Pediatric Quality Of Life Inventory Research Articles

Оцінка якості життя дітей після оперативних втручань на серці: дослідження та висновки

Studying the quality of life of patients, in particular children, is one of the important areas of modern medicine. Aim. To assess the quality of life of children who were operated on for congenital heart disease. Materials and methods. 100 children from the western regions of Ukraine aged 6 to 18 who were operated on for congenital heart disease and 100 of their parents were interviewed. A quantitative assessment of the quality of life of children was carried out using the international standardized Pediatric Quality of Life Inventory (PedsQLTM 4.0 Generic Core Scales, France) survey for children with cardiovascular disorders and their parents. In total, 57% (57 people) of boys and 43% (43 people) of girls took part in the study. The share of surveyed mothers is 84% (84 people), and fathers - 16% (16 people). Statistical processing of materials was performed using the Statistica 6.0 software package. The reliability of the difference in indicators was assessed using the Student's t-test. The average value (X) and the error of the average (s) were calculated. The difference of P<0.05 was considered statistically significant. Results. According to the children's questionnaire, when studying the quality of life of children, the indicators of the physical functioning scale had an average value of 67.31±19.52 points; according to parental questionnaires, the average value of this scale was 62.81±20.58 points. According to information from the child questionnaire, the psychosocial functioning scale had an average value of 71.06±15.17 points and 64.89±17.89 points according to the parental questionnaire. It was found that 62% of children rated the overall quality of their health better than their parents; 26% of children assessed the quality of their health almost the same as their parents; and only 12% of children rated the quality of their health worse than their parents. Conclusions. The overall assessment of the quality of life according to the children's questionnaire was 69.18±17.34 points, while according to the parental questionnaire it was 63.85±19.23 points. In some aspects, parents rated the physical and psychosocial quality of life of their children after heart surgery more negatively than the children themselves, but there was no significant difference (P˃0.05). No conflict of interests was declared by the authors.

Parental Stress and Child Quality of Life after Pediatric Burn

Parents’ emotions after their child’s burn might be influenced by the injury circumstances or demographic characteristics of the patient and family. Parents’ post-traumatic stress symptoms and their child’s distress may interact and affect emotional states. The psychosocial outcomes of parents were measured using the Impact of Event Scale-Revised, the CARe Burn Scale, and the Post-traumatic Growth Inventory-Brief. The psychosocial quality of life outcomes of the pediatric burn patients were measured using the Pediatric Quality of Life Inventory (PedsQL). Regression analysis was used to assess the relationship between patient psychosocial quality of life and the related parent scores. A total of 48 patients and parents participated, with 36 giving full data at 12 months. Parental post-traumatic stress symptoms were initially high, settling by six months, although outliers remained. Parents reported higher IESR scores if their child was female, if they felt helpless at the time of the incident, and if a language other than English was spoken in the home. Parents’ scores of their child’s psychosocial function were similar to their child’s self-scores. Parents who perceived poorer emotional functioning in their child reported higher IESR scores.

Acceptability and tolerability of long-acting injectable cabotegravir or rilpivirine in the first cohort of virologically suppressed adolescents living with HIV (IMPAACT 2017/MOCHA): a secondary analysis of a phase 1/2, multicentre, open-label, non-comparative dose-finding study

