Table 1.
Baseline characteristics of the patients
| Phase 1 | Phase 2 | |
|---|---|---|
| No. | 9 | 30 |
| Median age, y (range) | 59 (35-75) | 62 (37-79) |
| Sex (M/F) | 5/4 | 20/10 |
| PMF/PPV/PET | 7/2/0 | 16/8/6 |
| Median disease duration, mo (range) | 65 (7-124) | 27 (1-234) |
| Lille score, no. (%) | 7 (78) | 21 (70) |
| 0 | ||
| 1 | 2 (22) | 9 (30) |
| 2 | 0 (0) | 0 (0) |
| IWG-MRT, no. (%) | 3 (33.3) | 5 (16.7) |
| Low | ||
| Int-1 | 4 (44.4) | 8 (26.7) |
| Int-2 | 2 (22.2) | 9 (30) |
| High | 0 | 8 (26.6) |
| Median WBC × 109/L (range) | 12.2 (2.7-37.5) | 12.4 (4.1-50.3) |
| Median Hb g/L (range) | 126 (100-147) | 114 (75-173) |
| Median platelets × 109/L (range) | 250 (111-609) | 301 (99-1149) |
| Lactate dehydrogenase (mean ± SD) | 583.4 ± 169.8 | 977.4 ± 585.0 |
| JAK2V617F mutated, no. (%) | 7 (78) | 21 (70) |
| JAK2V617F allele burden (mean ± SD) | 55.4 ± 37.8 | 64.2 ± 20.8 |
| CD34+ cells, % (mean ± SD) | 0.24 ± 0.18 | 1.15 ± 1.73 |
| CD34+ cells × 106/L (mean ± SD) | 27.8 ± 34.4 | 96.8 ± 115.5 |
| Splenomegaly, cm from LCM (mean ± SD) | 14 (4-17) | 14 (2-25) |
| Previous therapy (%) | 8 (89) | 25 (83) |
| Transfusion dependent no. (%) | 2 (22.2) | 7 (23.3) |
| Pruritus, no. (%) | 4 (44.4) | 20 (67) |
| Constitutional symptoms no. (%) | 6 (66.7) | 26 (86.7) |
Int indicates intermediate; IWG-MRT, International Working Group for Myelofibrosis Research and Treatment; PET, previous essential thrombocythemia; PMF, primary myelofibrosis; and PPV, previous polycythemia vera.