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Scales currently validated to assess severity of chronic physical conditions in children are not well suited for limited-resource settings as they lack ease of use and interpretation. This study assessed the validity of the Global Assessment of Severity of Illness (GASI), a single-item scale designed for quick and simple assessment of illness severity in children with various chronic physical conditions. Study objectives were to examine validity, reliability, and responsiveness of the GASI. Clinicians assessed the severity of asthma, food allergy, epilepsy, diabetes, and juvenile arthritis in 55 children, and parents reported on children’s health-related quality of life. Area under the curve (AUC) computed by logistic regression and Kendall’s Tau-c (τc) assessed the strength of association between the GASI and other study measures. The kappa coefficient (κ), weighted kappa (κw), and McNemar’s test assessed stability in GASI ratings over time. The standardized response mean and Guyatt’s responsiveness index examined internal and external responsiveness of the GASI, respectively. The GASI correlated strongly with established severity scales (AUC = 0.83–0.96; τc = 0.57–0.78) and did not correlate with health-related quality of life (τc

Background With increasing psychiatric hospitalizations among adolescents and constrained hospital resources, there are times when youth are hospitalized in adult inpatient psychiatry units. Evidence on the prevalence of this practice and associated impacts is lacking. Aims We sought to explore the prevalence, determinants, and outcomes related to the hospitalization of adolescents aged 12-17 years on adult inpatient psychiatry units in Ontario. Methods Using health administrative data, we constructed a cohort of adolescents with an inpatient psychiatric admission in Ontario (2007-2011). We classified adolescents as having an admission to an adult psychiatry unit or to other inpatient units. Multivariable regression models were used to estimate prevalence ratios (PR) for factors associated with adult admission, as well as risk ratios (RR) for the impact of adult admission on length of stay, discharge against medical advice, and 30-day readmission. Results Over the study period, 22.6% of adolescents with a psychiatric hospitalization (n = 16 998) had an admission to an adult psychiatry unit. Older age (16 vs. 15 years: PR = 2.27, 95% CI = 2.07-2.48; 17 vs. 15 years: PR = 2.91, 95% CI = 2.66-3.18), rural residence (PR = 1.46, 95% CI = 1.38-1.55), psychotic (PR = 1.25, 95% CI = 1.15-1.36) or personality disorder (PR = 1.59, 95% CI = 1.41-1.80) diagnoses, and involuntary status (PR = 2.18, 95% CI = 2.05-2.31) were independently associated with adult admission. Adolescents admitted to adult units were more likely to be discharged against medical advice (RR = 1.77, 95% CI = 1.45-2.17). Conclusions Nearly one in four adolescent psychiatric admissions occurs on an adult psychiatric unit. These findings help to fill gaps in the prior literature, and highlight the need for further research to inform policy decisions and resource allocation for adolescent inpatient psychiatric care.

ImportanceHealth-related quality of life (HRQOL) is regarded as a key outcome for evaluating treatment efficacy. However, it is uncertain how HRQOL evolves after epilepsy surgery compared with medical therapy, such as whether it continues to improve over time, improves and then remains stable, or deteriorates after a period of time.ObjectiveTo assess trajectory of HRQOL over 2 years in children with drug-resistant epilepsy (DRE) treated with surgery compared with medical therapy.Design, Setting, and ParticipantsProspective cohort study assessing HRQOL longitudinally over 2 years. Participants were children recruited from 8 epilepsy centers in Canada from 2014 to 2019 with suspected DRE aged 4 to 18 years who were evaluated for surgery. Data were analyzed from May 2014 to December 2021.ExposuresEpilepsy surgery or medical therapy.Main Outcomes and MeasuresHRQOL was measured using the Quality of Life in Childhood Epilepsy Questionnaire (QOLCE)-55. HRQOL and seizure frequency were assessed at baseline, 6-month, 1-year, and 2-year follow-ups. Clinical, parent, and family characteristics were assessed at baseline. A linear mixed model was used to evaluate HRQOL over time, adjusting for baseline clinical, parent, and family characteristics.ResultsThere were 111 surgical and 154 medical patients (mean [SD] age at baseline was 11.0 [4.1] years; 118 [45%] were female). At baseline, HRQOL was similar among surgical and medical patients. HRQOL of surgical patients was 3.0 (95% CI, −0.7 to 6.8) points higher at 6-month, 4.9 (95% CI, 0.7 to 9.1) points higher at 1-year, and 5.1 (95% CI, 0.7 to 9.5) points higher at 2-year follow-ups compared with medical patients. Surgical patients experienced greater improvements in social functioning relative to medical patients, but not for cognitive, emotional, and physical functioning. At 2-year follow-up, 72% of surgical patients were seizure-free, compared with 33% of medical patients. Seizure-free patients reported higher HRQOL than those who were not.Conclusions and RelevanceThis study provided evidence on the association between epilepsy surgery and children’s HRQOL, with improvement in HRQOL occurring within the first year and remaining stable 2 years after surgery. By demonstrating that surgery improved seizure freedom and HRQOL, which has downstream effects such as better educational attainment, reduced health care resource utilization, and health care cost, these findings suggest that the high costs of surgery are justified, and that improved access to epilepsy surgery is necessary.

Health-related quality of life (HRQL) is compromised in children with epilepsy. We aimed to determine whether children diagnosed with epilepsy between ages 4-12 years who are exposed to a higher number of anti-seizure medication (ASM) over the first 2 years, have poorer HRQL 10 years after diagnosis.Data were obtained from 195 children enrolled in the Health-Related Quality of Life in Children with Epilepsy Study (HERQULES) in Canada. HRQL was measured using the Quality of Life in Childhood Epilepsy Questionnaire (QOLCE-55) completed by parents at baseline through to 10 years after diagnosis. The total number of ASM were reported by physicians four times in the first two years after diagnosis. Multivariable block-wise linear regression was used to assess the impact of ASM (categorized as none, one, or more than one), as well as clinical and family factors on children's HRQL 10 years after diagnosis.Children had a mean age of 7.9 ± 2.3 years at diagnosis and 92 (47%) were female. Mean QOLCE at baseline and 10 years was 72.04±14 and 78.7±16,respectively. Clinically meaningful improvement in HRQL from the 2 to 10-year follow-up was detected in 35% of children, reported similarly across all ASM treatment categories (p = .38). The number of ASM prescribed in the first two years was associated with HRQL at the 10-year follow-up, however this association was not significant when adjusting for clinical characteristics, family factors, and HRQL at the two-year follow-up (p = .75). Our data showed that HRQL at 2 years was the only variable associated with better HRQL scores at 10 years (p = .001).In children with new onset epilepsy, exposure to a higher number of ASM, when accounting for clinical and family factors as well as HRQL at 2 years, is not independently associated with lower long-term HRQL. Early HRQL was found to be a good indicator of long-term HRQL, despite the number of ASMs prescribed.

Background and Objective Children with inherited metabolic diseases often require complex and highly specialized care. Patient and family-centered care can improve health outcomes that are important to families. This study aimed to examine experiences of family caregivers (parents/guardians) of children diagnosed with inherited metabolic diseases with healthcare to inform strategies to improve those experiences. Methods A cross-sectional mailed survey was conducted of family caregivers recruited from an ongoing cohort study. Participants rated their healthcare experiences during their child’s visits to five types of healthcare settings common for inherited metabolic diseases: the metabolic clinic, the emergency department, hospital inpatient units, the blood laboratory, and the pharmacy. Participants provided narrative descriptions of any memorable negative or positive experiences. Results There were 248 respondents (response rate 49%). Caregivers were generally very or somewhat satisfied with the care provided at each care setting. Appropriate treatment, provider knowledge, provider communication, and care coordination were deemed essential aspects of satisfaction with care by the majority of participants across many settings. Memorable negative experiences were reported by 8–22% of participants, varying by setting. Among participants who reported memorable negative experiences, contributing factors included providers’ demeanor, lack of communication, lack of involvement of the family, and disregard of an emergency protocol letter provided by the family. Conclusions While caregivers’ satisfaction with care for children with inherited metabolic diseases was high, we identified gaps in family-centered care and factors contributing to negative experiences that are important to consider in the future development of strategies to improve pediatric care for inherited metabolic diseases. Supplementary Information The online version contains supplementary material available at 10.1007/s40271-021-00538-8.

BACKGROUND AND OBJECTIVES Referrals of transgender and gender-diverse (trans) youth to medical clinics for gender-affirming care have increased. We described characteristics of trans youth in Canada at first referral visit. METHODS Baseline clinical and survey data (2017–2019) were collected for Trans Youth CAN!, a 10-clinic prospective cohort of n = 174 pubertal and postpubertal youth RESULTS Of youth, 137 were transmasculine (assigned female) and 37 transfeminine (assigned male); 69.0% were aged 14 to 15, 18.8% Indigenous, 6.6% visible minorities, 25.7% from immigrant families, and 27.1% low income. Most (66.0%) were gender-aware before age 12. Only 58.1% of transfeminine youth lived in their gender full-time versus 90.1% of transmasculine (P < .001). Although transmasculine youth were more likely than transfeminine youth to report depressive symptoms (21.2% vs 10.8%; P = .03) and anxiety (66.1% vs 33.3%; P < .001), suicidality was similarly high overall (past-year ideation: 34.5%, attempts: 16.8%). All were in school; 62.0% reported strong parental gender support, with parents the most common support persons (91.9%). Two-thirds of families reported external gender-related stressors. Youth had met with a range of providers (68.5% with a family physician). At clinic visit, 62.4% were prescribed hormonal suppression or hormone therapy, most commonly depot leuprolide acetate. CONCLUSIONS Trans youth in Canada attending clinics for hormonal suppression or gender-affirming hormones were generally healthy but with depression, anxiety, and support needs.

