The PedsQL™ in pediatric rheumatology: Reliability,validity, and responsiveness of the Pediatric Quality of Life Inventory™ generic core scales and rheumatology module

Objective

The Pediatric Quality of Life Inventory (PedsQL) is a modular instrument designed to measure health‐related quality of life (HRQOL) in children and adolescents ages 2–18 years. The 23‐item PedsQL 4.0 Generic Core Scales are multidimensional child self‐report and parent proxy‐report scales developed as the generic core measure to be integrated with the PedsQL disease‐specific modules. The 22‐item PedsQL 3.0 Rheumatology Module was designed to measure pediatric rheumatology–specific HRQOL. This study was undertaken to demonstrate the reliability, validity, and responsiveness of the PedsQL 4.0 Generic Core Scales and the PedsQL 3.0 Rheumatology Module in pediatric rheumatology.

Methods

The 4 PedsQL 4.0 Generic Core Scales (physical, emotional, social, and school functioning) and the 5 PedsQL 3.0 Rheumatology Module scales (pain and hurt, daily activities, treatment, worry, and communication) were administered to 231 children and 244 parents (271 subjects accrued overall) recruited from a pediatric rheumatology clinic.

Results

Internal consistency reliability for the PedsQL Generic Core total scale score (α = 0.91 for child self report, α = 0.93 for parent proxy report), physical health summary score (α = 0.87 for child self report, α = 0.89 for parent proxy report), and psychosocial health summary score (α = 0.86 for child self report, α = 0.90 for parent proxy report) were acceptable for group comparisons. The Rheumatology Module scales also demonstrated acceptable reliability for group comparisons (α = 0.75–0.86 for child self report, α = 0.82–0.91 for parent proxy report). Validity was demonstrated using the known‐groups method. The PedsQL distinguished between healthy children and children with rheumatic diseases as a group. The responsiveness of the PedsQL was demonstrated through patient change over time as a result of clinical intervention.

Conclusion

The results demonstrate the reliability, validity, and responsiveness of the PedsQL 4.0 Generic Core Scales and the PedsQL 3.0 Rheumatology Module in pediatric rheumatology.

     

The PedsQL Multidimensional Fatigue Scale in pediatric rheumatology: reliability and validity

OBJECTIVE:. The PedsQL (Pediatric Quality of Life Inventory) is a modular instrument designed to measure health related quality of life (HRQOL) in children and adolescents ages 2-18 years. The recently developed 18-item PedsQL Multidimensional Fatigue Scale was designed to measure fatigue in pediatric patients and comprises the General Fatigue Scale (6 items), Sleep/Rest Fatigue Scale (6 items), and Cognitive Fatigue Scale (6 items). The PedsQL 4.0 Generic Core Scales were developed as the generic core measure to be integrated with the PedsQL Disease-Specific Modules. The PedsQL 3.0 Rheumatology Module was designed to measure pediatric rheumatology-specific HRQOL. Methods. The PedsQL Multidimensional Fatigue Scale, Generic Core Scales, and Rheumatology Module were administered to 163 children and 154 parents (183 families accrued overall) recruited from a pediatric rheumatology clinic. Results. Internal consistency reliability for the PedsQL Multidimensional Fatigue Scale Total Score (a = 0.95 child, 0.95 parent report), General Fatigue Scale (a = 0.93 child, 0.92 parent), Sleep/Rest Fatigue Scale (a = 0.88 child, 0.90 parent), and Cognitive Fatigue Scale (a = 0.93 child, 0.96 parent) were excellent for group and individual comparisons. The validity of the PedsQL Multidimensional Fatigue Scale was confirmed through hypothesized intercorrelations with dimensions of generic and rheumatology-specific HRQOL. The PedsQL Multidimensional Fatigue Scale distinguished between healthy children and children with rheumatic diseases as a group, and was associated with greater disease severity. Children with fibromyalgia manifested greater fatigue than children with other rheumatic diseases. CONCLUSION: The results confirm the initial reliability and validity of the PedsQL Multidimensional Fatigue Scale in pediatric rheumatology.     

