Quality of life in children at different stages of chronic kidney disease in a developing country.

BACKGROUND: Children with chronic kidney disease (CKD) require comprehensive assessments, including medical and quality of life (QoL) evaluations. Few studies have been conducted in developing countries. METHODS: This cross-sectional study included 2-18-year-old patients who were categorized into 4 groups: the CKD stage 2-3, stage 4-5, stage 5 with dialysis (D), and kidney transplantation (KT) groups. QoL was measured using the Pediatric Quality of Life Inventory™ (PedsQL™) version 4.0; relationships between different factors and QoL were determined using multivariable linear regression analysis. RESULTS: Eighty-seven patients (mean age: 13.3 (4.1) years) were included. The self-reported total scores were 77.5 (12.5), 78.9 (11.2), 77.4 (16.2), and 76.1 (10.9) in the stage 2-3, stage 4-5, stage 5D and KT groups, respectively. Parent-reported scores showed a weak-to-moderate correlation with self-reported scores (r = 0.12-0.42), with total scores of 71.8 (12.7), 69.5 (14.9), 63.4 (14.8), and 70.8 (18.1) in the stage 2-3, 4-5, 5D and KT groups, respectively. Multivariable linear regression revealed that the parent-reported score in the stage 5D group was 15.92 points lower than that in the stage 2-3 group (p = 0.02); the score in the low maternal education group was 10.13 points lower than that in the high maternal education group (p = 0.04). CONCLUSIONS: Parent-reported scores showed weak-to-moderate correlation with self-reported scores. Patients with CKD stage 5D and patients with low maternal education had lower QoL. Regular QoL assessment is recommended for patients with advanced CKD and those with socioeconomic vulnerabilities.

Influencing factors for pediatric eye disorders and health related quality of life: a cross-sectional study in Shanghai, China.

Background: Myopia, strabismus, and ptosis are common pediatric eye diseases, which have a negative impact on children and adolescents in terms of visual function, mental health, and health-related quality of life (HRQoL). Therefore, this study focused on those pediatric eye diseases by analyzing their risk factors and HRQoL for the comprehensive management of myopia, strabismus, and ptosis. Methods: A total of 363 participants (2-18 years old) were included in this study for risk factors analysis of myopia, strabismus, and ptosis. We collected demographic characteristics, lifestyle habits and eye care habits of these children and analyzed them by using univariable and multivariable logistic regression. In addition, we applied the Chinese version of Pediatric Quality of Life Inventory-Version 4.0 (PedsQL 4.0) to assess HRQoL in 256 children with strabismus and ptosis. Univariable and multivariable linear regression models were applied to evaluate potential influencing factors of HRQoL. Results: Of all the participants, 140 had myopia, 127 had strabismus, and 145 had ptosis. Based on the multivariable logistic regression analysis model, we found that the history of parental myopia and daily average near-distance eye usage time were risk factors for myopia, and increased body mass index (BMI) was identified as a risk factor for strabismus and ptosis. Individuals with ptosis possessed decreased HRQoL. The multivariable linear regression model suggested that daily average near-distance eye usage time, light intensity during visual tasks, and daily average sleep duration had potential influences on HRQoL. Conclusion: This is the first study to assess the risk factors and HRQoL of myopia, strabismus, and ptosis together. We identified risk factors for these common pediatric eye diseases to help doctors, parents, and teachers better manage them. Our study discovered that children with eye disorders exhibit a notably diminished HRQoL. Consequently, it emphasizes the necessity for increased social attention and mental health assistance for these children.

Rehabilitation for spinal muscular atrophy patients in China: a national cross-sectional study.