To describe fatigue in Duchenne muscular dystrophy (DMD) from patients' and parents' perspectives and to explore risk factors for fatigue in children and adolescents with DMD.A multicentre, cross-sectional study design was used. Seventy-one patients (all males; median age 12y, age range 5-17y) identified via the Canadian Neuromuscular Disease Registry, and their parents completed questionnaires. Subjective fatigue was assessed using the Pediatric Quality of Life Inventory Multidimensional Fatigue Scale by child self-report and parent proxy-report.Patients with DMD across ages and disease stages experienced greater fatigue compared to typically developing controls from published data. Sleep disturbance symptoms were associated with greater fatigue by child self-report (ρ=-0.42; p=0.003) and parent proxy-report (ρ=-0.51; p0.001). Depressive symptoms were associated with greater fatigue by child self-report (ρ=-0.46; p0.001) and parent proxy-report (ρ=-0.45; p0.001). Lower functional ability was associated with greater fatigue by parent proxy-report (ρ=0.26; p=0.03). Physical activity level, and musculoskeletal, respiratory, and cardiac function were not associated with fatigue.In paediatric DMD, sleep disturbance symptoms and depressive symptoms are potentially modifiable factors associated with fatigue, warranting additional investigation to facilitate the development of therapeutic strategies to reduce fatigue.Fatigue is a major issue in paediatric Duchenne muscular dystrophy (DMD) across ages and disease stages. Sleep disturbance and depressive symptoms are significantly associated with fatigue in paediatric DMD.FATIGA EN JÓVENES CON DISTROFIA MUSCULAR DE DUCHENNE: OBJETIVO: Describir la fatiga en la distrofia muscular de Duchenne (DMD) desde la perspectiva de los pacientes y los padres, y explorar los factores de riesgo de fatiga en niños y adolescentes con DMD. MÉTODO: Se utilizó un diseño transversal, con información de diversos centros. En total 71 pacientes (todos varones; edad media 12 años, rango de edad 5 a 17 años) identificados a través del Registro Canadiense de Enfermedades Neuromusculares, y sus padres completaron cuestionarios. La fatiga subjetiva se evaluó utilizando la Escala de Fatiga Multidimensional del Inventario de Calidad de Vida Pediátrica usando la información provista por los niños y por los padres. RESULTADOS: Los pacientes con DMD en todas las edades y etapas de la enfermedad experimentaron una mayor fatiga en comparación con los datos publicados en grupo de niños que que se desarrollan típicamente (controles). Los síntomas de alteración del sueño se asociaron con mayor fatiga reportada por el niño (ρ = -0,42; p = 0,003) y por los padres (ρ = -0,51; p0,001). Los síntomas de depresión se asociaron con mayor fatiga reportada por el niño (ρ = -0,46; p0,001) y por los padres (ρ = -0,45; p0,001). La capacidad funcional más baja se asoció con mayor fatiga reportada por los padres (ρ = 0,26; p = 0,03). El nivel de actividad física y la función musculoesquelética, respiratoria y cardíaca no se asociaron con la fatiga. INTERPRETACIÓN: En la DMD pediátrica, los síntomas de alteración del sueño y los síntomas de depresión son factores asociados con la fatiga. Estos factores son potencialmente modificables, lo que justifica una investigación adicional para facilitar el desarrollo de estrategias terapéuticas para reducir la fatiga.OBJETIVO: Descrever a fadiga na distrofia muscular de Duchenne (DMD) pela perspectiva de pacientes e pais, e explorar fatores de risco para fadiga em crianças e adolescentes com DMD. MÉTODO: Um desenho de estudo multicêntrico e transversal foi usado. Setenta e um pacientes (todos do sexo masculino; idade mediana 12a, variação da idade 5-17a) identificados via Registro Canadense de Doenças Neuromusculares, e seus pais, completaram os questionários. A fadiga subjetiva foi avaliada usando a Escada de Fadiga do Inventário Pediátrico de Qualidade de Vida Multidimensional por auto-relato da criança e relato dos pais.Pacientes com DMD de várias idades e estágios da doença apresentaram mais fadiga comparados os controles com desenvolvimento típico de dados publicados. Sintomas de distúrbios do sono foram associados com maior fadiga pelo auto-relato das crianças (ρ=-0,42; p=0,003) e pelo relato dos pais (ρ=-0,51; p0,001). Sintomas depressivos foram associados com maior fadiga no auto-relato das crianças (ρ=-0,46; p0,001) e pelo relato dos pais (ρ=-0,45; p0,001). Menor capacidade funcional se associou com maior fadiga de acordo com o relato dos pais (ρ=0,26; p=0,03). Nível de atividade física, e função músculo-esquelética, respiratória, e cardíaca não foram associadas com a fadiga. INTERPRETAÇÃO: Na DMD pediátrica, sintomas de distúrbios do sono e sintomas depressivos são fatores potencialmente modificáveis, que merecem investigação adicional para facilitar o desenvolvimento de estratégias terapêuticas para reduzir a fadiga.

Objective The aim of this study was to assess measurement equivalence in the 16-item short-form Quality of Life in Childhood Epilepsy Questionnaire (QOLCE-16) across age, sex, and time in a sample of children with newly diagnosed epilepsy. Methods Data came from 373 children participating in the Health-related Quality of Life in Children with Epilepsy Study (HERQULES), a multicenter prospective cohort study. Measurement equivalence was assessed using multigroup confirmatory factor analysis methods. Comparison groups were stratified by age (4–7 years vs. 8–12 years), sex (male vs. female), and time (at diagnosis vs. 24 months postdiagnosis). Results The QOLCE-16 demonstrated measurement equivalence at the level of strict invariance for each comparison group tested — age: χ2 (3, 274) = 429.6, p Significance The findings provide support for the robust psychometric profile of the QOLCE-16 as a reliable and valid measure of health-related quality of life for children with epilepsy. Demonstrating good properties and a multidimensional structure, the QOLCE-16 is an appropriate short measure for both clinicians and researchers wanting to obtain health-related quality of life information on children with epilepsy.

To develop an Arabic translation of the Quality of Life in Children with Epilepsy-55 questionnaire (QOLCE-55), and to assess its validity and reliability to be readily used in Arabic and Egyptian cultures.The original English version of the QOLCE-55 was translated into Arabic using a forward-backward translation method, and then a cross-sectional survey was conducted including 100 children with epilepsy aged 4-18 years. Caregivers of children completed the Arabic version of the QOLCE-55. Assessment of psychometric properties of the translated questionnaire was conducted using test-retest reliability, internal consistency, and convergent and divergent validity.The translated questionnaire showed excellent test-retest reliability with the intra-class correlation coefficient for all questionnaire domains, as well as the overall questionnaire ranging from 0.91 to 0.98. Cronbach alpha exceeded 0.7 denoting good internal consistency except for the emotional functioning scale. Convergent and divergent validity assessment showed that items of all domains significantly correlated with their scale scores with r 0.4 and these correlations were much higher than correlations with other scales' scores, consistent with good convergent and divergent validity. The mean total HRQOL score was 65.63 ± 8.79 with the highest score for social functioning domain and lowest score for physical functioning domain.The Arabic version of the QOLCE-55 can be considered a suitable, reliable, and valid tool to assess the HRQOL of children with epilepsy through their caregivers' reports.

Background Epilepsy extends far beyond seizures; up to 80% of children with epilepsy (CWE) may have comorbid cognitive or mental health problems, and up to 50% of parents of CWE are at risk for major depression. Past research has also shown that family environment has a greater influence on children’s and parents’ health-related quality of life (HRQOL) and mental health than epilepsy-related factors. There is a pressing need for low-cost, innovative interventions to improve HRQOL and mental health for CWE and their parents. The aim of this randomized controlled trial (RCT) is to evaluate whether an interactive online mindfulness-based intervention program, Making Mindfulness Matter (M3), can be feasibly implemented and whether it positively affects CWE’s and parents’ HRQOL and mental health (specifically, stress, behavioral, depressive, and anxiety symptoms). Methods This parallel RCT was planned to recruit 100 child-parent dyads to be randomized 1:1 to the 8-week intervention or waitlist control and followed over 20 weeks. The intervention, M3, will be delivered online and separately to parents and children (ages 4–10 years) in groups of 4–8 by non-clinician staff of a local community epilepsy agency. The intervention incorporates mindful awareness, social-emotional learning skills, and positive psychology. It is modeled after the validated school-based MindUP program and adapted for provision online and to include a parent component. Discussion This RCT will determine whether this online mindfulness-based intervention is feasible and effective for CWE and their parents. The proposed intervention may be an ideal vector to significantly improve HRQOL and mental health for CWE and their parents given its low cost and implementation by community epilepsy agencies. Trial registration ClinicalTrials.gov NCT04020484. Registered on July 16, 2019.

