Pediatric Health‐Related Quality of Life: Feasibility,Reliability and Validity of the PedsQL™ Transplant Module

The measurement properties of the newly developed Pediatric Quality of Life Inventory? (PedsQL?) 3.0 Transplant Module in pediatric solid organ transplant recipients were evaluated. Participants included pediatric recipients of liver, kidney, heart and small bowel transplantation who were cared for at seven medical centers across the United States and their parents. Three hundred and thirty‐eight parents of children ages 2–18 and 274 children ages 5–18 completed both the PedsQL? 4.0 Generic Core Scales and the Transplant Module. Findings suggest that child self‐report and parent proxy‐report scales on the Transplant Module demonstrated excellent reliability (total scale score for child self‐report α= 0.93; total scale score for parent proxy‐report α= 0.94). Transplant‐specific symptoms or problems were significantly correlated with lower generic HRQOL, supporting construct validity. Children with solid organ transplants and their parents reported statistically significant lower generic HRQOL than healthy children. Parent and child reports showed moderate to good agreement across the scales. In conclusion, the PedsQL? Transplant Module demonstrated excellent initial feasibility, reliability and construct validity in pediatric patients with solid organ transplants.     

Health-related quality-of-life in children with cystic fibrosis aged 5-years and associations with health outcomes

BackgroundThe impact of early cystic fibrosis (CF) on health-related quality-of-life (HRQOL) in preschool children is poorly characterised, and data on relationships between HRQOL and health outcomes in young children with CF are limited. We aimed to characterise and compare parent-proxy and child-reported HRQOL and evaluate relationships with clinical outcomes at age 5-years.MethodsSubjects were participating in the multi-centre Australasian Cystic Fibrosis Bronchoalveolar Lavage (ACFBAL) trial investigating BAL-directed versus standard CF therapy. Children aged 5-years and their parents rated HRQOL using the Pediatric Quality of Life Inventory (PedsQL™) and Cystic Fibrosis Questionnaire-Revised (CFQ-R) questionnaires.ResultsPedsQL and CFQ-R questionnaires were completed by 141 primary caregivers and 135 and 130 children, respectively. There were no differences in HRQOL between children randomised to BAL-directed versus standard CF therapy. Children with CF rated worse HRQOL than healthy children and there was poor parent-child concordance across HRQOL domains. Nutritional status, CF-CT scan score, forced expiratory volume in 1-second (FEV₁), and pulmonary exacerbations correlated with HRQOL at age 5-years. FEV₁ z-scores positively correlated with parent-proxy HRQOL in CFQ-R Respiratory (p = 0.018), Physical (<0.001), Emotional (p = 0.007) subscales and PedsQL Total-score (p = 0.021), Physical (p = 0.019) domains. Pulmonary exacerbation rates were inversely associated with parent-proxy CFQ-R Respiratory (p = 0.004), Physical (p = 0.022), PedsQL Total (p = 0.009) and Physical (p = 0.009) scores.ConclusionParent-reported HRQOL is a meaningful clinical endpoint to evaluate interventions in young children. Parent and child HRQOL reports provide different, complementary information. A preschool version of the CFQ-R is needed to assess relationships between HRQOL and clinical outcomes in young children.     

Health Status in Young Adults Two Decades After Pediatric Liver Transplantation

We conducted a cross‐sectional study of patients who underwent pediatric liver transplant (LT) between 1988 and 1992 to evaluate long‐term health status. Survivors completed socio‐demographic, medical and Health‐Related Quality of Life (HRQOL) surveys by mail including the SF‐36v2, PedsQL?4.0 Generic Core Scale, PedsQL? Cognitive Functioning Scale and PedsQL?3.0 Transplant Module. SF‐36 scores were converted to SF6D‐based utilities and risk factors for lower outcomes were assessed. Eighty‐five of 171 patients had survived. Fifty‐six were contacted with a response rate of 66%. Median age at LT was 0.86 years (IQR 0.58–3.0) and 64.3% had biliary atresia. Mean age at survey was 23.0 ± 4.4 years: 62% attended college, 68% lived with parents and 80% of those over 23 were employed. Patient health utilities were lower than norms (0.75 ± 0.12 vs. 0.82 ± 0.18, p < 0.01) and correlated with unemployment (p < 0.042), hospitalizations (p < 0.005) and lower education level (p < 0.016). Lower PedsQL?3.0 Transplant Module and PedsQL? 4.0 Generic Core Scale scores correlated with unemployment (p = 0.006, p = 0.009) and hospitalizations (p = 0.006, p = 0.02). Pediatric transplant recipients who survive to adulthood have lower physical HRQOL, measurable transplant‐related disability and lower health utility. Transplantation is life saving; however, physical and psychological sequelae continue to affect health status up to two decades later.     

