Background and aims: Very young children have a relatively high prevalence of morbidity and mortality. Health care and supportive technology has improved but may require difficult choices and decisions regarding the allocation of these resources in this age group. Cost-effective analysis (CEA) can inform these decisions and thus measurement of Health-Related Quality of Life (HRQoL) is becoming increasingly important. However, the components of HRQoL are likely to be specific to infants and young children. This study aimed to develop a bank of items to inform the possible development of a new proxy report instrument. Methods: A review of the literature was done to define the concepts, generate items and identify measures that might be an appropriate starting point of reference. The items generated from the cognitive interviews and systematic review were subsequently pruned by experts in the field of HRQoL and paediatrics over two rounds of a Delphi study. Results: Based on the input from the different sources, the greatest need for a new HRQoL measure was in the 0-3year age group. The item pool identified from the literature consisted of 36 items which was increased to 53 items after the cognitive interviews. The ranking of items from the first round of the Delphi study pruned this pool to 28 items for consideration. The experts further reduced this pool to 15 items for consideration in the second round. The experts also recommended that items could be merged due to their similar nature or construct. This process allowed for further reduction of items to 11 items which showed content validity and no redundancy. Conclusion: The need for an instrument to measure appropriate aspects of HRQoL in infants and young children became apparent as items included in existing measures did not cover the required spectrum. The identification of the final items was based on a sound conceptual model, acceptability to stakeholders and consideration of the observability of the item selected. The pruned item bank of 11 items needs to be subject to further testing with the target population to ensure validity and reliability before a new measure can be developed.

Background: The EQ-5D-Y Proxy is currently recommended for Health Related Quality of Life (HRQoL) measurement in children aged 4-8 years of age. However, it has only been validated in children over six years of age. The aim of this study was to investigate the performance of the EQ-5D-Y proxy version 1 in children between the ages of 3-6 years. Methods: A sample of 328 children between 3 and 6 years of age were recruited which included children who were either acutely-ill (AI), chronically-ill (CI) or from the general school going population (GP). The EQ-5D-Y Proxy Version 1 and the PedsQL questionnaires were administered at baseline. The EQ-5D-Y Proxy was administered telephonically 24 h later to children with chronic illnesses to establish test-retest reliability. The distribution of dimensions and summary scores, Cohen's kappa, the intraclass correlation coefficient, Pearson's correlation and Analysis of variance were used to explore the reliability, and validity of the EQ-5D-Y for each age group. A single index score was estimated using Latent scores and Adult EQ-5D-3 L values (Dolan). Results: The groups included 3-year olds (n = 105), 4-year olds (n = 98) and 5-years olds (n = 118). The dimension Looking after Myself had the greatest variability between age groups and had the highest rate of problems reported. Worried, Sad or Unhappy and Pain or Discomfort were not stable across time in test-retest analysis. The Visual Analogue Scale (VAS), and single index scores estimated using the latent values and Dolan tariff had good test retest (except for the latent value scores in a small number of 4-year olds). EQ-5D-Y scores for all ages had small to moderate correlations with PedsQL total score. The EQ-5D-Y discriminated well between children with a health condition and the general population for all age groups. Caregivers reported difficulty completing the Looking after Myself dimension due to age-related difficulties with washing and dressing. Conclusion: The dimension of Looking after Myself is problematic for these young children but most notably so in the 3 year old group. If one considers the summary scores of the EQ-5D-Y Proxy version 1 it appears to work well. Known group validity was demonstrated. Concurrent validity was demonstrated on a composite level but not for individual dimensions of Usual Activities or Worried, Sad or Unhappy.. The observable dimensions demonstrated stability over time, with the inferred dimensions (Pain or Discomfort and Worried, Sad or Unhappy) less so, which is to be expected. Further work is needed in exploring either the adaptation of the dimensions in the younger age groups.

