Baseline patient and disease characteristics
| Characteristic . | N = 18 . |
|---|---|
| Age, median (range), y | 2.1 (0.3-6.9) |
| Time from initial diagnosis to C1D1, median (range), d | 25 (6-80) |
| Sex, no. (%) | |
| Male | 11 (61) |
| Female | 7 (39) |
| Somatic mutation/genetic subtype, no. (%) | |
| PTPN11 | 13 (72) |
| NRAS | 3 (17) |
| KRAS | 1 (6) |
| NF1 | 1 (6) |
| Methylation class, no. (%) | |
| High | 11 (61) |
| Intermediate | 5 (28) |
| Low | 2 (11) |
| Karyotype, no. (%) | |
| Normal | 12 (67) |
| −7 | 3 (17) |
| −Y | 1 (6) |
| del(9) | 1 (6) |
| +21c | 1 (6) |
| WBC, median (range), ×109/L | 19.7 (4.3-59.0) |
| HbF, median (range) | 18.4 (0.7-59.1) |
| Elevated, no. (%) | 15 (83) |
| Normal for age, no. (%) | 3 (17) |
| Platelet count, median (range), ×109/L | 28 (7-85) |
| Patients receiving platelet transfusion, no. (%) | 12* (67) |
| Blasts in BM, median (range), % | 6 (0-19) |
| Spleen tip palpable below costal margin, median (range), cm | 4 (2-14) |
| Characteristic . | N = 18 . |
|---|---|
| Age, median (range), y | 2.1 (0.3-6.9) |
| Time from initial diagnosis to C1D1, median (range), d | 25 (6-80) |
| Sex, no. (%) | |
| Male | 11 (61) |
| Female | 7 (39) |
| Somatic mutation/genetic subtype, no. (%) | |
| PTPN11 | 13 (72) |
| NRAS | 3 (17) |
| KRAS | 1 (6) |
| NF1 | 1 (6) |
| Methylation class, no. (%) | |
| High | 11 (61) |
| Intermediate | 5 (28) |
| Low | 2 (11) |
| Karyotype, no. (%) | |
| Normal | 12 (67) |
| −7 | 3 (17) |
| −Y | 1 (6) |
| del(9) | 1 (6) |
| +21c | 1 (6) |
| WBC, median (range), ×109/L | 19.7 (4.3-59.0) |
| HbF, median (range) | 18.4 (0.7-59.1) |
| Elevated, no. (%) | 15 (83) |
| Normal for age, no. (%) | 3 (17) |
| Platelet count, median (range), ×109/L | 28 (7-85) |
| Patients receiving platelet transfusion, no. (%) | 12* (67) |
| Blasts in BM, median (range), % | 6 (0-19) |
| Spleen tip palpable below costal margin, median (range), cm | 4 (2-14) |
C, cycle; D, day; WBC, white blood cell.
At C2D1, 16 patients (90%) were platelet transfusion dependent.