Study protocol: longitudinal study of the transition of young people with complex health needs from child to adult health services.

BACKGROUND: Young people with complex health needs have impairments that can limit their ability to carry out day-to-day activities. As well as coping with other developmental transitions, these young people must negotiate the transfer of their clinical care from child to adult services. The process of transition may not be smooth and both health and social outcomes may suffer.Increasingly, policy-makers have recognised the need to ensure a smoother transition between children's and adult services, with processes that are holistic, individualised, and person-centred; however, there is little outcome data to support proposed models of care. This study aims to identify the features of transitional care that are potentially effective and efficient for young people with complex health needs making their transition. METHODS/DESIGN: Longitudinal cohort study. 450 young people aged 14 years to 18 years 11 months (with autism spectrum disorder and an additional mental health problem, cerebral palsy or diabetes) will be followed through their transition from child to adult services and will contribute data at baseline, 12, 24 and 36 months. We will collect data on: health and wellbeing outcomes (participation, quality of life, satisfaction with services, generic health status (EQ-5D-Y) and condition specific measure of disease control or management); exposure to proposed beneficial features of services (such as having a key worker, appropriate involvement of parents); socio-economic characteristics of the sample; use of condition-related health and personal social services; preferences for the characteristics of transitional care.We will us regression techniques to explore how outcomes vary by exposure to service features and by characteristics of the young people. These data will populate a decision-analytic model comparing the costs and benefits of potential alternative ways of organising transition services.In order to better understand mechanisms and aid interpretation, we will undertake qualitative work with 15 young people, including interviews, non-participant observation and diary collection. DISCUSSION: This study will evaluate the effect of service components of transitional care, rather than evaluation of specific models that may be unsustainable or not generalisable. It has been developed in response to numerous national and international calls for such evaluation.

Study protocol: determinants of participation and quality of life of adolescents with cerebral palsy: a longitudinal study (SPARCLE2).

BACKGROUND: Children and adults with impairments such as cerebral palsy have lower participation in life situations than able-bodied people. Less is known about their subjective perception of their lives, called their quality of life.During adolescence, rapid physical and psychological changes occur; although these may be more difficult for disabled than for able-bodied adolescents, little research has examined the lives of disabled adolescents.In 2003-4 a European Union funded project, SPARCLE, visited 818 children aged 8-12 years with cerebral palsy, sampled from population-based registers in nine European regions. The quality of life reported by these disabled children was similar to that of the general population but their participation was lower; levels of participation varied between countries even for children with similar severity of cerebral palsy.We are currently following up these children, now aged 13-17 years, to identify (i) to what extent contemporaneous factors (pain, impairment, psychological health and parental stress) predict their participation and quality of life, (ii) what factors modify how participation and quality of life at age 8-12 years are associated with participation and quality of life in adolescence, and (iii) whether differences between European countries in participation and quality of life can be explained by variations in environmental factors. METHODS/DESIGN: Trained researchers will visit families to administer questionnaires to capture the adolescents' type and severity of impairment, socio-demographic characteristics, participation, quality of life, psychological health, pain, environmental access and parental stress. We will use multivariable models (linear, logistic or ordinal) to assess how adolescent participation, quality of life, psychological health, pain, environmental access and parental stress, vary with impairment and socio-demographic characteristics and, where possible, how these outcomes compare with general population data. For participation and quality of life, longitudinal analyses will assess to what extent these are predicted by corresponding levels in childhood and what factors modify this relationship. Structural equation modelling will be used to identify indirect relationships mediated by other factors.

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, children's pain should be carefully assessed.

Space-time clustering analyses of occurrence of cerebral palsy in Northern England for births 1991 to 2003.

PURPOSE: The etiology of congenital cerebral palsy is unclear. Recent studies have suggested that maternal infection is involved. Indirect, but supportive, evidence for an infectious hypothesis would be provided by the finding of space-time clustering. METHODS: We analyzed all 906 cases born during the period 1991-2003 using a population-based registry from Northern England. We applied the K-function method for testing global space-time clustering and used residential addresses at the time of birth. The Knox method was used to determine the spatio-temporal range over which global clustering occurred. Tests were repeated using nearest neighbor thresholds to allow for variable population density. Additionally, Kulldorff's scan statistic identified specific space-time clusters. RESULTS: There was statistically significant evidence of global space-time clustering (p

Drooling Reduction Intervention randomised trial (DRI): comparing the efficacy and acceptability of hyoscine patches and glycopyrronium liquid on drooling in children with neurodisability.

