Table 3.
G-CSF administration and follow-up of patients with CN
| No. | Mutated gene | Age at first visit (y) | Follow-up period (mo) | Body weight (kg) | G-CSF |
HSCT∗ | |||
|---|---|---|---|---|---|---|---|---|---|
| Injection period (d) | Dosage (μg/kg) | bANC (×109 cells per L)† | pANC (×109 cells per L)‡ | ||||||
| 1 | ELANE | 13.5 | 1.6 | 33.5 | 9 | 7 | 0.53 | 2.37 | No |
| 2 | ELANE | 1.1 | 2.0 | 10.0 | 59 | 10-20 | 0.03 | 0.45 | No |
| 3 | ELANE | 1.8 | 20.8 | 12.5 | 14 | 10 | 0.65 | 2.01 | Yes |
| 4 | ELANE | 1.1 | 3.0 | 10 | — | — | — | — | No |
| 5 | ELANE(CyN) | 4.8 | 1.7 | 15 | — | — | — | — | No |
| 6 | ELANE | 1.4 | 2.3 | 11.5 | — | — | — | — | No |
| 7 | ELANE | 2.7 | 6.5 | 12 | 11 | 10 | 0.32 | 1.45 | Yes |
| 8 | ELANE | 1.4 | 2.4 | 9.5 | 9 | 5-10 | 0.34 | 2.41 | No |
| 9 | ELANE | 1.0 | 80.3 | 11.5 | 5 | 5-7.5 | 0.08 | 0.30 | No |
| 10 | ELANE | 0.2 | 8.1 | 6.6 | 15 | 7.5 | 0.47 | 5.01 | Yes |
| 11 | ELANE(CyN) | 6.7 | 0.5 | 23 | — | — | — | — | No |
| 12 | ELANE | 0.2 | 113.4 | 10 | 7 | 5-20 | 0.24 | 1.37 | No |
| 13 | ELANE | 1.5 | 7.8 | NA | — | — | — | — | No |
| 14 | ELANE | 13.4 | 12.0 | NA | — | — | — | — | Yes |
| 15 | ELANE | 1.5 | 29.9 | 12.5 | 53 | 7.5-15 | 0.91 | 2.11 | Yes |
| 16 | ELANE(CyN) | 10.1 | 2.1 | 26 | 10 | 5 | 0.45 | 10.33 | No |
| 17 | ELANE | 3.1 | 33.1 | 18 | 3 | 5 | 0.40 | 2.57 | Yes |
| 18 | ELANE | 0.5 | 0.5 | 6 | 9 | 10-15 | 0.04 | 0.90 | No |
| 19 | ELANE | 9.1 | 0.6 | 32 | 3 | 5 | 0.68 | 4.17 | No |
| 20 | ELANE | 5.7 | 0.6 | 20 | 11 | 5-10 | 0.26 | 1.32 | No |
| 21 | ELANE(CyN) | 0.4 | 33.5 | 15 | 1 | 5 | 0.46 | 8.38 | No |
| 22 | ELANE | 0.4 | 11.4 | 8 | 9 | 5 | 0.33 | 1.94 | No |
| 23 | ELANE | 2.5 | 0.9 | 11.5 | — | — | — | — | No |
| 24 | ELANE | 1.4 | 0.3 | 10 | — | — | — | — | Yes |
| 25 | ELANE | 1.0 | 60.0 | 9 | 93 | 5-20 | 0.05 | 0.98 | Yes |
| 26 | ELANE | 5.0 | 51.4 | 17 | 3 | 5 | 0.40 | 2.24 | No |
| 27 | ELANE | 2.5 | 19.6 | 12 | 50 | 5-7.5 | 0.18 | 1.71 | Yes |
| 28 | ELANE | 0.5 | 12.3 | 6 | 3 | 5 | 0.96 | 1.30 | No |
| 29 | ELANE | 0.8 | 1.5 | 8 | 4 | 5 | 0.90 | 1.07 | No |
| 30 | ELANE | 1.8 | 38.5 | 10 | 46 | 5-7.5 | 0.55 | 0.92 | Yes |
| 31 | ELANE | 6.5 | 15.4 | 16 | 9 | 5 | 0.42 | 0.55 | Yes |
| 32 | ELANE | 3.0 | 1.6 | 13 | 1 | 5 | 0.20 | — | No |
| 33 | ELANE | 1.1 | 1.4 | 10 | 10 | 5-10 | 0.11 | 3.81 | Yes |
| 34 | ELANE | 13.2 | 0.6 | 12 | 6 | 5-10 | 0.43 | 1.34 | No |
| 35 | ELANE | 6.6 | 3.3 | 22 | 7 | 5-10 | 0.18 | 2.37 | Yes |
| 36 | ELANE | 0.1 | 15.3 | 3 | 7 | 10-15 | 0.08 | 1.50 | No |
| 37 | HAX1 | 1.0 | 13.7 | 10 | 9 | 5-15 | 0.06 | 1.19 | No |
| 38 | HAX1 | 3.7 | 1.2 | 11 | 2 | 5 | 0.03 | 0.51 | No |
| 39 | HAX1 | 10.9 | 56.5 | 31 | 5 | 5 | 0.22 | 1.68 | No |
| 40 | HAX1 | 0.3 | 94.6 | 19.5 | 6 | 5 | 0.47 | 2.17 | No |
| 41 | CXCR4 | 8.9 | 59.4 | 35 | — | — | — | — | No |
| 42 | CXCR4 | 0.9 | 68.3 | 8.5 | 10 | 5 | 0.15 | 5.25 | No |
| 43 | CXCR4 | 5.1 | 1.9 | 19 | 8 | 5 | 0.10 | 7.39 | No |
| 44 | CXCR4 | 0.1 | 45.2 | 5 | 1 | 5 | 0.32 | 6.70 | No |
| 45 | CXCR4 | 7.3 | 3.7 | 22 | 1 | 5 | 0.92 | 1.36 | No |
| 46 | SBDS | 0.6 | 33.2 | 6.4 | — | — | — | — | No |
| 47 | SBDS | 5.1 | 26.2 | 15 | 10 | 7.5 | 0.42 | 22.16 | No |
| 48 | SBDS | 0.3 | 7.2 | 6 | — | — | — | — | No |
| 49 | SBDS | 1.7 | 4.1 | 9 | — | — | — | — | No |
| 50 | SBDS | 0.9 | 0.3 | 8.5 | 1 | 5 | 0.52 | 3.14 | No |
| 51 | SBDS | 0.3 | 1.4 | 5 | 4 | 5 | 0.36 | 1.32 | No |
| 52 | SBDS | 0.4 | 29.4 | 6 | — | — | — | — | No |
| 53 | SBDS | 0.6 | 0.8 | 7.8 | 1 | 5 | 0.37 | 2.39 | No |
| 54 | WAS | 6.4 | 6.5 | NA | — | — | — | — | No |
| 55 | G6PC3 | 0.8 | 13.1 | 8 | — | — | — | — | No |
| 56 | SPR54 | 3.1 | 2.9 | 14.5 | — | — | — | — | No |
—, not receiving G-CSF treatment.
∗
HSCT, hematopoietic stem cell transplantation.
†
bANC, last absolute baseline neutrophil count (×109 cells per L) before G-CSF initiation.
‡
pANC, peak ANC (×109 cells per L) within 10 days following G-CSF initiation.