BACKGROUND: The management of Spinal Muscular Atrophy (SMA) requires a multidisciplinary treatment approach, wherein rehabilitation constitutes an integral element. In this study, we examined the effects of rehabilitation among Chinese SMA patients and assessed the real-world efficacy of rehabilitation interventions. METHODS: We conducted a cross-sectional online survey on SMA patients from June 9, 2023, to June 30, 2023, through the Meier Advocacy & Support Center using data from the Center's database and electronic questionnaires. The rehabilitation situation of the participants over the past 14 months were investigated. Logistic binary regression was used to analyze the relationship between Pediatric Quality of Life Inventory(PedsQL™) scores and rehabilitation. RESULT: A total of 186 questionnaires were finally analyzed. Only 29 patients did not rehabilitated in the past 14 months. A significant correlation between age and type of rehabilitation, as well as between age and duration of rehabilitation. Patients receiving no rehabilitation or solely home-based rehabilitation exhibited a higher median age of 8.4 compared to those undergoing standard rehabilitation or a combination of standard and home-based rehabilitation, with a median age of 4.9 (z-score = -4.49, p-value < 0.001). In addition, long-term rehabilitation (OR = 0.314, 95%CI = 0.106-0.927, p = 0.04) were negatively correlated with lower PedsQL™ Neuromuscular Module scores, and PedsQL scores in the long-term rehabilitation group were higher than those in the short-term and no-rehabilitation groups (54.2 ± 15.1 vs. 45.9 ± 14.4 and 42.3 ± 14.3, p = 0.01), with the most significant difference observed in the physical function section (59.0 ± 15.8 vs. 46.8 ± 15.2 and 45.6 ± 15.9, p < 0.01). Mobility and exercise (OR = 0.26, 95%CI = 0.08-0.81, p = 0.02), as well as assistive technology (OR = 0.28, 95%CI = 0.10-0.82, p = 0.02), were independently associated with a lower score in a negative direction. CONCLUSION: The study found that long-term rehabilitation was linked to higher PedsQL scores in SMA patients, highlighting the need for standardized rehabilitation programs to enhance function and quality of life.

No Association between SARS-CoV-2 Infection and Quality of Life 6- and 12-Months After Infection.

OBJECTIVE: To assess the association between SARS-CoV-2 infection and long-term quality of life (QoL). METHODS: Prospective cohort study with 6- and 12-month follow-up conducted in 14 Canadian institutions. Children tested for SARS-CoV-2 between August 2020 and February 2022 were eligible. QoL was measured using PedsQLTM-4.0, overall health status scores 6- and 12-months after testing. RESULTS: Among SARS-CoV-2 positive and negative participants eligible for long-term follow-up, 74.8% (505/675) and 71.8% (1106/1541) at 6- and 59.0% (727/1233) and 68.1% (2520/3699) at 12-months, completed follow-up, respectively. Mean ± SD PedsQL™ scores did not differ between positive and negative groups; difference: -0.86 (95%CI: -2.33, 0.61) at 6- and -0.48 (95%CI: -1.6, 0.64) at 12-months, respectively. SARS-CoV-2 test-positivity was associated with higher social subscale scores. Although in bivariate analysis, overall health status at 6-month was higher among SARS-CoV-2 cases [difference: 2.16 (95%CI: 0.80, 3.53)], after adjustment for co-variates, SARS-CoV-2 infection was not independently associated with total PedsQL™ or overall health status at either time point. Parental perception of recovery did not differ based on SARS-CoV-2 test-status at either time point. CONCLUSIONS: SARS-CoV-2 infection was not associated with QoL, overall health status, or parental perception of recovery 6- and 12-months following infection. CLINICAL TRIAL REGISTRATION (IF ANY): N/A.

Selecting PedsQL items to derive the PedsUtil health state classification system to measure health utilities in children.

BACKGROUND: There is a lack of preference-based health-related quality of life (HRQoL) measures that consistently value health across a full range of child age groups. The PedsQL is a generic HRQoL instrument validated for children 2-18 years, but it is not preference-based. The objective of this study was to derive the PedsUtil health state classification system from the PedsQL as a basis for a preference-based HRQoL measure for children. METHODS: A two-step process was used to select PedsQL items to include in the health state classification system: 1) exclude poorly functioning items according to Rasch analysis in each of the previously established seven dimensions of the PedsUtil health state classification system and 2) select a single item to represent each dimension based on Rasch and psychometric analyses, as well as input from child health experts and parents. All secondary analyses were conducted using data from the Longitudinal Study of Australian Children (LSAC). Analyses were stratified by age group (i.e., 2-5 years, 6-13 years, and 14-17 years) to represent the different developmental stages of children and to reflect the study design of the LSAC. Rasch analyses were also performed on five random subsamples for each age group to enhance robustness of results. RESULTS: Twelve items were excluded from the PedsUtil health state classification system after the first step of the item selection process. An additional four items were excluded in the second step, resulting in seven items that were selected to represent the seven dimensions of the PedsUtil health state classification system: Physical Functioning ("participating in sports activity or exercise"), Pain ("having hurts or aches"), Fatigue ("low energy level"), Emotional Functioning ("worrying about what will happen to them"), Social Functioning ("other kids not wanting to be their friend"), School Functioning ("keeping up with schoolwork"), and School Absence ("missing school because of not feeling well"). CONCLUSIONS: The PedsUtil health state classification system was derived from the PedsQL based on several criteria and was constructed to be applicable to children two years and older. Research is ongoing to elicit preferences for the PedsUtil health state classification system to construct the PedsUtil scoring system.

