{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,23]],"date-time":"2026-09-23T12:22:46Z","timestamp":1790166166716,"version":"4.1.0"},"reference-count":42,"publisher":"Wiley","issue":"11","license":[{"start":{"date-parts":[[2025,6,17]],"date-time":"2025-06-17T00:00:00Z","timestamp":1750118400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Head &amp; Neck"],"published-print":{"date-parts":[[2025,11]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Background<\/jats:title>\n                    <jats:p>Time\/resource constraints might preclude a randomized controlled trial. Single\u2010arm oncology trials with historical controls are an alternative. With causal inference, treatment effect estimates can be computed in the absence of randomization.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>From a single\u2010arm trial of 39 head and neck squamous cell carcinoma patients treated with adjuvant nivolumab, we compare 2\u2010year disease\u2010free survival (DFS) to untreated historical controls. We resort to the potential outcomes framework known as Rubin's causal model (RCM). For time\u2010to\u2010event outcomes, RCM relies upon survival analysis regression with baseline covariates. We contrast the average treatment effect (ATE) estimated by three survival methods: Cox proportional hazards (CPH) versus machine learning alternatives, random survival forests (RSF), and Bayesian Additive Regression Trees (BART).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>The ATE in favor of nivolumab: CPH 0.202 (0.098\u20130.306); RSF 0.159 (0.070\u20130.248); and BART 0.268 (0.126\u20130.406).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>The uncertainty is considerable, yet all three methods show nivolumab is superior to control.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/hed.28215","type":"journal-article","created":{"date-parts":[[2025,6,17]],"date-time":"2025-06-17T05:14:24Z","timestamp":1750137264000},"page":"2990-2997","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":2,"title":["Treatment Effect Estimation With Potential Outcomes for a Single\u2010Arm Trial Compared With Historical Controls: A Case Study of Locally Recurrent Head and Neck Squamous Cell Carcinoma Patients Treated With Adjuvant Nivolumab"],"prefix":"10.1002","volume":"47","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-0802-3413","authenticated-orcid":false,"given":"Nusrat","family":"Harun","sequence":"first","affiliation":[{"name":"Cytel Inc  Cincinnati Ohio USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7780-6825","authenticated-orcid":false,"given":"Rodney","family":"Sparapani","sequence":"additional","affiliation":[{"name":"Division of Biostatistics Medical College of Wisconsin  Milwaukee Wisconsin USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0433-4193","authenticated-orcid":false,"given":"Purushottam W.","family":"Laud","sequence":"additional","affiliation":[{"name":"Division of Biostatistics Medical College of Wisconsin  Milwaukee Wisconsin USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Siva","family":"Sivaganesan","sequence":"additional","affiliation":[{"name":"Division of Statistics and Data Science, Department of Mathematical Sciences University of Cincinnati  Cincinnati Ohio USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jennifer L.","family":"Leddon","sequence":"additional","affiliation":[{"name":"Division of Hematology\/Oncology, Department of Internal Medicine University of Cincinnati  Cincinnati Ohio USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sulsal","family":"Haque","sequence":"additional","affiliation":[{"name":"Division of Hematology\/Oncology, Department of Internal Medicine University of Cincinnati  Cincinnati Ohio USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alice L.","family":"Tang","sequence":"additional","affiliation":[{"name":"Department of Otolaryngology\u2010Head and Neck Surgery University of Cincinnati  Cincinnati Ohio USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Trisha M.","family":"Wise\u2010Draper","sequence":"additional","affiliation":[{"name":"Division of Hematology\/Oncology, Department of Internal Medicine University of Cincinnati  Cincinnati Ohio USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2025,6,17]]},"reference":[{"key":"e_1_2_7_2_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00405-009-1070-0"},{"key":"e_1_2_7_3_1","doi-asserted-by":"publisher","DOI":"10.1097\/00005537-200003001-00001"},{"key":"e_1_2_7_4_1","doi-asserted-by":"publisher","DOI":"10.1002\/cncr.24595"},{"key":"e_1_2_7_5_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.oraloncology.2018.04.008"},{"key":"e_1_2_7_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(19)32591-7"},{"key":"e_1_2_7_7_1","doi-asserted-by":"publisher","DOI":"10.1158\/1078-0432.CCR-21-4554"},{"key":"e_1_2_7_8_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.oraloncology.2018.11.010"},{"key":"e_1_2_7_9_1","volume-title":"Causal Inference: What If","author":"Hernan M. 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