. Author manuscript; available in PMC: 2015 Sep 8.

Published in final edited form as: Stat Med. 2008 Oct 30;27(24):4895–4913. doi: 10.1002/sim.3329

Table 1.

Selection percentage of dose-schedule combination and number of patients treated for the leukemia trial. “Sce” and “Sch” denote the scenario and schedule numbers, respectively. Each pair in parentheses denotes the true probabilities of toxicity and efficacy (multiplied by 100) for the dose-schedule combination with the schedule given in the same row and the dose specified above in the same column. The underlined pairs are target dose-schedule combinations with a toxicity probability ≤ 0.3 and an efficacy probability ≥ 0.3. π₀₁ represents the joint probability of efficacy and no toxicity. Independence between toxicity and efficacy responses is assumed in generating the data. “None” denotes the percentage of the times that no combination is selected. “Tox” denotes the overall percentage of toxicity. “Eff” denotes the overall percentage of efficacy.

Dose
Sce	Sch	1	2	3	4	5	None	Tox	Eff
1	1	(10, 55)	(20, 58)	(30, 60)	(35, 62)	(50, 65)
π₀₁		.50	.46	.42	.40	.33
BIT-2d		41.1 (15.4)	23.7 (11.6)	4.5 (6.7)	.7 (3.8)	0 (1.5)
BIT-1d		42.9 (15.8)	11.9 (7.9)	1.7 (3.7)	.2 (1.8)	0 (.7)
CRM+AR		38.3 (11)	13.9 (8.3)	4.5 (7.2)	1.1 (3.9)	.3 (1.1)
1	2	(15, 58)	(25, 59)	(35, 61)	(45, 64)	(70, 70)
π₀₁		.49	.44	.40	.35	.21
BIT-2d		25.0 (11.9)	4.8 (6.0)	0 (2.3)	0 (.7)	0 (.1)	.2	20	58
BIT-1d		38.4 (18.7)	4.4 (6.7)	.5 (2.8)	0 (1.1)	0 (.3)	0	19	58
CRM+AR		30.7 (12)	8.0 (7.7)	2.7 (5.6)	.5 (2.4)	0 (.4)	0	23	59

2	1	(1, 15)	(1.5, 20)	(2, 25)	(2.5, 20)	(3, 15)
π₀₁		.15	.20	.25	.20	.15
BIT-2d		.4 (3.7)	1.7 (3.9)	3.5 (4.7)	1.9 (3.9)	.4 (3.4)
BIT-1d		.7 (5.4)	1.7 (5.9)	3.1 (7.9)	1.6 (5.9)	.9 (4.7)
CRM+AR		.7 (4.6)	2.5 (4.8)	4.0 (5.3)	1.2 (5.0)	.2 (7.1)
2	2	(9, 25)	(10, 45)	(11, 65)	(12, 85)	(35, 70)
π₀₁		.23	.41	.58	.75	.46
BIT-2d		2.2 (4.0)	11.0 (5.7)	29.1 (9.2)	49.1 (17.7)	.3 (3.7)	.4	9	52
BIT-1d		3.9 (5.4)	20.5 (6.8)	36.8 (8.0)	30.5 (7.0)	.3 (2.3)	0	7	39
CRM+AR		2.5 (6.9)	15.8 (6.8)	29.8 (7.5)	41.0 (7.5)	2.2 (7.5)	.1	9	40

3	1	(10, 15)	(12, 20)	(30, 25)	(35, 30)	(50, 40)
π₀₁		.14	.18	.18	.20	.20
BIT-2d		3.8 (5.6)	5.8 (6.0)	6.9 (5.3)	3 (3.7)	.2 (1.7)
BIT-1d		2.0 (8.6)	4.3 (8.4)	3.3 (6.3)	2.0 (3.4)	0 (1.1)
CRM+AR		2.7 (7.3)	6.4 (7.2)	3.7 (7.3)	2.6 (4.4)	.2 (1.5)
3	2	(12, 20)	(15, 55)	(40, 56)	(50, 58)	(70, 70)
π₀₁		.18	.47	.34	.29	.21
BIT-2d		4.8 (5.5)	68.3 (22.7)	2.9 (6.1)	0 (1.3)	0 (.1)	4.3	21	40
BIT-1d		5.9 (6.6)	75.3 (17.6)	6.4 (4.7)	.3 (1.6)	0 (.4)	.5	20	35
CRM+AR		5.2 (9.0)	69.2 (13)	6.6 (7.4)	1.5 (2.6)	.8 (.4)	1.1	22	35

