Table 2 Summary of phase III clinical trials evaluating the efficacy of ASCT in the era of novel agent induction therapy

From: Utilization of hematopoietic stem cell transplantation for the treatment of multiple myeloma: a Mayo Stratification of Myeloma and Risk-Adapted Therapy (mSMART) consensus statement

Clinical trial No. Induction Randomized arms ORR EFS/PFS OS
Palumbo et al. [20]. NDMM pts <65 y/o 402 Rd × 4 cycles MPR × 6 cycles →No maint CR (post consolidation): 18% Median PFS: 22 mos 5-year OS: 59%
MPR × 6 cycles →R-maint   Median PFS: 34 mos 5-year OS: 70%
Mel200 mg/m2 × 2 cycles → No maint CR (post consolidation): 23% Median PFS: 37 mos 5-year OS: 67%
Mel200 mg/m2 × 2 cycles → R-maint   Median PFS: 55 mos 5-year OS: 78%
Gay et al. [21]. NDMM pts ≤65 y/o 389 Rd × 4 cycles CRd × 6 cycles → R-maint CR: 27% Median PFS: 28 mos 4-year OS: 76%
CRd × 6 cycles → Rp-maint CR: 23% Median PFS: 24 mos 4-year OS: 68%
Mel200 mg/m2 × 2 cycles → R-maint CR: 33% Median PFS: 32 mos 4-year OS: 75%
Mel200 mg/m2 × 2 cycles → Rp-maint CR: 37% Median PFS: 38 mos 4-year OS: 77%
Cavo et al. [23]. NDMM pts ≤65 y/o 1503 VCd × 3–4 cycles VMP→+/− VRd → R-maint ≥VGPR: 75% 3-year PFS: 57% Median OS: NR
Mel200 mg/m2 × 1 or 2 →+/− VRd →R-maint ≥VGPR: 84% 3-year PFS: 64% Median OS: NR
Attal et al. [25]. NDMM pts ≤65 y/o 700 VRd × 3 cycles VRd × 5 cycles →R-maint CR: 48% MRD (−): 65% Median PFS: 36 mos 4-year OS: 82%
Mel200 mg/m2 →VRd × 2 cycles →R-maint CR: 59% MRD (−): 79% Median PFS: 50 mos 4-year OS: 81%
  1. CRd cyclophosphamide, lenalidomide, and dexamethasone, R-maint lenalidomide maintenance, Rp-maint lenalidomide and prednisone maintenance, VRd bortezomib, lenalidomide, and dexamethasone, MPR melphalan, prednisone and lenalidomide, VCd bortezomib, cyclophosphamide and dexamethasone, ORR overall response rate, EFS event-free survival, PFS progression-free survival, OS overall survival, CR complete remission, MRD (−) minimal residual disease negativity