Oxygen-Free Days as an Outcome Measure in Clinical Trials of Therapies for COVID-19 and Other Causes of New-Onset Hypoxemia.
Oxygen-Free Days as an Outcome Measure in Clinical Trials of Therapies for COVID-19 and Other Causes of New-Onset Hypoxemia.
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DOI：
10.1016/j.chest.2022.04.145
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发表时间：
2022-10
期刊：
影响因子：
9.6
通讯作者：
Fourth Accelerating COVID-19 Therapeutic Interventions and Vaccines (ACTIV-4) Host Tissue Investigators
中科院分区：
文献类型：
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作者：
Moskowitz A;Shotwell MS;Gibbs KW;Harkins M;Rosenberg Y;Troendle J;Merck LH;Files DC;de Wit M;Hudock K;Thompson BT;Gong MN;Ginde AA;Douin DJ;Brown SM;Rubin E;Joly MM;Wang L;Lindsell CJ;Bernard GR;Semler MW;Collins SP;Self WH;Fourth Accelerating COVID-19 Therapeutic Interventions and Vaccines (ACTIV-4) Host Tissue Investigators
死亡率历来是急性和危重症临床试验选择的主要结果。然而，过度依赖死亡率可能会限制可进行的试验的范围。为死亡率结果提供动力的试验通常需要大样本量，而仅仅关注死亡率未能认识到降低发病率对患者生命的重要性。COVID-19大流行突出表明，需要进行快速、有效的试验，以严格评估急性肺损伤住院患者的新疗法。无氧天数（OFDs）是临床试验的一个新结果，它是死亡率和新补充氧使用时间的综合结果。它旨在描述急性肺损伤在新低氧血症和补充氧气使用高发人群中的恢复情况。在这些人群中，OFDs捕获了急性肺损伤的两个以患者为中心的后果：死亡率和低氧性肺功能障碍。检测OFDs差异的能力通常大于其他临床试验结果，如死亡率和无呼吸机天数。OFDs是第四个加速COVID-19治疗干预和疫苗（ACTIV-4）宿主组织平台的主要结果，该平台评估了针对COVID-19住院成人和新发低氧血症的宿主对COVID-19反应的新疗法。本文概述了使用OFD作为临床试验结果的基本原理，提出了定义和分析OFD的标准化方法，并提供了使用OFD结果计算样本量的框架。
Mortality historically has been the primary outcome of choice for acute and critical care clinical trials. However, undue reliance on mortality can limit the scope of trials that can be performed. Large sample sizes are usually needed for trials powered for a mortality outcome, and focusing solely on mortality fails to recognize the importance that reducing morbidity can have on patients’ lives. The COVID-19 pandemic has highlighted the need for rapid, efficient trials to rigorously evaluate new therapies for hospitalized patients with acute lung injury. Oxygen-free days (OFDs) is a novel outcome for clinical trials that is a composite of mortality and duration of new supplemental oxygen use. It is designed to characterize recovery from acute lung injury in populations with a high prevalence of new hypoxemia and supplemental oxygen use. In these populations, OFDs captures two patient-centered consequences of acute lung injury: mortality and hypoxemic lung dysfunction. Power to detect differences in OFDs typically is greater than that for other clinical trial outcomes, such as mortality and ventilator-free days. OFDs is the primary outcome for the Fourth Accelerating COVID-19 Therapeutic Interventions and Vaccines (ACTIV-4) Host Tissue platform, which evaluates novel therapies targeting the host response to COVID-19 among adults hospitalized with COVID-19 and new hypoxemia. This article outlines the rationale for use of OFDs as an outcome for clinical trials, proposes a standardized method for defining and analyzing OFDs, and provides a framework for sample size calculations using the OFD outcome.
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影响因子：
2.5
作者：
Self WH;Stewart TG;Wheeler AP;El Atrouni W;Bistran-Hall AJ;Casey JD;Cataldo VD;Chappell JD;Cohn CS;Collins JB;Denison MR;de Wit M;Dixon SL;Duggal A;Edwards TL;Fontaine MJ;Ginde AA;Harkins MS;Harrington T;Harris ES;Hoda D;Ipe TS;Jaiswal SJ;Johnson NJ;Jones AE;Laguio-Vila M;Lindsell CJ;Mallada J;Mammen MJ;Metcalf RA;Middleton EA;Mucha S;O'Neal HR Jr;Pannu SR;Pulley JM;Qiao X;Raval JS;Rhoads JP;Schrager H;Shanholtz C;Shapiro NI;Schrantz SJ;Thomsen I;Vermillion KK;Bernard GR;Rice TW;Passive Immunity Trial for Our Nation (PassITON) Investigators
通讯作者：
Passive Immunity Trial for Our Nation (PassITON) Investigators
影响因子：
158.5
作者：
Mackle, Diane;Bellomo, M. N. Rinaldo;Young, Paul
通讯作者：
Young, Paul
影响因子：
120.7
作者：
Self, Wesley H.;Semler, Matthew W.;Brown, Samuel M.
通讯作者：
Brown, Samuel M.
DOI：
10.1056/nejmoa1711586
发表时间：
2018-03-01
期刊：
The New England journal of medicine
影响因子：
--
作者：
Self WH;Semler MW;Wanderer JP;Wang L;Byrne DW;Collins SP;Slovis CM;Lindsell CJ;Ehrenfeld JM;Siew ED;Shaw AD;Bernard GR;Rice TW;SALT-ED Investigators
通讯作者：
SALT-ED Investigators
影响因子：
120.7
作者：
Laterre, Pierre-Francois;Berry, Scott M.;Angus, Derek C.
通讯作者：
Angus, Derek C.