Predictors of 12 month functional outcomes and length of stay of severely injured children in NSW,Australia. A longitudinal multi-centre study

BackgroundThe majority of paediatric injury outcomes studies focus on mortality rather than the impact on long-term quality of life, health care use and other health-related outcomes. This study sought to determine predictors of 12-month functional and psychosocial outcomes for children sustaining major injury in NSW.MethodsThe study included all children < 16 years requiring intensive care or an injury severity score (ISS) ≥ 9 treated in NSW at a paediatric trauma centre (PTC). Children were identified through the three PTCs and NSW Trauma Registry. The paediatric Quality of Life Inventory (PedsQL) and EuroQol five-dimensional EQ-5D-Y were used to measure HRQoL post-injury, completed via parent/carer proxy recruited through NSW PTCs.ResultsThere were 510 children treated at the three NSW PTCs during the 15-month study period. The mean (SD) age was 6.7 (6.0) years, with a median NISS (New Injury Severity Score) of 11 (IQR: 9–18). Regression analysis showed worse psychosocial health at twelve months was associated with hospital length of stay (LoS) and number of body regions injured (F₂,₆₅ = 5.85, p = 0.005). Physical outcome was associated with LoS and intensive care unit (ICU) admission (F_2,66 = 13.48, p < 0.001). Hospital LoS was significantly associated with NISS and head injury (F_2,398 = 51.5, p < 0.001).ConclusionHospital length of stay and polytrauma are independent factors that negatively influence psychological and physical outcomes of children with major injuries. Early intervention to enable emotional well-being, discharge home and long-term follow up such as dedicated family support and rehabilitation at home could reduce preventable poor outcomes.     

Health-related quality of life in children with Hirschsprung disease and children with functional constipation: Parent-child variability

BackgroundHealth-related quality of life (HRQOL) is an important outcome among children with Hirschsprung Disease (HD), but there are challenges in interpreting findings in previous studies owing to the choice of a comparator group and informant. We compared parent-proxy versus child self-report HRQOL in children with HD to children with functional constipation (FC) and examined predictors of HRQOL.MethodsData of 126 children (5–18 years, 60.3% male, HD: n = 52, FC: n = 74) were acquired from the Pediatric Colorectal and Pelvic Learning Consortium. Demographics, clinical variables, HRQOL (Pediatric Quality of Life Inventory parent-proxy; child self-report) and functional outcomes (Baylor Continence Scale, Cleveland Clinic Constipation Scoring System) were collected.ResultsParent and child HRQOL was similar for both cohorts, with higher scores on physical functioning and lower scores on emotional and school functioning. For children with HD, demographics and clinical variables did not predict HRQOL in multivariable regression models. For children with FC, greater severity of constipation predicted lower HRQOL (parent-proxy: B = -2.14, p < 0.001; child: B = -1.75, p = 0.001). Parent-child agreement on HRQOL scores was poor to moderate in the HD group (intraclass correlations (ICC)=0.38–0.74), but moderate to excellent in the FC group (ICC=0.63–0.84). Furthermore, parents of children with FC and ≤10 years overestimated children's HRQOL (proportional OR 4.59 (1.63, 13.85); p = 0.004).ConclusionClinical symptoms and demographic factors did not predict HRQOL among children with HD, highlighting the need to examine other biopsychosocial factors to understand long term HRQOL. Low parent-child HRQOL agreement in children with HD demonstrates the importance of obtaining parent and child perspectives.Level of evidenceIII.Type of studyPrognosis study.     

