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Table 7 Numbers of patients allocated to doses in the six scenarios and three EffTox variants presented in Table 5

From: Implementing the EffTox dose-finding design in the Matchpoint trial

Scenario   Dose 1 Dose 2 Dose 3 Dose 4 Sum
1: Pr(Eff) 0.20 0.30 0.50 0.60  
monotonic, Pr(Tox) 0.03 0.05 0.10 0.30  
dose 4 Utility -0.33 -0.17 0.16 0.22  
optimal ESS=0.5 0.7 0.6 12.1 16.4 29.8
  ESS=1.3 0.2 0.2 9.8 19.6 29.8
  ESS=1.5 0.1 0.1 9.5 20.1 29.8
2: Pr(Eff) 0.40 0.60 0.75 0.79  
monotonic, Pr(Tox) 0.10 0.25 0.55 0.60  
dose 2 Utility -0.01 0.25 0.12 0.08  
optimal ESS=0.5 1.5 11.5 16.0 0.4 29.4
  ESS=1.3 0.8 11.6 16.9 0.6 29.9
  ESS=1.5 0.7 10.3 18.2 0.7 29.9
3: Pr(Eff) 0.25 0.40 0.60 0.60  
eff. plateau, Pr(Tox) 0.10 0.20 0.38 0.42  
dose 3 Utility -0.26 -0.04 0.15 0.11  
optimal ESS=0.5 1.1 2.8 21.8 3.7 29.4
  ESS=1.3 0.5 2.5 22.2 4.4 29.6
  ESS=1.5 0.4 2.0 22.1 5.2 29.7
4: Pr(Eff) 0.50 0.60 0.70 0.80  
tox. plateau, Pr(Tox) 0.20 0.20 0.20 0.20  
dose 4 Utility 0.12 0.28 0.43 0.57  
optimal ESS=0.5 0.5 1.0 19.3 9.2 30.0
  ESS=1.3 0.1 0.7 15.9 13.3 30.0
  ESS=1.5 0.1 0.3 15.9 13.7 30.0
5: Pr(Eff) 0.05 0.08 0.20 0.25  
all doses Pr(Tox) 0.05 0.08 0.12 0.14  
inactive Utility -0.58 -0.54 -0.34 -0.26  
  ESS=0.5 2.4 2.4 4.7 14.1 23.6
  ESS=1.3 1.5 1.9 4.7 15.3 23.4
  ESS=1.5 1.4 1.7 4.5 16.0 23.6
6 Pr(Eff) 0.05 0.08 0.12 0.25  
all doses Pr(Tox) 0.60 0.65 0.70 0.80  
too toxic Utility -0.78 -0.78 -0.76 -0.67  
and inactive ESS=0.5 2.6 3.0 4.0 0.9 10.5
  ESS=1.3 1.1 2.8 5.2 0.8 9.9
  ESS=1.5 1.0 2.8 5.3 0.9 10.0
  1. Dose i is the mean number of patients receiving dose-level i, for i=1,…,4. Row sums are also given in Sum. Sum ≠30 in some instances due to trials stopping early. Patients allocated to the optimal dose are given in bold and underlined for the admissible doses