ReviewPediatric research2018
Useful pharmacodynamic endpoints in children: selection, measurement, and next steps.
Review in Pediatric research, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
14 citing papers in PubMed.
- Pharmacokinetics of post-transplant cyclophosphamide and its associations with clinical outcomes in pediatric haploidentical hematopoietic stem cell transplantation.Biomarker research · 2025Article
- Monitoring Activity and Gait in Children (MAGIC) using digital health technologies.Pediatric research · 2024Article
- Personalized Dosing of Medicines for Children: A Primer on Pediatric Pharmacometrics for Clinicians.Paediatric drugs · 2024Review
- Pediatric Drug Development: Reviewing Challenges and Opportunities by Tracking Innovative Therapies.Pharmaceutics · 2023Review
- Pharmacokinetic pharmacodynamic modeling of analgesics and sedatives in children.Paediatric anaesthesia · 2023Review
- Current knowledge, challenges and innovations in developmental pharmacology: A combined conect4children Expert Group and European Society for Developmental, Perinatal and Paediatric Pharmacology White Paper.British journal of clinical pharmacology · 2022Review
- Relationship Between Anifrolumab Pharmacokinetics, Pharmacodynamics, and Efficacy in Patients With Moderate to Severe Systemic Lupus Erythematosus.Journal of clinical pharmacology · 2022Article
- Improving clinical paediatric research and learning from COVID-19: recommendations by the Conect4Children expert advice group.Pediatric research · 2022Article
- Navigating the Regulatory Landscape to Develop Pediatric Oncology Drugs: Expert Opinion Recommendations.Paediatric drugs · 2021Review
- Article
- How to Conduct Clinical Trials in Children: A Tutorial.Clinical and translational science · 2019Article
- Pharmacokinetic-Pharmacodynamic Modeling in Pediatric Drug Development, and the Importance of Standardized Scaling of Clearance.Clinical pharmacokinetics · 2019Review
- Improving pharmaceutical trials for children: a call to the pediatric academic community.Pediatric research · 2018Article
- Pharmacokinetic studies in children: recommendations for practice and research.Archives of disease in childhood · 2018Review
Corrections and comments
- Commented on by
Authors and funding
5 authors.
Funding
Abstract
Pharmacodynamic (PD) endpoints are essential for establishing the benefit-to-risk ratio for therapeutic interventions in children and neonates. This article discusses the selection of an appropriate measure of response, the PD endpoint, which is a critical methodological step in designing pediatric efficacy and safety studies. We provide an overview of existing guidance on the choice of PD endpoints in pediatric clinical research. We identified several considerations relevant to the selection and measurement of PD endpoints in pediatric clinical trials, including the use of biomarkers, modeling, compliance, scoring systems, and validated measurement tools. To be useful, PD endpoints in children need to be clinically relevant, responsive to both treatment and/or disease progression, reproducible, and reliable. In most pediatric disease areas, this requires significant validation efforts. We propose a minimal set of criteria for useful PD endpoint selection and measurement. We conclude that, given the current heterogeneity of pediatric PD endpoint definitions and measurements, both across and within defined disease areas, there is an acute need for internationally agreed, validated, and condition-specific pediatric PD endpoints that consider the needs of all stakeholders, including healthcare providers, policy makers, patients, and families.
Indexed as
Identifiers
What OpenQuestion holds
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.