Extracorporeal membrane oxygenation in patients with hepatopulmonary syndrome undergoing liver transplantation: A systematic review of the literature

BackgroundIsolated cases of extracorporeal membrane oxygenation (ECMO) in patients with hepatopulmonary syndrome (HPS) undergoing liver transplantation (LT) have been reported with increasing frequency. We aimed to systemically review and synthesize the available literature on ECMO use in this population.MethodsA systematic literature review of the PubMed, Web of Science, Cochrane Library, and Embase databases (end-of-search date: November 14, 2021) was conducted in accordance with the PRISMA statement. Eligible studies presented clinical parameters and outcomes of adult or pediatric patients with HPS receiving ECMO support at the time of, or following, LT.ResultsSixteen studies from 4 continents reporting on 17 patients who were initiated on ECMO prior to (n = 2), during (n = 1) or after LT (n = 14) were included. Nine of the 16 studies were published between 2019 and 2021. The median pre-LT PaO₂ was 38.0 mmHg (IQR 35.0–52.0). The median time from LT to ECMO initiation was 7 days (IQR, 3–12). Six patients (50%, n = 6 of 12) were extubated post-LT, before deterioration, development of refractory hypoxemia, and initiation of ECMO. Most patients were cannulated with a venovenous configuration (75%, n = 12 of 16). Most patients cannulated with a venoarterial or veno-arterial-venous strategy (75%, n = 3 of 4) had concurrent hemodynamic instability. The median total time on ECMO was 13 days (IQR 10–29). Using linear regression, for patients cannulated postoperatively, each day between LT and ECMO initiation was associated with a 3.5-day increase in total ECMO duration (95%CI: 2.23–4.73, p < 0.001, R² = 73.7%). The median postoperative intensive care unit length of stay was 40 days (IQR, 37–61) and hospital length of stay was 59.5 days (IQR 42–77). 82.4% of patients (14 of 17) survived to discharge.ConclusionsECMO is feasible in patients with HPS undergoing LT and appears to be associated with better outcomes compared to other causes of cardiopulmonary failure in LT patients. As the volume of experience grows, ECMO may become a central part of perioperative support in LT patients with severe HPS.     

Association between Kasai portoenterostomy at low caseload centres and transplant complications in children with biliary atresia

BackgroundKasai portoenterostomy (KPE) is the preferred treatment for biliary atresia (BA) patients. It has been shown that the center caseload of KPE impacts on native liver survival. We aimed to define the impact of KPE caseload on complications at the time of liver transplantation (LT).MethodsRetrospective data collection of LT for BA performed in our tertiary center between 2010 and 2018. The patients were grouped according to the caseload of the center that performed KPE: Group A (≥5 KPE/year) and Group B (<5 KPE/year). We analyzed total transplant time (TTT), hepatectomy time, amount of plasma and red blood cell (RBC) transfusions, occurrence of bowel perforations at LT.ResultsAmong 115 patients, Group A (n 44) and Group B (n 71) were comparable for age, sex, PELD score, TTT. The groups differed for: median hepatectomy time (57 min, IQR = 50–67; vs 65, IQR 55–89, p = 0.045); RBC transfusions (95 ml, IQR 0–250; vs 200 ml, IQR 70–500, p = 0.017); bowel perforations (0/44 vs 15/71, p = 0.001). One-year graft loss in Group A vs Group B was 1/44 vs 7/71 (p = 0.239), whereas deaths were 0/44 vs 5/71 respectively (p = 0.183); 5/15 patients who had a perforation eventually lost the graft.ConclusionsThis study found an association between KPE performed in low caseload center and the incidence of complications at LT. These patients tend to have a worse outcome. The centralization of KPE to referral center represents an advantage at the time of LT.Mini abstractWe studied the impact of Kasai portoenterostomy (KPE) caseload on complications at the time of liver transplantation (LT), in 115 patients. We found an association between KPE performed in low caseload center and increased bowel perforations and blood transfusions. We suggest to centralize to experienced center all children requiring KPE.     

