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Development of a core outcome set to determine the overall treatment success of acute uncomplicated appendicitis in children: a study protocol.

ABSTRACT: Introduction:In recent years, there has been growing interest in alternatives to appendicectomy. In particular, non-operative treatment of appendicitis, with antibiotics alone, has been proposed as a potential treatment. A small number of randomised controlled trials (RCTs) in adults and, more recently, children suggest that antibiotic treatment may be a valid alternative to appendicectomy. However, there is currently insufficient data to justify its widespread use. Prior to performing further efficacy studies of the treatment of appendicitis in children, it is imperative to identify the most relevant outcome measures for inclusion in the design of comparative studies. This is of particular importance when evaluating a novel treatment approach since the outcomes of importance may differ from those commonly reported with traditional therapies.A review of the relevant literature and electronic resources failed to identify a core outcome set (COS) for children with appendicitis. We aim to define a COS for the measurement of treatment interventions in children (<18 years) with acute appendicitis. Methods and analysis:This project will entail: (1) a systematic review to identify previously reported acute uncomplicated appendicitis treatment outcomes; (2) assembly of stakeholder panels (paediatric and adult surgeons, patients and parents); (3) a three-stage Delphi process; and (4) a final consensus meeting to complete the COS. Ethics and registration:COS development is part of CONservative TReatment of Appendicitis in Children - a randomised controlled Trial (Feasibility) (CONTRACT) study, for which full ethical approval for CONTRACT has been granted. The COS development study is registered with the COMET Initiative in May 2017 (http://www.comet-initiative.org/studies/details/987).

SUBMITTER: Sherratt FC

PROVIDER: S-EPMC5862231 | biostudies-literature | 2017

REPOSITORIES: biostudies-literature

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Development of a core outcome set to determine the overall treatment success of acute uncomplicated appendicitis in children: a study protocol.

Sherratt Frances C FC Eaton Simon S Walker Erin E Beasant Lucy L Blazeby Jane M JM Young Bridget B Crawley Esther E Wood Wendy W WW Hall Nigel J NJ

BMJ paediatrics open 20170811 1

<h4>Introduction</h4>In recent years, there has been growing interest in alternatives to appendicectomy. In particular, non-operative treatment of appendicitis, with antibiotics alone, has been proposed as a potential treatment. A small number of randomised controlled trials (RCTs) in adults and, more recently, children suggest that antibiotic treatment may be a valid alternative to appendicectomy. However, there is currently insufficient data to justify its widespread use. Prior to performing f ...[more]

PMID: 29637158

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Project description:ImportanceNonoperative management with antibiotics alone has the potential to treat uncomplicated pediatric appendicitis with fewer disability days than surgery.ObjectiveTo determine the success rate of nonoperative management and compare differences in treatment-related disability, satisfaction, health-related quality of life, and complications between nonoperative management and surgery in children with uncomplicated appendicitis.Design, setting, and participantsMulti-institutional nonrandomized controlled intervention study of 1068 children aged 7 through 17 years with uncomplicated appendicitis treated at 10 tertiary children's hospitals across 7 US states between May 2015 and October 2018 with 1-year follow-up through October 2019. Of the 1209 eligible patients approached, 1068 enrolled in the study.InterventionsPatient and family selection of nonoperative management with antibiotics alone (nonoperative group, n = 370) or urgent (≤12 hours of admission) laparoscopic appendectomy (surgery group, n = 698).Main outcomes and measuresThe 2 primary outcomes assessed at 1 year were disability days, defined as the total number of days the child was not able to participate in all of his/her normal activities secondary to appendicitis-related care (expected difference, 5 days), and success rate of nonoperative management, defined as the proportion of patients initially managed nonoperatively who did not undergo appendectomy by 1 year (lowest acceptable success rate, ≥70%). Inverse probability of treatment weighting (IPTW) was used to adjust for differences between treatment groups for all outcome assessments.ResultsAmong 1068 patients who were enrolled (median age, 12.4 years; 38% girls), 370 (35%) chose nonoperative management and 698 (65%) chose surgery. A total of 806 (75%) had complete follow-up: 284 (77%) in the nonoperative group; 522 (75%) in the surgery group. Patients in the nonoperative group were more often younger (median age, 12.3 years vs 12.5 years), Black (9.6% vs 4.9%) or other race (14.6% vs 8.7%), had caregivers with a bachelor's degree (29.8% vs 23.5%), and underwent diagnostic ultrasound (79.7% vs 74.5%). After IPTW, the success rate of nonoperative management at 1 year was 67.1% (96% CI, 61.5%-72.31%; P = .86). Nonoperative management was associated with significantly fewer patient disability days at 1 year than did surgery (adjusted mean, 6.6 vs 10.9 days; mean difference, -4.3 days (99% CI, -6.17 to -2.43; P < .001). Of 16 other prespecified secondary end points, 10 showed no significant difference.Conclusion and relevanceAmong children with uncomplicated appendicitis, an initial nonoperative management strategy with antibiotics alone had a success rate of 67.1% and, compared with urgent surgery, was associated with statistically significantly fewer disability days at 1 year. However, there was substantial loss to follow-up, the comparison with the prespecified threshold for an acceptable success rate of nonoperative management was not statistically significant, and the hypothesized difference in disability days was not met.Trial registrationClinicalTrials.gov Identifier: NCT02271932.

