To evaluate whether clinical or urodynamic response to Botox reduces progression to definitive surgery (bladder augmentation/incontinent diversion) in children with myelomeningocele (MMC).

We performed a retrospective cohort study of children with MMC treated with intravesical Botox at a tertiary pediatric center. Demographics, urodynamics, bladder morphology, vesicoureteral reflux (VUR), and treatment characteristics were collected. “Botox effect” was defined as clinical or urodynamic improvement after initial injection. The primary outcome was progression to augmentation or incontinent diversion. Time-to-event analyses used Kaplan-Meier and Cox regression.

Eighty-three patients were included (median follow-up 4.8 years). Twenty-five patients (30.1%) progressed to augmentation or diversion. Five-year freedom from progression was 73.9%. Patients demonstrating a Botox effect had lower progression to augmentation or diversion compared with non-responders (5-year freedom from progression: 81.4% vs 45.8%, P < .001). On multivariable analysis, Botox effect was independently protective against progression (HR 0.23, 95% CI 0.09-0.60), while presence of VUR was associated with increased risk (HR 4.69, 95% CI 1.79-12.29). Receipt of multiple Botox injections was also associated with improved outcomes.

In children with MMC, favorable initial response to Botox is strongly associated with reduced progression to augmentation or diversion. Future prospective studies with standardized definitions of response and long-term follow-up are needed to better define which patients derive durable benefit from Botox and to refine treatment strategies.