Select clinical trials in SMM
| Type of therapy . | Clinical trial design and outcome . | N of patients . | References . |
|---|---|---|---|
| Melphalan and prednisone (MP) | Retrospective cohort study of vincristine, adriamycin, and dexamethasone (VAD) vs MP. Because the treatment of MM remains palliative, chemotherapy should be withheld until symptoms | 23 SMM, 10 IMM | 60 |
| Initial vs delayed MP. Randomized-controlled trial. Similar response rate, response duration, and TTP of 12 months | 50 SMM and IMM (25/25) | 59 | |
| Initial vs delayed MP. TTP of about 12 months. No difference in OS (64 vs 71 months) | 145 DSSI | 61, 62 | |
| Observational study of delayed therapy: 54 DSS I. 2-year PFS 75%. Tumor specific OS 80% at 60 months | 54 DSSI | 71 | |
| Pamidronate or zolendronate | Single-arm, phase 2 trial; pamidronate vs observation. 5-year PFS 53% both arms. Skeletal-related events (SRE) 74% vs 39%, P = .009. Median OS 46 and 48 months | 177 SMM | 72,-74 |
| Open-label randomized controlled trial; zolendronate vs observation × 1 year. TTP not significant. SRE 55 vs 78%, P = .04. Zoledronate (ZLD) for 1 year decreased risk of skeletal-related disease, but TTP was similar (P = .83). OS no difference | 163 SMM | 63 | |
| Thalidomide | Single-arm, phase 2 trial phase 2 of thalidomide pamidronate. 4-year event-free survival (EFS) 60%. 4-year OS 91%. Median TTP 7 years; partial response (PR) identifies subset requiring earlier salvage therapy for symptomatic disease | 76 SMM | 64 |
| Single-arm pilot study of thalidomide. Median 35 months. OS 86 months. OS from treatment 49 months. Minimal response (MR) or better in 11/16. Microvessel density did not predict response | 19 SMM and I0 IMM | 66 | |
| Phase 2 of thalidomide. Patients were treated with thalidomide 100 to 200 mg. The response rate was 36% | 28 high risk SMM | 65 | |
| Phase 3 of thalidomide/ZLD vs ZLD | 68 SMM (35 to Thal/ZLD and 33 ZLD alone) | 67 | |
| Thalidomide +ZA vs ZA. 29 vs 14 months. 6-year OS >70% | 68 SMM | 67 | |
| IL-1 antagonist | Anakinra (IL-1 receptor antagonist). IL-1 antagonist +/− dexamethasone. Median PFS was 37.5 months. MR (n = 3), PR (n = 5). 8 patients stable on drug for 4 years | 47 SMM and IMM | 75 |
| Curcumin | Randomized, double-blind placebo-controlled crossover study. Administering 8 g dose of curcumin. Curcumin therapy decreased the free light-chain ratio, reduced the difference between clonal and monoclonal light-chain (dFLC) and involved free light-chain (iFLC) | 17 SMM | 76 |
| Lenalidomide and dexamethasone | Lenalidomide+dex vs observation. 2-year PFS 92% vs 30%, P < .001; 3-year OS 93% vs 76%, P < .04 | 119 high risk SMM | 5 |
| Type of therapy . | Clinical trial design and outcome . | N of patients . | References . |
|---|---|---|---|
| Melphalan and prednisone (MP) | Retrospective cohort study of vincristine, adriamycin, and dexamethasone (VAD) vs MP. Because the treatment of MM remains palliative, chemotherapy should be withheld until symptoms | 23 SMM, 10 IMM | 60 |
| Initial vs delayed MP. Randomized-controlled trial. Similar response rate, response duration, and TTP of 12 months | 50 SMM and IMM (25/25) | 59 | |
| Initial vs delayed MP. TTP of about 12 months. No difference in OS (64 vs 71 months) | 145 DSSI | 61, 62 | |
| Observational study of delayed therapy: 54 DSS I. 2-year PFS 75%. Tumor specific OS 80% at 60 months | 54 DSSI | 71 | |
| Pamidronate or zolendronate | Single-arm, phase 2 trial; pamidronate vs observation. 5-year PFS 53% both arms. Skeletal-related events (SRE) 74% vs 39%, P = .009. Median OS 46 and 48 months | 177 SMM | 72,-74 |
| Open-label randomized controlled trial; zolendronate vs observation × 1 year. TTP not significant. SRE 55 vs 78%, P = .04. Zoledronate (ZLD) for 1 year decreased risk of skeletal-related disease, but TTP was similar (P = .83). OS no difference | 163 SMM | 63 | |
| Thalidomide | Single-arm, phase 2 trial phase 2 of thalidomide pamidronate. 4-year event-free survival (EFS) 60%. 4-year OS 91%. Median TTP 7 years; partial response (PR) identifies subset requiring earlier salvage therapy for symptomatic disease | 76 SMM | 64 |
| Single-arm pilot study of thalidomide. Median 35 months. OS 86 months. OS from treatment 49 months. Minimal response (MR) or better in 11/16. Microvessel density did not predict response | 19 SMM and I0 IMM | 66 | |
| Phase 2 of thalidomide. Patients were treated with thalidomide 100 to 200 mg. The response rate was 36% | 28 high risk SMM | 65 | |
| Phase 3 of thalidomide/ZLD vs ZLD | 68 SMM (35 to Thal/ZLD and 33 ZLD alone) | 67 | |
| Thalidomide +ZA vs ZA. 29 vs 14 months. 6-year OS >70% | 68 SMM | 67 | |
| IL-1 antagonist | Anakinra (IL-1 receptor antagonist). IL-1 antagonist +/− dexamethasone. Median PFS was 37.5 months. MR (n = 3), PR (n = 5). 8 patients stable on drug for 4 years | 47 SMM and IMM | 75 |
| Curcumin | Randomized, double-blind placebo-controlled crossover study. Administering 8 g dose of curcumin. Curcumin therapy decreased the free light-chain ratio, reduced the difference between clonal and monoclonal light-chain (dFLC) and involved free light-chain (iFLC) | 17 SMM | 76 |
| Lenalidomide and dexamethasone | Lenalidomide+dex vs observation. 2-year PFS 92% vs 30%, P < .001; 3-year OS 93% vs 76%, P < .04 | 119 high risk SMM | 5 |