Linguistic, Content and Face Validity of the Swedish Version of a Quality-of-Life Assessment for Children, Teenagers and Adults with Spina Bifida.

Spina bifida includes a spectrum of different neural tube defects. Myelomeningocele is the most serious type and is associated with a risk of paralysis and sensory dysfunction below the affected level, bladder/bowel dysfunction, brain dysmorphology, and impaired health-related quality of life (HRQoL). The aim of this study was to describe the establishment of linguistic, content and face validity of the Swedish version of a Quality-of-Life Assessment for children (QUALAS-C, n = 10 items), teenagers (QUALAS-T, n = 10 items) and adults with spina bifida (QUALAS-A, n = 15 items) based on the original US English versions. The process included close collaboration with the original instrument developer and complied with international standards on patient-reported outcome measurements. The procedure includes forward translation, expert and patient/parent review and reconciliation, back translation, back translation review and cognitive debriefing interviews with 16 people with spina bifida aged 8 to 33, providing them with the possibility of evaluating the clarity, adequacy, and comprehensiveness of QUALAS-C, QUALAS-T and QUALAS-A, respectively. The interviews lasted a median of 15 min (range 8-16) for QUALAS-C, 10 min (range 9-15) for QUALAS-T and 24 min (range 9-38) for QUALAS-A. Four main issues/topics needed attention and discussion after both the forward and back translation. Following the back translation review, all issues were resolved. The patient feedback revealed recognition of the HRQoL issues included in QUALAS, and also difficulties in understanding some questions. After the patients' evaluation, four items were reworded for clarity. No study participant reported a wish to add to or remove questions from QUALAS. Hence, the Swedish versions of QUALAS became conceptually equivalent to the original US English versions and achieved linguistic, content and face validity. While empowering the voices of people with spina bifida, these results also enable their HRQoL to be properly assessed in research and clinical care in Sweden and in international studies.

Mortality rates, cause and risk factors in people with spina bifida, register-based study over five decades.

AIM: Care for people with spina bifida can be improved. This may be done by evaluating mortality rates and causes of death. METHODS: Between 1973 and 2021, 1735 people with spina bifida appeared in registers of the Swedish population. Survival rates and causes of death were calculated according to age and decade. RESULTS: Over almost 50 years, the prevalence of spina bifida decreased from 5.2 to 1.2 per 10 000 births. Mortality fell sharply during the first year of life, with survival rising from 75% to 94%. For children aged 2-18 years and adults, mortality rates were low and differences between decades were minimal. Causes of childhood deaths were congenital abnormalities, hydrocephalus and infections, the latter two also in adults. Adult causes also included self-inflicted injuries and substance abuse, with suicidal or unclear intent, both more common than in the general population. Bladder malignancies were also more frequent, although after reconstructive bladder surgery, mortality rates were similar. CONCLUSION: Survival in the first year of life increased in children with spina bifida, whereas there was no difference in survival rates between adults born between 1973 and 1999. For adults, proactive prevention methods regarding self-inflicted injury, substance abuse and bladder cancer are warranted.

The association of feeding difficulties and generic health-related quality of life among children born with esophageal atresia.

BACKGROUND: Children born with esophageal atresia experience feeding difficulties. This study investigates the association of feeding difficulties and generic health-related quality of life among children aged 2-7 and 8-17 years, born with esophageal atresia. METHODS: 108 families (n = 36 aged 2-7 years; n = 72 aged 8-17) answered a survey regarding difficulties in their child's mealtimes and a validated generic health-related quality of life instrument(PedsQL 4.0). Clinical data was collected from hospital records. The association of feeding difficulties and health-related quality of life was analysed trough Mann-Whitney U-test. Linear regression determined whether the number of concurrent feeding difficulties in the child decreased the health-related quality of life scores. P < 0.05 was considered significant. RESULTS: In children aged 2-7 and 8-17 years, to have a gastrostomy, to use a food infusion pump, need for energy-enriched food and eating small portions were respectively significantly associated with lower total health-related quality of life scores in the parent-reports (p < 0.05). Most of the feeding difficulties had a negative significant relationship with the domains of physical and social functioning. Additionally, in the older age group, long mealtimes and adult mealtime supervision were associated with lower scores in both child and parent reports. In both age groups, an increased number of feeding difficulties in the child decreased the total generic health-related quality of life scores (p < 0.01). CONCLUSION: Specific feeding difficulties are associated with low health-related quality of life among children with esophageal atresia. An increasing number of feeding difficulties is associated to decreasing health-related quality of life-scores. Further research is needed to understand these associations.

