Proxy-reported Health-related Quality Of Life Research Articles

Proxy-reported health-related quality of life of children with systemic lupus erythematosus and well-being of caregivers: a cross-sectional survey among caregivers in China.

This study comprehensively analyzes proxy-reported HRQoL of SLE children in China and identifies multiple associated factors. The caregiver's well-being was correlated to the children's HRQoL. We emphasize the necessity for comprehensive support for children with SLE, their caregivers, and extended family members. • Systemic lupus erythematosus (SLE) may impact the health-related quality of life for both the affected children and their families. • Most of such studies were conducted in developed countries. • This study provided comprehensive data on health-related quality of life of SLE children and wellbeing of their caregivers in China.

Health-related quality of life and caregiver burden of pediatric patients with inborn errors of metabolism in Japan using EQ-5D-Y, PedsQL, and J-ZBI.

Inborn errors of metabolism (IEM) are known with poor long-term health concerns; however, the health-related quality of life (HRQoL) and the burden placed on families remain unclear. This study investigated the self- and proxy-reported HRQoL of pediatric patients with IEM with or without developmental disabilities and the burden placed on their caregivers. Patients with IEM aged 8-15years and their caregivers were asked to respond to the Pediatric Quality of Life Inventory (PedsQL), EuroQoL five-dimension questionnaire for younger populations (EQ-5D-Y), and Japanese version of the Zarit Caregiver Burden Interview (J-ZBI). We compared EQ-5D-Y scores with matched EQ-5D-Y population norms. Intraclass correlation coefficients (ICC) for self and proxy HRQoL scores of those without developmental disabilities were calculated. Correlation coefficients of HRQoL proxy responses with J-ZBI score were estimated. We included 66 patients with IEM (mean age, 11.5years; males, 41.2%) in the study. The mean (± standard deviation) EQ-5D-Y scores without and with developmental disabilities were 0.957 (± 0.071) and 0.821 (± 0.175), respectively. The EQ-5D-Y scores significantly increased compared with the reference values (p < 0.01, effect size = 0.337). The ICC values were 0.331 and 0.477 for the EQ-5D-Y and PedsQL scores, respectively. HRQoL proxy scores had strong negative correlations with J-ZBI scores. The HRQoL of patients with IEM without developmental disabilities in our study was similar to that of the general Japanese population. The HRQoL of patients with IEM with developmental disabilities was low and associated with a tendency towards an increased burden of care.

Comparing patient- and proxy-reported health-related quality of life of frail older patients at the emergency department

PurposeTo examine agreement between patient- and proxy-reported Health-Related Quality of Life (HRQoL) scores, and their associations with clinical measures and patient demographics.MethodsWe examined the patient-proxy agreement in HRQoL within a cohort of community-dwelling frail older patients (Clinical Frailty Scale [CFS] score 4–6) planned for discharge from the Emergency Department (ED) of a 1700-bed tertiary hospital in Singapore. Patients were recruited as part of the Emergency Department Interventions for Frailty (EDIFY) program, a quasi-experimental study evaluating the effectiveness of a multicomponent frailty intervention (MFI) in maintaining or improving functional outcomes. HRQoL was measured using the EuroQoL five-dimensional questionnaire (EQ-5D-5L) for patients and proxies. We analysed a sample of dyadic-reported HRQoL to explore the inter-rater agreement using intraclass correlation coefficients (ICC), and the influencing factors of concordance.ResultsWe recruited 140 participants with 23 pairs of patient-proxy dyads having concurrent EQ-5D reported. There was poor inter-rater agreement between patient- and proxy-reported EQ-5D (ICC 0.18) with proxies perceiving poorer average HRQoL than patients. On the domain level, there was poor agreement in self-care and pain domains (ICC − 0.07 and 0.20), where proxies perceived more problems than patients. In multivariate regressions, lower frailty scores were associated with poorer agreement in EQ-5D scores (p < 0.05).ConclusionIn conclusion, our findings demonstrate differences between patient- and proxy-reported HRQoL outcomes among frail older patients, with significant discordance observed among patients who are “less frail”. Hence, proxy-reported responses should be interpreted with care especially when the abovementioned characteristics are present in an older patient.