Reliability,Validity, and Responsiveness of the Pediatric Quality of Life Inventory™ (PedsQL™) Generic Core Scales and Asthma Symptoms Scale in Vulnerable Children With Asthma

Background. Patient-reported outcomes such as health-related quality of life (HRQOL) are increasingly used as primary endpoints in clinical trials. The Pediatric Quality of Life Inventory? (PedsQL?) is widely used as a measure of HRQOL and may be a particularly suitable primary outcome in pediatric asthma clinical trials. Objectives. To examine the reliability, validity, and responsiveness to clinical change of the PedsQL? 4.0 Generic Core Scales and PedsQL? Asthma Module Asthma Symptoms Scale in a sample of vulnerable children with persistent asthma recruited from Federally Qualified Health Centers. Methods. Children (N = 252; ages 3 to 14 years) with persistent asthma (27% mild, 40.9% moderate, 32.1% severe) and their parents (93.7% mother, 83.3% Hispanic, 76.9% Spanish-speaking, 72.6% less than a high school diploma) enrolled in a clinical trial completed the PedsQL? 4.0 Generic Core Scales, the PedsQL? 3.0 Asthma Module Asthma Symptoms Scale, and a measure of asthma symptom frequency (used as an indicator of clinical change) at baseline and 3-month follow-up. Results. The PedsQL? demonstrated adequate internal consistency reliability and convergent and discriminative validity. Based on intra- and intersubject change, effect sizes, and standard errors of measurement, the PedsQL? demonstrated responsiveness to clinical change. Conclusions. For both child self-report and parent proxy-report, the PedsQL? Generic Core Scales Total Scale score and the PedsQL? Asthma Symptoms Scale are suitable for use as primary asthma clinical trial outcomes.     

Differences in disease outcomes between medicaid and privately insured children: possible health disparities in juvenile rheumatoid arthritis

OBJECTIVE: To determine the relationship between health insurance status and disease outcome in children with juvenile rheumatoid arthritis (JRA). METHODS: JRA patients followed at a tertiary pediatric rheumatology center were assessed for the number of active joints and number of joints with limited range of motion. Disease activity, patient well-being, and pain were measured. Disability was assessed by the Childhood Health Assessment Questionnaire, health-related quality of life by the Pediatric Quality of Life Inventory (PedsQL) Generic Core Scale, and the PedsQL Rheumatology Module. Health care resource utilization was estimated based on the number of billing events for health services coded in administrative databases; these databases also provided information on patient health insurance status. Children insured by Medicaid or similar state programs for low-income families were considered to have Medicaid status. Disease outcomes of children with Medicaid status was compared with that of children with private health insurance. RESULTS: Forty (14%) of the 295 children with JRA had Medicaid status. Patients with Medicaid status were more often of nonwhite race (P < or = 0.04) and more frequently had a polyarticular or systemic disease course (P = 0.04) compared with other patients (n = 255). After correction for differences in disease duration, race, JRA onset, and JRA course between groups, children with Medicaid status continued to have significantly higher disability (P < 0.0003), and lower mean PedsQL Generic Core Scale scores (P < 0.05), while health resource utilization appeared similar between groups. CONCLUSION: Despite apparently similar health resource utilization and joint involvement, Medicaid status is associated with significantly lower health-related quality of life and higher disability in JRA.     

