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. 2013 Aug 9;28(2):258–268. doi: 10.1038/leu.2013.220

Table 1. Prognostic impact of response level in patients with newly diagnosed multiple myeloma: transplant studies.

ReferenceResponse criteriaStudy 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.

a

Conversion to months for studies reporting other time increments.

b

From diagnosis P=0.0004.

c

From SCT.