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.

Development of a Health-State Classification System for the Pediatric Quality-of-Life Inventory Version 4.0 Generic Core Scales for Preference-Based Valuation in Australia.

OBJECTIVES: Pediatric Quality-of-Life Inventory Version 4.0 Generic Core Scales (PedsQL GCS), comprising 23 items covering 4 subscales (physical, emotional, social, and school functioning), is a widely applied generic measure of childhood health-related quality of life but does not provide health utilities for cost-effectiveness-based decision making. This study aimed to develop a reduced item version of PedsQL GCS amenable to health utility derivation in Australia. METHODS: Data sources were 2 cohorts of the Longitudinal Study of Australian Children, including proxy responses for all PedsQL GCS versions (Toddlers, Young Children, Children, and Teens), and the CheckPoint sample containing child self-report to the Children version. Three analytic samples were CheckPoint sample (n = 1874); Mallinson sample containing 1 measurement per child from one of the Young Children, Children, or Teens versions (n = 7855); and Toddlers sample (n = 7401). Exploratory and confirmatory factor analyses assessed dimensionality. Psychometric analyses used Rasch and classical criteria on 3 randomly selected subsamples (n = 500) per sample. Item selection prioritized psychometric performance in the CheckPoint sample, also considering performance in other samples and conceptual content. RESULTS: Dimensionality assessments did not generate an alternative empirical structure for the measure, and psychometric analyses were conducted on the original 4 subscales. The selected items were: "Get aches and pains" for physical functioning; "Feel sad/blue" for emotional functioning; "Other kids not friends" for social functioning; and "Keeping up with school work" for school functioning. CONCLUSIONS: The final 4-item set, pending further psychometric validation and valuation, can generate health utilities from the widely used PedsQL GCS to inform cost-effectiveness-based decision making.

Assessment of quality of life and physical activity in patients with oligoarticular juvenile idiopathic arthritis in remission.

The purpose of this study was to evaluate physical activity (PA) and health-related quality of life (HRQOL) in children with oligoarticular juvenile idiopathic arthritis (JIA) in remission in comparison with healthy peers and to determine the disease-related factors affecting PA levels. This study was conducted with 50 oligoarticular JIA patients in remission and 50 healthy peers between 9 and 14 years. Demographic and clinical characteristics, laboratory parameters, and treatments were noted from electronic medical records. HRQOL was assessed with the Pediatric Quality of Life Inventory (PedsQL). PA was evaluated with the Physical Activity Questionnaire for Children (PAQ-C). Oligoarticular JIA patients had significantly lower self-reported median PedsQL scores in the domains of school functioning and social functioning compared to the control group (67.5 (10) vs. 75 (25), p = 0.001 and 70 (15) vs. 85 (26.3), p < 0.001, respectively). The median PAQ-C score was 2.6 (1.1) in patients with JIA and 3 (0.9) in their healthy peers (p = 0.02). The PAQ-C score was 2.8 (1.2) in patients < 8 years at the disease onset and 2.3 (1) in those aged ≥ 8 years (p = 0.022). There was no significant difference in the number of affected joints, type of affected joint, MTX and biologic agent treatment, and remission with or without drugs with the total score of the PedsQL and PAQ-C. All PedsQL domains were positively correlated with the PAQ-C.  Conclusion: Oligoarticular JIA patients demonstrated lower PA and HRQOL scores compared to healthy controls despite favorable disease control. What is Known: • Oligoarticular JIA has fewer functional limitations and disabilities compared to other JIA subtypes. • As JIA can affect all aspects of a child's life, there is a need to improve the quality of life related to the disease. What is New: • It should be considered that patients with oligoarticular JIA may show lower PA and HRQOL scores compared to healthy controls despite favorable disease control. • Since there may be a relationship between PA and HRQOL, factors that may affect PA should be investigated to provide a holistic approach to JIA treatment.

Validity and reliability of Arabic version of pediatric migraine disability assessment scale (Child Self-Report versus Parent Proxy-Report): a multi-center study.