Background Despite the high burden of disease in younger children there are few tools specifically designed to estimate Health Related Quality of Life (HRQoL) in children younger than 3 years of age. A previous paper described the process of identifying a pool of items which might be suitable for measuring HRQoL of children aged 0–3 years. The current paper describes how the items were pruned and the final draft of the measure, Toddler and Infant (TANDI) Health Related Quality of Life, was tested for validity and reliability. Methods A sample of 187 caregivers of children 1–36 months of age were recruited which included children who were either acutely ill (AI), chronically ill (CI) or from the general school going population (GP). The TANDI, an experimental version of the EQ-5D-Y proxy, included six dimensions with three levels of report and general health measured on a Visual Analogue Scale (VAS) from 0 to 100. The content validity had been established during the development of the instrument. The TANDI, Ages and Stages Questionnaire (ASQ), Faces, Leg, Activity, Cry, Consolabilty (FLACC) or Neonatal Infant Pain Scale (NIPS) and a self-designed dietary information questionnaire were administered at baseline. The TANDI was administered 1 week later in GP children to establish test-retest reliability. The distribution of dimension scores, Cronbach’s alpha, rotated varimax factor analysis, Spearman’s Rho Correlation, the intraclass correlation coefficient, Pearson’s correlation, analysis of variance and regression analysis were used to explore the reliability, and validity of the TANDI. Results Concurrent validity of the different dimensions was tested between the TANDI and other instruments. The Spearman’s Rho coefficients were significant and moderate to strong for dimensions of activity and participation and significant and weak for items of body functions. Known groups were compared and children with acute illness had the lowest ranked VAS (median 60, range 0–100), indicating worse HRQoL. The six dimensions of the TANDI were tested for internal consistency and reliability and the Cronbach’s α as 0.83. Test-retest results showed no variance for dimension scores of movement and play, and high agreement for pain (83%), relationships (87%), communication (83%) and eating (74%). The scores were highly correlated for the VAS (ICC = 0.76; p < 0.001). Conclusion The TANDI was found to be valid and reliable for use with children aged 1–36 months in South Africa. It is recommended that the TANDI be included in future research to further investigate HRQoL and the impact of interventions in this vulnerable age group. It is further recommended that future testing be done to assess the feasibility, clinical utility, and cross-cultural validity of the measure and to include international input in further development.

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Background Health-Related Quality of Life (HRQoL) data together with clinical findings allow for monitoring of intervention efficacy and the effect on HRQoL. Children with Juvenile Idiopathic Arthritis (JIA) experience symptoms often persisting into adulthood, emphasising the need to track HRQoL. Objectives The aim of this study was to investigate psychometric properties of the EuroQol five-dimensional youth questionnaire (EQ-5D-Y) in children with JIA. Methods A cross-sectional, analytical study design was used. Children 8 to 15 years were recruited, completing the self-report EQ-5D-Y and two other HRQoL questionnaires. Known group validity was established by comparing the effect size between children with different disease severities. Concurrent validity was tested using Kruskal–Wallis to compare the ranking of scores on different questionnaires. Feasibility was assessed by number of missing responses and time to complete each questionnaire. Results All questionnaires were able to distinguish between children with different JIA severity. There was a significant difference in ranking of most Juvenile Arthritis Multidimensional Assessment Report dimension scores across EQ-5D-Y levels, ( p < 0.05), indicating concurrent validity. There was poor concurrent validity with the PedsQL dimensions tested with EQ-5D-Y, except for ‘pain’ ( p = 0.001). The EQ-5D-Y was the quickest to complete with no missing values. Conclusion This study showed that the EQ-5D-Y is valid and feasible in measuring HRQoL in JIA children and adequately responsive to detect change over time. Clinical implications It is quick and easy to use in a busy clinical setting, allowing for effective JIA management monitoring.

BackgroundIn estimating the impact of an intervention, ignoring the effect of improving the health of one member of the caregiver/child dyad on the Health Related Quality of Life (HRQoL) of the other member may lead to an underestimation of the utility gained. This may be particularly true for infants/young children and their caregivers. The aim of this study was to quantify the interaction between the child’s perceived general health as assessed by the newly developed Toddler and Infant Questionnaire (TANDI) on the reporting of the caregiver’s own HRQoL as assessed by the EQ-5D-3 L.MethodsA sample of 187 caregivers participated. A total of 60 caregivers of acutely-ill (AI) and 60 caregivers of chronically-ill (CI) children were recruited from a children’s hospital. The 67 caregivers of general population (GP) children were recruited at a pre-school. Each caregiver completed the proxy rating of their child’s HRQoL on the TANDI (The TANDI is an experimental HRQoL instrument, modelled on the EQ-5D-Y proxy, for children aged 1-36 months), which comprises of six dimensions of health and a rating of general health on a Visual Analogue Scale (VAS). The caregiver completed the EQ-5D-3 L, a self-report measure of their own HRQoL. Forward stepwise regression models were developed with 1) the VAS score of the caregiver and 2) the VAS score of the child as dependent variables. The independent variables for the caregiver included dummy variables for the presence or absence of problems on the EQ-5D-3 L and the VAS score of the child. The independent variables for the child included dummy variables for each TANDI dimension and the VAS of the caregiver.ResultsThe TANDI results indicated that in five of the six dimensions AI children had more problems than the other two groups and the GP children were reported to have a significantly higher VAS than the other two groups. The child’s VAS was significantly correlated with the caregiver’s VAS in all groups, but most strongly in the AI group. The preference based scores (using the UK TTO tariff) were only correlated in the AI group. The inclusion of the child’s VAS increased the variance accounted for 11% of the VAS score of the caregiver. Anxiety and depression was the only dimension which accounted for more variance (18%). Similarly the perceived health state, VAS of the caregiver accounted for 14% of the variance in the child’s VAS, second only to problems with play (25%).ConclusionThere does indeed appear to be a strong relationship between the VAS scores of the children and their caregivers. The perceived general health of the child influences the caregivers reporting of their general health, more than their own report of experiencing pain or discomfort or problems with mobility. Thus, improving the HRQoL of the very young child may improve the caregiver’s HRQoL as well. Conversely, if the caregiver has a lower perceived HRQoL this may result in a decrement in the reported VAS of the child, independent of the presence or absence of problems in the different dimensions. This imp...

