Health-related Quality Of Life In Pediatric Patients Research Articles

An analysis of health-related quality of life in children and adolescents after parotidectomy based on patient-reported outcomes.

To assess health-related quality of life (HRQoL) and its influencing factors in these pediatric patients undergoing parotidectomy. This was a cross-sectional study that included 37 children and adolescents (≤ 19years) with parotid gland tumors who were treated in Sichuan Cancer Hospital between January 2006 and November 2021. HRQoL was assessed using the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-C30 (EORTC QLQ-C30). The Wilcoxon rank sum test was used to analyze the factors influencing patients' HRQoL. 37 children and adolescents were included in the study, including 22 cases of benign tumors and 15 cases of malignant tumors. All patients underwent surgery, and some patients with malignant tumors received radiotherapy or chemotherapy. Malignancy, permanent facial palsy, and Frey syndrome were associated with worse HRQoL in children and adolescents with parotid gland tumors. Radiotherapy and no cervical lymph node dissection were associated with worse HRQoL in pediatric patients with malignancy. The surgical approach of parotid is not a factor influencing HRQoL. Factors associated with HRQoL in children and adolescents with parotid gland tumors include pathological types, permanent facial palsy, and Frey syndrome. In addition, factors affecting patients with malignancy include lateral lymph node dissection and radiotherapy.

Health-related quality of life among paediatric patients with coarctation of the aorta: an observational study

ABSTRACT Advancements in early diagnosis and paediatric cardiac surgery have improved the long-term survival of patients with congenital heart disease, necessitating a thorough assessment of their health-related quality of life (HRQoL). This study aimed to assess HRQoL in paediatric patients with coarctation of the aorta (CoA) (both as reported by patients and caregivers), and to evaluate associated factors. Patients aged 5–18 years diagnosed with CoA and their parents were enrolled at Bambino Gesù Children’s Hospital between September 2016 and December 2017. Socio-demographic characteristics were recorded using a family form, and the Pediatric Quality of Life Inventory (PedsQL) 3.0 cardiac module was used to evaluate HRQoL. Clinical data were retrieved from medical chart reviews. In this observational study, sixty-five pediatric patients (39 males, median [IQR] age 12 [9–14]) with CoA and their parents (65 mothers and 65 fathers) were enrolled. These patients exhibited overall good HRQoL. Mothers reported significantly lower total HRQoL scores compared to patient self-reports (p = .037), as well as treatment anxiety (p = .033), and cognitive problems (p = .021). Pediatric patients with CoA perceived their HRQoL better than their mothers did. Female sex and older age were associated with lower HRQoL scores.

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.

Self-Reported Health-Related Quality of Life of Children with Spinal Muscular Atrophy: Preliminary Insights from a Nationwide Patient Registry in Germany.

Spinal muscular atrophy (SMA) is a rare, severely debilitating neuromuscular disease characterized by a wide spectrum of progressive muscular atrophy and weakness. The objective of this pilot study was to estimate self-assessed health-related quality of life (HRQoL) of children with SMA. Children with SMA were recruited via the German national TREAT-NMD SMA patient registry and asked to self-complete the following rating-scales: KIDSCREEN-27, KINDL, the PedsQL 3.0 Neuromuscular Module (PedsQL 3.0 NMM), EQ-5D-5L, and the Health Utilities Index (HUI). Estimates were stratified by current best motor function of the lower limb and trunk (i.e., non-sitter, sitter, and walker) and SMA type (i.e., type I, II, and III). In total, 17 children with SMA (mean age: 9.88 years, SD: 4.33 years, range: 5-16 years; 59% female) participated in the study. Across examined strata, the mean KIDSCREEN-27 total score was estimated at between 48.24 and 83.81; the mean KINDL total score at between 60.42 and 76.73; the mean PedsQL 3.0 NMM total score at between 58.00 and 83.83; the mean EQ-5D-5L utility at between 0.31 and 0.99; and the mean HUI-derived utility at between -0.02 and 0.96. The results from this pilot study show that German children with SMA, despite significant physical disability, have surprisingly good HRQoL as assessed using KIDSCREEN-27. Yet, many reside in health states associated with low utility. The disease burden was generally higher among non-sitters compared with walkers, and SMA type I compared with type III, but more research is needed to further delineate this variability. Our preliminary findings contribute to the understanding of HRQoL in pediatric patients with SMA and should be helpful to inform the design of future studies of this patient population.