Long-acting injectable cabotegravir and rilpivirine have demonstrated safety, acceptability, and efficacy in adults living with HIV-1. The IMPAACT 2017 study (MOCHA study) was the first to use these injectable formulations in adolescents (aged 12-17 years) living with HIV-1. Herein, we report acceptability and tolerability outcomes in cohort 1 of the study. In this a secondary analysis of a phase 1/2, multicentre, open-label, non-comparative dose-finding study, with continuation of pre-study oral combination antiretroviral treatment (ART), 55 adolescents living with HIV-1 were enrolled to receive sequential doses of either long-acting cabotegravir or rilpivirine and 52 received at least two injections. Participants had a body weight greater than 35 kg and BMI less than 31·5 kg/m2 and had been on stable ART for at least 90 consecutive days with an HIV-1 viral load of less than 50 copies per mL at a participating IMPAACT study site. Participants had to be willing to continue their pre-study ART during cohort 1. The primary objectives of the study were to confirm doses for oral and injectable cabotegravir and for injectable rilpivirine in adolescents living with HIV. This analysis of participant-reported outcomes included a face scale assessment of pain at each injection and a Pediatric Quality of Life Inventory (PedsQL) at baseline and week 16 for participants in the USA, South Africa, Botswana, and Thailand. A subset of 11 adolescents and 11 parents or caregivers in the USA underwent in-depth interviews after receipt of one or two injections. This trial is registered at ClinicalTrials.gov, NCT03497676. Between March 19, 2019, and Nov 25, 2021, 55 participants were enrolled into cohort 1. Using the six-point face scale, 43 (83%) of participants at week 4 and 38 (73%) at week 8 reported that the injection caused "no hurt" or "hurts little bit", while only a single (2%) participant for each week rated the pain as one of the two highest pain levels. Quality of life was not diminished by the addition of one injectable antiretroviral. In-depth interviews revealed that parents and caregivers in the USA frequently had more hesitancy than adolescents about use of long-acting formulations, but parental acceptance was higher after their children received injections. High acceptability and tolerability of long-acting cabotegravir or rilpivirine injections suggests that these are likely to be favoured treatment options for some adolescents living with HIV. National Institutes of Health and ViiV Healthcare.

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.

Validation of the Sibling Perception Questionnaire in healthy siblings of children with chronic illnesses.

The purpose of the study was to assess the psychometric properties of the parent-report version of the Sibling Perception Questionnaire (SPQ) in well-siblings. Participants were 200 caregivers of healthy children ages 9-17 years who had a sibling (ages 0-18 years) diagnosed with cancer, diabetes, cystic fibrosis, epilepsy, spina bifida, or congenital heart disease. The SPQ had acceptable internal consistency reliability for the total score (α = 0.83) and displayed acceptable convergent validity as evidenced by medium to large positive correlations with the Strengths and Difficulties Questionnaire and Pediatric Quality of Life Inventory 4.0 (r's ranged from 0.39 to 0.56). The original four-factor model displayed a poor model fit (CFI = 0.6, RMSEA = 0.11, TLI = 0.55, SRMR = 0.14) in comparison to a revised, 14-item four-factor model (CFI = 0.92, RMSEA = 0.07, TLI = 0.90, SRMR = 0.06). The shortened, 14-item parent-report version of the SPQ largely demonstrated good psychometric properties and has the potential to reduce the burden of caregivers filling out the measure.

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

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. 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. We analyzed data for 766 patients across all PedsQL age group versions (1-12months through 13-18years). 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. 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.

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

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

Do preinjury life events moderate the outcomes of mild traumatic brain injuries in children? An A-CAP Study.

To examine preinjury life events as moderators of postconcussive symptoms (PCS) and quality of life (QoL) in children with pediatric mild traumatic brain injury (mTBI) versus orthopedic injury (OI). Participants were 633 children with mTBI and 334 with OI, ages 8-16.99, recruited from 5 pediatric emergency departments and followed for 6 months postinjury as part of a prospective cohort study. Preinjury life events were measured retrospectively using the Child and Adolescent Survey of Experiences, PCS using the Health and Behavior Inventory (HBI) and Post-Concussion Symptom Interview (PCS-I), and QoL using the Pediatric Quality of Life Inventory (PedsQL). Analyses involved longitudinal regression using restricted cubic splines, with group, positive and negative life events, and time as primary predictors. Covariates included age, sex, race, socioeconomic status, preinjury history (i.e., headache, migraine, previous concussion), and parent-rated retrospective PCS-I, HBI, and PedsQL scores. PCS and QoL were worse after mTBI than OI, but group differences declined with time (all p < .001). Group differences in PCS were larger at higher levels of positive life events, which predicted lower PCS (p= .03 to p < .001) and higher QoL (p = .048) after OI but not after mTBI. Negative life events predicted worse PCS and QoL in both groups (p = .002 to p < .001). Preinjury positive life events moderate outcomes after pediatric injury, with a protective effect seen in OI but not in mTBI. Negative life events are consistently associated with worse outcomes regardless of injury type.

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

BackgroundHeath-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.MethodsWe 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.ResultsIn 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.ConclusionsThe 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 registrationTrial registration name The Bridge and registration number NCT04631965 (https://clinicaltrials.gov/ct2/show/NCT04631965).