4	1	(9, 20)	(12, 25)	(15, 35)	(18, 45)	(20, 55)
π₀₁		.18	.22	.30	.37	.44
BIT-2d		2.1 (4.6)	3.9 (4.5)	9.9 (5.9)	15.3 (7.1)	12.5 (7.4)
BIT-1d		1.3 (7.1)	2.9 (6.3)	7.0 (6.4)	6.9 (5.5)	4.7 (3.6)
Ind YLJ		3.7 (5.4)	3.6 (5.1)	5.6 (5.2)	11.4 (6.0)	17.9 (6.6)
CRM+AR		1.7 (6.8)	3.9 (5.7)	7.7 (6.9)	13.0 (6.6)	9.7 (4.9)
4	2	(11, 50)	(30, 55)	(40, 57)	(50, 59)	(60, 61)
π₀₁		.45	.39	.34	.30	.24
BIT-2d		41.3 (15.2)	10.6 (8.6)	.9 (3.6)	.1 (1.0)	0 (.2)	3.4	19	45
BIT-1d		65.1 (19.6)	11.0 (7.5)	1.1 (2.8)	0 (.9)	0 (.2)	0	17	43
CRM+AR		52.2 (13)	9.5 (9.1)	1.7 (5.4)	.4 (1.6)	.1 (.2)	.1	20	44

5	1	(5, 5)	(8, 10)	(10, 40)	(28, 50)	(49, 70)
π₀₁		.05	.09	.36	.36	.36
BIT-2d		1.2 (3.6)	1.7 (3.9)	50.9 (14.5)	35.6 (13.0)	1.4 (7.4)
BIT-1d		.3 (4.1)	2.1 (4.2)	51.4 (10.6)	28.0 (6.9)	2.7 (2.6)
CRM+AR		.1 (4.8)	1.7 (5.1)	55.6 (11)	23.5 (9.2)	3.7 (3.9)
5	2	(8, 3)	(9, 8)	(10, 20)	(49, 70)	(60, 90)
π₀₁		.03	.08	.18	.36	.36
BIT-2d		.8 (3.3)	1.6 (3.5)	4.3 (4.2)	0 (2.4)	0 (3.1)	2.5	20	38
BIT-1d		.2 (5.0)	1.2 (5.8)	11.1 (9.2)	.1 (3.1)	1.5 (5.6)	1.4	18	31
CRM+AR		.2 (5.1)	.4 (4.9)	8.2 (7.7)	3.8 (6.5)	1.5 (1.5)	1.3	20	34

6	1	(1, 20)	(3, 40)	(4, 60)	(5, 70)	(40, 75)
π₀₁		.20	.39	.58	.67	.45
BIT-2d		.7 (3.5)	4.4 (4.7)	29.8 (10.8)	47.3 (14.8)	.4 (5.1)
BIT-1d		.2 (3.6)	8.1 (5.7)	34.0 (8.8)	35.2 (8.7)	.2 (3.2)
CRM+AR		.5 (4.6)	7.5 (5.7)	25.5 (6.8)	38.6 (7.7)	.9 (6.8)
6	2	(5, 22)	(8, 35)	(15, 65)	(20, 70)	(50, 80)
π₀₁		.21	.32	.55	.56	.40
BIT-2d		.6 (3.3)	1.2 (3.8)	11.6 (7.0)	3.4 (4.8)	0 (1.9)	.6	11	59
BIT-1d		.7 (5.4)	3.3 (6.8)	14.8 (9.9)	3.5 (5.1)	0 (2.1)	0	11	54
CRM+AR		.5 (5.3)	3.3 (5.5)	13.5 (7.3)	9.7 (6.9)	0 (3.4)	0	14	55

7	1	(1, 5)	(2, 10)	(3, 15)	(4, 20)	(5, 25)
π₀₁		.05	.10	.15	.19	.24
BIT-2d		0 (3.1)	0 (3.1)	.2 (3.3)	.4 (3.4)	.8 (3.5)
BIT-1d		0 (4.0)	.1 (4.8)	.3 (5.6)	.8 (7.4)	1.2 (7.8)
CRM+AR		0 (3.8)	0 (4.0)	.1 (4.5)	.3 (4.9)	.5 (7.8)
7	2	(1.5, 5)	(2.5, 25)	(3.5, 45)	(4.5, 65)	(5.5, 85)
π₀₁		.05	.24	.43	.62	.80
BIT-2d		0 (3.0)	.6 (3.5)	6.7 (5.3)	29.4 (10.7)	61.4 (20.9)	.5	4	51
BIT-1d		0 (3.1)	1.3 (3.7)	13.8 (5.6)	41.2 (8.8)	41.3 (8.8)	0	4	36
CRM+AR		0 (3.8)	.8 (5.1)	8.9 (6.9)	24.9 (11.0)	64.5 (8.2)	0	4	39

8	1	(8, 15)	(10, 35)	(12, 52)	(45, 55)	(55, 60)
π₀₁		.14	.32	.46	.30	.27
BIT-2d		2 (4.4)	21.4 (9.3)	53.8 (18.5)	1 (6.4)	0 (2.1)
BIT-1d		1.3 (4.9)	22.2 (8.5)	47.2 (10.6)	2.5 (3.5)	.3 (1.2)
CRM+AR		1.3 (6.4)	20.8 (8.2)	52.8 (11)	1.9 (6.2)	.1 (1.7)
8	2	(13, 15)	(20, 35)	(30, 52)	(50, 75)	(65, 78)
π₀₁		.13	.28	.36	.38	.27
BIT-2d		1.6 (3.7)	8.8 (5.5)	7.4 (5.8)	0 (2.0)	0 (.1)	4.0	21	44
BIT-1d		1.4 (6.9)	15.0 (11.9)	9.8 (7.5)	.2 (2.1)	0 (.4)	.1	20	40
CRM+AR		1.0 (7.9)	10.4 (8.2)	10.4 (7.1)	1.0 (3.1)	0 (.5)	.3	22	40

