A Bayesian adaptive design for two-stage clinical trials with survival data

Lifetime Data Analysis

Volumn 15, Issue 4, 2009, Pages 468-492

  Bandyopadhyay, Uttam ^a   Biswas, Atanu ^b   Bhattacharya, Rahul ^c  

^a UNIVERSITY OF CALCUTTA   (India)

^b INDIAN STATISTICAL INSTITUTE   (India)

^c WEST BENGAL STATE UNIVERSITY   (India)

Author keywords

Censoring; Clinical trials; Ethical design; Optimal design; Survival data; Two stage designs

Indexed keywords

ALGORITHM; ARTICLE; BAYES THEOREM; CLINICAL TRIAL; ETHICS; HUMAN; METHODOLOGY; STATISTICS; SURVIVAL;

ALGORITHMS; BAYES THEOREM; CLINICAL TRIALS AS TOPIC; HUMANS; RESEARCH DESIGN; SURVIVAL ANALYSIS;

EID: 70450239649     PISSN: 13807870     EISSN: None     Source Type: Journal    
DOI: 10.1007/s10985-009-9134-4     Document Type: Article

References (75)

1. Abdushukorov AA (1984) On some estimates of the distribution function under random censorship. In: Conference of young scientists. Math Inst Acad Sci. Urbek. SSR. Taschkent. VINTI No 8756 (in Russian).
2. Anderson PK, Borgan Ø, Gill RD, Keiding N (1993) Statistical models based on counting processes. Springer, New York.
3. Atkinson AC (1982) Optimal biased coin design for sequential clinical trials with prognostic factors. Biometrika 69: 61-67.
4. Atkinson AC, Biswas A (2005) Bayesian adaptive biased coin design for sequential clinical trials. Biometrics 61: 118-125.
5. Atkinson AC, Donev AN (1996) Optimum experimental designs. Oxford University Press, Oxford.
6. Ball FG, Smith AFM, Verdinelli I (1993) Biased coin designs with a Bayesian bias. J Stat Plan Inference 34: 403-421.
7. Bandyopadhyay U, Biswas A (2001) Adaptive designs for normal responses with prognostic factors. Biometrika 88: 409-419.
8. Bandyopadhyay U, Biswas A, Bhattacharya R (2007) Covariate adjusted two stage allocation design for binary responses in randomized clinical trials. Stat Med 26: 4386-4399.
9. Bartlett RH, Roloff DW, Cornell RG, Andrews AF, Dillon PW, Zwischenberger JB (1985) Extracorporeal circulation in neonatal respiratory failure: a prospective randomized trial. Pediatrics 76: 479-487.
10. Bauer M, Bauer P, Buddle M (1994) A simulation program for adaptive two stage designs. Comput Stat Data Anal 26: 351-371.
11. Bauer P, Brannath W, Posch M (2001) Flexible two-stage designs: an overview. Methods Inf Med 40: 117-121.
12. Biswas A, Dewanji A (2004) A randomized longitudinal play-the-winner design for repeated binary data. Aust N Z J Stat 46: 675-684.
13. Braekers R, Veraverbeke N (2001) The partial Kojiol-Green model with covariates. J Stat Plan Inference 92: 55-71.
14. Chen CH (1984) A correlation goodness-of-fit test for randomly censored data. Biometrika 71: 315-322.
15. Chen YY, Hollander M, Langberg NA (1982) Small-sample results for the Kaplan-Meier estimator. J Am Stat Assoc 77: 141-144.
16. Cheng CH, Lin GD (1987) Maximum likelihood estimation of a survival function under the Koziol-Green proportional hazards model. Stat Probab Lett 5: 75-80.
17. Cheung YK, Thall PF (2002) Monitoring the rates of composite events with censored data in phase I clinical trials. Biometrics 58: 89-97.
18. Coad DS (1992) Some results on estimation for two stage clinical trials. Sequential Anal 11: 299-311.
19. Coad DS (1994) Sequential estimation for two-stage and three-stage clinical trials. J Stat Plan Inference 43: 343-351.
20. Cox DR (1959) The analysis of exponentially distributed life-times with two types of failure. J R Stat Soc B 21: 411-421.
21. Csorgo S (1989) Testing the proportional hazards model of random censorship. In: Mandl P, Huskova M (eds) Proceedings of the fourth prague symposium on asymptotic statistics. Charles University Press, Prague, pp 41-53.
22. Csorgo S, Horvath L (1981) On the Koziol-Green model for random censorship. Biometrika 68: 391-401.