Adherence to medical treatment for Wilson’s disease in children and adolescents: a cohort study from Turkey

BackgroundThis study aimed to assess medication adherence and demographic, clinical, and psychopathological parameters such as quality of life, depression, and anxiety levels that can affect pediatrics with Wilson’s Disease (WD).MethodsA prospective cohort study was conducted at an outpatient clinic in Turkey among pediatric patients (2 to 18 years) with WD between November 2022 and April 2023. The Medication Adherence Report Scale (MARS-5) as a subjective and Medication Possession Ratio (MPR) as an objective assessment were scored. Physical, genetic and biochemical parameters, the Pediatric Quality of Life Inventory (PedsQL) for both parents and patients, Childhood Depression Inventory, State Trait Anxiety Inventory were also administered.ResultsA total of 30 pediatric outpatients who were prescribed D-penicillamine (n = 27) or trientine (n = 3) as chelators and zinc (n = 29) and pyridoxine (n = 19) as supplements were included. Proteinuria (n = 3), skin rash (n = 2), and gastrointestinal upset (n = 2) were observed. When the correlation between MARS-5 and duration of follow-up was examined, a significant negative correlation was found (p = 0.014). According to MPRs, non-adherence rates (missed doses ≥ 20%) were 29.6%, 17.2% and 5.3% for D-penicillamine, zinc and pyridoxine, respectively. PedsQL scores were higher than those of parents, with a positive correlation between them (p < 0.001). Also, there was a significant positive correlation between PedsQL and State Anxiety Inventory (p < 0.001). Comparing the change in urinary copper levels between different levels of treatment knowledge, significant differences were observed between high- and low levels (p = 0.043).ConclusionsOverall, nonadherence rates were 23.3% based on MARS-5 and 5.3–29.6% based on MPR. It is essential to consider factors such as the duration of follow-up, biochemical parameters, treatment knowledge, quality of life and anxiety as potential influencers of medication adherence.

Evaluation of Sleep Habits and Their Relationship With Quality of Life in Children With Epilepsy

BackgroundThe aim of this study was to investigate sleep habits, quality of life (QoL), and the relationship between them in children with epilepsy. MethodsIn this cross-sectional study, children aged two to 18 years being followed up for epilepsy were assessed using the Children's Sleep Habits Questionnaire (CSHQ) and the Pediatric Quality of Life Inventory (PedsQL). Pearson or Spearman correlation analysis was performed to examine the relationship between normally distributed and non-normally distributed variables, respectively. Linear regression analysis was used to examine independent variables associated with PedsQL total scale score. Level of significance was accepted as P < 0.05. ResultsThe study included 112 children with a mean age of 10.5 ± 4.4 years (51.8% female). The frequency of poor sleep habits was 96.4%. There was a good level of agreement between children's and parents' PedsQL total, physical health, and psychosocial health scores (P < 0.001). Correlation analysis between QoL and sleep parameters revealed negative correlations between total sleep score and self-assessed PedsQL total scale, physical health, and psychosocial health scores (P < 0.05) and parent-assessed PedsQL total scale and psychosocial health scores (P < 0.05). The results of linear regression analysis indicated that the factors most significantly associated with lower QoL were high CSHQ total sleep score and exclusively daytime seizures (P < 0.001). ConclusionsIt was found that children with epilepsy had poor sleep habits and low QoL and that poor sleep habits have a negative impact on QoL.

Can the Lower Extremity Functional Scale Be Used in Children and Adolescents? A Validation Study.

The lower extremity functional scale (LEFS) is a patient-reported outcome measure for assessment of lower extremity function. It has been validated in adults but not in children or adolescents. Patients 8 to 18 years of age who were treated for a lower limb fracture, injury, or other conditions were invited to join the study. LEFS and Pediatric Quality of Life Inventory 4.0 Generic Core Scales (PedsQL) were administered. Reliability and validity of the LEFS were determined for the entire cohort and for 2 age groups (children: 8 to 12 years; adolescents 13 to 18 years) using PedsQL as comparison. A total of 178 patients were recruited into this study. In the entire cohort, internal consistency of LEFS was excellent (0. 972) with acceptable floor (0%) and ceiling (12%) effects. Correlation between LEFS and PedsQL physical functioning component was high ( r =0.859). Construct validity was acceptable, with all 8 hypotheses demonstrating statistical significance. Factor analysis showed that item 15 (sitting for 1 hour) may contribute to measurement error in the pediatric population. Results remained similar when comparing the 2 age groups. The LEFS is overall an acceptable patient-reported outcome assessment of children and adolescents with various lower limbs disorders. Level II.