Examining health-related quality of life in ambulatory adult patients with chronic heart failure: insights from Malaysia using EQ-5D-5L.

Health-related quality-of-life (HRQoL) data for the chronic heart failure (HF) population in Malaysia are lacking. Using EQ-5D-5L, this study intended to describe their HRQoL, identify predictors of worse HRQoL, and derive EQ-5D-5L index scores for use in economic evaluations. A cross-sectional survey was conducted between April and September 2023 to collect EQ-5D-5L, sociodemographic, and clinical data from outpatients with HF across seven public specialist hospitals in Malaysia. Multivariable logistic and linear regression models were used to identify independent predictors of reported problems in the EQ-5D-5L dimensions, and predictors of index scores and EQ-VAS, respectively. EQ-5D-5L data from 424 outpatients of multi-ethnic background (mean age: 57.1years, 23.8% female, mean left ventricular ejection fraction: 35.7%, 89.7% NYHA class I-II) were collected using either Malay, English, or Chinese, achieving a 99.8% completion rate. Nearly half of the respondents reported issues in the Mobility, Usual Activities, and Pain/Discomfort dimensions. Mean EQ-5D-5L index was 0.820, lower than the general population, and significantly lower with NYHA class III-IV (0.747) versus NYHA class I (0.846) and NYHA class II (0.805). Besides NYHA class, independent predictors of worse HRQoL included Indian ethnicity, living alone, lower education, unemployment due to ill-health, and proxy-reported HRQoL, largely aligning with existing literature. Community-dwelling Malaysians with HF reported poorer HRQoL compared to the general population. The observed disparities in HRQoL among HF patients may be linked to specific patient characteristics, suggesting potential areas for targeted interventions. HRQoL assessment using EQ-5D-5L proves feasible and should be considered for routine implementation in local clinics.

Health-related quality scores in childhood interstitial lung disease: Good agreement between patient and caregiver reports.

Childhood interstitial lung disease (chILD) is a heterogeneous group of mostly chronic respiratory disorders. Assessment of health-related quality of life (HrQoL) in chILD has become increasingly important in clinical care and research. The aim of this study was to assess differences between patient-reported (self) and caregiver-reported (proxy) HrQoL scores. This study used data obtained from the chILD-EU Register. After inclusion (baseline), the patient's health status was followed up at predefined study visits. At each study visit, caregivers and patients were handed validated, age-specific HrQoL questionnaires. HrQoL data entered at baseline were used to compare self- and proxy-reported HrQoL scores. For the longitudinal analysis, we compared HrQoL scores between the baseline and the next follow-up visit. No differences between patient- and caregiver-reported HrQoL scores were found for school functioning, chILD-specific questionnaire score, and physical health summary score. Self-reported HrQoL scores were higher for the subscales emotional functioning (77.4 vs. 70.7; p < .001), social functioning (81.9 vs. 76.2; p < .001), as well as psycho-social summary score (76.5 vs. 71.8; p < .001) and total score (74.7 vs. 70.8; <.001). The longitudinal analysis showed that a significant change in a patient-reported HrQoL score resulted in a significant change in a caregiver-reported HrQoL score after a mean time of 11.0 months (SD 9.4). We found a good agreement between children- and caregiver-related HrQoL scores. In chILD, caregivers are able to sense changes in children's HrQoL scores over time and may be used as a proxy for children unable to complete HrQoL questionnaires.

Health-related quality of life for pediatric patients with end-stage kidney disease: A systematic review and meta-analysis of the Pediatric Quality of Life Inventory (PedsQL).