Pain and health-related quality of life in Duchenne muscular dystrophy: A multiple mediator analysis.

The objective was to investigate the serial mediating effects of daily activities, patient health communication, and disease-specific worry in the relationship between pain intensity and overall generic health-related quality of life (HRQOL) in pediatric patients with Duchenne muscular dystrophy from the patient perspective. Pain Intensity Item, Daily Activities Scale, Communication Scale and Worry Scale from Pediatric Quality of Life Inventory (PedsQL) Duchenne Muscular Dystrophy Module and PedsQL 4.0 Generic Core Scales were completed by 110 pediatric patients with Duchenne muscular dystrophy ages 8-17. A serial multiple mediator model analysis was conducted to test the hypothesized sequential mediating effects of daily activities, patient health communication, disease-specific worry as intervening variables in the association between the pain intensity predictor variable and overall generic HRQOL. Pain predictive effects on overall generic HRQOL were serially mediated by daily activities, patient health communication, and disease-specific worry. In a predictive analytics model utilizing hierarchical multiple regression analysis with age demographic covariate, patient-reported pain intensity, daily activities, patient health communication, and disease-specific worry accounted for 47% of the variance in overall generic HRQOL (P<0.001), representing a large effect size. Pain intensity, daily activities, patient health communication, and disease-specific worry explain in part the mechanism of pain predictive effects on overall generic HRQOL in pediatric patients with Duchenne muscular dystrophy. Identifying the mediators of pain intensity on overall generic HRQOL from the patient perspective may inform targeted clinical interventions and future patient-centered clinical research to improve overall daily functioning.

PCR128 Measurement of Health-Related Quality of Life in Pediatric Genetic Conditions: A Scoping Review

Measuring health-related quality of life (HRQoL) is key to patient-centered value assessment of genomic medicine interventions, including genomic sequencing and gene-based therapies. Our objective is to describe the empirical literature on HRQoL of pediatric patients with genetic conditions, as well as HRQoL and well-being of their family members.

Assessment of Quality of Life in Children With Pulmonary Hypertension Using Parent and Self-report Questionnaires

Pulmonary arterial hypertension (PAH) is a progressive disease characterized by elevation of pulmonary vascular resistance and right ventricular failure. By using advanced therapies to reduce mortality, clinicians focus on improving functional status and quality of life (QOL). The aim of our study was to assess health-related QOL of pediatric patients with PAH. Parents of all children (aged 2-18 years) and patients aged 5-18 years with an appropriate level of intellectual development completed general and cardiac-specific validated surveys (Pediatric Quality of Life Inventory 4.0 and Pediatric Quality of Life Inventory 3.0, respectively). Demographic and clinical information was collected to grade disease severity. Twenty-five children were enrolled, yielding 25 parent reports and 15 patient self-reports. The PAH group had significantly lower scores than healthy children in all domains. Patients with World Health Organization Functional Class I had significantly higher parent proxy scores in School Functioning (P=.029) and in Heart Problems and Symptoms domain (P=.014) Patients with tricuspid annular plane systolic excursion below -2 z score showed impairment in each parent proxy general domain and in the Cognitive Problems score of the Cardiac module (P=.006). In conclusion the QOL of patients with PAH was impaired in every domain compared with healthy children. Patients with reduced right ventricle systolic function showed significantly lower QOL in all core domains. These results point to the need for psychosocial rehabilitation in addition to somatic care to improve the QOL in this severely ill population.

Health-Related Quality of Life in Pediatric Inflammatory Bowel Disease Patients: A Narrative Review.