23. Dauxois J-Y, Kirmani SNUA (2003) On testing the proportionality of two cumulative incidence functions in a competing risks setup. Nonparametr Stat 16: 479-491.
24. de Una-Alvarez J, Gonzalez-Manteiga W (1998) Distributional convergence under proportional censorship when covariates are present. Stat Prob Lett 39: 305-315.
25. de Una-Alvarez J, Gonzalez-Manteiga W (1999) Strong consistency under proportional censorship when covariates are present. Stat Prob Lett 42: 283-292.
26. Durham S, Flournoy N, Li W (1998) A sequential design for maximising the probability of a favourable response. Can J Stat 26: 479-495.
27. Ebrahimi N (1985) Nonparametric estimation of survival functions for incomplete observations when the life time distribution is proportionally related to the censoring time distribution. Commun Stat Theory Methods 14: 2887-2898.
28. Ebrahimi N, Habibullah M (1992) Testing to determine the underlying distribution using incomplete observations when the life time distribution is proportionally related to the censoring time distribution. J Stat Comput Simul 40: 109-118.
29. Feigl P, Zelen M (1965) Estimation of exponential survival probabilities with concomitant information. Biometrics 21: 826-838.
30. Flehinger B, Louis T. (1971) A sequential treatment allocation in clinical trials. Biometrika 58: 419-426.
31. Freireich EJ etal (1963) The effect of 6-Mercaptopurine on the duration of steroid induced remission in 'Acute Leukemia'. Blood 21: 699-716.
32. Gather U, Pawlitschko J (1999) Estimating the survival function under a generalized Koziol-Green model with partially informative censoring. Metrika 48: 189-207.
33. Ghorai JK (1991) Estimation of a smooth quantile function under the proportional hazards model. Ann Inst Stat Math 43: 747-760.
34. Gijbels I, Veraverbeke N (1989) Quantile estimation in the proportion hazards model of random censorship. Commun Stat Theory Methods 18: 1645-1663.
35. Gross AJ, Clark VA (1975) Survival distributions: reliability applications in the biomedical sciences. Wiley, New York.
36. Hajek J, Sidak Z (1967) Theory of rank tests. Academic Press, New York.
37. Hallstrom A, Brooks MM, Peckovia M (1996) Logrank, play the winner, power and ethics. Stat Med 15: 2135-2142.
38. Henze N (1993) A quick omnibus test for the proportional hazards model of random censorship. Statistics 24: 253-263.
39. Herbst T (1992) Test of fit with the Koziol-Green model for random censorship. Stat Decis 10: 163-171.
40. Hollander M, Proschan F (1979) Testing to determine the underlying distribution using randomly censored data. Biometrics 35: 393-401.
41. Hsieh FY (1992) Comparing sample size formulas for trials with unbalanced allocation using the logrank test. Stat Med 11: 1091-1098.
42. Iglewicz B (1983) Alternative designs: sequential, multi-stage, decision theory and adaptive designs. In: Buyse ME, Staquet J, Sylvester RJ (eds) Cancer clinical trials: methods and practice. Oxford University Press, Oxford, pp 312-324.
43. Ivanova A (2003) A play-the-winner-type urn design with reduced variability. Metrika 58: 1-13.
44. Koziol JA, Green SB (1976) A Cramer-von Mises statistic for randomly censored data. Biometrika 63: 465-474.
45. Lawless JF (1976) Confidence interval estimation in the inverse power law model. Appl Stat 25: 128-138.
46. Lawrence J (2002) Strategies for changing the test statistic during a clinical trial. J Biopharm Stat 12: 193-205.
47. Liu Q, Chi GY (2001) On sample size and inference for two-stage adaptive designs. Biometrics 57: 172-177.
48. Liu Q, Proschan MA, Pledger GW (2002) A unified theory of two-stage adaptive designs. J Am Stat Assoc 97: 1034-1041.
49. Matthews JNS (2000) An introduction to randomized controlled clinical trials. Edward Arnold, London.