False-positive interpretations in respiratory cytopathology

The objective of the present study was to determine the reliability and validity of the Persian translation of the Pediatric Quality of Life Inventory (PedsQL?) 4.0 Generic Core Scales Young Adult Version in an Iranian sample of young adult patients with rheumatoid arthritis (RA). One hundred ninety-seven young adult patients with RA completed the 23-item PedsQL? and the 36-item Short-Form Health Survey (SF-36). Disease activity based on Disease Activity Score 28 was also measured. Internal consistency and test–retest reliability, as well as construct, discriminant, and convergent validity, were tested. Confirmatory factor analysis (CFA) was used to verify the original factor structure of the PedsQL?. Also, responsiveness to change in PedsQL? scores over time was assessed. Cronbach’s alpha coefficients ranged from α?=?0.82 to α?=?0.91. Test–retest reproducibility was satisfactory for all scales and the total scale score. The PedsQL proved good convergent validity with the SF-36. The PedsQL distinguished well between young adult patients and healthy young adults and also RA groups with different comorbidities. The CFA did not confirm the original four-factor model, instead, analyses revealed a best-fitting five-factor model for the PedsQL? Young Adult Version. Repeated measures analysis of variance indicated that the PedsQL scale scores for young adults increased significantly over time. The Persian translation of the PedsQL? 4.0 Generic Core Scales Young Adult Version demonstrated good psychometric properties in young adult patients with RA and can be recommended for the use in RA research in Iran.     

Children and Adolescents with Repaired Tetralogy of Fallot Report Quality of Life Similar to Healthy Peers

Objective. The study aims to evaluate and compare self‐reported and parent proxy‐reported quality of life (QOL) in pediatric patients with repaired tetralogy of Fallot (TOF) and determine relationships with residual disease. Design. QOL was prospectively evaluated in children/adolescents with repaired TOF and parents' proxy report using the Pediatric Quality of Life Inventory Generic Core and Cardiac Module scales. The scores were compared with published self and parent proxy‐reported normative data for children considered healthy, chronically ill, and with congenital heart disease. Recent clinical data were reviewed for correlations between QOL and residual disease severity. Results. Twenty child–parent pairs were assessed at median age of 10.9 years (range 8.4–18.7 years). Self‐report was higher than parent proxy report. Compared with peers, self‐reported QOL was higher than for chronically ill children (overall QOL 85 vs. 77, P= 0.007) and similar to healthy children (85 vs. 83, P= 0.44), while proxy report by parent was similar to parents of chronically ill children (overall QOL 77 vs. 74, P= 0.035). Despite moderate pulmonary regurgitation (mean 35%) and right ventricular dilation (mean 114 mL/m²), 76% had a New York Heart Association class of 1, normal B‐natriuretic peptide (24 pg/mL), reasonable exercise tolerance VO₂ max% predicted (mean 77%), and preserved right ventricular ejection fraction (mean 58%, range 44–80%). Overall QOL positively correlated with child's VO₂ max% predicted, when reported by child (r = 0.47, P < 0.05) and parent proxy (r = 0.63, P < 0.05). Conclusions. QOL in children/adolescents with repaired TOF is not proportional to the severity of their residual disease. Self‐reported QOL appears similar to healthy peers while parent proxy reported lower QOL. For both children and parents, QOL positively correlated with the child's exercise capacity. Therefore, comprehensive follow‐up should include cardiac rehabilitation and psychosocial evaluation to ensure an active lifestyle, improve health perception, and prevent later acquired heart disease.     

Quality of life in children with Type 1 diabetes: a comparison of general and diabetes‐specific measures and support for a unitary diabetes quality‐of‐life construct

Aims To assess the factor structure of the Pediatric Quality of Life Inventory (PedsQL) Diabetes Module and to compare the PedsQL general and diabetes‐specific quality of life (QOL) measures regarding psychometric properties and relations to relevant outcomes. Methods The instruments were completed by 447 children age 9 to 15.5 years with Type 1 diabetes > 1 year from four US paediatric diabetes clinics; parents completed the parallel parent‐proxy measures. Principal components factor analysis was used to examine the factor structure of the PedsQL diabetes module. Analyses of the generic and diabetes QOL measures included psychometric properties, parent–child correlations and correlations with depression, adherence and glycated haemoglobin (HbA_1c). Results The factor structure of the PedsQL diabetes module did not support the original five subscales. Both one‐ and two‐factor models were supported; however, parallel parent and child subscales did not emerge. While the generic and diabetes‐specific measures of QOL were moderately to highly correlated with each other, the constructs were differentially associated with relevant diabetes outcomes. Generic QOL was more highly associated with depression than diabetes QOL. Conversely, diabetes QOL was more highly associated with adherence and HbA_1c, although this was seen to a greater extent for parent‐proxy report than for child report. Conclusions Factor analysis of the PedsQL diabetes module supports the use of a total diabetes QOL score. Findings regarding the associations of the generic and diabetes modules with diabetes outcomes underscore the unique contribution provided by both generic and diabetes QOL.     