Transabdominal ultrasound of rectal diameter in healthy infants: a prospective cohort study during the first year of life.

AIM: Transabdominal rectal ultrasound (TRU) is used to measure transverse rectal diameter (TRD) in order to diagnose functional constipation (FC) and megarectum, and to evaluate treatment. The proposed cut-off value is 3.0 cm. Currently, no standardised values exist for children below the age of 4. We used repeated TRUs to establish reference TRD values in healthy infants and to describe rectal diameter in infants with FC. METHODS: This prospective observational cohort study enrolled healthy term babies from a maternity department. TRD measurements were taken at 2 and 12 months of age, and questionnaires completed in interviews helped diagnose FC according to Rome III criteria. RESULTS: Two hundred TRUs were performed on 110 infants (62 males). In infants without FC anytime, the mean TRD at 2 months was 1.56 (SD 0.32) cm and at 12 months 1.78 (0.47) cm, while the 95th percentiles were 2.26 and 2.64 cm, respectively. In 77 infants with two TRUs, the mean increase was 0.21 cm (95% confidence interval: 0.099-0.318). Thirteen infants were diagnosed with FC during the study period. At 2 and 12 months of age, there was no difference in TRD between infants with and without FC. CONCLUSION: TRD increased from 2 to 12 months. We suggest 2.3 cm as an upper limit for normal TRD at 2 months and 2.6 cm at 12 months. Infants diagnosed with FC did not have a greater TRD than infants without, either before or after treatment. Further studies are needed to evaluate the usefulness of TRU in infants with FC or megarectum.

Prevalence of Mental Health Problems, Associated Factors, and Health-Related Quality of Life in Children with Long-Gap Esophageal Atresia in Sweden.

BACKGROUND: Children with long-gap esophageal atresia (LGEA) face a high risk of digestive and respiratory morbidity, but their mental health outcomes have not been investigated. We aimed to identify the prevalence of mental health problems in children with LGEA, associated factors and health-related quality of life (HRQOL). METHODS: Twenty-six children with LGEA aged 3-17 were recruited nationwide in Sweden. One of their parents and adolescents aged 11-17 completed information on the child's mental health (Strength and Difficulties Questionnaire), generic (PedsQL 4.0) and condition-specific HRQOL (EA-QOL). Parents gave information on current child symptomatology. Mental health level was determined using validated norms; abnormal≥90 percentile/borderline≥80 percentile/normal. Elevated levels were considered borderline/abnormal. Data were analyzed using descriptives, correlation and Mann-Whitney-U test. Significance level was p < 0.05. RESULTS: Twelve children with LGEA aged 3-17 (46%) had elevated scores of ≥1 mental health domain in parent-reports, whereas 2 adolescents (15%) in self-reports. In parent-reports, 31% of the children had elevated levels of peer relationship problems, with associated factors being child sex male (p = 0.037), airway infections (p = 0.002) and disturbed night sleep (p = 0.025). Similarly, 31% showed elevated levels of hyperactivity/inattention, and associated factors were male sex (p = 0.005), asthma (p = 0.028) and disturbed night sleep (p = 0.036). Elevated levels of emotional symptoms, seen in 20%, were related to swallowing difficulties (p = 0.038) and vomiting problems (p = 0.045). Mental health problems correlated negatively with many HRQOL domains (p < 0.05). CONCLUSIONS: Children with LGEA risk mental health difficulties according to parent-reports, especially peer relationship problems and hyperactivity/inattention, with main risk factors being male sex, airway problems and sleep disturbances. This should be considered in follow-up care and research, particularly since their mental health problems may impair HRQOL. LEVELS OF EVIDENCE: Prognosis study, LEVEL II.

Postoperative morbidity and health-related quality of life in children with delayed reconstruction of esophageal atresia: a nationwide Swedish study.