The impact of posterior urethral valves on pediatric quality of life and family function: Prospective analysis of serial patients managed in a dedicated multidisciplinary clinic.

INTRODUCTION: Posterior urethral valves (PUV) is a chronic condition that can adversely impact child and family health related quality of life (HRQOL). Surprisingly, to date, the impact of this condition on the QOL of children with PUV has not been studied. Thus, our aim is to evaluate this important aspect of care using validated questionnaires in our institutional PUV clinic. METHODS: From December 2022 to January 2024, the PedsQL Inventory and associated family impact module was distributed in our PUV clinic. Higher scores reflect better HRQoL and less impact on family. Children >8 years old completed the tool themselves, otherwise parents completed the tools on behalf of the child. We compared PUV scores against reported psychometric data from healthy children and other chronic illnesses. RESULTS: A total of 112 families completed the questionnaires. The total HRQoL score was 80 ± 13. Overall, we noted that the domains reflecting psychosocial impact were significantly lower than those of physical well-being (76 ± 16 vs. 89 ± 15 for psychosocial and physical impact, respectively; p < 0.01). There was a significant difference in the psychosocial emotional impact for children managed with clean intermittent catheterization (CIC) compared to those who were not (69 ± 21 vs. 81 ± 16; p = 0.005) (Table 2). In addition, we noted that children with PUV scored similar to healthy controls, but higher than children with other chronic illnesses. With respect to the impact on family, overall, parent, and family impacts were 76 ± 19, 78 ± 21, and 81 ± 21, respectively. DISCUSSION: Chronic illness has been associated with a negative impact on HRQoL in both adults and children. We noted similar findings the present study, particularly for young children and those who are maintained on CIC. To our knowledge, this is the first study to evaluate the impact of PUV on a child's HRQoL as well as the impact on the family, and to document a negative psychosocial impact of CIC in this population. However, there are some important limitations to be addressed including relying on families to complete questionnaires accurately during busy clinic visits and the lack of controls from the same environment. CONCLUSION: When compared to healthy controls, PUV patients score similarly in HRQoL assessment. In addition, children <3years of age report a negative impact on physical well being, and CIC negatively impacts the emotional well-being. These data can be used to advocate for better mental health supports and social work for these children.

Cross-cultural adaptation, reliability, and validity of the Turkish Pediatric Quality of Life Inventory-Cognitive Functioning Scale (PedsQLTM-CFS) in children with cancer.

The Pediatric Quality of Life Inventory-Cognitive Functioning Scale (PedsQLTM-CFS) was developed as a brief, general, symptom-specific tool to measure cognitive function. The 6-item PedsQL™ Cognitive Functioning Scale and PedsQL 3.0 Cancer Module answered 369 parents and 330 children with 5-18 years. Parents also completed Behavior Rating Inventory of Executive Function (BRIEF). The PedsQL™ Cognitive Functioning Scale evidenced excellent reliability (parent proxy-report α = 0.980/Fleiss Kappa: 0.794; children self-report α = 0.963/Fleiss Kappa: 0.790). Both child self-report and parent proxy-report PedsQL™ Cognitive Functioning Scale scores exhibited significant correlations with all parent-report BRIEF summary and subscale scores (p < .05). Both child self-report and parent proxy-report PedsQL™ Cognitive Functioning Scale scores exhibited significant correlations with PedsQL 3.0 Cancer Module total score and subscale scores (p < .05). The PedsQLTM-CFS can be used in high-risk populations with substantial to perfect reliability, both in regards to total/subcategory scores as well as in children with cancer.

Health-related quality of life in children with childhood acute myeloid leukemia in China: A five-year prospective study.

