Achievement of the primary end point
| . | Response at C3D28 . | |
|---|---|---|
| cPR responders, n = 11 . | cPD nonresponders, n = 7 . | |
| Total, no./Total no. (%) | 11/18 (61) | 7/18 (39) |
| Age, median (range), y | 1.2 (0.3-4.7) | 3.0 (1.8-6.9) |
| Genetic subtype, no. (%)* | ||
| PTPN11 | 8 (73) | 5 (71) |
| KRAS | 1 (9) | 0 |
| NRAS | 2 (18) | 1 (14) |
| NF1 | 0 | 1 (14) |
| HbF, median (range)* | 16.5 (0.7-56.8) | 38 (10.0-59.1) |
| Elevated, no. (%) | 8 (73) | 7 (100) |
| Normal for age, no. (%) | 3 (27) | 0 |
| Karyotype, no. (%)* | ||
| Normal | 7 (64) | 5 (71) |
| −7 | 3 (27) | 0 |
| −Y | 0 | 1 (14) |
| +21c | 1 (9) | 0 |
| del(9) | 0 | 1 (14) |
| Methylation, no. (%)* | ||
| Low | 2 (18) | 0 |
| Intermediate | 5 (46) | 0 |
| High | 4 (36) | 7 (100) |
| . | Response at C3D28 . | |
|---|---|---|
| cPR responders, n = 11 . | cPD nonresponders, n = 7 . | |
| Total, no./Total no. (%) | 11/18 (61) | 7/18 (39) |
| Age, median (range), y | 1.2 (0.3-4.7) | 3.0 (1.8-6.9) |
| Genetic subtype, no. (%)* | ||
| PTPN11 | 8 (73) | 5 (71) |
| KRAS | 1 (9) | 0 |
| NRAS | 2 (18) | 1 (14) |
| NF1 | 0 | 1 (14) |
| HbF, median (range)* | 16.5 (0.7-56.8) | 38 (10.0-59.1) |
| Elevated, no. (%) | 8 (73) | 7 (100) |
| Normal for age, no. (%) | 3 (27) | 0 |
| Karyotype, no. (%)* | ||
| Normal | 7 (64) | 5 (71) |
| −7 | 3 (27) | 0 |
| −Y | 0 | 1 (14) |
| +21c | 1 (9) | 0 |
| del(9) | 0 | 1 (14) |
| Methylation, no. (%)* | ||
| Low | 2 (18) | 0 |
| Intermediate | 5 (46) | 0 |
| High | 4 (36) | 7 (100) |
C, cycle; D, day.
Percentage based on 11 responders with cPR and 7 nonresponders with cPD.