BACKGROUND: Pediatric Migraine Disability Assessment (PedMIDAS) is one of the most frequently used questionnaires to assess disability from migraine in pediatric patients. This work aimed to evaluate the validity and test-retest reliability of the Arabic version of the child self-report versus the parent proxy report PedMIDAS. We also aimed to test the agreement between children's and parents' reports of the scale. METHODS: PedMIDAS was subjected to translation and back-translation, then applied to 112 pediatric patients fulfilling the migraine diagnostic criteria. This cross-sectional study was conducted on two visits, one week apart. At visit 1, the following data were obtained from the included pediatric patients: disease duration, migraine type, current treatment regimen, monthly migraine days (MMD) during the last month preceding the enrollment, and migraine intensity using the visual analogue scale. Then, each child and his parent were independently asked to fill out PedMIDAS and Child Self-Report of the Pediatric Quality of Life Inventory™ 4.0 (PedsQL™) to test the convergent validity of PedMIDAS. At visit 2, each child was requested to complete PedMIDAS again, and so was the parent to evaluate test-retest reliability. RESULTS: Cronbach's alpha was estimated to be 0.94 for each instrument. For the child-self report PedMIDAS, the average measure intraclass correlation coefficient (ICC) value was 0.992 (95%CI = 0.989-0.995), while it was estimated to be 0.990 for the parent-proxy report with 95%CI = 0.985-0.993, indicating excellent test-retest reliability for both instruments. The child-self report and the parent-proxy report PedMIDAS scores were significantly correlated with MMD, VAS, and all domains of the corresponding PedsQL, supporting convergent validity for both instruments. Agreement between parent and child on disability grading categories of PedMIDAS was substantial (κ = 0.644). CONCLUSION: The Arabic version of PedMIDAS was a valid and reliable instrument to assess disability from migraine in Arabic-speaking pediatric patients with migraine. Parent reports can be valuable as a complement to child reports for a comprehensive assessment of migraine.

Psychometric Comparison of EQ-5D-Y, CHU-9D, and PedsQL 4.0 in Chinese Children and Adolescents With Functional Dyspepsia: A Multicenter Study.

OBJECTIVES: This study aimed to assess and compare psychometric properties of the 3 health-related quality of life (HRQOL) instruments EQ-5D Youth version (EQ-5D-Y), Child Health Utility 9D (CHU-9D), and Pediatric Quality of Life Inventory 4.0 (PedsQL 4.0) in children and adolescents with functional dyspepsia (FD) in China. METHODS: A consecutive sample of FD outpatients were recruited from 6 tertiary medical centers in Hangzhou. The patients self-completed the 3 instruments in random order. Their feasibility, acceptability, construct validity (convergent, divergent, and known-group validity), and sensitivity were assessed. Multiple linear regression was used for identifying HRQOL-associated factors. RESULTS: A total of 1100 patients (mean age, 9.2 years; girl, 56.8%) completed the survey with no missing responses. Ceiling effect was quite higher in EQ-5D-Y (60.9%) than CHU-9D (33.8%) and PedsQL 4.0 (1.0%). The EQ-5D-Y and CHU-9D utility scores and PedsQL 4.0 total score were highly correlated (|r| = 0.593-0.661), except for the EuroQol visual analog scale score (EQ-VAS). The intraclass correlation coefficient between the 2 utility scores was fair (0.542). Most conceptually similar dimensions among the 3 instruments showed moderate to high correlations (|r| > 0.3) as hypothesized. The difference was statistically significant for the 2 utility scores and PedsQL 4.0 total score in varied severity groups (P < .001), and PedsQL 4.0 total score had higher relative efficiency and effect size values. The child's age, severity of FD symptoms, and their guardian's education had significant impact on HRQOL (P < .001). CONCLUSIONS: EQ-5D-Y, CHU-9D, and PedsQL 4.0 demonstrated acceptable psychometric properties in Chinese children with FD. PedsQL 4.0 showed superior sensitivity and is recommended. EQ-5D-Y and CHU-9D utility scores were not interchangeable. The measurement properties of EQ-VAS need to be further explored.

