Table 1. Patient demographic and disease characteristics at baseline.
| Characteristic | CC-486 300 mg Once daily 14 days/cycle (n=28) | CC-486 300 mg Once daily 21 days/cycle (n=27) |
|---|---|---|
| Age (years), median (range) | 72.5 (51–85) | 70.0 (31–87) |
| RBC transfusion-dependent,a n (%) | 16 (57) | 16 (59) |
| Platelet transfusion-dependent,b n (%) | 4 (14) | 2 (7) |
| Hematology, median (range) | ||
| Hgb (g/dl) | 8.6 (6.4–13.0) | 8.7 (6.0–11.6) |
| ANC (109/l) | 1.3 (0–21.5) | 1.8 (0.4–30.3) |
| Platelets (109/l) | 69.0 (6.0–564.0) | 56.0 (8.0–362.0) |
| WBC (109/l) | 3.0 (0.9–26.2) | 3.5 (0.9–42.1) |
| MDS WHO classification, n (%) | ||
| RA/RARSc | 9 (32) | 9 (33) |
| RCMD/RCMD-RSc | 10 (36) | 7 (26) |
| RAEB-1 | 4 (14) | 4 (15) |
| RAEB-2 | 0 | 1d (4) |
| MDS-U | 3 (11) | 3 (11) |
| Del(5q) | 1 (4) | 1 (4) |
| Missing | 1 (4) | 2 (7) |
| IPSS risk classification, n (%) | ||
| Low | 6 (21) | 8 (30) |
| Intermediate-1 | 22 (79) | 19 (70) |
| IPSS-R risk classification, n (%) | ||
| Low/very low | 11 (39) | 14 (52) |
| Intermediate | 7 (25) | 7 (26) |
| High | 9 (32) | 6 (22) |
| Unknown | 1 (4) | 0 |
| Cytogenetics, n (%) | ||
| Normal/diploid | 12 (43) | 15 (56) |
| ⩾1 abnormality | 9 (32) | 10 (37) |
| Indeterminate | 6 (21) | 2 (7) |
| Prior treatment, n (%) | ||
| Erythropoiesis-stimulating agents | 16 (57) | 10 (37) |
| Granulocyte colony-stimulating factors | 5 (18) | 4 (15) |
| Other | 8 (29) | 5 (19) |
| Nonee | 9 (32) | 12 (44) |
Abbreviations: ANC, absolute neutrophil count; Hgb, hemoglobin; IPSS, International Prognostic Scoring System; MDS, myelodysplastic syndrome; MDS-U, myelodysplastic syndrome-unclassified; RA, refractory anemia; RAEB, refractory anemia with excess blasts; RARS, refractory anemia with ringed sideroblasts; RBC, red blood cell; RCMD, refractory cytopenia with multilineage dysplasia; RCMD-RS, refractory cytopenia with multilineage dysplasia and ringed sideroblasts; WBC, white blood cell.
Defined as receipt of ⩾4 units of packed RBC within 56 days of the first dose of CC-486.
Defined as receipt of ⩾2 platelet transfusions within 56 days of the first dose of CC-486.
Because of the limited number of patients in the study, these classifications were grouped prospectively.
Assessed as lower-risk MDS by the treating physician on the case report form.
Other than transfusions.