Emulating a trial of joint dynamic strategies: An application to monitoring and treatment of HIV-positive individuals

被引：18

作者：

Caniglia, Ellen C. ^{[1
,2
]}

Robins, James M. ^{[1
,3
]}

Cain, Lauren E. ^{[1
]}

Sabin, Caroline ^{[4
]}

Logan, Roger ^{[1
]}

Abgrall, Sophie ^{[5
]}

Mugavero, Michael J. ^{[6
]}

Hernandez-Diaz, Sonia ^{[1
]}

Meyer, Laurence ^{[7
]}

Seng, Remonie ^{[7
]}

Drozd, Daniel R. ^{[8
]}

Seage, George R., III ^{[1
]}

Bonnet, Fabrice ^{[9
]}

Le Marec, Fabien ^{[9
]}

Moore, Richard D. ^{[10
]}

Reiss, Peter ^{[11
]}

van Sighem, Ard ^{[11
]}

Mathews, William C. ^{[12
]}

Jarrin, Inma ^{[13
]}

Alejos, Belen ^{[13
]}

Deeks, Steven G. ^{[14
]}

Muga, Roberto ^{[15
]}

Boswell, Stephen L. ^{[16
]}

Ferrer, Elena ^{[17
]}

Eron, Joseph J. ^{[18
]}

Gill, John ^{[19
]}

Pacheco, Antonio ^{[20
]}

Grinsztejn, Beatriz ^{[20
]}

Napravnik, Sonia ^{[18
]}

Jose, Sophie ^{[4
]}

Phillips, Andrew ^{[4
]}

Justice, Amy ^{[21
]}

Tate, Janet ^{[21
]}

Bucher, Heiner C. ^{[22
]}

Egger, Matthias ^{[23
]}

Furrer, Hansjakob ^{[24
]}

Miro, Jose M. ^{[25
]}

Casabona, Jordi ^{[26
]}

Porter, Kholoud ^{[4
]}

Touloumi, Giota ^{[27
]}

Crane, Heidi ^{[8
]}

Costagliola, Dominique ^{[28
]}

Saag, Michael ^{[29
]}

Hernan, Miguel A. ^{[1
,3
,30
]}

机构：

[1] Harvard TH Chan Sch Publ Hlth, Dept Epidemiol, Boston, MA USA

[2] NYU, Dept Populat Hlth, Sch Med, 227 East 30th St, New York, NY 10016 USA

[3] Harvard TH Chan Sch Publ Hlth, Dept Biostat, Boston, MA USA

[4] UCL, London, England

[5] Hop Avicenne, AP HP, Bobigny, France

[6] Univ Alabama Birmingham, Sch Med, Birmingham, AL USA

[7] Univ Paris Sud, Inserm, Orsay, France

[8] Univ Washington, Seattle, WA 98195 USA

[9] Ctr Hosp Univ Bordeaux, Bordeaux, France

[10] Johns Hopkins Univ, Sch Med, Baltimore, MD USA

[11] Acad Med Ctr Geneeskunde, Amsterdam, Netherlands

[12] Univ Calif San Diego Hlth, Dept Med, San Diego, CA USA

[13] Inst Salud Carlos III, Natl Ctr Epidemiol, Madrid, Spain

[14] Univ Calif San Francisco, Sch Med, San Francisco, CA USA

[15] Germans Trias Hosp, Barcelona, Spain

[16] Fenway Hlth, Boston, MA USA

[17] Hosp Univ Bellvitge, Barcelona, Spain

[18] Univ N Carolina, Sch Med, Dept Med, Chapel Hill, NC USA

[19] Southern Alberta HIV Program, Calgary, AB, Canada

[20] Fundacao Oswaldo Cruz, Rio De Janeiro, Brazil

[21] Yale Univ, Sch Publ Hlth, New Haven, CT USA

[22] Univ Spital Basel, Basel, Switzerland

[23] Univ Bern, Inst Social & Prevent Med, Bern, Switzerland

[24] Univ Bern, Div Infect Dis, Bern Univ Hosp, Bern, Switzerland

[25] Univ Barcelona, Barcelona, Spain

[26] Inst Catala Oncol, Barcelona, Spain

[27] Athens Univ, Dept Hyg Epidemiol & Med Stat, Med Sch, Athens, Greece

[28] Univ Paris 06, Paris, France

[29] Univ Alabama Birmingham, Birmingham, AL USA

[30] Harvard MIT Div Hlth Sci & Technol, Boston, MA USA

来源：

STATISTICS IN MEDICINE | 2019年 / 38卷 / 13期

基金：

美国国家卫生研究院;

关键词：

causal inference; dynamic regime; joint treatment strategies; marginal structural model; no direct effect; COMBINED ANTIRETROVIRAL THERAPY; AIDS-DEFINING ILLNESS; IMMEDIATE INITIATION; TREATMENT REGIMES; REDUCE MORTALITY; COUNTRIES;

D O I：

10.1002/sim.8120

中图分类号：

Q [生物科学];

学科分类号：

07 ; 0710 ; 09 ;

摘要：

Decisions about when to start or switch a therapy often depend on the frequency with which individuals are monitored or tested. For example, the optimal time to switch antiretroviral therapy depends on the frequency with which HIV-positive individuals have HIV RNA measured. This paper describes an approach to use observational data for the comparison of joint monitoring and treatment strategies and applies the method to a clinically relevant question in HIV research: when can monitoring frequency be decreased and when should individuals switch from a first-line treatment regimen to a new regimen? We outline the target trial that would compare the dynamic strategies of interest and then describe how to emulate it using data from HIV-positive individuals included in the HIV-CAUSAL Collaboration and the Centers for AIDS Research Network of Integrated Clinical Systems. When, as in our example, few individuals follow the dynamic strategies of interest over long periods of follow-up, we describe how to leverage an additional assumption: no direct effect of monitoring on the outcome of interest. We compare our results with and without the "no direct effect" assumption. We found little differences on survival and AIDS-free survival between strategies where monitoring frequency was decreased at a CD4 threshold of 350 cells/mu l compared with 500 cells/mu l and where treatment was switched at an HIV-RNA threshold of 1000 copies/ml compared with 200 copies/ml. The "no direct effect" assumption resulted in efficiency improvements for the risk difference estimates ranging from an 7- to 53-fold increase in the effective sample size.

引用

页码：2428 / 2446

页数：19

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