Kobberø Hanne, Krhut Jan, Zvara Peter, Pedersen Torben Brøchner, Fode Mikkel, Nielsen Helle Hvilsted, Poulsen Mads Hvid
Department of Urology, Odense University Hospital, Odense, Denmark.
Department of Clinical Research, Research Unit of Urology, University of Southern Denmark, Odense, Denmark.
Neurourol Urodyn. 2025 Jun;44(5):1109-1119. doi: 10.1002/nau.70052. Epub 2025 Apr 14.
We present results of a two-arm pilot study assessing the feasibility of conducting a double-blind randomized controlled trial (RCT) to evaluate the efficacy of sacral neuromodulation (SNM) in patients with multiple sclerosis (MS) suffering from neurogenic lower urinary tract dysfunction (NLUTD).
Eligible subjects with refractory NLUTD and EDSS < 5 underwent SNM test phase. Those showing more than a 50% improvement of bladder variables, received implantable pulse generators (IPG) and were randomized to either treatment group (IPG ON) or to sham group (IPG OFF) for 1 month. During second month, all patients had the IPG ON until the end of the trial. The primary endpoints were trial feasibility, recruitment potential, and response rate at the end of SNM test phase. Secondary endpoint was safety. Changes in key bladder diary-derived variables and patient reported outcomes were recorded as well.
Thirty-two patients were screened, 17 were eligible and 13 were included in the SNM test phase. Eleven were considered responders and were implanted with IPG. Subsequently, six patients were randomized to the treatment group and five to the sham group. No serious adverse events were reported. In the intervention phase, both objective and subjective improvements were seen in the treatment group, while the symptoms in the sham group remained mostly unchanged. At study completion, six patients reported being completely satisfied, three were mostly satisfied, and two were indifferent to the treatment.
This pilot trial demonstrated feasibility of double-blind RCT assessing safety and efficacy of SNM in MS patients.
ClinicalTrials.gov NCT05380856.
我们展示了一项双臂试点研究的结果，该研究评估了进行双盲随机对照试验（RCT）以评估骶神经调节（SNM）对患有神经源性下尿路功能障碍（NLUTD）的多发性硬化症（MS）患者疗效的可行性。
符合条件的难治性NLUTD且扩展残疾状态量表（EDSS）<5的受试者接受SNM测试阶段。那些膀胱变量改善超过50%的受试者接受植入式脉冲发生器（IPG），并被随机分为治疗组（IPG开启）或假手术组（IPG关闭），为期1个月。在第二个月，所有患者的IPG都开启，直至试验结束。主要终点是试验的可行性、招募潜力以及SNM测试阶段结束时的反应率。次要终点是安全性。还记录了关键膀胱日记衍生变量和患者报告结局的变化。
筛选了32名患者，17名符合条件，13名纳入SNM测试阶段。11名被认为是反应者并植入了IPG。随后，6名患者被随机分为治疗组，5名被分为假手术组。未报告严重不良事件。在干预阶段，治疗组在客观和主观方面均有改善，而假手术组的症状大多保持不变。研究结束时，6名患者报告对治疗完全满意，3名基本满意，2名对治疗无差异。
这项试点试验证明了双盲RCT评估SNM对MS患者安全性和疗效的可行性。
ClinicalTrials.gov NCT05380856。