OBJECTIVE: Investigate whether hyoscine patch or glycopyrronium liquid is more effective and acceptable to treat drooling in children with neurodisability. DESIGN: Multicentre, single-blind, randomised controlled trial. SETTING: Recruitment through neurodisability teams; treatment by parents. PARTICIPANTS: Ninety children with neurodisability who had never received medication for drooling (55 boys, 35 girls; median age 4 years). EXCLUSION CRITERIA: medication contraindicated; in a trial that could affect drooling or management. INTERVENTION: Children were randomised to receive a hyoscine skin patch or glycopyrronium liquid. Dose was increased over 4 weeks to achieve optimum symptom control with minimal side-effects; steady dose then continued to 12 weeks. PRIMARY AND SECONDARY OUTCOMES: Primary outcome: Drooling Impact Scale (DIS) score at week-4. SECONDARY OUTCOMES: change in DIS scores over 12 weeks, Drooling Severity and Frequency Scale and Treatment Satisfaction Questionnaire for Medication; adverse events; children's perception about treatment. RESULTS: Both medications yielded clinically and statistically significant reductions in mean DIS at week-4 (25.0 (SD 22.2) for hyoscine and 26.6 (SD 16) for glycopyrronium). There was no significant difference in change in DIS scores between treatment groups. By week-12, 26/47 (55%) children starting treatment were receiving hyoscine compared with 31/38 (82%) on glycopyrronium. There was a 42% increased chance of being on treatment at week-12 for children randomised to glycopyrronium relative to hyoscine (1.42, 95% CI 1.04 to 1.95). CONCLUSIONS: Hyoscine and glycopyrronium are clinically effective in treating drooling in children with neurodisability. Hyoscine produced more problematic side effects leading to a greater chance of treatment cessation. TRIAL REGISTRATION NUMBERS: ISRCTN 75287237; EUDRACT: 2013-000863-94; Medicines and Healthcare Products Regulatory Agency: 17136/0264/001-0003.

Item response theory and structural equation modelling for ordinal data: Describing the relationship between KIDSCREEN and Life-H.

Both item response theory and structural equation models are useful in the analysis of ordered categorical responses from health assessment questionnaires. We highlight the advantages and disadvantages of the item response theory and structural equation modelling approaches to modelling ordinal data, from within a community health setting. Using data from the SPARCLE project focussing on children with cerebral palsy, this paper investigates the relationship between two ordinal rating scales, the KIDSCREEN, which measures quality-of-life, and Life-H, which measures participation. Practical issues relating to fitting models, such as non-positive definite observed or fitted correlation matrices, and approaches to assessing model fit are discussed. item response theory models allow properties such as the conditional independence of particular domains of a measurement instrument to be assessed. When, as with the SPARCLE data, the latent traits are multidimensional, structural equation models generally provide a much more convenient modelling framework.

Predictors of drop-out in a multi-centre longitudinal study of participation and quality of life of children with cerebral palsy.

