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. Author manuscript; available in PMC: 2019 Mar 30.
Published in final edited form as: Stat Med. 2018 Oct 25;38(7):1170–1189. doi: 10.1002/sim.8021

TABLE 1.

Simulation results using the nonparametric model for scenarios 1 to 6 from Thall and Cook.31(p, q): DLT risk and immune response probability. Dose levels that are safe (ppT) and have the highest immune response probability are in bold. %: rate of reporting a dose level. N: average number of patients treated. (D, R): average number of DLTs and immune responses. Note that the total rate of reporting any dose level is less than 100% when the chance that dose level 1 is considered too toxic is larger than 0 (eg, in scenarios 5 and 6)

Scenario Dose Level
1 2 3 4 5 Total
1 (p, q) (0.01,0.05) (0.02,0.2) (0.03,0.35) (0.04,0.6) (0.05,0.8)
% 2.1 6.2 3.7 4.3 83.7 100
N 7.47 8.48 8.16 8.05 8.01 40.17
(D,R) (0.08,0.38) (0.14,1.65) (0.24,2.89) (0.34,4.81) (0.43,6.39) (1.23,16.12)
2 (p, q) (0.01,0.57) (0.03,0.58) (0.06,0.6) (0.2,0.62) (0.32,0.64)
% 21.7 20.3 27.7 20 10.3 100
N 7.5 8.3 7.16 5.98 2.93 31.88
(D,R) (0.08,4.3) (0.28,4.84) (0.46,4.34) (1.23,3.66) (0.99,1.92) (3.04,19.06)
3 (p, q) (0.02,0.2) (0.03,0.4) (0.04,0.6) (0.06,0.68) (0.2,0.74)
% 4.1 5 12.9 24.9 53.1 100
N 7.86 8.34 8.3 8.36 8.46 41.32
(D,R) (0.14,1.55) (0.24,3.29) (0.32,5.03) (0.49,5.67) (1.71,6.27) (2.91,21.81)
4 (p, q) (0.01,0.52) (0.01,0.62) (0.02,0.71) (0.03,0.79) (0.03,0.86)
% 12.4 10.9 7.9 6.4 62.4 100
N 7.46 7.58 6.96 6.1 5.59 33.68
(D,R) (0.07,3.85) (0.11,4.68) (0.16,4.9) (0.14,4.87) (0.16,4.8) (0.63,23.09)
5 (p, q) (0.18,0.05) (0.22,0.2) (0.26,0.35) (0.3,0.47) (0.33,0.58)
% 15.6 24.1 24 15.6 9.9 89.2
N 11.65 10.57 8.35 5.26 2.58 38.4
(D,R) (2.09,0.6) (2.22,2.03) (2.2,3.04) (1.6,2.5) (0.89,1.53) (9,9.7)
6 (p, q) (0.08,0.15) (0.18,0.38) (0.25,0.52) (0.3,0.59) (0.35,0.62)
% 14.9 26.7 29.1 19.7 8.2 98.6
N 10 11.32 9.5 6 2.62 39.44
(D,R) (0.8,1.49) (2.03,4.25) (2.39,4.91) (1.82,3.5) (0.96,1.71) (7.99,15.85)