The reliability and validity of the pediatrics quality of life inventoryTM (PedsQL) diabetes module 3.0 for Turkish adolescents with type 1 diabetes

Adolescents might experience difficulties in compliance to diabetes management and their quality of life might be adversely affected. No specific quality of life scale is available in Turkey for adolescents aged 13–18 years with diabetes. The aim of the study was to examine the psychometric properties of the PedsQL^TM 3.0 Diabetes Module (PedsQL^TM 3.0 DM) Teen and Parent Reports (Ages 13–18) in Turkish adolescents with type 1 diabetes. One hundred and four adolescents aged 13–18 years with type 1 diabetes and their parents who presented at a diabetes center participated in this study. Content, construct, and concurrent validities, internal consistency reliability and item-total correlations were analyzed in order to determine the psychometric properties of the scales. Construct validity was evaluated with explanatory factor analysis. The factor structure of the Turkish version PedsQL^TM diabetes module consisted of four subscales and did not support the original five-factor structure. Concurrent validity was analysed with Spearman Correlation and a positive significant correlation was found between PedsQL^TM 3.0 DM and PedsQL^TM 4.0 Generic Core Scales. Cronbach's alpha was found to be 0.92 for child self-reports (CSR) and 0.88 for parent proxy-report (PPR) of PedsQL^TM 3.0 DM. The psychometric analyses of the Turkish version of PedsQL^TM 3.0 DM Teen and Parent Reports indicate high reliability and good content and construct validity for measuring diabetes-specific health related quality of life in Turkish adolescents with type 1 diabetes.     

Multicenter validation of a new quality of life measure in pediatric lupus

OBJECTIVE: Pediatric systemic lupus erythematosus (SLE) is a chronic fluctuating disease that significantly impacts quality of life (QOL). There is no pediatric SLE-specific health-related QOL (HRQOL) scale. Our objective was to develop and validate a new pediatric SLE-specific HRQOL scale. METHODS: We developed the Simple Measure of the Impact of Lupus Erythematosus in Youngsters (SMILEY) based on results of qualitative research of children with SLE and their parents. SMILEY has parallel child and parent reports with a 5-faces scale for responses. SMILEY comprises 4 domains: effect on self, limitations, social, and burden of SLE. In this cross-sectional study, we examined face, content, construct, and concurrent validity; internal consistency; test-retest reliability; and child-parent agreement for SMILEY. Children /=0.4); test-retest reliability (intraclass correlation coefficient [ICC] 0.9); and internal consistency (alpha = 0.9). Moderate agreement was found between child and parent SMILEY reports (ICC 0.7, r(s) = 0.5, P < 0.001). CONCLUSION: SMILEY is a brief, easily understood, valid, and reliable pediatric SLE-specific QOL scale. Because SMILEY assesses children's self-perception of QOL as impacted by SLE, we predict that it will have great utility in clinical practice, clinical trials, and outcomes research.     

Disease activity and fatigue in juvenile idiopathic arthritis

Objective

To examine the association between parent/proxy‐ and child‐reported fatigue and disease activity in children with polyarticular, extended oligoarticular, and persistent oligoarticular juvenile idiopathic arthritis (JIA).

Methods

We enrolled a cross‐sectional cohort of 309 children recruited from the Seattle Children's Hospital rheumatology clinic from 2009–2011. Parents and children completed the PedsQL Multidimensional Fatigue Scales. The parent/proxy, child, and/or physician provided additional disease activity data at each clinic visit, including active joint count, pain, and the Childhood Health Assessment Questionnaire (C‐HAQ). Disease activity was dichotomized as active or inactive using the American College of Rheumatology provisional criteria for clinically inactive disease. The Juvenile Arthritis Disease Activity Score (JADAS) was also calculated. Linear regression was used to examine the associations between fatigue and disease activity.

