Table 5 Simulation 1A subgroup discovery using random forest (five subgroups found)
From: Preventing false discovery of heterogeneous treatment effect subgroups in randomized trials
Single agent | Combination | ASD | |
|---|---|---|---|
Group 1: 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, previous treatment hormonal or chemo + hormonal, site bone only or other \( \widehat{\Delta}=0.06\left(-0.40,0.53\right) \) | |||
n = 11 | n = 10 | ||
Age (years) | 63.9 (±4.9) | 63.6 (±3.4) | 0.07 |
Stage 4 | |||
Yes | 11 (100.0%) | 10 (100.0%) | NaN |
Site | |||
Bone only | 2 (18.2%) | 0 (0.0%) | 0.67 |
Other | 9 (81.8%) | 10 (100.0%) | 0.67 |
Previous treatment | |||
Hormonal only | 6 (54.5%) | 9 (90.0%) | 0.86 |
Chemo + hormonal | 5 (45.5%) | 1 (10.0%) | 0.86 |
ECOG score | |||
0 | 4 (36.4%) | 5 (50.0%) | 0.28 |
1 | 7 (63.6%) | 5 (50.0%) | 0.28 |
Disease free >12 months adjuvant to recurrence | |||
No | 11 (100.0%) | 10 (100.0%) | 0 |
Group 3: not disease free >12 months adjuvant to recurrence, previous treatment hormonal or chemo + hormonal, site visceral \( \widehat{\Delta}=-0.57\left(-1.00,-0.08\right) \) | |||
n = 11 | n = 8 | ||
Age (years) | 67.3 (±5.2) | 64.2 (±4.8) | 0.62 |
Stage 4 | |||
No | 2 (18.2%) | 0 (0.0%) | 0.67 |
Yes | 9 (81.8%) | 8 (100.0%) | 0.67 |
Site | |||
Visceral | 11 (100.0%) | 8 (100.0%) | NaN |
Previous treatment | |||
Hormonal only | 7 (63.6%) | 6 (75.0%) | 0.25 |
Chemo + hormonal | 4 (36.4%) | 2 (25.0%) | 0.25 |
ECOG score | |||
0 | 6 (54.5%) | 2 (25.0%) | 0.63 |
1 | 5 (45.5%) | 6 (75.0%) | 0.63 |
Disease free >12 months adjuvant to recurrence | |||
No | 11 (100.0%) | 8 (100.0%) | 0 |
Group 4: not disease free >12 months adjuvant to recurrence, previous treatment none or chemo only, ECOG = 0 \( \widehat{\Delta}=-0.10\left(-0.48,0.28\right) \) | |||
n = 20 | n = 16 | ||
Age (years) | 63.7 (±6.4) | 63.1 (±5.7) | 0.1 |
Stage 4 | |||
No | 1 (5.0%) | 0 (0.0%) | 0.32 |
Yes | 19 (95.0%) | 16 (100.0%) | 0.32 |
Site | |||
Visceral | 11 (55.0%) | 10 (62.5%) | 0.15 |
Bone only | 4 (20.0%) | 2 (12.5%) | 0.2 |
Other | 5 (25.0%) | 4 (25.0%) | 0 |
Previous treatment | |||
None | 15 (75.0%) | 8 (50.0%) | 0.53 |
Chemo only | 5 (25.0%) | 8 (50.0%) | 0.53 |
ECOG score | |||
0 | 20 (100.0%) | 16 (100.0%) | 0 |
Disease free >12 months adjuvant to recurrence | |||
No | 20 (100.0%) | 16 (100.0%) | 0 |
Group 5: not disease free >12 months adjuvant to recurrence, previous treatment none or chemo only, ECOG = 1 \( \widehat{\Delta}=-0.62\left(-0.87,-0.37\right) \) | |||
n = 23 | n = 28 | ||
Age (years) | 66.1 (±6.2) | 66.5 (±4.8) | 0.07 |
Stage 4 | |||
No | 0 (0.0%) | 1 (3.6%) | 0.27 |
Yes | 23 (100.0%) | 27 (96.4%) | 0.27 |
Site | |||
Visceral | 12 (52.2%) | 16 (57.1%) | 0.1 |
Bone only | 2 (8.7%) | 5 (17.9%) | 0.27 |
Other | 9 (39.1%) | 7 (25.0%) | 0.31 |
Previous treatment | |||
None | 17 (73.9%) | 25 (89.3%) | 0.4 |
Chemo only | 6 (26.1%) | 3 (10.7%) | 0.4 |
ECOG score | |||
1 | 23 (100.0%) | 28 (100.0%) | 0 |
Disease free >12 months adjuvant to recurrence | |||
No | 23 (100.0%) | 28 (100.0%) | 0 |