Estimating Efficacy in a Randomized Trial With Product Nonadherence: Application of Multiple Methods to a Trial of Preexposure Prophylaxis for HIV Prevention

American Journal of Epidemiology

Volumn 182, Issue 10, 2015, Pages 848-856

  Murnane, Pamela M ^a,b   Brown, Elizabeth R ^a,c   Donnell, Deborah ^a,c   Coley, R Yates ^a   Mugo, Nelly ^a,e   Mujugira, Andrew ^a   Celum, Connie ^a,d   Baeten, Jared M ^a,d  

^a UNIVERSITY OF WASHINGTON   (United States)

^b Vagelos College of Physicians and Surgeons   (United States)

^c Fred Hutchinson Cancer Research Center   (United States)

^d UNIVERSITY OF WASHINGTON SCHOOL OF MEDICINE   (United States)

^e KENYA MEDICAL RESEARCH INSTITUTE   (Kenya)

Author keywords

HIV; HIV prevention; intention to treat analysis; medication adherence; randomized controlled trials

Indexed keywords

EMTRICITABINE PLUS TENOFOVIR DISOPROXIL; PLACEBO; ANTI HUMAN IMMUNODEFICIENCY VIRUS AGENT;

CONFIDENCE INTERVAL; DISEASE INCIDENCE; DISEASE TREATMENT; DRUG PRESCRIBING; DRUG USER; EFFICIENCY MEASUREMENT; HEALTH RISK; HUMAN IMMUNODEFICIENCY VIRUS; MEDICAL GEOGRAPHY; STUDY METHOD;

ADULT; AREA UNDER THE CURVE; ARTICLE; CD4 LYMPHOCYTE COUNT; COHABITATION; DOUBLE BLIND PROCEDURE; DRUG BLOOD LEVEL; DRUG EFFICACY; EDUCATIONAL STATUS; FEMALE; HETEROSEXUALITY; HUMAN; HUMAN IMMUNODEFICIENCY VIRUS 1 INFECTION; INFECTION PREVENTION; INTENTION TO TREAT ANALYSIS; MAJOR CLINICAL STUDY; MALE; MEDICATION COMPLIANCE; PRE-EXPOSURE PROPHYLAXIS; RANDOMIZED CONTROLLED TRIAL (TOPIC); RECEIVER OPERATING CHARACTERISTIC; SEROCONVERSION; TREATMENT REFUSAL; UNPROTECTED SEX; VIRUS LOAD; ACQUIRED IMMUNODEFICIENCY SYNDROME; AGE; CONTROLLED STUDY; HIV INFECTIONS; INCIDENCE; KENYA; METHODOLOGY; PATIENT SATISFACTION; PROCEDURES; RANDOMIZED CONTROLLED TRIAL; SEX DIFFERENCE; SOCIOECONOMICS; STATISTICAL MODEL; STATISTICS AND NUMERICAL DATA;

KENYA; UGANDA;

HUMAN IMMUNODEFICIENCY VIRUS;

ACQUIRED IMMUNODEFICIENCY SYNDROME; ADULT; AGE FACTORS; ANTI-HIV AGENTS; DOUBLE-BLIND METHOD; EMTRICITABINE, TENOFOVIR DISOPROXIL FUMARATE DRUG COMBINATION; FEMALE; HETEROSEXUALITY; HIV INFECTIONS; HUMANS; INCIDENCE; KENYA; MALE; MEDICATION ADHERENCE; MODELS, STATISTICAL; PATIENT SATISFACTION; PRE-EXPOSURE PROPHYLAXIS; RESEARCH DESIGN; SEX FACTORS; SOCIOECONOMIC FACTORS;

EID: 84947580609     PISSN: 00029262     EISSN: 14766256     Source Type: Journal    
DOI: 10.1093/aje/kwv202     Document Type: Article

References (32)