Purpose: This study aims to identify the key factors influencing health-related quality of life (HRQoL) of pediatric acute myeloid leukemia (AML) patients following their initial diagnosis and examine their impact on the five-year survival prognosis. Methods: A chart review and follow-up were conducted for children with AML who participated in a prospective cohort study between 2017 and 2020. We identified factors influencing HRQoL through Pediatric Quality of Life Inventory™ (PedsQL™ 4.0), PedsQL™ Cancer Module 3.0 (CM 3.0) and PedsQL™ Family Impact Module 2.0 (FIM 2.0), as well as assessed the impact of impaired HRQoL on the overall outcomes of patients. Results: Sixty-four subjects enrolled in the study had complete HRQoL outcome data, and 61 of them completed the 5-year follow-up. In CM 3.0, age was positively associated with parental proxy reports (p = 0.040), whereas divorced families were negatively associated with child self-reports (p = 0.045). A positive medical history correlates with FIM 2.0 (p = 0.025). Residence (p = 0.046), the occupation of caregivers (p = 0.014), disease severity (p = 0.024), and the only child (p = 0.029) exhibited statistically significant associations with the impairment of HRQoL. Impaired HRQoL scores shown by the PedsQL™4.0 parent proxy report (p = 0.013) and FIM 2.0 (p = 0.011) were associated with a reduced 5-year survival rate. Conclusions: This study demonstrated that early impairment of HRQoL in pediatric acute myeloid leukemia patients has predictive value for long-term prognosis. Once validated, these findings may provide some guidance to clinicians treating children with AML.

Caregiver reported long-term outcomes in children with major trauma and traumatic brain injuries: A single-centre retrospective study.

Introduction: We aim to investigate the functional outcomes and long-term health-related quality of life (HRQOL) in children with major trauma associated with traumatic brain injury (TBI). Method: We performed a retrospective review of records among patients >2 and ≤16 years old in a tertiary paediatric hospital between January 2014 and October 2019 with major trauma (Injury Severity Score of ≥16) and TBI of all severities. We recorded each child's Glasgow Outcome Scale-Extended Pediatric Version (GOS-E Peds) at 12 months post-injury and Pediatric Quality of Life Inventory (PedsQL) scores at 6 and 12 months post-injury based on the parent proxy-report scales. Results: We included 53 patients with a median age of 9.0 years old (interquartile range 2.3-15.5). Most injuries were due to falls (30, 56.6%) or road traffic collisions (15, 28.3%); 41 patients (77.3%) required intensive care while 30 patients (56.6%) underwent neurosurgical intervention. Most patients (43, 81.1%) had GOS-E Peds scores of ≤2 at 12 months post-injury. We reported a significant mean difference between the 6- and 12-month parent-reported scores for physical functioning (6.6, 95% confidence interval [CI] 0.3-12.8, P=0.041), psychosocial functioning (4.1, 95% CI 1.0-7.2, P=0.012) and overall scores (5.0, 95% CI 1.4-8.7, P=0.008). Compared with the validated PedsQL scores, our mean scores were higher across all domains at 12 months. Conclusion: With current standard of care, parents of children with major trauma and TBI reported gains in quality of life, physical, psychosocial and overall function between 6 and 12 months post-injury.

Measurement properties of the EQ-5D-Y-3L, PedsQL 4.0, and PROMIS-25 Profile v2.0 in pediatric patients with spinal muscular atrophy.

OBJECTIVE: The objective of this study was to examine the psychometric properties of the EQ-5D-Y-3 L, Patient Reported Outcomes Measurement System 25-item version profile v2.0 (PROMIS-25), and Pediatric Quality of Life Inventory™ version 4.0 Generic Core Scale (PedsQL 4.0) in Chinese pediatric patients with spinal muscular atrophy (SMA). METHODS: The data used in this study were obtained via a web-based cross-sectional survey. Parents of pediatric patients with SMA completed the proxy-reported EQ-5D-Y-3 L, PedsQL 4.0, and PROMIS-25 measures. Information about socioeconomic and health status was also obtained. The ceiling and floor effects, factorial structure, convergent validity, and known-group validity of the three measures were assessed. RESULTS: Three hundred and sixty-three parents of children aged from 5 to 12 completed the questionnaires. Strong floor effects were observed for the physical function components of the PROMIS-25 (41.3%) and PedsQL 4.0 (67.8%). For EQ-5D-Y-3 L, 84.6% of the respondents reported having "a lot of" problems with the dimensions "walking" and "looking after myself." Minimal ceiling or floor effects were observed for the EQ-5D-Y-3 L index value. The confirmatory factor analysis supported a six-factor structure for the PROMIS-25, but did not support a four-factor structure for the PedsQL 4.0. All hypothesized correlations of the dimensions among the three measures were confirmed, with coefficients ranging from 0.28 to 0.68. Analysis of variance showed that EQ-5D-Y-3 L demonstrated better known-group validity than the other two measures in 14 out of 16 comparisons. CONCLUSIONS: The EQ-5D-Y-3 L showed better discriminant power than the other two measures. The physical health dimensions of all three measures showed the significant floor effects. These findings provide valuable insights into the effectiveness of these measures at capturing and quantifying the impact of SMA on patients' health-related quality of life.

