Application of sample size re-estimation in clinical trials: A systematic review

Mano, Hirotaka; Tanaka, Yuji; Orihara, Shunichiro; Moriya, Junji

doi:10.1016/j.conctc.2023.101210

Cited by 1 publication

(1 citation statement)

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“…Outside of adaptive randomization alone, we found that three studies employed a group sequential design with blinded sample size re-estimation performed at one or two prespecified interim analyses [ 19 , 21 , 22 ]. A recent systematic review identified 27 adaptive trials that utilized sample size re-estimation, in particular, this design was used in Phase I/II trials wherein there was insufficient prior knowledge of treatment group [ 30 ]. Sample size re-estimation, however, can lead to an inflated type I error rate, and though methods for optimizing these statistical adaptations exist, they remain poorly standardized within the adaptive trial literature [ 31 ].…”

Section: Discussionmentioning

confidence: 99%

Adaptive clinical trials in surgery: A scoping review of methodological and reporting quality

Staibano,

Oulousian,

McKechnie

et al. 2024

PLoS ONE

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Importance Adaptive surgical trials are scarce, but adopting these methods may help elevate the quality of surgical research when large-scale RCTs are impractical. Objective Randomized-controlled trials (RCTs) are the gold standard for evidence-based healthcare. Despite an increase in the number of RCTs, the number of surgical trials remains unchanged. Adaptive clinical trials can streamline trial design and time to trial reporting. The advantages identified for ACTs may help to improve the quality of future surgical trials. We present a scoping review of the methodological and reporting quality of adaptive surgical trials. Evidence review We performed a search of Ovid, Web of Science, and Cochrane Collaboration for all adaptive surgical RCTs performed from database inception to October 12, 2023. We included any published trials that had at least one surgical arm. All review and abstraction were performed in duplicate. Risk of bias (RoB) was assessed using the RoB 2.0 instrument and reporting quality was evaluated using CONSORT ACE 2020. All results were analyzed using descriptive methods. Findings Of the 1338 studies identified, six trials met inclusion criteria. Trials were performed in cardiothoracic, oral, orthopedic, and urological surgery. The most common type of adaptive trial was group sequential design with pre-specified interim analyses planned for efficacy, futility, and/or sample size re-estimation. Two trials did use statistical simulations. Our risk of bias evaluation identified a high risk of bias in 50% of included trials. Reporting quality was heterogeneous regarding trial design and outcome assessment and details in relation to randomization and blinding concealment. Conclusion and relevance Surgical trialists should consider implementing adaptive components to help improve patient recruitment and reduce trial duration. Reporting of future adaptive trials must adhere to existing CONSORT ACE 2020 guidelines. Future research is needed to optimize standardization of adaptive methods across medicine and surgery.

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