Baseline disease characteristics
| Characteristic . | Placebo . | Zosuquidar . | All patients . |
|---|---|---|---|
| Total | 221 | 212 | 433 |
| Disease, no. (%) | |||
| De novo AML | 137 (62) | 123 (59) | 260 (61%) |
| RAEB-t (WHO-AML) | 7 (3) | 15 (7) | 22 (5%) |
| HR-RAEB (IPSS) | 6 (3) | 8 (4) | 14 (3%) |
| Secondary AML or MDS | 70 (32) | 63 (30) | 133 (31%) |
| Unknown/missing | 1 | 3 | 4 |
| Diagnostic karyotype, no. (%) | |||
| Evaluable | 173 (78.3) | 164 (77.0) | 337 (77.6) |
| Not evaluable/not submitted | 48 (21.7) | 48 (23.0) | 96 (22.4) |
| Cytogenetic risk classification, no. (%) | |||
| Favorable | 8 (5.3) | 4 (2.7) | 12 (4.0) |
| Intermediate | 68 (45.3) | 75 (50.3) | 143 (47.8) |
| Unfavorable | 74 (49.3) | 70 (47.0) | 144 (48.2) |
| Unknown/missing | 71 | 63 | 134 |
| Peripheral WBCs, ×103/μL | |||
| Median (IQR) | 3.9 (1.7-16.5) | 7.6 (3.2-26.3) | 5.5 (1.9-22.2) |
| Peripheral blast percentage | |||
| Median (IQR) | 10 (1-35) | 18 (3-46) | 14 (1-40) |
| Rh-123 efflux with or without zosuquidar | |||
| Median (IQR) | 2.0 (1.2-3.2) | 1.8 (1.2-3.1) | 2.0 (1.2-3.1) |
| Ratio > 1.3, no. (%) | 133 (70.4) | 123 (65.8) | 256 (68.1) |
| Unknown/missing | 32 | 25 | 57 |
| MRK 16 attaining | |||
| % positive, median (IQR) | 42 (18-87) | 32 (15-81) | 38 (15-84) |
| Unknown/missing | 48 | 32 | 80 |
| Characteristic . | Placebo . | Zosuquidar . | All patients . |
|---|---|---|---|
| Total | 221 | 212 | 433 |
| Disease, no. (%) | |||
| De novo AML | 137 (62) | 123 (59) | 260 (61%) |
| RAEB-t (WHO-AML) | 7 (3) | 15 (7) | 22 (5%) |
| HR-RAEB (IPSS) | 6 (3) | 8 (4) | 14 (3%) |
| Secondary AML or MDS | 70 (32) | 63 (30) | 133 (31%) |
| Unknown/missing | 1 | 3 | 4 |
| Diagnostic karyotype, no. (%) | |||
| Evaluable | 173 (78.3) | 164 (77.0) | 337 (77.6) |
| Not evaluable/not submitted | 48 (21.7) | 48 (23.0) | 96 (22.4) |
| Cytogenetic risk classification, no. (%) | |||
| Favorable | 8 (5.3) | 4 (2.7) | 12 (4.0) |
| Intermediate | 68 (45.3) | 75 (50.3) | 143 (47.8) |
| Unfavorable | 74 (49.3) | 70 (47.0) | 144 (48.2) |
| Unknown/missing | 71 | 63 | 134 |
| Peripheral WBCs, ×103/μL | |||
| Median (IQR) | 3.9 (1.7-16.5) | 7.6 (3.2-26.3) | 5.5 (1.9-22.2) |
| Peripheral blast percentage | |||
| Median (IQR) | 10 (1-35) | 18 (3-46) | 14 (1-40) |
| Rh-123 efflux with or without zosuquidar | |||
| Median (IQR) | 2.0 (1.2-3.2) | 1.8 (1.2-3.1) | 2.0 (1.2-3.1) |
| Ratio > 1.3, no. (%) | 133 (70.4) | 123 (65.8) | 256 (68.1) |
| Unknown/missing | 32 | 25 | 57 |
| MRK 16 attaining | |||
| % positive, median (IQR) | 42 (18-87) | 32 (15-81) | 38 (15-84) |
| Unknown/missing | 48 | 32 | 80 |
The only statistically significant differences in baseline disease characteristics between patients randomized to zosuquidar or placebo are for WBCs and peripheral blasts, with P values of .015 and .011, respectively.
Secondary AML indicates antecedent hematologic disease or chemotherapy or radiation therapy for a nonmyeloid malignancy; HR-RAEB, high-risk refractory anemia with excess blasts; RAEB-t, refractory anemia with excess blast in transformation; WHO, World Health Organization, WBC, white blood cell count; and Rh-123, rhodamine-123.