Table 3.
Larger studies of bortezomib containing regimens in light chain amyloidosis
| Study | Treatment regimen | Study design | N | Patient characteristics | Overall response (%) | Complete response (%) | PFS | OS |
|---|---|---|---|---|---|---|---|---|
| Kastritis 2010 [1] | Bortezomib with or without dexamethasone | Retrospective analysis | 94 | 19% untreated, 81% previously treated; 69% refractory | 71 | 25 | 25.5 months | 1-year survival: 76% |
| Palladini 2014 [2] |
BMD MD |
Matched case-control study |
87 87 |
Upfront therapy | 69 vs. 51 | 42 vs. 19 | Died 39% vs. 55% at median follow-up 26 months | |
| Reece 2011 [3] | Bortezomib1.6 mg/m2 once weekly vs. 1.3 mg/m2 twice weekly | Phase I/II study | 70 | Relapsed | 68.8 vs. 66.7 | 37.5 vs. 24.2 | 1-year PFS: 72.2% vs. 74.6% | 1-year survival rates: 93.8% vs. 84.0% |
| Kastritis 2017 [5] | VD vs. VCD | Retrospective comparison |
59 42 |
Upfront therapy | 68 vs. 78 (p = 0.26) | 27 vs. 21 | ND | 33 vs. 36 months (p = 0.45) |
| Palladini 2015 [6] | VCD | Retrospective study | 230 | Upfront therapy | 60 | 21 | Median time to SLT or death: 13 months | OS at 3 years: 55% |
| Venner 2014 [7] | VCD vs. CTD | Matched comparison | 69 vs. 69 | Upfront therapy | 71.0 vs. 79.7 (p = 0.32). | (40.5) vs. CTD (24.6), p = 0.046 | Median PFS: NR vs. 19.2 months (p = 0.028) | 1-year OS 96% vs. 92% (p = 0.40) |
BMD bortezomib plus melphalan plus dexamethasone, CTD cyclophosphamide, thalidomide, and dexamethasone, MD melphalan plus dexamethasone, ND not done, NR not reached, OS overall survival, PFS progression-free survival, SLT second-line therapy, VCD bortezomib plus cyclophosphamide plus dexamethasone, VD bortezomib plus dexamethasone, VGPR very good partial response