Wulfse Maaike, Vervoorn Mats T, Amelink Jantijn J G J, Ballan Elisa M, de Jager Saskia C A, Sluijter Joost P G, Doevendans Pieter A, Zwetsloot Peter-Paul M, Van der Kaaij Niels P
Department of Cardiothoracic Surgery, Division of Heart & Lungs, University Medical Center Utrecht, P.O. Box 85500, Utrecht, 3508, GA, The Netherlands.
Netherlands Heart Institute, Utrecht, The Netherlands.
J Cardiovasc Transl Res. 2025 Feb;18(1):209-220. doi: 10.1007/s12265-024-10563-1. Epub 2024 Oct 3.
Cell therapy, gene therapy, and tissue engineering have been explored as potential strategies to repair or regenerate damaged cardiac tissue. Despite the presence of encouraging preclinical data, clinical trials of regenerative cardiac therapies have yielded mixed results. Our study aimed to investigate the fate of all registered clinical trials within regenerative cardiac medicine, with the purpose of exploring the potential role of publication bias (or trial-completion bias), how published and unpublished research affects the field, and to draw lessons and recommendations for future clinical trials. In this analysis, we show that only a third of all registered trials has yielded results and that a significant number of trials are not completed. Furthermore, we identified significant heterogeneity in study design, study phase, funding, specific therapies used, primary outcome measures and methods of outcome assessment. These observations might hinder the successful translation of cardiac regenerative therapies into clinical practice.
细胞疗法、基因疗法和组织工程已被探索作为修复或再生受损心脏组织的潜在策略。尽管有令人鼓舞的临床前数据,但心脏再生疗法的临床试验结果却喜忧参半。我们的研究旨在调查心脏再生医学领域所有注册临床试验的结果,以探讨发表偏倚(或试验完成偏倚)的潜在作用、已发表和未发表的研究如何影响该领域,并为未来的临床试验吸取经验教训并提出建议。在这项分析中,我们表明所有注册试验中只有三分之一产生了结果,并且有相当数量的试验没有完成。此外,我们发现研究设计、研究阶段、资金、使用的特定疗法、主要结局指标和结局评估方法存在显著异质性。这些观察结果可能会阻碍心脏再生疗法成功转化为临床实践。