Ann Am Thorac Soc. 2023 Jul;20(7):931-943. doi: 10.1513/AnnalsATS.202303-258ST.
Continuous positive airway pressure (CPAP) is the first-line treatment for obstructive sleep apnea (OSA). Although CPAP improves symptoms (e.g., daytime sleepiness), there is a lack of high-quality evidence that CPAP prevents many long-term outcomes, including cognitive impairment, myocardial infarction, and stroke. Observational studies suggest that patients with symptoms may be particularly likely to experience these preventive benefits with CPAP, but ethical and practical concerns limited the participation of such patients in prior long-term randomized trials. As a result, there is uncertainty about the full benefits of CPAP, and resolving this uncertainty is a key priority for the field. This workshop assembled clinicians, researchers, ethicists, and patients to identify strategies to understand the causal effects of CPAP on long-term clinically important outcomes among patients with symptomatic OSA. Quasi-experimental designs can provide valuable information and are less time and resource intensive than trials. Under specific conditions and assumptions, quasi-experimental studies may be able to provide causal estimates of CPAP's effectiveness from generalizable observational cohorts. However, randomized trials represent the most reliable approach to understanding the causal effects of CPAP among patients with symptoms. Randomized trials of CPAP can ethically include patients with symptomatic OSA, as long as there is outcome-specific equipoise, adequate informed consent, and a plan to maximize safety while minimizing harm (e.g., monitoring for pathologic sleepiness). Furthermore, multiple strategies exist to ensure the generalizability and practicality of future randomized trials of CPAP. These strategies include reducing the burden of trial procedures, improving patient-centeredness, and engaging historically excluded and underserved populations.
持续气道正压通气(CPAP)是治疗阻塞性睡眠呼吸暂停(OSA)的一线方法。尽管 CPAP 可改善症状(例如白天嗜睡),但缺乏高质量证据表明 CPAP 可预防许多长期结局,包括认知障碍、心肌梗死和中风。观察性研究表明,有症状的患者可能特别有可能从 CPAP 中获得这些预防益处,但出于伦理和实际考虑,这些患者无法参与先前的长期随机试验。因此,CPAP 的全部益处尚不确定,解决这一不确定性是该领域的关键优先事项。本次研讨会召集了临床医生、研究人员、伦理学家和患者,以确定策略,了解 CPAP 对有症状的 OSA 患者长期临床重要结局的因果影响。准实验设计可以提供有价值的信息,并且比试验的时间和资源密集度要低。在特定条件和假设下,准实验研究可能能够从可推广的观察队列中提供 CPAP 有效性的因果估计。然而,随机试验是了解有症状患者 CPAP 因果效应的最可靠方法。只要存在特定结局的均衡、充分的知情同意以及最大化安全性同时最小化伤害的计划(例如,监测病理性嗜睡),就可以在伦理上纳入有症状的 OSA 患者进行 CPAP 随机试验。此外,还存在多种策略来确保未来 CPAP 随机试验的普遍性和实用性。这些策略包括减少试验程序的负担、提高以患者为中心的程度以及让历史上被排除和服务不足的人群参与进来。