Type | Journal Article |
---|---|
Author | Isabelle L. Smith |
Author | Jane E. Nixon |
Author | Linda Sharples |
URL | http://onlinelibrary.wiley.com/doi/abs/10.1002/sim.8882 |
Rights | © 2021 The Authors. Statistics in Medicine published by John Wiley & Sons, Ltd. |
Volume | n/a |
Issue | n/a |
Publication | Statistics in Medicine |
ISSN | 1097-0258 |
Date | 2021 |
Extra | _eprint: https://onlinelibrary.wiley.com/doi/pdf/10.1002/sim.8882 |
DOI | https://doi.org/10.1002/sim.8882 |
Accessed | 2/12/2021, 11:20:57 AM |
Library Catalog | Wiley Online Library |
Language | en |
Abstract | For clinical trials where participants pass through a number of discrete health states resulting in longitudinal measures over time, there are several potential primary estimands for the treatment effect. Incidence or time to a particular health state are commonly used outcomes but the choice of health state may not be obvious and these estimands do not make full use of the longitudinal assessments. Multistate models have been developed for some diseases and conditions with the purpose of understanding their natural history and have been used for secondary analysis to understand mechanisms of action of treatments. There is little published on the use of multistate models as the primary analysis method and potential implications on design features, such as assessment schedules. We illustrate methods via analysis of data from a motivating example; a Phase III clinical trial of pressure ulcer prevention strategies. We clarify some of the possible estimands that might be considered and we show, via a simulation study, that under some circumstances the sample size could be reduced by half using a multistate model based analysis, without adversely affecting the power of the trial. |
Date Added | 2/12/2021, 11:20:57 AM |
Modified | 2/25/2021, 8:34:44 AM |
Type | Journal Article |
---|---|
Author | Stephen Senn |
Author | Lynda Stevens |
Author | Nish Chaturvedi |
URL | https://pubmed.ncbi.nlm.nih.gov/10734289 |
Volume | 19 |
Pages | 861-877 |
Publication | Stat Med |
Date | 2000 |
Extra | Citation Key: sen00rep tex.citeulike-article-id= 13265118 tex.citeulike-linkout-0= http://dx.doi.org/10.1002/(SICI)1097-0258(20000330)19:6%3C861::AID-SIM407%3E3.0.CO;2-F tex.posted-at= 2014-07-14 14:09:49 tex.priority= 0 |
DOI | 10.1002/(SICI)1097-0258(20000330)19:6<861::AID-SIM407>3.0.CO;2-F |
Date Added | 7/7/2018, 1:38:33 PM |
Modified | 6/12/2020, 6:11:53 AM |
Type | Journal Article |
---|---|
Author | Julio M. Singer |
Author | Dalton F. Andrade |
URL | http://dx.doi.org/10.2307/2533973 |
Volume | 53 |
Pages | 729-735 |
Publication | Biometrics |
Date | 1997 |
Extra | Citation Key: sin97reg tex.citeulike-article-id= 13264865 tex.citeulike-linkout-0= http://dx.doi.org/10.2307/2533973 tex.posted-at= 2014-07-14 14:09:44 tex.priority= 0 |
DOI | 10.2307/2533973 |
Date Added | 7/7/2018, 1:38:33 PM |
Modified | 11/8/2019, 8:01:59 AM |
problems with using baseline measurement as covariate
Type | Journal Article |
---|---|
Author | J. N. S. Matthews |
URL | http://dx.doi.org/10.1002/sim.4780120105 |
Volume | 12 |
Pages | 27-37 |
Publication | Stat Med |
Date | 1993 |
Extra | Citation Key: mat93ref tex.citeulike-article-id= 13264584 tex.citeulike-linkout-0= http://dx.doi.org/10.1002/sim.4780120105 tex.posted-at= 2014-07-14 14:09:38 tex.priority= 0 |
DOI | 10.1002/sim.4780120105 |
Date Added | 7/7/2018, 1:38:33 PM |
Modified | 11/8/2019, 8:01:59 AM |
Type | Journal Article |
---|---|
Author | J. N. S. Matthews |
Author | Douglas G. Altman |
Author | M. J. Campbell |
Author | Patrick Royston |
URL | http://dx.doi.org/10.1136/bmj.300.6719.230 |
Volume | 300 |
Pages | 230-235 |
Publication | BMJ |
Date | 1990 |
Extra | Citation Key: mat90ana tex.citeulike-article-id= 13264583 tex.citeulike-linkout-0= http://dx.doi.org/10.1136/bmj.300.6719.230 tex.posted-at= 2014-07-14 14:09:38 tex.priority= 0 Letter to editor by S. Senn in same issue |
DOI | 10.1136/bmj.300.6719.230 |
Date Added | 7/7/2018, 1:38:33 PM |
Modified | 11/8/2019, 8:01:59 AM |