IntroductionLittle is known about cystic fibrosis (CF) in low-middle income settings. This study aimed to describe the spectrum and outcomes of CF in South Africa (SA) from the recently established SA CF registry (SACFR).MethodsDemographic, diagnosis and clinical data was extracted from the SACFR. Cross-sectional univariable and multivariable regression analysis of best forced expiratory volume in 1 s (FEV1; age≥6 years) and nutrition (all ages) in 2018 was conducted to investigate factors associated with severe lung disease (SLD; FEV1 ≤3.0 z-score) and undernutrition.ResultsBy December 2018, ancestry of 447 individuals included in the SACFR was Caucasian (315; 70%), mixed (87; 19%) and black African (41; 9%). Median diagnosis age was 7.6 months (IQR 2.7,37.1). Genotype was p.Phe508del homozygous (220; 49%); p.Phe508del heterozygous (144; 32%) and neither p.Phe508del or unknown Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) variant in 83 (19%); the second most frequent CFTR variant was 3120+1G>A, common in black Africans. Median age of patients in 2018 was 14.7 years (IQR 7.4,24.4). SLD was independently associated with chronic methicillin resistant S.aureus (MRSA) (aOR 16.75; 95% CI 1.74–161.50), undernutrition (aOR 5.20; 95% CI 2.23–12.13) and age (aOR 2.23 per 10-years; 95% CI 1.50–3.31). Undernutrition was associated in univariable analysis with low weight at diagnosis, non-caucasian ancestry, chronic P.aeruginosa infection and lower socioeconomic status.ConclusionInterventions targeting MRSA infection and nutrition are needed to improve CF outcomes in SA. Most people with CF in SA are eligible for highly effective CFTR modulator therapy.

Objective The aim of this study is a head-to-head comparison of the instrument performance and responsiveness of the EQ-5D-Y-3L and the expanded English version of the EQ-5D-Y-5L in children/adolescents receiving acute orthopaedic management in South Africa. Methods Children/adolescents aged 8–15 years completed the EQ-5D-Y-5L, EQ-5D-Y-3L, self-rated health (SRH) question and PedsQL at baseline. The EQ-5D-Y-5L, EQ-5D-Y-3L and SRH question were repeated after 24 and 48 h. Performance of the EQ-5D-Y-5L and EQ-5D-Y-3L was determined by comparing feasibility (missing responses), redistribution of dimensions responses, discriminatory power, concurrent validity, and responsiveness. Results Eighty-three children/adolescents completed baseline measures and seventy-one at all three time-points. Reporting of 11111 decreased by 20% from the EQ-5D-Y-3L to the EQ-5D-Y-5L. Informativity of dimensions improved on average by 0.267 on the EQ-5D-Y-5L with similar evenness. There was a range of 11–27% inconsistent responses when moving from the EQ-5D-Y-3L to the EQ-5D-Y-5L. There was a low to moderate and significant association on the EQ-5D-Y-3L and EQ-5D-Y-5L to similar items on the PedsQL and SRH scores. Percentage change over time was greater for the EQ-5D-Y-5L (range 0–182%) than EQ-5D-Y-3L (range 0–100%) with the largest reduction for both measures between 0 and 48 h. For those who respondents who showed an improved SRH the EQ-5D-Y-5L and EQ-5D-Y-3L showed significant paired differences. Conclusion The English version of the EQ-5D-Y-5L appears to be a valid and responsive extension of the EQ-5D-Y-3L for children receiving acute orthopaedic management. The expanded levels notably reduce the ceiling effect and has greater discriminatory power. Concurrent validity of the EQ-5D-Y-3L and EQ-5D-Y-5L was low to moderate with similar PedsQL items and SRH. The EQ-5D-Y-5L generally showed greater change than the EQ-5D-Y-3L across all dimensions with the greatest change observed for 0–48 h. Responsiveness was comparable across the EQ-5D-Y-3L and EQ-5D-Y-5L for those with improved SRH. Greater sensitivity to change may be observed on comparison of utility scores, once preference-based value sets are available for the EQ-5D-Y-5L.