BACKGROUND: SPARCLE is a study across nine European regions which examines the predictors of participation and quality of life of children with cerebral palsy. Children and their families were initially interviewed in 2004/2005 when the children were aged 8-12 years (SPARCLE1); they were approached again in 2009/2010 at age 13-17 years (SPARCLE2). The objective of this report is to assess potential for bias due to family non-response in SPARCLE2. Logistic regression was used to assess whether socio-demographic factors, parental stress and child impairment were related to non-response, both overall and by category (failure to trace families, death of child, traced families declining to participate). RESULTS: Of the 818 families who participated in SPARCLE1, 224/818 (27%) did not participate in SPARCLE2. 51/818 (6%) were not traced. Among the 767 traced families, 32/767 (4%) children with cerebral palsy had died, seven children had been incorrectly diagnosed as having cerebral palsy, thirteen families had moved out of the region and one family had language problems. Of the remaining 714 families, 120/714 (17%) declined to participate. Drop-out between SPARCLE1 and SPARCLE2 varied significantly between regions; families were more difficult to trace and more likely to decline to participate if the parents' educational qualifications, as recorded in SPARCLE1, were lower; they were also more likely to decline to participate if SPARCLE1 recorded that they were more stressed or if they had not completed a SPARCLE1 stress questionnaire. CONCLUSIONS: To reduce the risk of bias, all SPARCLE2 analyses should allow for factors (region and walking ability) which determined the sampling strategy, either by adjusting for these factors or by using sampling weights. Further analyses should be performed, adjusting for additional factors that were associated with non-response: parents' educational qualifications, family structure and parental stress. To allow for differential non-response in studies which sample from population registers, such registers should routinely record socio-demographic information.

Access of children with cerebral palsy to the physical, social and attitudinal environment they need: a cross-sectional European study.

PURPOSE: The UN Convention on the Rights of Persons with Disabilities requires states 'to ensure to persons with disabilities access, on an equal basis with others, to the physical environment, transportation, information and communications.' We explored whether this convention was respected for disabled children in Europe. METHOD: One thousand one-hundred and seventy-four children aged 8-12 years were randomly selected from population-based registers of children with cerebral palsy in eight European regions. 743 children joined the study; one further region recruited 75 children from multiple sources. Researchers visited these 818 children and administered the European Child Environment Questionnaire, which records parents' perceptions of availability of the physical, social and attitudinal environment needed in home, school and community. Multilevel, multivariable regression related child access on these domains to their impairments and socio-demographic characteristics. RESULTS: Children with more impaired walking ability had less access to the physical environment, transport and social support they needed than other children. They also experienced less favourable attitudes from family and friends. However, attitudes of teachers and therapists were similar for children with all levels of impairment. The access of children, across all impairment severities, to their needed environment showed significant variation between regions (p ≤ 0.0001), some regions consistently providing better access on most or all domains. CONCLUSION: European states need to substantially improve environmental access for disabled children in order to meet their obligations under UN Conventions. In some regions, many environmental factors should and realistically could be changed. Legislation and regulation should be directed to making this happen. Local environmental planners and health and social service providers should listen carefully to parents to address mismatches between policy intentions and parental experience.

A qualitative study of the health-related quality of life of disabled children.

This qualitative study investigated what disabled children thought most important in their lives and examined how well their priorities are represented in KIDSCREEN, a generic health-related quality of life (HRQoL) instrument. Participants were a subgroup of families who had previously taken part in a study of quality of life and participation in children with cerebral palsy (CP) using KIDSCREEN. This subgroup was sampled purposively, using children's scores on KIDSCREEN and demographic characteristics. Twenty-eight children (15 males, 13 females; age range 8y 3mo-13y 5mo) and 35 parents were interviewed. Ten children were at Gross Motor Function Classification System Level I, 15 were at Levels II or III, and three were at Levels IV or V. Eleven children had unilateral spastic CP, 16 had bilateral spastic C P, and one child had dyskinetic C P. Interviews were audio-recorded and transcribed verbatim. The analysis was based on the constant comparative method and focused largely on the children's data, though the parent data were drawn upon to illuminate the children's data. Four overarching areas of HRQoL were identified: social relationships; home and school environment; self and body; and recreational activities and resources. These generally mapped well to the dimensions and items in KIDSCREEN. The precedence children gave to environmental, social, interpersonal, health, and functional concerns corresponded well with the balance of these items in KIDSCREEN. However, children had some specific priority areas that were not represented in KIDSCREEN. These included: relationships with family members other than parents; inclusion and fairness; home life and neighbourhood; pain and discomfort; environmental accommodation of needs; and recreational resources other than finances and time. We recommend that further consideration be given to inclusion of these areas in the assessment of HRQoL of disabled children.

Severe food-allergic reactions in children across the UK and Ireland, 1998-2000.