Objective To examine longitudinal changes and predictors of depression and anxiety 2 years following resective epilepsy surgery, compared to no surgery, in children with drug-resistant epilepsy (DRE). Method This multicenter cohort study involved 128 children and adolescents with DRE (48 surgical, 80 nonsurgical; 8–18 years) who completed self-report measures of depression and anxiety at baseline and follow-up (6-month, 1-year, 2-year). Child demographic (age, sex, IQ) and seizure (age at onset, duration, frequency, site and side) variables were collected. Results Linear mixed-effects models controlling for age at enrolment found a time by treatment by seizure outcome interaction for depression. A negative linear trend across time (reduction in symptoms) was found for surgical patients, irrespective of seizure outcome. In contrast, the linear trend differed depending on seizure outcome in nonsurgical patients; a negative trend was found for those with continued seizures, whereas a positive trend (increase in symptoms) was found for those who achieved seizure freedom. Only a main effect of time was found for anxiety indicating a reduction in symptoms across patient groups. Multivariate regressions failed to find baseline predictors of depression or anxiety at 2-year follow-up in surgical patients. Older age, not baseline anxiety or depression, predicted greater symptoms of anxiety and depression at 2-year follow-up in nonsurgical patients. Conclusion Children with DRE reported improvement in anxiety and depression, irrespective of whether they achieve seizure control, across the 2 years following surgery. In contrast, children with DRE who did not undergo surgery, but achieved seizure freedom, reported worsening of depressive symptoms, which may indicate difficulty adjusting to life without seizures and highlight the potential need for ongoing medical and psychosocial follow-up and support.

Purpose The objective of this study was to delineate the long-term impact of pediatric convulsive status epilepticus (CSE) on health-related quality of life (HRQOL) in children recently diagnosed with epilepsy. Methods Children with newly-diagnosed epilepsy were recruited between 2004–2007 through a Canada-wide population-based study, the Health-Related Quality of Life Study in Children with Epilepsy Study (HERQULES). Eligible children were 4–12 years of age at epilepsy diagnosis; consequently, children with the more catastrophic syndromes and epileptic encephalopathies typically diagnosed at younger ages were ineligible. Participants were followed over 10 years, and neurologists identified those with CSE in the first two years after epilepsy diagnosis. HRQOL was self-reported by adolescents and young adults (AYAs) in the long-term, and reported by parents at multiple time points throughout the 10-year follow-up. Results A total of 204 AYAs were followed over the long-term, 12 of whom had a history of CSE. Parents of those with CSE reported poorer HRQOL in their children two years after the epilepsy diagnosis, (Cohen’s d = 0.58, p = .037), though not at the 10-year follow-up (d = 0.28, p = .19). Results from AYAs’ self-reports at the 10-year follow-up were similar, such that mean HRQOL scores were poorer for AYAs with CSE, though this difference was not significant (d = 0.39, p = .11). Results were similar when adjusting for HRQOL at the time of epilepsy diagnosis. Conclusion These findings suggest that compromised HRQOL in the short-term after CSE may resolve over the long-term. It will be important for future studies to focus on patient-reported outcomes in reporting the long-term sequelae of CSE.

Objective This study estimated trajectories of health-related quality of life (HRQOL) over a 10-year period among children newly diagnosed with epilepsy. We also modeled the characteristics of children, parents, and families associated with each identified trajectory. Methods Data came from the HERQULES (Health-Related Quality of Life in Children With Epilepsy Study), a Canada-wide prospective cohort study of children (aged 4-12 years) with newly diagnosed epilepsy. Parents reported on their children's HRQOL at diagnosis, and at 0.5-, 1-, 2-, 8-, and 10-year follow-ups using the Quality of Life in Childhood Epilepsy Questionnaire-55. Trajectories of HRQOL were identified using latent class growth models. Characteristics of children, parents, and families at the time of diagnosis that were associated with each trajectory were identified using multinomial logistic regression. Results A total of 367 children were included. Four unique HRQOL trajectories were identified; 11% of the cohort was characterized by low and stable scores, 18% by intermediate and stable scores, 35% by intermediate scores that increased then plateaued, and 43% by high scores that increased then plateaued. Absence of comorbidities, less severe epilepsy, and better family environment (greater satisfaction with family relationships and fewer family demands) at the time of diagnosis were associated with better long-term HRQOL trajectories. Although the analyses used estimates for missing values and accounted for any nonrandom attrition, the proportion of children with poorer HRQOL trajectories may be underestimated. Significance Children with new onset epilepsy are heterogenous and follow unique HRQOL trajectories over the long term. Overall, HRQOL improves for the majority in the first 2 years after diagnosis, with these improvements sustained over the long term.

AIM: To compare long-term psychosocial and functional outcomes of young adults with uncomplicated childhood-onset epilepsy (COE) to population norm controls utilizing a controlled prospective cohort study. METHOD: Psychosocial and functional outcomes were assessed at 10-year follow-up. Fifty-three young adults (27 males, 26 females) with COE (n=21 remission; 18y 1mo–30y 9mo; mean age 23y 4mo [SD 3y 4mo]; mean age of epilepsy onset 12y [SD 3y 2mo]) were compared to 55 (23 males, 32 females) first-degree cousin controls (18y 5mo–29y 8mo; mean age 23y 6mo [SD 3y]). Seizure remission status and baseline comorbidities (attention-deficit/hyperactivity disorder [ADHD], depressive disorders, anxiety disorders, and academic problems) were examined as possible risk factors for significant differences in functional outcomes. RESULTS: Poorer functional outcomes, indicated by patient rated cognition and overall disability, were evident among young adults with epilepsy compared to controls (all p-values

© 2019, Springer Nature Switzerland AG. Purpose: To generate foundational knowledge in the creation of a quality-of-life instrument for patients who are clinically diagnosed as being in a vegetative or minimally conscious state but are able to communicate by modulating their brain activity (i.e., behaviourally nonresponsive and covertly aware). The study aimed to identify a short list of key domains that could be used to formulate questions for an instrument that determines their self-reported quality of life. Methods: A novel two-pronged strategy was employed: (i) a scoping review of quality-of-life instruments created for patient populations sharing some characteristics with patients who are behaviourally nonresponsive and covertly aware was done to compile a set of potentially relevant domains of quality of life; and (ii) a three-round Delphi consensus process with a multidisciplinary panel of experts was done to determine which of the identified domains of quality of life are most important to those who are behaviourally nonresponsive and covertly aware. Five expert groups were recruited for this study including healthcare workers, neuroscientists, bioethicists, quality-of-life methodologists, and patient advocates. Results: Thirty-five individuals participated in the study with an average response rate of 95% per round. Over the three rounds, experts reached consensus on 34 of 44 domains (42 domains were identified in the scoping review and two new domains were added based on suggestions by experts). 22 domains were rated as being important for inclusion in a quality-of-life instrument and 12 domains were deemed to be of less importance. Participants agreed that domains related to physical pain, communication, and personal relationships were of primary importance. Based on subgroup analyses, there was a high degree of consistency among expert groups. Conclusions: Quality of life should be a central patient-reported outcome in all patient populations regardless of patients’ ability to communicate. It remains to be determined how covertly aware patients perceive their circumstances and quality of life after suffering a life-altering injury. Nonetheless, it is important that any further dialogue on what constitutes a life worth living should not occur without direct patient input.

Objective The objective of the study was to validate the parent-proxy reported Quality of Life in Childhood Epilepsy Questionnaire (QOLCE) in a sample of young adults with a history of childhood-onset epilepsy, allowing for the utilization of a consistent informant (the parent) across the youths' stages of development. The 55-item (QOLCE-55) and 16-item versions (QOLCE-16) were evaluated. Methods Data came from 134 young adults (aged 18.0 to 28.5 years) with childhood-onset epilepsy, recruited through community and tertiary care centers across Canada. Confirmatory factor analysis (CFA) was used to assess the higher-order factor structure of the QOLCE. Cronbach's alpha was used to evaluate internal consistency. Convergent validity was assessed by intraclass correlation coefficients (ICC) with the youth self-reported Quality of Life in Epilepsy Questionnaire (QOLIE-31-P). Results The higher-order factor structure of the QOLCE-55 and QOLCE-16 demonstrated adequate fit: QOLCE-55 comparative fit index (CFI) = 0.968, Tucker–Lewis index (TLI) = 0.966; and root mean square of approximation (RMSEA) = 0.061; QOLCE-16 CFI = 0.966, TFI = 0.959, RMSEA = 0.141. Higher-order factor loadings were strong, ranging from 0.71 to 0.90. Internal consistency was excellent for the total score (αQOLCE-55 = 0.97; αQOLCE-16 = 0.93) and good-excellent for each subscale (α > 78). Convergent validity was moderate to good for the total score (ICC > 0.72) and each subscale (ICC > 0.51). Significance These findings provide support for the use of the QOLCE-55 and QOLCE-16 among young adults with a history of childhood-onset epilepsy. Utilizing a consistent measure and informant across the stages of development is essential to reliably evaluate change over time.