Impact of steroids and steroid-sparing agents on quality of life in children with nephrotic syndrome.

BACKGROUND: Steroids and/or steroid-sparing medications are commonly used for nephrotic syndrome treatment; however, the impact of these medications on health-related quality of life over time is not well described. METHODS: Longitudinal cohort is up to 5 years where children were assessed with baseline and annual Pediatric Quality of Life Inventory questionnaire. A mixed-effects linear regression determined differences in scores among children receiving steroids and/or steroid-sparing agents for at least 30 days compared with those not on medication at 1, 3, 6, and 12 months prior to assessment. RESULTS: Among 295 children, 64% were male, with a median age of 3.7 (interquartile range [IQR], 2.7, 5.9) years at diagnosis, and comprised 25% Europeans, 40% South Asians, and 8% East/Southeast Asians. Adjusted HRQOL scores were reduced among children taking steroids and steroid-sparing agents among 705 HRQOL measures (median 2 [IQR, 1, 3] per child). Compared to children without medication, steroid and steroid-sparing agent use up to 12 months prior to assessment were associated with an overall HRQOL drop of 3.17 (95% confidence interval [CI], - 5.25, - 1.08) and 3.18 (95% CI, - 5.24, - 1.12), respectively, after adjustment. Functioning domain scores were reduced by 4.41 points (95% CI, - 6.57, - 2.25) in children on steroids, whereas fatigue domain scores were reduced by 5.47 points (95% CI, - 9.28, - 1.67) in children on steroid-sparing agents after adjustment. CONCLUSIONS: HRQOL is consistently decreased in children receiving steroids and steroid-sparing agents, with differential effects on functioning and fatigue. Counseling families on possible effects of prolonged treatment periods is important in the management of childhood nephrotic syndrome.

Validity and reliability of the French version of the Pediatric Quality of Life Inventory™ brain tumor module.

INTRODUCTION: Assessing health-related quality of life (HRQoL) is an increasingly important aspect of standard care in pediatric oncology. Currently, there is a gap in the availability of French questionnaires to assess the quality of life of French-speaking pediatric brain tumor (PBT) patients, which has important implications in the care of this population. The first aim of this study was to translate the original English Pediatric Quality of Life Inventory™ (PedsQL) brain tumor module version into French. The second aim was to describe the stability, repeatability and convergent validity of the French PedsQL brain tumor module. METHODS: A total of 61 PBT patients were included in this study. Among them, 15 children and 20 parents participated in the translation process. As part of the validation study, 48 children and 48 parents answered the PedsQL brain tumor module twice, and the PedsQL generic core scales and the patient-reported outcomes measurement information system (PROMIS-37 pediatric profile v2.0) questionnaire were administered once to the participants. The mean age of the 25 boys and 23 girls was 8.3 ± 4.8 years. For temporal stability, we used intraclass correlation coefficients (ICCs), for repeatability, we used the Bland and Altman method to assess the accuracy at a 1-week interval, and we used Pearson's correlation coefficients for convergent validity between the PedsQL brain tumor module, PedsQL general module and the PROMIS. RESULTS: Temporal stability for the parent proxy-reports (average ICC = 0.98) and the child self-reports (average ICC = 0.98) were excellent. There was a high absolute stability over a 1-week interval for the parent proxy-reports (ICC > 0.96) and child self-reports (ICC > 0.96). Convergent validity between parent proxy-reports and child self-reports was supported by positive correlations for five subscales. Children reported higher scores in cognitive problems and the movement and balance parameters than their parents and reported lower scores on the worry parameter than their parents. CONCLUSION: The strong psychometric properties of the French version of the PedsQL brain tumor module indicate that it is a validate and reliable questionnaire to measure HRQoL in PBT patients. The availability of a French version of the PedsQL brain tumor module supports the wider dissemination of the assessment of HRQOL in PBT patients.