Project description:BACKGROUND:Urinary tract infections (UTIs) are the second most common infection presenting in the community. Clinical guidelines and decision aids assist health practitioners to treat a UTI; however, treatment practices vary due to patient needs and context of presentation. Numerous trials have evaluated the effectiveness of treatment interventions for UTI; however, it is difficult to compare the results between trials due to inconsistencies between reported outcomes. Poor choice of outcome measures can lead to impairment of evidence synthesis due to the inability to compare outcomes between trials with similar aims. Transparency in selecting and reporting outcomes can be mitigated through the development of an agreed minimum set of outcomes that should be reported in clinical trials, referred to as a core outcome set (COS). This paper presents the protocol for the development of a COS for interventions in the treatment of uncomplicated UTI in adults. METHODS:This COS development consists of three phases. Phase 1 is a systematic review, which aims to identify the core outcomes that have been reported in trials and systematic reviews of interventions treating uncomplicated UTI in adults. Phase 2 consists of a three-round online Delphi survey with stakeholders in the area of treatment interventions for UTI. The aim of this online Delphi survey is to achieve consensus on the importance of the outcomes emerging from Phase 1 of this research. Phase 3 is a consensus meeting to finalise the COS that should be reported in trials evaluating the effectiveness of interventions for the treatment of UTI. DISCUSSION:It is hoped that the development of a COS for interventions for the treatment of uncomplicated UTI in adults will be adopted as a minimum set of outcomes that should be reported and measured within this context. If the findings from clinical trials related to treatment interventions for UTI are to impact on policy and practice, it is important that the findings from different treatment interventions are comparable across trials.

Project description:INTRODUCTION:This article presents an overview of a prospective randomised controlled non-inferiority study designed to evaluate the safety and effectiveness of non-operative management (NOM) with operative management in children with acute uncomplicated appendicitis (AUA). Here, we present the study protocol for this APRES study, a multicentre Australian study. The rationale and details of future analysis, in particular, non-inferiority calculations, cost-effectiveness, feasibility and acceptability of each intervention. DESIGN:A multicentre, prospective randomised controlled clinical trial, conducted in 2 Australian tertiary paediatric hospitals. PARTICIPANTS:Children who meet the inclusion criteria of an age between 5 and 15?years and a clinical diagnosis of AUA will be invited to participate, and after consent will be randomised via a computer-based program into treatment groups. The study started in June 2016, and the target recruitment is 220 patients. INTERVENTIONS:Children in the control group will be treated with prophylactic antibiotics and appendicectomy, and those in the intervention group will be treated with antibiotic therapy alone. Primary outcome measures include unplanned or unnecessary operation and complications at 30?days. Secondary outcomes include longer term complications within 1 year, length of stay, time off work and school analgesic requirements and cost. ANALYSIS:Data analyses will be on the intention-to-treat principle using non-inferiority analysis. Analysis will include the Pearson ?2 test for categorical variables and independent sample t-test or Mann-Whitney test for continuous variables. Non-inferiority for NOM will be tested using 1-sided Wald tests with an ? level of 0.05. ETHICS AND DISSEMINATION:The research has been approved by the Human Research Ethics Committee of the Sydney Children's Hospital Network. In addition, results will be reported through academic journals, seminars and conference presentations. TRIAL REGISTRATION NUMBERS:NCT02795793; ACTRN12616000788471.

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Development of a core outcome set to determine the overall treatment success of acute uncomplicated appendicitis in children: a study protocol.

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Development of a core outcome set to determine the overall treatment success of acute uncomplicated appendicitis in children: a study protocol.

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