TY - JOUR
T1 - Appendectomy versus non-operative treatment for acute uncomplicated appendicitis in children
T2 - Study protocol for a multicentre, open-label, non-inferiority, randomised controlled trial
AU - Hall, Nigel J.
AU - Eaton, Simon
AU - Abbo, Olivier
AU - Arnaud, Alexis P.
AU - Beaudin, Marianne
AU - Brindle, Mary
AU - Bütter, Andreana
AU - Davies, Dafydd
AU - Jancelewicz, Tim
AU - Johnson, Kathy
AU - Keijzer, Richard
AU - Lapidus-Krol, Eveline
AU - Offringa, Martin
AU - Piché, Nelson
AU - Rintala, Risto
AU - Skarsgard, Erik
AU - Svensson, Jan F.
AU - Ungar, Wendy J.
AU - Wester, Tomas
AU - Willan, Andrew R.
AU - Zani, Augusto
AU - St Peter, Shawn D.
AU - Pierro, Agostino
N1 - Publisher Copyright:
© Article author(s) (or their employer(s) unless otherwise stated in the text of the article) 2017. All rights reserved.
PY - 2017/12
Y1 - 2017/12
N2 - 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 nonoperative 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.
AB - 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 nonoperative 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.
UR - http://www.scopus.com/inward/record.url?scp=85034598685&partnerID=8YFLogxK
U2 - 10.1136/bmjpo-2017-000028
DO - 10.1136/bmjpo-2017-000028
M3 - Article
AN - SCOPUS:85034598685
SN - 2399-9772
VL - 1
JO - BMJ Paediatrics Open
JF - BMJ Paediatrics Open
IS - 1
M1 - Y
ER -