Table 2.
Efficacy outcomes and follow-up status
| All patients | Phase 2 dose | |
|---|---|---|
| (N = 48) | (N = 45) | |
| Overall response rate | 77% (95%CI: 63, 88) | 78% (95%CI: 63, 89) |
| ≥VGPR Response Rate | 35% (95%CI: 22, 50) | 38% (95%CI: 24, 53) |
| sCR | 3 | 3 |
| CR | 0 | 0 |
| VGPR | 14 | 14 |
| PR | 20 | 18 |
| MR | 9 | 8 |
| SD | 2 | 2 |
| Overall Response Rate by FISH | ||
| High risk | 88% (95%CI: 47, 100) | 88% (95%CI: 47, 100) |
| Standard risk | 75% (95%CI: 59, 87) | 76% (95%CI: 59, 88) |
| Median overall survivala | NA | NA |
| 12 Months | 100% | 100% |
| Median progression-free survivala | NA (95%CI: 31.3, NA) | NA (95%CI: 31.3, NA) |
| Median duration of responsea | NA (95%CI: 30.3, NA) | NA (95%CI: 30.3, NA) |
| Median time to response | 1.9 mos (range: 0.9–4.8) | 1.9 mos (range: 0.9–4.8) |
| Patients with progression | 11 (22.9%) | 11 (24.4%) |
| Patients alive | 46 (95.8%) | 43 (95.6%) |
| Median follow-up (alive patients) | 25.6 mos (range: 12.3–44.6) |
25.1 mos (range: 12.3–40.7) |
| Last cycle administered | 7 (range: 3–46) | 7 (range: 3–44) |
| On treatment | 8 | 7 |
| Reason For ending treatment | ||
| Refused further treatment | 3 (7.5%) | 2 (5.3%) |
| Adverse event | 3 (7.5%) | 3 (7.9%) |
| Disease progression | 10 (25.0%) | 10 (26.3%) |
| Alternate treatment | 19 (47.5%) | 18 (47.4%) |
| Lack of response | 5 (10.4%) | 5 (11.1%) |
CI confidence interval, mo month, NA not attained
a Kaplan–Meier