David Geffen School of Medicine at UCLA, Los Angeles, California, USA.
Advanced Urology Center of New York, New York, New York, USA.
Neurourol Urodyn. 2024 Nov;43(8):1765-1775. doi: 10.1002/nau.25538. Epub 2024 Jul 8.
This pooled analysis of randomized controlled studies investigated the safety and efficacy of onabotulinumtoxinA in male and female patients with overactive bladder (OAB).
Data were pooled from four similarly designed trials in North America and Europe. Adults with idiopathic OAB for ≥6 months inadequately managed by at least one anticholinergic were randomized 1:1 or 2:1 to receive onabotulinumtoxinA 100 U or matched placebo in Cycle 1 and could request open-label retreatment with onabotulinumtoxinA 100 U at ≥12 weeks. Efficacy outcomes at Week 12 included the primary endpoint of mean urinary incontinence (UI) episodes per day and other variables, such as the proportion of patients with ≥50% reduction in daily UI episodes. Safety was assessed by monitoring treatment-emergent adverse events (TEAEs). Analyses by sex were descriptive. Males were further analyzed by benign prostatic hyperplasia (BPH) diagnosis status.
In the pooled population (N = 1564), there were 194 males (12.4%) and 1370 females (87.6%). Mean number of baseline UI episodes per day was 4.9 in males and 5.5 in females. At Week 12, numerically greater mean reductions from baseline in number of daily UI episodes were observed with the onabotulinumtoxinA 100 U group (females: -3.0; males: -2.2) versus placebo (females: -1.1; males: -1.3). Achievement of ≥50% reduction in daily UI episodes was numerically greater with onabotulinumtoxinA 100 U (females: 64.8%; males: 61.2%) versus placebo (females: 30.6%; males: 44.8%), and numerically higher in males without BPH (onabotulinumtoxinA: 65.1%; placebo: 50.9%) versus with BPH (onabotulinumtoxinA: 54.3%; placebo: 36.6%). A total of 34.7% of males and 39.4% of females experienced at least one TEAE in the first 12 weeks during treatment Cycle 1. Urinary tract infection rate was 13.1% in females and 4.2% in males; incidence of hematuria was 6.8% in males and 1.1% in females. Incidence of urinary retention (defined as incomplete emptying, requiring catheterization) was 2.7% in females and 4.7% in males.
OnabotulinumtoxinA 100 U was efficacious and well tolerated in men and women with OAB, including in males with and without BPH. No new safety findings were identified when data were analyzed by sex.
本项汇集分析的随机对照研究调查了在男性和女性膀胱过度活动症（OAB）患者中使用肉毒毒素 A 治疗的安全性和疗效。
数据来自北美和欧洲四项类似设计的试验。入组为至少接受一种抗胆碱能药物治疗但病情仍未得到充分控制的、患有特发性 OAB 且病史超过 6 个月的成年人，按照 1:1 或 2:1 的比例随机分组，分别接受肉毒毒素 A 100U 或匹配安慰剂治疗（第 1 周期），并可在至少 12 周时请求开放标签的肉毒毒素 A 100U 再治疗。第 12 周的疗效终点包括平均每日尿失禁（UI）发作次数这一主要终点，以及其他变量，如每日 UI 发作次数减少≥50%的患者比例。通过监测治疗中出现的不良事件（TEAEs）来评估安全性。按性别进行的分析为描述性分析。对男性进一步按良性前列腺增生（BPH）诊断情况进行分析。
在汇集的人群中（N=1564），男性 194 例（12.4%），女性 1370 例（87.6%）。男性和女性基线时的平均每日 UI 发作次数分别为 4.9 次和 5.5 次。第 12 周时，与安慰剂相比，肉毒毒素 A 100U 组男性（-2.2）和女性（-3.0）的每日 UI 发作次数均有更大程度的平均减少。每日 UI 发作次数减少≥50%的患者比例在肉毒毒素 A 100U 组也更大（女性：64.8%；男性：61.2%），而安慰剂组（女性：30.6%；男性：44.8%）更小，在无 BPH 的男性中比例更高（肉毒毒素 A：65.1%；安慰剂：50.9%），而在有 BPH 的男性中比例更低（肉毒毒素 A：54.3%；安慰剂：36.6%）。在第 1 周期的前 12 周内，共有 34.7%的男性和 39.4%的女性出现至少一次治疗期间不良事件。女性的尿路感染发生率为 13.1%，男性为 4.2%；血尿发生率在男性中为 6.8%，在女性中为 1.1%。女性的尿潴留发生率（定义为不完全排空，需要导尿）为 2.7%，男性为 4.7%。
在男性和女性 OAB 患者中，肉毒毒素 A 100U 治疗有效且耐受良好，包括有和无 BPH 的男性。按性别分析时未发现新的安全性发现。
