Appendectomy Versus Non-Operative Treatment For Acute Non-Perforated Appendicitis in Children (APPY)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT02687464|
Recruitment Status : Recruiting
First Posted : February 22, 2016
Last Update Posted : December 4, 2017
Rationale and Aim: The standard treatment for acute appendicitis in children is appendectomy. An increasing body of evidence from the adult literature suggests that acute appendicitis may be treated effectively with antibiotics alone, avoiding the need for surgery. The aim of this study is to investigate the effectiveness of non-operative treatment of acute appendicitis in children.
Study design: Pragmatic, parallel-group, unmasked, non-inferiority multicentre randomized controlled trial (RCT).
Patient allocation: Children will be randomly allocated (age 5-16 years) with a diagnosis of acute appendicitis to either laparoscopic appendectomy or treatment with antibiotics. Randomization will be performed using stratification to ensure equal distribution between groups of presenting clinical and demographic features that may influence outcome including gender, duration of symptoms and center.
Interventions: One group of children will undergo laparoscopic appendectomy that is the current standard treatment for children with acute appendicitis. The other group will be treated with intravenous antibiotics. A treatment pathway specifically designed for this study will be used.
Primary Outcome: To be meaningful to parents of, and clinicians treating, children with acute appendicitis, the primary outcome is treatment failure defined as: (i) any additional intervention related to appendicitis requiring general anesthesia within 1 year of randomization (including recurrence of appendicitis after non-operative treatment, which we will treat with appendectomy) or (ii) negative appendectomy. Secondary outcomes are: (i) complications; (ii) time to discharge following randomization; (iii) number and duration of hospital admissions.
Sample size and data analysis: The proposed RCT has a 20% non-inferiority margin to test the null hypothesis that treatment with antibiotics is inferior to appendectomy. Based on data from collaborating centers and a pilot study that we have performed, we expect recruitment of 978 children in total (90% power) over 19 months allowing for drop out. Follow-up will be for 12 months.
|Condition or disease||Intervention/treatment|
|Appendicitis||Other: Non-operative treatment Procedure: Appendectomy|
Show Detailed Description
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||978 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Multicentre, Randomized Controlled Trial Comparing Appendectomy Versus Non-Operative Treatment For Acute Non-Perforated Appendicitis in Children|
|Study Start Date :||January 2016|
|Estimated Primary Completion Date :||January 2019|
|Estimated Study Completion Date :||January 2020|
Experimental: Non-operative treatment group
IV fluids, minimum 12 hrs IV antibiotics, minimum 12 hrs clear PO fluids only, regular clinical review. Discharge within 24 hrs after randomization, if study criteria met. If stable but not adequate improvement for discharge, non-operative management continues. If not improved by 48 hrs, appendectomy will be done. Discharge home once vital signs are within normal limits, light oral diet tolerated, adequate oral pain relief and mobile. Total 10 days of antibiotics (IV and oral) following randomization will be given. Antibiotics used vary between centers and will be current standard of care in that center; improving study feasibility and increased generalization of results.
Other: Non-operative treatment
will receive a minimum of 12 hours intravenous antibiotics and then receive oral antibiotics once they have shown clinical improvement (a total course of 10 days of antibiotics (intravenous and oral). They will be discharged home once they meet a standardized set of criteria to be used in all centers.
The choice of antibiotics will vary between centers and will be the antibiotic regimen that is current standard of care in that center allowing each center to maintain current protocols will improve study feasibility and increase generalization of the results.
Active Comparator: Appendectomy group
Laproscopic appendectomy within 18 hrs of randomization. IV antibiotics given from time of randomization and continued post-operatively per the standardized treatment regimen: children with visibly normal appendix or non-perforated acute appendicitis will receive no further antibiotics; children with perforated appendicitis will continue IV antibiotics for a minimum of 3 days and then per local practice. The type of antibiotics used in each center will be identical to those used in the non-operative treatment group.
will undergo laparoscopic appendectomy within 18 hours of randomization and will receive intravenous antibiotics per each site's standard care, from the time of randomization and post-operatively according to a defined and standardized treatment regimen based on consensus for this trial.
Children with a visibly normal appendix or non-perforated acute appendicitis will receive no further antibiotics; children with perforated appendicitis will continue to receive intravenous antibiotics for a minimum of 3 days, and then per local practice. The type of antibiotics used in each center will be identical to those used in the non-operative treatment group and are not specified by the study.
Other Name: Laparoscopic appendectomy
- treatment failure [ Time Frame: within 1 year of randomization ]The primary outcome is treatment failure. Treatment failure is defined as: (i) additional intervention related to appendicitis requiring general anesthesia within 1 year of randomization (this includes the recurrence of appendicitis after non-operative treatment, which will be treated with appendectomy) or (ii) negative appendectomy. This definition of the primary outcome will capture all important parameters in both treatment groups including specifically: failure of antibiotic treatment requiring appendectomy, significant complication (defined as requiring general anaesthesia) in either treatment group, recurrence of acute appendicitis (treated by appendectomy) and negative appendectomy.
- treatment efficacy [ Time Frame: within 1 year of randomization ]Secondary outcomes are objective measures of treatment efficacy that fulfil important core areas of relevance to clinicians and patients (pathophysiological manifestations, life impact, resource use and death)
- complications [ Time Frame: within 1 year of randomization ]complications: adverse events related to non-operative treatment of appendicitis or appendectomy which require additional interventions without general anesthesia, during the first year following randomization
- time to discharge [ Time Frame: 1 year from randomization ]time to discharge home after randomization measured in hours
- number and duration of hospital admissions [ Time Frame: within 1 year of randomization ]number and duration of hospital admissions related to appendicitis, appendectomy or their complications during the first year following randomization
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02687464
|Contact: Shawn D St. Peter, MDfirstname.lastname@example.org|
|Contact: Pete Muenks, MAemail@example.com|
|United States, Missouri|
|Children's Mercy Kansas City||Recruiting|
|Kansas City, Missouri, United States, 64108|
|Contact: Shawn D St. Peter, MD 816-234-3575 firstname.lastname@example.org|
|Contact: Pete Muenks, MA 816-983-6670 email@example.com|
|Principal Investigator:||Shawn D St. Peter, MD||Children's Mercy Hospital Kansas City|