Table 1.
Studies comparing high-dose melphalan and autologous stem cell transplantation with chemotherapy for multiple myeloma.
| Reference | N | Treatment | Response | EFS* | OS* | Notes |
|---|---|---|---|---|---|---|
| Attal et al.39 | 200 | VMCP/BVAP vs. MEL 140 + TBI (8Gy) IFN-a maintenance until relapse |
ASCT vs. chemo CR 22% vs. 5% VGPR 16% vs. 9% P<.001 |
ASCT vs. chemo 27 vs. 18 mo P=0.01 |
ASCT vs. chemo NR vs. 37.4 mo P=0.03 |
|
| Child et al.40 | 407 | BCAM vs. VMCP → Cy+G- CSF mobilization → MEL 200 IFN maintenance in both groups N=8 received TBI+MEL140 |
ASCT vs. chemo CR-rate 44% vs. 8% P<0.001 PR-rate 42% vs. 40% P=0.72 |
PFS ASCT vs. chemo 31.6 mo vs. 19.6 mo P<0.001 |
ASCT vs. chemo 54.1 mo vs. 42.3 mo P=0.04 |
17% of patients in chemotherapy arm crossed over to ASCT arm, usually after disease progression, hence the actually survival benefit may be higher. |
| Fermand et al.41 | 179 | VAMP vs. LomustineVP- 16+Cy+Mel140+T BI (1,200 cGy) & ASCT IFN offered to both groups in remission |
Early ASCT vs. chemo CR 18% vs. 5% |
Early ASCT vs. chemo 39 mo vs. 13 mo |
Early ASCT vs. chemo 64.6 mo vs. 64 mo P=0.92 |
Late ASCT offered to chemotherapy group at progression or resistant disease after 6 cycles of VMCP. Distribution of periods spent without chemotherapy was longer in early ASCT group. |
| Blade et al.42 |
186 | VBMCP/VBAD vs. MEL 200 or MEL 140+TBI (12Gy) Responding patients in both groups received maintenance with IFN + Dex until relapse |
ASCT vs. chemo CR 30% vs. 11% P=0.002 |
PFS ASCT vs. chemo 42 mo vs. 33 mo P=0.57 |
ASCT vs. chemo 61 mo vs. 66 mo P=0.89 |
Only patients with chemo-sensitive disease were enrolled. |
| Fermand et al.43 | 190 | VMCP vs. CHOP+G-CSF for collection → VAMP → MEL200 or MEL140 + Bu IFN-a proposed to both arms |
ASCT vs. chemo CR +MRD 36% vs. 19.8% |
ASCT vs. chemo 25.3 mo vs. 18.7 mo P=0.07 |
ASCT vs. chemo 47.8 mo vs. 47.6 mo P=0.91 |
TwiSTT ASCT vs. chemo 25.1 mo vs. 16.6 mo P=0.033 |
| Barlogie et al.44 | 516 | MEL140+TBI (12Gy) vs. VBMCP Responding patients randomized to IFN × 4 yrs vs. Obs |
ASCT vs. chemo CR 11% vs. 11% |
ASCT vs. chemo 17% vs. 14% |
ASCT vs. chemo 38% vs. 38% |
Legend:
V, vincristine; M, melphalan; C & Cy, cyclophosphamide; P, prednisone; B, carmustine (BCNU); A, adriamycin (H, hydroxydaunorubicin); IFN, interferon; MEL, melphalan; G-CSF, granulocyte colony-stimulating factor; TBI, total-body irradiation; IFN-a, interferon-alpha; Dex, dexamethasone; O, oncovin (vincristine); Bu, busulfan
ASCT, autologous stem cell transplantation; Chemo, chemotherapy; CR, complete remission; PFS, progression-free survival; MRD, minimal residual disease; TwiSTT; the period of time without symptoms, treatment, and treatment toxicity; EFS, event-free survival; OS, overall survival
*
Median values