Randomized controlled trials - The what, when, how and why.

IF 2 3区 医学 Q2 PEDIATRICS
Luis H Braga, Forough Farrokhyar, M İrfan Dönmez, Caleb P Nelson, Bernhard Haid, Kathy Herbst, Massimo Garriboli, Salvatore Cascio, Anka Nieuwhof-Leppink, Martin Kaefer, Darius J Bägli, Nicholas Kalfa, Christina Ching, Magdalena Fossum, Luke Harper
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引用次数: 0

Abstract

Randomized controlled trials (RCTs) are at the top of the pyramid of evidence as they offer the best answer on the efficacy of a new treatment. RCTs are true experiments in which participants are randomly allocated to receive a certain intervention (experimental group) or a different intervention (comparison group), or no treatment at all (control or placebo group). Randomization, along with other methodological features such as blinding and allocation concealment, safeguard against biases. This review will focus on parallel group RCT design as it is the most common design in the field of Pediatric Urology. RCTs can be designed using a superiority, equivalency, or non-inferiority hypothesis, and are usually preceded by a pilot, where the trial protocol is implemented in a small number of patients, mimicking the larger, definitive study. Even though regarded as the best available option to bring out scientific data, RCTs might be prone to mislead. If RCTs are small and underpowered, a difference of even one single event between groups, may completely change the trial results. To safeguard against RCTs weakness, a fragility concept of statistical significance was developed and called the Fragility Index (FI). RCTs may not be appropriate, ethical, or feasible for all surgical interventions. They may have limitations such as prohibitive cost and unrealistic large sample sizes. Nearly 60 % of surgical research questions cannot be answered by RCTs. Therefore, clinical practice should be based on the best available evidence on a given topic, regardless of the study design. However, even in these situations, conclusions drawn from observational studies must be interpreted with caution.

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来源期刊
Journal of Pediatric Urology
Journal of Pediatric Urology PEDIATRICS-UROLOGY & NEPHROLOGY
CiteScore
3.70
自引率
15.00%
发文量
330
审稿时长
4-8 weeks
期刊介绍: The Journal of Pediatric Urology publishes submitted research and clinical articles relating to Pediatric Urology which have been accepted after adequate peer review. It publishes regular articles that have been submitted after invitation, that cover the curriculum of Pediatric Urology, and enable trainee surgeons to attain theoretical competence of the sub-specialty. It publishes regular reviews of pediatric urological articles appearing in other journals. It publishes invited review articles by recognised experts on modern or controversial aspects of the sub-specialty. It enables any affiliated society to advertise society events or information in the journal without charge and will publish abstracts of papers to be read at society meetings.
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