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Intradetrusor Injections of Botulinum Toxin Type A in Children With Spina Bifida: A Multicenter Study
Juliette Hascoet1, Benoit Peyronnet1, Véronique Forin2
1Department of Urology, University of Rennes, Rennes, France.
Insights
Intradetrusor injections of botulinum toxin type A (IDBTX-A) showed a 66% clinical success rate in children with spina bifida, but a low 30% global success rate due to poor urodynamic outcomes.
Area of Science:
- Pediatric Urology
- Neurology
- Pharmacology
Background:
- Spina bifida often leads to neurogenic detrusor overactivity (NDO) and urinary incontinence in children.
- Management of NDO in this population frequently involves clean intermittent catheterization (CIC) and anticholinergic medications.
- Intradetrusor injections of botulinum toxin type A (IDBTX-A) are an alternative treatment option for refractory NDO.
Purpose of the Study:
- To evaluate the effectiveness of IDBTX-A in pediatric patients with spina bifida.
- To determine the clinical and urodynamic success rates of IDBTX-A.
- To identify factors predictive of successful IDBTX-A treatment.
Main Methods:
- Retrospective study of 53 children (<16 years) treated with IDBTX-A between 2002 and 2016 across 6 institutions.
- Primary endpoint: success defined by both clinical (no incontinence, urgency, <8 CICs/day) and urodynamic (resolved detrusor overactivity, normal compliance) improvement lasting ≥12 weeks.
- Univariate analysis was used to assess predictive factors for success.
Main Results:
- The overall success rate for the first IDBTX-A injection was 30%.
- Clinical success was achieved in 66% of patients, significantly associated with maximum urethral closure pressure.
- Urodynamic success was 34%; maximum cystometric capacity and compliance improved, while detrusor pressure tended to decrease.
Conclusions:
- IDBTX-A provided significant clinical improvement in 66% of pediatric spina bifida patients but demonstrated poor urodynamic outcomes.
- The overall global success rate was low at 30%, highlighting limitations in achieving complete resolution of NDO.
- A substantial proportion of patients (43.4%) eventually required augmentation cystoplasty, and 38.3% continued with botulinum toxin injections long-term.
Objective:
To assess the effectiveness of intradetrusor injections of botulinum toxin type A (IDBTX-A) in children with spina bifida.
Methods:
All patients aged less than 16 years old who underwent IDBTX-A between 2002 and 2016 at 6 institutions were included in a retrospective study. Our primary endpoint was the success rate of IDBTX-A defined as both clinical improvement (no incontinence episodes between clean intermittent catheterizations [CICs], absence of urgency, and less than 8 CICs per day) and urodynamic improvement (resolution of detrusor overactivity and normal bladder compliance for age) lasting ≥12 weeks. Predictive factors of success were assessed through univariate analysis.
Results:
Fifty-three patients with a mean age of 8.5 years were included. All patients were under CIC and 88.7% had received anticholinergics with either poor efficacy or bothersome adverse events. The global success rate of the first injection (clinical and urodynamic) was 30%. Patients with closed spinal dysraphism had a significantly better success rate than patients with myelomeningocele (P = .002). The clinical success rate was 66% and was significantly associated with maximum urethral closure pressure (34 cm H2O vs 54.4 cm H2O, P = .02). The urodynamic success rate was 34%. Maximum cystometric capacity (P <.0001) and compliance (P = .01) significantly improved after the first IDBTX-A and maximum detrusor pressure tended to decrease (P = .09) except in the subgroup of patients with poor compliance. After a mean follow-up of 3.7 years, 23 patients (43.4%) required augmentation cystoplasty. Excluding 6 patients who were lost to follow-up, 38.3% of patients were still undergoing botulinum toxin injections at last follow-up.
Conclusion:
In this series, despite the fact that IDBTX-A enabled clinical improvement in 66% patients, urodynamic outcomes were poor resulting in a low global success rate (30%).
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