Prospective Evaluation of Health-Related Quality-of-Life in Children with Craniosynostosis.

To investigate health-related quality of life (HRQL) in children aged 2 to 7 years, who have undergone surgery for craniosynostosis. Cross-sectional survey. A tertiary pediatric academic medical center. Children with craniosynostosis who underwent surgical correction, and who were 2-7 years old at the time of the study. Children from families that did not speak English were excluded. Caregivers were asked to fill out the Pediatric Quality of Life Inventory (PedsQL) Core Parent Report and the PedsQL Cognitive Functioning Scale. PedsQL: Psychosocial Health Summary Score, Physical Health Summary Score, Total Core Score, Cognitive Functioning Scale Score. Scores range from 0 to 100, with higher scores reflecting greater QoLSubject factors: comorbidities, syndromic status, type of craniosynostosis, type of surgery. The study included 53 subjects, of whom 13.2% had a syndrome. Core and cognitive scores did not depend on presence of a syndrome or suture involved. Subjects who underwent posterior cranial distraction achieved higher Total Core Scores than subjects who underwent open vault remodeling. Among subjects with sagittal craniosynostosis, there was a tendency for higher scores among children who underwent minimally-invasive surgery compared to those who underwent open vault remodeling. This study demonstrates similar HRQL among children with and without a syndrome, higher HRQL among children undergoing posterior cranial distraction than those undergoing open vault remodeling, and trends towards higher HRQL in children with sagittal craniosynostosis who underwent minimally-invasive surgery compared to those who underwent open vault remodeling.

L-serine treatment in patients with GRIN-related encephalopathy: a phase 2A, non-randomized study.

GRIN-related disorders are rare developmental encephalopathies with variable manifestations and limited therapeutic options. Here, we present the first non-randomized, open-label, single-arm trial (NCT04646447) designed to evaluate the tolerability and efficacy of L-serine in children with GRIN genetic variants leading to loss-of-function. In this phase 2A trial, patients aged 2-18 years with GRIN loss-of-function pathogenic variants received L-serine for 52 weeks. Primary end points included safety and efficacy by measuring changes in the Vineland Adaptive Behavior Scales, Bayley Scales, age-appropriate Wechsler Scales, Gross Motor Function-88, Sleep Disturbance Scale for Children, Pediatric Quality of Life Inventory, Child Behavior Checklist and the Caregiver-Teacher Report Form following 12 months of treatment. Secondary outcomes included seizure frequency and intensity reduction and EEG improvement. Assessments were performed 3 months and 1 day before starting treatment and 1, 3, 6 and 12 months after beginning the supplement. Twenty-four participants were enrolled (13 males/11 females, mean age 9.8 years, SD 4.8), 23 of whom completed the study. Patients had GRIN2B, GRIN1 and GRIN2A variants (12, 6 and 5 cases, respectively). Their clinical phenotypes showed 91% had intellectual disability (61% severe), 83% had behavioural problems, 78% had movement disorders and 58% had epilepsy. Based on the Vineland Adaptive Behavior Composite standard scores, nine children were classified as mildly impaired (cut-off score > 55), whereas 14 were assigned to the clinically severe group. An improvement was detected in the Daily Living Skills domain (P = 0035) from the Vineland Scales within the mild group. Expressive (P = 0.005), Personal (P = 0.003), Community (P = 0.009), Interpersonal (P = 0.005) and Fine Motor (P = 0.031) subdomains improved for the whole cohort, although improvement was mostly found in the mild group. The Growth Scale Values in the Cognitive subdomain of the Bayley-III Scale showed a significant improvement in the severe group (P = 0.016), with a mean increase of 21.6 points. L-serine treatment was associated with significant improvement in the median Gross Motor Function-88 total score (P = 0.002) and the mean Pediatric Quality of Life total score (P = 0.00068), regardless of severity. L-serine normalized the EEG pattern in five children and the frequency of seizures in one clinically affected child. One patient discontinued treatment due to irritability and insomnia. The trial provides evidence that L-serine is a safe treatment for children with GRIN loss-of-function variants, having the potential to improve adaptive behaviour, motor function and quality of life, with a better response to the treatment in mild phenotypes.

Evaluation of the effect of palatoplasty on the quality of life and speech outcomes in patients with velocardiofacial syndrome.

