Onabotulinum toxin-A vs extended release tolterodine in the management of idiopathic overactive bladder in children: A Pilot Randomized Controlled Trial

  • Charlotte Melling

Student thesis: Doctor of Medicine


Abstract Introduction Children who fail standard anti-cholinergic therapy for idiopathic overactive bladder (IOAB) may be offered alternative oral therapies, or intravesical injection of botulinum toxin A (BtA). This thesis presents the first RCT to evaluate an oral anti-cholinergic compared with BtA for IOAB in children. An audit of current practice, alongside a description of bladder diary completion by, and urodynamic (UDS) findings in, children with IOAB are also presented. Methods A national audit of practice of UK paediatric urologists was completed. A pilot randomized controlled trial to inform on the feasibility of comparing oral anticholinergic with BtA for children with IOAB was performed. A post-hoc exploratory analysis of the subjective (bladder diaries) and objective (urodynamics) data were undertaken. Results 81% of UK paediatric urologists use BtA for children with IOAB. 98 children were enrolled in a pilot RCT, demonstrating rates of: randomization (74%) and attrition (4.3%). 48-hour frequency/volume charts were fully completed by 8%. 100% and 76% of participants accepted baseline and post-intervention UDS. Compared with tolterodine, BtA improved bladder capacity by median 38mls, reduced number of detrusor contractions by three, but increased post-void residual volumes by 52mls. There was one UTI requiring hospital treatment in the BtA group Conclusions Use of BtA in the management of IOAB in children by UK paediatric urologists is commonplace, but there are wide variations in the use of urodynamics and patient-reported outcomes. A pilot RCT to compare oral anticholinergic therapy with BtA has suggested a larger RCT is safe and feasible, with modifications. The return of subjective, patient-reported outcomes was inadequate to assess treatment response. Objective data analysis of urodynamics allowed a thorough description of urodynamic findings in children with IOAB, and a comparison of the two treatment arms. Despite trends towards cystometric improvements in bladder capacity and detrusor overactivity with BtA, there was no clear demonstration of superiority over tolterodine. As equipoise remains, an appropriately powered RCT is required to further investigate treatment options for IOAB in children.
Date of Award31 Dec 2020
Original languageEnglish
Awarding Institution
  • The University of Manchester
SupervisorAnju Goyal (Supervisor)


  • Pilot RCT
  • Urodynamics
  • Overactive bladder
  • Children
  • Anticholinergic
  • Onabotulinum toxin A
  • Tolterodine

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