Pediatric end stage renal disease health-related quality of life differs by modality: a PedsQL ESRD analysis

We previously validated the 34-item PedsQL 3.0 End Stage Renal Disease (ESRD) Module designed to measure pediatric ESRD-specific health-related quality of life (HRQOL) in children and adolescents receiving maintenance dialysis or with a renal transplant. The study reported here was undertaken to assess for potential HRQOL differences between ESRD modality in children with ESRD and their parents using the PedsQL 3.0 ESRD Module. Parents of patients with a renal transplant reported a significantly higher HRQOL for their children than parents of pediatric patients receiving dialysis on all ESRD Module Scales except the Perceived Physical Appearance Scale, with the majority of the effect sizes in the medium range. Pediatric renal transplant patients self-reported comparable HRQOL to pediatric patients receiving dialysis across the ESRD Module Scales, with the exception of the Family and Peer Interaction Scale, in which pediatric renal transplant patients self-reported significantly higher HRQOL than pediatric patients receiving dialysis. Our cross-sectional data suggest that parents of children with ESRD observe a positive impact from renal transplantation on the majority of HRQOL domains compared to dialysis, whereas children self-report generally non-significant small effect size differences in favor of renal transplantation. These findings suggest that the PedsQL ESRD 3.0 Module may be used to identify ESRD- and modality-specific challenges that impact pediatric patient HRQOL. Electronic supplementary material  The online version of this article (doi:) contains supplementary material, which is available to authorized users.     

Quality of Life and Psychological Well-Being in Children and Adolescents After Renal Transplantation

BackgroundKidney transplantation is the predominant treatment option in patients with end-stage renal disease. The aim of this study was to evaluate the effect of the presence of a psychiatric disorder on the quality of life in children and adolescents after kidney transplantation.MethodsA total of 43 patients aged 6 to 18 years were included in the study. All participants and their parents were asked to complete the Pediatric Quality of Life Inventory (PedsQL), and families only, the Strengths and Challenges Questionnaire. Psychiatric symptoms and disorders of the patients were evaluated according to the Schedule for Mood Disorders and Schizophrenia for School-Age Children/Now and Lifetime Turkish Version. Patients were divided into 2 groups according to psychiatric symptoms and disorders.ResultsThe most common psychiatric disorder was attention deficit and hyperactivity disorder (26%). The questionnaires filled out by the patients revealed a lower Total PedsQL Score (P = .003), PedsQL Physical Functionality Score (P = .019), and PedsQL Social Functioning Score (P = .016) in patients with psychiatric disorders. When the parents filled out the questionnaires, the Total PedsQL Score was similar in both groups. The PedsQL Emotional Functionality Score (P = .001) and PedsQL School Functionality Score (P = .004) were significantly lower in patients with psychiatric disorders. The Strengths and Difficulties Questionnaire revealed significantly higher total (P = .014) and hyperactivity/inattention subscale scores (P = .001) in those with a psychiatric disorder.ConclusionsPsychiatric disorders in kidney transplant patients adversely affect the quality of life.     

Child self-reported quality of life in pediatric intestinal failure

BackgroundRecent studies have focused on parent-reported health-related quality of life (HRQOL) in children with intestinal failure (IF). However, there is a paucity of data on HRQOL from the perspective of the child with IF.MethodsA prospective study of child self-reported HRQOL was performed in a regional intestinal rehabilitation program from 2015 to 2019. The PedsQL 4.0 Generic Core Scales were administered annually to children with IF ages five years and older along with their parents. Survey data was stratified by age and compared with parent-proxy scores and reference populations of healthy and chronically ill children. Linear mixed-effect models were constructed to identify associations with child self-reported HRQOL.ResultsA total of 140 surveys were administered to 69 children and their parents. Median child age at survey was 8 (IQR 6–10) years. Child self-reported HRQOL scores increased with each increasing age range. Children reported higher HRQOL scores compared to parent-proxy data in all age groups. Children with IF had lower HRQOL scores compared to healthy children in all survey dimensions (p < 0.001) and to children with chronic illness in the school and social functioning dimensions (p < 0.05). In adjusted analysis, longer remnant bowel length was independently associated with decreased HRQOL scores in children (p < 0.05).ConclusionsChildren with IF reported better HRQOL compared to parent-proxy data. While these HRQOL scores improved with age, they remain significantly lower than healthy and chronically ill peers. The association between bowel length and child-reported HRQOL deserves further investigation.Level of evidenceLevel II     