Results

Associations among fatigue, clinically inactive disease, and the JADAS were not statistically significant after controlling for pain. In the multivariable models of fatigue, the C‐HAQ and parent/child‐reported disease activity were significantly associated with fatigue; however, only the C‐HAQ remained significantly associated after adjustment for pain. The C‐HAQ and parent/child‐reported disease activity explained 17% and 30% of the variance in fatigue for the parent/proxy‐ and child‐reported multivariable models, respectively.

Conclusion

In this cohort, functional ability, as measured by the C‐HAQ, was significantly associated with fatigue. Child‐ and parent/proxy‐reported pain were important confounders of the relationship between fatigue and disease activity. Routinely incorporating pain and fatigue into interventional and observational trials of JIA will enable better delineation of the relationships between these variables.     

Disease control and health‐related quality of life in juvenile idiopathic arthritis

Objective

To examine variability in health‐related quality of life (HRQOL) in children with juvenile idiopathic arthritis (JIA) experiencing no or minimal clinical symptoms, and in a subgroup with polyarticular JIA treated with biologic agents for 12 months.

Methods

We defined 3 samples using a database of patients ages 2–18 years with JIA (n = 524; patient visits [PV] = 2,354): visits (PV = 2,155) with no or minimal clinical symptoms on at least 1 of 4 measures (active joint count, pain, physician global disease rating, Childhood Health Assessment Questionnaire); visits (PV = 941) with no or minimal symptoms on all 4 measures; and children (n = 31) with polyarticular JIA treated with biologic agents for 12 months. HRQOL was measured using the Pediatric Quality of Life Inventory (PedsQL) and the percentage of patients with suboptimal HRQOL was determined.

Results

In PV with a PedsQL score, suboptimal HRQOL by self‐report occurred in 362 (20.6%) PV with at least 1 indicator of minimal symptoms, and in 64 (7.9%) PV with all 4 measures indicating minimal symptoms (519 [25.7%] and 95 [10.7%], respectively, by parent report). For children with polyarticular JIA treated for 12 months with biologic agents, 7 (25.9%) patients by self‐report and 10 (35.7%) patients by parent report were in the suboptimal range of HRQOL.

Conclusion

A substantial percentage of patients with JIA who report no or mild clinical symptoms experience suboptimal HRQOL. This is also true for patients with polyarticular JIA treated with biologic agents for 12 months. Although disease activity and clinical symptoms are related to HRQOL, considerable unexplained variation in HRQOL exists. HRQOL needs to be assessed independently regardless of clinical status.

     

Insomnia and quality of life in children referred for limb pain

OBJECTIVE: Children with limb pain have significantly diminished quality of life. Although this could result directly from the pain, we investigated the extent to which associated insomnia may contribute. METHODS: A consecutive series of pediatric rheumatology clinic patients (age 3-18 yrs) who presented for initial evaluation of limb pain were offered participation. Parents and children, as appropriate, completed the Pediatric Sleep Questionnaire and Pediatric Quality of Life Inventory (PedsQL 4.0). Validated measures of pain duration and current pain level were provided by the children. Subjects were judged to have substantial insomnia if they had at least 2 of the following symptoms: difficulty falling asleep at night, waking more than twice on average, trouble falling back to sleep, or waking in the morning feeling unrefreshed. Linear regression was used to model the total PedsQL 4.0 score on insomnia, pain duration, and pain level. RESULTS: Seventy-four subjects were recruited (47 girls, mean age 10 +/-3.9); 25 (33%) had juvenile idiopathic arthritis and 40 (54%) had insomnia. A low PedsQL 4.0 score was predicted by insomnia (p < 0.001), but not by pain duration or level (each p > 0.10). Neither pain level nor duration differed significantly between subjects with or without insomnia (each p > 0.10). CONCLUSION: Significant insomnia may affect half of the children who present to a pediatric rheumatology clinic for limb pain. Quality of life in this setting may depend more on insomnia than on current level or duration of pain.     