BACKGROUND: In 10-15% of children with esophageal atresia (EA) delayed reconstruction of esophageal atresia (DREA) is necessary due to long-gap EA and/or prematurity/low birth weight. They represent a patient subgroup with high risk of complications. We aimed to evaluate postoperative morbidity and health-related quality of life (HRQOL) in a Swedish national cohort of children with DREA. METHODS: Postoperative morbidity, age-specific generic HRQOL (PedsQL™ 4.0) and condition-specific HRQOL (The EA-QOL questionnaires) in children with DREA were compared with children with EA who had primary anastomosis (PA). Factors associated with the DREA group's HRQOL scores were analyzed using Mann-Whitney U-test and Spearman's rho. Clinical data was extracted from the medical records. Significance level was p < 0.05. RESULTS: Thirty-four out of 45 families of children with DREA were included and 30 returned the questionnaires(n = 8 children aged 2-7 years; n = 22 children aged 8-18 years). Compared to children with PA(42 children aged 2-7 years; 64 children aged 8-18 years), there were no significant differences in most early postoperative complications. At follow-up, symptom prevalence in children aged 2-7 with DREA ranged from 37.5% (heartburn) to 75% (cough). Further digestive and respiratory symptoms were present in ≥ 50%. In children aged 8-18, it ranged from 14.3% (vomiting) to 40.9% (cough), with other digestive and airway symptoms present in 19.0-27.3%. Except for chest tightness (2-7 years), there were no significant differences in symptom prevalence between children with DREA and PA, nor between their generic or condition-specific HRQOL scores (p > 0.05). More children with DREA underwent esophageal dilatations (both age groups), gastrostomy feeding (2-7 years), and antireflux treatment (8-18 years), p < 0.05. Days to hospital discharge after EA repair and a number of associated anomalies showed a strong negative correlation with HRQOL scores (2-7 years). Presence of cough, airway infection, swallowing difficulties and heartburn were associated with lower HRQOL scores (8-18 years), p < 0.05. CONCLUSIONS: Although children with DREA need more treatments, they are not a risk group for postoperative morbidity and impaired HRQOL compared with children with PA. However, those with a long initial hospital stay, several associated anomalies and digestive or respiratory symptoms risk worse HRQOL. This is important information for clinical practice, families and patient stakeholders.

Factors of family impact in a Swedish-German cohort of children born with esophageal atresia.

BACKGROUND: After repair of esophageal atresia (EA), childhood survivors commonly present with digestive and respiratory morbidity, and around 55% have associated anomalies. Although it is known that these problems can reduce health-related quality of life in children with EA, less is understood about the impact on the family. We aimed to identify factors related to family impact in children with EA. METHODS: One parent each of a child with EA (2-18 years) in 180 families from Sweden and Germany answered the PedsQL™ Family Impact Module as the dependent variable. The independent variables were the child's parent-reported health-related quality of life as measured by PedsQL™ 4.0, current symptoms, school situation, and parent/family characteristics together with child clinical data from the medical records. RESULTS: Stepwise multivariable regression analysis showed a multifactorial model of the total family impact scores (R2 = 0.60), with independent factors being the child's overall generic health-related quality of life, school-absence ≥ 1/month, severe tracheomalacia, a family receiving carer's allowance, and a parent with no university/college education, p < 0.05. Logistic regression analysis showed that an increased number of symptoms in the child the preceding 4 weeks lowered the family impact scores; however, the child's feeding (R2 = 0.35) and digestive symptoms (R2 = 0.25) explained more in the variation of scores than the child's respiratory symptoms (R2 = 0.09), p < 0.0001. CONCLUSIONS: Family functioning may be a contributing factor to the maintenance of child health. The study findings suggest multifactorial explanations to family impact in children with EA, which are essential when optimizing the support to these families in clinical and psychosocial practice. Future research should explore experiences of family impact from all family members' perspectives and multicenter studies are warranted to understand better the effectiveness of psychosocial-educational interventions to families of children with EA.

Linguistic and content validity of the Swedish version of the PedsQL™ gastrointestinal symptoms scales and symptoms module for paediatric patients.

AIM: To describe the process of linguistic and content validity of the Swedish version of the PedsQL™ Gastrointestinal Symptoms Scales and Symptoms Module, measuring health-related quality of life (HRQOL) in children with gastrointestinal (GI) disorders. METHODS: The establishment of linguistic and content validity was carried out in compliance with international standards on patient-reported outcome measurements. The process included forward translation, expert review and reconciliation, backward translation, backward translation review and interviews with 15 children aged 5-18 years with GI tract symptoms and 20 parents of children with GI tract symptoms aged 2-18 years. RESULTS: The Swedish version of the PedsQL™ Gastrointestinal Symptoms Scales and Symptoms Module (child report 5-18 years, parent report for children 2-18 years) was achieved without major difficulties. Eight issues needed discussion after forward translation, and there was one change after backward translation and three revisions following patient and parent testing. CONCLUSION: A conceptually equivalent Swedish language version of PedsQL™ Gastrointestinal Symptoms Scale and Symptoms Module for children aged 2-18 years old was developed. This enables improved HRQOL evaluations in children with GI disorders in Sweden. Future research using a larger sample is recommended to evaluate validity and reliability of the Swedish language version of the module.