Objective Diagnosis of epilepsy is known to impact health-related quality of life (HRQOL) of children with new-onset epilepsy and can also influence their conceptualization and valuation of HRQOL construct, also known as response shift. This study investigates the presence of response shift in a cohort of children with new-onset epilepsy. Methods Data are from the HEalth-Related QUality of Life in children with Epilepsy Study, a prospective cohort study of 373 children with new-onset epilepsy. Hypotheses about the presence of reconceptualization, reprioritization, and recalibration response shift were tested in the Quality of Life in Childhood Epilepsy (QOLCE-55) Questionnaire, a parent-reported, disease-specific HRQOL measure, using Oort's structural equation model between baseline and 1-year follow-up. Model fit was assessed using log-likelihood ratio test, root mean square error of approximation, and comparative fit index. Results Small positive uniform recalibration response shift effects were observed on physical, emotional, and social functioning domains of the QOLCE-55, but negligibly small negative nonuniform recalibration response shift effect was observed on social functioning domain. There was no significant change in overall QOLCE-55 scores over time after adjusting for response shift effects. Significance Parents of children with new-onset epilepsy are likely to positively recalibrate (upward bias) their assessments of their children's HRQOL over a 1-year period after diagnosis. This study highlights the potential benefits of response shift as a desired consequence in parents' perception of changes in HRQOL of children with new-onset epilepsy.

To determine whether epilepsy surgery improved health-related quality of life (HRQoL) and whether seizure freedom after surgery mediated the improvement in HRQoL.This multicenter cohort study compared HRQoL after epilepsy surgery to pharmacological management in children with drug-resistant epilepsy (DRE). HRQoL was measured using the Quality of Life in Childhood Epilepsy (QOLCE) questionnaire at baseline and 1-year follow-up. The mediator between treatment type and HRQoL was seizure freedom.Two hundred and thirty-seven patients were recruited (surgery group: n=147 [92 males, 45 females]; pharmacological group: n=90 [53 males, 37 females]). Mean age at seizure onset was 6 years (SD 4y 4mo) in the surgical group and 6 years 1 month (SD 4y) in the pharmacological group. The odds ratio of seizure freedom was higher for the surgery versus pharmacological group (β=4.24 [95% confidence interval {CI}: 2.26-7.93], p0.001). Surgery had no direct effect on total QOLCE score at 1-year (β=0.24 [95% CI -2.04 to 2.51], p=0.839) compared to pharmacological management, but had an indirect effect on total QOLCE that was mediated by seizure freedom (β=0.92 [95% CI 0.19-1.65], p=0.013), adjusting for baseline total QOLCE score. Surgery had a direct effect on improving social function (p=0.043), and an indirect effect on improving physical function (p=0.016), cognition (p=0.042), social function (p=0.012) and behavior (p=0.032), mediated by seizure freedom.Greater seizure freedom achieved through epilepsy surgery mediated the improvement in HRQoL compared to pharmacological management in children with DRE.Seizure freedom is higher after pediatric epilepsy surgery compared to pharmacologically managed epilepsy. Surgery indirectly improves health-related quality of life (HRQoL) mediated by seizure freedom compared to pharmacological management. Surgery has a direct effect on improving social function relative to pharmacological management. Baseline HRQoL was an important predictor of HRQoL after treatment.LA AUSENCIA DE CRISES EPILÉPTICAS EN NIÑOS MEJORA LA CALIDAD DE VIDA RELACIONADA CON LA SALUD DESPUÉS DE LA CIRUGÍA DE EPILEPSIA: OBJETIVO: Determinar si la cirugía de epilepsia mejora la calidad de vida relacionada con la salud (HRQoL) y si la ausencia de crises epilépticas después de la cirugía intervino en la mejora.Estudio de cohorte, multicéntrico que evaluó la HRQoL después de la cirugía de epilepsia comparándolo con el manejo farmacológico, en niños con epilepsia farmacorresistente (DRE). La HRQoL se midió utilizando el cuestionario de calidad de vida en la infancia (QOLCE) al inicio y después de un año de seguimiento. El mediador entre el tipo de tratamiento y HRQoL fue la ausencia de crises epilépticas.Se reclutaron 237 pacientes (grupo de cirugía: n 147 [92 varones, 45 mujeres]; grupo farmacológico: n 90 [53 varones, 37 mujeres]). La edad promedio al inicio de las crisis epilépticas fue de 6 años (DE 4 años 4 meses) en el grupo quirúrgico y 6 años 1 mes (DE 4 años) en el grupo farmacológico. La relación de probabilidad de la ausencia de crises epilépticas fue mayor para el grupo de cirugía frente a farmacológico (β = 4,24 [intervalo de confianza (IC) 95 %: 2.26-7.93], p0.001). La cirugía no tiene ningún efecto directo sobre la puntuación total de QOLCE a 1 año (β = 0,24 [IC del 95% -2,04 a 2,51], p=0,839) en comparación con el tratamiento farmacológico, pero tiene un efecto indirecto en el QOLCE total que fue mediado por la ausencia de crises epilépticas (β = 0,92 [IC del 95% 0,19-1,65], p=0,013), ajustando la puntuación total de QOLCE. La cirugía tuvo un efecto directo en la mejora de la función social (p=0.043), y un efecto indirecto en la mejora de la función física (p=0.016), la cognición (p=0,042), la función social (p=0.012) y el comportamiento (p=0.032), mediado por la ausencia de crises epilépticas.En este estudio encontramos que en niños con DRE la ausencia mas significativa de crises epilépticas, lograda a través de la cirugía de epilepsia, mediaba una mejor HRQoL en comparación con el manejo farmacológico.FICAR SEM CRISES EPILÉPTICAS MELHORA A QUALIDADE DE VIDA RELACIONADA À SAÚDE APÓS CIRURGIA PARA EPILEPSIA EM CRIANÇAS: OBJETIVO: Determinar se a cirurgia para epilepsia melhora a qualidade de vida relacionada à saude (QVRS) e se ficar sem crises epilépticas após a cirurgia media a melhora na QVRS. MÉTODO: Este estudo de coorte multicêntrico comparou a QVRS após cirurgia para epilepsia ao manejo farmacológico em criancas com epilepsia resistente a drogas (ERD). A QVRS foi medida usando o questionário Qualidade de Vida na Epilepsia Infantil (QVEI) na linha de base e no acompanhamento após 1 ano. O mediador entre o tipo de tratamento e a QVRS foi estar sem crises epilépticas. RESULTADOS: Duzentos e trinta e sete pacientes foram recrutados (grupo cirurgia: n=147 [92 do sexo masculino, 45 do sexo feminino]; grupo farmacológico: n=90 [53 do sexo masculino, 37 do sexo feminino]). A média de idade no início das crises foi 6 anos (DP 4a 4m) no grupo cirúrgico e 6 anos e 1 mês (DP 4a) no grupo farmacológico. A taxa de risco de estar sem crises foi maior no grupo cirurgia versus farmacológico (β=4,24 [intervalo de confiança a 95% {IC}: 2,26-7,93], p0,001). A cirurgia não teve efeito direto no QVEI total após 1 ano (β=0,24 [IC 95% -2,04 a 2,51], p=0,839) comparada ao manejo farmacológico, mas teve efeito indireto no QVEI total que foi medido pelo fato de estar livre de crises (β=0,92 [IC 95% 0,19-1,65], p=0,013), ajustando para o escore total QVEI na linha de base. A cirurgia teve efeito direto na melhora da função social (p=0,043), e efeito indireto na melhora da função física (p=0,016), cognição (p=0,042), função social (p=0.012) comportamento (p=0,032), mediada pelo fato de estar sem crises. INTERPRETAÇÃO: A maior chance de ficar sem crises, atingida por meio da cirurgia, mediou a melhora na QVRS em comparação com o manejo farmacológico em crianças com ERD.

There is limited information about the effectiveness of transition programs for youth moving from pediatric to adult care with any chronic disease. Two Delphi studies and National Institute for Health and Care Excellence (NICE) guidelines about transition for epilepsy have suggested few critical outcome measures for transition. A single large prospective study found that the most important transition program elements were appropriate parent involvement, promotion of health self-efficacy, and meeting the adult team before transfer. Two Cochrane reviews of the value of transition for epilepsy found insufficient evidence to establish or refute the value of various programs, although evaluation of a few programs suggested a great deal of family/patient satisfaction. The cost of transition programs and their cost effectiveness have also not been established except for renal transplantation where transition programs were associated with fewer losses of the transplanted kidneys, a cost-effective outcome. Published data on the overall cost of care for children and adults with epilepsy may be helpful to establish a business plan for a transition program, and are briefly reviewed. Establishing cost effectiveness of transition programs for epilepsy would promote their establishment and viability. However, a number of studies will be needed based on the nature of the program, the healthcare system where it is carried out, and the type of epilepsy. In fee-for-service health systems, the reevaluation of patients with epilepsy prior to transfer may be sufficient to cover the costs of the transition program, whereas in single payer systems, there may be positive downstream health or societal benefits that justify the costs. A theoretical framework for comprehensive evaluation of epilepsy transition programs is needed. The Triple Aim Framework seems applicable with focus on population health, patient experiences, and cost and has the potential to assess transition interventions in the context of system-wide improvements in healthcare. Transition programs in general have not been well evaluated, and very little evaluation data exist regarding transition programs for epilepsy. We recommend more evaluative research using rigorous methodology to comprehensively assess these programs.

