The addition of mirabegron in pediatric lower urinary tract dysfunction: A large single-center experience.

OBJECTIVE: To study the efficacy and safety of mirabegron, beta-3 adrenergic agonist, in a large real-world sample of pediatric patients with neurogenic bladder or non-neurogenic lower urinary tract dysfunction as monotherapy or concurrent medication. METHODS: We reviewed children who were prescribed mirabegron for any indication (neurogenic bladder, non-neurogenic lower urinary tract dysfunction, among others) between 2018 and 2024 at our institution. We reviewed data on treatment indications, concurrent treatments, side effects, and efficacy for individual symptoms and overall improvement. Symptoms and side effects were determined from clinical documentation at follow-up encounters after mirabegron initiation. We assessed time-to-improvement and performed univariate and multivariable Cox regression to identify covariates that affect efficacy. RESULTS: In total, 192 patients including 101 (53%) females at a median age of 10 years (IQR 7, 14) were prescribed mirabegron and median follow-up was 12 months (IQR 6, 22). Most common indications included non-neurogenic lower urinary dysfunction (46%), and neurogenic bladder (42%). Side effects occurred in 25 patients (13%), including headaches in 7 patients (4%), nausea in 7 patients (4%), and hypertension in 3 patients (2%). Within the population, 124 (65%) of patients had partial improvement or greater in their symptoms with a median time-to-improvement of 7 months. Age at diagnosis, sex, indication for treatment, comorbid behavioral conditions, or concurrent medications were not found to be predictive of symptom improvement within the sample. CONCLUSIONS: Mirabegron appears to be a safe and effective treatment for pediatric patients and should be considered as mono-therapy or in addition to other anticholinergic medication in lower urinary tract dysfunction. CLINICAL TRIAL REGISTRATION: Not required, as this is an observational study.