Table 1 A Baseline patient characteristics. B Best response rates to first-line therapy.
|  |  | HMA IL | HMA/Ven 1 L | P-value | HMA/VEN R/R cohort |
|---|---|---|---|---|---|
A | Â | Â | Â | Â | Â |
N | Â | 1127 | 35 | Â | 31 |
Age (years) | mean | 68.4 | 67.8 | 0.76 | 70.9 |
Gender | Male | 66% | 71% | 0.50 | 74% |
Race | White | 90% | 97% | 0.66 | 94% |
t-MDS | Â | 24% | 23% | 0.86 | 26% |
WHO 2016 | MDS-SLD/MLD | 18% | 4% | 0.04 | 19% |
| Â | MDS-RS | 6% | 4% | Â | 3% |
| Â | MDS-EB1 | 33% | 9% | Â | 36% |
| Â | MDS-EB2 | 39% | 78% | Â | 42% |
IPSS-R | Intermediate | 31% | 17% | 0.22 | 45% |
| Â | High | 31% | 37% | Â | 32% |
| Â | Very High | 38% | 46% | Â | 23% |
Myeloblasts (%) | Mean | 8 | 13 | <0.005 | 9% |
Hgb (g/dL) | Mean | 9 | 9 | 1.00 | 9.9 |
WBC (k/uL) | Mean | 4 | 10.6 | <0.005 | 3.1 |
ANC (k/uL) | Mean | 1.8 | 4.1 | <0.005 | 1.5 |
Platelets (k/uL) | mean | 96 | 100 | 0.80 | 120 |
Somatic Mutations (n = 546 sequenced) | SF3B1 | 5% | 0 | 0.30 | 10% |
| Â | TET-2 | 16% | 23% | 0.30 | 23% |
| Â | IDH-1 | 3% | 3% | 0.70 | 6% |
| Â | IDH-2 | 5% | 14% | 0.056 | 0 |
| Â | ASXL-1 | 21% | 46% | 0.002 | 42% |
| Â | TP53 | 27% | 34% | 0.60 | 23% |
| Â | NRAS | 4% | 11% | 0.07 | 3% |
B | Â | Â | Â | Â | Â |
|  | HMA IL | HMA/Ven 1 L | P-value |  |  |
N | 1127 | 35 | Â | Â | Â |
ORR | 40% | 77% | <0.005 | Â | Â |
CR | 13% | 34% | Â | Â | Â |
mCR | 11% | 37% (62%+ HI) | Â | Â | Â |
PR | 1% | 3% | Â | Â | Â |
HI | 15% | 3% | Â | Â | Â |
ASXL1 MT | N = 106 | N = 16 |  |  |  |
ORR | 32% | 87% | <0.005 | Â | Â |
CR | 8% | 44% | Â | Â | Â |
TP53 MT | N = 137 | N = 12 |  |  |  |
ORR | 44% | 75% | 0.038 | Â | Â |
CR | 17% | 25% | 0.47 | Â | Â |
