ArticleStatistical methods in medical research2018
Unified approach for extrapolation and bridging of adult information in early-phase dose-finding paediatric studies.
Article in Statistical methods in medical research, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers.
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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
9 citing papers in PubMed.
- Anticoagulant prescribing trends, bleeding events, and reversal agent use in pediatric patients: A retrospective, real-world study.PloS one · 2025Article
- A semi-mechanistic dose-finding design in oncology using pharmacokinetic/pharmacodynamic modeling.Pharmaceutical statistics · 2022Article
- The Use of Translational Modelling and Simulation to Develop Immunomodulatory Therapy as an Adjunct to Antibiotic Treatment in the Context of Pneumonia.Pharmaceutics · 2021Article
- Paediatric Medicines in Europe: The Paediatric Regulation-Is It Time for Reform?Frontiers in medicine · 2021Article
- An adaptive power prior for sequential clinical trials - Application to bridging studies.Statistical methods in medical research · 2020Article
- A phase I dose-finding design with incorporation of historical information and adaptive shrinking boundaries.PloS one · 2020Article
- Developmental Pharmacodynamics and Modeling in Pediatric Drug Development.Journal of clinical pharmacology · 2019Review
- Recommendations for the design of therapeutic trials for neonatal seizures.Pediatric research · 2019Article
- Recent advances in methodology for clinical trials in small populations: the InSPiRe project.Orphanet journal of rare diseases · 2018Review
Corrections and comments
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Authors and funding
8 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The number of trials conducted and the number of patients per trial are typically small in paediatric clinical studies. This is due to ethical constraints and the complexity of the medical process for treating children. While incorporating prior knowledge from adults may be extremely valuable, this must be done carefully. In this paper, we propose a unified method for designing and analysing dose-finding trials in paediatrics, while bridging information from adults. The dose-range is calculated under three extrapolation options, linear, allometry and maturation adjustment, using adult pharmacokinetic data. To do this, it is assumed that target exposures are the same in both populations. The working model and prior distribution parameters of the dose-toxicity and dose-efficacy relationships are obtained using early-phase adult toxicity and efficacy data at several dose levels. Priors are integrated into the dose-finding process through Bayesian model selection or adaptive priors. This calibrates the model to adjust for misspecification, if the adult and pediatric data are very different. We performed a simulation study which indicates that incorporating prior adult information in this way may improve dose selection in children.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.