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Risk and benefit for targeted therapy agents in paediatric phase I trials in oncology: a systematic review and
Joanna Ostrowska1, Karolina Strzebonska1, Marcin Waligora2
1REMEDY, Research Ethics in Medicine Study Group, Department of Bioethics and Health Psychology, Jagiellonian University Medical College, Krakow, Poland.
Introduction:
Paediatric phase I oncology trials establish safe dosing and provide preliminary efficacy data for investigational anti-cancer agents in children. The aim of the proposed systematic review is to identify the risk and benefit ratio for targeted therapy agents in paediatric Phase I trials in oncology.
Methods And Analysis:
We will search Embase and PubMed for paediatric Phase I cancer trials published from 2 March 2015 to 30 June 2026. Eligible studies are dose-escalation trials enrolling participants under 21 years with solid or haematological malignancies, testing targeted therapy agents. We will measure two co-primary outcomes: (1) risk-proportion of participants experiencing drug-related adverse events of grade 3, 4 or 5; and (2) benefit-objective response rate. Where data permit, proportions will be pooled using random-effects meta-analysis.
Ethics And Dissemination:
This study will use publicly available data; therefore, ethics committee approval is not required. Results from the systematic review will be published in a peer-reviewed journal and presented at relevant conferences.
Prospero Registration Number:
CRD420261408673.
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