Health-related quality of life (HRQoL) studies demonstrate the impact of end-stage renal disease (ESRD) on the physical and psychosocial development of children. While several instruments are used to measure HRQoL, few have standardized domains specific to pediatric ESRD. This review examines current evidence on self and proxy-reported HRQoL among pediatric patients with ESRD, based on the Pediatric Quality of Life Inventory (PedsQL) questionnaires. Following PRISMA guidelines, we conducted a systematic review and meta-analysis on HRQoL using the PedsQL 4.0 Generic Core Scale (GCS) and the PedsQL 3.0 ESRD Module among 5- to 18-year-old patients. We queried PubMed, Embase, Web of Science, CINAHL, and Cochrane databases. Retrospective, case-controlled, and cross-sectional studies using PedsQL were included. Of 435 identified studies, 14 met inclusion criteria administered in several countries. Meta-analysis demonstrated a significantly higher total HRQoL for healthy patients over those with ESRD (SMD:1.44 [95% CI: 0.78-2.09]) across all dimensional scores. In addition, kidney transplant patients reported a significantly higher HRQoL than those on dialysis (PedsQL GCS, SMD: 0.33 [95% CI: 0.14-0.53]) and (PedsQL ESRD, SMD: 0.65 [95% CI: 0.39-0.90]) concordant with parent-proxy reports. Patients with ESRD reported lower HRQoL in physical and psychosocial domains compared with healthy controls, while transplant and peritoneal dialysis patients reported better HRQoL than those on hemodialysis. This analysis demonstrates the need to identify dimensions of impaired functioning and produce congruent clinical interventions. Further research on the impact of individual comorbidities in HRQoL is necessary for developing comprehensive, integrated, and holistic treatment programs.

Patient and Caregiver Outcomes After Onasemnogene Abeparvovec Treatment: Findings from the Cure SMA 2021 Membership Survey.

Onasemnogene abeparvovec (OA) is the only gene replacement therapy currently approved for spinal muscular atrophy (SMA) treatment. We sought to assess real-world patient and caregiver outcomes after OA treatment for SMA. Patients who received OA were identified from the 2021 Cure SMA Membership Survey. Those treated at 6-23months of age were matched to non-patients treated with OA on the basis of age at the time of survey and survival motor neuron2 gene copy number. Patient characteristics, motor milestones, and resource and supportive care use, as well as caregiver proxy-reported health-related quality of life (HRQOL), were described. Caregiver unmet needs and HRQOL were also assessed. Of the 614 patients in the survey, 64 received OA, and 17 were matched with 28 non-OA-treated patients. In general, a greater percentage of OA-treated patients achieved various motor milestones, including 100% sitting without support and 58.8% walking with assistance. OA-treated patients also had numerically lower rates of hospitalization and surgery. None required tracheostomy with a ventilator. The rate of using oxygen or a breathing machine for more than 16h was also lower for OA-treated patients. OA-treated patients had less frequent trouble swallowing. HRQOL was reported to be similar to non-OA-treated patients. Caregivers of OA-treated patients reported better patient mobility scores and less work impairment. The study suggests that treatment with OA is associated with greater rates of motor milestone achievements and less resource and supportive care use for patients with SMA treated at 6-23months of age in the real world. For caregivers, it may also potentially reduce unmet needs, improve HRQOL, and reduce work impairment.

Relationship between survivorship care plans and unmet information needs, quality of life, satisfaction with care, and propensity to engage with, and attend, follow-up care.

The impact of survivorship care plans (SCPs) on the proximal and distal outcomes of adult and childhood cancer survivors, and parent proxies, is unclear. This study aimed to determine the relationship between SCP receipt and these outcomes. A cross-sectional survey of adult and childhood cancer survivors (and parent proxies for survivors aged younger than 16years) across Australia and New Zealand was conducted. Multivariate regression models were fitted to measure the impact of SCP receipt on proximal (unmet information needs and propensity to engage with, and attend, cancer-related follow-up care) and distal outcomes (quality of life and satisfaction with cancer-related follow-up care) with control for cancer history and sociodemographic factors. Of 1123 respondents, 499 were adult cancer survivors and 624 were childhood cancer survivors (including 222 parent proxies). We found that SCP receipt was predictive of greater attendance at, and awareness of, cancer-related follow-up care (adult: odds ratio [OR],2.46; 95% CI,1.18-5.12; OR,2.38; 95% CI,1.07-5.29; child/parent: OR,2.61; 95% CI,1.63-4.17; OR,1.63; 95% CI,1.06-2.50; respectively). SCP receipt also predicted fewer unmet information needs related to "follow-up care required" and "possible late effects" (adult: OR, 0.44; 95% CI,0.20-0.96; OR,0.29; 95% CI,0.13-0.64; child/parent: OR,0.46; 95% CI,0.30-0.72; OR,0.57; 95% CI, 0.38-0.85; respectively). In terms of distal outcomes, SCP receipt predicted a better global quality of life for adult cancer survivors (β,0.08; 95% CI,-0.01-7.93), proxy-reported health-related quality of life (β,0.15; 95% CI,0.44-7.12), and satisfaction with follow-up care for childhood cancer survivors (OR,2.93; 95% CI,1.64-5.23). Previous studies have shown little impact of SCPs on distal end points. Results suggest that SCPs may be beneficial to cancer survivors' proximal and distal outcomes.

