Trials of FDA-approved novel agents for the treatment of relapsed or refractory multiple myeloma
| Study name | Phase | N | Median age, y | Number of prior regimens | Treatment | Response rates | Survival | Reference |
|---|---|---|---|---|---|---|---|---|
| Bortezomib (BTZ) | ||||||||
| APEX | III | 333 | 62 (10th, 90th percentiles, 48, 74) | Median, 2 (range, 1–4+) | BTZ | ORR: 38%; CR: 6%; PR: 32% | 1-y OS: 80% | (30) |
| MMY-3021 | III | 74 | 64.5 (range, 38–86) | 1 prior: 65%; >1 prior: 35% | i.v. BTZ | ORR: 42%; CR: 8%; PR: 34% | PFS: 8.0 mo | (25) |
| 1-y OS: 76.7% | ||||||||
| MMY-3021 | III | 148 | 64.5 (range, 42–88) | 1 prior: 62%; >1 prior: 38% | s.c. BTZ | ORR: 42%; CR: 6%; PR: 36% | PFS: 10.2 mo | (25) |
| 1-y OS: 72.6% | ||||||||
| Lenalidomide (Len) | ||||||||
| MM-009 | III | 177 | 64 (range, 36–86) | 1 prior: 38.4%; >1 prior: 61.6% | Len–Dex | ORR: 61.0%; CR: 14.1%; nCR: 10.2%; PR: 36.7% | OS: 29.6 mo | (31) |
| MM-010 | III | 176 | 63 (range, 33–84) | 1 prior: 31.8%; >1 prior: 68.2% | Len–Dex | ORR: 60.2%; CR: 15.9%; nCR: 8.5%; PR: 35.8% | OS: 36+ mo | (32) |
| NCT00378209 | II | 64 | 65 (range, 32–83) | Median, 2 (range, 1–3) | Len–BTZ–Dex | ORR: 64%; CR: 11%; nCR: 14%; VGPR: 3%; PR: 36% | PFS: 9.5 mo | (33) |
| OS: 30 mo | ||||||||
| Carfilzomib (CFZ) | ||||||||
| PX-171-003-A1 | II | 266 | 63 (range, 37–87) | Median, 5 (range, 1–20) | CFZ, 20 mg/m2 then 27 mg/m2 | ORR: 23.7%; CR: 0.4%; VGPR: 5.1%; PR: 18.3% | PFS: 3.7 mo | (34, 35) |
| OS: 15.6 mo | ||||||||
| NCT01351623 | II | 44 | 63 (range, 45–86) | Median, 5 (range, 1–11) | CFZ, 20 mg/m2 then 56 mg/m2 with slower (30-min) infusion | ORR: 55%; CR: 2%; VGPR: 21%; PR: 31% | PFS: 4.1 mo | (35) |
| OS: 20.3 mo | ||||||||
| ASPIRE | III | 396 | 64 (range, 38–87) | Median, 2 (range, 1–3) | CFZ–Len–Dex | ORR: 87.1%; sCR: 14.1%; CR: 17.7%; VGPR: 38.1%; PR: 17.2% | PFS: 26.3 mo | (36) |
| 2-y OS: 73.3% | ||||||||
| Pomalidomide (Pom) | ||||||||
| MM-002 | II | 108 | 61 (range, 37–88) | >2 prior: 95% | Pom | ORR: 18%; CR: 2%; PR: 16% | PFS: 2.7 mo | (37) |
| OS: 13.6 mo | ||||||||
| MM-002 | II | 113 | 64 (range, 34–88) | >2 prior: 95% | Pom–Dex | ORR: 33%; CR: 3%; PR: 30% | PFS: 4.2 mo | |
| OS: 16.5 mo | (37) | |||||||
| MM-003 | III | 302 | 64 (range, 35–84) | Median, 5 (range, 2–14) | Pom–Dex | ORR: 31%; sCR or CR: 1%; VGPR: 5%; PR: 26% | PFS: 4.0 mo | (38) |
| OS: 12.7 mo | ||||||||
| Panobinostat (PAN) | ||||||||
| PANORAMA 2 | II | 55 | 61 (range, 41–88) | Median, 4 (range, 2–11) | PAN–BTZ–Dex | ORR: 34.5%; nCR: 1.8%; PR: 32.7% | PFS: 5.4 mo | (17) |
| PANORAMA 1 | III | 387 | 63 (range, 56–69) | 1 prior: 51%; 2–3 prior: 49% | PAN–BTZ–Dex | ORR: 60.7%; CR: 11%; nCR: 17%; PR: 33% | PFS: 11.99 mo | (19) |
| OS: 33.64 mo | ||||||||
Abbreviations: APEX, Assessment of Proteasome Inhibition for Extending Remissions; CR, complete response; Dex, dexamethasone; i.v., intravenous; nCR, near complete response; NR, not reported; PR, partial response; s.c., subcutaneous; sCR, stringent complete response; VGPR, very good partial response.