Table 1.
Patient demographics and baseline characteristics
| Characteristic | R/I to imatinib or dasatinib* (n = 33†) | Newly diagnosed (n = 25) |
|---|---|---|
| Age, y | ||
| <12 | 12 (36.4) | 6 (24.0) |
| 12 to <18 | 21 (63.6) | 19 (76.0) |
| Median (range) | 13 (2-17) | 13 (10-16) |
| Female | 12 (36.4) | 12 (48.0) |
| Prior antineoplastic TKI therapies | ||
| Imatinib | 31 (93.9) | NA |
| Dasatinib | 2 (6.1) | NA |
| Intolerant | ||
| Imatinib | 6 (18.2)‡ | NA |
| Dasatinib | 0 | NA |
| Resistant | ||
| Imatinib | 28 (84.8)‡ | NA |
| Dasatinib | 2 (6.1) | NA |
| BCR-ABL1IS at baseline | ||
| ≤0.0032 | 1 (3.0) | 0 |
| >0.0032 to ≤0.01 | 1 (3.0) | 0 |
| >0.01 to ≤0.1 | 5 (15.2) | 0 |
| >0.1 to ≤1 | 10 (30.3) | 0 |
| >1 to ≤10 | 9 (27.3) | 0 |
| >10 | 5 (15.2) | 25 (100) |
| Atypical transcripts | 1 (3.0) | 0 |
| Missing | 1 (3.0) | 0 |
| Known BCR-ABL1 mutation at baseline, n/m§ | 3/29 | NA |
Data are presented as number (%) unless otherwise specified.
IS, International Scale; NA, not applicable.
Patients were resistant to and/or intolerant of 1 previous TKI, either imatinib or dasatinib.
One additional patient was enrolled in this cohort but did not receive nilotinib treatment and was excluded from the analyses.
Three patients were both imatinib intolerant and imatinib resistant and are counted in both categories.
Numerator (n) is the number of patients with known baseline mutations. Denominator (m) is the number of patients with an evaluable baseline mutational assessment. Mutations detected at baseline were E255K and E255V in 1 patient, G250E and E255K in 1 patient, and L387M in 1 patient.