Health-related quality of life of children with neurofibromatosis type 1: Analysis of proxy-rated PedsQL and CHQ questionnaires

This study aims to (1) investigate health-related quality of life (HRQoL) in children with Neurofibromatosis Type 1 (NF1) using the Pediatric Quality of Life inventory (PedsQL) and the Child Health Questionnaire (CHQ); and (2) compare the psychometric properties and content of these questionnaires in NF1 patients. PedsQL and CHQ proxy-reports were administered to parents/caregivers of 160 patients with NF1 aged 5-12 years. HRQoL scores were compared with Dutch population norms using independent t-tests. Psychometric properties (feasibility and reliability) were assessed by floor/ceiling effects and Cronbach's alpha coefficient. A principal component analysis (PCA) with varimax rotation was performed to identify the data's internal structure. By content mapping, we identified unique constructs of each questionnaire. Proxy-reported HRQoL was significantly lower on all PedsQL subscales for children aged 5-7 years, and on 4/6 subscales for children aged 8-12 years compared to norms. Significantly lower HRQoL was reported on 6/14 CHQ subscales (children 5-7 years) and 9/14 subscales (children 8-12 years). The PedsQL showed slightly better feasibility and reliability. The PCA identified two components, representing psychosocial and physical aspects of HRQoL, explaining 63% of total variance. Both questionnaires showed relevant loadings on both components. The CHQ subscales concerning parents and family were considered unique contributions. Proxy-reported HRQoL of children with NF1 is significantly lower compared to norms on multiple domains. Both questionnaires adequately measure HRQoL in children with NF1. However, the PedsQL has slightly better psychometric properties, while the CHQ covers a unique dimension of HRQoL associated with disease impact on parents and family.

Epilepsy and proxy-reported health-related quality of life in children and young people with non-ambulatory cerebral palsy.

To assess the association between epilepsy characteristics and proxy-reported health-related quality of life (HRQoL) in children and young people with non-ambulatory cerebral palsy (CP) and seizures. This was a cross-sectional study of 164 children and young people (74 females, 90 males; mean age 10 years 6 months, range 2-21 years, SD 5 years 5 months). Caregivers completed the Child Health Index of Life with Disabilities (CPCHILD) in an outpatient setting. We utilized univariable linear regression and multivariable modeling to study relationships between variables and CPCHILD scores. Gross Motor Function Classification System levels were 37% IV and 63% V. Sociodemographic factors included the Child Opportunity Index (median 51, interquartile range [IQR] 25-80). A median of 2 (IQR 1-3) antiseizure medications (ASMs) were used, and days with seizures ranged from 0 (30%) to 28 (20%) days in the previous 4 weeks. Total CPCHILD scores decreased 2.3 points for each ASM (95% confidence interval [CI] -4.1 to -0.42). Compared to persons with focal epilepsy, those with generalized epilepsy had lower total CPCHILD scores (-5.7; 95% CI -11 to -0.55). Number of days with seizures was not associated with total CPCHILD scores. Proxy-reported HRQoL was affected by epilepsy-specific features in children and young people with severe CP. Health-related quality of life (HRQoL) was lower with increasing numbers of antiseizure medications. Overall quality of life (QoL) scores were lower by a similar amount, independent of seizure frequency. HRQoL was lower in persons with recent hospital admissions for epilepsy.