Long-term surgical and patient-reported outcomes of Hirschsprung Disease

BackgroundInformation is needed regarding the complex relationships between long-term functional outcomes and health-related quality of life (HRQoL) in Hirschsprung's Disease (HSCR). We describe long-term outcomes across multiple domains, completing a core outcome set through to adulthood.MethodsHSCR patients operated at a single center over a 35-year period (1978–2013) were studied. Patients completed detailed questionnaires on bowel and urologic function, and HRQOL. Patients with learning disability (LD) were excluded. Outcomes were compared to normative data. Data are reported as median [IQR] or mean (SD).Results186 patients (median age 28 [18–32] years; 135 males) completed surveys. Bowel function was reduced (BFS 17 [14–19] vs. 19 [19–20], p < 0•0001;$\eta$² = 0•22). Prevalence and severity of fecal soiling and fecal awareness improved with age (p < 0•05 for both). Urinary incontinence was more frequent than controls, most of all in 13–26y females (65% vs. 31%,p = 0•003). In adults, this correlated independently with constipation symptoms (OR 3.18 [1.4–7.5],p = 0.008). HRQoL outcomes strongly correlated with functional outcome: 42% of children demonstrated clinically significant reductions in overall PedsQL score, and poor bowel outcome was strongly associated with impaired QOL (B = 22•7 [12•7–32•7],p < 0•001). In adults, GIQLI scores were more often impacted in patients with extended segment disease. SF-36 scores were reduced relative to population level data in most domains, with large effect sizes noted for females in General Health (g = 1.19) and Social Wellbeing (g = 0.8).ConclusionFunctional impairment is common after pull-through, but bowel function improves with age. Clustering of poor functional outcomes across multiple domains identifies a need for early recognition and long-term support for these patients.     

A cross-cultural adaptation of the Upper Limb Functional Index in French Canadian

Study designClinical measurement.IntroductionThe Upper Limb Functional Index (ULFI) is a self-report questionnaire assessing activity limitations/participation restrictions resulting from an upper limb musculoskeletal disorder (UL-MSD). It is suitable for use in a rehabilitation context where clinicians have important time constraints due to a heavy caseload. However, no French version was available until now.Purpose/methodsTo perform a cross-cultural adaptation of the ULFI in French Canadian and examine the psychometric properties and clinical applicability of the adapted version (ULFI-FC) among 50 bilingual patients.ResultsThe ULFI-FC showed high internal consistency (Cronbach α = 0.93), good convergent validity with the original ULFI (r = 0.85) and with the French Canadian version of the Disabilities of the Arm, Shoulder and Hand (r = −0.85) and good applicability.ConclusionThis study supports the suitability of the ULFI-FC for use in a busy rehabilitation setting for French-speaking patients with UL-MSD.Level of evidenceN/A.     

Behavior Problems and Cognitive Function in Pediatric Liver Transplant Recipients