Parent-child-agreement on health-related quality of life and its determinants in patients born with Esophageal Atresia: a Swedish-German cross-sectional study.

BACKGROUND: The aim was to compare parent and child-reported health-related quality of life (HRQOL) of children born with esophageal atresia (EA) and determine factors that affect the level of parent-child agreement. METHODS: We included 63 parent-child dyads of children born with EA aged 8-18 from Germany and Sweden. The generic PedsQL 4.0™ questionnaire and the condition-specific EA QOL questionnaire were used to assess children's HRQOL from parents' and children's perspectives. The PedsQL™ Family Impact Module was used to assess parental HRQOL and Family Functioning. RESULTS: On an individual level, intra-class correlation coefficients indicated strong levels of parent-child agreement (.61-.97). At the group level, the analyses showed no significant differences between the responses of parents and children. When a disagreement occurred, parents were more likely to rate generic HRQOL lower than the children (19-35%) and condition-specific HRQOL higher than the children (17-33%). Findings of the binary logistic regression analyzes showed that the child's age, gender, and country (Germany vs. Sweden) were significant predictors of parent-child agreement in condition-specific HRQOL. We did not identify any significant variables that explain agreement for the generic HRQOL. CONCLUSION: The parent-child agreement is mostly good, suggesting that parent-reports are a reliable source of information. However, discrepancies may occur and can be explained by the child's age, gender, and country (Sweden vs. Germany). Both perspectives are essential sources for treating EA patients and should not be considered right or wrong. Instead, this information broadens the perspective on pediatric EA patients.

A scoring system for predicting downgrading and resolution of high-grade infant vesicoureteral reflux.

AIM: Aim of the study was to provide a scoring system for predicting downgrading and resolution of infantile high-grade vesicoureteral reflux (VUR). METHODS: Eighty-nine infants (65 boys) with high-grade VUR (grade 4-5) diagnosed at median age 2.5 months and followed to 39 months had repeated investigations of VUR grade, renal damage/function and bladder function. Recurrent urinary tract infections (UTIs) were registered. Risk variables collected at 1 year were analysed as independent factors for spontaneous resolution to grades ≤2 and 0, using univariable/multivariable logistic regression. RESULTS: A scoring system was built with a total of 14 points from four independent risk factors (sex, breakthrough UTI, type of renal damage and subnormal glomerular filtration rate). Children with persistent VUR (grade 3-5) had higher scores compared with the group with spontaneous resolution (grade 0-2) (mean 7.9 vs. 4.5, P < .0001). A score of ≥8 points indicated a low probability of VUR resolution (≤14%). The model was considered excellent based on area under the ROC curve (0.82) and showed satisfactory internal validity. CONCLUSION: This model provides a practical tool in the management of infants born with high-grade reflux. High scores at one year of age indicate a high risk of persistent dilated reflux.

Bladder/bowel dysfunction in pre-school children following febrile urinary tract infection in infancy.

BACKGROUND: An association between bladder-bowel dysfunction (BBD) and urinary tract infection (UTI) is well-known. However, a question less explored is whether children with UTI early in life also have increased prevalence of BBD after they are toilet-trained. In this study, consecutively selected children with pyelonephritis during their first year of life were assessed for BBD at pre-school age. METHODS: Ninety-two children (51 boys) hospitalized due to pyelonephritis during their first year of life were assessed for BBD at median age 5.4 years. A validated BBD questionnaire, along with urine flow and residual volume measurements, was used for diagnosing BBD. During follow-up, the group was well-characterized regarding renal status, vesicoureteral reflux (VUR), and recurrent UTI. RESULTS: BBD was diagnosed in 35/92 (38%), of which the majority was sub-diagnosed with dysfunctional voiding (DV). There was a strong association between BBD and recurrent UTI during follow-up (p < 0.0001), but only a slight association with VUR status at presentation. Nevertheless, in the group with both BBD and VUR, recurrent UTI was four times higher (12/13, 92%) than in children who had neither VUR nor BBD (23%), (p = 0.0008). BBD was also associated with kidney damage (p = 0.017). CONCLUSION: In children with pyelonephritis during the first year of life, 38% had BBD at pre-school age, regardless of whether they had VUR or not. The study shows an important association between BBD and recurrent UTI, so an assessment of BBD is therefore recommended for pre-school children with UTI, especially when they have history of pyelonephritis during infancy.

Parent-Reported Feeding Difficulties among Children Born with Esophageal Atresia: Prevalence and Early Risk Factors.

