Baseline characteristics of patients treated at 12 μg/kg per day (ITT population)
| Characteristic . | Relapsed/refractory (N = 25) . | Treatment-naïve (N = 5) . |
|---|---|---|
| Age, median (range), y | 70.0 (55-83) | 74.0 (67-81) |
| Sex, n (%) | ||
| Female | 10 (40) | 3 (60) |
| Male | 15 (60) | 2 (40) |
| ECOG performance status, n (%) | ||
| 0 | 2 (8) | 1 (20) |
| 1 | 18 (72) | 2 (40) |
| 2 | 5 (20) | 2 (40) |
| Platelet count (×109/L), n (%) | ||
| Median (range) | 75.0 (16-651) | 36.0 (16-330) |
| >100 | 11 (44) | 2 (40) |
| >50-100 | 5 (20) | 0 |
| ≥20-50 | 8 (32) | 2 (40) |
| <20 | 1 (4) | 1 (20) |
| Hemoglobin, median (range), g/L | 85.0 (63-141) | 75.0 (57-113) |
| Dependent on RBC transfusion, n (%) | 2 (8) | 1 (20) |
| Time since diagnosis, median (range), mo | 43.5 (0-162) | 22.6 (10-152) |
| MF subtype, n (%) | ||
| Primary MF | 16 (64) | 4 (80) |
| Postpolycythemia MF | 5 (20) | 1 (20) |
| Postessential thrombocythemia MF | 4 (16) | 0 |
| DIPSS-plus risk score, n (%) | ||
| Intermediate 1 | 4 (16) | 0 |
| Intermediate 2 | 13 (52) | 4 (80) |
| High risk | 8 (32) | 1 (20) |
| Splenomegaly, n (%) | 16 (64) | 2 (40) |
| Spleen size by MRI/CT, cm3 | (n = 23) | (n = 5) |
| Median (range) | 2091.0 (197-5321) | 1616.0 (724-3183) |
| MPN-SAF tumor symptom score | (n = 20) | (n = 5) |
| Median (range) | 71.0 (6-209) | 48.0 (23-87) |
| Mean (SD) | 78.9 (51.39) | 51.0 (22.99) |
| Prior therapies for MPN | ||
| Median (range) | 2.0 (1.0-8.0) | 0 |
| Mean (SD) | 2.4 (1.58) | 0 |
| Karyotype, n (%) | ||
| Normal | 19 (76) | 4 (80) |
| Abnormal | 6 (24) | 1 (20) |
| Very high-risk for GIPSS/MIPSSv2 | 3 (12) | 1 (20) |
| Unfavorable for GIPSS/MIPSSv2 | 2 (8) | 0 |
| 20q-only | 1 (4) | 0 |
| Unfavorable for DIPSS-plus | 4 (16) | 1 (20) |
| Not reported | 1 (4) | 0 |
| Driver mutations, n (%) | (n = 17) | (n = 5) |
| JAK2 V617F | 16 (64) | 3 (60) |
| CALR exon 9 | 0 | 2 (40) |
| MPL exon 10 | 1 (4) | 0 |
| High-risk molecular mutations, n (%) | 10 (40) | 2 (40) |
| ASXL1 | 5 (20) | 2 (40) |
| SRSF2 | 2 (8) | 0 |
| U2AF1 Q157 | 4 (16) | 1 (20) |
| IDH1 | 1 (4) | 0 |
| IDH2 | 1 (4) | 0 |
| Characteristic . | Relapsed/refractory (N = 25) . | Treatment-naïve (N = 5) . |
|---|---|---|
| Age, median (range), y | 70.0 (55-83) | 74.0 (67-81) |
| Sex, n (%) | ||
| Female | 10 (40) | 3 (60) |
| Male | 15 (60) | 2 (40) |
| ECOG performance status, n (%) | ||
| 0 | 2 (8) | 1 (20) |
| 1 | 18 (72) | 2 (40) |
| 2 | 5 (20) | 2 (40) |
| Platelet count (×109/L), n (%) | ||
| Median (range) | 75.0 (16-651) | 36.0 (16-330) |
| >100 | 11 (44) | 2 (40) |
| >50-100 | 5 (20) | 0 |
| ≥20-50 | 8 (32) | 2 (40) |
| <20 | 1 (4) | 1 (20) |
| Hemoglobin, median (range), g/L | 85.0 (63-141) | 75.0 (57-113) |
| Dependent on RBC transfusion, n (%) | 2 (8) | 1 (20) |
| Time since diagnosis, median (range), mo | 43.5 (0-162) | 22.6 (10-152) |
| MF subtype, n (%) | ||
| Primary MF | 16 (64) | 4 (80) |
| Postpolycythemia MF | 5 (20) | 1 (20) |
| Postessential thrombocythemia MF | 4 (16) | 0 |
| DIPSS-plus risk score, n (%) | ||
| Intermediate 1 | 4 (16) | 0 |
| Intermediate 2 | 13 (52) | 4 (80) |
| High risk | 8 (32) | 1 (20) |
| Splenomegaly, n (%) | 16 (64) | 2 (40) |
| Spleen size by MRI/CT, cm3 | (n = 23) | (n = 5) |
| Median (range) | 2091.0 (197-5321) | 1616.0 (724-3183) |
| MPN-SAF tumor symptom score | (n = 20) | (n = 5) |
| Median (range) | 71.0 (6-209) | 48.0 (23-87) |
| Mean (SD) | 78.9 (51.39) | 51.0 (22.99) |
| Prior therapies for MPN | ||
| Median (range) | 2.0 (1.0-8.0) | 0 |
| Mean (SD) | 2.4 (1.58) | 0 |
| Karyotype, n (%) | ||
| Normal | 19 (76) | 4 (80) |
| Abnormal | 6 (24) | 1 (20) |
| Very high-risk for GIPSS/MIPSSv2 | 3 (12) | 1 (20) |
| Unfavorable for GIPSS/MIPSSv2 | 2 (8) | 0 |
| 20q-only | 1 (4) | 0 |
| Unfavorable for DIPSS-plus | 4 (16) | 1 (20) |
| Not reported | 1 (4) | 0 |
| Driver mutations, n (%) | (n = 17) | (n = 5) |
| JAK2 V617F | 16 (64) | 3 (60) |
| CALR exon 9 | 0 | 2 (40) |
| MPL exon 10 | 1 (4) | 0 |
| High-risk molecular mutations, n (%) | 10 (40) | 2 (40) |
| ASXL1 | 5 (20) | 2 (40) |
| SRSF2 | 2 (8) | 0 |
| U2AF1 Q157 | 4 (16) | 1 (20) |
| IDH1 | 1 (4) | 0 |
| IDH2 | 1 (4) | 0 |
CT, computed tomography; ECOG, Eastern Cooperative Oncology Group; GIPSS, Genetically Inspired Prognostic Scoring System; MIPSS, Mutation-Enhanced International Prognostic Scoring System, version 2; MRI, magnetic resonance imaging; SAF, symptom assessment form; SD, standard deviation.