Dataset Information

OnabotulinumtoxinA for the treatment of neurogenic detrusor overactivity in children.

ABSTRACT:

Aims

This study evaluated whether one (or more) of three doses of onabotulinumtoxinA were safe and effective to treat neurogenic detrusor overactivity (NDO) in children.

Methods

This was a 48-week prospective, multicenter, randomized, double-blind study in children (aged 5-17 years) with NDO and urinary incontinence (UI) receiving one onabotulinumtoxinA treatment (50, 100, or 200 U; not to exceed 6 U/kg). Primary endpoint: change from baseline in daytime UI episodes. Secondary endpoints: change from baseline in urine volume at first morning catheterization, urodynamic measures, and positive response on the treatment benefit scale. Safety was also assessed.

Results

There was a similar reduction in urinary incontinence from baseline to Week 6 for all doses (-1.3 episodes/day). Most patients reported positive responses on the treatment benefit scale (75.0%-80.5%). From baseline to Week 6, increases were observed in urine volume at first morning clean intermittent catheterization (50 U, 21.9 ml; 100 U, 34.9 ml; 200 U, 87.5 ml; p = 0.0055, 200 U vs. 50 U) and in maximum cystometric capacity (range 48.6-63.6 ml) and decreases in maximum detrusor pressure during the storage phase (50 U, -12.9; 100 U, -20.1; 200 U, -27.3 cmH₂ O; p = 0.0157, 200 U vs. 50 U). The proportion of patients experiencing involuntary detrusor contractions dropped from baseline (50 U, 94.4%; 100 U, 88.1%; 200 U, 92.6%) to Week 6 (50 U, 61.8%; 100 U, 44.7%; 200 U, 46.4%). Safety was similar across doses; urinary tract infection was most frequent.

Conclusions

OnabotulinumtoxinA was well tolerated and effective for the treatment of NDO in children; 200 U showed greater efficacy in reducing bladder pressure and increasing bladder capacity.

SUBMITTER: Austin PF

PROVIDER: S-EPMC7839517 | biostudies-literature | 2021 Jan

REPOSITORIES: biostudies-literature

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Publications

OnabotulinumtoxinA for the treatment of neurogenic detrusor overactivity in children.

Austin Paul F PF Franco Israel I Dobremez Eric E Kroll Pawel P Titanji Wilson W Geib Till T Jenkins Brenda B Hoebeke Piet B PB

Neurourology and urodynamics 20201211 1

<h4>Aims</h4>This study evaluated whether one (or more) of three doses of onabotulinumtoxinA were safe and effective to treat neurogenic detrusor overactivity (NDO) in children.<h4>Methods</h4>This was a 48-week prospective, multicenter, randomized, double-blind study in children (aged 5-17 years) with NDO and urinary incontinence (UI) receiving one onabotulinumtoxinA treatment (50, 100, or 200 U; not to exceed 6 U/kg). Primary endpoint: change from baseline in daytime UI episodes. Secondary end ...[more]

PMID: 33305474

Similar Datasets

Project description:ObjectiveTo evaluate the effects of onabotulinumtoxinA on patient-reported outcomes including health-related quality of life (HRQOL), treatment satisfaction, and treatment goal attainment in patients with urinary incontinence (UI) due to neurogenic detrusor overactivity (NDO).MethodsIn this multicenter, double-blind, randomized, placebo-controlled, phase III, 52-week study (ClinicalTrials.gov NCT00311376), patients with UI due to NDO who were not adequately managed with anticholinergic therapy were treated with intradetrusor injections of onabotulinumtoxinA (200 or 300 U) or placebo (0.9% saline). HRQOL measures included the Incontinence Quality of Life (I-QOL) Questionnaire total score, and the 3 domain scores (avoidance and limiting behavior, psychosocial, and social embarrassment), the modified Overactive Bladder Patient Satisfaction with Treatment Questionnaire (OAB-PSTQ), and Patient Global Assessment. Assessments were made at baseline, posttreatment week 6 (primary time point), week 12, and at 12-week intervals.ResultsPatients (mean age of 46 years with 30.5 weekly UI episodes at baseline) were randomized to receive placebo (n = 149) or onabotulinumtoxinA (200 U [n = 135] or 300 U [n = 132]). At week 6, improvements from baseline in I-QOL Questionnaire total score were greater (p < 0.001) in both onabotulinumtoxinA-treated groups vs placebo. Responses to the OAB-PSTQ also demonstrated greater mean improvements from baseline (p < 0.001) in both onabotulinumtoxinA-treated groups vs placebo at week 6. Patients who received onabotulinumtoxinA also reported greater improvement in the Patient Global Assessment than those in the placebo group (p ≤ 0.001 vs placebo).ConclusionsPatients with UI due to NDO reported greater improvement in HRQOL and treatment satisfaction with onabotulinumtoxinA than with placebo consistently across several patient-reported outcome instruments.Classification of evidenceThis study provides Class I evidence that onabotulinumtoxinA intradetrusor injections (200 or 300 U) can improve quality of life measures in patients with NDO not adequately managed with anticholinergic therapy.

