Sample size formulae for two-stage randomized trials with survival outcomes

Biometrika

Volumn 98, Issue 3, 2011, Pages 503-518

  Li, Zhiguo ^a   Murphy, Susan A ^b  

^a DUKE UNIVERSITY MEDICAL CENTER   (United States)

^b UNIVERSITY OF MICHIGAN   (United States)

Author keywords

Dynamic treatment regime; Sample size calculation; Sequential multiple assignment randomized trial; Weighted Kaplan Meier estimator; Weighted log rank test

Indexed keywords

EID: 80052282229     PISSN: 00063444     EISSN: 14643510     Source Type: Journal    
DOI: 10.1093/biomet/asr019     Document Type: Article

References (28)

1. CHOW S.C., SHAO J. & WANG H. (2005). Sample Size Calculations in Clinical Research. London: Chapman and Hall.
2. ENG, K.H. & KOSOROK, M.R. (2005). A sample size formula for the supremum log-rank statistic. Biometrics 61, 86-91. (Pubitemid 40380967)
3. FENG, W. & WAHED, A.S. (2008). A supremum log-rank test for comparing adaptive treatment strategies and corresponding sample size formula. Biometrika 95, 695-707.
4. FENG, W. & WAHED, S.A. (2009). Sample size for two-stage studies with maintenance therapy. Statist. Med., 28, 2028-41.
5. GUO X. & TSIATIS A.A. (2005). A weighted risk set estimator for survival distributions in two-stage randomization designs with censored survival data. Int. J. Biostatistics 1, 1-15.
6. HOLLAND P.W. (1986). Statistics and causal inference. J. Am. Statist. Assoc. 81, 945-60.
7. LAVORI P.W., & D AWSON R. (2003). Dynamic treatment regimes: practical design considerations. Clin. Trials 1, 9-20.
8. LAVORI P.W., D AWSON R. & ROTH A.J. (2000). Flexible treatment strategies in chronic disease: clinical and research implications. Biol. Psychiat. 48, 605-14.
9. LIEBERMAN J.A., STROUP T. S. , M CEVOY J.P., SWARTZ M.S., ROSENHECK R.A., PERKINS D.O., KEEFE R.S., DAVIS S.M., DAVIS, C.E., LEBOWITZ B.D., SEVERE J., & HSIAO J.K. (2005). Clinical Antipsychotic Trials of Intervention Effectiveness (CATIE) Investigators. Effectiveness of antipsychotic drugs in patients with chronic schizophrenia. New Engl. J. Med. 53, 1209-23.
10. LOKHNYGINA Y. & H ELTERBRAND J.D. (2007). Cox regression methods for two-stage randomization designs. Biometrics 63, 422-8.
11. LUNCEFORD J.K., DAVIDIAN M. & TSIATIS, A.A. (2002). Estimation of survival distributions of treatment strategies in two-stage randomization designs in clinical trials. Biometrics 58, 48-57. (Pubitemid 34194487)
12. MCKAY J.R., LYNCH K.G., SHEPARD D.S., RATICHEK S., MORRISON R., KOPPENHAVER J. & PETTINATI H.M. (2004). The effectiveness of telephone-based continuing care in the clinical management of alcohol and cocaine use disorders: 12-month outcomes. J. Consult. Clin. Psychol. 72, 967-79. (Pubitemid 39658531)
13. MARLOWE D.B., FESTINGER D.S., ARABIA P. L., D UGOSH K.L., BENASUTTI K.M., CROFT J.R. & MCKAY J.R. (2008). Adaptive interventions in drug court: a pilot experiment. Criminal Justice Rev. 33, 343-60.
14. MIYAHARA S. & WAHED A.S. (2010). Weighted Kaplan-Meier estimators for two-stage treatment regimes. Statist. Med. 29, 2581-91.
15. MURPHY S.A. (2005). An experimental design for the development of adaptive treatment strategies. Statist. Med. 24, 1455-81. (Pubitemid 40716347)
16. MURPHY S.A., LYNCH K.G., OSLIN D., MCKAY J.R. & TENHAV E T. (2007). Developing adaptive treatment strategies in substance abuse research. Drug Alcohol Dependence 88, S24-S30. (Pubitemid 46541624)
17. MURPHY S.A., VA N D E R LAAN M.J., ROBINS J.M., CPPRG (2001). Marginal mean models for dynamic regimes. J. Am. Statist. Assoc. 96, 1410-23. (Pubitemid 33695590)
18. OETTING A.I., LEVY J.A., WEISS R.D. & MURPHY S.A. (2007). Statistical methodology for a SMART design in the development of adaptive treatment strategies. In Causality and Psychopathology: Finding the Determinants of Disorders and their Cures, Ed. P.E. Shrout. Arlington, VA: American Psychiatric Publishing, Inc.
19. PETO R. & PETO J. (1972). Asymptotically efficient rank invariant test procedures (with discussion). J. R. Statist. Soc. A 96, 1410-23.
20. ROBINS J.M. (1986). A new approach to causal inference in mortality studies with sustained exposure periods-application to control of the healthy worker survivor effect. Comp. Math. Appl. 14, 1393-512.
21. ROBINS J.M. (1997). Causal Inference From Complex Longitudinal Data. Lecture Notes in Statistics, Berkane M. Ed. Berlin, Heidelberg, New York: Springer, 120, 69-117.
22. ROBINS J.M., ROTNITZKY A. & ZHAO L.P. (1994). Estimation of regression coefficients when some regressors are not always observed. J. Am. Statist. Assoc. 89, 846-66.
23. ROGER, B.N. (1998). An Introduction to Copulas. Berlin, Heidelberg, New York: Springer, 1998.
24. RUSH A.J., FAVA M., WISNIEWSKI S.R., LAVORI P.W., T RIVEDI M.H., SACKEIM H.A., THASE M.E., NIERENBERG A.A., QUITKIN F.M. & KASHNER T. M. (2004) Sequenced treatment alternatives to relieve depression (STAR*D): rationale and design. Contr. Clin. Trials 25, 119-42. (Pubitemid 38230109)
25. SCHOENFELD, D.A. (1981). The asymptotic properties of nonprametric tests for comparing survival distributions. Biometrika 68, 316-9.
26. STONE, R.M., BERG, D.T., GEORGE, S.L., DODGE, R.K., PACIUCCI, P.A., SCHULMAN, P., LEE, E.J., MOORE, J.O., POWELL, B.L. & SCHIFFER, C.A. (1995). Granulocyte-macrophage colony-stimulating factor after initial chemotherapy for elderly patients with primary acute myeloge-nous leukemia. New Engl. J. Med. 332, 1671-7.
27. TUMMARELLO, D., MARI, D., GRAZIANO, F., ISIDORI, P., CETTO, G., PASINI, F., SANTO, A., & CELLERINO, R. (1997). A randomized, controlled phase III study of cyclophosphamide, doxorubicin, and vincristine with Etoposide (CAVE) or Teniposide (CAV-T), followed by recombinant interferon-α maintenance therapy or observation, in small cell lung carcinoma patients with complete responses. Cancer 80, 2222-9. (Pubitemid 28007392)
28. WAHED, A.S. & TSIATIS, A.A. (2006). Semiparametric efficient estimation of survival distribution in two-stage randomization designs in clinical trials with censored data. Biometrika 93, 163-77.