Optimal individualized decision rules from a multi-arm trial: A comparison of methods and an application to tailoring inter-donation intervals among blood donors in the UK.
Statistical methods in medical research
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Xu, Y., Wood, A., Sweeting, M. J., Roberts, D. J., & Tom, B. (2020). Optimal individualized decision rules from a multi-arm trial: A comparison of methods and an application to tailoring inter-donation intervals among blood donors in the UK.. Statistical methods in medical research, 29 (11), 3113-3134. https://doi.org/10.1177/0962280220920669
There is a growing interest in precision medicine where individual heterogeneity is incorporated into decision-making and treatments are tailored to individuals to provide better healthcare. One important aspect of precision medicine is the estimation of the optimal individualized treatment rule (ITR) that optimizes the expected outcome. Most methods developed for this purpose are restricted to the setting with two treatments, while clinical studies with more than two treatments are common in practice. In this work, we summarize methods to estimate the optimal ITR in the multi-arm setting and compare their performance in large-scale clinical trials via simulation studies. We then illustrate their utilities with a case study using the data from the INTERVAL trial, which randomly assigned over 20000 male blood donors from England to one of the three inter-donation intervals (12-week, 10-week, and 8-week) over 2 years. We estimate the optimal individualized donation strategies under three different objectives. Our findings are fairly consistent across five different approaches that are applied: when we target the maximization of the total units of blood collected, almost all donors are assigned to the 8-week inter-donation interval, whereas if we aim at minimizing the low haemoglobin deferral rates, almost all donors are assigned to donate every 12 weeks. However, when the goal is to maximize the utility score that “discounts” the total units of blood collected by the incidences of low haemoglobin deferrals, we observe some heterogeneity in the optimal inter-donation interval across donors and the optimal donor assignment strategy is highly dependent on the trade-off parameter in the utility function.
Medical Research Council programme MC_UU_00002/2
British Heart Foundation (RG/13/13/30194)
British Heart Foundation (RG/18/13/33946)
External DOI: https://doi.org/10.1177/0962280220920669
This record's URL: https://www.repository.cam.ac.uk/handle/1810/304021
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