Health-related quality of life and subjective well-being among children aged 9-12 years in Shandong Province, China.

PURPOSE: To investigate the health-related quality of life (HRQoL) and subjective well-being (SWB) of children aged 9-12 years in eastern China, and examine concordance within child self-reported and parent proxy-assessed. METHODS: Data was collected from 9 to 12 years old children (including their parents) in Shandong Province in 2018. Participants self-completed a hard-copy questionnaire including Child Health Utility 9D (CHU9D), Pediatric Quality of Life Inventory (PedsQL)™ 4.0 Short Form 15 Generic Core Scales (hereafter the PedsQL™), Student's Life Satisfaction Scale (SLSS), as well as information on socio-demographic characteristics and self-report health status. Spearman's correlation coefficients and the difference between sub-groups were conducted to assess and compare the agreement on HRQoL and SWB instruments. Exploratory factor analysis (EFA) was used to ascertain the number of unique underlying latent factors that were associated with the items covered by the two generic HRQoL and the SWB instruments. The concordance of child self-reported and parent proxy-assessed was analyzed using weighted kappa coefficient and Bland-Altman plots. RESULTS: A total of 810 children and 810 parents were invited to participate in the survey. A valid sample of 799 (98.6%) children and 643 (79.4%) parents completed the questionnaire. The child self-reported mean scores were CHU9D = 0.87, PedsQL™ = 83.47, and SLSS = 30.90, respectively. The parent proxy-assessed mean scores were PedsQL™ = 68.61 and SLSS = 31.23, respectively. The child self-reported PedsQL™ was moderately correlated with the CHU9D (r = 0.52). There was a weak correlation between CHU9D and SLSS (r = 0.27). The EFA result found 3 factors whilst seven SLSS items grouped into a standalone factor (factor 3), and the nine dimensions of CHU9D shared two common factors with the PedsQL™ (factor 1 and factor 2). A low level of concordance was observed across all comparisons and in all domains (weighted kappa < 0.20) between parents and their children. Furthermore, a high level of discordance was observed between child self-reported and father proxy-assessed. CONCLUSIONS: CHU9D and PedsQL™ instruments have a higher agreement in measuring the HRQoL in children. CHU9D/PedsQL™ and SLSS instruments showed a low agreement and EFA result suggested that measuring SWB in children potentially may provide further information, which might be overlooked by using HRQoL instruments exclusively. Concordance of child self-reported and parent proxy-assessed was poor. Overall, mother-child concordance was higher than father-child concordance.

Pattern and determinants of health-related quality of life of adolescents with congenital heart disease in Cameroon: A single-center cross-sectional study.

Background: Health-related quality of life (HRQoL) assessment is necessary for the management of patients with congenital heart diseases (CHD). No study has yet been reported on Cameroonian adolescents. The aim of this study was to evaluate the profile of and look for determinants of HRQoL in adolescents with CHD in Cameroon. Methods: This was a cross-sectional study with prospective recruitment carried out on 71 adolescents diagnosed with CHD aged 12 to 18 years and recruited at the Douala General Hospital. Sociodemographic and clinical data were collected using a structured questionnaire. HRQoL was assessed using the pediatric quality of life inventory (PedsQL4.0) for child and parent reports. Multivariate linear regression was used to assess the determinants of HRQoL. Differences were considered significant for p < 0.05. Results: Mean age of participants was 15 ± 2 years with 54.9% women. Mean physical and psychosocial functioning scores were 50.7 ± 13.9 and 60.5 ± 9.6 for parent report and 49.5 ± 13.4 and 59.1 ± 9.1 for child report respectively; with no significant difference according to gender. Distribution of functioning scores according to anatomical complexity showed no significant difference while it was lower in patients with a greater physiological severity and to those with no surgical intervention compared to the others. After multivariate adjustments, physiological stage 3 or 4 was negatively associated while cardiac intervention was positively associated with HRQoL. Conclusion: CHD adolescents exhibit a low level of quality of life. Cardiac intervention positively affects HRQoL and should be targeted in the reduction of HRQoL burden from CHD in Cameroon.

