Defining outcomes following congenital diaphragmatic hernia using standardised clinical assessment and management plan (SCAMP) methodology within the CDH EURO consortium.

Treatment modalities for neonates born with congenital diaphragmatic hernia (CDH) have greatly improved in recent times with a concomitant increase in survival. In 2008, CDH EURO consortium, a collaboration of a large volume of CDH centers in Western Europe, was established with a goal to standardize management and facilitate multicenter research. However, limited knowledge on long-term outcomes restricts the identification of optimal care pathways for CDH survivors in adolescence and adulthood. This review aimed to evaluate the current practice of long-term follow-up within the CDH EURO consortium centers, and to review the literature on long-term outcomes published from 2000 onward. Apart from having disease-specific morbidities, children with CDH are at risk for impaired neurodevelopmental problems and failure of educational attainments which may affect participation in society and the quality of life in later years. Thus, there is every reason to offer them long-term multidisciplinary follow-up programs. We discuss a proposed collaborative project using standardized clinical assessment and management plan (SCAMP) methodology to obtain uniform and standardized follow-up of CDH patients at an international level.

Outcome for biliary atresia patients treated at a low-volume centre.

OBJECTIVES: The importance of case load for treatment of extrahepatic biliary atresia (BA) is debated. The aim of this study was to register results of BA treatment in a small volume centre. METHODS: Retrospective chart review study of patients with BA treated from 2000 to 2017. The institutional review board approved the study. RESULTS: Forty-five babies were identified of which 42 (93%) are alive. Forty-one patients had a Kasai portoenterostomy (PE), two had a hepaticojejunostomy and two had a primary liver transplantation. The age at PE/hepaticojejunostomy was median 63 (4-145 days). Seven surgeons performed the operations, and the median duration of the diagnostic work-up was 8 (3-24) days. Clearance of jaundice was achieved in 23/43 (53%) babies, and 3- and 5-year native liver survival was 47% and 40%, respectively. Clearance of jaundice post-PE/hepaticojejunostomy was a strong predictor of native liver survival (adjusted OR: 0.027; 95%; p = .009). Plasma level of conjugated bilirubin at time of referral was also a significant predictor of native liver survival (adjusted OR: 1.053; p = .017). CONCLUSION: A small volume centre may achieve satisfactory results for BA patients. The study has, however, identified factors that may further improve results; earlier referral, optimizing diagnostic work-up and establishing one dedicated surgical team.

Comparison of clinical outcomes after total transanal and laparoscopic assisted endorectal pull-through in patients with rectosigmoid Hirschsprung disease.

BACKGROUND: Total transanal (TERPT) and laparoscopic endorectal pull-through (LERPT) are the most common procedures to treat rectosigmoid Hirschsprung's disease (HD). Since few studies have compared the two methods, we aimed to assess clinical outcomes after TERPT and LERPT in this cross-sectional study. METHODS AND PATIENTS: All patients with rectosigmoid HD operated with TERPT and LERPT between 2001 and 2018 were eligible. Peri-operative data were registered from patients' records, and bowel function was assessed according to the Krickenbeck classification. RESULTS: 91/97 (94%) patients were included; 46 operated with TERPT and 45 with LERPT. Bowel function was assessed in 80 patients at median seven (4-17) years. There was no difference in functional outcome between the procedures. Unplanned procedures under general anesthesia were frequent; 28% after TERPT and 49% after LERPT (p = 0.04). 11% of TERPT and 29% of LERPT patients got botulinum toxin injections (p = 0.03). In the TERPT group, patients operated in the neonatal period had poorer outcome (78%) than those operated later (24%) (p = 0.005). No difference in operative time, length of hospital stay, and rate of early and late complications was found between the procedures. CONCLUSION: There was no difference in long-term bowel function in patients with rectosigmoid HD operated with TERPT or LERPT. More LERPT patients had an unplanned procedure under general anesthesia, mostly due to obstructive symptoms. LEVEL OF EVIDENCE: III.

Development of a core outcome set for congenital pulmonary airway malformations: study protocol of an international Delphi survey.

INTRODUCTION: A worldwide lack of consensus exists on the optimal management of asymptomatic congenital pulmonary airway malformation (CPAM) even though the incidence is increasing. Either a surgical resection is performed or a wait-and-see policy is employed, depending on the treating physician. Management is largely based on expert opinion and scientific evidence is scarce. Wide variations in outcome measures are seen between studies making comparison difficult thus highlighting the lack of universal consensus in outcome measures as well. We aim to define a core outcome set which will include the most important core outcome parameters for paediatric patients with an asymptomatic CPAM. METHODS AND ANALYSIS: This study will include a critical appraisal of the current literature followed by a three-stage Delphi process with two stakeholder groups. One surgical group including paediatric as well as thoracic surgeons, and a non-surgeon group including paediatric pulmonologists, intensive care and neonatal specialists. All participants will score outcome parameters according to their level of importance and the most important parameters will be determined by consensus. ETHICS AND DISSEMINATION: Electronic informed consent will be obtained from all participants. Ethical approval is not required. After the core outcome set has been defined, we intend to design an international randomised controlled trial: the COllaborative Neonatal NEtwork for the first CPAM Trial, which will be aimed at determining the optimal management of patients with asymptomatic CPAM.