DOI
10.1016/S2352-4642(24)00328-6
In this Viewpoint, we discuss the challenges facing perinatal clinical researchers, many of which are unique to this field, and how traditional two-arm randomised trials using frequentist analysis might no longer be fit for purpose for perinatology. We propose a solution: the adoption of adaptive platform trials (APTs) with Bayesian methodology to address perinatal research questions to improve outcomes of preterm birth. APTs use a master protocol as a foundation to efficiently assess multiple interventions simultaneously for a particular disease. APTs can study these interventions in a perpetual manner, with interventions allowed to enter or leave the platform on the basis of preplanned decision algorithms. In this Viewpoint, we outline the ways in which APTs can overcome some of the issues facing perinatal clinical research, and the challenges and essential requirements for the design and implementation of perinatal APTs that should be considered.
DOI
10.1186/s13063-024-08072-2
Randomised controlled trials (RCTs) aim to estimate the causal effect of one or more interventions relative to a control. One type of outcome that can be of interest in an RCT is an ordinal outcome, which is useful to answer clinical questions regarding complex and evolving patient states. The target parameter of interest for an ordinal outcome depends on the research question and the assumptions the analyst is willing to make. This review aimed to provide an overview of how ordinal outcomes have been used and analysed in RCTs.
DOI
10.1136/archdischild-2024-326874
The largest randomised controlled trial (RCT) of neonatal caffeine therapy, the Caffeine for Apnea of Prematurity (CAP) trial, evaluated caffeine citrate at a loading dose of 20 mg/kg and a maintenance dose of 5–10 mg/kg/day.1 Although caffeine is among the most commonly used neonatal medications, practice varies widely, and higher dosing has been reported.2 We hypothesised that caffeine practice may have evolved since the last survey conducted in the Australian and New Zealand Neonatal Network (ANZNN),3 despite limited data supporting higher caffeine doses.2 We aimed to describe the current use of caffeine in very preterm infants as a fundamental step towards large RCTs of caffeine dosing.