Polysomnographic titration of non-invasive ventilation in motor neurone disease (3TLA): study protocol for a randomised controlled trial.

IF 2 4区医学 Q3 MEDICINE, RESEARCH & EXPERIMENTAL Trials Pub Date : 2025-01-06 DOI:10.1186/s13063-024-08464-4

David J Berlowitz, Dominic Rowe, Mark E Howard, Amanda Piper, Marnie Graco, Sabine Braat, Bhajan Singh, Tanara Viera Souza, Natasha Lannin, Alistair McLean, Abbey Sawyer, Kate A Carey, Yasmin Ahamed

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Abstract

Background: Non-invasive ventilation (NIV) uses positive pressure to assist people with respiratory muscle weakness or severe respiratory compromise to breathe. Most people use this treatment during sleep when breathing is most susceptible to instability. The benefits of using NIV in motor neurone disease (MND) are well-established. However, uptake and usage are low (~ 19%) and there is no consensus on how to best implement NIV in MND in Australia. Consequently, clinical practice models are highly variable. Our team has recently provided evidence that specific and individualised NIV titration using a sleep study (polysomnography; PSG) leads to better outcomes in people with MND. However, for this clinical practice model to result in sustained benefits, evidence of effectiveness across multiple sites, as well as culture and practice change, must occur.

Methods: A two-arm, assessor-blinded, individual participant randomised controlled trial in MND care centres across Australia will be undertaken. Two-hundred and forty-four participants will be randomised (1:1) to either the intervention group (PSG-assisted commencement of NIV settings; PSG) or a control group (sham PSG). Participants will be asked to use their NIV device for 7 weeks and will then return for follow-up assessments. Respiratory, sleep and patient-reported outcome measures will be collected at baseline and follow-up. The primary aim is to determine if the proportion of participants using NIV for > 4 h/day during the intervention period is higher in the PSG than the control group. A process evaluation, health economic evaluation and 12-month cohort follow-up will be undertaken and reported separately.

Discussion: The results of this trial will demonstrate the effects of PSG-assisted titration of NIV on usage of NIV in people with MND. We hypothesise that the PSG intervention will improve synchrony between the user and the machine, which will lead to greater NIV usage compared to the control group.

Trial registration: ClinicalTrials.gov NCT05136222. Registered on November 25, 2021.

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运动神经元疾病（3TLA）无创通气的多导睡眠滴定法：随机对照试验的研究方案。

背景：无创通气（NIV）使用正压通气来帮助呼吸肌无力或严重呼吸损害患者呼吸。大多数人在睡眠时使用这种疗法，因为睡眠时呼吸最容易不稳定。在运动神经元疾病（MND）中使用NIV的益处是公认的。然而，采用率和使用率都很低（约19%），对于如何在澳大利亚的MND中最好地实施NIV还没有达成共识。因此，临床实践模式是高度可变的。我们的团队最近提供的证据表明，使用睡眠研究(多导睡眠仪；多导睡眠监测（PSG）对MND患者的治疗效果更好。然而，为了使这种临床实践模式产生持续的效益，必须出现跨多个地点的有效性证据，以及文化和实践的变化。方法：将在澳大利亚的MND护理中心进行一项双臂、评估者盲法、个体参与者随机对照试验。244名参与者将被随机分配（1:1）到干预组(psg辅助开始NIV设置；或对照组（假PSG）。参与者将被要求使用他们的NIV设备7周，然后将返回进行后续评估。将在基线和随访时收集呼吸、睡眠和患者报告的结果测量值。主要目的是确定在干预期间PSG中使用NIV的参与者比例是否高于对照组。将分别进行过程评价、卫生经济评价和12个月队列随访并进行报告。讨论：本试验的结果将证明psg辅助NIV滴定对MND患者使用NIV的影响。我们假设PSG干预将改善用户和机器之间的同步，这将导致与对照组相比更多的NIV使用。试验注册：ClinicalTrials.gov NCT05136222。于2021年11月25日注册。

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来源期刊

Trials 医学-医学：研究与实验

CiteScore

3.80

自引率

4.00%

发文量

966

审稿时长

6 months

期刊介绍： Trials is an open access, peer-reviewed, online journal that will encompass all aspects of the performance and findings of randomized controlled trials. Trials will experiment with, and then refine, innovative approaches to improving communication about trials. We are keen to move beyond publishing traditional trial results articles (although these will be included). We believe this represents an exciting opportunity to advance the science and reporting of trials. Prior to 2006, Trials was published as Current Controlled Trials in Cardiovascular Medicine (CCTCVM). All published CCTCVM articles are available via the Trials website and citations to CCTCVM article URLs will continue to be supported.