Project description:BackgroundNeurogenic detrusor overactivity (NDO) affects the quality of life (QoL) of millions of individuals worldwide. The purpose of this study was to assess the efficacy and safety of onabotulinumtoxinA in patients with NDO using a network meta-analytic approach, which can also quantify and compare the efficacy of onabotulinumtoxinA across different dosages.MethodsPubMed, EMBASE, and the Controlled Trials Register were searched to identify randomized controlled trials comparing onabotulinumtoxinA to a control for NDO in adult patients. The primary outcome was the mean number of urinary incontinence (UI) episodes per week. Urodynamic parameters included the maximum cystometric capacity (MCC) and the maximum detrusor pressure (MDP). The safety of onabotulinumtoxinA was determined by the incidence of various frequent adverse events (AEs). Two authors extracted data independently, and the statistical analyses were performed using RevMan 5.1.0 software.ResultsA total of 1,915 patients from six randomized controlled trials were included in this meta-analysis. The onabotulinumtoxinA-treated groups had a significantly decreased mean number of urinary incontinence episodes per week (at week 6) (onabotulinumtoxinA200U: MD: -10.72, 95% CI: -13.4 to -8.04, P<0.00001; 300 U: MD: -11.42, 95% CI: -13.91 to -8.93, P<0.00001), MDP (200 U: MD: -33.46, 95% CI: -39.74 to -27.18, P<0.00001; 300 U: MD: -31.72, 95% CI: -37.69 to -25.75, P<0.00001), and greater increased MCC (200 U: MD: 141.30, 95% CI: 121.28 to 161.32, P<0.00001; 300 U: MD: 151.39, 95% CI: 130.43 to 172.34, P<0.00001) compared to the placebo-treated groups. However, there were no significant differences between the onabotulinumtoxinA-treated groups for the number of weekly UI episodes at 6 weeks (MD: 0.08, 95% CI: -2.57 to 2.73, P = 0.95). Similarly, we also observed that there were no significant differences in MCC (MD: -9.97, 95% CI: -33.15 to 13.20, P = 0.40) and MDP (MD: -1.86, 95% CI: -8.09 to 4.37, P = 0.56). Considering the AEs, the onabotulinumtoxinA-treated groups were often associated with more complications, including urinary tract infections (UTIs) (RR: 1.47, 95% CI: 1.29 to 1.67, P<0.00001), urinary retention (RR: 5.58, 95% CI: 3.53 to 8.83, P<0.00001), hematuria (RR: 1.70, 95% CI: 1.01 to 2.85, P = 0.05), and muscle weakness (RR: 2.59, 95% CI: 1.36 to 4.91, P = 0.004).ConclusionsOnabotulinumtoxinA can significantly reduce the frequency of urge urinary incontinence and improve urodynamic parameters (MCC and MDP) in patients with NDO at 6 weeks after treatment. This meta-analysis indicates that onabotulinumtoxinA is effective and safe for treating patients with NDO compared to placebo. Additionally, we did not observe any statistical or clinical differences in efficacy between 300 and 200 U dosages.

Project description:IntroductionThe COVID-19 pandemic has imposed an additional pressure on health systems worldwide. Patients with neurogenic detrusor overactivity (NDO) were especially vulnerable to inadequate care. This study aims to evaluate the impact of the suspension of NDO treatment with Botulinum Toxin (BONT-A) due to the COVID-19 pandemic.MethodsCross-sectional study of patients with spinal cord injury and NDO, who underwent BONT-A treatment in 2018 or 2019 and, whose administration programed for 2020 or 2021 was suspended. The study protocol was divided into two parts. Phase 1 consisted of data collection from the clinical processes and in phase 2 a standardized telephone questionnaire was applied. Information was collected at 3 time points: (1) before the last BONT-A treatment, (2) after the last BONT-A treatment and (3) at the time of the telephone call. Statistical analysis used the McNemar and the Wilcoxon test with a p-value ⩽ 0.05 as level of significance.Results21 patients with mean age of 42.0 years and disease duration of 16.9 years were included. On average patients were undergoing treatment with BONT-A for 7.6 years and mean inter-treatment frequency was 2.3 years. Mean time since the last BONT-A administration was 2.3 years and mean reported BONT-A effect duration was 11.9 months. A significant increase in the percentage of patients with involuntary urinary loss (p = 0.004) and urgency (p = 0.031) was found. A significant decrease in mean catheterization interval from 4.5 to 3.6 h (p = 0.002) and an increase in daily oxybutynin dosage from 8.5 to 12.1 mg (p = 0.002) was also found.DiscussionThe COVID-19 pandemic originated clinical worsening of patients undergoing regular BONT-A treatment for NDO. These patients presented a significant increase in involuntary urinary loss, urgency and medication dosage and a decrease in catheterization interval. Thus, interruption of intravesical BONT-A treatment severely affected these patients and needs to be avoided.

Dataset Information

OnabotulinumtoxinA for the treatment of neurogenic detrusor overactivity in children.

Aims

Methods

Results

Conclusions

Publications

OnabotulinumtoxinA for the treatment of neurogenic detrusor overactivity in children.

Similar Datasets

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