Background Neonatal research evaluates many different outcomes using multiple measures. This can prevent synthesis of trial results in meta-analyses, and selected outcomes may not be relevant to former patients, parents and health professionals. Objective To define a core outcome set (COS) for research involving infants receiving neonatal care in a high-income setting. Design Outcomes reported in neonatal trials and qualitative studies were systematically reviewed. Stakeholders were recruited for a three-round international Delphi survey. A consensus meeting was held to confirm the final COS, based on the survey results. Participants Four hundred and fourteen former patients, parents, healthcare professionals and researchers took part in the eDelphi survey; 173 completed all three rounds. Sixteen stakeholders participated in the consensus meeting. Results The literature reviews identified 104 outcomes; these were included in round 1. Participants proposed 10 additional outcomes; 114 outcomes were scored in rounds 2 and 3. Round 1 scores showed different stakeholder groups prioritised contrasting outcomes. Twelve outcomes were included in the final COS: survival, sepsis, necrotising enterocolitis, brain injury on imaging, general gross motor ability, general cognitive ability, quality of life, adverse events, visual impairment/blindness, hearing impairment/deafness, retinopathy of prematurity and chronic lung disease/bronchopulmonary dysplasia. Conclusions and relevance A COS for clinical trials and other research studies involving infants receiving neonatal care in a high-income setting has been identified. This COS for neonatology will help standardise outcome selection in clinical trials and ensure these are relevant to those most affected by neonatal care.

CONTEXT: Nonoperative treatment (NOT) with antibiotics alone of acute uncomplicated appendicitis (AUA) in children has been proposed as an alternative to appendectomy. OBJECTIVE: To determine safety and efficacy of NOT based on current literature. DATA SOURCES: Three electronic databases. STUDY SELECTION: All articles reporting NOT for AUA in children. DATA EXTRACTION: Two reviewers independently verified study inclusion and extracted data. RESULTS: Ten articles reporting 413 children receiving NOT were included. Six, including 1 randomized controlled trial, compared NOT with appendectomy. The remaining 4 reported outcomes of children receiving NOT without a comparison group. NOT was effective as the initial treatment in 97% of children (95% confidence interval [CI] 96% to 99%). Initial length of hospital stay was shorter in children treated with appendectomy compared with NOT (mean difference 0.5 days [95% CI 0.2 to 0.8]; P = .002). At final reported follow-up (range 8 weeks to 4 years), NOT remained effective (no appendectomy performed) in 82% of children (95% CI 77% to 87%). Recurrent appendicitis occurred in 14% (95% CI 7% to 21%). Complications and total length of hospital stay during follow-up were similar for NOT and appendectomy. No serious adverse events related to NOT were reported. LIMITATIONS: The lack of prospective randomized studies limits definitive conclusions to influence clinical practice. CONCLUSIONS: Current data suggest that NOT is safe. It appears effective as initial treatment in 97% of children with AUA, and the rate of recurrent appendicitis is 14%. Longer-term clinical outcomes and cost-effectiveness of NOT compared with appendicectomy require further evaluation, preferably in large randomized trials, to reliably inform decision-making.

Objective Congenital duodenal obstruction (CDO) comprising duodenal atresia or stenosis is a rare congenital anomaly requiring surgical correction in early life. Identification of variation in surgical and postoperative practice in previous studies has been limited by small sample sizes. This study aimed to prospectively estimate the incidence of CDO in the UK, and report current management strategies and short-term outcomes. Design Prospective population-based, observational study for 12 months from March 2016. Setting Specialist neonatal surgical units in the UK. Main outcome measures Incidence of CDO, associated anomalies and short-term outcomes. Results In total, 110 cases were identified and data forms were returned for 103 infants giving an estimated incidence of 1.22 cases per 10 000 (95% CI 1.01 to 1.49) live births. Overall, 59% of cases were suspected antenatally and associated anomalies were seen in 69%. Operative repair was carried out mostly by duodenoduodenostomy (76%) followed by duodenojejunostomy (15%). Postoperative feeding practice varied with 42% having a trans-anastomotic tube placed and 88% receiving parenteral nutrition. Re-operation rate related to the initial procedure was 3% within 28 days. Two infants died within 28 days of operation from unrelated causes. Conclusion This population-based study of CDO has shown that the majority of infants have associated anomalies. There is variation in postoperative feeding strategies which represent opportunities to explore the effects of these on outcome and potentially standardise approach. Short-term outcomes are generally good.

Background Appendectomy is considered the gold standard treatment for acute appendicitis. Recently the need for surgery has been challenged in both adults and children. In children there is growing clinician, patient and parental interest in non-operative treatment of acute appendicitis with antibiotics as opposed to surgery. To date no multicentre randomised controlled trials that are appropriately powered to determine efficacy of non-operative treatment (antibiotics) for acute appendicitis in children compared with surgery (appendectomy) have been performed. Methods Multicentre, international, randomised controlled trial with a non-inferiority design. Children (age 5–16 years) with a clinical and/or radiological diagnosis of acute uncomplicated appendicitis will be randomised (1:1 ratio) to receive either laparoscopic appendectomy or treatment with intravenous (minimum 12 hours) followed by oral antibiotics (total course 10 days). Allocation to groups will be stratified by gender, duration of symptoms (> or <48 hours) and centre. Children in both treatment groups will follow a standardised treatment pathway. Primary outcome is treatment failure defined as additional intervention related to appendicitis requiring general anaesthesia within 1 year of randomisation (including recurrent appendicitis) or negative appendectomy. Important secondary outcomes will be reported and a cost-effectiveness analysis will be performed. The primary outcome will be analysed on a non-inferiority basis using a 20% non-inferiority margin. Planned sample size is 978 children. Discussion The APPY trial will be the first multicentre randomised trial comparing non-operative treatment with appendectomy for acute uncomplicated appendicitis in children. The results of this trial have the potential to revolutionise the treatment of this common gastrointestinal emergency. The randomised design will limit the effect of bias on outcomes seen in other studies. Trial registration number clinicaltrials.gov: NCT02687464. Registered on Jan 13th 2016.