Safety, Efficacy and Health Related Quality of Life of Autologous Myoblast Transplantation for Treatment of Urinary Incontinence in Children With Bladder Exstrophy-Epispadias Complex

Purpose: Children with bladder exstrophy-epispadias complex undergoing en-dourethral autologous myoblast transplantation to treat urinary incontinence were evaluated at 4 years of followup regarding the safety, efficacy and durability of the procedure, and health related quality of life. Materials and Methods: Seven boys underwent autologous myoblast transplantation between May and December 2006. All patients had persistent urinary incontinence after bladder neck reconstruction and bulking agent injection. Patients were followed for 4 years after autologous myoblast transplantation regarding clinical outcomes and cystometric, urodynamic, uroflowmetric and urethrocystoscopic evaluations. Health related quality of life was also measured before treatment and at final followup. Results: No evidence of urinary obstruction was observed. Five children (71%) were completely continent and 2 (29%) were socially dry with complete daytime dryness at final followup. Health related quality of life was improved significantly. Urodynamic studies revealed a progressive increase in bladder capacity (p <0.001). Mean detrusor leak point pressure showed a 27 cm H2O (158%) increase during 4-year followup. Uroflowmetry parameters of voided volume and average maximum flow rate were improved significantly (p <0.001). Conclusions: The 4-year outcomes demonstrate that autologous myoblast transplantation for urinary incontinence in children with bladder exstrophy-epispadias complex is relatively reliable, reproducible, safe and effective with minimal morbidity. This novel treatment represents a promising therapeutic approach in patients with urinary incontinence. Further randomized trials with larger numbers of patients and longer followup are needed.