Patient-centred outcomes following non-operative treatment or appendicectomy for uncomplicated acute appendicitis in children.

While non-operative treatment has emerged as an alternative to surgery for the treatment of uncomplicated acute appendicitis in children, comparative patient-centred outcomes are not well documented. We investigated these in a feasibility randomised trial. Of 57 randomised participants, data were available for 26. Compared with appendicectomy, children allocated to non-operative treatment reported higher short-term quality of life scores, shorter duration of requiring analgesia, more rapid return to normal activities and shorter parental absence from work. These preliminary data suggest differences exist in recovery profile and quality of life between these treatments that are important to measure in a larger RCT. Trial registration number is ISRCTN15830435.

At risk child: a contemporary analysis of injured children in London and the South East of England: a prospective, multicentre cohort study.

Background: Injury is a leading health burden in children yet relatively little is reported about the contemporary risks they face. Current national registry data may under-represent the true burden of injury to children. We aim to analyse contemporary patterns of paediatric trauma and identify current factors putting children at risk of injury. Methods: A 3-month prospective multicentre cohort evaluation of injured children across the London Major Trauma System was performed. All children receiving a trauma team activation; meeting National Institute for Health and Care Excellence CT head criteria; or admitted/transferred out due to trauma were included. Data were collected on demographics, mechanism and location of injury, and body region injured. The primary outcome was in-hospital mortality and secondary outcome was safeguarding concerns. Results: 659 children were included. Young children were more likely to be injured at home (0-5 years old: 70.8%, n=167 vs adolescents: 15.6%, n=31). Adolescents were more likely to be injured in the street (42.7%, n=85). Head trauma caused over half of injuries in 0-5 years old (51.9%, n=121). Falls were common and increasingly prevalent in younger children, causing 56.6% (n=372) of injuries. In adolescents, penetrating violence caused more than one in five injuries (21.9%, n=50). Most injured children survived (99.8%, n=658), however, one in four (26.1%, n=172) had safeguarding concerns and a quarter of adolescents had police, third sector or external agency involvement (23.2%, n=53). Conclusions: This study describes modern-day paediatric trauma and highlights the variance in injury patterns in young children and adolescents. Importantly, it highlights differences in actual rates of injuries compared with those reported from current national registry data. We must understand real risks facing 21st century children to effectively safeguard future generations. The results provide an opportunity to reassess the current approach to injury prevention, child and adolescent safeguarding, and public health campaigns for child safety.

Conservative treatment for uncomplicated appendicitis in children: the CONTRACT feasibility study, including feasibility RCT.

BACKGROUND: Although non-operative treatment is known to be effective for the treatment of uncomplicated acute appendicitis in children, randomised trial data comparing important outcomes of non-operative treatment with those of appendicectomy are lacking. OBJECTIVES: The objectives were to ascertain the feasibility of conducting a multicentre randomised controlled trial comparing the clinical effectiveness and cost-effectiveness of a non-operative treatment pathway with appendicectomy for the treatment of uncomplicated acute appendicitis in children. DESIGN: This was a mixed-methods study, which included a feasibility randomised controlled trial, embedded and parallel qualitative and survey studies, a parallel health economic feasibility study and the development of a core outcome set. SETTING: This study was set in three specialist NHS paediatric surgical units in England. PARTICIPANTS: Children (aged 4-15 years) clinically diagnosed with uncomplicated acute appendicitis participated in the feasibility randomised controlled trial. Children, their families, recruiting clinicians and other health-care professionals involved in caring for children with appendicitis took part in the qualitative study. UK specialist paediatric surgeons took part in the survey. Specialist paediatric surgeons, adult general surgeons who treat children, and children and young people who previously had appendicitis, along with their families, took part in the development of the core outcome set. INTERVENTIONS: Participants in the feasibility randomised controlled trial were randomised to a non-operative treatment pathway (broad-spectrum antibiotics and active observation) or appendicectomy. MAIN OUTCOME MEASURES: The primary outcome measure was the proportion of eligible patients recruited to the feasibility trial. DATA SOURCES: Data were sourced from NHS case notes, questionnaire responses, transcribed audio-recordings of recruitment discussions and qualitative interviews. RESULTS: Overall, 50% (95% confidence interval 40% to 59%) of 115 eligible patients approached about the trial agreed to participate and were randomised. There was high acceptance of randomisation and good adherence to trial procedures and follow-up (follow-up rates of 89%, 85% and 85% at 6 weeks, 3 months and 6 months, respectively). More participants had perforated appendicitis than had been anticipated. Qualitative work enabled us to communicate about the trial effectively with patients and families, to design and deliver bespoke training to optimise recruitment and to understand how to optimise the design and delivery of a future trial. The health economic study indicated that the main cost drivers are the ward stay cost and the cost of the operation; it has also informed quality-of-life assessment methods for future work. A core outcome set for the treatment of uncomplicated acute appendicitis in children and young people was developed, containing 14 outcomes. There is adequate surgeon interest to justify proceeding to an effectiveness trial, with 51% of those surveyed expressing a willingness to recruit with an unchanged trial protocol. LIMITATIONS: Because the feasibility randomised controlled trial was performed in only three centres, successful recruitment across a larger number of sites cannot be guaranteed. However, the qualitative work has informed a bespoke training package to facilitate this. Although survey results suggest adequate clinician interest to make a larger trial possible, actual participation may differ, and equipoise may have changed over time. CONCLUSIONS: A future effectiveness trial is feasible, following limited additional preparation, to establish appropriate outcome measures and case identification. It is recommended to include a limited package of qualitative work to optimise recruitment, in particular at new centres. FUTURE WORK: Prior to proceeding to an effectiveness trial, there is a need to develop a robust method for distinguishing children with uncomplicated acute appendicitis from those with more advanced appendicitis, and to reach agreement on a primary outcome measure and effect size that is acceptable to all stakeholder groups involved. TRIAL REGISTRATION: Current Controlled Trials ISRCTN15830435. FUNDING: This project was funded by the National Institute for Health Research (NIHR) Health Technology Assessment programme and will be published in full in Health Technology Assessment; Vol. 25, No. 10. See the NIHR Journals Library website for further project information.