Inflammatory bowel disease (IBD) is a chronic autoimmune condition that can have a wide range of symptoms among pediatric patients. Although clinical symptoms like hematochezia, diarrhea, and abdominal pain are commonly addressed, health-related quality of life (HRQOL) is often overlooked in patients with IBD and pediatric patients with chronic disease in general. Examining HRQOL can help improve patient outcomes, but it has been studied sparingly. In this review, we aim to compare HRQOL between pediatric patients suffering from IBD and healthy children, as well as those suffering from other illnesses. We searched through peer-reviewed primary literature related to IBD and HRQOL and selected 10 articles from the PubMed database to be reviewed. Our inclusion criteria included articles published after the year 2000 in English, primary studies, and those that corresponded to the aim of this review. Case reports and secondary and tertiary articles were excluded from our review. We found that patients with IBD reported worse HRQOL in terms of overall health and in various subdomains, including physical health and fatigue, compared to their healthy counterparts. However, children with IBD demonstrated a comparable HRQOL with children suffering from functional abdominal pain (FAP) and obesity. Additionally, children with IBD displayed a greater HRQOL than pediatric patients with gastroesophageal reflux disease (GERD) and chronic constipation. In addressing the aim of this review, we found that children with IBD had a lower HRQOL when compared to healthy children, but a comparable or greater HRQOL than other sick children. Some factors associated with a reduced HRQOL include disease activity, age, fatigue, gender, psychological variables, and associated symptoms. Going forward, HRQOL should be considered by practitioners when caring for pediatric IBD patients in a clinical setting as it can help improve patient care. More studies need to be conducted to further explore HRQOL in pediatric patients. This can help implement early psychosocial interventions in children to reduce the disease burden.

Chronic pain, psychological distress, and quality of life in males with Duchenne muscular dystrophy.

To describe chronic pain in Duchenne muscular dystrophy (DMD) from children's/adolescents' perspectives, explore patient variables associated with self-reported pain, and examine the relationship between chronic pain, psychological functioning, and health-related quality of life (HRQoL). This observational study included a paediatric cohort (aged 8-18 years; median age 9 years 4 months) with DMD under multidisciplinary care (n=45). Clinical data of the latest visit were extracted from the electronic health record and assessment of pain, psychological distress, and HRQoL were performed during the same visit. Thirty-two patients reported pain during the previous 4 weeks, and 18 reported persistent or recurrent chronic pain. Average pain intensity of chronic pain was mild, with regions of the legs (n=12), lower back (n=6), hips (n=6), and shoulder (n=6) most frequently affected. Older age, higher body mass index, being non-ambulatory, wheelchair dependency, and spinal deformities were contextual variables related to the presence of chronic pain. Furthermore, chronic pain was significantly associated with psychological distress and reduced HRQoL in paediatric patients with DMD. Chronic pain in paediatric DMD is frequent and widespread, highlighting the need for pain to be addressed in the routine care of affected young people. Chronic pain may make a significant contribution to psychological distress and impaired HRQoL in paediatric patients with DMD. Chronic pain differs in aetiology, scope, and nature compared with acute pain in paediatric Duchenne muscular dystrophy (DMD). Older age, higher body mass index, being non-ambulatory, wheelchair dependency, and spinal deformities are important patient variables. Chronic pain is significantly associated with psychological distress and reduced health-related quality of life in paediatric DMD.

PB2227: HEALTH-RELATED QUALITY OF LIFE OVER TIME IN PEDIATRIC PATIENTS WITH SICKLE CELL DISEASE ADMITTED FOR VASO-OCCLUSIVE CRISIS.

