Table 5.
Efficacy
| Hematologic Improvement Response Rate (IWG 2000) | ||||
| Baseline Cell-Line Cytopenia | n (%) | |||
| Trilineage | 4/16 (25) | |||
| Bilineage | 1/13 (8) | |||
| Unilineage | 7/14 (50) | |||
| Hematologic Responses by Cell-Line Cytopenia (IWG 2000) | ||||
| Number of Patients |
Major Response n (%) |
Minor Response n (%) |
Total Response n (%) |
|
| HI-E | 38 | 5 (13) | 4 (11) | 9 (24) |
| HI-N | 26 | 9 (35) | 2 (8) | 11 (42) |
| HI-P | 24 | 4 (17) | 8 (33) | 12 (50) |
| Hematologic Improvement (HI) Response by Patient Demographics & MDS Disease Characteristics | ||||
|
HI-E Response n (%) |
HI-N Response n (%) |
HI-P Response n (%) |
||
| Number of Patients (%) | 9/38 (24) | 11/26 (42) | 12/24 (50) | |
| Gender | ||||
| Female | 2/13 (15) | 3/8 (38) | 3/9 (33) | |
| Male | 7/25 (28) | 8/18 (44) | 9/15 (60) | |
| Baseline FAB | ||||
| RA | 6/20 (30) | 5/12 (42) | 7/11 (64) | |
| RARS | 1/6 (17) | 2/4 (50) | 1/1 (100) | |
| RAEB | 2/8 (25) | 4/7 (57) | 3/7 (43) | |
| RAEB-t | 0/3 (0) | 0/3 (0) | 0/3 (0) | |
| CMML | 0/1 (0) | 0 | 0/1 (0) | |
| Unknown | 0 | 0 | 1/1 (100) | |
| Baseline Karyotype | ||||
| Normal | 3/20 (15) | 6/16 (38) | 5/14 (36) | |
| Abnormal | 6/18 (33) | 5/10 (50) | 7/10 (70) | |
| Transfusion Requirements and Clinical Symptoms Improvement (IWG 2000) | ||||
| Phase 1: Comparison of Decreased Transfusion Requirements and Clinical Symptoms Improvement | ||||
|
All Dose Levels Combined n (%) |
||||
| 50% Decreased Transfusion Requirements | 3/15 (20) | |||
| Clinical Symptoms Improvement | 7/21 (33) | |||
| Phase 2a: Comparison of Decreased Transfusion Requirements and Clinical Symptoms Improvement by Dose Schedule | ||||
|
Dose Schedule 1 Days 1–5 of 21-Day Treatment Cycle n (%) |
Dose Schedule 2 Days 1–3 of 21-Day Treatment Cycle n (%) |
Total n (%) |
||
| 50% Decreased Transfusion Requirements | 1/5 (20) | 1/11 (9) | 2/16 (13) | |
| Clinical Symptoms Improvement | 9/10 (90) | 3/12 (25) | 12/22 (55) | |
Abbreviations: RA, refractory anemia; RARS, refractory anemia with ringed sideroblasts; RAEB, refractory anemia with excess blasts; RAEB-t, refractory anemia with excess blasts in transformation; CMML, chronic myelomonocytic leukemia; IWG, International Working Group.