Appendicitis is usually treated with an operation to remove the appendix. But we have learned, from other research, that some children with appendicitis may not need an operation, and could be treated with antibiotics instead. To find out how these two different treatments compare with one another, we need to do a big study. First, though, we need to see if doing that kind of study would even be possible (or 'feasible'). We carried out a feasibility study that had several parts. First, we did a small study with children who had appendicitis, whereby children were randomly allocated to have either antibiotics or an operation, with an equal chance of having either treatment. Second, we asked parents and health-care staff about why they wanted, or did not want, to take part in that small study. This helped us to understand how to make a bigger future study as acceptable as possible to children, families and surgeons. Third, we asked parents, patients and surgeons what they think are the most important things ­ or 'outcomes' ­ we should look at in future research on children who have appendicitis. From that, we developed a list of outcomes that should be included in our future big study, so we can be certain that the research we do is likely to help parents and surgeons. Overall, we established that a future big study is feasible and we have plenty of information to help us with how to plan it best, so that it has the greatest possible chance of success. We were also guided in all of these steps of the research by a group of parents, children and young people, some of whom had appendicitis and some of whom did not.

CONservative TReatment of Appendicitis in Children: a randomised controlled feasibility Trial (CONTRACT).

OBJECTIVE: To establish the feasibility of a multicentre randomised controlled trial to assess the effectiveness and cost-effectiveness of a non-operative treatment pathway compared with appendicectomy in children with uncomplicated acute appendicitis. DESIGN: Feasibility randomised controlled trial with embedded qualitative study to inform recruiter training to optimise recruitment and the design of a future definitive trial. SETTING: Three specialist paediatric surgery centres in the UK. PATIENTS: Children (aged 4-15 years) with a clinical diagnosis of uncomplicated acute appendicitis. INTERVENTIONS: Appendicectomy or a non-operative treatment pathway (comprising broad-spectrum antibiotics and active observation). MAIN OUTCOME MEASURES: Primary outcome measure was the proportion of eligible patients recruited. Secondary outcomes evaluated adherence to interventions, data collection during follow-up, safety of treatment pathways and clinical course. RESULTS: Fifty per cent of eligible participants (95% CI 40 to 59) approached about the trial agreed to participate and were randomised. Repeated bespoke recruiter training was associated with an increase in recruitment rate over the course of the trial from 38% to 72%. There was high acceptance of randomisation, good patient and surgeon adherence to trial procedures and satisfactory completion of follow-up. Although more participants had perforated appendicitis than had been anticipated, treatment pathways were found to be safe and adverse event profiles acceptable. CONCLUSION: Recruitment to a randomised controlled trial examining the effectiveness and cost-effectiveness of a non-operative treatment pathway compared with appendicectomy for the treatment of uncomplicated acute appendicitis in children is feasible. TRIAL REGISTRATION NUMBER: ISRCTN15830435.

Pneumoperitoneum in the Setting of Pneumatosis Intestinalis in Children: Is Surgery Always Indicated?