BackgroundLiver transplant (LT) is a lifesaving treatment providing excellent clinical outcomes. However, data regarding behavioral and cognitive issues after LT are sparse in Asia. This study aimed to investigate behavior and cognitive problems among pediatric LT recipients.MethodsWe used the Child Behavior Checklist (CBCL) to evaluate behavior problems and/or the Wechsler Intelligence Scale for Children, Third Edition (WISC-III) to assess cognitive function. Participants were children aged 2 to 18 years who were treated with LT for at least 2 years.ResultsWe included 77 children with a median age of 7.8 years (interquartile range, 5.0-10.9). All children were evaluated with the CBCL, and 26 children were available for WISC-III assessment. Approximately one-third (34%) of the children had a total CBCL score above a clinical cutoff for significant behavior problems, and 29% of school-aged children had abnormal competence scores in a clinically significant range. Most of the evaluated children (17 of 26; 65%) had a normal full-scale intelligence quotient. Lower cognitive function was associated with having a single parent (P < .01). Higher behavior problems were associated with lower parental education level (P = .01) and correlated with longer post-transplant duration (Spearman’s rho = 0.443; P < .001).ConclusionsSignificant numbers of children have behavior problems after LT. Most children have normal cognitive function, although a larger sample size is required to confirm this result. Long-term support for cognitive and behavior problems after LT should be implemented, particularly in children with single parents and lower parental education level.     

Effect of Pre-Transplant Covid-19 Exposure on Post-Liver Transplant Clinical Outcomes

BackgroundCOVID-19 has led to an unprecedented global health crisis. This situation caused an immediate reduction in solid organ transplantation activity. This study aimed to present the follow-up results of patients with chronic liver disease who underwent liver transplantation (LT) after a history of COVID-19 infection.MethodsSociodemographic characteristics and clinicopathological data of 474 patients who underwent LT at Inonu University Liver Transplant Institute between March 11, 2020 and March 17, 2022 were prospectively recorded and analyzed retrospectively. Among these, the data of 35 patients with chronic liver disease who were found to be exposed to COVID-19 infection in the pre-LT period were analyzed for this study.ResultsThe median body mass index, Child score, and Model for end-stage liver disease/ Pediatric end-stage liver disease scores of the 35 patients were calculated as 25.1 kg/m² (IQR: 7.4), 9 points (IQR: 4), and 16 points (IQR: 10), respectively. Graft rejection occurred in 4 patients at a median of 25 days post-transplant. Five patients underwent retransplantation at a median of 25 days post-transplant. The most common cause of retransplantation is early hepatic artery thrombosis. There were 5 deaths during postoperative follow-up. Mortality developed in 5 (14.3%) patients exposed to COVID-19 infection in the pretransplant period, whereas mortality occurred in 56 (12.8%) patients not exposed to COVID-19 infection. There was no statistically significant difference in mortality between the groups (P = .79).ConclusionsThe results of this study showed that exposure to COVID-19 before LT does not affect post-transplant patients and graft survival.     

Quality of life in paediatric burn patients with non-severe burns

BackgroundBurns are common worldwide, and the vast majority are non-severe burns of less than 20% of the total body surface area (TBSA). In Australia, paediatric burns account for a third of all burn admissions, thus understanding the quality-of-life outcomes after a non-severe burn in children is important.MethodsThis retrospective cohort study describes a paediatric cohort from Western Australia with non-severe burns occurring between 2018 and 2020 and characterises the child’s quality-of-life outcomes which is measured using the Paediatric quality of life survey (PedsQL). The PedsQL included a parent-report and child-report assessment, each with a physical function domain and a psychosocial function domain which comprised of an emotional, a social and a school category.ResultsData collected from 249 patients; 50.6% were male, 45.6% were toddlers. The most common cause was scald (48.19%), the majority had burns smaller than 5% TBSA (91.97%), and most included visible areas such as head, neck or hands (77.51%). The parent-report PedsQL scores were significantly different for both physical and psychosocial domains between the different age groups (p = 0.002, p = 0.001, respectively) and for burn cause (p = 0.004, p = 0.005, respectively). For child-reported scores we found evidence of an effect of burn cause across both domains that did not reach a statistical significance (p = 0.076, p = 0.078, respectively). The psychosocial functions in both the parent-report and the self-report were significantly different for the socioeconomic status groups (p = 0.015, p = 0.032, respectively). Quality of life scores were critically low in 16.46% of paediatric burn patients at three months after burn.ConclusionParent-reported and child-reported psychosocial function was significantly poorer in higher socioeconomic groups, for older children and for those with flame burns. About 16% of patients had scores below the critical cut off. These data provide insight into the quality-of-life outcomes of paediatric patients with non-severe burns, allowing future studies to investigate burn prevention strategies and services to help paediatric burn patients in their recovery.     

