Table 2.
Characteristics of patients by baseline gene expression cluster in combined phase I and phase II cohorts
| Characteristics | Resistant cluster (n = 27) | Sensitive cluster (n = 95) | p value |
|---|---|---|---|
| Median age (range) | 63 (29.1–83.4) | 61 (23.4–86.1) | 0.76 |
| Sex | 0.37 | ||
| Male (%) | 19 (70%) | 56 (59%) | |
| Female (%) | 8 (30%) | 39 (41%) | |
| Cytogenetic risk | 0.5 | ||
| Poor (%) | 14 (52%) | 38 (40%) | |
| Intermediate (%) | 12 (44%) | 47 (49%) | |
| Unevaluable (%) | 1 (4%) | 10 (11%) | |
| PB blasts at screening (%), median (range) | 72 (10–99) | 4 (0–86) | < 0.0001 |
| BM blasts at screening (%), median (range) | 71 (16–95) | 26 (2–90) | < 0.0001 |
| Platelet count (K/μL) at screening, median (range) | 25 (2–342) | 41 (4–289) | 0.7 |
| Hemoglobin (g/dL) at screening, median (range) | 9.3 (6.0–11.7) | 9.2 (6.5–14.4) | 0.33 |
| WBC (K/μL) at screening, median (range) | 9.9 (1–34) | 1.8 (0–76) | 0.0005 |
| LINE-1 maximum demethylation %, mean ± SE | 15.4 ± 2.3 | 26.3 ± 1.4 | 0.0002 |
| RAS mutation | 12/27 (44%) | 9/89 (10%) | 0.0002 |
| IDH mutation | 6/27 (22%) | 17/89 (19%) | 0.78 |
| z4-score mean ± SD | − 2.4 ± 1.26 | 1.90 ± 1.85 | < 0.0001 |
| Methylation cluster | < 0.0001 | ||
| CIMP-like | 18 (67%) | 23 (24%) | |
| Intermediate | 9 (33%) | 23 (24%) | |
| Normal-like | 0 | 38 (40%) | |
| Unknown | 0 | 11 (12%) | |
| Response | |||
| CR rate (%) | 0 | 16/95 (17%) | 0.02 |
| CRc rate (%) | 0 | 28/95 (29%) | 0.0005 |
| Median survival days (range) | 154 (12–506) | 237 (8–1088+) | 0.003 |
| 2-year survival rate | 0 | 20/95 (21%) | 0.0065 |