Statistical methods in recent HIV noninferiority Trials: Reanalysis of 11 trials

PLoS ONE

Volumn 6, Issue 9, 2011, Pages

  Flandre, Philippe ^a  

^a INSERM   (France)

Author keywords

[No Author keywords available]

Indexed keywords

ANTIRETROVIRUS AGENT; DARUNAVIR; LOPINAVIR; PROTEINASE INHIBITOR; RALTEGRAVIR; RITONAVIR;

ARTICLE; CLINICAL TRIAL (TOPIC); COMBINATION CHEMOTHERAPY; CONFIDENCE INTERVAL; HIGHLY ACTIVE ANTIRETROVIRAL THERAPY; HUMAN; HUMAN IMMUNODEFICIENCY VIRUS INFECTION; HYPOTHESIS; INTENTION TO TREAT ANALYSIS; MONOTHERAPY; NONINFERIORITY TRIAL; OUTCOMES RESEARCH; SAMPLE SIZE; STATISTICAL ANALYSIS; METHODOLOGY; RANDOMIZED CONTROLLED TRIAL (TOPIC); STATISTICAL MODEL;

CONFIDENCE INTERVALS; HIV INFECTIONS; HUMANS; MODELS, STATISTICAL; RANDOMIZED CONTROLLED TRIALS AS TOPIC; RESEARCH DESIGN;

EID: 80052538046     PISSN: None     EISSN: 19326203     Source Type: Journal    
DOI: 10.1371/journal.pone.0022871     Document Type: Article

References (39)

