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Table 6 Simulation results evaluating Rule 2 when the threshold probability is set at 0.10

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 A B C B C
40 −0.20 0.00 0.0625 0.0585 −0.0123 30.6959 13.8404 29.9078 92.23 % 34.59 %
  −0.15 0.00 0.0601 0.0311 −0.0120 31.2312 18.3044 29.5173 79.84 % 35.56 %
  −0.10 0.00 0.0567 0.0107 −0.0119 32.3427 22.4957 29.6238 63.89 % 35.43 %
  −0.05 0.00 0.0506 −0.0027 −0.0126 33.5772 26.5306 29.7194 48.34 % 35.68 %
  0.00 0.00 0.0480 −0.0122 −0.0123 34.5352 29.3818 29.5852 35.89 % 35.74 %
  0.05 0.00 0.0386 −0.0166 −0.0109 35.7946 32.2020 29.7191 25.14 % 35.03 %
  0.10 0.00 0.0337 −0.0178 −0.0107 36.7805 34.2756 29.8086 17.75 % 34.52 %
  0.15 0.00 0.0300 −0.0176 −0.0122 37.6091 35.9146 29.5542 12.22 % 35.64 %
  0.20 0.00 0.0268 −0.0180 −0.0111 38.1120 36.8978 29.8112 8.92 % 34.91 %
100 −0.20 0.00 0.0704 0.0581 −0.0229 66.7855 15.4632 65.8818 99.60 % 42.83 %
  −0.15 0.00 0.0673 0.0252 −0.0230 68.2876 23.4374 66.0635 96.28 % 42.43 %
  −0.10 0.00 0.0601 0.0007 −0.0229 71.7312 36.8674 66.5112 84.36 % 42.43 %
  −0.05 0.00 0.0571 −0.0160 −0.0231 75.3970 51.5977 65.8379 64.18 % 43.02 %
  0.00 0.00 0.0467 −0.0237 −0.0240 81.2572 66.4872 65.8151 42.25 % 42.95 %
  0.05 0.00 0.0379 −0.0255 −0.0233 86.5612 76.7592 66.9099 27.22 % 41.95 %
  0.10 0.00 0.0338 −0.0241 −0.0242 89.8261 83.4433 64.7309 18.46 % 43.92 %
  0.15 0.00 0.0279 −0.0229 −0.0232 92.3335 88.1690 66.3140 12.66 % 42.48 %
  0.20 0.00 0.0234 −0.0208 −0.0245 94.1323 91.5214 65.5106 8.98 % 43.34 %
  1. pA, pB and pC refer to the posterior means of response probability in arms A, B and C, respectively; Δ=0. 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)