吡非尼酮预防急性呼吸窘迫综合征的纤维化：PIONEER 研究方案。

IF 2 3区医学 Q3 MEDICINE, RESEARCH & EXPERIMENTAL

Contemporary clinical trials Pub Date : 2025-03-14 DOI:10.1016/j.cct.2025.107883

Giacomo Monti , Matteo Marzaroli , Maria Teresa Tucciariello , Brian Ferrara , Francesco Meroi , Cristina Nakhnoukh , Massimo Zambon , Giovanni Borghi , Fabio Guarracino , Marco Manazza , Valentina Ajello , Alessandro Belletti , Cesare Biuzzi , Valentina Plumari , Matteo Filippini , Raffaele Cuffaro , Gabriele Racanelli , Domenico Pontillo , Simon Rauch , Federico Mattia Oliva , Luca Cabrini

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引用次数: 0

摘要

背景：肺纤维化是急性呼吸窘迫综合征（ARDS）的主要并发症。吡非尼酮是一种被批准治疗特发性肺纤维化的药物。它可以减轻ards相关的纤维化，减少长时间通气的需要。因此，我们旨在评估吡非尼酮对ARDS患者无呼吸机天数的影响。方法：在一项多中心、随机、双盲、安慰剂对照试验中，我们计划随机分配130名患有ARDS的有创通气成人接受吡非尼酮或安慰剂治疗，治疗时间长达28 天。主要预后指标为28 天存活天数和无呼吸机天数。次要结局包括无ICU天数、住院天数均为28 天、ICU死亡率和住院死亡率。我们还将评估ICU出院时高分辨率CT扫描的纤维增生性变化和生活质量。数据分析将以意向治疗为基础。讨论：该试验正在进行中，目前正在招募。这将是首个调查吡非尼酮与安慰剂相比是否能减少ARDS患者存活和无呼吸机天数的随机对照研究。它的双盲多中心设计将提供内部效度，最小的偏差，和一定程度的外部效度。如果我们的假设得到证实，这种治疗方法将证明对这种干预进行更大规模的试验是合理的。试验注册：该试验在ClinicalTrials.gov上注册，试验标识为NCT05075161。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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Pirfenidone to prevent fibrosis in acute respiratory distress syndrome: The PIONEER study protocol

Background

Pulmonary fibrosis is a major complication of the Acute Respiratory Distress Syndrome (ARDS). Pirfenidone is an approved treatment for idiopathic pulmonary fibrosis. It may attenuate ARDS-related fibrosis and decrease the need for prolonged ventilation. Accordingly, we aimed to evaluate the effect of pirfenidone on ventilator-free days in patients with ARDS.

Methods

In a multi-center, randomized, double-blind, placebo-controlled trial, we plan to randomly assign 130 adults invasively ventilated for ARDS to receive pirfenidone or placebo for up to 28 days. The primary outcome is days alive and ventilator free at 28 days. Secondary outcomes include ICU-free days, hospital free days all at 28 day, ICU mortality and hospital mortality. We will also assess fibroproliferative changes on high-resolution CT scans at ICU discharge and quality of life. Data analysis will be on an intention-to-treat basis.

Discussion

The trial is ongoing and currently recruiting. It will be the first randomized controlled study to investigate whether, compared to placebo, pirfenidone increases the number of days alive and ventilator-free in patients with ARDS. Its double-blind multicenter design will provide internal validity, minimal bias, and a degree of external validity. If our hypothesis is confirmed, this treatment would justify larger trials of this intervention.

Trial registration: This trial was registered on ClinicalTrials.gov with the trial identification NCT05075161.

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来源期刊

Contemporary clinical trials 医学-药学

CiteScore

3.70

自引率

4.50%

发文量

281

审稿时长

44 days

期刊介绍： Contemporary Clinical Trials is an international peer reviewed journal that publishes manuscripts pertaining to all aspects of clinical trials, including, but not limited to, design, conduct, analysis, regulation and ethics. Manuscripts submitted should appeal to a readership drawn from disciplines including medicine, biostatistics, epidemiology, computer science, management science, behavioural science, pharmaceutical science, and bioethics. Full-length papers and short communications not exceeding 1,500 words, as well as systemic reviews of clinical trials and methodologies will be published. Perspectives/commentaries on current issues and the impact of clinical trials on the practice of medicine and health policy are also welcome.