近日，美国约翰·霍普金斯医院Mark G. Luciano团队进行了分流治疗特发性常压脑积水的随机试验。这一研究成果发表在2025年9月16日出版《新英格兰医学杂志》上。

特发性常压脑积水是一种以老年人步态、平衡、认知和膀胱控制功能受损为特征的神经系统疾病。这种疾病可以通过分流手术治疗，但分流的效果尚不清楚。

研究组进行了一项双盲、随机、安慰剂对照试验，参与者根据脑脊液（CSF）引流改善步态速度的基础上选择分流手术。参与者被随机分配到无创可调分流阀的开放分流阀设置（开启压力，110毫米水）或安慰剂阀设置（开启压力，400毫米水）。主要观察指标是术后3个月步态速度的变化。次要结果是3个月时Tinetti量表总分的变化（范围0 - 28；分数越低表明步态和平衡状况较差），蒙特利尔认知评估（MoCA）评分（范围0 - 30；分数越低表明认知状况较差），膀胱过度活动问卷评分（范围0 - 100；分数越高表明尿失禁情况较差）。

共有99名参与者接受随机分组并接受指定的干预措施。在3个月时，开路分流组的步态速度有所增加（平均[±SD]变化，0.23±0.23 m / s；在49名参与者中评估），安慰剂组的步态速度不变（平均变化，0.03±0.23 m / s；在49名参与者中评估），导致治疗差异为0.21 m / s（95%置信区间，0.12至0.31;P<0.001）。开路分流组的Tinetti量表评分（平均变化，2.9分对0.5分；P=0.003）的改善明显大于安慰剂组，但MoCA评分（1.3分对0.3分）或膀胱过度活动问卷评分（3.3分对1.5分）的改善不明显。关于不良事件，安慰剂组更多的参与者报告跌倒（46%对24%），脑出血的比例相同（两组均为2%），开放分流组更多的参与者出现硬膜下出血（12%对2%）和体位性头痛（59%对28%）。

研究结果表明，在对暂时性脑脊液引流有反应的特发性常压脑积水患者中，分流术在3个月时显著改善了步态速度、步态和平衡，但在认知或尿失禁方面没有改善。

附：英文原文

Title: A Randomized Trial of Shunting for Idiopathic Normal-Pressure Hydrocephalus

Author: Mark G. Luciano, Michael A. Williams, Mark G. Hamilton, Heather L. Katzen, Nickolas A. Dasher, Abhay Moghekar, Jun Hua, Jan Malm, Anders Eklund, Naomi Alpert Abel, Ahmed M. Raslan, Benjamin D. Elder, Jesse J. Savage, Daniel L. Barrow, Kiarash Shahlaie, Hailey Jensen, Thomas J. Zwimpfer, Jessica Wollett, Daniel F. Hanley, Richard Holubkov

Issue&Volume: 2025-09-16

Abstract:

Background

Idiopathic normal-pressure hydrocephalus is a neurologic disorder characterized by impaired gait, balance, cognition, and bladder control in older adults. The disorder is treated with shunt surgery, but the effectiveness of shunting is unclear.

Methods

We conducted a double-blind, randomized, placebo-controlled trial involving participants selected for shunt surgery on the basis of gait-velocity improvement with cerebrospinal fluid (CSF) drainage. Participants were randomly assigned to an open-shunt valve setting (opening pressure, 110 mm of water) or a placebo valve setting (opening pressure, >400 mm of water) of a noninvasively adjustable shunt. The primary outcome was the change in gait velocity 3 months after surgery. Secondary outcomes were the change at 3 months in the Tinetti scale total score (range, 0 to 28; lower scores indicate worse gait and balance), Montreal Cognitive Assessment (MoCA) score (range, 0 to 30; lower scores indicate worse cognition), and Overactive Bladder Questionnaire score (range, 0 to 100; higher scores indicate worse urinary incontinence).

Results

A total of 99 participants underwent randomization and received the assigned intervention. At 3 months, gait velocity had increased in the open-shunt group (mean [±SD] change, 0.23±0.23 m per second; assessed in 49 participants) and was unchanged in the placebo group (mean change, 0.03±0.23 m per second; assessed in 49 participants), resulting in a treatment difference of 0.21 m per second (95% confidence interval, 0.12 to 0.31; P<0.001). A significantly greater improvement in the open-shunt group than the placebo group was seen for the Tinetti scale score (mean change, 2.9 points vs. 0.5 points; P=0.003) but not the MoCA score (1.3 points vs. 0.3 points) or the Overactive Bladder Questionnaire score (3.3 points vs. 1.5 points). The results regarding adverse events were mixed, with more participants in the placebo group reporting falls (46% vs. 24%), an equal percentage having cerebral bleeding (2% in both groups), and more participants in the open-shunt group having subdural bleeding (12% vs. 2%) and positional headaches (59% vs. 28%).

Conclusions

Among participants with idiopathic normal-pressure hydrocephalus who had a response to temporary CSF drainage, shunting resulted in significant improvements at 3 months in gait velocity and a measure of gait and balance but not in measures of cognition or incontinence.

DOI: NJ202509160000007

Source: https://www.nejm.org/doi/full/10.1056/NEJMoa2503109