Table 1.
Author | Phase Primary end point Regimen | Patient population with RRMM | Toxicities | Results |
---|---|---|---|---|
Stewart et al23 | III Randomized PFS KRd vs Rd |
396 KRd patients vs 396 Rd patients | AE grade 3 or higher in 83.7% KRd and 80.7% Rd | ORR 87.1% KRd vs 66.7% Rd Median PFS 26.3 months KRd vs 17.6 months Rd 24-month OS 73.3% KRd vs 65% Rd |
Dimopoulos et al25 | III Randomized PFS Kd vs Vd |
464 Kd patients vs 465 Vd patients | Higher rate of grade 3–4 anemia, hypertension, thrombocytopenia in Kd | ORR 77% Kd vs 63% Vd Median PFS 18.7 months Kd vs 9.4 months Vd |
Wang et al22 | II ORR, PFS, DOR KRd |
54 KRd patients | Lymphopenia, neutropenia, thrombocytopenia and anemia | ORR 76.9% KRd Median DOR 22.1 months KRd Median PFS 15.4 months KRd |
Fiala et al35 | II Efficacy, ORR KLd |
23 KLd patients | Infections, hypertension, MI, thrombocytopenia, anemia | ORR 83% KLd Median EFS 7.4 months |
Berdeja et al32 | I/II MTD, ORR FKd |
33 FKd patients | Dyspnea, neuropathy, cardiac toxicities | ORR 82% FKd Median PFS 9.7 months Median OS 24.7 months |
Martin et al72 | Ib MTD IK |
11 IK patients | Anemia, neutropenia, pneumonia | ORR 80% IK |
Vesole et al30 | I MTD, safety QUAD |
17 QUAD patients | Neutropenia, anemia, thrombocytopenia | MTD not reached No DLT observed ORR 53% QUAD Median PFS 12 months QUAD |
Jakubowiak et al37,38 | I MTD, safety, efficacy SKd |
18 SKd patients | Thrombocytopenia, neutropenia, anemia, gastrointestinal disorders | MTD not reached 1 DLT of cardiac amyloidosis 75%≥ MR SKd |
Shah et al26 | I MTD, safety KPd |
32 KPd patients | Anemia, dypsnea | ORR 50% KPd Median PFS 7.2 months KPd |
Abbreviations: AE, adverse event; DLT, dose-limiting toxicities; DOR, duration of response; EFS, event free survival; FKd, panobinostat carfilzomib dexamethasone; IK, isatuximab carfilzomib; Kd, carfilzomib dexamethasone; KLd, carfilzomib pegylated liposomal doxorubicin dexamethasone; KPd, carfilzomib pomalidomide dexamethasone; KRd, carfilzomib lenalidomide dexamethasone; MI, myocardial infarction; MR, minimal response; MTD, maximum tolerated dose; ORR, overall response rate; OS, overall survival; PFS, progression-free survival; QUAD, carfilzomib lenalidomide vorinostat dexamethasone; Rd, lenalidomide dexamethasone; RRMM, relapsed and/or refractory multiple myeloma; SKd, selinexor carfilzomib dexamethasone; Vd, bortezomib dexamethasone.