A comparison of parent-reported and self-reported psychosocial function scores of the PedsQL for children with non-severe burn

BackgroundQuality of life of paediatric patients after burn injury is often assessed through parents who may score differently to their child. Non-severe burns are the most common type of burn injury in Western Australia, however, despite low severity and high survival rates, they can cause long term physical and psychosocial problems which need to be detected early in order to provide patients with optimal holistic care.MethodsDemographic and clinical data were collected from paediatric patients (5–16-year-old) with non-severe burns (<20% total body surface area), and Paediatric quality of life (PedsQL) questionnaires were collected from both the patient and their parent. Two cohorts of patients were assessed: first, those at approximately six months after burn, and second, those more than one-year after burn. Differences between parent-scores and self-scores were analysed using multivariate linear regression to assess the relationship between risk factors and observed differences in PedsQL scores.ResultsParents reported poorer Psychosocial Function (PSF) for younger children (p = 0.01) and for patients from higher socioeconomic status areas (p = 0.05) compared to their children. In the ‘Early Recovery Cohort’, female patients had significantly different scores to their parents (p < 0.01). In the ‘Late Recovery Cohort’, parents rated older patients lower than they rated themselves (p = 0.03).ConclusionAge at burn, socioeconomic status, and female gender may increase the discrepancy in quality-of-life assessments between parents and patients.     

Health-related quality of life in pediatric patients with ESRD

As part of creating a pediatric ESRD-specific Health-Related Quality of Life (HRQOL) assessment instrument, we established pilot data with the PedsQL 4.0 Generic Core Scales in 96 pediatric patients with ESRD receiving hemodialysis (HD), peritoneal dialysis (PD) or with a renal transplant (TX). Patient age ranges were: 2–4 (8; PD n=3, TX n=5), 5–7 (12; HD n=9, TX n=3), 8–12 (25; HD n=5, PD n=6, TX n=14), 13–18 (51; HD n=18, PD n=10, TX n=23). PedsQL ESRD data were compared to healthy children (n=131 child report; n=145 parent report) and across HD/PD/TX. For all domains, ESRD patient HRQOL scores were significantly lower than healthy controls. Transplant patients reported better physical and psychosocial health than dialysis patients. No difference was noted between HD and PD patients for any PedsQL domain. Our data demonstrate that the PedsQL 4.0 Generic Core Scales is a useful measurement instrument to screen for HRQOL impairment in pediatric patients with ESRD. We suggest that the ESRD-specific HRQOL instrument in development is needed to help define condition-specific HRQOL differences between modalities.     

Health-related quality of life after conservatively and surgically-treated paediatric proximal humeral fractures

PurposeThe health-related quality of life (HRQoL) after conservatively versus surgically treated paediatric proximal humeral fractures is poorly understood. We assessed the HRQoL after this injury and asked if HRQoL was associated with age, radiological classification or treatment chosen.MethodsWe identified 228 patients who were treated for proximal humeral fractures between 2004 and 2017. These patients completed the Quick Disabilities of the Arm, Shoulder and Hand (Quick-DASH) (primary outcome), the Paediatric Quality of Life Inventory (PedsQL) and questions regarding patient satisfaction. Fractures were classified radiologically following the Paediatric Comprehensive AO Classification.ResultsWe were able to follow-up on 190 children; 147 (mean age 8.7 years (0.8 to 15.7)) sustained a metaphyseal and 43 (mean age 11.6 years (3.7 to 15.8)) sustained a Salter Harris type I or II injury. Most fractures (90%) were simple, 10% were multifragmentary. In total, 137 children (72%) were treated nonoperatively, 51 (27%) were treated by elastic stable intramedullary nailing (ESIN). After a median follow-up of 7.6 years (0.8 to 14.3) there was an overall mean Quick-DASH of 4.3 (SD 9.3) for girls and 1.2 (SD 3.1) for boys. The mean function score of the PedsQL was 94.7 (SD 11.1) for girls and 98.0 (SD 6.0) for boys. The mean psychosocial score of the PedsQL was 92.0 (SD 11.1) for girls and 94.1 (SD 11.6) for boys. Most children (79%) were very satisfied with the cosmetic result and 74% were very satisfied with the treatment overall. Surgery and female sex were associated with lower satisfaction.ConclusionIn this cohort of 190 patients, where immobilization for mildly displaced fractures, and closed reduction and ESIN was used for displaced fractures, there was equally excellent mid- and long-term HRQoL when assessed by the Quick-Dash and the PedsQL.Level of EvidenceTherapeutic, Level IV     