9	1	(8, 20)	(10, 50)	(40, 40)	(45, 40)	(60, 35)
π₀₁		.18	.45	.24	.22	.14
BIT-2d		6 (6.8)	84.7 (33.9)	4.9 (6.8)	.6 (3.0)	0 (.6)
BIT-1d		8.6 (6.2)	78.3 (16.0)	6.8 (4.5)	1.4 (2.0)	0 (.5)
CRM+AR		3.2 (9.5)	88.2 (21.0)	2.3 (9.0)	.9 (3.7)	.3 (.8)
9	2	(50, 40)	(60, 50)	(70, 55)	(80, 70)	(90, 75)
π₀₁		.20	.20	.17	.14	.08
BIT-2d		1.3 (5.7)	.1 (1.9)	0 (.2)	0 (0)	0 (0)	2.4	21	44
BIT-1d		1.3 (13.8)	0 (2.2)	0 (.2)	0 (0)	0 (0)	3.6	29	41
CRM+AR		.6 (14)	.3 (1.1)	.2 (.2)	.7 (0)	.5 (0)	2.8	27	41

10	1	(40, 5)	(50, 15)	(60, 25)	(70, 35)	(80, 45)
π₀₁		.03	.08	.10	.11	.09
BIT-2d		1.4 (7.1)	.6 (4.5)	.2 (1.0)	0 (.1)	0 (.0)
BIT-1d		2.2 (7.2)	5.5 (4.4)	.8 (.8)	0 (.1)	0 (0)
CRM+AR		0 (14)	0 (2.4)	0 (.7)	.4 (.1)	.2 (.1)
10	2	(50, 10)	(60, 25)	(70, 40)	(80, 55)	(90, 70)
π₀₁		.05	.10	.12	.11	.07
BIT-2d		.2 (3.6)	0 (.8)	0 (.0)	0 (0)	0 (0)	97.6	47	11
BIT-1d		.9 (7.9)	.2 (2.7)	0 (.2)	0 (.0)	0 (0)	90.4	48	12
CRM+AR		0 (13)	.1 (1.5)	.3 (.3)	.1 (0)	0 (0)	98.9	49	9

11	1	(2, 5)	(4, 10)	(6, 15)	(20, 30)	(30, 20)
π₀₁		.05	.10	.14	.24	.14
BIT-2d		.2 (3.4)	.6 (3.7)	1.3 (4.0)	6.5 (5.8)	.5 (3.2)
BIT-1d		0 (4.3)	.5 (5.5)	.8 (6.4)	4.9 (9.0)	.6 (4.0)
CRM+AR		.2 (4.3)	.3 (4.5)	.8 (5.0)	3.9 (6.9)	.9(6.9)
11	2	(5, 20)	(7, 40)	(8, 60)	(45, 50)	(55, 40)
π₀₁		.19	.37	.55	.28	.18
BIT-2d		2.2 (4.5)	20.5 (9.7)	66.8 (20.1)	.7 (4.4)	0 (1.0)	.7	13	38
BIT-1d		3.6 (4.9)	27.8 (8.3)	60.4 (12.1)	1.2 (3.4)	0 (1.5)	.2	13	32
CRM+AR		2.2 (6.2)	21.2 (8.2)	69.0 (11)	1.3 (6.5)	.2 (6.5)	0	16	33

12	1	(1, 5)	(2, 25)	(3, 45)	(4, 65)	(5, 85)
π₀₁		.05	.25	.44	.62	.81
BIT-2d		0 (3.0)	.3 (3.4)	5.1 (4.9)	26.5 (10.6)	65.8 (23.8)
BIT-1d		0 (3.1)	1.3 (3.7)	11.2 (5.5)	36.3 (8.4)	42.4 (9.2)
CRM+AR		0 (3.7)	.8 (5.1)	6.1 (6.5)	22.0 (7.7)	66.9 (7.7)
12	2	(5, 5)	(10, 28)	(20, 48)	(30, 65)	(40, 70)
π₀₁		.05	.25	.38	.46	.42
BIT-2d		0 (3.0)	.4 (3.3)	1.4 (3.5)	.4 (3.0)	0 (1.4)	.1	8	60
BIT-1d		0 (3.7)	1.5 (8.1)	5.2 (9.9)	1.9 (5.0)	.2 (1.9)	0	11	49
CRM+AR		0 (4.3)	1.0 (5.6)	2.2 (7.2)	1.0 (5.9)	0 (5.9)	0	10	50