Table 1.
Patient demographics and clinical characteristics by study phase.
| Dose escalation (n = 9) | Dose expansion (n = 12) | Total (n = 21) | |
|---|---|---|---|
| Age (y) | 69 (51–81) | 74 (51–85) | 73 (51–85) |
| Sex, F | 2 (22.2%) | 3 (25.0%) | 5 (23.8%) |
| Prior ruxolitinib | 8 (88.9%) | 11 (91.7%) | 19 (90.5%) |
| DIPSS | |||
| Int-2 | 6 (66.7%) | 7 (58.3%) | 13 (61.9%) |
| High | 3 (33.3%) | 5 (41.7%) | 8 (38.1%) |
| JAK2V617 | 6 (66.7%) | 9 (75.0%) | 15 (71.4%) |
| VAF% | 55 (5–84) | 47 (21–96) | 47 (5–96) |
| CALR | 3 (33.3%) | 1 (8.3%) | 4 (19.0%) |
| Spleen length cm | 10 (0–19) | 11 (0–24) | 10 (0–24) |
| Platelet count × 109/L | 114 (42–290) | 107 (27–695) | 114 (27–695) |
| Platelet count <100 × 109/L | 4 (44.4%) | 6 (50.0%) | 10 (47.6%) |
| Platelet count <50 × 109/L | 1 (11.1%) | 3 (25.0%) | 4 (19.0%) |
| RBC transfusion dependent | 5 (55.6%) | 9 (75.0%) | 14 (66.7%) |
| PB blasts % | 1 (0–21) | 3 (0–8) | 2 (0–21) |
| BMF grade | |||
| Mild | — | 1 (8.3%) | 1 (4.8%) |
| Moderate | — | 3 (25.0%) | 3 (14.3%) |
| Severe | 9 (100%) | 8 (66.7%) | 17 (81.0%) |
| TSS | 10.5 (3–26) | 16.5 (3–39) | 14.0 (3–39) |
| Normal karyotype | 4 (44.4%) | 6 (54.5%) | 10 (50.0%)a |
| Abnormal karyotype | 5 (55.6%) | 5 (45.5%) | 10 (50.0%)a |
| Adverse Karyotype | 0 | 3 (27.3%) | 3 (15%)a |
Note: Median (range) or n (%) values are presented.
Abbreviations: DIPSS, dynamic international prognostic score; PB, peripheral blood; RBC, red blood cell; VAF, variant allele frequency.
aBaseline karyotype available in 20/21 patients (escalation cohort: 9, expansion cohort: 11).