Self-reported respiratory and gastrointestinal outcomes in children with isolated congenital diaphragmatic hernia: A prospective multicentre study.

OBJECTIVE: To evaluate medium-term self-reported respiratory and gastrointestinal (GI) outcomes in children with congenital diaphragmatic hernia (CDH). DESIGN: Self-reported respiratory and GI outcomes correlated with prenatal severity indicators. SETTING: Prospective study at three fetal medicine units. POPULATION: Families of children prenatally diagnosed with isolated, left-sided CDH surviving for >1 year. METHODS: Families received validated questionnaires for GI outcomes (Infant Gastroesophageal Reflux Questionnaire Revised, I-GERQ-R, for infants aged <2 years, or Paediatric Gastro-oesophageal Symptom and Quality of Life Questionnaire, PGSQ, for children aged aged 2-8 years or >9 years) and respiratory outcomes (preschool respiratory outcome questionnaire, for children aged ≤5 years, or the International Study of Asthma and Allergies in Childhood asthma questionnaire, for children aged 6-8 years or ≥9 years). Prenatal data collected from the medical records included lung size (percentage observed/expected lung-to-head ratio, O/E LHR %), liver position, fetal endoluminal tracheal occlusion (FETO) gestational age (GA) at delivery, and perinatal data included birthweight, location, patch repair and respiratory support. MAIN OUTCOME MEASURES: The GI and respiratory scores were correlated with O/E LHR using linear and logistic regression models. Univariate analysis was used to evaluate associations with perinatal variables. RESULTS: We obtained 142 responses from 342 families (representing a response rate of 45%). The baseline characteristics of participants and non-participants were comparable. No correlations between perinatal variables and respiratory or GI scores were identified. Children aged ≤5 years with lower O/E LHR values reported higher respiratory scores (P = 0.0175); this finding was not reported in older children. Overall, the children who underwent FETO (n = 51) had GI (P = 0.290) and respiratory (P = 0.052) scores that were comparable with those of children who were expectantly managed. CONCLUSIONS: Families and children with prenatally diagnosed CDH reported fewer respiratory symptoms with increasing age. There was no correlation between O/E LHR or the use of FETO and self-reported outcomes.

Acute-on-chronic liver failure in children with biliary atresia awaiting liver transplantation.

OBJECTIVES: Acute-on-chronic liver failure (ACLF) is an acute decompensation of cirrhosis complicated by other organ failure and is associated with increased mortality and morbidity. ACLF has not been studied in children with biliary atresia (BA), which is the commonest indication for pediatric liver transplantation (LT) worldwide. This study aims to evaluate ACLF and outcomes in children with BA while awaiting deceased donor LT. METHODS: This was a subanalysis of the dataset from a prospective cohort study of patients aged 0-18 years who underwent portoenterostomy for BA and were listed for LT at King's College Hospital, London, between 1999 and 2003. Outcomes included the development of ACLF, mortality, and complications. RESULTS: Ninety-nine (41 male) children were included, and follow-up was 10 [6.0-15.0] years. A total of 20/99 children developed ACLF. ACLF was associated with increased mortality while awaiting LT (20% vs 4%; P = 0.03). There were no associations between biochemical parameters at listing and death. Increased bilirubin levels 3 months post-portoenterostomy was predictive of development of ACLF (AUROC = 0.72, P < 0.01). Age at LT and time on the waiting list in the ACLF subgroup were both lower compared to the non-ACLF group (P > 0.05). Sepsis and gastrointestinal bleeding were the commonest precipitants of ACLF. Complications included ascites, hepatic encephalopathy, and hepatorenal syndrome; the ACLF subgroup required multisystem support and longer intensive care unit stay. CONCLUSIONS: ACLF in children with BA awaiting deceased donor LT carries increased mortality and morbidity. This warrants stratification of patients for earlier wait-listing and prioritization for LT.

AST-to-platelet ratio index in non-invasive assessment of long-term graft fibrosis following pediatric liver transplantation.

Long-term graft fibrosis occurs in the majority of pediatric liver transplant recipients. Serial biopsies to monitor graft health are impractical and invasive. The APRI has been evaluated in pediatric liver disease, but not in the context of post-transplantation fibrosis. We aimed to investigate the validity of APRI as a predictor of long-term graft fibrosis in pediatric liver transplant recipients. This was a retrospective, observational study of a cohort of children who underwent liver transplantation at King's College Hospital between 1989 and 2003, with a relevant dataset available. Protocol liver biopsies were performed at 10-yr follow-up and fibrosis was graded using the Ishak scoring system, with S3-6 denoting "significant fibrosis." APRI was calculated concurrently with biopsy. A total of 39 asymptomatic patients (20 males; median age at transplant, 1.43 yr) underwent protocol liver biopsies at a median of 10.39 yr post-transplantation. APRI was associated with significant fibrosis (p = 0.012). AUROC for APRI as a predictor of significant fibrosis was 0.74 (p = 0.013). The optimal cutoff APRI value for significant fibrosis was 0.45 (sensitivity = 0.67; specificity = 0.79; PPV = 0.67; NPV = 0.79). APRI appears to be a useful non-invasive adjunct in the assessment of significant graft fibrosis in the long-term follow-up of pediatric liver transplant survivors.

Complete Resection of Necrotic Bowel Improves Survival in NEC Without Compromising Enteral Autonomy.