1. Bartlett JA, Fath MJ, Demasi R, Hermes A, Quinn J, et al. (2006) An updated systematic overview of triple combination therapy in antiretroviral-naive HIV-infected adults. AIDS 20: 2051-2064.
2. Thompson MA, Aberg JA, Cahn P, Montaner JS, Rizzardini G, et al. (2010) Antiretroviral treatment of adult HIV infection: 2010 recommendations of the International AIDS Society-USA panel. JAMA 304: 321-333.
3. Gallant JE, DeJesus E, Arribas JR, Pozniak AL, Gazzard B, et al. (2006) Tenofovir DF, emtricitabine, and efavirenz vs. zidovudine, lamivudine, and efavirenz for HIV. N Engl J Med 354: 251-260.
4. Ortiz R, Dejesus E, Khanlou H, Voronin E, van Lunzen J, et al. (2008) Efficacy and safety of once-daily darunavir/ritonavir versus lopinavir/ritonavir in treatment-naive HIV-1-infected patients at week 48. AIDS 22: 1389-1397.
5. Sax PE, Tierney C, Collier AC, Fischl MA, Mollan K, et al. (2009) Abacavir-lamivudine versus tenofovir-emtricitabine for initial HIV-1 therapy. N Engl J Med 361: 2230-2240.
6. Cahn PFJ, Grinsztejn B, et al. Efficacy and safety at 48 weeks of once-daily vs twice-daily DRV/r in treatment-experienced HIV-1+ patients with no DRV resistance associated mutations: the ODIN Trial. Program and abstracts of the 17th Conference on Retroviruses and Opportunistic Infections; February 16-19, 2010; San Francisco, California. Abstract 57. (2010).
7. Gulick RM, Ribaudo HJ, Shikuma CM, Lustgarten S, Squires KE, et al. (2004) Triple-nucleoside regimens versus efavirenz-containing regimens for the initial treatment of HIV-1 infection. N Engl J Med 350: 1850-1861.
8. Division of Antiviral Drug Products OoDEI, Center for Drug Evaluation and Research (CDER), Food and Drug Administration Guidance for Industry: Antiretroviral Drugs Using Plasma HIV RNA Measurements-Clinical Considerations for Accelerated and Traditional Approval. Available at: http://www.fda.gov/cder/guidance/3647fnl.pdf. Accessed June 18, 2004.
9. Reynes JLA, Pulido F, et al. Lopinavir/ritonavir combined with raltegravir demonstrated similar antiviral efficacy and safety as lopinavir/ritonavir combined with tenofovir disoproxil fumarate/emtricitabine in treatment-naïve HIV-1 infected subjects. Program and abstracts of the XVIII International AIDS Conference; July 18-23, 2010; Vienna, Austria. Abstract MOAB0101.
10. Sierra-Madero J, Villasis-Keever A, Mendez P, Mosqueda-Gomez JL, Torres-Escobar I, et al. (2010) Prospective, randomized, open label trial of Efavirenz vs Lopinavir/Ritonavir in HIV+ treatment-naive subjects with CD4+<200 cell/mm3 in Mexico. J Acquir Immune Defic Syndr 53: 582-588.
11. Arribas JR, Horban A, Gerstoft J, Fatkenheuer G, Nelson M, et al. (2010) The MONET trial: darunavir/ritonavir with or without nucleoside analogues, for patients with HIV RNA below 50 copies/ml. AIDS 24: 223-230.
12. Katlama C, Valantin MA, Algarte-Genin M, Duvivier C, Lambert-Niclot S, et al. (2010) Efficacy of darunavir/ritonavir maintenance monotherapy in patients with HIV-1 viral suppression: a randomized open-label, noninferiority trial, MONOI-ANRS 136. AIDS 24: 2365-2374.
13. Martinez E, Larrousse M, Llibre JM, Gutierrez F, Saumoy M, et al. (2010) Substitution of raltegravir for ritonavir-boosted protease inhibitors in HIV-infected patients: the SPIRAL study. AIDS 24: 1697-1707.
14. Hill A, Sabin C, (2008) Designing and interpreting HIV noninferiority trials in naive and experienced patients. AIDS 22: 913-921.
15. Piaggio G, Elbourne DR, Altman DG, Pocock SJ, Evans SJ, (2006) Reporting of noninferiority and equivalence randomized trials: an extension of the CONSORT statement. JAMA 295: 1152-1160.
16. ICH topic E9. Statistical principles for clinical trials (E9). Geneva SICoHoTRfRoPfHU Available athttp://wwichorg/LOB/media/MEDIA485Pdf.
17. Jones B, Jarvis P, Lewis JA, Ebbutt AF, (1996) Trials to assess equivalence: the importance of rigorous methods. BMJ 313: 36-39.
18. Brittain E, Lin D, (2005) A comparison of intent-to-treat and per-protocol results in antibiotic non-inferiority trials. Stat Med 24: 1-10.
19. Blackwelder WC, (1982) "Proving the null hypothesis" in clinical trials. Control Clin Trials 3: 345-353.
20. Dunnett CW, Gent M, (1977) Significance testing to establish equivalence between treatments, with special reference to data in the form of 2×2 tables. Biometrics 33: 593-602.
21. Chan IS, Zhang Z, (1999) Test-based exact confidence intervals for the difference of two binomial proportions. Biometrics 55: 1202-1209.
22. Farrington CP, Manning G, (1990) Test statistics and sample size formulae for comparative binomial trials with null hypothesis of non-zero risk difference or non-unity relative risk. Stat Med 9: 1447-1454.
23. Miettinen O, Nurminen M, (1985) Comparative analysis of two rates. Stat Med 4: 213-226.
24. Wilson EB, (1927) Probable inference, the law of succession, and statistical inference. Journal of the American Statistical Association 22: 209-212.
25. Santner TJ, Snell MK, (1980) Small-sample confidence intervals for p1-p2 and p1/p2 in 2×2 contingency Tables. Journal of the American Statistical Association 75: 386-394.
26. De Castro N, Braun J, Charreau I, Pialoux G, Cotte L, et al. (2009) Switch from enfuvirtide to raltegravir in virologically suppressed multidrug-resistant HIV-1-infected patients: a randomized open-label trial. Clin Infect Dis 49: 1259-1267.
27. Eron JJ, Young B, Cooper DA, Youle M, Dejesus E, et al. (2010) Switch to a raltegravir-based regimen versus continuation of a lopinavir-ritonavir-based regimen in stable HIV-infected patients with suppressed viraemia (SWITCHMRK 1 and 2): two multicentre, double-blind, randomised controlled trials. Lancet 375: 396-407.
28. Meynard JL, Bouteloup V, Landman R, Bonnard P, Baillat V, et al. (2010) Lopinavir/ritonavir monotherapy versus current treatment continuation for maintenance therapy of HIV-1 infection: the KALESOLO trial. J Antimicrob Chemother 65: 2436-2444.
29. Zajdenverg R, Podsadecki TJ, Badal-Faesen S, Andrade-Villanueva J, Gathe J, et al. (2010) Similar safety and efficacy of once- and twice-daily lopinavir/ritonavir tablets in treatment-experienced HIV-1-infected subjects at 48 weeks. J Acquir Immune Defic Syndr 54: 143-151.
30. Richert L, Bouteloup V, Thiebaut R, Chene G, (2008) Methodological issues of non-inferiority trials in HIV-infected patients: a need for consensus? AIDS 22: 996-997.
31. Guideline on the choice of Non-inferiority margin. London EEMAEJ.
32. Points to consider on switching between superiority and non-inferiority. London EEMAEJ.
33. Daar ES, Tierney C, Fischl MA, Sax PE, Mollan K, et al. (2011) Atazanavir Plus Ritonavir or Efavirenz as Part of a 3-Drug Regimen for Initial Treatment of HIV-1: A Randomized Trial. Ann Intern Med 154: 445-456.
34. Wiens BL, (2002) Choosing an equivalence limit for noninferiority or equivalence studies. Control Clin Trials 23: 2-14.
35. Agresti A, Coull BA, (1998) Approximate is better than 'exact' for interval estimation of binomial proportions. The American Statistician 52: 119-126.
36. Agresti A, Min Y, (2001) On small-sample confidence intervals for parameters in discrete distributions. Biometrics 57: 963-971.
37. Roebruck P, Kuhn A, (1995) Comparison of tests and sample size formulae for proving therapeutic equivalence based on the difference of binomial probabilities. Stat Med 14: 1583-1594.
38. Newcombe RG, (1998) Interval estimation for the difference between independent proportions: comparison of eleven methods. Stat Med 17: 873-890.
39. Barker L, Rolka H, Rolka D, Brown C, (2001) Equivalence testing for binomial random variables: which test to use? The American Statistician 55: 279-287.