{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,29]],"date-time":"2026-07-29T11:09:16Z","timestamp":1785323356430,"version":"3.55.0"},"reference-count":21,"publisher":"Oxford University Press (OUP)","issue":"3","license":[{"start":{"date-parts":[[2018,1,22]],"date-time":"2018-01-22T00:00:00Z","timestamp":1516579200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/academic.oup.com\/journals\/pages\/open_access\/funder_policies\/chorus\/standard_publication_model"}],"funder":[{"DOI":"10.13039\/100000002","name":"NIH","doi-asserted-by":"publisher","award":["P30-CA016672"],"award-info":[{"award-number":["P30-CA016672"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000002","name":"NIH","doi-asserted-by":"publisher","award":["P30-CA077598"],"award-info":[{"award-number":["P30-CA077598"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2018,9,1]]},"abstract":"<jats:title>Summary<\/jats:title>\n               <jats:p>Traditional paradigms for clinical translation are challenged in settings where multiple contemporaneous therapeutic strategies have been identified as potentially beneficial. Platform trials have emerged as an approach for sequentially comparing multiple trials using a single protocol. The Ebola virus disease outbreak in West Africa represents one recent example which utilized a platform design. Specifically, the PREVAIL II master protocol sequentially tested new combinations of therapies against the concurrent, optimal standard of care (oSOC) strategy. Once a treatment demonstrated sufficient evidence of benefit, the treatment was added to the oSOC for all future comparisons (denoted as segments throughout the manuscript). In the interest of avoiding bias stemming from population drift, PREVAIL II considered only within-segment comparisons between the oSOC and novel treatments and failed to leverage data from oSOC patients in prior segments. This article describes adaptive design methodology aimed at boosting statistical power through Bayesian modeling and adaptive randomization. Specifically, the design uses multi-source exchangeability models to combine data from multiple segments and adaptive randomization to achieve information balance within a segment. When compared to the PREVAIL II design, we demonstrate that our proposed adaptive platform design improves power by as much as 51% with limited type-I error inflation. Further, the adaptive platform effectuates more balance with respect to the distribution of acquired information among study arms, with more patients randomized to experimental regimens.<\/jats:p>","DOI":"10.1111\/biom.12841","type":"journal-article","created":{"date-parts":[[2018,1,23]],"date-time":"2018-01-23T04:46:42Z","timestamp":1516682802000},"page":"1082-1094","source":"Crossref","is-referenced-by-count":50,"title":["A Multi-Source Adaptive Platform Design for Testing Sequential Combinatorial Therapeutic Strategies"],"prefix":"10.1093","volume":"74","author":[{"given":"Alexander M.","family":"Kaizer","sequence":"first","affiliation":[{"name":"Division of Biostatistics, University of Minnesota , Minneapolis, Minnesota , U.S.A."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Brian P.","family":"Hobbs","sequence":"additional","affiliation":[{"name":"The University of Texas MD Anderson Cancer Center , Houston, Texas , U.S.A."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6147-3168","authenticated-orcid":false,"given":"Joseph S.","family":"Koopmeiners","sequence":"additional","affiliation":[{"name":"Division of Biostatistics, University of Minnesota , Minneapolis, Minnesota , U.S.A."}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"286","published-online":{"date-parts":[[2018,1,22]]},"reference":[{"key":"2024011406444520500_biom12841-bib-0001","doi-asserted-by":"crossref","DOI":"10.1016\/S0140-6736(14)61734-7","article-title":"Randomised controlled trials for ebola: Practical and ethical issues","volume":"384","author":"Adebamowo","year":"2014","journal-title":"Lancet"},{"key":"2024011406444520500_biom12841-bib-0002","doi-asserted-by":"crossref","first-page":"231","DOI":"10.1002\/sim.4780140302","article-title":"Adaptive assignment versus balanced randomization in clinical trials: A decision analysis","volume":"14","author":"Berry","year":"1995","journal-title":"Statistics in Medicine"},{"key":"2024011406444520500_biom12841-bib-0003","doi-asserted-by":"crossref","first-page":"1177","DOI":"10.1111\/j.0006-341X.2000.01177.x","article-title":"Sequential designs for phase i clinical trials with late-onset toxicities","volume":"56","author":"Cheung","year":"2000","journal-title":"Biometrics"},{"key":"2024011406444520500_biom12841-bib-0004","doi-asserted-by":"crossref","first-page":"1906","DOI":"10.1093\/infdis\/jiw061","article-title":"Design of a randomized controlled trial for ebola virus disease medical countermeasures: PREVAIL II, the ebola MCM study","volume":"213","author":"Dodd","year":"2016","journal-title":"The Journal of Infectious Diseases"},{"key":"2024011406444520500_biom12841-bib-0005","doi-asserted-by":"crossref","first-page":"430","DOI":"10.1177\/1740774513483934","article-title":"Adaptive adjustment of the randomization ratio using historical control data","volume":"10","author":"Hobbs","year":"2013","journal-title":"Clinical Trials"},{"key":"2024011406444520500_biom12841-bib-0006","article-title":"Controlled multi-arm platform design using predictive probability","author":"Hobbs","year":"2016","journal-title":"Statistical Methods in Medical Research"},{"key":"2024011406444520500_biom12841-bib-0007","doi-asserted-by":"crossref","first-page":"639","DOI":"10.1214\/12-BA722","article-title":"Commensurate priors for incorporating historical