Hall, Nigel, Sherratt, Frances C., Eaton, Simon, Reading, Isabel, Walker, Erin, Chorozoglou, Maria, Beasant, Lucy, Wood, Wendy, Stanton, Michael, Corbett, Harriet, Rex, Dean, Hutchings, Natalie, Dixon, Elizabeth, Grist, Simon, Van't Hoff, William, Crawley, Esther M., Blazeby, Jane M. and Young, Bridget (2021) Conservative treatment for uncomplicated appendicitis in children: the CONTRACT feasibility study, including feasibility RCT. Health Technology Assessment, 25 (10). (doi:10.3310/hta25100).
Abstract
Background
Whilst non-operative treatment is known to be effective for the treatment of uncomplicated acute appendicitis in children, comparative randomised trial data reporting important outcomes compared to appendicectomy are lacking.
Objectives
To ascertain the feasibility of conducting a multi-centre randomised controlled trial (RCT) testing the effectiveness and cost-effectiveness of a non-operative treatment pathway compared to appendicectomy for the treatment of uncomplicated acute appendicitis in children.
•Design
Mixed methods study including: a feasibility RCT; embedded and parallel qualitative and survey studies; parallel health economic feasibility study; development of a core outcome set.
Setting
Three specialist NHS Paediatric Surgical Units in England
Participants
Children (aged 4-15 years) clinically diagnosed with uncomplicated acute appendicitis participated in the feasibility RCT. Children, their families, recruiting clinicians and other healthcare 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 core outcomes set development.
Interventions
Participants in the feasibility RCT were randomised to a non-operative treatment pathway (broad-spectrum antibiotics and active observation) or appendicectomy.
Main outcome measures
Primary outcome measure was the proportion of eligible patients recruited to the feasibility trial.
Data sources
NHS casenotes, questionnaire responses, transcribed audio recordings of recruitment discussions and qualitative interviews
Results
Overall, 50% (95%CI 40-59) of 115 eligible participants 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 rates of 89%, 85% and 85% at six weeks, three months and six 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; design and deliver bespoke training to optimise recruitment; and understand how to optimise 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, and has 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
Since the feasibility RCT was only performed in three centres we cannot guarantee successful recruitment across a larger number of sites. However, our 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 moved over time.
Conclusions
A future effectiveness trial is feasible following limited additional preparation to establish appropriate outcome measures and case identification. We recommend a limited package of qualitative work be included to optimise recruitment at new centres in particular.
Future work
Prior to proceeding to an effectiveness trial we need to: develop a robust method for distinguishing children with uncomplicated acute appendicitis from those with more advanced appendicitis; reach agreement on a primary outcome measure and effect size that is acceptable to all stakeholder groups involved.
Study registration
ISRCTN15830435.
Funding details
NIHR HTA programme
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