Treatment trials for patients with SMM
| Reference . | Study . | Therapy . | N . | TTP . | OS . |
|---|---|---|---|---|---|
| Hjorth et al, 19934 | RCT | Initial vs delayed MP | 50 SMM and IMM | 12 mo | No difference |
| Riccardi et al, 1994 and 20005,6 | RCT | Initial vs delayed MP | 145 DSS I | ∼12 mo | No difference 64 mo vs 71 mo |
| Peest et al, 199578 | Observational | Delayed MP | 54 DSS I | 2-y PFS 75% | Tumor-specific OS 80% at 60 mo |
| Martin et al, 20027 | Pilot | Pamidronate | 5 SMM and 7 IMM | 2-y TTP 25% | |
| Musto et al, 20038 and D’Arena et al, 20119 | RCT | Pamidronate vs observation | 177 SMM | 5-y PFS 53% both arms; SRE 74% vs 39%, P = .009 | Median OS 46 mo and 48 mo |
| Musto et al, 200810 | RCT | Zoledronate vs observation × 1 y | 163 SMM | TTP: 67 mo vs 59 mo, P = NS SRE: 55% vs 78%, P = .04 | OS not different |
| Barlogie et al, 200811 | Phase 2 | Thalidomide pamidronate | 76 SMM | 4-y EFS 60% | 4-y OS 91% |
| Rajkumar et al, 200112,13 | Phase 2 | Thalidomide | 19 SMM and 10 IMM | Median 35 mo | OS: 86 mo OS from treatment: 49 mo |
| Weber et al, 200314 | Phase 2 | Thalidomide | 28 high-risk SMM | NA | NA |
| Witzig et al, 201348 | RCT | Thalidomide + ZA vs ZA | 68 SMM | 29 mo vs 14 mo | 6 y > 70% |
| Lust et al, 200979 | Ph 2 | Interleukin-1 receptor antagonist ± dexamethasone | 47 SMM and IMM | 37 mo | |
| Golombick et al, 200980 | Crossover | Curcumin vs placebo | 17 SMM | ||
| Mateos et al, 201315 | RCT | Lenalidomide + dexamethasone × 9 mo → lenalidomide maintenance × 15 mo vs observation | 119 SMM | 2-y PFS: 92% vs 50%, P < .001 | 3-y OS: 93% vs 76%, P = .04 |
| Reference . | Study . | Therapy . | N . | TTP . | OS . |
|---|---|---|---|---|---|
| Hjorth et al, 19934 | RCT | Initial vs delayed MP | 50 SMM and IMM | 12 mo | No difference |
| Riccardi et al, 1994 and 20005,6 | RCT | Initial vs delayed MP | 145 DSS I | ∼12 mo | No difference 64 mo vs 71 mo |
| Peest et al, 199578 | Observational | Delayed MP | 54 DSS I | 2-y PFS 75% | Tumor-specific OS 80% at 60 mo |
| Martin et al, 20027 | Pilot | Pamidronate | 5 SMM and 7 IMM | 2-y TTP 25% | |
| Musto et al, 20038 and D’Arena et al, 20119 | RCT | Pamidronate vs observation | 177 SMM | 5-y PFS 53% both arms; SRE 74% vs 39%, P = .009 | Median OS 46 mo and 48 mo |
| Musto et al, 200810 | RCT | Zoledronate vs observation × 1 y | 163 SMM | TTP: 67 mo vs 59 mo, P = NS SRE: 55% vs 78%, P = .04 | OS not different |
| Barlogie et al, 200811 | Phase 2 | Thalidomide pamidronate | 76 SMM | 4-y EFS 60% | 4-y OS 91% |
| Rajkumar et al, 200112,13 | Phase 2 | Thalidomide | 19 SMM and 10 IMM | Median 35 mo | OS: 86 mo OS from treatment: 49 mo |
| Weber et al, 200314 | Phase 2 | Thalidomide | 28 high-risk SMM | NA | NA |
| Witzig et al, 201348 | RCT | Thalidomide + ZA vs ZA | 68 SMM | 29 mo vs 14 mo | 6 y > 70% |
| Lust et al, 200979 | Ph 2 | Interleukin-1 receptor antagonist ± dexamethasone | 47 SMM and IMM | 37 mo | |
| Golombick et al, 200980 | Crossover | Curcumin vs placebo | 17 SMM | ||
| Mateos et al, 201315 | RCT | Lenalidomide + dexamethasone × 9 mo → lenalidomide maintenance × 15 mo vs observation | 119 SMM | 2-y PFS: 92% vs 50%, P < .001 | 3-y OS: 93% vs 76%, P = .04 |
EFS, event-free survival; MP, melphalan-prednisone; NA, not applicable; NS, not significant; RCT, randomized controlled trial; ZA, zoledronic acid.