information in clinical trials using general and generalized linear models","volume":"7","author":"Hobbs","year":"2012","journal-title":"Bayesian Analysis"},{"key":"2024011406444520500_biom12841-bib-0008","doi-asserted-by":"crossref","first-page":"407","DOI":"10.1016\/S1473-3099(16)00132-8","article-title":"Non-randomised ebola trials: Lessons for optimal outbreak research","volume":"16","author":"Ippolito","year":"2016","journal-title":"The Lancet Infectious Diseases"},{"key":"2024011406444520500_biom12841-bib-0009","article-title":"Dynamic multi-resolution smoothing using multi-source exchangeability models","author":"Kaizer","year":"2017","journal-title":"Biostatistics"},{"key":"2024011406444520500_biom12841-bib-0010","doi-asserted-by":"crossref","first-page":"38","DOI":"10.1177\/1740774513500589","article-title":"A bayesian adaptive phase i\u2013ii clinical trial for evaluating efficacy and toxicity with delayed outcomes","volume":"11","author":"Koopmeiners","year":"2014","journal-title":"Clinical Trials"},{"key":"2024011406444520500_biom12841-bib-0011","doi-asserted-by":"crossref","first-page":"5","DOI":"10.1177\/1740774509356002","article-title":"Summarizing historic information on controls in clinical trials","volume":"7","author":"Neuenschwander","year":"2010","journal-title":"Clinical Trials"},{"key":"2024011406444520500_biom12841-bib-0012","doi-asserted-by":"crossref","first-page":"1761","DOI":"10.1002\/sim.3978","article-title":"Response-adaptive randomization for clinical trials with adjustment for covariate imbalance","volume":"29","author":"Ning","year":"2010","journal-title":"Statistics in Medicine"},{"key":"2024011406444520500_biom12841-bib-0013","doi-asserted-by":"crossref","first-page":"1448","DOI":"10.1056\/NEJMoa1604330","article-title":"A randomized, controlled trial of zmapp for ebola virus infection","volume":"375","author":"The PREVAIL II Writing Group, for the Multi-National PREVAIL II Study Team","year":"2016","journal-title":"The New England Journal of Medicine"},{"key":"2024011406444520500_biom12841-bib-0014","doi-asserted-by":"crossref","first-page":"39","DOI":"10.1177\/1740774515620145","article-title":"Statistical considerations for a trial of ebola virus disease therapeutics","volume":"13","author":"Proschan","year":"2016","journal-title":"Clinical Trials"},{"key":"2024011406444520500_biom12841-bib-0015","doi-asserted-by":"crossref","first-page":"34","DOI":"10.1093\/annonc\/mdw413","article-title":"Statistical controversies in clinical research: Basket trials, umbrella trials, and other master protocols: A review and examples","volume":"28","author":"Renfro","year":"2016","journal-title":"Annals of Oncology"},{"key":"2024011406444520500_biom12841-bib-0016","doi-asserted-by":"crossref","first-page":"2092","DOI":"10.1056\/NEJMoa1411680","article-title":"Clinical illness and outcomes in patients with ebola in sierra leone","volume":"371","author":"Schieffelin","year":"2014","journal-title":"New England Journal of Medicine"},{"key":"2024011406444520500_biom12841-bib-0017","doi-asserted-by":"crossref","first-page":"1621","DOI":"10.1093\/annonc\/mdv238","article-title":"Statistical controversies in clinical research: Scientific and ethical problems with adaptive randomization in comparative clinical trials","volume":"26","author":"Thall","year":"2015","journal-title":"Annals of Oncology"},{"key":"2024011406444520500_biom12841-bib-0018","doi-asserted-by":"crossref","first-page":"859","DOI":"10.1016\/j.ejca.2007.01.006","article-title":"Practical bayesian adaptive randomisation in clinical trials","volume":"43","author":"Thall","year":"2007","journal-title":"European Journal of Cancer"},{"key":"2024011406444520500_biom12841-bib-0019","author":"WHO","year":"2016","journal-title":"Ebola virus disease"},{"key":"2024011406444520500_biom12841-bib-0020","doi-asserted-by":"crossref","first-page":"219","DOI":"10.1111\/j.1467-9876.2011.01006.x","article-title":"Phase ii trial design with bayesian adaptive randomization and predictive probability","volume":"61","author":"Yin","year":"2012","journal-title":"Journal of the Royal Statistical Society, Series C (Applied Statistics)"},{"key":"2024011406444520500_biom12841-bib-0021","doi-asserted-by":"crossref","first-page":"181","DOI":"10.1177\/1740774508091815","article-title":"Bayesian adaptive design for targeted therapy development in lung cancer\u2013a step toward personalized medicine","volume":"5","author":"Zhou","year":"2008","journal-title":"Clinical Trials"}],"container-title":["Biometrics"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.wiley.com\/onlinelibrary\/tdm\/v1\/articles\/10.1111%2Fbiom.12841","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/academic.oup.com\/biometrics\/article-pdf\/74\/3\/1082\/55684455\/biometrics_74_3_1082.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"syndication"},{"URL":"https:\/\/academic.oup.com\/biometrics\/article-pdf\/74\/3\/1082\/55684455\/biometrics_74_3_1082.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,1,26]],"date-time":"2024-01-26T18:06:22Z","timestamp":1706292382000},"score":1,"resource":{"primary":{"URL":"https:\/\/academic.oup.com\/biometrics\/article\/74\/3\/1082-1094\/7525793"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2018,1,22]]},"references-count":21,"journal-issue":{"issue":"3","published-online":{"date-parts":[[2018,1,22]]},"published-print":{"date-parts":[[2018,9,1]]}},"URL":"https:\/\/doi.org\/10.1111\/biom.12841","relation":{},"ISSN":["0006-341X","1541-0420"],"issn-type":[{"value":"0006-341X","type":"print"},{"value":"1541-0420","type":"electronic"}],"subject":[],"published-other":{"date-parts":[[2018,9]]},"published":{"date-parts":[[2018,1,22]]}}}