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临床研究类型及其在疫情后正确解读的指导。

Clinical study types and guidance for their correct post-pandemic interpretation.

机构信息

Manuel Martínez-Sellés. Cardiology Service, Hospital General Universitario Gregorio Marañón, C/ del Dr. Esquerdo 46, 28007, Madrid. Spain.

出版信息

Rev Esp Quimioter. 2024 Jun;37(3):203-208. doi: 10.37201/req/003.2024. Epub 2024 Feb 26.

Abstract

Randomized clinical trials (RCTs) are key to the advancement of medicine and microbiology, but they are not the only option. Observational studies provide information on long-term efficacy and safety, are less expensive, allow the study of rare events, and obtain information more quickly than RCTs. On the other hand, they are more vulnerable to confounding factors. Prospective exploratory pilot studies share many aspects with RCTs but are not subject to supervision by external commissions or mandatory registration. Multitesting can pervert the balance of publications in favor of the desired effect. Bonferroni's reasoning shows that if 10 studies are performed with an ineffective antibiotic, the probability that at least one will show P <0.05 might be 40%. Scenarios in which there is intensive pressure to perform research, such as the recent pandemic, might result in many research teams trying to study the effect of an antimicrobial. Even if the drug has no efficacy, if 100 research teams conduct a study to assess its usefulness, it might be virtually certain that at least one will get a P value <0.05. If the other studies (with P >0.05) are not published, the scientific commu nity would consider that there is strong evidence in favor of its usefulness. In conclusion, RCTs are a very good source of clinical information, but are not the only one. The systematic registration of all research can and should be applied to all types of clinical studies.

摘要

随机临床试验(RCTs)是医学和微生物学发展的关键,但它们不是唯一的选择。观察性研究提供了关于长期疗效和安全性的信息,成本较低,允许研究罕见事件,并比 RCTs 更快地获得信息。另一方面,它们更容易受到混杂因素的影响。前瞻性探索性试点研究与 RCTs 有许多共同之处,但不受外部委员会的监督或强制性注册的限制。多重检验可能会扭曲出版物的平衡,有利于预期的效果。Bonferroni 的推理表明,如果对一种无效的抗生素进行 10 项研究,那么至少有一项研究显示 P<0.05 的概率可能为 40%。在需要进行研究的压力很大的情况下,例如最近的大流行,可能会有许多研究团队试图研究一种抗菌药物的效果。即使该药物没有疗效,如果 100 个研究团队进行一项研究来评估其有用性,那么几乎可以肯定至少有一个研究团队会得到 P 值<0.05。如果其他研究(P>0.05)没有发表,科学界会认为有强有力的证据支持其有用性。总之,RCTs 是临床信息的一个很好的来源,但不是唯一的来源。对所有研究的系统登记可以而且应该适用于所有类型的临床研究。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/8cd0/11094635/0ef6a191d6f2/revespquimioter-37-203-g001.jpg

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