Baseline characteristics
| . | R/I cohort∗ n = 33 . | ND cohort n = 25 . |
|---|---|---|
| Median age (range), y | 13 (2-17) | 13 (10-16) |
| Patients aged 1 to <12 y, n (%) | 12 (36.4) | 6 (24.0) |
| Patients aged 12 to <18 y, n (%) | 21 (63.6) | 19 (76.0) |
| Female, n (%) | 12 (36.4) | 12 (48.0) |
| Male, n (%) | 21 (63.6) | 13 (52.0) |
| Median BMI SDS, (range) | −0.57 (−3.4 to 3.4) | −0.09 (−2.8 to 2.1) |
| Median height SDS, (range) | −0.56 (−4.3 to 1.2) | 0.06 (−0.9 to 1.7) |
| Pubertal status, n (%) | ||
| Tanner stage 1 (prepubertal) | 9 (27.3) | 4 (16.0) |
| Tanner stage 2-4 | 15 (45.5) | 17 (68.0) |
| Tanner stage 5† | 8 (24.2) | 4 (16.0) |
| Missing, n (%) | 1 (3.0) | 0 |
| Prior antineoplastic TKI therapies, n (%) | ||
| Imatinib | 31 (93.9) | N/A |
| Dasatinib | 2 (6.1) | N/A |
| Intolerant of imatinib/dasatinib, n (%)‡ | 6 (18.2)/0 | N/A |
| Resistant to imatinib/dasatinib, n (%)‡ | 28 (84.8)/2 (6.1) | N/A |
| BCR::ABL1IS, n (%) | ||
| ≤0.0032 | 1 (3.0) | 0 |
| >0.0032 to ≤0.01 | 1 (3.0) | 0 |
| >0.01 to ≤0.1 | 5 (15.2) | 0 |
| CCyR at baseline, n (%) | 14 (42.4) | 0 |
| Known BCR::ABL1 mutation at baseline, n/m§ | 3/29 | 0/25 |
| Presence of other chromosomal abnormalities in Ph+ metaphases, n (%) | 2 (6.1)‖ | 3 (12.0)¶ |
| Presence of chromosomal abnormalities in Ph− metaphases, n (%) | 2 (6.1)# | 0 |
| . | R/I cohort∗ n = 33 . | ND cohort n = 25 . |
|---|---|---|
| Median age (range), y | 13 (2-17) | 13 (10-16) |
| Patients aged 1 to <12 y, n (%) | 12 (36.4) | 6 (24.0) |
| Patients aged 12 to <18 y, n (%) | 21 (63.6) | 19 (76.0) |
| Female, n (%) | 12 (36.4) | 12 (48.0) |
| Male, n (%) | 21 (63.6) | 13 (52.0) |
| Median BMI SDS, (range) | −0.57 (−3.4 to 3.4) | −0.09 (−2.8 to 2.1) |
| Median height SDS, (range) | −0.56 (−4.3 to 1.2) | 0.06 (−0.9 to 1.7) |
| Pubertal status, n (%) | ||
| Tanner stage 1 (prepubertal) | 9 (27.3) | 4 (16.0) |
| Tanner stage 2-4 | 15 (45.5) | 17 (68.0) |
| Tanner stage 5† | 8 (24.2) | 4 (16.0) |
| Missing, n (%) | 1 (3.0) | 0 |
| Prior antineoplastic TKI therapies, n (%) | ||
| Imatinib | 31 (93.9) | N/A |
| Dasatinib | 2 (6.1) | N/A |
| Intolerant of imatinib/dasatinib, n (%)‡ | 6 (18.2)/0 | N/A |
| Resistant to imatinib/dasatinib, n (%)‡ | 28 (84.8)/2 (6.1) | N/A |
| BCR::ABL1IS, n (%) | ||
| ≤0.0032 | 1 (3.0) | 0 |
| >0.0032 to ≤0.01 | 1 (3.0) | 0 |
| >0.01 to ≤0.1 | 5 (15.2) | 0 |
| CCyR at baseline, n (%) | 14 (42.4) | 0 |
| Known BCR::ABL1 mutation at baseline, n/m§ | 3/29 | 0/25 |
| Presence of other chromosomal abnormalities in Ph+ metaphases, n (%) | 2 (6.1)‖ | 3 (12.0)¶ |
| Presence of chromosomal abnormalities in Ph− metaphases, n (%) | 2 (6.1)# | 0 |
BMI, body mass index; N/A, not applicable.
Patients were resistant to and/or intolerant of 1 prior TKI, either imatinib or dasatinib.
Patients had completed puberty before study enrollment.
Three patients were both intolerant of and resistant to imatinib and are counted once as resistant and once as intolerant.
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.
Data were available for 30 patients with R/I CML-CP. The abnormalities in Ph+ metaphases were 47,XX, t(9,22)(Q34;Q11.2), +der(22)t(9;22)[18]/46, XX[2] in 1 patient and a 3-way translocation between chromosome 4, 9, and 22 in 1 patient.
The abnormalities in Ph+ metaphases were chromosome-3 abnormality in 1 patient, 46,IDEM,add(14)(Q22) OR inv(14)(Q22Q32)[14] in 1 patient, and trisomy-19 in 1 patient.
Data were available for 30 patients with R/I CML-CP. The abnormalities in Ph− metaphases were 46, XX, del(17)(p?) and t(11;17) (1 patient each).