Efficacy and safety of mirabegron for the treatment of bladder dysfunction in the pediatric population: A prospective observational study

Standard treatments for paediatric lower urinary tract dysfunction, including anticholinergics and intravesical botulinum toxin, may be limited by adverse effects and suboptimal response. Mirabegron, a β3-adrenergic receptor agonist, offers an alternative mechanism of action; however, paediatric evidence remains limited. This study aimed to evaluate its safety and clinical efficacy.

A prospective observational study included patients treated with mirabegron between November 2023 and November 2024, with follow-up until November 2025. Mirabegron was used as second- or third-line therapy. Clinical response was assessed based on changes in urinary symptoms (frequency, urgency, nocturnal enuresis, and daytime urinary incontinence) and categorised as complete improvement (resolution), partial improvement (reduction), or no change. Adverse events were recorded. In a subgroup, urodynamic parameters were assessed before and after treatment.

Fifteen patients were included (mean age 12 years), with a mean treatment duration of 12 months. Clinical improvement was observed in 78% of patients. Paired urodynamic studies were available in seven patients, showing significant improvements in maximum cystometric capacity, bladder compliance, and detrusor pressure during filling. Two patients experienced mild adverse events. One patient discontinued treatment due to difficulty swallowing the tablet. No clinically relevant cardiovascular changes were observed.

Mirabegron demonstrated a favourable safety profile and was associated with clinically meaningful improvement in symptoms and urodynamic parameters, supporting its use in selected paediatric patients with lower urinary tract dysfunction.