title: Prospective Pilot Study of Mirabegron in Pediatric Patients with Overactive Bladder.
abstract: Antimuscarinics are the pharmacologic mainstay of overactive bladder (OAB) management, but side effects limit their use. Mirabegron, a new molecule with a distinct mechanism of action (β3-adrenoreceptor agonist), was recently approved as monotherapy for idiopathic OAB in adults but has not been studied in the pediatric population.
  
  
  
  To evaluate the efficacy and safety of mirabegron to treat urinary incontinence in children with idiopathic OAB who were refractory to and/or intolerant of antimuscarinics.
  
  
  
  A prospective off-label study using mirabegron was conducted. Pediatric patients without symptom improvement under behavioral and medical therapies and/or with significant side effects with at least two different antimuscarinic agents were recruited.
  
  
  
  Our primary outcome was better reported efficacy than with the use of prior anticholinergic medication. Secondary end points were tolerability, safety, and satisfaction. Efficacy and tolerability were assessed with voiding diaries, postvoid residuals, urine cultures, electrocardiogram, and vital signs. Families were questioned for continence, side effects, compliance, and Patient Perception of Bladder Condition (PPBC) questionnaire. The Wilcoxon rank sum test and Wilcoxon signed rank test were used for statistical analysis.
  
  
  
  A total of 58 patients were recruited at a median age of 10.1 yr and were on mirabegron for a median of 11.5 mo. Median bladder capacity improved from 150ml to 200ml (p<0.001). Continence improved in 52 of 58, with 13 being completely dry. Median PPBC improved from 4.0 to 2.0 (p<0.001). Eight patients reported mild or moderate side effects. Absence of a placebo group is a limitation of the study.
  
  
  
  Mirabegron, a novel first-in-class therapy, appeared as a safe and effective alternative for children with idiopathic OAB refractory to antimuscarinics.
  
  
  
  We evaluated the efficacy and safety of mirabegron to treat incontinence in pediatric patients. Continence, median voided volumes, and quality of life were improved after the introduction of mirabegron, and few side effects were reported.
authors.affiliation: Division of Urology, CHU de Québec-Université Laval, Québec, Québec, Canada.
authors.forename: Anne-Sophie
authors.initials: AS
authors.lastname: Blais
authors.affiliation: Division of Urology, CHU de Québec-Université Laval, Québec, Québec, Canada.
authors.forename: Geneviève
authors.initials: G
authors.lastname: Nadeau
authors.affiliation: Division of Urology, CHU de Québec-Université Laval, Québec, Québec, Canada.
authors.forename: Katherine
authors.initials: K
authors.lastname: Moore
authors.affiliation: Division of Urology, CHU de Québec-Université Laval, Québec, Québec, Canada.
authors.forename: Lucie
authors.initials: L
authors.lastname: Genois
authors.affiliation: Division of Urology, CHU de Québec-Université Laval, Québec, Québec, Canada. Electronic address: stephane.bolduc@fmed.ulaval.ca.
authors.forename: Stéphane
authors.initials: S
authors.lastname: Bolduc
mesh_terms: D000083:Acetanilides / Q000009:adverse effects / Q000627:therapeutic use*; D000293:Adolescent; D058667:Adrenergic beta-3 Receptor Agonists / Q000009:adverse effects / Q000627:therapeutic use*; D002648:Child; D005260:Female; D006801:Humans; D008297:Male; D018727:Muscarinic Antagonists / Q000627:therapeutic use; D056687:Off-Label Use; D017060:Patient Satisfaction; D010865:Pilot Projects; D011446:Prospective Studies; D019233:Retreatment; D011795:Surveys and Questionnaires; D013844:Thiazoles / Q000009:adverse effects / Q000627:therapeutic use*; D017211:Treatment Failure; D053201:Urinary Bladder, Overactive / Q000150:complications / Q000188:drug therapy*; D014549:Urinary Incontinence / Q000209:etiology
chemical_list: D000083:Acetanilides; D058667:Adrenergic beta-3 Receptor Agonists; D018727:Muscarinic Antagonists; D013844:Thiazoles; C520025:mirabegron
keywords: Anticholinergic; Children; Mirabegron; Overactive bladder; β-3 Agonist