Table 1.
Baseline Demographics and Disease Characteristics (SAF)
| 20 mg (n=16) | 40 mg (n=16) | 80 mg (n=24) | 120 mg (n=70) | 200 mg (n=103) | 300 mg (n=20) | 450 mg (n=3) | |
|---|---|---|---|---|---|---|---|
| Age (years), median (IQR) | 64·5 (58, 71) | 62 (54, 66 ) | 62 (47, 70) | 59·5 (51, 69) | 64 (49, 70) | 64 (46, 69) | 64 (50, 71) |
| Sex, n | |||||||
| Male | 6 (38%) | 11 (69%) | 11 (46%) | 32 (46%) | 52 (51%) | 14 (70%) | 3 (100%) |
| Female | 10 (63%) | 5 (31%) | 13 (54%) | 38 (54%) | 51 (50%) | 6 (30%) | 0 |
| Cytogenetic risk status, na | |||||||
| Favorable | 0 | 0 | 2 (8%) | 1 (1%) | 3 (3%) | 0 | 1 (33%) |
| Intermediate | 13 (81%) | 5 (31%) | 11 (46%) | 42 (60%) | 64 (62%) | 8 (40%) | 0 |
| Unfavorable | 2 (13%) | 9 (56%) | 7 (29%) | 12 (17%) | 17 (17%) | 7 (35%) | 2 (67%) |
| AML disease history | |||||||
| Duration of disease (months), median (IQR) | 10·6 (7·2, 16·1) | 7·1 (5·1, 11·7) | 16·8 (8·3, 29) | 9·0 (4·7, 16·6) | 8·3 (3·9, 13·8) | 7·3 (2·7, 16·5) | 6·3 (3·5, 11·9) |
| Prior stem cell transplant, n | |||||||
| 0 | 11 (69%) | 13 (81%) | 15 (63%) | 49 (70%) | 71 (69%) | 18 (90%) | 2 (67%) |
| 1 | 4 (25%) | 2 (13%) | 9 (38%) | 20 (29%) | 29 (28%) | 2 (10%) | 1 (33%) |
| ≥2 | 1 (6%) | 1 (6%) | 0 | 1 (1%) | 3 (3%) | 0 | 0 |
| Lines of prior AML therapy, n | |||||||
| 1 | 3 (19%) | 5 (31%) | 5 (21%) | 17 (24%) | 36 (35%) | 7 (35%) | 2 (67%) |
| 2 | 3 (19%) | 1 (6%) | 5 (21%) | 22 (31%) | 28 (27%) | 7 (35%) | 0 |
| ≥3 | 10 (63%) | 10 (63%) | 14 (58%) | 31 (44%) | 39 (38%) | 6 (30%) | 1 (33%) |
| Prior TKI therapy, n | |||||||
| No | 8 (50%) | 12 (75%) | 19 (79%) | 48 (69%) | 82 (80%) | 18 (90%) | 2 (67%) |
| Yes | 8 (50%) | 4 (25%) | 5 (21%) | 22 (31%) | 21 (20%) | 2 (10%) | 1 (33%) |
| TKI therapy usagea,b, n | |||||||
| Sorafenib | 6 (75%) | 4 (100%) | 5 (100%) | 19 (83%) | 18 (82%) | 1 (50%) | 1 (100%) |
| PLX 3397 | 2 (25%) | 2 (50%) | 1 (20%) | 3 (13%) | 0 | 0 | 0 |
| Quizartinib | 0 | 0 | 0 | 1 (4%) | 4 (18%) | 1 (50%) | 0 |
| Crenolanib | 1 (13%) | 0 | 0 | 1 (4%) | 0 | 0 | 0 |
| FLT3 mutation type, n c | |||||||
| FLT3-ITD | 12 (75%) | 6 (38%) | 10 (42%) | 47 (67%) | 79 (77%) | 8 (40%) | 0 |
| FLT3-D835 | 1 (6%) | 0 | 1 (4%) | 6 (9%) | 3 (3%) | 1 (5%) | 1 (33%) |
| FLT3-ITD and -D835 | 1 (6%) | 2 (13%) | 1 (4%) | 3 (4%) | 8 (8%) | 0 | 1 (33%) |
AML, acute myeloid leukemia; FLT3, FMS-like tyrosine kinase; IQR, interquartile range; SAF, safety analysis set; TKI, tyrosine kinase inhibitor.
a
Values represent the proportion of patients who had received prior TKI therapy
b
Patients may have received more than one prior TKI;
c
FLT3 mutation status was determined by a local laboratory.