Paediatric patients report lower health-related quality of life in daily clinical practice compared to new normative PedsQLTM data.

AIM: To compare Health-Related Quality of Life (HRQOL) of paediatric patients with newly collected HRQOL data of the general Dutch population, explore responses to individual items and investigate variables associated with HRQOL. METHODS: Children (8-12y) and adolescents (13-17y) from the general population (N = 966) and from a paediatric population (N = 1209) completed the Pediatric Quality of Life Inventory (PedsQLTM ) online via the KLIK Patient-Reported Outcome Measures portal. PedsQLTM scale scores were compared between groups with independent t tests, by age group and gender. Responses to PedsQLTM items were explored using descriptive analyses. Linear regression analyses were performed to determine which variables were associated with HRQOL. RESULTS: Paediatric patients reported worse HRQOL than the general population on all PedsQLTM scales (p ≤ .001, d = 0.20-1.03), except social functioning, and a high proportion reported problems on PedsQLTM items, for example, 'I have trouble sleeping'. Younger age, female gender and school absence were negatively associated with HRQOL (ß = -0.37-0.10, p ≤ .008). CONCLUSION: Paediatric patients reported lower HRQOL than the general population, and school absence, female gender and younger age were associated with lower HRQOL. The results underline the importance to structurally monitor paediatric patients' HRQOL in clinical practice to detect problems and offer the right help on time.

Reliability and validity of the Thai pediatric quality of life inventory™ 3.0 neuromuscular module.

BACKGROUND: Neuromuscular disorders (NMDs) are chronic illnesses that adversely impact the lives of patients and their families. The Pediatric Quality of Life™ 3.0 Neuromuscular Module (PedsQL™ 3.0 NMM) was designed to assess health-related quality of life (HRQoL) among children with NMDs. The objective of this cross-sectional study is to evaluate the reliability and validity of the PedsQL™ 3.0 NMM Thai version. METHODS: Formal permission to translate the PedsQL™ 3.0 NMM into Thai language was granted by the inventor, and the translation process followed linguistic translation guidelines. The PedsQL™ 3.0 NMM Thai version was administered to children with NMD and their parents/caregivers at the Division of Neurology, Department of Pediatrics, Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok, Thailand. Re-test was performed within 2-4 weeks after the initial test. RESULTS: One hundred and three children with NMD and their parents or caregivers were enrolled. Internal reliability as measured by Cronbach's alpha was > 0.7 (total score: child α = 0.88, parent α = 0.91). Test-retest reliability showed good agreement with an intraclass correlation coefficient (ICC) of 0.69 and 0.82 for the total score of the child report and the parent report, respectively. The mean (SD) quality of life total score for the child self-report was 74.9 (13.9) among ambulatory patients, and 60.7 (15.2) among non-ambulatory patients (maximum quality of life score is 100). The mean total quality of life score for the parent proxy-report was 70 (14.5) among ambulatory patients, and 55.2 (18.3) among non-ambulatory patients. The child total score was in good agreement with the parent/caregiver total score. CONCLUSIONS: PedsQL™ 3.0 NMM Thai version is a reliable and valid measure of HRQoL in Thai children with NMDs.

The comparison of perceived health-related quality of life between Australian children with severe specific language impairment to age and gender-matched peers.