Screening for Quality of Life in a Neurology Tic Clinic Using Quality Improvement Methodology.

BACKGROUND: Tic disorders in children often co-occur with other disorders that can significantly impact functioning. Screening for quality of life (QoL) can help identify optimal treatment paths. This quality improvement (QI) study describes implementation of a QoL measure in a busy neurology clinic to help guide psychological intervention for patients with tics. METHODS: Using QI methodology outlined by the Institute for Healthcare Improvement, this study implemented the PedsQL Generic Core (4.0) in an outpatient medical clinic specializing in the diagnosis and treatment of tic disorders. Assembling a research team to design process maps and key driver diagrams helped identify gaps in the screening process. Conducting several plan-do-study-act cycles refined identification of patients appropriate to receive the measure. Over the three-year study, electronic health record notification tools and data collection were increasingly utilized to capture patients' information during their visit. RESULTS: Over 350 unique patients were screened during the assessment period. Electronic means replaced paper measures as time progressed. The percentage of patients completing the measure increased from 0% to 51.9% after the initial implementation of process improvement, advancing to 91.6% after the introduction of electronic measures. This average completion rate was sustained for 15 months. CONCLUSIONS: Using QI methodology helped identify the pragmatics of implementing a QoL assessment to enhance screening practices in a busy medical clinic. Assessment review at the time of appointment helped inform treatment and referral decisions.

Factors Related to the Quality of Life in Children with Transfusion-Dependent Thalassemia.

Objective: Increased survival rate of patients with Transfusion-dependent Thalassemia (TDT) should be in line with their good quality of life (QoL). The study aimed to analyze the relationship between sociodemographic factors and clinical characteristics with the QoL of children with TDT. Methods: A cross-sectional study was conducted at Hasan Sadikin General Hospital from December 2022 to February 2023. A total of 158 eligible subjects aged 5-18 years with TDT were included in the analysis. QoL assessment was performed using child self-report and parent-proxy report questionnaires, along with physical examination findings. Bivariate and multivariate analyses were conducted to analyze the data. Results: A total of 158 subjects who met the research criteria were included in the analysis. Of 58.9% of children with TDT had a low adherence rate to iron chelating therapy (ICT). School function had the lowest score in QoL based on child-self report and parent proxy. Gender (p<0,05) and adherence to ICT (p<0,05) were significantly associated with lower quality of life. Conclusion: Female and adherence to ICT were predictors of children with TDT's QoL.

Scoring Abdominal Symptoms in People with Cystic Fibrosis.

(1) Background: The introduction of highly effective CFTR-modulating therapies (HEMT) has changed the course of the disease for many people with Cystic Fibrosis (pwCF). Attention previously focused on life-threatening conditions of the respiratory system has broadened, bringing the involvement of the digestive system into the clinical and scientific focus. This emphasized the need for sensitive tools to capture and quantify changes in abdominal symptoms (AS), ideally applying patient-reported outcome measures (PROMs). (2) Methods: The present review focuses on studies addressing AS assessment deriving from the multi-organic abdominal involvement in pwCF. Among 5224 publications retrieved until Nov. 2022, 88 were eligible, and 39 were finally included. (3) Results: The review reveals that for a long time, especially before HEMT availability, AS in pwCF were assessed by single questions on abdominal complaints or non-validated questionnaires. PROMs focusing on quality of life (QOL) including a few GI-related questions were applied. Likewise, PROMs developed and partially validated for other non-CF GI pathologies, such as chronic inflammatory bowel diseases, irritable bowel syndrome, gastroesophageal reflux, constipation, or pancreatitis, were implemented. (4) Conclusions: Only lately, CF-specific GI-PROMs have been developed and validated following FDA guidelines, showing high sensitivity to changes and capturing marked and statistically significant reductions in the burden of AS achieved with HEMT implementation.

Health-related quality of life and self-reported health status in adolescents with chronic health conditions before transfer of care to adult health care: an international cohort study.

