Efficacy outcomes in the efficacy analysis set, according to IWG 2006 response criteria
| Efficacy outcomes . | MDS substudy efficacy analysis set (N = 18) . | 95% CI . |
|---|---|---|
| Primary end point, n (%) | ||
| CR + PR | 7 (38.9) | (17.3-64.2) |
| Secondary end points | ||
| Time to CR + PR, median mo (min, max) | 1.87 (1.0, 5.6) | (58.6-96.4) |
| Duration of CR + PR,∗ median mo (min, max) | NR (1.9, NR) | |
| Probability of patients maintaining CR + PR,∗ n (%) | ||
| At 3 mo | 85.7 | |
| At 24 mo | 68.6 | |
| At 60 mo | 68.6 | |
| Best overall response | ||
| ORR† | 15 (83.3) | (58.6-96.4) |
| CR | 7 (38.9) | (17.3-64.3) |
| PR | 0 | (0.0-18.5) |
| mCR | 8 (44.4) | (21.5-96.2) |
| SD | 2 (11.1) | (1.4-34.7) |
| PDis | 1 (5.6) | (0.1-27.3) |
| Time to OR,† median months (min, max) | 0.99 (0.9, 4.6) | |
| HI in non-CR/PR patients, n (%) | ||
| Erythrocyte lineage | 2 (18.2)‡ | |
| Platelet lineage | 2 (25.0)§ | |
| Neutrophil lineage | 4 (57.1)‖ | |
| Any HI lineage | 4 (36.4)¶ | |
| OS,∗ median months, [95% CI] (min, max) | 35.7 [13.1, NE] (3.7#, 88.7#) | |
| OS rate,∗ (%) | ||
| 1 y | 86.9 | |
| 3 y | 46.3 | |
| 5 y | 46.3 | |
| 7 y | 46.3 |
| Efficacy outcomes . | MDS substudy efficacy analysis set (N = 18) . | 95% CI . |
|---|---|---|
| Primary end point, n (%) | ||
| CR + PR | 7 (38.9) | (17.3-64.2) |
| Secondary end points | ||
| Time to CR + PR, median mo (min, max) | 1.87 (1.0, 5.6) | (58.6-96.4) |
| Duration of CR + PR,∗ median mo (min, max) | NR (1.9, NR) | |
| Probability of patients maintaining CR + PR,∗ n (%) | ||
| At 3 mo | 85.7 | |
| At 24 mo | 68.6 | |
| At 60 mo | 68.6 | |
| Best overall response | ||
| ORR† | 15 (83.3) | (58.6-96.4) |
| CR | 7 (38.9) | (17.3-64.3) |
| PR | 0 | (0.0-18.5) |
| mCR | 8 (44.4) | (21.5-96.2) |
| SD | 2 (11.1) | (1.4-34.7) |
| PDis | 1 (5.6) | (0.1-27.3) |
| Time to OR,† median months (min, max) | 0.99 (0.9, 4.6) | |
| HI in non-CR/PR patients, n (%) | ||
| Erythrocyte lineage | 2 (18.2)‡ | |
| Platelet lineage | 2 (25.0)§ | |
| Neutrophil lineage | 4 (57.1)‖ | |
| Any HI lineage | 4 (36.4)¶ | |
| OS,∗ median months, [95% CI] (min, max) | 35.7 [13.1, NE] (3.7#, 88.7#) | |
| OS rate,∗ (%) | ||
| 1 y | 86.9 | |
| 3 y | 46.3 | |
| 5 y | 46.3 | |
| 7 y | 46.3 |
NR, not estimable; OR, objective response; ORR, objective response rate; PDis, progressive disease; SD, stable disease.
Kaplan-Meier estimate of duration of CR + PR.
OR comprised CR, PR, or mCR.
% based on number of patients with mCR and pretreatment hemoglobin <11 g/dL (N1 = 11).
% based on number of patients with mCR and pretreatment platelet count <100 × 109/L (N2 = 8).
% based on patients with mCR and pretreatment absolute neutrophil count <1.0 × 109/L (N3 = 7).
% based on patients with mCR and who satisfy the pretreatment criteria for HI-erythrocyte, HI-platelet, or HI-neutrophil (N4 = 11).
Censored observation.