INTRODUCTION: We aimed to describe the prevalence of observable feeding difficulties during mealtimes among children with repaired esophageal atresia (EA) and to determine their early predictors. MATERIALS AND METHODS: A survey, based on parents' reports and concerning difficulties in EA children's nutritional intake, was performed with the help of 114 families of 2 to 17-year-old EA patients. Neonatal and clinical/surgical data were collected from medical records. Comparisons were made of the prevalence n (%) of feeding difficulties between children in three age groups (2 to 7 years, 8 to 12 years, or 13 to 17 years of age) using the Mantel-Haenszel chi-square test. Logistic regression identified outcome predictors (odds ratio: 95% confidence interval). Predictors with p ≤ 0.1 in the univariable analysis were included in multiple regression analysis (p < 0.05). RESULTS: Seventy-five percent of the young children aged 2 to 7, (median number of feeding difficulties: 2), 61% of school-aged children aged 8 to 12 (median number of feeding difficulties: 1), and 60% patients in the teenage group, aged 13 to 17, (median number of feeding difficulties: 1), reported feeding difficulties. Surgical complications after EA repair independently predicted children having a gastrostomy (p ≤ 0.01), using a food infusion pump (p ≤ 0.01), taking small portions to facilitate eating (p = 0.01), and needing >30 minutes to finish a main meal (p = 0.02). Congenital independent predictors were VACTERL, low birth weight, and preterm birth. CONCLUSION: Parentally observed feeding difficulties were commonly reported during early childhood, although prevalence decreases in older age groups. Several congenital and surgical factors were identified as independent predictors of complicated nutritional intake patterns.

Urological results and patient satisfaction in adolescents after surgery for proximal hypospadias in childhood.

BACKGROUND: Proximal hypospadias repair is associated with a considerable complication risk. Long-term follow-up is required to present realistic expectations in pre-operative counseling. OBJECTIVE: To investigate adolescents after childhood surgery for proximal hypospadias in a prospective cohort study describing the urological outcome, complication rates and patient satisfaction with penile appearance. STUDY DESIGN: 39 adolescents ≥14 years with penoscrotal to perineal hypospadias and primary urethroplasty (tubularized incised plate (TIP), preputial flap as Onlay or tubularized (Duckett)) from 1996 to 2005 at a single center were evaluated. The clinical assessment, at Md 16.5 years (14-25), included voiding history, genital examination including the Hypospadias Objective Scoring Evaluation (HOSE), uroflowmetry plus chart data from previous urinary flows and evaluation of patient satisfaction using the Penile Perception Score (PPS). RESULTS: Twenty-nine patients with penoscrotal and 10 with scrotal/perineal hypospadias underwent surgery with TIP (N = 14), Onlay (N = 14) and Duckett (N = 11). Uroflows improved significantly compared with prepubertal maximal flows. Impaired flow rate (<10 mL/s) was found in 14% (5/36). Fifty-one percent (20/39) required reoperations, 29% (4/14) of TIP, 50% (7/14) of Onlay and 82% (9/11) of Duckett (p = 0.0062). Median penile length in adolescence was 8.7 cm (4.0-11.0). Forty-four percent (12/27) of patients were dissatisfied with penile length. Patients were 'satisfied' or 'very satisfied' with meatal position and shape despite HOSE for meatal position being 11% (4/38) distal, 76% (29/38) proximal glanular and 13% (5/38) coronal. TIP patients had more curvature at puberty than Duckett (p = 0.0062). Patients that had a decurvature procedure had shorter penile length (p = 0.019). DISCUSSION: A high complication rate is previously described, predominantly within the first years. Our study shows 50% of reoperations were performed after >3 years, illustrating the need for long-term follow-up. Patient satisfaction with a deviant meatal position is rarely reported [1,2]. Our results support a conservative approach to an asymptomatic retracted meatus. Limitations of this descriptive study are the non-comparable groups and the retrospective data for correlation, impeding evaluation of prognostic outcome-factors. The shorter penile length found in patients after plication, and increased curvature after TIP, is therefore merely descriptive. However, the findings are in line with earlier publications suggesting limited use of TIP, and plication (recommending ventral lengthening instead) to avoid penile shortening and curvature in these cases [3-5]. CONCLUSIONS: The urological long-term outcome after proximal hypospadias repair is good, although late reoperations are common. In adolescence, patients were dissatisfied with the short penile length but satisfied with meatal position, indicating that in proximal hypospadias, preserving penile length and correcting curvature are prioritized over a distal meatus.

Changes in differential renal function after pyeloplasty in infants and children.

