Skip to main content

Table 6 Simulation 1A subgroup discovery using gradient random forest (five subgroups found)

From: Preventing false discovery of heterogeneous treatment effect subgroups in randomized trials

 

Single agent

Combination

ASD

Group 1: not disease free >12 months adjuvant to recurrence

\( \widehat{\Delta}=-0.05\left(-0.30,0.20\right) \)

 

n = 35

n = 38

 

 Age (years)

64.6 (±5.6)

65.0 (±5.1)

0.08

 Stage 4

  No

1 (2.9%)

0 (0.0%)

0.24

  Yes

34 (97.1%)

38 (100.0%)

0.24

 Site

  Visceral

19 (54.3%)

15 (39.5%)

0.3

  Bone only

5 (14.3%)

6 (15.8%)

0.04

  Other

11 (31.4%)

17 (44.7%)

0.28

 Previous treatment

  None

24 (68.6%)

19 (50.0%)

0.38

  Chemo only

6 (17.1%)

8 (21.1%)

0.1

  Hormonal only

3 (8.6%)

4 (10.5%)

0.07

  Chemo + hormonal

2 (5.7%)

7 (18.4%)

0.4

 ECOG score

  0

15 (42.9%)

14 (36.8%)

0.12

  1

20 (57.1%)

24 (63.2%)

0.12

 Disease free >12 months adjuvant to recurrence

  Yes

35 (100.0%)

38 (100.0%)

0

Group 2: not disease free >12 months adjuvant to recurrence

\( \widehat{\Delta}=-0.01\left(-0.42,0.39\right) \)

 

n = 11

n = 15

 

 Age (years)

63.9 (±4.9)

64.2 (±4.1)

0.06

 Stage 4

  Yes

11 (100.0%)

15 (100.0%)

0

 Site

  Other

11 (100.0%)

15 (100.0%)

0

 Previous treatment

  Chemo only

2 (18.2%)

5 (33.3%)

0.35

  Hormonal only

5 (45.5%)

9 (60.0%)

0.29

  Chemo + hormonal

4 (36.4%)

1 (6.7%)

0.78

 ECOG score

  0

3 (27.3%)

8 (53.3%)

0.55

  1

8 (72.7%)

7 (46.7%)

0.55

 Disease free >12 months adjuvant to recurrence

  No

11 (100.0%)

15 (100.0%)

0

Group 3: not disease free >12 months adjuvant to recurrence

\( \widehat{\Delta}=-0.33\left(-0.92,0.25\right) \)

 

n = 12

n = 6

 

 Age (years)

65.7 (±7.6)

65.0 (±4.4)

0.12

 Stage 4

  Yes

12 (100.0%)

6 (100.0%)

0

 Site

  Other

12 (100.0%)

6 (100.0%)

0

 Previous treatment

  None

12 (100.0%)

6 (100.0%)

0

 ECOG score

  0

5 (41.7%)

1 (16.7%)

0.57

  1

7 (58.3%)

5 (83.3%)

0.57

 Disease free >12 months adjuvant to recurrence

  No

12 (100.0%)

6 (100.0%)

0

Group 4: not disease free >12 months adjuvant to recurrence

\( \widehat{\Delta}=-0.23\left(-0.61,0.15\right) \)

 

n = 22

n = 14

 

 Age (years)

63.7 (±6.2)

62.3 (±5.1)

0.24

 Stage 4

  No

1 (4.5%)

0 (0.0%)

0.31

  Yes

21 (95.5%)

14 (100.0%)

0.31

 Site

  Visceral

17 (77.3%)

12 (85.7%)

0.22

  Bone only

5 (22.7%)

2 (14.3%)

0.22

 Previous treatment

  None

10 (45.5%)

7 (50.0%)

0.09

  Chemo only

5 (22.7%)

5 (35.7%)

0.29

  Hormonal only

4 (18.2%)

1 (7.1%)

0.34

  Chemo + hormonal

3 (13.6%)

1 (7.1%)

0.21

 ECOG score

  0

22 (100.0%)

14 (100.0%)

0

 Disease free >12 months adjuvant to recurrence

  No

22 (100.0%)

14 (100.0%)

0

Group 5: not disease free >12 months adjuvant to recurrence

\( \widehat{\Delta}=-0.64\left(-0.90,-0.39\right) \)

 

n = 20

n = 27

 

 Age (years)

67.3 (±4.5)

66.5 (±5.1)

0.15

 Stage 4

  No

2 (10.0%)

1 (3.7%)

0.25

  Yes

18 (90.0%)

26 (96.3%)

0.25

 Site

  Visceral

17 (85.0%)

22 (81.5%)

0.09

  Bone only

3 (15.0%)

5 (18.5%)

0.09

 Previous treatment

  None

10 (50.0%)

20 (74.1%)

0.51

  Chemo only

4 (20.0%)

1 (3.7%)

0.52

  Hormonal only

4 (20.0%)

5 (18.5%)

0.04

  Chemo + hormonal

2 (10.0%)

1 (3.7%)

0.25

 ECOG score

  1

20 (100.0%)

27 (100.0%)

0

 Disease free >12 months adjuvant to recurrence

  No

20 (100.0%)

27 (100.0%)

0

  1. ASD absolute standardized difference, ECOG Eastern Cooperative Oncology Group