Children diagnosed with velocardiofacial syndrome (VCFS) suffer from various disabilities. Palatal abnormalities, as well as speech and language impairment, adversely affect a child's quality of life (QoL) and are some of the most distressing aspects for the parents of these children. The present study aimed to explore the effect of palatoplasty on the health-related quality of life (HRQoL) and speech outcomes in children with VCFS. The study recruited 20 patients (N = 20) with VCFS and connected speech, aged 3 years or older, having either undiagnosed submucous cleft palate (SMCP) or velopharyngeal insufficiency (VPI), and requiring primary cleft palate surgery or revision surgery. Speech assessment was conducted prior to palatoplasty and 6 months after the surgery. Intelligibility and hypernasality were evaluated using the Cleft Audit Protocol for Speech - Augmented (CAPS-A). The parent proxy-report form of the Pediatric Quality of Life Inventory (PedsQL™) was used to evaluate and compare the HRQoL of the VCFS patients before and after palatoplasty. Significant improvement in the HRQoL scores was achieved after the surgery across all domains (physical, emotional, social, and school functioning), especially in the emotional and social dimensions (p < 0.000). The post-operative speech assessment based on CAPS-A demonstrated improvement in speech intelligibility and hypernasality in the majority of patients. Given that children with VCFS face various medical and social problems, suitable palatal interventions are beneficial, improving both the speech ability and QoL of these children.

Evaluation of quality of life in children with precocious puberty.

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. 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. 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). 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.

Caregiver strategies supporting community participation among children and youth with or at risk for disabilities: a mixed-methods study.

The purpose of this mixed-methods study is to examine the role of caregiver strategies to support community participation among children and youth with disabilities and those at risk, from the caregiver perspective. For the quantitative phase, we tested the hypothesized positive effect of participation-focused caregiver strategies on the relationship(s) between participation-related constructs and community participation attendance and involvement. For the qualitative phase, we solicited caregiver perspectives to explain the quantitative findings. An explanatory sequential mixed-methods design (QUAN > qual) was used. For the quantitative phase, we conducted secondary analyses of data collected during a second follow-up phase of a longitudinal cohort study, including 260 families of children and youth (mean age: 13.5 years) with disabilities and those at risk [i.e., 120 families of children and youth with craniofacial microsomia (CFM); 140 families of children and youth with other types of childhood-onset disabilities]. Data were collected through the Participation and Environment Measure-Children and Youth, the Pediatric Quality of Life Inventory, and the Child Behavior Checklist and analyzed using structural equation modeling. For the qualitative phase, we conducted semi-structured interviews with eight caregivers of children and youth with disabilities and those at risk (i.e., three caregivers of children and youth with CFM; five caregivers of children and youth with other childhood-onset disabilities). Interviews were transcribed verbatim and inductively content-analyzed. Our model reached acceptable to close model fit [CFI = 0.952; RMSEA = 0.068 (90% CI = 0.054-0.082); SRMR = 0.055; TLI = 0.936], revealing no significant effect of the number of participation-focused caregiver strategies on the relationships between participation-related constructs (e.g., activity competence, environment/context) and community participation in terms of attendance and involvement. The qualitative findings revealed three main categories for how caregivers explained these quantitative results: (1) caregiver workload and supports needed for implementing strategies; (2) caregivers careful strategy quality appraisal; and (3) community setting characteristics hindering successful strategy implementation. The findings suggest that the insignificant effect of the number of caregiver strategies may be explained by the intensified need for caregiver effort and support to develop and implement quality strategies that are responsive to community setting characteristics.

Association of an In-Hospital Desirability of Outcomes Ranking Scale With Postdischarge Health-Related Quality of Life: A Secondary Analysis of the Life After Pediatric Sepsis Evaluation.

