Comparative efficacy versus effectiveness of initial antiretroviral therapy in clinical trials versus routine care.
BACKGROUND
The applicability of clinical trial findings (efficacy) to the routine care setting (effectiveness) may be limited because of study eligibility criteria and volunteer bias. Although well-chronicled in many conditions, the efficacy versus effectiveness of antiretroviral therapy (ART) remains understudied.


METHODS
A retrospective study of the University of Alabama at Birmingham 1917 Clinic Cohort evaluated ART-naive patients who started ART from 1 January 2000 through 31 December 2006. Patients received ART through clinical trials or routine care. Multivariable logistic and linear regression models were fit to evaluate factors associated with virological failure (virological failure was defined as a viral load >50 copies/mL) and change from baseline CD4+ cell count 6 and 12 months after ART initiation. Sensitivity analyses evaluated the impact of missing data on outcomes.


RESULTS
Among 570 patients starting ART during the study period, 121 (21%) enrolled in clinical trials, and 449 (79%) received ART via routine care. ART receipt through routine care was not associated with viral failure at either 6 months (odds ratio [OR], 1.00; 95% confidence interval [CI], 0.54-1.86) or 12 months (OR, 1.56; 95% CI, 0.80-3.05) in primary analyses. No statistically significant differences in CD4+ cell count responses at 6 and 12 months were observed.


CONCLUSIONS
Although marked differences in efficacy versus effectiveness have been observed in the therapeutic outcomes of other conditions, our analyses found no evidence of such divergence among our patients who initiated antiretroviral therapy for human immunodeficiency virus infection.
Routman
Justin S
JS
Divisions of Infectious Diseases, Department of Medicine, University of Alabama School of Medicine, University of Alabama at Birmingham, Birmingham, AL 35294-2050, USA.
Willig
James H
JH
Westfall
Andrew O
AO
Abroms
Sarah R
SR
Varshney
Mohit
M
Adusumilli
Sunil
S
Allison
Jeroan J
JJ
Savage
Karen G
KG
Saag
Michael S
MS
Mugavero
Michael J
MJ
D000328:Adult; D044966:Anti-Retroviral Agents / Q000008:administration & dosage*; D018791:CD4 Lymphocyte Count; D005260:Female; D006678:HIV / Q000302:isolation & purification; D015658:HIV Infections / Q000188:drug therapy* / Q000453:epidemiology / Q000276:immunology / Q000821:virology; D006801:Humans; D008297:Male; D008875:Middle Aged; D015999:Multivariate Analysis; D012367:RNA, Viral / Q000097:blood; D012044:Regression Analysis; D012189:Retrospective Studies; D012307:Risk Factors; D012680:Sensitivity and Specificity; D016896:Treatment Outcome; D019562:Viral Load
D044966:Anti-Retroviral Agents; D012367:RNA, Viral