Prospective,comparative effectiveness trial of cow's milk elimination and swallowed fluticasone for pediatric eosinophilic esophagitis

Eosinophilic esophagitis (EoE) is a chronic, immune‐mediated disease in which food antigens play a key role. Current therapeutic options are limited to long‐term steroid medication and dietary elimination of multiple foods, each of which is challenging. Our objective was to compare single food elimination of cow's milk to swallowed fluticasone in pediatric EoE patients. This is a prospective, comparative effectiveness trial of newly diagnosed EoE patients (ages 2–18 years) treated with swallowed fluticasone (n = 24) or elimination of cow's milk (n = 20). The dual outcome measures of repeat esophageal biopsy (6–8 weeks) and change in Pediatric Quality of Life Inventor (PedsQL) EoE Module and Symptoms Scales were used to assess response to treatment. After 6–8 weeks of treatment, peak esophageal eosinophil counts decreased to below the threshold of 15 eosinophils/high‐power field in 64% of patients treated with cow's milk elimination and 80% of patients treated with swallowed fluticasone (P = 0.4). Mean PedsQL EoE Module total scores (69 vs. 82; P < 0.005) and Total Symptoms scores (58 vs. 75; P = 0.001) showed significant improvement with cow's milk elimination. Among children treated with swallowed fluticasone, mean PedsQL EoE Module total scores (64 vs. 75; P < 0.05) and Total Symptoms scores (58 vs. 69; P < 0.01) were also significantly improved after 6–8 weeks of therapy. Removal of cow's milk from the diet is an effective single food elimination treatment for pediatric patients with EoE as assessed by statistically significant histologic and symptomatic improvement. Cow's milk elimination may be more desirable for EoE patients who do not want to take chronic, long‐term steroid medications.     

Factors influencing the quality of life of Moroccan patients with juvenile idiopathic arthritis

The aim of our study is to investigate the factors influencing the quality of life, assessed by the Pediatric Quality of Life Inventory 4.0 (PedsQL4) Generic Score Scales, in Moroccan patients with juvenile idiopathic arthritis. This is a cross-sectional study conducted between January and June 2012, covering children with juvenile idiopathic arthritis (JIA) seen at the consultations of El Ayachi Hospital and Children’s Hospital of the University Hospital of Rabat. Quality of life is assessed by the PedsQL4 which is a questionnaire composed of 23 items, completed by the child and the parent; the response to each item ranges from 0 to 100, so that higher scores indicate a better quality of life. The functional impact is assessed by the Childhood Health Assessment Questionnaire (CHAQ), and the disease activity by the number of tender and swollen joints, visual analogue scale (VAS) activity, erythrocyte sedimentation rate (ESR), and C-reactive protein. Forty-seven patients are included; the average age of the patients is 11?±?3.35 years, and 40.4 % are females, with a median disease duration of 4 (2; 6) years. The oligoarticular form presents 26.7 %, the systemic form 24.4 %, and the enthesic form 22.2 %. The median of PedsQL4 is 80.43 (63.19; 92.93), and the median of the CHAQ is 0 (0; 1). Our study shows that some clinical and biological characteristics have significant effects on PedsQL by both parent and child reports. This study suggests that the achievement of the quality of life of our patients with JIA depends on the disease activity measured by swollen joints, the number of awakenings, parent VAS, physician VAS, patient VAS, and the ESR.     

Stress and quality of life among parents of children with congenital heart disease referred for psychological services

Objective

The study examined parent stress and health‐related quality of life (HRQOL) among families of children with congenital heart disease (CHD) referred for psychological services.

Methods

Parents of 54 children (85% boys) aged 3 to 13 (M_age = 7.48, SD = 2.38) completed measures to assess parenting stress (Parenting Stress Index – Short Form; Pediatric Inventory for Parents) and the PedsQL Family Impact Module. Medical information was retrieved from medical record review.

Results

Half of parents of children with single ventricle anatomy had clinically significant levels of parenting stress. Parents of children with single ventricle anatomy reported more frequent illness‐related stress and more difficulty dealing with illness‐related stress than parents of children with two ventricle anatomy. Younger gestational age at birth and referral for attention or behavior problems were associated with greater likelihood of parent at‐risk psychosocial functioning.