QOL-05. Cardiorespiratory fitness and health-related quality of life amongst survivors of childhood central nervous system tumours.

INTRODUCTION: Due to recent advancements in surgical techniques, imaging and adjuvant therapies, survival rates for children with central nervous system (CNS) tumours have improved. Research priorities have now shifted to focus on late-effects and quality of survivorship amongst childhood CNS tumour survivors. Our study aimed to assess levels of cardiorespiratory fitness and health-related quality of life (HRQoL) amongst survivors of childhood CNS tumours. A secondary aim was to investigate potential relationships between cardiorespiratory fitness and HRQoL. METHODS: Participants were recruited from the National Children’s Cancer Service in CHI Crumlin. Inclusion criteria included: diagnosis of a primary CNS tumour, aged between 6-17 years, between 3 months and 5 years post completion of oncology treatment, independent mobility, deemed clinically appropriate to participate by treating oncologist. Cardiorespiratory fitness was assessed using the six-minute walk test. HRQoL was assessed with the PedsQL Generic Core Scales, Version 4.0, both self-report and parent proxy report forms were used. RESULTS: 20 participants (n=9 male) were recruited with a mean age of 12.34 (SD = 3.46) years. Mean time since completion of oncology treatment was 2.31 (SD = 1.38) years. Mean 6-minute walk distance (6MWD) was 482.75 (SD = 50.04) metres, equating to the 5.55th (SD = 6.83th) percentile overall. 6MWD was significantly reduced compared to predicted 6MWD (t = -12.52, p <.001, 95% CI [-163.52, -116.68]). Parent proxy-reported HRQoL was significantly reduced compared to healthy population norms (t = -5.82, p <0.001, 95% CI [-25.76, -12.17]). A strong positive correlation exists between 6MWD and both parent-proxy (Pearson’s r = 0.533, p = 0.015) and child-reported HRQoL (r = 0.580, p = 0.007). CONCLUSION: Survivors of childhood CNS tumours present with impaired levels of cardiorespiratory fitness and HRQoL compared to healthy population norms. Higher levels of cardiorespiratory fitness are associated with higher levels of HRQoL.

Caregiver burden, and parents' perception of disease severity determine health-related quality of life in paediatric patients with intoxication-type inborn errors of metabolism

BackgroundLiving with a non-acute (phenylketonuria) or acute (e.g. urea cycle disorders, organic acidurias) intoxication-type inborn error of metabolism (IT-IEM) can have a substantial impact on health-related quality of life (HrQoL) of paediatric patients and their families. Parents take primary responsibility for treatment monitoring and experience worry and fear about their child's health status. Quantitative evidence on parental psychological factors which may influence the HrQoL of patients with IT-IEM are sparse to non-existent. MethodsIn this multicenter survey study 50 parents of IT-IEM patients (ages 5–19) assessed the severity of their child's disease, reported on caregiver burden, and proxy-rated their child's HrQoL. Additionally, 35 patient self-reports on HrQoL were obtained (n = 16 female patients, n = 19 male patients). Multiple linear regressions were conducted to examine the predictive power of child age, sex, medical diagnosis type (acute / non-acute), parental perceived disease severity and caregiver burden on patients' HrQoL. Mediation analyses were used to investigate the relation of caregiver burden and parental ratings of disease severity with patients' HrQoL. ResultsSignificant regression models for self-reported [F(5,34) = 10.752, p < .001, R2 adj.. = 0.59] and parent proxy reported HrQoL [F(5,49) = 20.513, p < .001, R2 adj.. = 0.67] emerged. High caregiver burden and perceived disease severity predicted significantly lower patient self- and proxy-reported HrQoL while type of diagnosis (acute versus non-acute) did not. Female sex predicted significantly lower self-reported HrQoL. High caregiver burden was the mediating factor between high perceived severity of the child's disease and lower proxy- by parent rated HrQoL. ConclusionDetecting elevated burden of care and providing support for parents seems crucial to prevent adverse consequences for their children's HrQoL. Intervention studies are needed, to assess which support programs are most efficient.