One-year impact of a bowel management program in treating fecal incontinence in patients with anorectal malformations

BackgroundMany patients with anorectal malformations (ARM) need a bowel management program (BMP) to manage lifelong problems of fecal incontinence or severe constipation. We aimed to evaluate the sustainability of the results in such a program.MethodsA single-institution retrospective review was performed in children with ARM who attended our BMP (2015–2019). Standardized definitions and validated tools were used to assess fecal continence (Baylor Continence Scale), constipation (Cleveland Constipation Scoring System), urinary symptoms (Vancouver Symptoms Score), and the Pediatric Quality of Life (PedsQL) and health-related quality of life (HRQOL) at the start of BMP and 1-year after completion of the program.Results222 patients with ARM at a median age of 6.7 (IQR, 4.9–10.1) years were identified. All (100%) soiled at intake with 149 (67.1%) patients being treated with rectal or antegrade enemas and 73 (32.9%) with oral laxatives. At 1 year 150 (70.4%) were clean, 72.7% were on enemas and 27.3% were on laxatives (p = 0.08). 109 out of 148 (73.6%) patients were clean on enemas. A further 41 out of 66 (62.1%) patients were continent on laxatives with voluntary bowel movements and clean. In the group that was clean, there was improvement in Baylor Continence Scale (25 vs. 13.0, p < 0.000000002), Vancouver (11 vs. 6, p = 0.0110) scores, and clinically relevant improvement in the total PedsQL HRQL (78–85) and the PedsQL HRQL physical function (86–92) and psychosocial domain (77–82). There was no improvement in Cleveland (10 vs. 9, p = 0.31) score.ConclusionAn intensive BMP offers significant benefits in the treatment of fecal incontinence in ARM. It appears to also improve urinary incontinence and urinary voiding as well as the patient's quality of life. These changes are sustainable over at least one year.     

Most children experience resolution of idiopathic pediatric rectal prolapse with bowel management alone

BackgroundRecent studies in children with idiopathic rectal prolapse report up to 48% require surgical intervention to manage refractory disease. We sought to examine outcomes of our non-surgical approach to managing rectal prolapse using a bowel management program.MethodsA retrospective review was performed for all children with the diagnosis of rectal prolapse between 2011 and 2020. Children with a rectal polyp or hemorrhoid were excluded.Results47 children with rectal prolapse were identified (median age at diagnosis of 4 years (IQR 3,7.75); age ≤ 4 years n = 30; age > 4 years n = 17). Associated diagnoses included constipation (n = 45, 96%) and psychiatric diagnoses (n = 7, 14%). Children underwent a bowel management program including stimulant laxatives in 44 (94%) and osmotic laxatives in 2 (4%). Median follow-up time was 181 days (IQR 77, 238). Median time to resolution of rectal prolapse was 9 months (IQR 4, 13) with a maximum time to resolution of 31 months. We compared children ≤ 4 years old (Group A) to those > 4 years old (Group B). Psychiatric diagnoses were less common in Group A (3.5 vs. 38.9%, p = 0.003). Median time to spontaneous resolution was 6.5 months (IQR 3.5, 9.5) in Group A versus 13.5 (IQR 4, 16) months in Group B, p = 0.13. No differences in surgical intervention were identified. Three (6.4%) patients required surgery for prolapse.ConclusionsA bowel management program is an effective treatment for most children with rectal prolapse. This data suggests that surgical intervention is unnecessary in most children.Level of evidenceIII.