This chapter examines the lives of caregivers in different settings and in different stages of the life course. It suggests that the comparative study of different caregiving experiences might assist in better understanding the key elements that shape caregivers’ lives: Those factors that assist family members in shouldering the burden of care and those that may exacerbate the negative consequences of providing such care. The chapter identifies the potentially important aspects of the caregiving process that may have implications for future studies of caregiving in a variety of different circumstances. It discusses the utility of the stress process for understanding variations in psychological distress among caregivers and explains the impact of social stressors on psychological distress. The chapter also examines the impact of stressful life experiences varies substantially according to which caregivers. It is interesting to note that these correlations are weakest among those individuals who provide care to individuals with psychiatric or developmental problems.

Aim To identify important explanatory variables of four patient-reported outcomes (PROs): vision-related quality of life (VRQoL), preference-based health-related quality of life (HRQoL), social support and community integration and depressive symptoms. Methods Cross-sectional study conducted at one ophthalmic practice in a hospital setting. Patients with a diagnosis of glaucoma or glaucoma suspect (n = 250) were sequentially recruited. Patients with language restrictions were excluded. Data were collected through medical chart reviews and face-to-face interviews. The PROs were measured using validated tools. Candidate models for predicting PROs from explanatory variables were constructed using linear and logistic regression, as well as classification and regression trees. Through leave-one-out cross-validation, the performance of each model was assessed in terms of mean absolute error. Results Use of mobility aids, best corrected visual acuity (BCVA), income, and living arrangements were most predictive of VRQoL, social support, and community integration. Use of mobility aids was also most predictive of the presence of depressive symptoms, and BCVA with preference-based HRQoL. Conclusion Although promising associations were discovered, the models based on commonly collected clinical variables had limited ability to accurately predict individual patient PROs. Thus, although this study identifies clinical and demographic variables that are most predictive of PROs, routine collection of PROs in clinical practice may be necessary to obtain a complete picture of the quality of life of glaucoma patients.

This study investigated clinical and family characteristics associated with health-related quality of life in children with Duchenne muscular dystrophy. Families of 176 boys with Duchenne muscular dystrophy were identified and mailed questionnaires via the Canadian Neuromuscular Disease Registry. Multiple linear regressions analyses were used to examine the relationship between clinical and family characteristics and child-self and parent-proxy reported health-related quality of life. Greater fatigue and use of wheelchairs were consistently associated with worse health-related quality of life independent of other factors. Higher household income and parent having a postsecondary degree were associated with better health-related quality of life in some of the measures. A greater clinical focus on and efforts to reduce fatigue could lead to improvement of health-related quality of life in the Duchenne muscular dystrophy population. This study also sets the ground for longitudinal studies where changes in health-related quality of life can be monitored over time.

Summary Objective The aim of this study was to examine measurement equivalence of the newly developed Quality of Life in Childhood Epilepsy Questionnaire (QOLCE-55) across age, sex, and time in a representative sample of children with newly diagnosed epilepsy. Methods Data come from 373 children enrolled in the Health-related Quality of Life in Children with Epilepsy Study (HERQULES), a multisite prospective cohort study. Measurement equivalence was examined using a multiple-group confirmatory factor analysis framework, whereby increasingly stringent parameter constraints are imposed on the model. Comparison groups were stratified based on age (4–7 years vs. 8–12 years), sex (male vs. female), and time (measurement of health-related quality of life at diagnosis vs. 24 months later). Results The QOLCE-55 demonstrated measurement equivalence at the level of strict invariance for each model tested—age: χ2 (3,123) = 4,097.3, p < 0.001; Comparative Fit Index (CFI) = 0.968; Root Mean Square Error of Approximation (RMSEA) = 0.042 (0.038, 0.045); sex: χ2 (3,124) = 4,188.3, p < 0.001; CFI = 0.964; RMSEA = 0.044 (0.040, 0.047); and time: χ2 (3,121) = 5,185.0, p < 0.001; CFI = 0.965; RMSEA = 0.046 (0.043, 0.048). Significance These findings suggest that items comprising the QOLCE-55 are perceived similarly among groups stratified by age, sex, and time and provide further evidence supporting the validity of the scale in children with epilepsy. Health professionals and researchers should be confident that group comparisons made using the QOLCE-55 are unbiased and that any group differences detected are meaningful; that is, not related to differences in the interpretation of items by informants. Future research replicating these findings is encouraged.

Objectives This review aimed to describe social outcomes in adulthood for people with a history of childhood-onset epilepsy and identify factors associated with these outcomes; focused on educational attainment, employment, income/financial status, independence/living arrangement, romantic relationships, parenthood, and friendships. Methods A comprehensive search of MEDLINE, EMBASE, and PsycINFO was conducted, as well as forward and backward citation tracking. A total of 45 articles met inclusion criteria. Random effects meta-analyses were conducted, and subgroup analyses evaluated outcomes for people with epilepsy (PWE) with good prognosis (e.g., normal intelligence, ‘epilepsy-only’) and poor prognosis (e.g., intellectual disability, Dravet syndrome), and those who underwent epilepsy surgery in childhood. Results Among all PWE, 73% (95% confidence interval [CI]: 64–82%) completed secondary school education, 63% (95%CI: 56–70%) were employed; 74% (95%CI: 68–81%) did not receive governmental financial assistance; 32% (95%CI: 25–39%) were in romantic relationships; 34% (95%CI: 24–45%) lived independently; 21% (95%CI:12–33%) had children, and 79% (95%CI: 71–87%) had close friend(s). People with epilepsy often fared worse relative to healthy controls. Among PWE with a good prognosis, a comparable number of studies reported similar/better outcomes relative to controls as reported poorer outcomes. The most consistent predictor of poorer outcomes was the presence of cognitive problems; results of studies evaluating seizure control were equivocal. Conclusion People with epilepsy with a good prognosis may show similar social outcomes as controls, though robust conclusions are difficult to make given the extant literature. Seizure control does not guarantee better outcomes. There is a need for more studies evaluating prognostic factors and studies with control groups to facilitate appropriate comparisons.

OBJECTIVE: Parental depression significantly impacts children's health and well-being. This study aimed to (1) estimate the prevalence of depressive symptoms, at six time points, among mothers over the first 10 years after their child was diagnosed with epilepsy; (2) identify trajectories of maternal depressive symptoms over time; and (3) identify baseline factors associated with each trajectory. METHODS: Data came from the Health-Related Quality of Life in Children with Epilepsy Study (HERQULES), a Canada-wide prospective cohort study. Data on child, parent, and family characteristics were collected at the time of diagnosis, and follow-ups at 0.5, 1, 2, 8, and 10 years. Maternal depressive symptoms were measured using the Center for Epidemiological Studies Depression Scale. Trajectories of depressive symptoms were evaluated using latent class growth modeling, and multinomial logistic regression was used to identify baseline factors associated with each trajectory. RESULTS: A total of 356 mothers participated in the study, of whom 57% scored in the at-risk range for major depression disorder (period-prevalence). Four unique trajectories were identified as follows: "Low-Stable" (29% of mothers), "Intermediate-Stable" (46%), "High-Stable" (20%), and "High-Decreasing" (5%). Positive family environment was consistently associated with a better trajectory of depressive symptoms over time; other significant factors included type of seizures, child cognitive comorbidity, maternal age, and maternal education. SIGNIFICANCE: A substantial proportion of mothers of children with epilepsy are at risk for depression, and this risk is stable over the long term. Family environment at the time of diagnosis has long-term and persistent effects and may be an ideal target for interventions.

Objectives This systematic review aimed to 1) describe the quality of life (QOL) of parents of children with childhood-onset epilepsy (CWE), 2) identify factors associated with parental QOL, and 3) evaluate the association between parents' QOL and children's psychological well-being. Methods We conducted a comprehensive search of MEDLINE, EMBASE, and PsycINFO and conducted forward and backward citation tracking. A total of 15 articles met inclusion criteria. Parents' QOL was compared with population norms, healthy controls, and parents of children with other chronic conditions. Factors associated with parental QOL were systematically evaluated. Results Heterogeneity in study design and reporting prevented a meta-analytic synthesis of results. The majority of studies found that parents of CWE had poorer QOL relative to healthy controls or population norms and similar QOL as parents of children with other chronic conditions. In addition, poorer parental QOL was consistently associated with greater parental anxiety and depressive symptoms and poorer socioeconomic status and child QOL. Mothers had poorer QOL relative to fathers. Seizure control was not consistently associated with parental QOL. Results highlight the impact of family environment and psychosocial factors. Significance This review suggests that parents of CWE have compromised QOL. The results are in line with previous research showing the interdependent nature of psychosocial and medical factors, with psychosocial factors playing a critical role in child and parental QOL and well-being. Interventions targeting the family unit are warranted, and healthcare providers should be aware of the bidirectional relationship of epilepsy, family environment, and child/parent health and well-being.