BACKGROUND: Heath-related quality of life (HRQoL) is lower in adolescents with chronic health conditions compared to healthy peers. While there is evidence of some differences according to the underlying condition and gender, differences by measure and country are poorly understood. In this study we focus on the differences in HRQoL in adolescents with various chronic medical conditions in the year before transfer of care to adult health services. We also study the associations of two different HRQoL measurements to each other and to self-reported health. METHODS: We recruited 538 adolescents from New Children`s Hospital, Helsinki, Finland, and the Royal Children`s Hospital, Melbourne, Australia in 2017-2020. We used two validated HRQoL measurement instruments, Pediatric Quality of Life Inventory (PedsQL) and 16D, and a visual analog scale (VAS) for self-reported health status. RESULTS: In total, 512 adolescents (50.4% female, mean age 17.8 [SD 1.2] years), completed the survey measures. Higher HRQoL was reported in males than females in both countries (PedsQL 79.4 vs. 74.1; 16D 0.888 vs. 0.846), and in adolescents from Finland than Australia (80.6 vs. 72.2 and 0.905 vs. 0.825, p < 0.001 for all). Adolescents with diabetes, rheumatological, nephrological conditions and/or organ transplants had higher HRQoL than adolescents with neurological conditions or other disease syndromes (p < 0.001). PedsQL and 16D scores showed a strong correlation to each other (Spearman correlation coefficient r = 0.81). Using the 7-point VAS (1-7), 52% (248 of 479) considered their health status to be good (6-7) and 10% (48 of 479) rated it poor (1-2). Better self-reported health was associated with higher HRQoL. CONCLUSIONS: The HRQoL of transition aged adolescents varies between genders, diagnostic groups, and countries of residence. The association between self-reported health and HRQoL suggests that brief assessment using the VAS could identify adolescents who may benefit from in-depth HRQoL evaluation. TRIAL REGISTRATION: Trial registration name The Bridge and registration number NCT04631965 ( https://clinicaltrials.gov/ct2/show/NCT04631965 ).

Measuring health-related quality of life in children with suspected genetic conditions: validation of the PedsQL proxy-report versions.

PURPOSE: Measuring health-related quality of life (HRQoL) of children with suspected genetic conditions is important for understanding the effect of interventions such as genomic sequencing (GS). The Pediatric Quality of Life Inventory (PedsQL) is a widely used generic measure of HRQoL in pediatric patients, but its psychometric properties have not yet been evaluated in children undergoing diagnostic GS. METHODS: In this cross-sectional study, we surveyed caregivers at the time of their child's enrollment into GS research studies as part of the Clinical Sequencing Evidence Generating Research (CSER) consortium. To evaluate structural validity of the PedsQL 4.0 Generic Core Scales and PedsQL Infant Scales parent proxy-report versions, we performed a confirmatory factor analysis of the hypothesized factor structure. To evaluate convergent validity, we examined correlations between caregivers' reports of their child's health, assessed using the EQ VAS, and PedsQL scores by child age. We conducted linear regression analyses to examine whether age moderated the association between caregiver-reported child health and PedsQL scores. We assessed reliability using Cronbach's alpha. RESULTS: We analyzed data for 766 patients across all PedsQL age group versions (1-12 months through 13-18 years). Model fit failed to meet criteria for good fit, even after modification. Neither age group (categorical) nor age (continuous) significantly moderated associations between PedsQL scores and caregiver-reported child health. Cronbach's alphas indicated satisfactory internal consistency for most PedsQL scales. CONCLUSION: The PedsQL Generic Core Scales and Infant Scales may be appropriate to measure HRQoL in pediatric patients with suspected genetic conditions across a wide age range. While we found evidence of acceptable internal consistency and preliminary convergent validity in this sample, there were some potential problems with structural validity and reliability that require further attention.

Evaluation of quality of life in children with precocious puberty.

