Table 3 Operating Characteristics of the two-stage PO-TITE-CRM (with true DLT rates that imply 2a is more toxic than 2b) based on 10000 simulated trials. Definitions: DLT: Dose-limiting toxicity. P(select): Probability of selecting a dose as the TD25. Bold values indicate the correct decision

Scenario

Prior DLT

0.01

0.04

0.08

0.16

0.25

0.35

9: TD25 @-1

True DLT rate

0.25

0.4

0.45

0.55

0.5

0.6

P(select)

0.67

0.19

0.05

0

0.01

0

0.08

% of patients

39

32

20

6

3

0

Mean number of patients

10.19

8.43

5.27

1.6

0.68

0.07

10: TD25 @0

True DLT rate

0.12

0.25

0.4

0.5

0.45

0.55

P(select)

0.23

0.52

0.2

0.02

0.02

0

0.01

% of patients

18

36

29

11

6

1

Mean number of patients

5.24

10.64

8.82

3.16

1.85

0.24

11: TD25 @1

True DLT rate

0.09

0.12

0.25

0.45

0.4

0.5

P(select)

0.02

0.2

0.55

0.09

0.14

0.01

<0.01

% of patients

4

20

34

21

17

3

Mean number of patients

1.16

6.43

11.07

6.83

5.6

1.07

12: TD25 @2a

True DLT rate

0.06

0.09

0.12

0.25

0.15

0.45

P(select)

0

0.01

0.08

0.44

0.33

0.14

<0.01

% of patients

1

11

16

30

24

18

Mean number of patients

0.48

3.78

5.24

10.1

7.9

6.07

13: TD25 @2b

True DLT rate

0.03

0.06

0.09

0.35

0.25

0.4

P(select)

0

0

0.15

0.31

0.43

0.11

0

% of patients

1

11

18

30

28

14

Mean number of patients

0.25

3.5

5.9

9.82

9.14

4.54

14: TD25 @3

True DLT rate

0.01

0.03

0.06

0.12

0.09

0.25

P(select)

0

0

0

0.13

0.09

0.78

0

% of patients

0

10

11

19

16

43

Mean number of patients

0.1

3.13

3.51

5.88

5.06

13.13

15: Equal steps in DLT rate

True DLT rate

0.05

0.1

0.15

0.25

0.2

0.3

P(select)

0

0.02

0.12

0.32

0.27

0.26

<0.01

% of patients

2

13

19

27

22

18

Mean number of patients

0.54

4.02

5.93

8.56

6.89

5.75

16: All toxic

True DLT rate

0.5

0.6

0.65

0.75

0.7

0.8

P(select)

0.27

0

0

0

0

0

0.73

% of patients

56

27

15

2

0

0

Mean number of patients

9.01

4.28

2.39

0.38

0.05

0