Health-Related Quality of Life in Adolescents and Young Adults with Heart Failure

<h3>Purpose</h3> In adult heart failure populations, health-related quality of life (HRQOL) is predictive of changes in health status and outcomes including mortality and hospitalization. Despite a growing population of pediatric and young adult patients with heart failure, HRQOL in this population has not been well described. This study aimed to describe self- and proxy-reported HRQOL in adolescents and young adults (AYA) with heart failure. <h3>Methods</h3> AYAs (12-24 years) with heart failure or a significant post-heart transplant complication and a caregiver were enrolled at a single center. AYA/parent dyads completed a self- or proxy-reported version of the PedsQL 4.0 Generic Core. Group comparisons based on primary cardiac diagnosis leading to heart failure were conducted. Patient demographic and disease characteristics were examined as correlates of HRQOL. <h3>Results</h3> 53 patient-caregiver dyads participated. Patients were 64% male; 76% White. Post-transplant with life threatening complication (26%), single ventricle heart disease (SVHD) (23%), anthracycline induced cardiomyopathy (21%) and dilated cardiomyopathy (DCM) (17%) were the most common diagnoses. Disease burden was high with 42% experiencing extra-cardiac disease and 34% having >11 hospitalizations. AYAs with transplant complications, SVHD, and DCM and their caregivers endorsed significantly impaired total HRQOL when compared to population norms. Conversely, AYAs with anthracycline cardiomyopathy and their caregivers reported normative HRQOL that was significantly better than the other diagnostic groups (p=0.04). Across all diagnostic groups, female sex (p=0.01) and white race (p=0.01) were associated with poorer physical functioning HRQOL. <h3>Conclusion</h3> Many AYAs with heart failure and their caregivers report impaired HRQOL, however certain cardiac diagnoses and demographic groups were associated with better reported HRQOL. Further studies are needed to understand mediators of risk for poor HRQOL in this population, as well as the role of potential resiliency focused interventions to improve HRQOL.

Patient-Proxy Agreement Regarding Health-Related Quality of Life in Survivors with Lymphoma: A Propensity-Score Matching Analysis.

Objective: To assess the difference between lymphoma survivors' self- and proxy-reported health-related quality of life (HRQoL) and its association with socioeconomic and health statuses. Methods: The data used in this study were obtained from a nationwide cross-sectional online survey in 2019. Information about participants' demographics, health status and HRQoL were collected. The propensity-score matching (PSM) method was used to control the effect of potential confounders on selection bias. A chi-squared test, one-way analysis of variance, and multiple linear regression models were used to assess the relationship between HRQoL and response type adjusted to respondents' background characteristics. Results: Out of the total 4400 participants, data of 2350 ones were elicited for analysis after PSM process. Patients' self-reported outcomes indicated a slightly better physical, role and emotional functioning than proxy-reported outcomes. Regression analysis showed that patients, who were older, unemployed, and who received surgery, were more likely to report a lower HRQoL. Further analysis demonstrated that proxy-reported patients who had completed treatment were more likely to report a higher HRQoL than those who were being treated. Conclusions: Our study demonstrates that the agreement between self- and proxy-reported HRQoL is low in patients with lymphoma and the heterogeneities of HRQoL among patients with different types of aggressive NHL (Non-Hodgkin's lymphoma) is large. Differences in self- and proxy-reported HRQoL should be considered by oncologists when selecting and deciding the optimal care plan for lymphoma survivors.