Background: Sickle cell disease (SCD) is the most common inherited hemoglobinopathy characterized by chronic hemolytic anemia, vaso-occlusive crises (VOCs), multi-organ damage and an impaired health-related quality of life (HRQoL). Previous studies have shown that the occurrence of VOCs affects the HRQoL substantially in children with SCD. However, there is limited evidence about the HRQoL changes over time following hospitalization for VOC. Aims: The aim of this study is to evaluate the longitudinal impact of hospital admission for VOCs on the HRQOL in pediatric patients with SCD. Methods: In this retrospective cohort study, children between the age of 8 and 18 years with SCD treated at the Amsterdam University Medical Centers were included. As part of standard care, these children complete once a year Patient Reported Outcomes measures (PROMs) on the KLIK PROM portal (www.hetklikt.nu). HRQoL was measured with the Pediatric Quality of Life Inventory (PedsQL) (score ranging from 0-100, a higher score indicates better QoL). Data were collected from the medical files of patients, who filled out at least two PedsQL questionnaires between 2012 and 2021. The effect of the occurrence and frequency of hospital admissions for VOCs on the HRQoL was evaluated using a linear mixed model 3, 6, 9 and 12 months after hospital admission, adjusted for age and SCD genotype. Results: In total, 109 children were included with a median age of 11.8 years (IQR: 9.2 – 14.2), including 72 patients (66%) with the severe genotype: HbSS or HbSβ0. During follow-up, 45 patients (41%) were admitted to the hospital for a VOC. If hospitalization occurred 6 and 12 months prior to the PedsQoL measurement, a decrease of 3.61 (p=0.049) and 3.80 points (p=0.007) in HRQoL respectively, was seen compared to patients without hospitalization. As for the frequency of VOC admission in the preceding 12 months, a decrease of 2.28 points in HRQoL was observed for every additional admission (p=0.012). Summary/Conclusion: The negative longitudinal association between hospital admission for VOC and HRQoL in pediatric patients with SCD gets worse and persists up to 12 months. These findings highlight the importance of monitoring using PROMS in clinical practice and extra attention and support for the negative consequences of the hospital admission for VOCs, affecting the HRQoL.

QOL-15. Life happens wherever you are! Use of Avatar AV1 to enhance Health-Related Quality of Life, Sense of Belonging and Social Inclusion in Children and Adolescents with Chronic Illnesses

BACKGROUND: Due to regular hospital check-ups, inpatient treatments, or a weakened immune system, children with brain tumors experience frequent and long absences from school and social activities. Returning to school presents a challenge for these patients, as they experience reduced health-related quality of life (HRQOL), decreased sense of belonging and a lack of social inclusion in class. To prevent social and emotional problems, telepresence systems such as the Avatar AV1 are described as promising approach for pediatric patients. OBJECTIVES: To sustainably improve social inclusion in times of illness-related absence for these patients, the first study in Austria investigating effects of the use of the Avatar is pursuing both, a qualitative and quantitative approach. METHODS: To examine effects on social inclusion, sense of belonging and HRQOL in pediatric patients, interviews were conducted with patients, their parents, teachers and classmates within the qualitative approach and questionnaires were administered at three times (before -, after 6 months Avatar-use and 3 months after returning the Avatar) within the quantitative approach. The sample consists of pediatric patients (6 to 18 years). RESULTS: Categories from n=24 interviews indicate that a positive attitude towards and identification with the Avatar as well as the patients’ psychological condition and social inclusion into class before the illness play major roles. Preliminary findings from the ongoing longitudinal quantitative survey indicate that the Avatar has significant positive and stabilizing effects on HRQOL, sense of belonging and social inclusion of pediatric patients. CONCLUSION: This study is the first to describe the impact of Avatar use on social inclusion in children with brain tumors. To strengthen the sense of belonging in these children, the pedagogical-interactional component needs to be brought in focus. Through pedagogical-didactical adaptions, a routine handling of and a positive attitude towards the telepresence system, pediatric patients highly benefit from the Avatar.

Risk factors for impaired health-related quality of life in a cohort of pediatric patients with inborn metabolic diseases

In the last decade, health-related quality of life (HrQoL) has become an increasingly important outcome parameter in children and adolescents with chronic health conditions; among them are pediatric patients with inborn metabolic diseases (IMDs). Hence, knowledge on this topic is increasing, but findings on non-medical influences on the HrQoL of IMD patients are still scarce. In the present study, we retrospectively evaluated the self-reported generic HrQoL of a cohort of pediatric patients (ages 7 to 17 years) with diverse IMDs (n = 204) and explored associations between HrQoL and psychosocial and medical characteristics of the patients. We aimed to identify risk factors for impaired HrQoL to improve and tailor support for the patients and economize resources. Generic HrQoL was assessed with the KINDL-R questionnaire. We compared the HrQoL scores to published German normative data and analyzed the impact of demographic variables and intellectual and psychosocial functioning on the HrQoL. Moreover, we examined the influence of the diagnostic category and the health impairment (as judged by the physicians) on our patients’ HrQoL. Overall, the HrQoL of the adolescent patients was comparable to the HrQoL of the norm group. Disorders of intellectual development, impaired psychosocial functioning, and a severe health impairment were associated with lower HrQoL scores.Conclusion: We recommend evaluating these factors in children and adolescents with IMDs to identify patients at risk for impaired HrQoL.What is Known:• Studies on HrQoL in pediatric patients with IMDs mainly focused on subgroups with specific diagnoses and found normal HrQoL in some of those subgroups.• In healthy children and adolescents as well as in pediatric patients with various chronic diseases, associations between psychosocial factors and HrQoL are well known.What is New:• Impaired psychosocial functioning, disorders of intellectual development, and a significant disease and/or treatment burden are risk factors for impaired HrQoL in pediatric patients with IMDs.• Evaluating these factors in children and adolescents with IMDs can help identify patients and families in need of enhanced psychological support.

