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Table 3 Simulation results for dropping treatment arms based on the first rule (R1) and the absolute bias for such arms in the estimated treatment effect at the time of dropping decision– all priors on pk (k=A,B,C) are non-informative beta(1, 1) priors, when decision threshold is set at 0.95

From: Evaluation of a multi-arm multi-stage Bayesian design for phase II drug selection trials – an example in hemato-oncology

Sample size

True benefit

Posterior mean estimate biases

Enrolled sample sizes

% Early stopping

 

dB

dC

pA

pB

pC

nA

nB

nC

A

% early B

% early C

40

−0.20

0.00

−0.0051

0.0305

−0.0063

36.3504

15.0121

36.3180

15.32 %

96.09 %

15.15 %

 

−0.15

0.00

−0.0058

0.0061

−0.0057

36.2548

21.3829

36.3305

15.43 %

79.69 %

15.19 %

 

−0.10

0.00

−0.0063

−0.0066

−0.0062

36.3540

27.9657

36.3594

15.20 %

53.98 %

15.36 %

 

−0.05

0.00

−0.0056

−0.0076

−0.0072

36.2719

33.0725

36.2428

15.44 %

30.66 %

15.86 %

 

0.00

0.00

−0.0050

−0.0057

−0.0063

36.2726

36.4040

36.4653

15.77 %

15.11 %

14.85 %

 

0.05

0.00

−0.0073

−0.0034

−0.0049

36.2420

38.1107

36.3738

15.75 %

7.33 %

15.23 %

 

0.10

0.00

−0.0077

−0.0011

−0.0049

36.2214

39.1845

36.3761

15.91 %

2.92 %

14.88 %

 

0.15

0.00

−0.0044

−0.0008

−0.0070

36.4279

39.6107

36.2580

14.86 %

1.35 %

15.47 %

 

0.20

0.00

−0.0044

−0.0016

−0.0060

36.3945

39.8325

36.3640

15.06 %

0.55 %

15.29 %

100

−0.20

0.00

−0.0206

0.0305

−0.0205

84.2873

15.4339

84.3159

22.80 %

99.99 %

22.82 %

 

−0.15

0.00

−0.0188

−0.0007

−0.0203

84.7484

24.5031

84.4050

21.95 %

99.27 %

22.22 %

 

−0.10

0.00

−0.0202

−0.0212

−0.0197

84.4487

43.6230

84.5338

22.31 %

85.84 %

22.44 %

 

−0.05

0.00

−0.0191

−0.0276

−0.0199

84.8712

66.7741

84.3454

21.99 %

52.46 %

22.57 %

 

0.00

0.00

−0.0216

−0.0207

−0.0216

84.0536

84.2135

83.8033

22.86 %

22.67 %

23.36 %

 

0.05

0.00

−0.0203

−0.0115

−0.0196

84.6003

93.1786

84.7271

22.28 %

8.66 %

22.24 %

 

0.10

0.00

−0.0194

−0.0050

−0.0202

84.6521

97.3946

84.1845

21.94 %

3.08 %

22.76 %

 

0.15

0.00

−0.0194

−0.0023

−0.0202

84.8085

98.9283

84.6060

22.00 %

1.20 %

22.06 %

 

0.20

0.00

−0.0199

−0.0024

−0.0215

84.5757

99.4983

84.1044

22.02 %

0.56 %

22.94 %

  1. pA, pB and pC refer to the posterior means of response probability in arms A, B and C, respectively; p0=0.3 (minimum required treatment response rate). Bold data refer to the null hypothesis of absence of any treatment difference (dB=dC=0), or to the alternative hypothesis of an expected true 0.15 benefit of treatment B (dB=0.15)