Aim Pneumatosis intestinalis (PI) is a condition in which multiple gas-filled cysts form within the wall of the gastrointestinal tract in either the subserosa or submucosa. The presence of pneumoperitoneum in the presence of PI can present a therapeutic dilemma. The aim of our study was to review our experience and management of this condition. Methods A single-center retrospective study of consecutive children (> 1 year) presenting with a pneumoperitoneum and evidence of PI (2009-2015). Demographics, case notes, microbiology, and imaging were reviewed. Results Seven patients were identified (four males; age range 5-14 years). Four children had global developmental delay and were percutaneous endoscopic gastrostomy or jejunostomy fed, one was immunocompromised (acute lymphoblastic leukemia). The others had encephalitis and eosinophilic gastroenteritis. One patient proceeded to an exploratory laparotomy; no perforation was identified, pneumatosis of the colon was observed, and a loop ileostomy was formed. The remaining six were managed conservatively and made nil by mouth with intravenous antibiotics commenced. Five of the six had a computed tomography (CT) scan which revealed PI and free air with no other worrying signs. One died from nongastrointestinal causes, while the remaining five had feeds reintroduced uneventfully. Conclusion Free air in the setting of PI may represent rupture of the gas cysts and not always transmural perforation. Surgery may not always be indicated and conservative management may suffice. A CT scan can be useful for excluding other intra-abdominal pathological findings and continued clinical assessment is essential.

Early experience of thoracoscopic aortopexy for severe tracheomalacia in infants after esophageal atresia and tracheo-esophageal fistula repair.

BACKGROUND: Aortopexy is the most effective treatment for severe tracheomalacia associated with esophageal atresia with distal tracheo-esophageal fistula (EA/TOF). In the last few years, the thoracoscopic approach has been proposed, but the number of patients treated is limited. The purpose of this study is to review our initial experience with thoracoscopic aortopexy. PATIENTS AND METHODS: A retrospective review of medical records was performed on EA/TOF patients undergoing thoracoscopic aortopexy at the Great Ormond Street Hospital for Sick Children (London, United Kingdom) from January 2009 to May 2012. Patient demographics, indication, perioperative course, and long-term results when available were noted. RESULTS: Four patients underwent a successful thoracoscopic aortopexy, with no operation being converted. No morbidity or mortality was associated with the procedure. Length of postoperative stay ranged from 2 to 4 days. All patients were relieved of their symptoms, and no recurrence was noted. CONCLUSIONS: Thoracoscopic aortopexy is a feasible and successful treatment for severe tracheomalacia in EA/TOF patients. The complication rate may be lower than after the open procedure and is more satisfactory in terms of cosmetic appearance. However, we need a larger and prospective study with a longer follow-up to confirm these preliminary results.

Paediatric laparoscopic Heller's cardiomyotomy: a single centre series.

BACKGROUND: The optimal management of oesophageal achalasia remains unclear in the paediatric population due to the rarity of the disease. This study reviews the institutional experience of the laparoscopic Heller's cardiomyotomy (HC) procedure and attempts to define the most appropriate treatment. METHODS: A retrospective review of children undergoing HC at a single institution was performed. Demographics, pre-operative investigations, and interventions were reviewed. Post-operative outcomes and follow up were evaluated. Data is expressed as median (range). RESULTS: Twenty-eight children were included (13 male, 15 female) whose median age was 13 (3.2-17.4) years. Nine children underwent a pre-operative oesophageal balloon dilatation (OBD) a median of 1(1-6) times. Others included botulinum toxin injection (n=1) and Nifedipine (n=1). All had a pre-operative upper gastrointestinal contrast series, and twenty-five had upper gastrointestinal endoscopy and manometry. All had laparoscopic HC with no conversions, and ten had a concomitant fundoplication. Post-operative intervention occurred in eight (28%) incorporating OBD (n=7), of whom four required a redo HC. One patient underwent a redo without intervening OBD. Follow-up was for a median of 0.83 (0-5) years with fourteen children discharged from surgical follow-up. Twenty-seven have thus far had a good outcome. CONCLUSION: This study comprises the largest series of paediatric laparoscopic HC reported to date. It is effective with or without a fundoplication and is the best long term treatment modality available. OBD for persisting symptoms following HC may obviate the need for redo myotomy.

Tension gastrothorax in children: introducing a management algorithm.

Tension gastrothorax is a life-threatening condition and presents dramatically. It develops when the stomach herniates through a diaphragmatic defect into the thorax and distends with gas and/or fluid causing mediastinal shift. It is commonly misdiagnosed as tension pneumothorax which results in significant morbidity and mortality. In this article we report our experience of three cases with a spectrum of aetiology and a comprehensive literature review of tension gastrothorax in children. We propose an algorithm for the management of tension gastrothorax.

Gastroschisis with Hirschsprung's disease: a therapeutic dilemma.

The authors report a case of a neonate with gastroschisis, which, after repair, was further complicated by the diagnosis of Hirschsprung's disease. The authors discuss the diagnostic and management dilemmas posed by the coexistence of these two conditions for the clinician.