The Association Between Functional Status and Health-Related Quality of Life Following Discharge from the Pediatric Intensive Care Unit.

Despite one third of children with acquired brain injury (ABI) experiencing new functional impairments following critical care admission, there is limited research investigating the impact of new functional impairments on overall health-related quality of life (HRQOL) or among important HRQOL domains. We aimed to investigate the association between new functional impairments, measured by the Functional Status Scale (FSS), and HRQOL in pediatric patients with ABI after critical care. We conducted a secondary analysis of a prospective observational study of 275 children aged 2months to 18years with ABI. The primary exposure evaluated was change in FSS from baseline at hospital discharge, categorized per prior work (no change, 1-2 point increase, and ≥ 3 point increase). The primary outcome was overall HRQOL 6months after hospital discharge, measured by the Pediatric Quality of Life Inventory (PedsQL) total score. Secondary outcomes were PedsQL domain scores. PedsQL total and domain scores were transformed into age-standardized z scores for analyses. Multiple linear regression models evaluated the association between FSS change category and HRQOL (overall and domain z scores) when controlling for demographic and clinical characteristics and were reported as β-coefficients with 95% confidence intervals. Complete data were analyzed for 195 (71%) children, including 127 with traumatic brain injury. New functional impairment was common with 32 (16%) patients experiencing FSS increases ≥ 3, 50 (26%) patients with FSS increases of 1-2 points, and 113 (58%) patients with no change from prehospital baseline. The majority of children (63%) demonstrated HRQOL ratings ≥ 1 standard deviation below healthy age-based standards (z scores ≤ - 1). Regression models demonstrated older age, female sex, presence of comorbidities, and preadmission cardiopulmonary resuscitation were all significantly associated with poorer overall HRQOL (all p < 0.05). FSS increase ≥ 3 at discharge was significantly associated with worse overall HRQOL at follow-up (β = - 1.07; 95% confidence interval = - 1.63 to - 0.52) when controlling for the aforementioned significant factors, and significantly improved model fit (p value for change = 0.001). Similar findings in secondary analyses were found for physical domain scores, with FSS increase showing a significant association with worse physical HRQOL scores and improvements in model fit. Change in FSS was not significantly associated with other HRQOL domain scores (emotional, social, school, psychosocial). Many children with ABI after critical care experience new functional impairments (FSS increases) and worse HRQOL than healthy peers. FSS increase at discharge is a significant risk factor for worse HRQOL in the months after hospital discharge and improves HRQOL models beyond illness and demographic variables alone.

Health-related quality of life (HRQoL) among pediatric patients with neurofibromatosis type 1 (NF1) and plexiform neurofibroma (PN) in the United States (U.S.).

