Abstract
BACKGROUND: Dose-expansion cohort (DEC) has been increasingly used as the pivotal trial to demonstrate preliminary efficacy and safety of target anticancer drugs. We aimed to investigate the reporting quality in the published articles of DECs. METHODS: We retrospectively identified the published articles of DEC pivotal trials of the FDA-approved targeted anticancer drugs between Jan 1st, 2012 and March 31st, 2023. All published articles were evaluated by using the CONSORT Dose-finding Extension (CONSORT-DEFINE) guidance. RESULTS: Forty-one articles on DEC trials were identified, of which 32 (78.0%) were published in journals with a high impact factor (≥ 50). The median proportion of adequately reported items in the evaluated 41 articles was 83.7% (with an interquartile range of 79.1% to 88.4%). Inadequacies were observed in several areas of DEC trial reporting. Specifically, planned cohort size (item 3a8), dose modification and discontinuation (item 5b), sample size calculation (item 7a), interim analysis (item 7b), adjusted analyses (item 12b) for efficacy endpoints, data monitoring (item 26), safety review committee (item 26a), and the role of funders (item 25) were mentioned in only 4 (9.8%), 17 (41.5%), 30 (73.2%), 14 (34.1%), 30 (73.2%), 6 (14.6%), 4 (9.8%), and 12 (29.3%) articles, respectively. Additionally, inconsistencies were noted in the definition of DEC across different articles, revealing irregular naming methods being used. CONCLUSIONS: Despite being published in high-impact journals, DEC pivotal trials were inadequately reported. There is still a need to improve the completeness and transparency in reporting DEC trials to facilitate external review and evaluation of the DEC evidence.