these, 743 (63%) agreed to participate; 1 further region recruited 75 children from multiple sources. Thus, there were 818 children in the study. INTERVENTIONS Not applicable. MAIN OUTCOME MEASURE Participation in life situations. RESULTS For the hypothesized associations, the models confirmed that higher participation was associated with better availability of environmental items. Higher participation in daily activities-mealtimes, health hygiene, personal care, and home life-was significantly associated with a better physical environment at home (P<.01). Mobility was associated with transport and physical environment in the community. Participation in social roles (responsibilities, relationships, recreation) was associated with attitudes of classmates and social support at home. School participation was associated with attitudes of teachers and therapists. Environment explained between 14% and 52% of the variation in participation. CONCLUSIONS The findings confirmed the social model of disability. The physical, social, and attitudinal environment of disabled children influences their participation in everyday activities and social roles.