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The inverse-probability-of-censoring weighting (IPCW) adjusted win ratio statistic: an unbiased estimator in the presence of independent censoring.
Dong, Gaohong; Mao, Lu; Huang, Bo; Gamalo-Siebers, Margaret; Wang, Jiuzhou; Yu, GuangLei; Hoaglin, David C.
Afiliação
  • Dong G; iStats Inc ., Long Island City, New York, USA.
  • Mao L; Department of Biostatistics and Medical Informatics, University of Wisconsin , Madison, Wisconsin, USA.
  • Huang B; Pfizer Inc ., Groton, Connecticut, USA.
  • Gamalo-Siebers M; Eli Lilly & Company , Indianapolis, Indian, USA.
  • Wang J; ImmunoGen Inc ., Waltham, Massachusetts, USA.
  • Yu G; Eli Lilly & Company , Indianapolis, Indian, USA.
  • Hoaglin DC; Department of Population and Quantitative Health Sciences, University of Massachusetts Medical School , Worcester, Massachusetts, USA.
J Biopharm Stat ; 30(5): 882-899, 2020 09 02.
Article em En | MEDLINE | ID: mdl-32552451
ABSTRACT
The win ratio method has received much attention in methodological research, ad hoc analyses, and designs of prospective studies. As the primary analysis, it supported the approval of tafamidis for the treatment of cardiomyopathy to reduce cardiovascular mortality and cardiovascular-related hospitalization. However, its dependence on censoring is a potential shortcoming. In this article, we propose the inverse-probability-of-censoring weighting (IPCW) adjusted win ratio statistic (i.e., the IPCW-adjusted win ratio statistic) to overcome censoring issues. We consider independent censoring, common censoring across endpoints, and right censoring. We develop an asymptotic variance estimator for the logarithm of the IPCW-adjusted win ratio statistic and evaluate it via simulation. Our simulation studies show that, as the amount of censoring increases, the unadjusted win proportions may decrease greatly. Consequently, the bias of the unadjusted win ratio estimate may increase greatly, producing either an overestimate or an underestimate. We demonstrate theoretically and through simulation that the IPCW-adjusted win ratio statistic gives an unbiased estimate of treatment effect.
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Projetos de Pesquisa / Ensaios Clínicos como Assunto Tipo de estudo: Observational_studies / Prognostic_studies / Risk_factors_studies Limite: Humans Idioma: En Ano de publicação: 2020 Tipo de documento: Article

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Projetos de Pesquisa / Ensaios Clínicos como Assunto Tipo de estudo: Observational_studies / Prognostic_studies / Risk_factors_studies Limite: Humans Idioma: En Ano de publicação: 2020 Tipo de documento: Article