BACKGROUND: Children with specific language impairment often present with multiple comorbidities, which may adversely affect both participation in play and academic performance, potentially impacting a child's health-related quality of life. This study 1) explored the suitability of the Pediatric Quality of Life Inventory™ Version 4.0 Generic Core Scales (PedsQL™) for use with a typically developing Australian control group, and 2) compared the health-related quality of life between a control group and Australian children with severe specific language impairment. METHODS: Health-related quality of life data collected as part of a broader study of 43 children with severe specific language impairment (males = 35, age range 5-16, mean age = 8.79+/- 2.92) enrolled at a special school were used to explore previously unreported findings. Typically developing gender and age matched (+/- 3 months) peers were recruited from local schools. The PedsQL™ child self-report and proxy-report were individually or interviewer-administered to the control group as required, and then compared to the group with specific language impairment. RESULTS: The PedsQL™ was reliable and feasible for use with the control group (N = 43, males = 35, age range = 5-16 years, mean age = 8.74+/- 2.94 years). Control group performance was as expected as per the manual. Parents of the control group scored their children significantly higher than did the children themselves on all scales except the emotional functioning scale. Both the control group children and their parents scored themselves significantly higher on all scales, compared to children with severe specific language impairment and their parents. CONCLUSIONS: The PedsQL™ was suitable for use with the control group. Further, the recruitment of a control group provided additional clarity on the extent a severe specific language impairment impacts on an Australian child's perceived health-related quality of life, compared to the manual cut-off scores. Severe specific language impairment significantly impacts negatively on the health-related quality of life of Australian children across all domains, particularly when compared to an age and gender-matched group of peers. These results warrant the inclusion of health-related quality of life evaluations in the assessment of these children along with a multidisciplinary approach.

Quality of life after living donor liver transplant for biliary atresia in Japan.

BACKGROUND: Health-related quality of life (HRQOL) is an important outcome in solid organ transplantation. This study evaluated and explored the factors of generic and transplant-specific HRQOL in Japanese pediatric and adolescent patients with biliary atresia (BA) after living donor liver transplant (LDLT). METHODS: A cross-sectional survey using anonymous questionnaires was completed between April and July 2015. Patient medical records were accessed. The Japanese version of Pediatric Quality of Life InventoryTM Generic Core Scales and Transplant Modules (child self-report and parent proxy-report) was administered. RESULTS: Participants consisted of 75 patients (mean age at survey, 9.6 years) and 74 parents. Japanese patients reported higher generic and transplant-specific HRQOL (total score) than that reported by US patients with BA after LT (US I; age at survey, 7.2 years) and by US patients after solid organ transplant (US II; age at survey, 11.3 years; LT, 53.8%; effect size, 0.55-0.96). Japanese parents, however, rated their children's generic HRQOL (total score) similar to that rated by the US I and II parents (0.13 and 0.30, respectively) and reported lower transplant-specific HRQOL (total score) than that reported by US II (0.26). Although the number of types of prescribed drugs was a common factor in HRQOL, most demographic and medical factors (e.g. child's age at survey and consultation frequency) varied with reporter (i.e. patients and parents). CONCLUSIONS: The levels and factors of generic and transplant-specific HRQOL of Japanese pediatric and adolescent patients with BA after LDLT varied with reporter (i.e. patients or parents).

Quality of life in children with infantile hemangioma: a case control study.

BACKGROUND: Infantile hemangioma (IH) is the most common vascular tumor in children. It is controversial whether IHs has effects on the quality of life (QOL) in patients of whom IH poses no threat or potential for complication. Thus, we conducted this study to evaluate the q QOL in patients with IH and find the predictors of poor QOL. METHODS: The PedsQL 4.0 Genetic Core Scales and the PedsQL family information form were administered to parents of children with IH and healthy children both younger than 2-year-old. The quality-of-life instrument for IH (IH-QOL) and the PedsQL 4.0 family impact module were administered to parents of children with IH. We compared the PedsQL 4.0 Genetic Core Scales (GCIS) scores of the two groups. Multiple step-wise regression analysis was used to determine factors that influenced QOL in children with IH and their parents. RESULTS: Except for physical symptom, we found no significant difference in GCIS between patient group and healthy group (P = 0.409). The internal reliability of IH-QOL was excellent with the Cronbach's alpha coefficient for summary scores being 0.76. Multiple step-wise regression analysis showed that the predictors of poor IH-QOL total scores were hemangioma size, location, and mother's education level. The predictors of poor FIM total scores were hemangioma location and father's education level. The predictors of poor GCIS total scores were children's age, hemangioma location and father's education level. CONCLUSION: The findings support the feasibility and reliability of the Chinese version of IH-QOL to evaluate the QOL in children with IH and their parents. Hemangioma size, location and education level of mother are important impact factors for QOL in children with IH and their parents.

