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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