INTRODUCTION: Ureteropelvic junction obstruction (UPJO) is one of the most common causes of hydronephrosis in pediatric populations. Many need surgical intervention. The aim of surgery is preserving renal function and reducing symptoms such as urinary tract infections and pain. OBJECTIVES: The objectives were to evaluate differential renal function (DRF) in infants and children after surgery for UPJO and to identify factors predicting postoperative improvement. The difference in outcomes between patients with antenatal hydronephrosis and those diagnosed later was evaluated. STUDY DESIGN: A total of 85 children (63 boys and 22 girls) aged 0-16 years, treated for UPJO with dismembered pyeloplasty, were followed up as per a structured protocol including ultrasounds and renal scans (MAG-3) pre-operatively and three and 18 months postoperatively. Five children with bilateral or single kidney UPJO were excluded. Patient records were retrospectively reviewed, and the patients were grouped as per prenatal (group 1, n = 23) or postnatal (group 2, n = 57) diagnosis. Univariable and multivariable logistic regression analyses searching for factors predicting >5% postoperative improvement in DRF on the obstructed side were performed. Factors included in analyses were age at diagnosis and surgery, sex, type of presentation, cause of obstruction, estimated glomerular filtration rate, pre-operative DRF, anteroposterior diameter (APD), APD/renal parenchymal thickness, and grade of hydronephrosis as per the Onen alternative grading system (grade 1-4). RESULTS: Pre-operative DRF on the obstructed side was a mean of 42% (standard deviation, 12), with no difference between the groups. The median age at surgery was 0.9 (0.2-10) and 8.1 (0.6-16) years in groups 1 and 2, respectively (P < 0.001). The majority had unchanged DRF 18 months postoperatively, 19 (27%) patients improved by >5%, and one deteriorated. The proportion of patients with improved DRF was higher in group 1 (n = 10; 45%, P = 0.026). Anteroposterior diameter, APD/parenchymal thickness, pre-operative DRF, and antenatal diagnosis were predictors in the univariable analyses, and high APD (odds ratio [OR] = 1.1, P = 0.0023), antenatal diagnosis (OR = 0.23, P = 0.048), and low pre-operative DRF (OR = 0.90, P = 0.0045) built the best model of independent factors predicting improvement in DRF in multivariable analyses (Summary Figure). DISCUSSION: The limitation of the study is that it is retrospective, but it has the advantage of a uniform follow-up protocol, including patients from a five-year period, with few lost to follow-up. The results can be of interest in evaluating factors of importance for predicting recovery of function in obstructive uropathies in children. CONCLUSION: The majority of children had preserved or improved function after surgery for UPJO. Those with an antenatal diagnosis displayed a greater ability to catch up in DRF, and high APD, antenatal diagnosis, and low pre-operative DRF were independent predictive factors of an improvement in renal function after pyeloplasty.

Bladder/bowel dysfunction at school age is seen in children with high-grade vesicoureteral reflux and lower urinary tract dysfunction in infancy.

AIM: In approximately one third of cases, congenital high-grade vesicoureteral reflux (VUR) diagnosed during infancy is seen together with lower urinary tract dysfunction (LUTD), characterised by a high-capacity bladder and incomplete emptying. In an earlier study, 20 of these infants were treated with clean intermittent catheterisation during a 3-year period and with surgical treatment of the VUR before catheterisation was ended. In the present study, bladder function was evaluated in these children at school age. METHODS: Bladder function was evaluated in the 20 children at a mean age of 7.3 years using a validated voiding-bowel questionnaire with scores (cut-off score 7) and a urine flow/residual study. RESULTS: Four children (20%) had a normal voiding function at follow-up, whereas 11 (55%) had a clear bladder/bowel dysfunction (scores 7-19) and five (25%) had a mild dysfunction (score 6). Ten (63%) of the children with any dysfunction were recognised as dysfunctional voiding. Recurrent febrile urinary tract infections were correlated with the scores of faecal questions (P = .041), but for total scores P = .058. CONCLUSION: The follow-up of bladder function in children at 7.3 years, diagnosed with high-grade VUR and LUTD in infancy, revealed bladder/bowel dysfunction of varying severity in the majority of cases.

Long-Term Effectiveness of Antireflux Surgery in Esophageal Atresia Patients.