Health-Related Quality of Life and Health Resource Utilization in Patients with Primary Immunodeficiency Disease Prior to and Following 12 Months of Immunoglobulin G Treatment.

PURPOSE: Health-related quality of life (HRQOL) has not been examined in patients with predominant antibody deficiency both pre- and post-immunoglobulin G (IgG) treatment initiation. HRQOL and health resource utilization (HRU) were assessed in newly diagnosed patients with primary immunodeficiency disease (PIDD) pre- and 12 months post-IgG treatment initiation. METHODS: Adults (age ≥18 years) completed the 36-item Short Form Health Survey, version 2; pediatric patients (PP)/caregivers completed the Pediatric Quality of Life Inventory (PedsQL). Scores were compared with normative data from the US general population (GP) and patients with other chronic conditions (OCC). RESULTS: Seventeen adult patients (APs), 8 PPs, and 8 caregivers completed baseline assessments. APs had significantly lower baseline mean physical component summary scores versus GP (37.4 vs 50.5, p < 0.01) adults with chronic back pain (44.1, p < 0.05) or cancer (44.4, p < 0.05) and lower mental component summary scores versus GP (41.6 vs 49.2, p < 0.05). PPs had lower PedsQL total (63.1 vs 82.7), physical summary (64.5 vs 84.5), and psychosocial summary (62.5 vs 81.7) scores versus GP. Post-IgG treatment, 14 APs, 6 PPs, and 8 caregivers completed assessments. Hospital admissions (0.2 versus 1.8, p < 0.01), serious infections (3.3 versus 10.9, p < 0.01) and antibiotic prescriptions (3.0 versus 7.1; p < 0.01) decreased significantly overall. While APs reported significant improvement in role-physical (p = 0.01), general health (p < 0.01), and social functioning (p = 0.02) and caregivers in vitality (p < 0.01), PPs did not. CONCLUSIONS: Pre-IgG treatment, patients with PIDD experienced diminished HRQOL versus GP and patients with OCC; post-treatment, HRU decreased and certain HRQOL aspects improved for APs and caregivers.

Health-related quality of life in children with chronic immune thrombocytopenia in China.

BACKGROUND: The concept of health-related quality of life (HRQoL) was brought up decades ago and has been well utilized in many different areas. Regarding immune thrombocytopenia (ITP) management, much work has been done to emphasize the necessity of taking HRQoL into consideration. However, data on HRQoL of children with chronic ITP remain rare. METHODS: This is a cross-sectional study. Children with chronic ITP aged from 2 to 18 and their parents were recruited. Participants completed the Pediatric Quality of Life Inventory™ (PedsQL™) and Kids' ITP Tools (KIT) questionnaires at only one time. The Pearson's correlation was examined between these measures for the pooled samples. RESULTS: A total of 42 families participated. Mean child self-report scores of KIT and PedsQL™ were 78.60 (SD = 12.40) and 85.13 (SD = 14.12), respectively, corresponding to parent proxy report scores, which were 73.40 (SD = 19.96) and 85.10 (SD = 13.56), respectively. Mean score of KIT parent impact report was only 40.39 (SD = 19.96). Significantly higher KIT scores (self-report and parent proxy) were observed in children with PLT more than 30 × 10*9/L compared to others, and this difference was even more noticeable in the PedsQL™ parent proxy report group (p < 0.001). As with intravenous immunoglobulin, the statistics difference appeared only in KIT child self-report group (p = 0.03), while for bone marrow examination, the difference appeared only in PedsQL™ parent proxy report group (p = 0.01). A negative relationship was apparent between duration of disease and scores. Gender and use of corticosteroids had no impact on the KIT and PedsQL™ scores here. Internal consistency reliability was demonstrated with Cronbach's alpha for all scales above the acceptable level of 0.89 (range from 0.88 to 0.97). There was a substantial concordance (p < 0.001) between the child and parent proxy scores (ICC for KIT is 0.59, ICC for PedsQL™ is 0.85). Meanwhile, KIT scores are correlated with PedsQL™ (r = 0.75 for child self report, r = 0.61 for parent proxy report). CONCLUSIONS: ITP affects HRQoL of children and parents. Parents are much more concerned with the disease than their children, which seriously influence their HRQoL as a result. The cross-culture translated KIT is reliable and valid with acceptable correlation to the PedsQL™. KIT provides valuable information of childhood ITP and will be a reliable outcome measure for further clinical research on HRQoL.

