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. 2019 Apr 16;19(2):73–92. doi: 10.1007/s40268-019-0269-9

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