Risk and benefit for targeted therapy agents in paediatric phase I trials in oncology: a systematic review and meta-analysis protocol

Abstract

Abstract 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.