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

OBJECTIVE: To assess the association between Severe Acute Respiratory Syndrome Coronavirus 2 (SARS-CoV-2) infection and long-term quality of life (QoL). METHODS: Prospective cohort study with 6- and 12-months 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 PedsQL-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-months 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.

Insights into Pediatric Sleep Disordered Breathing: Exploring Risk Factors, Surgical Interventions, and Physical and Scholastic Performance at Follow-Up.

(1) Background: Sleep-disordered breathing represents a growing public health concern, especially among children and adolescents. The main risk factors for pediatric sleep-disordered breathing in school-age children are tonsillar and adenoid hypertrophy. Adenoidectomy, often in combination with tonsillectomy, is the primary treatment modality for pediatric sleep-disordered breathing. This study aims to comprehensively investigate various risk and protective factors in children with sleep-disordered breathing undergoing adenotonsillar or adenoidal surgeries. We also aim to explore the differences in neuropsychological profiles. (2) Methods: This is an observational, retrospective cohort study. We collected information on adenoidectomy or adenotonsillectomy in children referred to our center. We reviewed the clinical history and preoperative visits and collected data through a telephone questionnaire. The Pediatric Sleep Questionnaire (PSQ) and the Pediatric Quality of Life Inventory (PedsQL) screen sleep-disordered breathing and quality of life, respectively. The data were statistically analyzed using SPSS version 22.0 for Windows (SPSS Inc., Chicago, IL, USA). (3) Results: The study involved 138 patients, but only 100 children participated. A higher percentage of patients with sleep-disordered breathing were observed to have mothers who smoked during pregnancy. A smaller proportion of patients with sleep-disordered breathing habitually used a pacifier. A rise in physical score was associated with a reduced PSQ at follow-up (p = 0.051). An increase in the overall academic score was related to a decrease in the PSQ at follow-up (p < 0.001). A more significant proportion of patients undergoing adenotonsillectomy were observed to have a history of prematurity and cesarean birth. (4) This comprehensive study delves into the intricate interplay of risk and protective factors impacting children with sleep-disordered breathing undergoing adenotonsillectomy and adenoidectomy.

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.

Evaluation of quality of life in PKU before and after introducing tetrahydrobiopterin (BH4); a prospective multi-center cohort study.

BACKGROUND: Phenylketonuria (PKU) is a rare inborn error of metabolism caused by phenylalanine hydroxylase enzyme (PAH) deficiency. Treatment constitutes a strict Phe restricted diet with unpalatable amino acid supplements. Residual PAH activity enhancement with its cofactor tetrahydrobiopterin (BH4) is a novel treatment which increases dietary tolerance in some patients and permits dietary relaxation. Relaxation of diet may improve health related quality of life (HRQoL). This prospective cohort study aims to evaluate HRQoL of patients with PKU and effects of BH4 treatment on HRQoL. METHODS: Patients aged 4years and older, diagnosed through newborn screening and early and continuously treated, were recruited from eight metabolic centers. Patients and mothers completed validated generic and chronic health-conditions HRQoL questionnaires (PedsQL, TAAQOL, and DISABKIDS) twice: before and after testing BH4 responsivity. Baseline results were compared to the general population. Data collected after BH4 testing was used to find differences in HRQoL between BH4 unresponsive patients and BH4 responsive patients after one year of treatment with BH4. Also a within patient comparison was performed to find differences in HRQoL before and after treatment with BH4. RESULTS: 69/81 (85%) patients completed the questionnaires before BH4 responsivity testing, and 45/69 (65%) participated again after testing. Overall PKU patients demonstrated normal HRQoL. However, some significant differences were found when compared to the general population. A significantly higher (thus better) score on the PedsQL was reported by children 8-12 years on physical functioning and by children 13-17 years on total and psychosocial functioning. Furthermore, adult patients reported significantly lower (thus worse) scores in the TAAQOL cognitive domain. 10 patients proved to be responsive to BH4 treatment; however improvement in their HRQoL after relaxation of diet could not be demonstrated.