10042 Background: PNs occur in 30-50% of pediatric patients with NF1, often resulting in debilitating pain and dysfunction. Children with NF1 PN report significantly worse HRQoL than the general population, though real-world evidence is limited. Research is needed to better characterize HRQoL among this patient population in the US. Methods: Patients ages 8-18 years with NF1 PN in the US who were treatment naïve or new users of selumetinib (≤1 month of use) were recruited through the Children’s Tumor Foundation to participate with their caregivers in an online cross-sectional survey in December 2020 and January 2021. Caregivers of similar patients ages 2-7 years also participated. Measures included the Pediatric Quality of Life Inventory (PedsQL; Acute version), EQ-5D-Y, Pain Interference Index (PII), Numeric Rating Scale (NRS-11), and the Patient-Reported Outcomes Measurement Information System (PROMIS) mobility and upper extremity functioning subscales. Patients provided self-reported responses; caregivers provided proxy responses and patient demographic and clinical characteristics. Results: 61 patients and 82 caregivers responded to the survey. Median (range) age of patients was 12.0 (8-14) years, and 53.7% were female. Most were treatment naïve (97.6%), white/Caucasian (85.4%), and had an NF1 and PN diagnosis for &gt; 5 years (80.5% and 68.3%, respectively). On the PedsQL (range: 0-100; higher = better; mean scores typically &gt; 80 among healthy patients), mean patient scores were 50.3 (school functioning), 56.1 (emotional functioning), 60.7 (social functioning), and 63.7 (physical functioning); the mean total score was 58.5.Caregiver-proxy mean scores were similar, ranging from 54.0 for school functioning to 65.0 for physical functioning, with a mean total score of 59.1. On the EQ-5D-Y, more than half of patients reported experiencing “some” or “a lot” of problems with pain or discomfort (65.6%) and with feeling worried, sad or unhappy (62.3%). Among patients with pain in the last 7 days, mean scores on the PII (range: 0-6; higher = more interference) were 3.0 for patients and 2.7 for caregiver proxies. Almost 75% of them reported moderate or severe pain on the NRS-11. Among patients with movement difficulty in the past 7 days, mean t-scores from the PROMIS scales (distribution mean = 50; higher = better) were 40.2 for mobility and 39.5 for upper extremity functioning among patients and 36.0 and 29.1, respectively, among caregivers. Within dyads, patients generally reported better functioning, on average, than their caregiver proxies. Conclusions: The humanistic burden of NF1 PN among this pediatric patient population is substantial, especially regarding pain, emotional functioning, and physical functioning. Results highlight an unmet need to be addressed for improving HRQoL in pediatric patients with NF1 and PN.

Cognitive functioning and pain interference mediate pain predictive effects on health-related quality of life in pediatric patients with Neurofibromatosis Type 1

The objective was to investigate the serial mediating effects of perceived cognitive functioning and pain interference in daily living in the relationship between perceived pain and overall generic health-related quality of life (HRQOL) in children, adolescents, and young adults with Neurofibromatosis Type 1 (NF1). The Pain, Cognitive Functioning, and Pain Impact Scales from the PedsQL Neurofibromatosis Type 1 Module and the PedsQL 4.0 Generic Core Scales were completed in a multi-site national study by 323 patients ages 5-25 and 335 parents. A serial multiple mediator model analysis was conducted to test the hypothesized sequential mediating effects of cognitive functioning and pain interference as intervening variables in the association between pain as a predictor variable and overall generic HRQOL. Pain predictive effects on overall generic HRQOL were serially mediated by cognitive functioning and pain interference. In predictive analytics models utilizing hierarchical multiple regression analyses with age and gender demographic covariates, pain, cognitive functioning and pain interference accounted for 66% of the variance in patient-reported generic HRQOL and 57% of the variance in parent proxy-reported generic HRQOL (P<0.001), reflecting large effect sizes. Cognitive functioning and pain interference explain in part the mechanism of pain predictive effects on overall generic HRQOL in pediatric patients with NF1. Identifying NF1-specific pain, cognitive functioning, and pain interference as salient predictors of overall generic HRQOL from the patient and parent perspective facilitates a family-centered orientation to the comprehensive care of children, adolescents, and young adults with NF1.

Colectomy and health-related quality of life in children with ulcerative colitis.

