Table 1.

Trials of FDA-approved novel agents for the treatment of relapsed or refractory multiple myeloma

Study namePhaseNMedian age, yNumber of prior regimensTreatmentResponse ratesSurvivalReference
Bortezomib (BTZ)
APEXIII33362 (10th, 90th percentiles, 48, 74)Median, 2 (range, 1–4+)BTZORR: 38%; CR: 6%; PR: 32%1-y OS: 80%(30)
MMY-3021III7464.5 (range, 38–86)1 prior: 65%; >1 prior: 35%i.v. BTZORR: 42%; CR: 8%; PR: 34%PFS: 8.0 mo(25)
1-y OS: 76.7%
MMY-3021III14864.5 (range, 42–88)1 prior: 62%; >1 prior: 38%s.c. BTZORR: 42%; CR: 6%; PR: 36%PFS: 10.2 mo(25)
1-y OS: 72.6%
Lenalidomide (Len)
MM-009III17764 (range, 36–86)1 prior: 38.4%; >1 prior: 61.6%Len–DexORR: 61.0%; CR: 14.1%; nCR: 10.2%; PR: 36.7%OS: 29.6 mo(31)
MM-010III17663 (range, 33–84)1 prior: 31.8%; >1 prior: 68.2%Len–DexORR: 60.2%; CR: 15.9%; nCR: 8.5%; PR: 35.8%OS: 36+ mo(32)
NCT00378209II6465 (range, 32–83)Median, 2 (range, 1–3)Len–BTZ–DexORR: 64%; CR: 11%; nCR: 14%; VGPR: 3%; PR: 36%PFS: 9.5 mo(33)
OS: 30 mo
Carfilzomib (CFZ)
PX-171-003-A1II26663 (range, 37–87)Median, 5 (range, 1–20)CFZ, 20 mg/m2 then 27 mg/m2ORR: 23.7%; CR: 0.4%; VGPR: 5.1%; PR: 18.3%PFS: 3.7 mo(34, 35)
OS: 15.6 mo
NCT01351623II4463 (range, 45–86)Median, 5 (range, 1–11)CFZ, 20 mg/m2 then 56 mg/m2 with slower (30-min) infusionORR: 55%; CR: 2%; VGPR: 21%; PR: 31%PFS: 4.1 mo(35)
OS: 20.3 mo
ASPIREIII39664 (range, 38–87)Median, 2 (range, 1–3)CFZ–Len–DexORR: 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-002II10861 (range, 37–88)>2 prior: 95%PomORR: 18%; CR: 2%; PR: 16%PFS: 2.7 mo(37)
OS: 13.6 mo
MM-002II11364 (range, 34–88)>2 prior: 95%Pom–DexORR: 33%; CR: 3%; PR: 30%PFS: 4.2 mo
OS: 16.5 mo(37)
MM-003III30264 (range, 35–84)Median, 5 (range, 2–14)Pom–DexORR: 31%; sCR or CR: 1%; VGPR: 5%; PR: 26%PFS: 4.0 mo(38)
OS: 12.7 mo
Panobinostat (PAN)
PANORAMA 2II5561 (range, 41–88)Median, 4 (range, 2–11)PAN–BTZ–DexORR: 34.5%; nCR: 1.8%; PR: 32.7%PFS: 5.4 mo(17)
PANORAMA 1III38763 (range, 56–69)1 prior: 51%; 2–3 prior: 49%PAN–BTZ–DexORR: 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.