AIMS: Controversy persists regarding operative strategy for necrotising enterocolitis (NEC). Some surgeons advocate resecting all necrotic bowel, whilst others defunction with a stoma, leaving diseased bowel in situ to preserve bowel length. We reviewed our institutional experience of both approaches. METHODS: Neonates undergoing laparotomy for NEC May 2015-2019 were identified. Data extracted from electronic records included: demographics, neonatal Sequential Organ Failure Assessment (nSOFA) score at surgery, operative findings, and procedure performed. Neonates were assigned to two groups according to operative strategy: complete resection of necrotic bowel (CR) or necrotic bowel left in situ (LIS). Primary outcome was survival, and secondary outcome was enteral autonomy. Outcomes were compared between groups. RESULTS: Fifty neonates were identified. Six were excluded: 4 with NEC totalis and 2 with no visible necrosis or histological confirmation of NEC. Of the 44 remaining neonates, 27 were in the CR group and 17 in the LIS group. 32 neonates survived to discharge (73%). On univariate analysis, survival was associated with lower nSOFA score (P = 0.003), complete resection of necrotic bowel (OR 9.0, 95% CI [1.94-41.65]), and being born outside the surgical centre (OR 5.11 [1.23-21.28]). On Cox regression multivariate analysis, complete resection was still strongly associated with survival (OR 4.87 [1.51-15.70]). 28 of the 32 survivors (88%) achieved enteral autonomy. There was no association between operative approach and enteral autonomy (P = 0.373), or time to achieve this. CONCLUSION: Complete resection of necrotic bowel during surgery for NEC significantly improves likelihood of surviving without negatively impacting remaining bowel function. LEVEL OF EVIDENCE: III.

Questionnaire-based service evaluation of the efficacy and usefulness of SEREN: a structured education programme for children and young people diagnosed with type 1 diabetes mellitus.

OBJECTIVES: To evaluate the usefulness and effectiveness of a new structured education module for children with type 1 diabetes: Structured Education Reassuring Empowering Nurturing (SEREN) 'Diabetes at Diagnosis'. DESIGN: Retrospective questionnaire-based service evaluation. SETTING: 12/14 paediatric diabetes centres across Wales took part. PARTICIPANTS: Children diagnosed with type 1 diabetes 1 year before (pre-SEREN group) and 1 year after the introduction of SEREN (post-SEREN group) were selected using a national diabetes register. RESOURCE: 'Diabetes at Diagnosis' delivers structured education to empower children and families with self-management of type 1 diabetes. EVALUATION: Primary outcomes were patient-reported effectiveness and user-friendliness of the educational resources and quality of life (PedsQL). Age-appropriate child and parent questionnaires were provided. Clinical outcomes included glycated haemoglobin (HbA1c) at 6 and 12 months, service engagement and diabetes-related hospital admissions in the first year. RESULTS: 89/106 responded pre-SEREN and 108/115 post-SEREN, with no demographic differences at diagnosis. Parent scores for educational package evaluation significantly improved post-SEREN, with a non-significant trend towards improved results in children. PedsQL scores were similar. There was no change in HbA1c overall. Subgroup analyses at 12 months showed a trend towards a lower HbA1c in key stage 1-2 (62 vs 58 mmol/mol, p=0.06) and increased HbA1c in key stage 3-4 (56 vs 66 mmol/mol, p=0.009). There were no differences in hospital admissions or missed clinic appointments. CONCLUSIONS: This is an evaluation of the only standardised type 1 diabetes structured education programme in use for children throughout Wales. This module improved parent-reported outcomes and showed a non-significant trend towards improved usefulness in children, without a difference in a PedsQL scores overall. Ongoing evaluation of the cohort who received subsequent SEREN modules may show the long-term benefit of the programme.

Patch repair of congenital diaphragmatic hernia is not at risk of poor outcomes.

PURPOSE: Recurrence of congenital diaphragmatic hernia (CDH) was retrospectively evaluated after correction with or without a patch in an institution where tension-free repair is advocated. METHODS: Demographics and outcomes of patients with a postero-lateral CDH repaired (2000-2016) were analyzed (univariate tests and binary logistic regression adjusting for time since start of study, gender, defect side, liver herniation, patch, surgical approach, absence of postero-lateral rim and length of follow-up). RESULTS: Of 203 patients, 107 received a patch (P), and 96 were not patched (NP). Groups were not different for gestational age birthweight, gender, defect side and minimally invasive approach rate. Preoperative ECMO incidence (P:29.9% vs. NP:2.1%, p < 0.01), liver herniation (P:57.0% vs. NP:22.9%, p < 0.01) and absence of a postero-lateral rim (P:61.7% vs. NP:8.3%, p < 0.01) were higher in the P group. The mortality rate was 10.8% (P:15.0% vs. NP:6.2%, p = 0.07). Recurrence was not different (P:9.3% vs. NP:4.2%, p = 0.15). Multivariate analysis showed that recurrence was higher after thoracoscopy compared to open (OR = 12.2 [2.2-68], p < 0.01); neither the use of patch (OR = 2.3, [0.5-10.4], p = 0.28) nor any other factors were associated with recurrence. CONCLUSION: In this single centre series where tension-free repair was advocated, patch repair of CDH was not associated with higher recurrence, though access route was. TYPE OF STUDY: Cohort Study. LEVEL OF EVIDENCE: Level III.