OBJECTIVE: This study aimed to investigate the quality of life of patients with central precocious puberty (CPP) who required treatment and premature thelarche (PT) followed up without treatment and to compare the groups with and without treatment among themselves and with healthy children. DESIGN, PATIENTS AND MEASUREMENT: This study is designed as a case-control study. A total of 193 children including 59 children with CPP, 53 children with PT, 81 healthy children and their parents were included in the study. A questionnaire was applied to evaluate the sociodemographic characteristics that would affect the quality of life. The 'Pediatric Quality of Life Inventory (PedsQL)' was used to assess the quality of life. RESULTS: The PedsQL total scale score was 78.10 ± 17.13, 79.35 ± 11.54 and 79.52 ± 14.65, the psychosocial health summary score was 78.86 ± 16.83, 79.40 ± 12.54 and 79.94 ± 14.94 and physical health summary score was 75.81 ± 20.69, 79.41 ± 15.04 and 78.25 ± 17.52 in CPP, PT and control groups, respectively; however, there was no statistical difference (p > .05). In the scale administered to the parents, scores were similar in the three groups. No difference was found between CPP, PT and control groups in terms of sociodemographic data in the study (p > .05). CONCLUSION: Unlike previous studies, in this study the effects of sociodemographic characteristics and whether treatment was initiated or not on quality of life were investigated. Although the scale scores of the CPP group were lower than the PT and control group, there was no statistically significant difference, indicating that quality of life was not negatively affected in the CPP group receiving treatment.

Translation, transcultural adaptation, and validation of PedsQL 3.0 Sickle Cell Disease Module into Brazilian Portuguese.

INTRODUCTION: Despite the high prevalence of sickle cell disease (SCD) in Brazil, no studies have described the validation of an SCD-specific health-related quality-of-life (HRQoL) instrument in children. We validated PedsQL 3.0 Sickle Cell Disease Module (PedsQL-SCD) for Brazilian Portuguese, and cross-validated it with PedsQL 4.0 Generic Core Scale (PedsQL-GCS) in children with SCD. METHODS: PedsQL-SCD was translated and culturally adapted using forward and reverse translations. PedsQL-SCD and PedsQL-GCS were tested in children and adolescents with SCD aged 2-18 years and their caregivers. Validity was assessed using the Pearson and intraclass correlation coefficients, and reliability measured with Cronbach's alpha. RESULTS: PedsQL-SCD was validated in 206 children with SCD (median age 14 years, range: 8-18) and 201 caregivers. Among patients and caregivers, the mean total score for PedsQL-SCD was 65.7 and 64.1, respectively. The mean total score for PedsQL-GCS was 73.1 and 68.9 among patients and caregivers, respectively. The internal consistency for PedsQL-SCD and PedsQL-GCS was good; Cronbach's alpha coefficients ranged from .59-.93 to .64-.83 among patients and from .60-.95 to .65-.85 among caregivers, respectively. Most intercorrelations between PedsQL-SCD and PedsQL-GCS, for patients and caregivers, had medium to large effect sizes (range: .23-.63 and .27-.64, respectively). Pain and pain impact domains of PedsQL-SCD and physical dimension of PedsQL-GCS had the highest cross-correlation (.63 and .6 for patients; .63 and .64 for caregivers, respectively), confirming convergent construct validity. CONCLUSION: PedsQL-SCD is a valid, culturally appropriate measure to assess HRQoL in children with SCD in Brazil and is well-correlated PedsQL-GCS.

Health-related quality of life in children with coeliac disease and in their families: A long-term follow-up study.

OBJECTIVES: The aim of the study was to assess long-term health-related quality of life (HRQoL) in children and adolescents with coeliac disease (CD), and their parents. METHODS: We re-evaluated prospectively the HRQoL and clinical characteristics of 80 families, assessed 5 years earlier, using a disease-specific questionnaire, the CD Dutch Questionnaire (CDDUX), and a generic questionnaire, the Paediatric Quality of Life Inventory (PedsQL). RESULTS: After a 10-year follow-up, there was no significant change in the total CDDUX and PedsQL scores in children and their parents when compared to the evaluation conducted 5 years earlier. The total CDDUX score reflected a neutral QoL, while for the generic PedsQL was good-very good. The only significant decrease after 5 years was the PedsQL subdomain Emotional functioning. Patients who admitted voluntarily eating gluten reported lower score in CDDUX Diet. Lower scores in subdomain "Physical functioning" (PedsQL) were reported in patients with positivity of TTG or associated diseases. CONCLUSIONS: The CDDUX score indicated a consistently stable and neutral QoL perception among coeliac patients and caregivers, even after 10-year postdiagnosis, suggesting minimal fluctuations in the impact of CD on disease-specific health domains over time. Furthermore, the consistently good PedsQL score could be a reflection of the resilience of coeliac families in coping with this chronic condition. Gluten-free diet compliance was confirmed to be determinant of HRQoL in the long term. The study confirms the importance of extending surveillance on these patients, possibly using different questionnaires, to assess QoL from different perspectives.