Restorative proctocolectomy and ileal pouch-anal anastomosis (IPAA) is the recommended elective surgery for children with ulcerative colitis (UC). The aim of this study was to evaluate functional and long-term health-related quality of life (HRQoL) outcomes of surgery in paediatric patients with UC. We reviewed the hospital records of all paediatric patients who had undergone surgery for UC between January 2009 and December 2016 in the Units of Paediatric Gastroenterology and Surgery, of both the University Hospital of Messina, and the Gaslini Children's Hospital of Genoa. Surgical treatment was represented by restorative proctocolectomy and laparoscopic IPAA. Patients and parents were interviewed by telephone before and after surgery and responded to the modified IMPACT III questionnaire about health outcomes and HRQoL. The questionnaire was scored on a five-point scale with higher scores indicating a better HRQoL. The total score ranged from 35 (worst HRQoL) to 175 (best HRQoL). Data were obtained in 30 patients (16 males), with a median age of 12 (range 3- 16). The median amount of time elapsed after the operation was 3 years (range 1-4.5). Preoperative scores were very low in all 4 domains of the questionnaire. Postoperatively, HRQoL measures improved significantly (p<0.05), on symptoms, school attendance, social activities, and emotional aspects. Overall, nearly all were completely satisfied with the outcome of surgery. Our data confirmed that surgical treatment improves the overall HRQoL in paediatric patients with UC.

Speech difficulties and patient health communication mediating effects on worry and health-related quality of life in children, adolescents, and young adults with Neurofibromatosis Type 1.

The objective was to investigate the serial mediating effects of speech difficulties, patient health communication, and disease-specific worry in the relationship between neurofibromatosis (NF) symptoms (pain and skin symptoms) and total generic health-related quality of life (HRQOL) in children, adolescents, and young adults with NF Type 1 (NF1) from the patient perspective. The Speech, Communication, Worry, Pain, Skin Itch Bother, and Skin Sensations Scales from the Pediatric Quality of Life Inventory (PedsQL) NF1 Module and the PedsQL 4.0 Generic Core Scales were completed in a multi-site national study by 305 patients ages 5-25 years. A serial multiple mediator model analysis was conducted to test the hypothesized sequential mediating effects of speech difficulties, health communication, and worry as intervening variables in the association between NF1 symptoms and HRQOL. Symptoms predictive effects on total generic HRQOL were serially mediated by speech difficulties, patient health communication, and worry. In predictive analytics models utilizing hierarchical multiple regression analyses with age and gender demographic covariates, the pain, skin itch bother, and skin sensations multiple mediator models accounted for 61%, 59%, and 56% of the variance in generic HRQOL (p < .001), reflecting large effect sizes. Speech difficulties, patient health communication, and disease-specific worry explain in part the mechanism of symptoms predictive effects on total generic HRQOL in pediatric patients with NF1. Identifying NF1-specific predictors and serial mediators of total generic HRQOL in pediatric patients with NF1 from the patient perspective enables a patient-centered comprehensive care approach for children, adolescents, and young adults with NF1.

Health-Related Quality of Life (HRQL) Trajectories during Treatment for Advanced Stage Pediatric Hodgkin Lymphoma (HL)

Introduction: The treatment of advanced stage HL typically includes dose-dense chemotherapy with or without involved field radiation. However, little is known about the HRQL of pediatric patients during initial treatment. We describe the HRQL trajectory over time by child and parent-proxy rater and examine baseline patient characteristics associated with the trajectory.Methods: Children and adolescents, ages 5-17.9 years newly diagnosed with advanced stage HL and enrolled in Children's Oncology Group AHOD 1331, and their parents were co-enrolled in an embedded study to assess HRQL (n=310). Children (age ≥11 years) and parent proxies (of children 5-17.9 years) reported on the child's global HRQL using the Child Health Rating Inventories (CHRIs) at four times: (1) baseline, (2) cycle 2, (3) cycle 5, and (4) end of therapy, approximately 6-7 months following initial diagnosis. The 7-item CHRIs-Global yields scores that range from 0-100, with higher scores indicating better HRQL. A repeated measures linear regression model was fit with categorical time (reference, baseline), rater, child race, ethnicity, and continuous age. An interaction of rater and time was tested and predicted means were plotted.Results: 97% of age-eligible patients and parents completed baseline HRQL assessments with 93% completing planned follow-ups. Median child age was 15.5 years (q1=5.4, q3=18.9) and 50.3% were female. Most children were white (76.1%) and non-Hispanic (82.6%). There was no significant interaction between rater and time (Figure), so this was subsequently excluded from the model. Scores improved slightly at time 2 (β=2.5, 95%CI=0.3, 4.6) and 3 (β=2.8, 95%CI=0.3, 5.3) compared to baseline; larger improvements were seen by time 4 (β=10.6, 95%CI=7.9, 13.3) after completion of therapy. Children reported higher HRQL than their parent proxies (β=5.6, 95%CI=3.9, 7.2), males had higher HRQL than females (β=4.6, 95%CI=1.0, 8.3), and older age was associated with lower HRQL (β=-0.8, 95%CI=-1.5, -0.1). There was no significant effect of race or ethnicity.Conclusions: Completion rates of HRQL were high across all time periods and for both raters. HRQL, impaired at baseline, likely from the disease process, improved slightly during treatment, with larger improvements by the end of initial therapy. Future research will examine how clinical and treatment factors impact the HRQL trajectory. [Display omitted] DisclosuresParsons:Seattle Genetics: Research Funding. Rodday:Seattle Genetics: Research Funding. Henderson:Seattle Genetics: Research Funding.