Conclusions

Among children referred for psychological services, many parents report significant stress and significant negative impact of the child's medical condition on the family. Results underscore the need to consider assessing parent psychosocial functioning and providing additional support for parents of children with CHD.     

Health related quality of life in Middle Eastern children with beta-thalassemia

ABSTRACT: BACKGROUND: Thalassemia is a common disorder worldwide with a predominant incidence in Mediterranean countries, North Africa, the Middle East, India, Central Asia, and Southeast Asia. Whilst substantial progress has been made towards the improvement of Health related quality of life (HRQoL) in western countries, scarce evidence-based data exists on HRQol of thalassemia children and adolescents living in developing countries. Patients and methods We studied 60 thalassemia children from Middle Eastern countries with a median age of 10 years (range 5 to 17 years). HRQoL was assessed with the Pediatric Quality of Life Inventory (PedsQL) 4.0. The Questionnaire was completed at baseline by all patients and their parents. The agreement between child-self and parent-proxy HRQoL reports and the relationship between HRQoL profiles and socio-demographic and clinical factors were investigated. RESULTS: The scores of parents were generally lower than those of their children for Emotional Functioning (mean 75 vs 85; p = 0.002), Psychosocial Health Summary (mean 70.3 vs 79.1; p = 0.015) and the Total Summary Score (mean 74.3 vs 77.7 p = 0.047). HRQoL was not associated with ferritin levels, hepatomegaly or frequency of transfusions or iron chelation therapy. Multivariate analysis showed that a delayed start of iron chelation had a negative impact on total PedsQL scores of both children (p = 0.046) and their parents (p = 0.007). CONCLUSIONS: The PedsQL 4.0 is a useful tool for the measurement of HRQoL in pediatric thalassemia patients. This study shows that delayed start of iron chelation has a negative impact on children's HRQOL.     

Predictors of Health-Related Quality of Life in Children with Cyanotic Heart Disease Who Underwent Palliative and Total Repair

Background: Studies on predictors of health-related quality of life (HRQOL) in pediatric patients with cyanotic heart disease who are waiting for the next stage and those who have undergone total repair are scarce. Therefore, we aimed to identify such predictors in children who received the modified Blalock–Taussig shunt (MBTS) and those who underwent total repair. Methods: In this historical cohort and concurrent follow-up study, data of children who underwent MBTS at the age of 0–3 years between January 2005 and December 2016 at a super-tertiary care hospital in Southern Thailand were obtained. Children who were alive in December 2017 were recruited to evaluate the quality of life at least 1 year after their operation. Between January and December 2018, the “Pediatric Quality of Life Inventory 4.0 Generic Core Scales” with both child self-report and parent proxy-report scores were used to examine the HRQOL. Multivariate linear regression analysis was performed to identify independent predictors of HRQOL. Beta-coefficient (β) and 95% confidence intervals (95% CIs) were calculated and considered statistically significant at p < 0.05. Results: Among the 380 enrolled children, 148 died, 122 survived and waited for total repair, and 110 survived after total repair. In the multivariate analysis, chronic lung disease was a common predictor of lower physical and psychosocial HRQOL reported by the parents (β [95% CI]: −0.42 [−0.81, −0.03] and −0.49 [−0.89, −0.09], respectively). Total repair was a predictor of higher physical HRQOL according to both parents and children (β [95% CI]: 0.33 [0.09, 0.57] and 0.70 [0.36, 1.03], respectively). A predictor of higher psychosocial HRQOL reported by the parents was younger age during MBTS surgery compared with older age (β [95% CI]: 0.012 [0.001, 0.022]). In the total repair subgroup, undergoing the Fontan procedure (vs. Glenn procedure) was a predictor for lower physical HRQOL reported by the parents (β [95% CI]: −0.82 [−1.52, −0.13]). Higher socioeconomic status was a predictor of both physical and psychosocial HRQOL (β [95% CI]: 0.018 [0.001, 0.034] and 0.012 [0.0001, 0.04], respectively). Conclusions: Successful total repair was a predictor of higher physical HRQOL, and younger age during MBTS surgery was a predictor of higher psychological HRQOL in children with cyanotic heart disease. Higher socioeconomic status was a predictor of both physical and psychological HRQOL following total repair [Thai Clinical Trials Registry: TCTR20161221003].     