50. Muller H, Schafer H (2001) Adaptive group sequential designs for clinical trials: combining the advantages of adaptive and of classical group sequential approaches. Biometrics 57: 886-891.
51. Pawlitschko J (1999) A comparison of survival function estimators in the Koziol-Green model. Statistics 32: 277-291.
52. Posch M, Bauer P (1999) Adaptive two stage designs and the conditional error function. Biometrical J 41: 689-696.
53. Prentice RL (1973) Exponential survival with censoring and explanatory variables. Biometrika 61: 539-544.
54. Proschan MA, Hunsberger SA (1995) Designed extension of studies based on conditional power. Biometrics 51: 1315-1324.
55. Proschan MA, Liu Q, Hunsberger S (2003) Practical midcourse sample size modification in clinical trials. Control Clin Trials 24: 4-15.
56. Rosenberger WF (2003) Adaptive survival trials. In: Chow S-C (eds) Encyclopedia of biopharmaceutical statistics, 2nd edn. Dekkar, New York, pp 16-17.
57. Rosenberger WF, Lachin JL (2002) Randomization in clinical trials: theory and practice. Wiley, New York.
58. Rosenberger WF, Seshaiyer P (1997) Adaptive survival trials. J Biopharm Stat 7: 617-624.
59. Rout CC, Rocke DA, Levin J, Gouws E, Reddy D (1993) A reevaluation of the role of crystalloid preload in the prevention of hypotension associated with spinal anesthesia for elective cesarean section. Anesthesiology 79: 768-776.
60. Sposto R, Krailo MD (1987) Use of unequal allocation in survival trials. Stat Med 7: 629-637.
61. Stute W (1992) Strong consistency under the Koziol-Green model. Stat Probab Lett 14: 313-320.
62. Subramanian S (2000) Efficient estimation of regression coefficients and baseline hazard under proportionality of conditional hazard. J Stat Plan Infer 84: 81-94.
63. Tamura RN, Faries DE, Andersen JS, Heiligenstein JH (1994) A case study of an adaptive clinical trials in the treatment of out-patients with depressive disorder. J Am Stat Assoc 89: 768-776.
64. The Trials of Hypertension Prevention Collaborative Research Group (1992) The effects of nonpharmacologic interventions on blood pressure of persons with high normal levels. J Am Med Assoc 267: 1213-1220.
65. Veraverbeke N, Cadarso-Suarez C (2000) Estimation of the conditional distribution in a conditional Koziol-Green model. Test 9: 97-122.
66. Ware JH (1989) Investigating therapies of potentially great benefit: ECMO. Stat Sci 4: 298-340.
67. Wei LJ, Durham S (1978) The randomized play-the-winner rule in medical trials. J Am Stat Assoc 73: 838-843.
68. Yao Q, Wei LJ (1996) Play the winner for phase II and III clinical trials. Stat Med 15: 2413-2423.
69. Zeymer U, Suryapranata H, Monassier JP, Opolski G, Davies J, Rasmanis G, Linssen G, Tebbe U, Schroder R, Tiemann R, Machnig T, Neuhaus K-L, forthe ESCAMI Investigators (2001a) The Na+/H+ exchange inhibitor eniporide as an adjunct to early reperfusion therapy for acute myocardial infraction. J Am Coll Cardiol 38: 1644-1651.
70. Zeymer U, Suryapranata H et al (2001b) Evaluation of the safety and cardioprotective effects of eniporide, a specific Sodium/Hydrogen exchange inhibitor, given as adjunctive therapy to reperfusion in patients with acute myocardial infraction. Heart Drug 1: 71-76.
71. Zelen M (1959) Factorial experiments in life testing. Technometrics 1: 269-288.
72. Zelen M (1969) Play-the-winner rule and controlled clinical trial. J Am Stat Assoc 64: 131-146.
73. Zhang L, Rosenberger WF (2007) Response-adaptive randomization for survival trials: the parametric approach. J R Stat Soc Ser C 34: 562-569.
74. Zheng G, Gastwirth JL (2001) On the Fisher information in randomly censored data. Stat Probab Lett 52: 421-426.
75. Zheng G, Gastwirth JL (2003) Fisher information in ordered randomly censored data with applications to characterization problems. Stat Sin 13: 507-517.