Pain, skin sensations symptoms, and cognitive functioning predictors of health-related quality of life in pediatric patients with Neurofibromatosis Type 1.

The aim was to investigate pain, skin sensations symptoms and patient self-reported, and parent proxy-reported cognitive functioning as predictors of generic health-related quality of life (HRQOL) in pediatric patients with Neurofibromatosis Type 1 (NF1) from the perspectives of patients and parents. The Pain, Skin Itch Bother, Skin Sensations, and Cognitive Functioning Scales from the PedsQL™ Neurofibromatosis Type 1 Module and the PedsQL™ Generic Core Scales were completed in a multi-site national study by 323 patients and 335 parents. Patients were 5-25years of age. Pain and skin symptoms and cognitive functioning were tested for bivariate and multivariate linear associations with generic HRQOL. Pain, skin itch bother, skin sensations, and cognitive functioning were associated with decreased HRQOL in bivariate analyses (Ps < 0.001). In predictive analytics models, utilizing hierarchical multiple regression analyses controlling for demographic covariates, pain, skin itch bother, skin sensations, and cognitive functioning as a group accounted for 61 percent of the variance in patient-reported generic HRQOL (P < 0.001), reflecting a large effect size. For parent proxy-report, the predictor variables as a group accounted for 53% of the variance in generic HRQOL. Pain, skin symptoms, and patient self-reported and parent proxy-reported cognitive functioning are key predictors of generic HRQOL in pediatric patients with NF1. Delineating NF1-specific symptoms and cognitive functioning as high-priority predictors from the patient and parents perspective enhances a family-centered approach in clinical research, clinical trials, and clinical practice intended to improve the global generic HRQOL of pediatric patients with NF1.

Parental Distress and Quality of Life in Pediatric Inflammatory Bowel Disease: Implications for the Outpatient Clinic.

The interrelation between the course of inflammatory bowel disease (IBD) in children and parent's distress, and the subsequent impact this may have on health-related quality of life (HRQOL) of the child is unclear. Therefore, we investigated patient's HRQOL and parental distress and the association between the course of IBD, parental distress, and HRQOL of pediatric patients with IBD. Pediatric patients with IBD (8-18 years) and parents were invited. Patients completed the Pediatric Quality of Life Inventory, and parents the Distress Thermometer for Parents, simultaneously. Disease course was expressed as current clinical disease activity or months since last IBD flare. Patient's HRQOL and parental distress were compared to healthy controls. In total, 87 patients (71% response rate, 59% boys, median age 15.2 years) and parents were included. Patients had an impaired total HRQOL (β = 0.125, P = 0.010), driven by lower physical (0.196, P = 0.001) and school (β = 0.232, P < 0.001) functioning. Parents of children with IBD exhibited comparable levels of distress to parents of healthy children on the total problem and most subdomain problem scores (practical, social, emotional, physical, and cognitive), yet experienced more frequent parenting problems (P = 0.025). More severe disease course (months since last IBD flare) was indirectly associated, through parental distress, with decreased HRQOL of patients. Worse disease course is directly associated with increased distress of parents and indirectly with lower HRQOL of children and adolescents with IBD. Distress of parents may be considered in management of pediatric IBD to improve HRQOL of children.