Table 1.
Demographics of patients by cohort (N=188). Data expressed as median (range).
| Cohort A Conventional dosing (N = 53) |
Cohort B Model-Based Excel tool (N = 76) |
Cohort C Model-Based Bayesian tool (N = 59) |
||||
|---|---|---|---|---|---|---|
| Malignant Disease | Non-malignant Disease | Malignant Disease | Non-malignant Disease | Malignant Disease | Non-malignant Disease | |
| Number of subjects (n) | 25 | 28 | 40 | 36 | 42 | 17 |
| Weight (kg) | 33 (7–101) | 18 (3–61) | 24 (8–98) | 10.1 (4.9–62) | 17.8 (8.1–150) | 10.2 (5.8–79) |
| Age (years) | 8.8 (0.21–29) | 4.9 (0.1–21) | 6.2 (0.9–24) | 1.2 (0.24–19) | 5.9 (0.9–20) | 1.4 (0.2–17) |
| Gender (M/F) | 14/11 | 17/11 | 28/12 | 19/17 | 28/14 | 13/4 |
| Malignant disease | ||||||
| Acute myelogenous leukemia | 16 | 17 | 11 | |||
| Chronic myelogenous leukemia | 3 | 3 | 1 | |||
| Juvenile myelomonocytic leukemia | 3 | 3 | 4 | |||
| Myelodysplastic syndrome | 3 | 0 | 2 | |||
| Acute lymphoblastic leukemia | 0 | 9 | 10 | |||
| Neuroblastoma | 0 | 8 | 10 | |||
| Other | 4 | |||||
| Non-malignant disease | ||||||
| Primary immune deficiencies | 13 | 23 | 9 | |||
| Inborn errors of metabolism | 4 | 7 | 3 | |||
| Hemoglobinopathies | 8 | 6 | 3 | |||
| Congenital neutropenia | 3 | 0 | 2 | |||
| Predefined cAUC target (mg*h/L) | 86 (58–86) | 58 (58–86) | 82 (62–90) | 62 (29–82) | 83 (60–90) | 60 (17–85) |
| Observed cAUC target (mg*h/L) | 80 (43–110) | 72 (53–108) | 78 (54–170) | 63 (24–102) | 83 (59–94) | 61 (15–85) |
M, male; F, female.