Efficacy
| . | Dose level . | |||
|---|---|---|---|---|
| 200-1400 mg/d . | 2000-4000 mg/d . | 5000-6000 mg/d . | All doses combined . | |
| HI response rates (efficacy evaluable population) | ||||
| IWG 2000 | ||||
| HI-E | 0/5 (0) | 3/11 (27) | 3/13 (23) | 6/29 (21) |
| HI-N | 0/5 (0) | 0/7 (0) | 4/7 (57) | 4/19 (21) |
| HI-P | 2/7 (29) | 2/7 (29) | 3/8 (38) | 7/21 (33) |
| Unilineage | 1/6 (17) | 1/5 (20) | 1/3 (33) | 3/14 (21) |
| Bilineage | 0/5 (0) | 0/9 (0) | 3/11 (27) | 3/25 (12) |
| Comparison of HI-E response rates | ||||
| IWG 2000 | 0/5 (0) | 3/11 (27) | 3/13 (23) | 6/29 (21) |
| IWG 2006 | 3/5 (60) | 5/11 (45) | 6/13 (45) | 14/29 (48) |
| Comparison of RBC transfusion independence and reduction | ||||
| IWG 2006 | ||||
| RBC transfusion independence | 3/5 (60) | 2/10 (20) | 3/8 (38) | 8/23 (35) |
| RBC transfusions reduced by 4 units/8 weeks | 3/5 (60) | 5/10 (50) | 6/8 (75) | 14/23 (61) |
| IWG 2000 | ||||
| RBC transfusions reduced by 50% | 2/5 (40) | 2/10 (20) | 5/8 (63) | 12/23 (52) |
| . | Dose level . | |||
|---|---|---|---|---|
| 200-1400 mg/d . | 2000-4000 mg/d . | 5000-6000 mg/d . | All doses combined . | |
| HI response rates (efficacy evaluable population) | ||||
| IWG 2000 | ||||
| HI-E | 0/5 (0) | 3/11 (27) | 3/13 (23) | 6/29 (21) |
| HI-N | 0/5 (0) | 0/7 (0) | 4/7 (57) | 4/19 (21) |
| HI-P | 2/7 (29) | 2/7 (29) | 3/8 (38) | 7/21 (33) |
| Unilineage | 1/6 (17) | 1/5 (20) | 1/3 (33) | 3/14 (21) |
| Bilineage | 0/5 (0) | 0/9 (0) | 3/11 (27) | 3/25 (12) |
| Comparison of HI-E response rates | ||||
| IWG 2000 | 0/5 (0) | 3/11 (27) | 3/13 (23) | 6/29 (21) |
| IWG 2006 | 3/5 (60) | 5/11 (45) | 6/13 (45) | 14/29 (48) |
| Comparison of RBC transfusion independence and reduction | ||||
| IWG 2006 | ||||
| RBC transfusion independence | 3/5 (60) | 2/10 (20) | 3/8 (38) | 8/23 (35) |
| RBC transfusions reduced by 4 units/8 weeks | 3/5 (60) | 5/10 (50) | 6/8 (75) | 14/23 (61) |
| IWG 2000 | ||||
| RBC transfusions reduced by 50% | 2/5 (40) | 2/10 (20) | 5/8 (63) | 12/23 (52) |
HI indicates hematologic improvement; HI-E, HI-erythroid; HI-N, HI-neutrophils; HI-P, HI-platelets; and RBC, red blood cells.