Challenges in Results Robustness of Trials with Missing Data for the Primary Endpoint: Insights from Coronary Balloon/Stent Trials.

IF 2.7 4区医学 Q2 HEALTH CARE SCIENCES & SERVICES

Risk Management and Healthcare Policy Pub Date : 2025-03-27 eCollection Date: 2025-01-01 DOI:10.2147/RMHP.S511449

Xinyue Lang, Yanyan Zhao, Yingxuan Zhu, Lei Song, Chuangshi Wang, Duoer Wang, Chilie Danzeng, Yang Wang, Wei Li

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引用次数: 0

Abstract

Background: To assess the consequence of different degrees of missing primary endpoint data for randomized controlled trials and to find the influence factors.

Methods: PubMed, Cochrane Library, EMBASE and ClinicalTrials.gov were searched up to Nov 30, 2023. We included trials of the drug-coated balloon/drug-eluted stent with angiographic outcomes as the primary endpoint. The tipping-point analysis was used to deal with the missing data for the primary endpoint. The inconsistency rate, tipping-point standardized effect size (SES) and tipping-point ratio were used to assess the result robustness.

Results: A total of 101 trials were included, which had 109 trial comparisons. Among them, 89 (81.7%) comparisons had superior/non-inferior conclusions (H₀ rejected); 85 (78.0%) comparisons had a missing rate of ≥10%, and 30 (27.5%) comparisons had a missing rate of ≥20%. For H₀ rejected comparisons with a missing rate of ≥10%, the median of inconsistency rate, tipping-point SES and tipping-point ratio was 32.2% (IQR 19.7%, 45.4%), 0.90 (IQR 0.17, 1.79) and -1.53 (IQR -2.43, -0.39). A higher missing rate and a larger (worse) observed-target SES were associated with a more unreliable result.

Conclusion: A high dropout rate and inflated target effect size could cause an unreliable result. We emphasize a robust evaluation of the results for clinical trials with missing data for the primary endpoint.

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缺少主要终点数据的试验结果的稳健性挑战：来自冠状动脉球囊/支架试验的见解

背景：评估随机对照试验中不同程度的主要终点数据缺失的后果，并寻找影响因素。方法：检索截至2023年11月30日的PubMed、Cochrane Library、EMBASE和ClinicalTrials.gov。我们纳入了以血管造影结果为主要终点的药物包被球囊/药物洗脱支架试验。引爆点分析用于处理主要终点的缺失数据。采用不一致率、临界点标准化效应大小（SES）和临界点比率来评估结果的稳健性。结果：共纳入101项试验，其中109项试验比较。其中89个（81.7%）比较得出优/非劣结论（H0拒绝）；85例（78.0%）缺失率≥10%，30例（27.5%）缺失率≥20%。对于缺失率≥10%的H0拒绝比较，不一致率、临界点SES和临界点比率的中位数分别为32.2% （IQR为19.7%,45.4%）、0.90 （IQR为0.17,1.79）和-1.53 （IQR为-2.43,-0.39）。更高的缺失率和更大（更差）的观察到的目标SES与更不可靠的结果相关。结论：高辍学率和过高的目标效应会导致结果不可靠。我们强调对缺少主要终点数据的临床试验结果进行强有力的评估。

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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来源期刊

Risk Management and Healthcare Policy Medicine-Public Health, Environmental and Occupational Health

CiteScore

6.20

自引率

2.90%

发文量

242

审稿时长

16 weeks

期刊介绍： Risk Management and Healthcare Policy is an international, peer-reviewed, open access journal focusing on all aspects of public health, policy and preventative measures to promote good health and improve morbidity and mortality in the population. Specific topics covered in the journal include: Public and community health Policy and law Preventative and predictive healthcare Risk and hazard management Epidemiology, detection and screening Lifestyle and diet modification Vaccination and disease transmission/modification programs Health and safety and occupational health Healthcare services provision Health literacy and education Advertising and promotion of health issues Health economic evaluations and resource management Risk Management and Healthcare Policy focuses on human interventional and observational research. The journal welcomes submitted papers covering original research, clinical and epidemiological studies, reviews and evaluations, guidelines, expert opinion and commentary, and extended reports. Case reports will only be considered if they make a valuable and original contribution to the literature. The journal does not accept study protocols, animal-based or cell line-based studies.