Table 2. Hematologic response and transfusion independence.
| Parameter |
Treatment schedule n responders/N evaluable (%) |
||
|---|---|---|---|
| CC-486 300 mg once daily 14 days/cycle (n=28) | CC-486 300 mg once daily 21 days/cycle (n=27) | Total (N=55) | |
| Overall response (CR, PR, any HI, TI)a | 10/28 (36) | 11/27 (41) | 21/55 (38) |
| CRb | 1/7(14) | 0/5 | 1/12 (8.3) |
| PR | 0/5 | 0/3 | 0/7 |
| Any HI | 7/28 (25) | 10/27 (37) | 17/55 (31) |
| HI-E | 4/25 (16) | 8/25 (32) | 12/50 (24) |
| HI-P | 4/18 (22) | 3/15 (20) | 7/33 (21) |
| HI-N | 3/10 (30) | 0/6 | 3/16 (19) |
| Marrow CR | 0/7 | 3/5 (60) | 3/12 (25) |
| RBC TIc | |||
| Sustained for 56 days | 5/16 (31) | 6/16 (38) | 11/32 (34) |
| Sustained for 84 days | 2/16 (13) | 5/16 (31) | 7/32 (22) |
| Platelet TId | 0/4 | 0/2 | 0/6 |
Abbreviations: CR=complete remission; HI=hematologic improvement; HI-E=hematologic improvement-erythroid; HI-P=hematologic improvement-platelet; HI-N=hematologic improvement-neutrophil; IWG=International Working Group; PR=partial remission; TI=transfusion independence. IWG 2006 criteria.28
Patients are counted only once for overall response, but may be counted more than once in individual response categories. Marrow CR was not included in overall response. Lower-risk patients with <5% bone marrow blasts at baseline were not evaluable for CR or PR.
Subjects who had a CR are not counted for PR, any HI or marrow CR.
To be evaluated for RBC TI, patients must have been RBC transfusion-dependent at baseline and been on-study at least 56 days. RBC transfusion dependence at baseline was defined as receipt of ⩾4 units of packed RBC within 56 days of the first dose of CC-486. To be evaluated for platelet TI, patients must have been platelet transfusion-dependent at baseline and been on-study at least 56 days. Platelet transfusion dependence at baseline was defined as receipt of ⩾2 platelet transfusions within 56 days of the first dose of CC-486.
To be evaluated for platelet TI, patients must have been platelet transfusion-dependent at baseline and been on-study at least 56 days. Platelet transfusion dependence at baseline was defined as receipt of ⩾2 platelet transfusions within 56 days of the first dose of CC-486.