特发性肺纤维化药物随机对照试验的资格标准：一项基于注册的研究,European Respiratory Journal

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特发性肺纤维化药物随机对照试验的资格标准：一项基于注册的研究
European Respiratory Journal ( IF 16.6 ) Pub Date : 2023-05-05 , DOI: 10.1183/13993003.02163-2022
Yet H Khor _{1,

2,

3,

4} , Max Schulte _{2,

3,

5} , Kerri A Johannson ₆ , Veronica Marcoux ₇ , Jolene H Fisher ₈ , Deborah Assayag ₉ , Helene Manganas ₁₀ , Nasreen Khalil ₁₁ , Martin Kolb ₁₂ , Christopher J Ryerson _{11,

13} , , , Gerard Cox , Charlene D Fell , Andrea S Gershon , Nicole Goh , Andrew J Halayko , Stacey Lok , Julie Morisset , Mohsen Sadatsafavi , Shane Shapera , Teresa To , Pearce G Wilcox , Alyson W Wong

Affiliation

Respiratory Central Clinical School, Monash University, Melbourne, Australia
Department of Respiratory and Sleep Medicine, Austin Health, Heidelberg, Australia.
Institute for Breathing and Sleep, Heidelberg, Australia.
Faculty of Medicine, University of Melbourne, Melbourne, Australia.
School of Health Sciences, Swinburne University of Technology, Melbourne, Australia.
Department of Medicine, University of Calgary, Calgary, AB, Canada.
Department of Medicine, University of Saskatchewan, Saskatoon, SK, Canada.
Department of Medicine, University of Toronto, Toronto, ON, Canada.
Department of Medicine, McGill University, Montreal, QC, Canada.
Département de Médecine, Centre Hospitalier de l'Université de Montréal, Montreal, QC, Canada.
Department of Medicine, University of British Columbia, Vancouver, BC, Canada.
Department of Medicine, Firestone Institute for Respiratory Health, McMaster University, Hamilton, ON, Canada.
Centre for Heart Lung Innovation, St. Paul's Hospital, Vancouver, BC, Canada.

背景

关于特发性肺纤维化 (IPF) 的随机对照试验 (RCT) 的普遍性知之甚少。我们评估了 III 期 IPF RCT 的资格标准以确定它们在临床登记中的代表性，并根据资格标准计算了用力肺活量 (FVC) 的变化。

方法

在超过 60% 的 IPF RCT 中使用的通用资格标准是通过文献检索确定的，并适用于来自澳大利亚和加拿大前瞻性登记处的 IPF 患者。还评估了额外的 6 分钟步行距离 (6MWD) 和先前疾病进展的不同测量标准。根据个体和复合标准的资格，使用联合纵向生存模型来比较 FVC 下降。

结果

在 990 名 IPF 患者中，527 名 (53%) 在第一次就诊时符合所有常见的 RCT 资格标准，包括 343 名具有明确的 IPF 和 184 名具有放射学可能的普通间质性肺炎模式而没有组织学证实（即临时 IPF ）。尼达尼布和吡非尼酮具有里程碑意义的随机对照试验符合条件的患者百分比为 19-50%。将 6MWD ≥150 m 和先前疾病进展的不同测量值添加到复合共同标准中，将符合资格的患者百分比分别降低至 52% (n=516) 和 4–18% (n=12–61)。符合复合共同标准的患者 1 年 FVC 下降速度低于未达到标准的患者（–90对–103 毫升，p=0.01）。与临时 IPF 相比，确定 IPF 通常具有更快速的 1 年 FVC 下降。

结论

先前 IPF RCT 的资格标准对临床 IPF 人群的普遍适用性有限，FVC 下降在符合条件和不符合条件的人群之间存在差异。

"点击查看英文标题和摘要"

Eligibility criteria from pharmaceutical randomised controlled trials of idiopathic pulmonary fibrosis: a registry-based study

Background

Little is known about generalisability of randomised controlled trials (RCTs) for idiopathic pulmonary fibrosis (IPF). We evaluated eligibility criteria for phase III IPF RCTs to determine their representativeness in clinical registries, and calculated forced vital capacity (FVC) changes according to eligibility criteria.

Methods

Common eligibility criteria used in >60% of IPF RCTs were identified from a literature search and applied to patients with IPF from prospective Australian and Canadian registries. Additional pre-specified criteria of 6-min walk distance (6MWD) and different measures of preceding disease progression were also evaluated. Joint longitudinal-survival modelling was used to compare FVC decline according to eligibility for individual and composite criteria.

Results

Out of 990 patients with IPF, 527 (53%) met all common RCT eligibility criteria at the first clinic visit, including 343 with definite IPF and 184 with radiological probable usual interstitial pneumonia pattern without histological confirmation (i.e. provisional IPF). The percentages of eligible patients for landmark RCTs of nintedanib and pirfenidone were 19–50%. Adding 6MWD ≥150 m and different measures of preceding disease progression to the composite common criteria reduced the percentages of patients meeting eligibility to 52% (n=516) and 4–18% (n=12–61), respectively. Patients meeting the composite common criteria had less-rapid 1-year FVC decline than those who did not (–90 versus –103 mL, p=0.01). Definite IPF generally had more-rapid 1-year FVC decline compared to provisional IPF.

Conclusions

Eligibility criteria of previous IPF RCTs have limited generalisability to clinical IPF populations, with FVC decline differing between eligible and ineligible populations.

更新日期：2023-05-05

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