The association between birth weight and proxy-reported health-related quality of life among children aged 5\u2009\u2212\u200910 years old: A linked data analysis

BackgroundBirth weight has a substantial effect on children’s cognitive development, physical capability, and emotional development, which in turn impact on Health-Related Quality of Life (HRQoL). Generally, evidence indicates that children born with low birth weight tend to have poorer proxy-reported HRQoL, particularly at school age. However, there is limited evidence on whether variation in HRQoL exists across the entire range of possible birth weights. This study aimed to examine the association between birth weight and proxy-reported HRQoL among children aged 5–10 years old.MethodsData from the 1973–78 cohort of the Australian Longitudinal Study on Women’s Health were linked with state-based Perinatal Data Collections and the Mothers and their Children’s Health study for 1,589 mothers and 2,092 children aged 5 − 10 years old. Generalized estimating equations were used to model the association between birth weight and proxy-reported HRQoL measured by the Pediatric Quality of Life Inventory 4.0. Results are presented as odds ratios with 95 % confidence intervals.ResultsIn this study, 15.61 % of children were at risk of impaired proxy-reported HRQoL. Each 100-gram increase in birth weight was associated with a 3 % reduction in the odds of impaired HRQoL (AOR = 0.97; 95 % CI: 0.94, 0.99). However, there was only limited evidence of an effect within the normal birth weight range (AOR = 0.97; 95 % CI: 0.94, 1.01).ConclusionsThe findings indicate that increased birth weight was protective against impaired HRQoL, although there was limited evidence of variability within the normal birth weight range. This study contributes to the existing literature by not only emphasizing the impact of low birth weight on children’s health and health-related outcomes but also by focusing on the variability within the normal birth weight range, particularly in a setting where low birth weight is less prevalent.

Caregiver burden and proxy-reported outcomes of people without natural speech: a cross-sectional survey study

ObjectiveTo examine interrelations between care-related burden on informal caregivers and their proxy assessments of outcomes in people without natural speech.DesignA cross-sectional survey.SettingData were collected in January 2019 from a postal...

Evaluation of caregiver burden and coping strategies in parents of paediatric familial Mediterranean fever patients in relation to illness severity, therapy and health-related quality of life.

The study aimed to determine the caregiver burden and coping strategies in caregivers of familial Mediterranean fever (FMF) patients in relation to illness severity, therapy and health-related quality of life (HRQoL). The study included 171 paediatric FMF patients and their caregivers (parents). The caregivers were asked to complete a socio-demographic form, the Zarit caregiver burden interview (ZCBI) and the Brief COPE. The patients and their caregivers were asked to complete the KINDer Lebensqualitätsfragebogen questionnaire (self-report and proxy report, respectively) for assessing HRQoL. The patients were categorised according to their disease activity (mild, moderate or severe) and the presence or absence of anti-IL-1 therapy. The mean ZCBI score of the caregivers was 44.7 ± 13.5. ZCBI and COPE scores did not differ significantly between the caregivers of FMF patients receiving and not receiving anti-IL-1 therapy. However, dysfunctional COPE (p = 0.039) and ZCBI (p = 0.021) scores showed a significant difference between the caregivers in relation to patient's disease severity. ZCBI scores were positively correlated with dysfunctional coping (p = 0.01). Self-reported HRQoL disease module scores were lower for the patients who received anti-IL-1 therapy than for those did not (p = 0.009). Proxy-reported (p < 0.001) and self-reported (p = 0.043) HRQoL disease module scores were lower for the patients with severe disease activity. As the caregiver burden increases, parents tend to use a dysfunctional coping strategy. Good control of disease activity with administration of medical therapy can reduce the disease severity, thereby decrease the caregiver burden, and secondly help to reduce the usage of dysfunctional coping in caregivers.

Cognitive functioning and pain interference mediate pain predictive effects on health-related quality of life in pediatric patients with Neurofibromatosis Type 1

The objective was to investigate the serial mediating effects of perceived cognitive functioning and pain interference in daily living in the relationship between perceived pain and overall generic health-related quality of life (HRQOL) in children, adolescents, and young adults with Neurofibromatosis Type 1 (NF1). The Pain, Cognitive Functioning, and Pain Impact Scales from the PedsQL Neurofibromatosis Type 1 Module and the PedsQL 4.0 Generic Core Scales were completed in a multi-site national study by 323 patients ages 5-25 and 335 parents. A serial multiple mediator model analysis was conducted to test the hypothesized sequential mediating effects of cognitive functioning and pain interference as intervening variables in the association between pain as a predictor variable and overall generic HRQOL. Pain predictive effects on overall generic HRQOL were serially mediated by cognitive functioning and pain interference. In predictive analytics models utilizing hierarchical multiple regression analyses with age and gender demographic covariates, pain, cognitive functioning and pain interference accounted for 66% of the variance in patient-reported generic HRQOL and 57% of the variance in parent proxy-reported generic HRQOL (P<0.001), reflecting large effect sizes. Cognitive functioning and pain interference explain in part the mechanism of pain predictive effects on overall generic HRQOL in pediatric patients with NF1. Identifying NF1-specific pain, cognitive functioning, and pain interference as salient predictors of overall generic HRQOL from the patient and parent perspective facilitates a family-centered orientation to the comprehensive care of children, adolescents, and young adults with NF1.