PURPOSE: Epilepsy in childhood extends far beyond seizures and affects child and parental well-being. The long-term impact of childhood-onset epilepsy on parental well-being is unknown. This study assessed health-related quality of life (HRQOL) in mothers 10 years after their child's diagnosis of epilepsy. METHODS: Data come from the Health-Related Quality of Life in Children with Epilepsy Study, a multicenter prospective cohort study of children with newly diagnosed epilepsy. Mothers completed a mailed questionnaire at the 10-year follow-up, which included the Short-Form Health Survey (SF-12-v2) to evaluate the physical and mental health components of their HRQOL. Block-wise linear regressions identified child/epilepsy, maternal/family, and maternal psychosocial factors associated with mothers' HRQOL. RESULTS: A total of 159 mothers participated in this study (46% of the sample assessed at baseline). At follow-up, 69% of youth had been seizure free for the past 5 years. Mothers scored similarly to population norms (mean: 50, SD: 10) on the mental health subscale (mean: 49.5, SD: 9.3) and significantly better on the physical health subscale (mean: 53.0, SD: 7.6). Better family resources were associated with higher (better) scores on the physical health subscale (B = 0.20; 95% CI 0.03, 0.36). Better family functioning (B = 0.34; 95% CI 0.06, 0.62), fewer maternal depressive symptoms (B = 0.33; 95% CI 0.20, 0.47), and perception of less stress (B = 0.70; 95% CI 0.52, 0.88) were associated with higher (better) scores on the mental health subscale. CONCLUSION: Ten years after the diagnosis of epilepsy in children, the HRQOL of mothers was similar to reports from women in the general population. This study identified factors contributing to better maternal HRQOL and highlights the importance of family environment over epilepsy-related variables.

Objective The aim of this study was to develop and validate a brief version of the Quality of Life in Childhood Epilepsy Questionnaire (QOLCE). A secondary aim was to compare the results described in previously published studies using the QOLCE-55 with those obtained using the new brief version. Methods Data come from 373 children involved in the Health-related Quality of Life in Children with Epilepsy Study, a multicenter prospective cohort study. Item response theory (IRT) methods were used to assess dimensionality and item properties and to guide the selection of items. Replication of results using the brief measure was conducted with multiple regression, multinomial regression, and latent mixture modeling techniques. Results IRT methods identified a bi-factor graded response model that best fits the data. Thirty-nine items were removed, resulting in a 16-item QOLCE (QOLCE-16) with an equal number of items in all 4 domains of functioning (Cognitive, Emotional, Social, and Physical). Model fit was excellent: Comparative Fit Index = 0.99; Tucker-Lewis Index = 0.99; root mean square error of approximation = 0.052 (90% confidence interval [CI] 0.041-0.064); weighted root mean square = 0.76. Results that were reported previously using the QOLCE-55 and QOLCE-76 were comparable to those generated using the QOLCE-16. Significance The QOLCE-16 is a multidimensional measure of health-related quality of life (HRQoL) with good psychometric properties and a short-estimated completion time. It is notable that the items were calibrated using multidimensional IRT methods to create a measure that conforms to conventional definitions of HRQoL. The QOLCE-16 is an appropriate measure for both clinicians and researchers wanting to record HRQoL information in children with epilepsy.

ObjectivesMethodologically, to assess the feasibility of participant recruitment and retention, as well as missing data in studying mental disorder among children newly diagnosed with chronic physical conditions (ie, multimorbidity). Substantively, to examine the prevalence of multimorbidity, identify sociodemographic correlates and model the influence of multimorbidity on changes in child quality of life and parental psychosocial outcomes over a 6-month follow-up.DesignProspective pilot study.SettingTwo children’s tertiary-care hospitals.ParticipantsChildren aged 6–16 years diagnosed in the past 6 months with one of the following: asthma, diabetes, epilepsy, food allergy or juvenile arthritis, and their parents.Outcome measuresResponse, participation and retention rates. Child mental disorder using the Mini International Neuropsychiatric Interview at baseline and 6 months. Child quality of life, parental symptoms of stress, anxiety and depression, and family functioning. All outcomes were parent reported.ResultsResponse, participation and retention rates were 90%, 83% and 88%, respectively. Of the 50 children enrolled in the study, the prevalence of multimorbidity was 58% at baseline and 42% at 6 months. No sociodemographic characteristics were associated with multimorbidity. Multimorbidity at baseline was associated with declines over 6 months in the following quality of life domains: physical well-being, β=−4.82 (–8.47, –1.17); psychological well-being, β=−4.10 (–7.62, –0.58) and school environment, β=−4.17 (–8.18, –0.16). There was no association with parental psychosocial outcomes over time.ConclusionsPreliminary evidence suggests that mental disorder in children with a physical condition is very common and has a negative impact on quality of life over time. Based on the strong response rate and minimal attrition, our approach to study child multimorbidity appears feasible and suggests that multimorbidity is an important concern for families. Methodological and substantive findings from this pilot study have been used to implement a larger, more definitive study of child multimorbidity, which should lead to important clinical implications.

Objectives. To investigate if an association exists between being born large for gestational age (LGA) and verbal ability or externalizing behaviour problems at ages 4-5 years. Method. A secondary analysis was conducted using the National Longitudinal Survey of Children and Youth, including singleton births in 2004-2005 followed till 4-5 years (n=1685). LGA was defined as a birth weight > 90th percentile. Outcomes included poor verbal ability (scoring < 15th percentile on the Revised Peabody Picture Vocabulary Test) and externalizing behaviour problems (scoring > 90th percentile on externalizing behaviour scales). Multivariable logistic regression with longitudinal standardized funnel weights and bootstrapping estimation were used. Results. Infants born LGA were not found to be at increased risk for poor verbal ability (aOR: 1.16 [0.49,2.72] and aOR: 0.83 [0.37,1.87] for girls and boys, resp.) or externalizing behaviour problems (aOR: 1.24 [0.52,2.93] and aOR: 1.24 [0.66,2.36] for girls and boys, resp.). Social factors were found to impact developmental attainment. Maternal smoking led to an increased risk for externalizing behaviour problems (aOR: 3.33 [1.60,6.94] and aOR: 2.12 [1.09,4.13] for girls and boys, resp.). Conclusion. There is no evidence to suggest that infants born LGA are at increased risk for poor verbal ability or externalizing behaviour problems.

In pediatric chronic illness, improving health-related quality of life (HRQOL) has become one of the most important goals of disease management. Duchenne muscular dystrophy (DMD) is a debilitating, progressive and chronic neuromuscular disorder affecting boys. The purpose of this review is to provide an overview of published research on HRQOL in the pediatric DMD population, describe the instruments used and summarize the study findings. The databases searched were Medline, Embase and PsycInfo. The literature search yielded 167 articles, of which 19 were included in this review. The studies were published between 2005 and 2013 across nine countries. Thirteen different generic and disease-specific measures were used, the most common being the Pediatric Quality of Life 4.0 Generic Core module. HRQOL in boys with DMD is worse than that of healthy peers and children with other chronic illnesses, especially in the physical domains. Boys who are at a more severe stage of the disease reported worse physical HRQOL but not necessarily psychosocial HRQOL than boys at a less severe stage. Traditional clinical outcome measures correlated well only with physical HRQOL. Parents’ proxy-reports of their sons’ HRQOL and the boys’ self-reports had poor concordance. More research is needed to assess trends in HRQOL over time and to elucidate factors that affect HRQOL.

Background: Patient-centered health care for children with inborn errors of metabolism (IEM) and their families is important and requires an understanding of patient experiences, needs, and priorities. IEM-specific patient groups have emerged as important voices within these rare disease communities and are uniquely positioned to contribute to this understanding. We conducted qualitative interviews with IEM patient group representatives to increase understanding of patient and family experiences, needs, and priorities and inform patient-centered research and care. Methods: We developed a sampling frame of patient groups representing IEM disease communities from Canada, the United States, and United Kingdom. With consent, we interviewed participants to explore their views on experiences, needs, and outcomes that are most important to children with IEM and their families. We analyzed the data using a qualitative descriptive approach to identify key themes and sub-themes. Results: We interviewed 18 organizational representatives between February 28 and September 17, 2014, representing 16 IEMs and/or disease categories. Twelve participants voluntarily self-identified as parents and/or were themselves patients. Three key themes emerged from the coded data: managing the uncertainty associated with raising and caring for a child with a rare disease; challenges associated with the affected child’s life transitions, and; the collective struggle for improved outcomes and interventions that rare disease communities navigate. Conclusion: Health care providers can support children with IEM and their families by acknowledging and reducing uncertainty, supporting families through children’s life transitions, and contributing to rare disease communities’ progress toward improved interventions, experiences, and outcomes.