1. Joint United Nations Programme on HIV/AIDS. Global Report: UNAIDS Report on the Global AIDS Epidemic 2013. Geneva, Switzerland: Joint United Nations Programme on HIV/AIDS; 2013.
2. Baeten JM, Donnell D, Ndase P, et al. Antiretroviral prophylaxis for HIV prevention in heterosexual men and women. N Engl J Med. 2012;367(5):399-410.
3. Grant RM, Lama JR, Anderson PL, et al. Preexposure chemoprophylaxis for HIV prevention in men who have sex with men. N Engl J Med. 2010;363(27):2587-2599.
4. Thigpen MC, Kebaabetswe PM, Paxton LA, et al. Antiretroviral preexposure prophylaxis for heterosexual HIV transmission in Botswana. N Engl J Med. 2012;367(5):423-434.
5. Choopanya K, Martin M, Suntharasamai P, et al. Antiretroviral prophylaxis for HIV infection in injecting drug users in Bangkok, Thailand (the Bangkok Tenofovir Study): a randomised, double-blind, placebo-controlled phase 3 trial. Lancet. 2013;381(9883):2083-2090.
6. Marrazzo JM, Ramjee G, Richardson BA, et al. Tenofovir-based preexposure prophylaxis for HIV infection among African women. N Engl J Med. 2015;372(6):509-518.
7. Van Damme L, Corneli A, Ahmed K, et al. Preexposure prophylaxis for HIV infection among African women. N Engl J Med. 2012;367(5):411-422.
8. Rubin DB. Estimating causal effects of treatments in randomized and nonrandomized studies. J Educ Psychol. 1974;66(5):688-701.
9. Greenland S, Robins JM. Identifiability, exchangeability, and epidemiological confounding. Int J Epidemiol. 1986;15(3):413-419.
10. Frangakis CE, Rubin DB. Principal stratification in causal inference. Biometrics. 2002;58(1):21-29.
11. Robins JM, Finkelstein DM. Correcting for noncompliance and dependent censoring in an AIDS clinical trial with inverse probability of censoring weighted (IPCW) log-rank tests. Biometrics. 2000;56(3):779-788.
12. Robins JM, Tsiatis AA. Correcting for non-compliance in randomized trials using rank-preserving structural failure time models. Commun Stat Theory Methods. 1991;20(8):2609-2631.
13. Imbens G, Rubin DB. Bayesian inference for causal effects in randomized experiments with noncompliance. Ann Stat. 1997;25(1):305-327.
14. Loeys T, Goetghebeur E. A causal proportional hazards estimator for the effect of treatment actually received in a randomized trial with all-or-nothing compliance. Biometrics. 2003;59(1):100-105.
15. Angrist JD, Imbens GW, Rubin DB. Identification of causal effects using instrumental variables. J Am Stat Assoc. 1996;91(434):444-455.
16. Donnell DJ, Baeten JM, Hong T, et al. Correlation between pill counts and biologic effects in an HIV-1 prevention clinical trial: implications for measuring adherence. AIDS Behav. 2013;17(2):632-639.
17. Donnell D, Baeten JM, Bumpus NN, et al. HIV protective efficacy and correlates of tenofovir blood concentrations in a clinical trial of PrEP for HIV prevention. J Acquir Immune Defic Syndr. 2014;66(3):340-348.
18. Celum C, Morrow RA, Donnell D, et al. Daily oral tenofovir and emtricitabine-tenofovir preexposure prophylaxis reduces herpes simplex virus type 2 acquisition among heterosexual HIV-1-uninfected men and women: a subgroup analysis of a randomized trial. Ann Intern Med. 2014;161(1):11-19.
19. Follmann DA. On the effect of treatment among treatment compliers: an analysis of the Multiple Risk Factor Intervention Trial. J Am Stat Assoc. 2000;95(452):1101-1109.
20. Haberer JE, Baeten JM, Campbell J, et al. Adherence to antiretroviral prophylaxis for HIV prevention: a substudy cohort within a clinical trial of serodiscordant couples in East Africa. PLoS Med. 2013;10(9):e1001511.
21. Akaike H. A new look at the statistical model identification. IEEE Trans Automat Contr. 1974;19(6):716-723.
22. Gould MK, Ananth L, Barnett PG, et al. A clinical model to estimate the pretest probability of lung cancer in patients with solitary pulmonary nodules. Chest. 2007;131(2):383-388.
23. Wacholder S, Gail MH, Pee D, et al. Alternative variance and efficiency calculations for the case-cohort design. Biometrika. 1989;76(1):117-123.
24. Cole SR, Hernán MA. Constructing inverse probability weights for marginal structural models. Am J Epidemiol. 2008;168(6):656-664.
25. Hernán MA, Brumback B, Robins JM. Marginal structural models to estimate the causal effect of zidovudine on the survival of HIV-positive men. Epidemiology. 2000;11(5):561-570.
26. Harrell FE. Regression Modeling Strategies - With Applications to Linear Models, Logistic Regression, and Survival Analysis. 1st ed. (Springer Series in Statistics). New York, NY: Springer-Verlag New York; 2001.
27. Rubin DB. More powerful randomization-based p-values in double-blind trials with non-compliance. Stat Med. 1998;17(3):371-385.
28. Dai JY, Gilbert PB, Hughes JP, et al. Estimating the efficacy of preexposure prophylaxis for HIV prevention among participants with a threshold level of drug concentration. Am J Epidemiol. 2013;177(3):256-263.
29. Hill JL, Brooks-Gunn J, Waldfogel J. Sustained effects of high participation in an early intervention for low-birth-weight premature infants. Dev Psychol. 2003;39(4):730-744.
30. Jo B, Stuart EA. On the use of propensity scores in principal causal effect estimation. Stat Med. 2009;28(23):2857-2875.
31. Joffe MM, Ten Have TR, Brensinger C. The compliance score as a regressor in randomized trials. Biostatistics. 2003;4(3):327-340.
32. Shrier I, Steele RJ, Verhagen E, et al. Beyond intention to treat: what is the right question? Clin Trials. 2014;11(1):28-37.