Health-related quality of life in children and adolescents with cyclic vomiting syndrome: a comparison with published data on youth with irritable bowel syndrome and organic gastrointestinal disorders.

OBJECTIVE: To evaluate health-related quality of life (HRQoL) in children with cyclic vomiting syndrome (CVS) and to compare child self-reports with those of their parents and with published reports of children with irritable bowel syndrome (IBS), children with organic gastrointestinal disorders, and a healthy control group. STUDY DESIGN: Sixty-eight children aged 5-18 years with CVS confirmed in a gastroenterology clinic completed the Pediatric Quality of Life Inventory (PedsQL). Eighty-two parents completed the parent-proxy PedsQL for children aged 2-18 years. These results were compared with published data for children with IBS, organic gastrointestinal disorders, and a healthy control group using ANOVA. Intraclass correlation was used to evaluate concordance between child and parent reports of HRQoL. RESULTS: HRQoL reported on the PedsQL by children with CVS was lower than that reported by children with IBS (P < .01) and healthy controls (P < .001), but did not differ from that reported by children with organic gastrointestinal disorders. Children with CVS also had lower HRQoL compared with healthy controls by parent-proxy report on the PedsQL (P < .001). Correlations between HRQoL reports by parents and children were moderate to good (intraclass correlation coefficients, 0.504-0.805; P < .01). Duration of CVS episodes, delay in CVS diagnosis, and number of school days missed due to CVS were associated with lower parent-rated HRQoL (P = .01). CONCLUSION: Children with CVS reported lower HRQoL compared with those with IBS, and both parents and children reported lower HRQoL compared with healthy controls. Parent and child ratings of HRQoL converged. Improved recognition of CVS and school support might help mitigate the impact of CVS on HRQoL.

Health related quality of life in patients with biliary atresia surviving with their native liver.

OBJECTIVES: To quantify health related quality of life (HRQOL) of patients with biliary atresia with their native livers and compare them with healthy children and patients with biliary atresia post-liver transplant (LT) and to examine the relationship between HRQOL and medical variables. STUDY DESIGN: A cross-sectional HRQOL study of patients with biliary atresia with their native livers (ages 2-25 years) was conducted and compared with healthy and post-LT biliary atresia samples using Pediatric Quality of Life Inventory 4.0 child self and parent proxy reports, a validated measure of physical/psychosocial functioning. RESULTS: 221 patients with biliary atresia with native livers (54% female, 67% white) were studied. Patient self and parent proxy reports showed significantly poorer HRQOL than healthy children across all domains (P < .001), particularly in emotional and psychosocial functioning. Child self and parent proxy HRQOL scores from patients with biliary atresia with their native livers and post-LT biliary atresia were similar across all domains (P = not significant). Child self and parent proxy reports showed moderate agreement across all scales, except social functioning (poor to fair agreement). On multivariate regression analysis, black race and elevated total bilirubin were associated with lower Total and Psychosocial HRQOL summary scores. CONCLUSIONS: HRQOL in patients with biliary atresia with their native livers is significantly poorer than healthy children and similar to children with post-LT biliary atresia. These findings identify significant opportunities to optimize the overall health of patients with biliary atresia.