Table 1. Prognostic impact of response level in patients with newly diagnosed multiple myeloma: transplant studies.
Reference Response criteria Study type | Treatment | Response | n | PFS/TTP/EFS (median, moa/rate, %) | OS (median, moa/rate, %) | |||||
---|---|---|---|---|---|---|---|---|---|---|
mEFS | 5 yr | P-value | mOS | 5 yr | P-value | |||||
Lahuerta et al.26 mEBMT Prospective | GEM2000→HD-SCT→IFN+prednisone (maint) | Post induction | CR nCR PR SD PD | 101 96 346 63 26 | 56 47 43 41 20 | 53 49 34 33 12 | 0.05 CR versus PR | NR NR NR NR 25 | 78 65 63 56 25 | 0.02 CR versus SD |
Post transplant | CR nCR PR SD PD | 278 124 175 30 25 | 61 40 34 44 13 | 52 27 23 27 4 | <0.01 CR versus nCR, PR and SD | NR NR 61 NR 15 | 74 63 50 57 24 | ⩽0.01 CR versus nCR, PR, and SD; 0.04 nCR versus PR | ||
PFS | 4 yr | P-value | OS | 4 yr | P-value | |||||
Dytfeld et al.114 mEBMT Phase 2 | VDD±SCT VDD+SCT (subgroup) | Post induction Post induction | ⩾VGPR <VGPR ⩾VGPR <VGPR | 25 15 16 14 | — — | 67.2 46.3 77.8 53.8 | 0.08 0.036 | — — | 86.5 58.2 92.9 64.3 | 0.04 0.027 |
mPFS | P-value | mOS | P-value | |||||||
Gertz et al.115 Retrospective | T or R regimen→SCT | Post induction | PR <PR | 232 54 | 22.1 13.1 | <0.001 | 73.5 30.4 | — | <0.001 | |
Moreau et al.27 mEBMT Ph 3 post hoc | VD or VAD ±DCEP→SCT | Post induction | ⩾VGPR <VGPR | 125 357 | 41.2 29.0 | <0.0001 | — | — | — | |
mEFS | 5 yr | P-value | OS | 5 yr | P-value | |||||
Harousseau et al.28 EBMT Pooled analysis (2 prospective studies) | VAD→double SCT→no treatment, Pam or Pam+T | Post transplant | ⩾VGPR <VGPR | 445 288 | 42 32 | 34 26 | 0.005 | — | 74 61 | 0.0017 |
mOSb | 4 yrc | P-value | ||||||||
Kapoor et al.116 IMWG Retrospective | SCT | Post transplant | sCR CR nCR | 115 28 46 | — | — | — | NR 59 53 | 86 60 — | 0.007 |
mOS | 4 yr | P-value | ||||||||
Barlogie et al.57 EBMT Retrospective | TT2 TT1 Tandem SCT | 36-mo landmark 36-mo landmark 36-mo landmark | Sust-CR Non-CR Lost-CR Sust-CR Non-CR Lost-CR Sust-CR Non-CR Lost-CR | 256 211 39 44 88 33 197 252 60 | — | — | — | NR 67.2 19.2 127.2 72 34.8 76.8 46.8 21.6 | 83 59 19 — — — — — — | <0.0001 Sust-CR versus non-CR and <0.0001 versus lost-CR; <0.0001 non-CR versus lost-CR 0.06 Sust-CR versus non-CR and 0.001 versus lost-CR; 0.04 <0.0001 Sust-CR versus non- CR/lost-CR; 0.0002 non- CR versus lost-CR |
mPFS | P-value | mOS | P-value | |||||||
Alegre et al.117 Observational | HD-ASCT±IFN-α maint | Post induction | CR PR SD PD | 56 153 25 25 | 35 28 20 8 | — | 0.001 CR/PR versus SD/PD | 39 36 24 12 | — | 0.001 CR/PR versus SD/PD |
EFS | 5 yr | P-value | OS | 5 yr | P-value | |||||
Galli et al. 29 mEBMT Observational | TT1 | Post induction Post ASCT1 Post ASCT2 | CR ⩾VGPR PR/SD SD CR <VGPR CR <VGPR | 17 29 81 18 43 53 51 28 | — | 65 54 24 27 32 30 33 34 | 0.025 CR versus <CR 0.006 ⩾VGPR versus PR/SD | — | 63 63 47 56 50 58 59 63 | 0.31 CR versus <CR |
Abbreviations: DCEP, dexamethasone, cyclophosphamide, etoposide, and cisplatin; GEM2000, induction therapy with six alternating 5-week cycles of vincristine, carmustine, cyclophosphamide, melphalan and prednisone/vincristine, carmustine, doxorubicin and dexamethasone→HD-SCT; HD-ASCT, high-dose therapy with autologous stem cell transplant; IFN, interferon; IMWG, International Myeloma Working Group; maint, maintenance therapy; mEBMT, modified European Group for Blood and Marrow Transplantation; mEFS, median event-free survival; mOS, median overall survival; mPFS, median progression-free survival; nCR, near complete response; NR, not reached; Pam, pamidronate; PD, progressive disease; R, lenalidomide; sCR, stringent complete response; SCT, stem cell transplant; SD, stable disease; Sust, sustained; T, thalidomide; TT; total therapy; VAD, vincristine, doxorubicin, dexamethasone; VD, bortezomib, dexamethasone; VDD, bortezomib, pegylated liposomal doxorubicin, dexamethasone; VGPR, very good partial response; yr, years.
Conversion to months for studies reporting other time increments.
From diagnosis P=0.0004.
From SCT.