Baracaldo-Santamaría Daniela, Feliciano-Alfonso John Edwin, Ramirez-Grueso Raul, Rojas-Rodríguez Luis Carlos, Dominguez-Dominguez Camilo Alberto, Calderon-Ospina Carlos Alberto
Pharmacology Unit, Department of Biomedical Sciences, School of Medicine and Health Sciences, Universidad del Rosario, Bogotá 111221, Colombia.
School of Medicine, Universidad El Bosque, Bogotá 110121, Colombia.
J Clin Med. 2023 Jun 29;12(13):4371. doi: 10.3390/jcm12134371.
Multiple drugs currently used in clinical practice have been approved by regulatory agencies based on studies that utilize composite endpoints. Composite endpoints are appealing because they reduce sample size requirements, follow-up periods, and costs. However, interpreting composite endpoints can be challenging, and their misuse is not uncommon. Incorrect interpretation of composite outcomes can lead to misleading conclusions that impact patient care. To correctly interpret composite outcomes, several important questions should be considered. Are the individual components of the composite outcome equally important to patients? Did the more and less important endpoints occur with similar frequency? Do the component endpoints exhibit similar relative risk reductions? If these questions receive affirmative answers, the use and interpretation of the composite endpoint would be appropriate. However, if any component of the composite endpoint fails to satisfy the aforementioned criteria, interpretation can become difficult, necessitating additional steps. Regulatory agencies acknowledge these challenges and have specific considerations when approving drugs based on studies employing composite endpoints. In conclusion, composite endpoints are valuable tools for evaluating the efficacy and net clinical benefit of interventions; however, cautious interpretation is advised.
目前临床实践中使用的多种药物已基于采用复合终点的研究获得监管机构的批准。复合终点很有吸引力，因为它们减少了样本量要求、随访期和成本。然而，解释复合终点可能具有挑战性，而且其被误用的情况并不少见。对复合结果的错误解释可能会导致产生影响患者护理的误导性结论。为了正确解释复合结果，应考虑几个重要问题。复合结果的各个组成部分对患者同样重要吗？更重要和较不重要的终点出现的频率相似吗？组成终点是否表现出相似的相对风险降低？如果这些问题得到肯定的回答，那么复合终点的使用和解释将是合适的。然而，如果复合终点的任何组成部分不符合上述标准，解释可能会变得困难，这就需要采取额外的步骤。监管机构认识到这些挑战，在基于采用复合终点的研究批准药物时会有特定的考量。总之，复合终点是评估干预措施疗效和净临床效益的有价值工具；然而，建议谨慎解释。
