Hospital de São João, Porto, Portugal.
i3S Institute of Health Research and Innovation, University of Porto, Porto, Portugal.
J Med Econ. 2023 Jan-Dec;26(1):200-207. doi: 10.1080/13696998.2023.2165366.
To compare the efficacy and safety of abobotulinumtoxinA (aboBoNT-A) and onabotulinumtoxinA (onaBoNT-A) for the treatment of refractory neurogenic detrusor overactivity (NDO), using an indirect treatment comparison (ITC).
A systematic literature review was used to identify randomized controlled trials (RCTs) that evaluated botulinum toxin type A for the treatment of refractory NDO. Treatments were compared using a Bucher ITC approach. Efficacy outcomes were reduction in number of weekly urinary incontinence (UI) episodes at 6, 12, and 24 weeks of follow-up. The safety outcome was the proportion of patients with treatment-emergent urinary tract infections (TE-UTIs) during follow-up. Subgroup/sensitivity analyses were performed to investigate the impact of heterogeneity.
Fifteen studies of botulinum toxin type A were identified. Among these, onaBoNT-A 200 U was the only botulinum toxin type A considered an appropriate comparator for aboBoNT-A 600 U and 800 U. As such, six RCTs that evaluated onaBoNT-A or aboBoNT-A were included in the ITC. In base-case analyses, there were no statistically significant differences between aboBoNT-A and onaBoNT-A in terms of UI episodes or TE-UTIs. Numerically, the trend favored aboBoNT-A (either dose) for all endpoints and time points. At 12 and 24 weeks, the difference in reduction of UI episodes per week was considered clinically relevant when comparing aboBoNT-A 800 U with onaBoNT-A 200 U, but not when comparing the lower dose of aboBoNT-A (600 U) with onaBoNT-A 200 U. Results from subgroup/sensitivity analyses were consistent with the base case.
Heterogeneity across studies was observed; however, strong consistency of trends across analyses suggests the impact of heterogeneity is low.
There may be potential advantages of aboBoNT-A over onaBoNT-A, in terms of UI reduction, in patients with refractory NDO. More confirmatory studies are needed owing to the sparsity of current evidence.
使用间接治疗比较(ITC)比较阿博特毒素 A(aboBoNT-A)和奥那博毒素 A(onaBoNT-A)治疗难治性神经源性逼尿肌过度活动(NDO)的疗效和安全性。
系统文献综述用于确定评估 A 型肉毒毒素治疗难治性 NDO 的随机对照试验(RCT)。使用 Bucher ITC 方法比较治疗方法。疗效结局是在随访的 6、12 和 24 周时每周尿失禁(UI)发作次数的减少。安全性结局是在随访期间出现治疗后尿路感染(TE-UTI)的患者比例。进行亚组/敏感性分析以调查异质性的影响。
确定了 15 项 A 型肉毒毒素的研究。其中,onaBoNT-A 200 U 是唯一一种被认为适合与 aboBoNT-A 600 U 和 800 U 进行比较的肉毒毒素 A。因此,纳入了 6 项评估 onaBoNT-A 或 aboBoNT-A 的 RCT 进行 ITC。在基础分析中,aboBoNT-A 和 onaBoNT-A 在 UI 发作或 TE-UTI 方面没有统计学上的显著差异。从数值上看,趋势有利于 aboBoNT-A(任何剂量)在所有终点和时间点。在 12 和 24 周时,当比较 aboBoNT-A 800 U 与 onaBoNT-A 200 U 时,每周 UI 发作减少量的差异被认为具有临床意义,但当比较 aboBoNT-A 的较低剂量(600 U)与 onaBoNT-A 200 U 时则不然。亚组/敏感性分析的结果与基础情况一致。
研究之间存在异质性;然而,分析中趋势的高度一致性表明,异质性的影响较低。
在难治性 NDO 患者中,aboBoNT-A 在减少 UI 方面可能优于 onaBoNT-A。由于目前证据有限,需要更多的确认性研究。