To develop a desirability of outcome ranking (DOOR) scale for use in children with septic shock and determine its correlation with a decrease in 3-month postadmission health-related quality of life (HRQL) or death. Secondary analysis of the Life After Pediatric Sepsis Evaluation prospective study. Twelve U.S. PICUs, 2013-2017. Children (1 mo-18 yr) with septic shock. None. We applied a 7-point pediatric critical care (PCC) DOOR scale: 7: death; 6: extracorporeal life support; 5: supported by life-sustaining therapies (continuous renal replacement therapy, vasoactive, or invasive ventilation); 4: hospitalized with or 3: without organ dysfunction; 2: discharged with or 1: without new morbidity to patients by assigning the highest applicable score on specific days post-PICU admission. We analyzed Spearman rank-order correlations (95% CIs) between proximal outcomes (PCC-DOOR scale on days 7, 14, and 21, ventilator-free days, cumulative 28-day Pediatric Logistic Organ Dysfunction-2 (PELOD-2) scores, and PICU-free days) and 3-month decrease in HRQL or death. HRQL was measured by Pediatric Quality of Life Inventory 4.0 or Functional Status II-R for patients with developmental delay. Patients who died were assigned the worst possible HRQL score. PCC-DOOR scores were applied to 385 patients, median age 6 years (interquartile range 2, 13) and 177 (46%) with a complex chronic condition(s). Three-month outcomes were available for 245 patients (64%) and 42 patients (17%) died. PCC-DOOR scale on days 7, 14, and 21 demonstrated fair correlation with the primary outcome (-0.42 [-0.52, -0.31], -0.47 [-0.56, -0.36], and -0.52 [-0.61, -0.42]), similar to the correlations for cumulative 28-day PELOD-2 scores (-0.51 [-0.59, -0.41]), ventilator-free days (0.43 [0.32, 0.53]), and PICU-free days (0.46 [0.35, 0.55]). The PCC-DOOR scale is a feasible, practical outcome for pediatric sepsis trials and demonstrates fair correlation with decrease in HRQL or death at 3 months.

Non-Pharmacologic Intensive Interdisciplinary Pain Treatment in Pediatrics: Impact on Symptoms, Daily Functioning, and the Family Unit.

Objectives: To assess non-pharmacologic treatment outcomes pertaining to health-related quality of life (HRQoL) in youth with chronic idiopathic pain and their families. Methods: We conducted a retrospective cohort study of 115 youth with chronic idiopathic pain enrolled in a non-pharmacologic, hospital-based intensive interdisciplinary pain treatment (IIPT) program. HRQoL measures for the patient (Pediatric Quality of Life Inventory [PedsQL] short form) and family unit (PedsQL Family Impact) were collected on admission and discharge as part of routine clinical care. Changes in PedsQL scores were calculated using the Wilcoxon signed-rank test. Multivariable linear regression was used to explore factors associated with patient-level HRQoL. Results: Both individuals and the family unit reported that their HRQoL improved in all domains by program completion. Improvements in pain and allodynia were present for program participants at the time of completion as well as at the 3-month follow-up, suggesting durability of these effects. Conclusions: A non-pharmacologic IIPT program is a compelling treatment for pediatric and adolescent chronic idiopathic pain, for both patients and the family unit. Patients participating in this program had positive treatment outcomes with significantly improved subjective and objective measures of physical, emotional, social, and cognitive function.

How do gene mutation diversity and disease severity scoring affect physical capacity and quality of life in children/adolescents with Familial Mediterranean Fever?

ObjectivesThe aim of this study is to examine how gene mutation diversity and disease severity affect physical capacity and quality of life in children/adolescents with Familial Mediterranean Fever (FMF). MethodsEighty children/adolescents (42 female, 38 male) diagnosed with FMF according to Tell-Hashomer diagnostic criteria were included in this study. Disease severity score (PRAS), running speed and agility and strength subtests of Bruininks-Oseretsky Test of Motor Proficiency Second Edition Short Form (BOT-2 SF), Physical Activity Questionnaire, Pediatric Quality of Life Inventory 3.0 Arthritis Module (PedsQL) was used for evaluation. Participants were divided into 2 groups as M694V and other mutations according to MEFV gene mutation and were divided into 3 groups as mild, moderate and severe according to PRAS. ResultsWhen the data were compared between groups; in terms of gene mutation, a significant difference was observed in treatment subtest of PedsQL-parent form in favor of the M694V gene mutation group (p<0.05). In terms of PRAS, significant difference was seen in the pain, treatment subtests and total score of the PedsQL-child form, and in the pain, treatment, worry subtests and total score of the PedsQL-parent form in favor of the mild group (p<0.05). ConclusionsMEFV gene mutations in children and adolescents with FMF did not differ on physical capacity and quality of life. PRAS was not effective on physical parameters, but quality of life decreased as the severity score increased. Encouraging children/adolescents with FMF to participate in physical activity and to support them psychosocially can be important to improve their quality of life.