Baseline characteristics of patients treated with tagraxofusp at 12 μg/kg per day
| Characteristic . | Treatment naïve (n = 15) . | Relapsed/refractory (n = 22) . |
|---|---|---|
| Age, median (range), y | 64.0 (48-82) | 70.5 (60-87) |
| Female sex, n (%) | 3 (20) | 7 (32) |
| ECOG PS, n (%) | ||
| 0 | 2 (13) | 2 (9) |
| 1 | 11 (73) | 17 (77) |
| 2 | 2 (13) | 3 (14) |
| Platelets, median (range), ×109/L | 76.0 (10-339) | 109.5 (7-788) |
| Hemoglobin, median (range), g/L | 86.0 (72-134) | 94.5 (67-123) |
| WBC count, median (range), ×109/L | 16.3 (2-34) | 10.3 (3-99) |
| Monocyte count, median (range), ×109/L | 3.0 (0-10) | 3.9 (0-44) |
| Splenomegaly,∗ n (%) | 5 (33) | 8 (36) |
| Hepatomegaly, n (%) | 3 (20) | 4 (18) |
| Baseline RBC-transfusion dependence | 0 | 0 |
| CMML type, n (%)† | ||
| CMML-1 | 8 (53) | 14 (64) |
| CMML-2 | 7 (47) | 8 (36) |
| MD-CMML, WBCs of <13 × 109/L, n (%) | 7 (47) | 12 (55) |
| MP-CMML, WBCs of ≥13 × 109/L, n (%) | 8 (53) | 10 (46) |
| No. of previous therapies for MPN, median (range) | 0 (0-0) | 1 (1-7) |
| Cytogenetic risk category‡ as reported by sites n (%) | ||
| High | 7 (47) | 4 (18) |
| Intermediate | 4 (27) | 3 (14) |
| Low | 3 (20) | 13 (59) |
| Other | 1 (7) | 1 (5) |
| Cytogenetic risk category (CPSS-Mol), n (%) | ||
| High | 5 (33) | 4 (18) |
| Intermediate 2 | 7 (47) | 9 (41) |
| Intermediate 1 | 2 (13) | 6 (27) |
| Low | 1 (7) | 3 (14) |
| Patients with no mutations | 11 (73) | 14 (64) |
| Patients with mutations | 4 (26) | 8 (36) |
| Median number of molecular mutations per patient (range) | 3 (1-5) | 2 (1-4) |
| Types of mutation, n (%) | ||
| TET2 | 1 (7) | 4 (18) |
| TET2mut/ASXL1 WT | 0 | 2 (9) |
| ASXL1 | 4 (27) | 5 (23) |
| IDH2 | 0 | 1 (5) |
| SRSF2 | 2 (13) | 4 (18) |
| CBL | 1 (7) | 0 |
| NRAS | 2 (13) | 2 (9) |
| NF1 | 1 (7) | 0 |
| RUNX | 0 | 1 (5) |
| SETBP1 | 1 (7) | 1 (5) |
| Characteristic . | Treatment naïve (n = 15) . | Relapsed/refractory (n = 22) . |
|---|---|---|
| Age, median (range), y | 64.0 (48-82) | 70.5 (60-87) |
| Female sex, n (%) | 3 (20) | 7 (32) |
| ECOG PS, n (%) | ||
| 0 | 2 (13) | 2 (9) |
| 1 | 11 (73) | 17 (77) |
| 2 | 2 (13) | 3 (14) |
| Platelets, median (range), ×109/L | 76.0 (10-339) | 109.5 (7-788) |
| Hemoglobin, median (range), g/L | 86.0 (72-134) | 94.5 (67-123) |
| WBC count, median (range), ×109/L | 16.3 (2-34) | 10.3 (3-99) |
| Monocyte count, median (range), ×109/L | 3.0 (0-10) | 3.9 (0-44) |
| Splenomegaly,∗ n (%) | 5 (33) | 8 (36) |
| Hepatomegaly, n (%) | 3 (20) | 4 (18) |
| Baseline RBC-transfusion dependence | 0 | 0 |
| CMML type, n (%)† | ||
| CMML-1 | 8 (53) | 14 (64) |
| CMML-2 | 7 (47) | 8 (36) |
| MD-CMML, WBCs of <13 × 109/L, n (%) | 7 (47) | 12 (55) |
| MP-CMML, WBCs of ≥13 × 109/L, n (%) | 8 (53) | 10 (46) |
| No. of previous therapies for MPN, median (range) | 0 (0-0) | 1 (1-7) |
| Cytogenetic risk category‡ as reported by sites n (%) | ||
| High | 7 (47) | 4 (18) |
| Intermediate | 4 (27) | 3 (14) |
| Low | 3 (20) | 13 (59) |
| Other | 1 (7) | 1 (5) |
| Cytogenetic risk category (CPSS-Mol), n (%) | ||
| High | 5 (33) | 4 (18) |
| Intermediate 2 | 7 (47) | 9 (41) |
| Intermediate 1 | 2 (13) | 6 (27) |
| Low | 1 (7) | 3 (14) |
| Patients with no mutations | 11 (73) | 14 (64) |
| Patients with mutations | 4 (26) | 8 (36) |
| Median number of molecular mutations per patient (range) | 3 (1-5) | 2 (1-4) |
| Types of mutation, n (%) | ||
| TET2 | 1 (7) | 4 (18) |
| TET2mut/ASXL1 WT | 0 | 2 (9) |
| ASXL1 | 4 (27) | 5 (23) |
| IDH2 | 0 | 1 (5) |
| SRSF2 | 2 (13) | 4 (18) |
| CBL | 1 (7) | 0 |
| NRAS | 2 (13) | 2 (9) |
| NF1 | 1 (7) | 0 |
| RUNX | 0 | 1 (5) |
| SETBP1 | 1 (7) | 1 (5) |
Data presented are for the intent-to-treat population.
ECOG PS, Eastern Cooperative Oncology Group performance status; WT, wild type.
Splenomegaly as assessed by physical examination.
Patient had a 2016 WHO–defined diagnosis of CMML. Patients with CMML-0 were not eligible.
Based on CPSS, Groupe Français des Myélodysplasies, Mayo Molecuular Model, or European LeukemiaNet risk stratification systems.