{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,12,10]],"date-time":"2025-12-10T08:18:08Z","timestamp":1765354688561,"version":"3.40.5"},"reference-count":24,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2019,12,30]],"date-time":"2019-12-30T00:00:00Z","timestamp":1577664000000},"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":["Pharmaceutical Statistics"],"published-print":{"date-parts":[[2020,5]]},"abstract":"<jats:title>Summary<\/jats:title><jats:p>The design of a clinical trial is often complicated by the multi\u2010systemic nature of the disease; a single endpoint often cannot capture the spectrum of potential therapeutic benefits. Multi\u2010domain outcomes which take into account patient heterogeneity of disease presentation through measurements of multiple symptom\/functional domains are an attractive alternative to a single endpoint. A multi\u2010domain test with adaptive weights is proposed to synthesize the evidence of treatment efficacy over numerous disease domains. The test is a weighted sum of domain\u2010specific test statistics with weights selected adaptively via a data\u2010driven algorithm. The null distribution of the test statistic is constructed empirically through resampling and does not require estimation of the covariance structure of domain\u2010specific test statistics. Simulations show that the proposed test controls the type I error rate, and has increased power over other methods such as the O'Brien and Wei\u2010Lachin tests in scenarios reflective of clinical trial settings. Data from a clinical trial in a rare lysosomal storage disorder were used to illustrate the properties of the proposed test. As a strategy of combining marginal test statistics, the proposed test is flexible and readily applicable to a variety of clinical trial scenarios.<\/jats:p>","DOI":"10.1002\/pst.1993","type":"journal-article","created":{"date-parts":[[2019,12,30]],"date-time":"2019-12-30T11:08:01Z","timestamp":1577704081000},"page":"315-325","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":3,"title":["A flexible multi\u2010domain test with adaptive weights and its application to clinical trials"],"prefix":"10.1002","volume":"19","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-8320-6992","authenticated-orcid":false,"given":"Yang","family":"Zhao","sequence":"first","affiliation":[{"name":"Gilead Sciences Foster City California"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Qifeng","family":"Yu","sequence":"additional","affiliation":[{"name":"Sanofi R&amp;D Framingham Massachusetts"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Stephen L.","family":"Lake","sequence":"additional","affiliation":[{"name":"Wave Life Sciences Lexington Massachusetts"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2019,12,30]]},"reference":[{"key":"e_1_2_8_2_1","doi-asserted-by":"publisher","DOI":"10.1002\/pst.301"},{"key":"e_1_2_8_3_1","doi-asserted-by":"publisher","DOI":"10.1080\/10543406.2018.1489402"},{"volume-title":"Multiple sclerosis functional composite (MSFC) administration and scoring manual","year":"2001","author":"Fischer JS","key":"e_1_2_8_4_1"},{"key":"e_1_2_8_5_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000004743"},{"key":"e_1_2_8_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jacc.2007.10.034"},{"key":"e_1_2_8_7_1","doi-asserted-by":"publisher","DOI":"10.2307\/2531158"},{"key":"e_1_2_8_8_1","doi-asserted-by":"publisher","DOI":"10.1002\/sim.3182"},{"key":"e_1_2_8_9_1","doi-asserted-by":"publisher","DOI":"10.1002\/pst.268"},{"key":"e_1_2_8_10_1","doi-asserted-by":"publisher","DOI":"10.1080\/01621459.1984.10478093"},{"key":"e_1_2_8_11_1","doi-asserted-by":"publisher","DOI":"10.1002\/sim.4780110903"},{"key":"e_1_2_8_12_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0108784"},{"key":"e_1_2_8_13_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0378-3758(99)00032-4"},{"key":"e_1_2_8_14_1","doi-asserted-by":"publisher","DOI":"10.1093\/biostatistics\/4.2.223"},{"key":"e_1_2_8_15_1","doi-asserted-by":"publisher","DOI":"10.1002\/gepi.2001.21.s1.s479"},{"key":"e_1_2_8_16_1","doi-asserted-by":"publisher","DOI":"10.1002\/sim.6591"},{"key":"e_1_2_8_17_1","doi-asserted-by":"publisher","DOI":"10.1214\/aoms\/1177732979"},{"key":"e_1_2_8_18_1","doi-asserted-by":"publisher","DOI":"10.1093\/biomet\/52.1-2.203"},{"key":"e_1_2_8_19_1","first-page":"690","article-title":"Evaluation of survival data and two new rank order statistics arising in its consideration","volume":"50","author":"Mantel N","year":"1966","journal-title":"Cancer Chemother Rep"},{"key":"e_1_2_8_20_1","doi-asserted-by":"crossref","first-page":"187","DOI":"10.1111\/j.2517-6161.1972.tb00899.x","article-title":"Regression models and life tables","volume":"34","author":"Cox DR","year":"1972","journal-title":"J R Stat Soc"},{"key":"e_1_2_8_21_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jpeds.2004.01.046"},{"key":"e_1_2_8_22_1","doi-asserted-by":"publisher","DOI":"10.1002\/sim.3495"},{"key":"e_1_2_8_23_1","doi-asserted-by":"publisher","DOI":"10.1161\/01.STR.27.11.2136"},{"key":"e_1_2_8_24_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1541-0420.2008.01067.x"},{"key":"e_1_2_8_25_1","doi-asserted-by":"publisher","DOI":"10.1002\/bimj.200710456"}],"container-title":["Pharmaceutical Statistics"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.wiley.com\/onlinelibrary\/tdm\/v1\/articles\/10.1002%2Fpst.1993","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/pst.1993","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/pst.1993","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/pst.1993","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,7,29]],"date-time":"2024-07-29T09:39:32Z","timestamp":1722245972000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/pst.1993"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2019,12,30]]},"references-count":24,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2020,5]]}},"alternative-id":["10.1002\/pst.1993"],"URL":"https:\/\/doi.org\/10.1002\/pst.1993","archive":["Portico"],"relation":{},"ISSN":["1539-1604","1539-1612"],"issn-type":[{"type":"print","value":"1539-1604"},{"type":"electronic","value":"1539-1612"}],"subject":[],"published":{"date-parts":[[2019,12,30]]},"assertion":[{"value":"2018-08-20","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2019-11-21","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2019-12-30","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}