Assessing micro- vs macro-costing approaches for treating appendicitis in children with appendicectomy or non-operatively.

OBJECTIVES: We conducted a health economic sub-study within a feasibility RCT comparing a non-operative treatment pathway as an alternative to appendicectomy for the treatment of uncomplicated acute appendicitis in children. The objectives were to understand and assess data collection tools and methods and to determine indicative costs and benefits assessing the feasibility of conducting a full economic evaluation within the definitive trial. METHODS: We compared different methods of estimating treatment costs including micro-costing, hospital administrative data (PLICS) and health system (NHS) reference costs. We compared two different HRQoL instruments (CHU-9D and EQ-5D-5L) in terms of data completeness and sensitivity to change over time, including potential ceiling effects. We also explored how the timing of data collection and duration of the analysis could affect QALYs (Quality Adjusted Life Years) and the results of the cost-utility analysis (CUA) within the future RCT. RESULTS: Using a micro-costing approach, the total per treatment costs were in alignment with hospital administrative data (PLICS). Average health system reference cost data (macro-costing using NHS costs) could potentially underestimate these treatment costs, particularly for non-operative treatment. Costs incurred following hospital discharge in the primary care setting were minimal, and limited family borne costs were reported by parents/carers. While both HRQoL instruments performed relatively well, our results highlight the problem of ceiling effect and the importance of the timing of data collection and the duration of the analysis in any future assessment using QALYs and CUA. CONCLUSIONS: We highlighted the importance of obtaining accurate individual-patient cost data when conducting economic evaluations. Our results suggest that timing of data collection and duration of the assessment are important considerations when evaluating cost-effectiveness and reporting cost per QALY. CLINICAL TRIAL REGISTRATION: Current Controlled Trials ISRCTN15830435.

Urodynamic Outcomes in Children after Single and Multiple Injections for Overactive and Low Compliance Neurogenic Bladder Treated with Abobotulinum Toxin A.

PURPOSE: Intradetrusor botulinum toxin is an established part of the treatment pathway for pediatric patients with neurogenic bladder. We determined the urodynamic effect of single and multiple administrations of abobotulinum toxin A in pediatric patients with neurogenic bladder, and determined the urodynamic efficacy of abobotulinum toxin A in low compliance vs overactive bladders. MATERIALS AND METHODS: We conducted a single center retrospective review of all pediatric patients with neurogenic bladder treated with abobotulinum toxin A. Videourodynamic data on cystometric capacity, maximum neurogenic detrusor overactivity pressure and compliance were gathered before and after the first abobotulinum toxin A administration and after the last administration. Patients were divided into low compliance and overactive bladder groups depending on initial videourodynamics findings. Paired t-test was used to compare videourodynamic outcomes before vs after abobotulinum toxin A injection. The Mann-Whitney U test was used to compare bladder groups. RESULTS: A total of 30 patients were included in the study. Of these patients 15 (50%) received multiple abobotulinum toxin A injections. There were 16 patients (53%) with overactive bladder. Abobotulinum toxin A administration significantly improved cystometric capacity (p <0.0001) and maximum neurogenic detrusor overactivity (p=0.0001). Overall, compliance did not change significantly (p=0.25). There was no significant difference in urodynamic parameters between first and last abobotulinum toxin A injections. Improvement in cystometric capacity (p=0.05) and maximum neurogenic detrusor overactivity (p=0.25) was similar between the low compliance and overactive bladder groups. Compliance significantly improved in the low compliance group vs the overactive bladder group (p=0.016). CONCLUSIONS: Intradetrusor abobotulinum toxin A improves cystometric capacity and maximum neurogenic detrusor overactivity in pediatric patients with neurogenic bladder. This effect is maintained over multiple injections. Compliance is significantly improved in patients with low compliance bladder vs overactive bladder.

Intra-thoracic appendicitis in a child with Down's syndrome.

Morgagni congenital diaphragmatic hernias are rare. They are associated with chromosomal anomalies, especially Down's syndrome. Acute appendicitis is the most common surgical emergency in childhood, but accurate diagnosis can be a challenge if encountered out of the usual anatomical position. This is the first case report of acute appendicitis within a Morgagni hernia in a child with Down's syndrome.