Objective Our objectives were: (1) to examine the association between maternal, fetal, and placental phenotypes of preterm delivery and medically indicated early delivery of singletons during the late preterm and early term periods; and (2) to identify the specific maternal, fetal, and placental conditions associated with these early deliveries. Design Retrospective study. Setting City of London and Middlesex County, Ontario, Canada. Sample Singleton live deliveries, at 34–41 weeks of gestation to women in London and Middlesex. Methods We obtained data from a city-wide perinatal database (2002–2011; n = 25 699). We used multinomial logistic regression for multivariable analyses. Main outcome measure The outcome was the occurrence of medically indicated late preterm (34–36 weeks of gestation) and early term (37–38 weeks of gestation) delivery, versus delivery at full term (39–41 weeks of gestation). Results After controlling for confounding factors, all phenotypes were associated with increased odds of medically indicated late preterm and early term delivery. Within the maternal phenotype, chronic maternal medical conditions were associated with increased odds of medically indicated early term delivery (e.g. for gastrointestinal disease, adjusted odds ratio, aOR 1.72, 95% CI 1.47–2.00; for anaemia, aOR 1.40, 95% CI 1.20–1.63), but not late preterm delivery. Conclusions The aetiology of medically indicated early delivery close to full term is heterogeneous. Patterns of associations suggest slightly different conditions underlying the late preterm and early term phenotypes, with chronic maternal medical conditions being associated with early term delivery but not with late preterm delivery. These results have implications for the prevention of early delivery as well as the identification of high-risk groups among those born early. Tweetable Abstract The aetiology of medically indicated late preterm and early term delivery is heterogeneous.

SummaryObjective To develop and validate a shortened version of the Quality of Life in Childhood Epilepsy Questionnaire (QOLCE). A secondary aim was to compare baseline risk factors predicting health-related quality of life (HRQoL) in children newly diagnosed with epilepsy, as identified using the original and shortened version. Methods Data came from the Health-Related Quality of Life in Children with Epilepsy Study (HERQULES, N = 373), a multicenter prospective cohort study. Principal component analysis reduced the number of items from the original QOLCE, and factor analysis was used to assess the factor structure of the shortened version. Convergent and divergent validity was assessed by correlating the Child Health Questionnaire (CHQ) with the shortened QOLCE. Multiple regression identified risk factors at diagnosis for HRQoL at 24 months. Results A four-factor, higher-order, 55-item solution was obtained. A total of 21 items were removed. The final model represents functioning in four dimensions of HRQoL: Cognitive, Emotional, Social, and Physical. The shortened QOLCE demonstrated acceptable fit: Bentler's Comparative Fit Index = 0.944; Tucker-Lewis Index = 0.942; root mean square approximation = 0.058 (90% CI: 0.056–0.061); weighted root mean square residuals (WRMR) = 1.582, and excellent internal consistency (α = 0.96, subscales α > 0.80). Factor loadings were good (first-order: λ = 0.66–0.93; higher-order λ = 0.66–0.85; p

Aim This study investigated the relationship between quality of life (QoL) and health-related quality of life (HRQoL) and assessed factors other than health that contribute to differences in QoL in young males with Duchenne muscular dystrophy (DMD). Method In this cross-sectional study, QoL and HRQoL measures were completed by 98 parents and 85 children. The Quality of My Life (QoML) questionnaire measured QoL and HRQoL as single-items, and the Pediatric Quality of Life 4.0 Generic Core (PedsQL) questionnaire was used as a multidimensional measure of HRQoL. Simple regression was used to examine the relationship between single-item measures of HRQoL and QoL. Multivariable regression was used to investigate factors that may contribute to difference in QoL and HRQoL. Results While ratings of QoL and HRQoL were significantly correlated with one another, HRQoL only accounted for 21% and 44% of the variability in QoL by child- and parent-reports respectively. None of the factors measured contributed ratings of the child's QoL to be much higher than HRQoL. Interpretation QoL and HRQoL are related but distinct constructs as rated by children with DMD and their parents. Further research is needed to elucidate factors outside HRQoL that contribute to QoL. What this paper adds Quality of life (QoL) and health-related quality of life are distinct concepts rated by young males with Duchenne muscular dystrophy (DMD) and their parents. Factors outside of ‘health’ contribute to overall QoL in the paediatric population with DMD. This article's abstract has been translated into Spanish and Portuguese. Follow the links from the abstract to view the translations.

The diagnosis of epilepsy in children is known to impact the trajectory of their health-related quality of life (HRQOL) over time. However, there is limited knowledge about variations in longitudinal trajectories across multiple domains of HRQOL. This study aims to characterize the heterogeneity in HRQOL trajectories across multiple HRQOL domains and to evaluate predictors of differences among the identified trajectory groups in children with new-onset epilepsy. Data were obtained from the Health Related Quality of Life in Children with Epilepsy Study (HERQULES), a prospective multi-center study of 373 children newly diagnosed with new-onset epilepsy who were followed up over 2years. Child HRQOL and family factors were reported by parents, and clinical characteristics were reported by neurologists. Group-based multi-trajectory modeling was adopted to characterize longitudinal trajectories of HRQOL as measured by the individual domains of cognitive, emotional, physical, and social functioning in the 55-item Quality of Life in Childhood Epilepsy Questionnaire (QOLCE-55). Multinomial logistic regression was used to assess potential factors that explain differences among the identified latent trajectory groups. Three distinct HRQOL trajectory subgroups were identified in children with new-onset epilepsy based on HRQOL scores: "High" (44.7%), "Intermediate" (37.0%), and "Low" (18.3%). While most trajectory groups exhibited increasing scores over time on physical and social domains, both flat and declining trajectories were noted on emotional and cognitive domains. Less severe epilepsy, an absence of cognitive and behavioral problems, lower parental depression scores, better family functioning, and fewer family demands were associated with a "Higher" or "Intermediate" HRQOL trajectory. The course of HRQOL over time in children with new-onset epilepsy appears to follow one of three different trajectories. Addressing the clinical and psychosocial determinants identified for each pattern can help clinicians provide more targeted care to these children and their families.

SummaryObjective The aim of this study was to validate the newly developed shortened Quality of Life in Childhood Epilepsy Questionnaire (QOLCE-55) in a sample of children with drug-resistant epilepsy. Methods Data came from 136 children enrolled in the Impact of Pediatric Epilepsy Surgery on Health-Related Quality of Life Study (PEPSQOL), a multicenter prospective cohort study. Confirmatory factor analysis was used to assess the higher-order factor structure of the QOLCE-55. Convergent and divergent validity was assessed by correlating subscales of the KIDSCREEN-27 with the QOLCE-55. Measurement equivalence of the QOLCE-55 was evaluated using multiple-group confirmatory factor analysis of children with drug-resistant epilepsy from PEPSQOL versus children with new-onset epilepsy from HERQULES (Health-Related Quality of Life in Children with Epilepsy Study). Results The higher-order factor structure of the QOLCE-55 demonstrated adequate fit: Comparative Fit Index (CFI) = 0.948; Tucker-Lewis Index (TLI) = 0.946; Root Mean Square of Approximation (RMSEA) = 0.060 (90% confidence interval [CI] 0.054–0.065); Weighted Root Mean Square Residuals (WRMR) = 1.247. Higher-order factor loadings were strong, ranging from λ = 0.74 to 0.81. Internal consistency reliability was excellent (α = 0.97, subscales α > 0.82). QOLCE-55 subscales demonstrated moderate to strong correlations with similar subscales of the KIDSCREEN-27 (ρ = 0.43–0.75) and weak to moderate correlations with dissimilar subscales (ρ = 0.25–0.42). The QOLCE-55 demonstrated partial measurement equivalence at the level of strict invariance – χ2 (2,823) = 3,727.9, CFI = 0.961, TLI = 0.962, RMSEA = 0.049 (0.044, 0.053), WRMR = 1.834. Significance The findings provide support for the factor structure of the QOLCE-55 and contribute to its robust psychometric profile as a reliable and valid measure. Researchers and health practitioners should consider the QOLCE-55 as a viable option for reducing respondent burden when assessing health-related quality of life in children with epilepsy.

The objective of this study was to examine the association between convulsive status epilepticus (CSE) and health-related quality of life (HRQL) during a 24-month follow-up in a multisite incident cohort of children with epilepsy.Data were collected in the Health-Related Quality of Life Study in Children with Epilepsy Study from 374 families of children with newly diagnosed epilepsy. The Quality of Life in Childhood Epilepsy (QOLCE) Questionnaire was used to evaluate parent-reported child HRQL. Hierarchical linear regression was used to examine the relationship between CSE and HRQL at 24 months postepilepsy. A total of 359 families completed the 24-month assessment.Twenty-two children (6.1%) had experienced CSE during the follow-up. Children with and without CSE were similar, except a larger proportion of children with CSE had partial seizures (p0.001). Controlling for clinical, demographic, and family characteristics, CSE was significantly associated with poorer HRQL (β = -4.65, p = 0.031). The final model explained 47% of the variance in QOLCE scores.The findings suggested that not only do children with CSE have significantly poorer HRQL compared with their non-CSE counterparts, but that this factor is independent of the effects of demographic and clinical features known to affect HRQL.

