Baseline characteristics of the CLAG-M/sorafenib study cohort and historical cohort of patients treated with CLAG-M
| . | CLAG-M/sorafenib . | CLAG-M . | P value∗ . | ||
|---|---|---|---|---|---|
| All patients (N = 81) (%) . | Phase 1 cohort (n = 46) (%) . | RP2D cohort (n = 41) (%) . | All patients (N = 76) (%) . | ||
| Age, median (range), y | 50 (21-60) | 48 (22-60) | 50 (21-60) | 46 (18-60) | .16 |
| ECOG performance status | 1.00 | ||||
| 0-1 | 75 (93) | 43 (93) | 38 (93) | 71 (93) | |
| 2-3 | 6 (7) | 3 (7) | 3 (7) | 5 (7) | |
| Laboratory values at diagnosis | |||||
| White blood cell count | 10.2 (0.7-183.3) | 9.68 (0.94-183.3) | 11.1 (0.73-351.0) | 5.53 (0.58-240.4) | .17 |
| Platelet count | 47 (9-361) | 47 (9-257) | 58 (12-351) | 49 (6-794) | .39 |
| Creatinine | 0.84 (0.44-1.63) | 0.84 (0.44-1.63) | 0.84 (0.47-1.45) | 0.85 (0.34-1.37) | .89 |
| Bilirubin | 0.6 (0.2-1.6) | 0.6 (0.2-1.6) | 0.5 (0.3-1.6) | 0.6 (0-2.2) | .99 |
| TRM score, median (range) | 2.08 (0.19-12.26) | 2.06 (0.20-12.26) | 2.28 (0.20-8.52) | 1.99 (0.01-10.95) | .33 |
| Disease | .62 | ||||
| AML | 66 (81) | 39 (85) | 33 (80) | 64 (84) | |
| MDS-EB-2 | 12 (15) | 7 (15) | 8 (20) | 12 (16) | |
| Other | 3 (4) | 0 | 0 | 0 | |
| Secondary disease† | 12 (15) | 6 (13) | 6 (15) | 15 (20) | .62 |
| Cytogenetic risk (MRC) | .71 | ||||
| Favorable | 4 (5) | 1 (2) | 3 (7) | 9 (12) | |
| Intermediate | 59 (73) | 36 (78) | 29 (71) | 46 (62) | |
| Adverse | 18 (22) | 9 (20) | 9 (22) | 17 (23) | |
| Unknown | 0 | 0 | 0 | 8 (11) | |
| Cytogenetic/molecular risk (ELN 2017) | .02 | ||||
| Favorable | 24 (30) | 14 (30) | 11 (27) | 30 (39) | |
| NPM1 | 16 | 9 | 7 | 20 | |
| CEBPA | 6 | 4 | 2 | 5 | |
| Intermediate | 25 (31) | 13 (28) | 16 (39) | 14 (18) | |
| Adverse | 31 (38) | 18 (39) | 14 (34) | 24 (32) | |
| Unknown | 1 (1) | 1 (2) | 0 | 8 (11) | |
| FLT3-ITD, n (%) | 21 (26) | 11 (24) | 13 (32) | 9 (13) | .03 |
| FLT3-TKD, n (%) | 4 (5) | 3 (7) | 2 (5) | 5 (19) | .11 |
| . | CLAG-M/sorafenib . | CLAG-M . | P value∗ . | ||
|---|---|---|---|---|---|
| All patients (N = 81) (%) . | Phase 1 cohort (n = 46) (%) . | RP2D cohort (n = 41) (%) . | All patients (N = 76) (%) . | ||
| Age, median (range), y | 50 (21-60) | 48 (22-60) | 50 (21-60) | 46 (18-60) | .16 |
| ECOG performance status | 1.00 | ||||
| 0-1 | 75 (93) | 43 (93) | 38 (93) | 71 (93) | |
| 2-3 | 6 (7) | 3 (7) | 3 (7) | 5 (7) | |
| Laboratory values at diagnosis | |||||
| White blood cell count | 10.2 (0.7-183.3) | 9.68 (0.94-183.3) | 11.1 (0.73-351.0) | 5.53 (0.58-240.4) | .17 |
| Platelet count | 47 (9-361) | 47 (9-257) | 58 (12-351) | 49 (6-794) | .39 |
| Creatinine | 0.84 (0.44-1.63) | 0.84 (0.44-1.63) | 0.84 (0.47-1.45) | 0.85 (0.34-1.37) | .89 |
| Bilirubin | 0.6 (0.2-1.6) | 0.6 (0.2-1.6) | 0.5 (0.3-1.6) | 0.6 (0-2.2) | .99 |
| TRM score, median (range) | 2.08 (0.19-12.26) | 2.06 (0.20-12.26) | 2.28 (0.20-8.52) | 1.99 (0.01-10.95) | .33 |
| Disease | .62 | ||||
| AML | 66 (81) | 39 (85) | 33 (80) | 64 (84) | |
| MDS-EB-2 | 12 (15) | 7 (15) | 8 (20) | 12 (16) | |
| Other | 3 (4) | 0 | 0 | 0 | |
| Secondary disease† | 12 (15) | 6 (13) | 6 (15) | 15 (20) | .62 |
| Cytogenetic risk (MRC) | .71 | ||||
| Favorable | 4 (5) | 1 (2) | 3 (7) | 9 (12) | |
| Intermediate | 59 (73) | 36 (78) | 29 (71) | 46 (62) | |
| Adverse | 18 (22) | 9 (20) | 9 (22) | 17 (23) | |
| Unknown | 0 | 0 | 0 | 8 (11) | |
| Cytogenetic/molecular risk (ELN 2017) | .02 | ||||
| Favorable | 24 (30) | 14 (30) | 11 (27) | 30 (39) | |
| NPM1 | 16 | 9 | 7 | 20 | |
| CEBPA | 6 | 4 | 2 | 5 | |
| Intermediate | 25 (31) | 13 (28) | 16 (39) | 14 (18) | |
| Adverse | 31 (38) | 18 (39) | 14 (34) | 24 (32) | |
| Unknown | 1 (1) | 1 (2) | 0 | 8 (11) | |
| FLT3-ITD, n (%) | 21 (26) | 11 (24) | 13 (32) | 9 (13) | .03 |
| FLT3-TKD, n (%) | 4 (5) | 3 (7) | 2 (5) | 5 (19) | .11 |
CEBPA, CCAAT enhancer-binding protein alpha; EB, excess blasts; ECOG, Eastern Cooperative Oncology Group; MDS, myelodysplastic syndrome; MRC, Medical Research Council; TRM, treatment-related mortality score.
Comparison CLAG-M/sorafenib RP2D cohort vs CLAG-M cohort.
Defined as AML transformed from antecedent hematologic disorder or AML/myelodysplastic syndrome in a patient who had previously received cytotoxic therapy.