INTRODUCTION: Gastroesophageal reflux disease (GERD) is a common morbidity after esophageal atresia (EA) repair, and the antireflux procedure (ARP) is a way of treating GERD symptoms. The aim of this study was to evaluate whether reflux index (Ri) and growth were improved by ARP. MATERIALS AND METHODS: Ninety-nine individuals with EA treated at the Queen Silvia Children's Hospital in Gothenburg, Sweden, between 1997 and 2010 were followed prospectively according to a structured care program. Twenty-four-hour pH-metry (Ri) and growth were studied at birth, then at 1, 7, and 15 years of age. All the patients included had reached 7 years of age. RESULTS: Preoperatively, Ri was significantly higher (32%) in the ARP than the non-ARP group (10%). Postoperatively, no difference was seen between the groups. However, at 7 and 15 years of age, Ri was significantly higher in the ARP group than in the non-ARP group. Weight (standard deviation scores) was significantly lower in the ARP group at 1 and 7 years of age when compared with the non-ARP group, but these differences were not seen at birth and at 15 years of age. In a multivariate analysis, only birth weight remained a significant factor for low weight at 7 years of age. At 15 years of age, no risk factors for low body weight were found. CONCLUSION: In the long term, ARP is not effective in reducing GERD as measured as Ri in EA patients. The ARP group had significantly lower weight at 1 and 7 years of age than the non-ARP group, but this was not the case at the age of 15.

Health-related quality of life among children, adolescents, and adults with bladder exstrophy-epispadias complex: a systematic review of the literature and recommendations for future research.

PURPOSE: Bladder exstrophy-epispadias complex (BEEC) is a rare spectrum of genitourinary malformations. Children risk long-term urinary and genital dysfunctions. To achieve a comprehensive understanding, this study aimed to review the literature on generic and disease-specific health-related quality of life (HRQOL) in BEEC patients, and methodologies used. METHODS: A literature search was conducted in Pubmed/CINAHL/Embase/PsycINFO/Cochrane, from inception to May 2018. A meta-analysis of HRQOL in BEEC patients compared to healthy references was performed. RESULTS: Twenty-one articles (published 1994-2018), describing HRQOL of children and adolescents (n = 5) and adults only (n = 5), or integrated age populations (n = 11), were identified (median sample size 24, loss to follow-up 43%, response rate 84%). Overall HRQOL was reduced in BEEC patients compared to healthy references in 4/4 studies. Impaired physical or general health in BEEC patients has been described in 9 articles, diminished mental health in 11, restricted social health in 10, and sexual health/functioning or body perception impairments in 13 articles. Urinary incontinence was the most common factor related to worse HRQOL (12 studies). In six studies, HRQOL was better than healthy norms. In eligible studies (n = 5), the pooled estimate of the effect of BEEC indicated worse HRQOL for children and adults (0 > effect sizes < 0.5). Thirty-six HRQOL assessments were used, none developed and validated for BEEC. CONCLUSIONS: HRQOL in BEEC patients may be negatively impacted, particularly considering mental and social HRQOL. Sexual health/functioning or body perception impairments may be present in adolescents and adults. However, HRQOL is heterogeneously assessed and subsequent findings are differently reported. Additional research is warranted and can be improved.

Generic Health-Related Quality of Life after Repair of Esophageal Atresia and Its Determinants within a German-Swedish Cohort.

AIM: Despite advances of outcomes of esophageal atresia (EA), knowledge on patients' health-related quality of life (HRQoL) is sparse. Due to the heterogeneity of EA, larger cohorts need to be investigated to ensure reliability of data. Aim was to determine generic HRQoL after EA repair in a Swedish-German cohort. PATIENTS AND METHODS: Ethical approval was obtained. A total of 192 patients (2-18 years; 134 Swedish; 58 German) were included. Clinical data were reviewed. EA was classified in "severe" and "mild/moderate." Pediatric Quality of Life Inventory 4.0 Generic Core Scale (PedsQL 4.0) was used in appropriate versions (2-7 years; 8-18 years; self- [SR] and proxy report [PR]) to determine generic HRQoL. RESULTS: Swedish and German samples were clinically and demographically comparable. HRQoL was lower in "severe EA" versus "mild/moderate" (2-18 years; total score; PR 85.6 vs. 73.6; p < 0.001) and Gross A versus Gross C type EA (2-7 years; total score; PR 61.0 vs. 79.3; p = 0.035). Total HRQoL was higher in the Swedish versus German sample (2-18 years; total score; PR 82.3 vs. 72.7; p = 0.002). HRQoL was impaired in the German sample versus healthy population (2-18 years; total score; PR 72.7 vs. 82.7; p = 0.001). In German patients (8-18 years), HRQoL was higher in SR versus PR (80.7 vs. 74.7; p = 0.044). Patients' age and presence of VACTERL association or isolated anorectal malformations did not affect HRQoL. Various differences were detected regarding different dimensions of PedsQL 4.0. CONCLUSION: In this first international study, we found several differences in perception of generic HRQoL. HRQoL appears to be determined by the type of EA and severity rather than patients' age or the presence of typical associated malformations. Country-specific differences may be culturally dependent, but further investigations are suggested. A condition-specific instrument validated for EA may provide additional insights.

