Patient characteristics
| Characteristic . | DC cohort (separate capsules) . | FDC cohort (FDC tablet) . | Phase 2 overall (N = 80) . | ||||
|---|---|---|---|---|---|---|---|
| Sequence A (n = 25) . | Sequence B (n = 25) . | Total (n = 50) . | Sequence A (n = 16) . | Sequence B (n = 14) . | Total (n = 30) . | ||
| Mean age, y (SD) | 69.3 (11.2) | 70.2 (10.5) | 69.7 (10.7) | 69.9 (12.1) | 69.4 (9.0) | 69.6 (10.6) | 69.7 (10.6) |
| Median (range) | 69 (32-87) | 72 (41-86) | 71.5 (32-87) | 71 (40-90) | 70 (53-82) | 70.5 (40-90) | 71 (32-90) |
| Sex | |||||||
| Male | 20 (80) | 21 (84) | 41 (82) | 12 (75) | 8 (57) | 20 (67) | 61 (76) |
| Female | 5 (20) | 4 (16) | 9 (18) | 4 (25) | 6 (43) | 10 (33) | 19 (24) |
| Ethnic origin | |||||||
| White | 24 (96) | 22 (88) | 46 (92) | 14 (88) | 14 (100) | 28 (93) | 74 (93) |
| Black or African American | 1 (4) | 1 (4) | 2 (4) | 0 (0) | 0 (0) | 0 (0) | 2 (2.5) |
| Other | 0 (0) | 2 (8) | 2 (4) | 2 (12) | 0 (0) | 2 (7) | 4 (5) |
| Median weight, kg (range) | 82 (40-122) | 87 (55-118) | 85 (40-122) | 76 (49-100) | 83 (42-98) | 80 (42-100) | 83 (40-122) |
| Mean BSA, m2 (SD) | 1.9 (0.3) | 2.0 (0.2) | 2.0 (0.3) | 1.9 (0.2) | 1.9 (0.3) | 1.9 (0.2) | 2.0 (0.3) |
| ECOG performance status | |||||||
| 0 | 13 (52) | 9 (36) | 22 (44) | 7 (44) | 6 (43) | 13 (43) | 35 (44) |
| 1 | 9 (36) | 15 (60) | 24 (48) | 9 (56) | 5 (36) | 14 (47) | 38 (48) |
| 2 | 3 (12) | 1 (4) | 4 (8) | 0 (0) | 3 (21) | 3 (10) | 7 (9) |
| Disease and IPSS category | |||||||
| MDS intermediate 1 | 10 (40) | 10 (40) | 20 (40) | 9 (56) | 6 (43) | 15 (50) | 35 (44) |
| MDS intermediate 2 | 6 (24) | 7 (28) | 13 (26) | 3 (19) | 3 (21) | 6 (20) | 19 (24) |
| MDS high risk | 4 (16) | 4 (16) | 8 (16) | 1 (6) | 0 (0) | 1 (3) | 9 (11) |
| CMML | 5 (20) | 4 (16) | 9 (18) | 3 (19) | 5 (36) | 8 (27) | 17 (21) |
| Prior HMA | 2 (8) | 1 (4) | 3 (6) | 1 (6) | 3 (21) | 4 (13) | 7 (9) |
| RBC transfusion dependence | 9 (36) | 13 (52) | 22 (44) | 11 (69) | 5 (36) | 16 (53) | 38 (47) |
| >5% bone marrow blasts | 14 (61) | 14 (56) | 28 (58) | 8 (53) | 5 (36) | 13 (45) | 41 (53) |
| Characteristic . | DC cohort (separate capsules) . | FDC cohort (FDC tablet) . | Phase 2 overall (N = 80) . | ||||
|---|---|---|---|---|---|---|---|
| Sequence A (n = 25) . | Sequence B (n = 25) . | Total (n = 50) . | Sequence A (n = 16) . | Sequence B (n = 14) . | Total (n = 30) . | ||
| Mean age, y (SD) | 69.3 (11.2) | 70.2 (10.5) | 69.7 (10.7) | 69.9 (12.1) | 69.4 (9.0) | 69.6 (10.6) | 69.7 (10.6) |
| Median (range) | 69 (32-87) | 72 (41-86) | 71.5 (32-87) | 71 (40-90) | 70 (53-82) | 70.5 (40-90) | 71 (32-90) |
| Sex | |||||||
| Male | 20 (80) | 21 (84) | 41 (82) | 12 (75) | 8 (57) | 20 (67) | 61 (76) |
| Female | 5 (20) | 4 (16) | 9 (18) | 4 (25) | 6 (43) | 10 (33) | 19 (24) |
| Ethnic origin | |||||||
| White | 24 (96) | 22 (88) | 46 (92) | 14 (88) | 14 (100) | 28 (93) | 74 (93) |
| Black or African American | 1 (4) | 1 (4) | 2 (4) | 0 (0) | 0 (0) | 0 (0) | 2 (2.5) |
| Other | 0 (0) | 2 (8) | 2 (4) | 2 (12) | 0 (0) | 2 (7) | 4 (5) |
| Median weight, kg (range) | 82 (40-122) | 87 (55-118) | 85 (40-122) | 76 (49-100) | 83 (42-98) | 80 (42-100) | 83 (40-122) |
| Mean BSA, m2 (SD) | 1.9 (0.3) | 2.0 (0.2) | 2.0 (0.3) | 1.9 (0.2) | 1.9 (0.3) | 1.9 (0.2) | 2.0 (0.3) |
| ECOG performance status | |||||||
| 0 | 13 (52) | 9 (36) | 22 (44) | 7 (44) | 6 (43) | 13 (43) | 35 (44) |
| 1 | 9 (36) | 15 (60) | 24 (48) | 9 (56) | 5 (36) | 14 (47) | 38 (48) |
| 2 | 3 (12) | 1 (4) | 4 (8) | 0 (0) | 3 (21) | 3 (10) | 7 (9) |
| Disease and IPSS category | |||||||
| MDS intermediate 1 | 10 (40) | 10 (40) | 20 (40) | 9 (56) | 6 (43) | 15 (50) | 35 (44) |
| MDS intermediate 2 | 6 (24) | 7 (28) | 13 (26) | 3 (19) | 3 (21) | 6 (20) | 19 (24) |
| MDS high risk | 4 (16) | 4 (16) | 8 (16) | 1 (6) | 0 (0) | 1 (3) | 9 (11) |
| CMML | 5 (20) | 4 (16) | 9 (18) | 3 (19) | 5 (36) | 8 (27) | 17 (21) |
| Prior HMA | 2 (8) | 1 (4) | 3 (6) | 1 (6) | 3 (21) | 4 (13) | 7 (9) |
| RBC transfusion dependence | 9 (36) | 13 (52) | 22 (44) | 11 (69) | 5 (36) | 16 (53) | 38 (47) |
| >5% bone marrow blasts | 14 (61) | 14 (56) | 28 (58) | 8 (53) | 5 (36) | 13 (45) | 41 (53) |
Data are n (%) unless otherwise specified.
BSA, body surface area; DC, dose confirmation; FDC, fixed dose combination; ECOG, Eastern Cooperative Oncology Group; IPSS, International Prognostic Scoring System; RBC, red blood cell; SD, standard deviation.