AIM: Medical and lay concerns about food allergy are increasing. Whilst food allergy may be becoming more common, fatal reactions to food in childhood are very rare and their rate is not changing. We sought to establish how common severe reactions are. METHODS: Prospective survey, 1998 to 2000, of hospital admissions for food-allergic reactions-conducted primarily through the British Paediatric Surveillance Unit, covering the 13 million children in the United Kingdom and Ireland. RESULTS: 229 cases reported by 176 physicians in 133 departments, yielding a rate of 0.89 hospital admissions per 100,000 children per year. Sixty-five per cent were male, 41% were under 4 y and 60% started at home. Main allergens were peanut (21%), tree nuts (16%), cow's milk (10%) and egg (7%). Main symptoms were facial swelling (76%), urticaria (69%), respiratory (66%), shock (13%), gastrointestinal (4%). Fifty-eight cases were severe. Three were fatal, six near fatal, and 8 of these 9 had asthma with wheeze being the life-threatening symptom. Three near-fatal cases received excess intravenous epinephrine. None of the non-fatal reactions resulted in mental or physical impairment. Seven of 171 non-severe and 6/58 severe cases might have had a worse outcome if epinephrine auto-injectors had been unavailable. Six of the severe cases might have benefited if auto-injectors had been more widely prescribed. CONCLUSION: In the United Kingdom and Ireland, the incidence of severe reactions is low. The study highlights that: asthma is a strongly significant risk factor for a severe reaction and therefore warrants optimal management; severe wheeze is a prominent feature of severe reactions and warrants optimal management; intravenous epinephrine should be used with great care if needed. Epinephrine auto-injectors do not always prevent death, but our study design and data do not allow a definite statement about whether overall they are beneficial.

Participation of children with cerebral palsy is influenced by where they live.

The study aimed to determine whether degree of participation of children with cerebral palsy (CP) is influenced by where they live, as predicted by the social model of disability. Ninety-two per cent children with CP resident in Northern England and born 1991-1996 were entered into the study. Participation was measured by the Lifestyle Assessment Score and its six component domain scores. Regression analysis was used to investigate variations in participation. There were 443 children (265 male, 178 female; mean age 4 years 8 months [SD 1 year 1 month] at time of assessment) in the study. In the regression analysis the following factors remained significant with regard to level of participation: type of CP (167 with hemiplegia, and of those remaining 240 with bilateral spasticity); intellectual impairment (105 with IQ<50, 113 with IQ 50 to 70, and 225 with IQ>70); presence of seizures (115 with active epilepsy); walking disability (114 unable to walk, 81 restricted and needing aids, 186 restricted but unaided, 62 unrestricted); communication problems (61 no formal communication, 51 use alternative formal methods, 126 some delay or difficulty, 205 no communication problems). After adjustment for these factors, there were significant variations with regard to level of participation in the Lifestyle Assessment Score by district of residence. The magnitude of these variations in Lifestyle Assessment Score between districts is similar to that accounted for by severe intellectual impairment. Similar models were obtained for four of the six domain scores. For one of these four, restriction of social interaction, the significant variation between districts was minimally influenced by the underlying type of CP, walking ability, or presence of seizures. Higher levels of participation among children with CP are associated with residence in certain districts. This is not attributable to variations in case-mix or functional capacity of the children. Participation of children with disability is partly a product of their environment.

Identification and description of environmental factors that influence participation of children with cerebral palsy.

Physical, social, and attitudinal environment may restrict participation in children with cerebral palsy (CP). Here we discuss existing/possible approaches in order to identify and describe this environment. We used a critical review of evidence from the World Health Organization Literature Review on Environmental Factors; a search of electronic databases; and talked to specialists in order to find unpublished papers and 'grey' literature. Both children with disabilities and their parents identified a range of barrier and facilitator factors. These included psychosocial pressures (family, school), financial difficulties, and inadequate public services. Observational studies suggest that building structure, loss of income, and provision of specific equipment have a direct impact on levels of child participation. Some available instruments attempt to capture environmental factors by client survey or objective measurement; most relate to adult contexts, but there are a few child-specific instruments for surveying attitudes of children to peers with disabilities and for observation of the school environment. Defining and measuring potential environmental determinants of participation for children with CP needs further development; and here we propose how this might be done.