Objective Patients with epilepsy (PWE) are more likely to have unmet healthcare needs than the general population. This systematic review assessed the reasons for unmet needs in PWE. Methods Medline, Embase, PsycINFO, Cochrane, and Web of Science databases were searched using keywords relating to unmet healthcare needs, treatment barriers, and access to care. The search included all countries, adult and pediatric populations, survey and qualitative studies, but excluded non-English articles and articles published before 2001. Reasons for unmet needs were extracted. Results Nineteen survey and 22 qualitative studies were included. Three survey and five qualitative studies excluded patients with comorbidities. There were twice as many studies on unmet mental healthcare needs than unmet physical care needs in PWE. Poor availability of health services, accessibility issues, and lack of health information contributed to unmet needs in both Western and developing countries. Lack of health services, long wait lists, uncoordinated care, and difficulty getting needed health information were prevalent in the United States (US) as well as countries with a universal healthcare system. However, unmet needs due to costs of care were reported more commonly in studies from the US. Significance This systematic review identified reasons for unmet needs in PWE across different countries, which will inform specific interventions required to address these unmet needs. Unmet needs may have been underestimated due to exclusion of PWE with comorbidities in some studies. Additional studies are needed to understand the contribution of comorbidities on unmet needs and their interaction with caregiver and family factors.

Background: The aim of this study was to elucidate the role of gestational age in determining the risk of neonatal morbidity among infants born late preterm (34-36 weeks) and early term (37-38 weeks) compared with those born full term (39-41 weeks) by examining the contribution of gestational age within the context of biological determinants of preterm birth.Methods: This was a retrospective cohort study. The sample included singleton live births with no major congenital anomalies, delivered at 34-41 weeks of gestation to London-Middlesex (Canada) mothers in 2002-11. Data from a city-wide perinatal database were linked with discharge abstract data. Multivariable models used modified Poisson regression to directly estimate adjusted relative risks (aRRs). The roles of gestational age and biological determinants of preterm birth were further examined using mediation and moderation analyses.Results: Compared with infants born full term, infants born late preterm and early term were at increased risk for neonatal intensive care unit triage/admission [late preterm aRR = 6.14, 95% confidence interval (CI) 5.63, 6.71; early term aRR = 1.54, 95% CI 1.41, 1.68] and neonatal respiratory morbidity (late preterm aRR = 6.16, 95% CI 5.39, 7.03; early term aRR = 1.46, 95% CI 1.29, 1.65). The effect of gestational age was partially explained by biological determinants of preterm birth acting through gestational age. Moreover, placental ischaemia and other hypoxia exacerbated the effect of gestational age on poor outcomes.Conclusions: Poor outcomes among infants born late preterm and early term are not only due to physiological immaturity but also to biological determinants of preterm birth acting through and with gestational age to produce poor outcomes. © The Author 2013; all rights reserved.

OBJECTIVES: Fever is a common reason for an emergency department visit and misconceptions abound. We assessed the effectiveness of an interactive Web-based module (WBM), read-only Web site (ROW), and written and verbal information (standard of care [SOC]) to educate caregivers about fever in their children. METHODS: Caregivers in the emergency department were randomized to a WBM, ROW, or SOC. Primary outcome was the gain score on a novel questionnaire testing knowledge surrounding measurement and management of fever. Secondary outcome was caregiver satisfaction with the interventions. RESULTS: There were 77, 79, and 77 participants in the WBM, ROW, and SOC groups, respectively. With a maximum of 33 points, Web-based interventions were associated with a significant mean (SD) pretest to immediate posttest gain score of 3.5 (4.2) for WBM (P < 0.001) and 3.5 (4.1) for ROW (P < 0.001) in contrast to a nonsignificant gain score of 0.1 (2.7) for SOC. Mean (SD) caregiver satisfaction scores (out of 32) for the WBM, ROW, and SOC groups were 22.6 (3.2), 20.7 (4.3), and 17 (6.2), respectively. All groups were significantly different from one another in the following rank: WBM > ROW > SOC (P < 0.001). CONCLUSIONS: Web-based interventions are associated with significant improvements in caregiver knowledge about fever and high caregiver satisfaction. These interventions should be used to educate caregivers pending the demonstration of improved patient-centered outcomes.

SummaryObjective The objective was to test whether the five-domain structure of the Measure of Processes of Care (MPOC-20) was observed in a sample of children with epilepsy and, if not, to propose adaptations to improve its utility in this population. Methods Data came from the Health-Related Quality of Life in Children with Epilepsy Study (HERQULES)—a multicenter prospective cohort study (n = 374) following children 4–12 years of age for 2 years after diagnosis. Confirmatory factor analysis (CFA) tested the applicability of the five domains/factors in a sample of children with epilepsy approximately 6 months following diagnosis (n = 311). Goodness-of-fit statistics were used to examine sources of ill model fit, and modification indices guided the model modification process where there was strong theoretical rationale for changes. Results The five-factor model described by the originators of the MPOC-20 was found to be inadmissible in children with epilepsy, with four of the five factors demonstrating high correlations (r > 0.85). Upon merging the intercorrelated factors, a two-factor solution with a mediocre fit emerged (Root Mean Square Error of Approximation (RMSEA) = 0.080, Comparative Fit Index (CFI) = 0.902, Standardized Root Mean Square Residual (SRMR) = 0.060). Modification indices identified four items as the source of poor model fit. Removing these four items and reperforming the CFA resulted in an adequate model fit and a revised 16-item MPOC (RMSEA = 0.057, CFI = 0.958, SRMR = 0.036). The two factors are “Family/Care Provider Interaction” and “Providing Information.” Significance Results suggest that the MPOC-16 better reflects family-centered care (FCC) in children with epilepsy than the original MPOC-20. The benefit of having fewer factors is that scoring is simpler and the interpretation of the results is easier. This was the first investigation of the factor structure of the MPOC-20 on a sample entirely composed of children with epilepsy. These results add to evidence that the factor structure (and how family-centered care is delivered and perceived) differs across treatment environments and treatment populations.

SummaryObjective Health-related quality of life (HRQL) is compromised in children with epilepsy. The current study aimed to identify correlates of HRQL in children with drug resistant epilepsy. Methods Data came from 115 children enrolled in the Impact of Pediatric Epilepsy Surgery on Health-Related Quality of Life Study (PEPSQOL), a multicenter prospective cohort study. Individual, clinical, and family factors were evaluated. HRQL was measured using the Quality of Life in Childhood Epilepsy Questionnaire (QOLCE), a parent-rated epilepsy-specific instrument, with composite scores ranging from 0 to 100. A series of univariable linear regression analyses were conducted to identify significant associations with HRQL, followed by a multivariable regression analysis. Results Children had a mean age of 11.85 ± 3.81 years and 65 (56.5%) were male. The mean composite QOLCE score was 60.18 ± 16.69. Child age, sex, age at seizure onset, duration of epilepsy, caregiver age, caregiver education, and income were not significantly associated with HRQL. Univariable regression analyses revealed that a higher number of anti-seizure medications (p = 0.020), lower IQ (p = 0.002), greater seizure frequency (p = 0.048), caregiver unemployment (p = 0.010), higher caregiver depressive and anxiety symptoms (p < 0.001 for both), poorer family adaptation, fewer family resources, and a greater number of family demands (p < 0.001 for all) were associated with lower HRQL. Multivariable regression analysis showed that lower child IQ (β = 0.20, p = 0.004), fewer family resources (β = 0.43, p = 0.012), and caregiver unemployment (β = 6.53, p = 0.018) were associated with diminished HRQL in children. Significance The results emphasize the importance of child cognition and family variables in the HRQL of children with drug-resistant epilepsy. The findings speak to the importance of offering comprehensive care to children and their families to address the nonmedical features that impact on HRQL.

The aim of this study was to utilize bootstrapping to investigate the robustness of latent class trajectories and risk factors of depressive symptoms among mothers of children with epilepsy. Data were obtained from a national prospective cohort study (2004–09) of children newly diagnosed with epilepsy and their families in Canada (n = 339). Latent classes of depressive symptom trajectories were modeled using a semi-parametric group-based trajectory modeling approach. Multinomial logistic regression identified risk factors predicting trajectory group membership. Four trajectories were identified: low stable, borderline, moderate increasing, and high decreasing. Goodness of fit, posterior probabilities, and parameter estimates obtained with bootstrapping were not significantly different from the original sample. Calculation of the root mean square error demonstrated minimal non-ignorable bias for three parameter estimates, which was subsequently removed with additional sampling. Risk factors identified were identical for the original sample and the bootstrap, and differences in odds ratios, as calculated with the method of variance estimation recovery, were not significant. As examined using a bootstrapping procedure, group-based trajectory modeling offers a robust methodology to uncover potential heterogeneity in populations and identify high-risk individuals.