Electronic Thesis and Dissertation Repository

Degree

Master of Science

Program

Epidemiology and Biostatistics

Supervisor

Dr George Rodrigues

Abstract

Research question

Is it feasible to conduct a phase III RCT to compare OnabotulinumtoxinA injections to oxybutynin as primary therapy in pediatric neurogenic bladder?

Methods

Patients on a stable oxybutynin regimen were recruited for a pilot RCT and underwent randomization to either OnabotulinumtoxinA or continuation of oxybutynin. Primary outcomes included an a priori defined feasibility and acceptability assessment. Secondary outcomes included continence, urodynamic parameters, side effects and QOL.

Results

The study enrolled 8 subjects in the OnabotulinumtoxinA group and 6 in the oxybutynin group. The recruitment rate was 75 % and the dropout rate was 6.6 %. There were 2 minor protocol deviations. There were no side effects in the botulinum group compared to 66.7% in the oxybutynin group (p=0.02). The clinical and QOL outcomes were comparable.

Conclusion

It is feasible and safe to conduct a phase III trial to investigate the efficacy of primary OnabotulinumtoxinA compared to oxybutynin therapy.

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