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. 2018 Jan;24(1):10.18553/jmcp.2018.24.1.29. doi: 10.18553/jmcp.2018.24.1.29
Key Trials Patient Characteristics Treatment Comparator Harms (Treatment Arm)
ASPIRE
Open-label RCT
Phase 3
Carfilzomib (CFZ)
• Median age: 64
• ECOG = 2: 9.5%
• ISS Stage III: 20%
• Previous SCT: 57%
• High risk: 12.6%
• Prior regimens (median): 2
• Prior BOR: 65.8%
• Prior LEN: 19.8%
CFZ+LEN+DEX
(n = 396)
LEN+DEX
(n = 396)
• Discontinued d/t AEs: 15%
• SAEs: 60%
• Tx-related deaths: 2%
• Median f/u: 32.3 m • Median f/u: 31.5 m
• OS HR: 0.79 (95% CI: 0.63-0.99; P = 0.04)
• PFS HR: 0.69 (95% CI: 0.57-0.83)
• Median PFS: 26.3 m
• ORR: 87.1%
• Median PFS: 17.6 m
• ORR: 66.7%, P < 0.001
CASTOR
Open-label RCT
Phase 3
Daratumumab (DAR)
• Median age: 64
• ECOG = 2: NR
• ISS Stage III: 22%
• Previous SCT: 61%
• del(17p): 10%
• Prior regimens (median): 2
• Prior BOR: 66%
• Prior LEN: 76%
DAR+BOR+DEX
(n = 251)
BOR+DEX
(n = 247)
• Discontinued d/t AEs: 7%
• SAEs: 76%
• Tx-related deaths: 5%
• Median f/u: 7.4 m
• Deaths: 11.6% • Deaths: 14.6%
• PFS HR: 0.39 (95% CI: 0.28-0.53; P < 0.001)
• Median PFS: NR
• ORR: 82.9%
• Median PFS: 7.2 m
• ORR: 63.2%
POLLUX
Open-label RCT
Phase 3
Daratumumab (DAR)
• Median age: 65
• ECOG = 2: 5%
• ISS Stage III: 20%
• Previous SCT: 63%
• del(17p): 8%
• Prior regimens (median): 1
• Prior BOR+LEN: 15%
DAR+LEN+DEX
(n = 286)
LEN+DEX
(n = 283)
• Discontinued d/t AEs: 8%
• SAEs: 49%
• Tx-related deaths: 4%
• Median f/u: 13.5 m
• Deaths: 10.5% • Deaths: 15.9%
• PFS HR: 0.37 (95% CI: 0.27-0.52; P < 0.001)
• Median PFS: NR
• ORR: 92.9%
• Median PFS: 18.4 m
• ORR: 76.4%
ELOQUENT-2
Open-label RCT
Phase 3
Elotuzumab (ELO)
• Median age: 66
• ECOG = 2: 9%
• ISS Stage III: 21%
• Previous SCT: 54%
• del(17p): 32%
• Prior regimens (median): 2
• Prior BOR: 70%
• Prior LEN: 6%
ELO+LEN+DEX
(n = 321)
LEN+DEX
(n = 325)
• Discontinued d/t AEs: 13%
• SAEs: 65%
• Tx-related deaths: 2%
• Median f/u: 24.5 m
• OS HR: 0.71 (95% CI: 0.54-0.93)
• PFS HR: 0.70 (95% CI: 0.57-0.85; P < 0.001)
• Median PFS: 19.4 m
• ORR: 79%
• Median PFS: 14.9 m
• ORR: 66%, P < 0.001
TOURMALINE-MM1
Double-blind RCT
Phase 3 (unpublished)
Ixazomib (IX)
• Median age: 66
• ECOG = 2: 6%
• ISS Stage III: 13%
• Previous SCT: 57%
• High risk: 19%
• Prior regimens (median): 2
• Prior BOR: 69%
• Prior LEN: 12%
IX+LEN+DEX
(n = 360)
Placebo+LEN+DEX
(n = 362)
• Discontinued d/t AEs: 13%
• SAEs: 40%
• Tx-related deaths: NR
• Median f/u (PFS): 23 m
• Deaths: 22.5% • Deaths: 24.8%
• PFS HR: 0.74 (95% CI: 0.59-0.94; P = 0.012)
• Median PFS: 20.6 m
• ORR: 78%
• Median PFS: 14.7 m
• ORR: 72%, P < 0.001
PANORAMA-1
Double-blind RCT
Phase 3
Panobinostat (PAN)
• Median age: 63
• ECOG = 2: 5%
• ISS Stage III: 22%
• Previous SCT: 58%
• 1 prior regimen: 51%
• Prior BOR+DEX: 38%
• Prior LEN: 21%
PAN+BOR+DEX
(n = 387)
Placebo+BOR+DEX
(n = 381)
• Discontinued d/t AEs: 36%
• SAEs: 60%
• Tx-related deaths: 3%
• Median f/u: 6.4 m • Median f/u: 5.9 m
• OS HR: 0.87 (95% CI: 0.69-1.10; P = 0.26)
• PFS HR: 0.63 (95% CI: 0.52-0.76; P < 0.0001)
• Median PFS: 11.99 m
• ORR: 60.7%
• Median PFS: 8.08 m
• ORR: 54.6%, P = 0.09

AE = adverse event; BOR = bortezomib; CI = confidence interval; del(17p) = deletion in 17 p region of tumor protein 53 gene; DEX = dexamethasone; d/t = due to; ECOG=Eastern Cooperative Oncology Group; f/u = follow-up; HR = hazard ratio; ISS = International Staging System; LEN = lenalidomide; NR = not reported; ORR = objective response rate; OS = survival; PFS = progression-free survival; RCT = randomized controlled trial; SAE = serious adverse event; SCT = stem cell transplant; Tx = treatment.