The impact of long-term ventilator-use on health-related quality of life and the mental health of children with neuromuscular diseases and their families: need for a revised perspective?

BackgroundLife extension by medical interventions and health-related quality of life (HRQOL) are sometimes conflicting aspects of medical care. Long-term ventilation in children with neuromuscular disease is a well-established life-extending procedure and often at the center of this conflict. HRQOL and the mental health of affected children and their families become even more important in respect to emerging therapies in neuromuscular diseases with longer life-expectancy of treated patients and considerable costs of medical treatment.MethodsWe performed a questionnaire survey in a total of forty-three families of children with neuromuscular disease treated in the University Medical Center Hamburg-Eppendorf and the Children’s Hospital Altona. We evaluated self- and proxy-reported HRQOL and mental health outcomes of affected children and their parents using validated and age-appropriate instruments.ResultsCompared to normative data, children with neuromuscular diseases and their families experienced a lower HRQOL and mental health. However, there was no additional negative influence on the overall HRQOL by ventilator use.ConclusionsAs ventilator use was not responsible for the reduction of HRQOL and mental health our data contributes an important aspect to the discussion about life-prolonging procedures, in particular mechanical ventilation, in severly disabled patients.

What predicts the proxy-reported health-related quality of life of adolescents with cerebral palsy in Bangladesh?

BackgroundThe health-related quality of life (HRQoL) of adolescents with CP in low and middle-income countries is often poor, as is the case in Bangladesh. This exploratory study examined what factors predict the proxy-reported HRQoL of adolescents with CP in rural Bangladesh, a typical low- and middle-income country (LMIC).MethodsAdolescents with CP (10 to 18y) were identified using the Bangladesh Cerebral Palsy Register. HRQoL was assessed using the Cerebral Palsy Quality of Life-Teens proxy-report questionnaire (CPQoL-Teens), adolescent mental health using the Strengths and Difficulty Questionnaire (SDQ) and caregiver mental health using the Depression, Anxiety and Stress Scale (DASS-21). Theoretical and statistical interests (i.e. bivariate analysis, p < 0.05) identified potential predictors which were entered into hierarchical multiple linear regression (HMLR) models in order of clinical significance; HMLR related adolescent clinical characteristics, adolescent and caregiver mental health and proxies of socioeconomic status to CPQoL-Teens dimensions.ResultsOne hundred fifty-four adolescents with CP (mean age 15y 1mo, SD 1y 8mo, female 31.2%) participated in this study.Twenty-four factors were identified to explore for relationship to adolescent proxy-reported HRQoL. Fifteen of the factors correlated to one or more CPQoL-Teens dimension; strongest correlation was between ‘feelings about functioning’ and motor impairment (r = 0.545). Nine were predictive of CPQoL-Teens dimensions; adolescent sex, school attendance, severity of motor impairment, hearing and speech impairment, mother’s education, primary caregiver depression and stress, and having a sanitary latrine at home resulting in score changes of between 0.79 (95% CI 0.24 to 1.35) to 35.1 (95% CI 6.03 to 64.22).ConclusionsMany of the factors predicting the proxy-reported HRQoL of adolescents with CP are amenable to intervention, and have the potential to improve adolescent wellbeing. Several determinants are priorities of the sustainable development goals (SDGs); these findings should inform resource prioritization to improve the wellbeing of adolescents with CP in Bangladesh and other LMICs.