Development and initial validation of composite parent- and child-centered disease assessment indices for juvenile idiopathic arthritis

Objective

To develop and validate a parent‐centered and a child‐centered composite disease assessment index for juvenile idiopathic arthritis (JIA): the Juvenile Arthritis Parent Assessment Index (JAPAI) and the Juvenile Arthritis Child Assessment Index (JACAI), respectively.

Methods

The JAPAI and the JACAI include 4 measures: parent/child rating of overall well‐being, pain, physical function, and health‐related quality of life (HRQOL). Validation analyses were conducted on nearly 5,000 patients and included assessment of construct validity, discriminant validity, responsiveness to change, and reliability. Besides the 4‐item version, a 3‐item version of both indices, which did not include HRQOL, was tested.

Results

The JAPAI and the JACAI demonstrated good construct validity, yielding high correlations with the Juvenile Arthritis Disease Activity Score and moderate correlations with physician global rating and joint counts. Correlations obtained for the JAPAI and the JACAI and for the 4‐item and the 3‐item versions were comparable. Factorial analysis by principal component analysis showed that both indices are monodimensional. Both the JAPAI and JACAI discriminated well between different disease states and courses and between different levels of American College of Rheumatology Pediatric criteria in a clinical trial, and revealed fair responsiveness to clinical change. Internal consistency was satisfactory, with a Cronbach's alpha of >0.80 in all but 1 of the patient samples tested.

Conclusion

The JAPAI and the JACAI were found to be valid instruments for assessment of disease status in JIA and suitable surrogates of physicians' evaluations. Both indices are potentially applicable in clinical practice, observational studies, and therapeutic trials.     

Facilitating patient-centered care: the development of illustrated multidimensional patient-reported outcome measures for children/adolescents with juvenile idiopathic arthritis

Clinical Rheumatology - To assess the validity, reliability, comprehensibility, and responsiveness to change of an illustrated child/parent multidimensional patient-reported outcome measures...     

Quality of life of asthmatic children and adolescents: Portuguese translation,adaptation, and validation of the questionnaire “Pediatric Quality of Life (PedsQL) Asthma Module”

Objective: The objectives of the study were to translate, validate, and verify the psychometric properties of the Portuguese version of the instrument “Pediatric Quality of Life Asthma Module” (PedsQL Asthma) culturally adapted for the Brazilian culture. Methods: After being translated to Portuguese and being culturally adapted, the questionnaire was answered by 200 asthmatic children and adolescents (aged 2–18) as well as the adults responsible for them. Validation required the use of the following instruments: PedsQL Asthma Children (applied to children and adolescents), PedsQL Asthma Parents (applied to adults responsible for children and adolescents), Pediatric Asthma Quality of Life (PAQLQ), Asthma Control Test (ACT) or Childhood Asthma Control Test (C-ACT), as well as socioeconomic and personal information questionnaires. A group of 45 clinically stable children repeated the questionnaires 15–60 days after answering the first questionnaire. Results: Correlations between the scores of PedsQL Children and PedsQL Parents (r = 0.67), PedsQL Children and PAQLQ (r = 0.66), and PedsQL Parents and PAQLQ (r = 0.64) were moderate and significant. Correlations were higher for men (r = 0.72) when analyzing the children's and parents’ answers to PedsQL according to gender. The 5- to 7-year-old age group had the strongest correlations with PAQLQ (r = 0.79). Cronbach's alpha coefficient for PedsQL Children and Parents had values of 0.85 and 0.87, respectively. A high concordance was observed in both tests at different times, with kappa values of 0.89 and 0.87 for PedsQL Children and Parents, respectively. Conclusion: The instrument used in this study was considered valid, consistent, and reproducible and has acceptable psychometric properties for the Brazilian population.