Health-related quality of life experiences in children and adolescents born with esophageal atresia: A Swedish-German focus group study.

BACKGROUND: Esophageal atresia (EA) is a rare malformation of the esophagus, which needs surgical treatment. Survival rates have reached 95%, but esophageal and respiratory morbidity during childhood is frequent. Child and parent perspectives and cultural and age-specific approaches are fundamental in understanding children's health-related quality of life (HRQoL) and when developing a pediatric HRQoL questionnaire. We aimed to increase the conceptual and cross-cultural understanding of condition-specific HRQoL experiences among EA children from Sweden and Germany and investigate content validity for an EA-specific HRQoL questionnaire. METHODS: Eighteen standardized focus groups (FGs) with 51 families of EA children aged 2-17 years in Sweden (n = 30 families) and Germany (n = 21 families) were used to explore HRQoL experiences, which were content analyzed into HRQoL domains. The Swedish HRQoL domains were analyzed first and used as framework to evaluate HRQoL content reported in the German FGs. HRQoL experiences were then categorized as physical, social, and emotional HRQoL burden or resource. RESULTS: One thousand nine hundred eight HRQoL statements were recorded. All nine EA-specific HRQoL domains identified in the Swedish FGs (eating, social relationships, general life issues, communication, body issues, bothersome symptoms, confidence, impact of medical treatment, and additional difficulties due to concomitant anomalies) were recognized in the FGs held in Germany, and no additional EA-specific HRQoL domain was found. The HRQoL dimensions referenced physical burden (n = 655, 34.5%), social burden (n = 497, 26.0%), social resources (n = 303, 15.9%), emotional burden (n = 210, 11.0%), physical resources (n = 158, 8.3%), and emotional resources (n = 85, 4.5%). CONCLUSION: This first international FG study to obtain the EA child and his or her parents' perspective on HRQoL suggests Swedish-German qualitative comparability of the HRQoL domains and content validity for a cross-cultural EA-specific HRQoL questionnaire. EA children make positive and negative HRQoL experiences, but prominently related to physical and social burden, which underlines appropriate follow-up care and future research.

The Esophageal-Atresia-Quality-of-life Questionnaires: Feasibility, Validity and Reliability in Sweden and Germany.

OBJECTIVES: Esophageal atresia (EA) is a rare malformation characterized of discontinuity of the esophagus, concurrent with or without a tracheoesophageal fistula (TEF). We report the feasibility validity and reliability of a condition-specific quality-of-life (QOL) tool for EA/TEF children, the age-adapted EA-QOL-questionnaires, when used in Sweden and Germany. METHODS: A total of 124 families of children with EA/TEF participated in the study; 53 parents completed the EA-QOL-questionnaire for children aged 2 to 7 years; 62 children/71 parents the EA-QOL-questionnaire for children 8 to 17 years. Feasibility was determined from the percentage of missing item responses. Based on clinical data and previously validated generic QOL-instruments (PedsQL 4.0, DISABKIDS-12), the final EA-QOL scores were evaluated against hypotheses of validity (known-groups/concurrent/convergent) and reliability (internal consistency/retest reliability of scores for 3 weeks). Significant level was P < 0.05. RESULTS: In the questionnaire for EA/TEF children aged 2 to 7 years, 16/18 items were completed with missing values <6% (range 0%-7.5%), and in the questionnaire for 8 to 17-year-olds, 24/24 child-reported items (range 0%-4.8%) and 21/24 parent-reported items (range 0%-7.0%). In both age-specific EA-QOL-questionnaires, desirable standards for known-groups and concurrent validity were fulfilled; digestive symptoms and feeding difficulties negatively impacted EA-QOL-Total-scores (P < 0.001), and as hypothesized, in 2 to 7-year-olds, respiratory symptoms decreased EA-QOL-Total-scores (P = 0.002). Correlations between the EA-QOL and generic QOL questionnaires supported convergent validity. Internal consistency reliability was satisfactory. The level of agreements of EA-QOL-scores between the field- and retest study were good to excellent. CONCLUSIONS: The overall psychometric performance of the EA-QOL-questionnaires for EA/TEF children is satisfactory and can enhance outcome evaluations in future research and clinical practice.