Results from recent clinical trials in patients with light-chain amyloidosis
| Reference . | Treatment regimen . | Newly diagnosed vs previously treated . | No. of patients . | Heme PR, % . | Heme CR, % . | Organ response, % . | Adverse events, ≥ grade 3, % . | OS from study entry . |
|---|---|---|---|---|---|---|---|---|
| Palladini et al6 | Mel 0.22 mg/d, Dex 40 mg/d, days 1-4 of 28 | Newly diagnosed, SCT ineligible | 46 | 67 | 33 | 48 | 11 | NA |
| Dispenzieri et al13 | Lenalidomide 25 mg/d, 21 of 28 days; Dex 40 mg, days 1-4, 15-18 added if no response after 3 cycles | Phase 2, mixed | 23 | 41 | NA | 23 | 86 | NA |
| Sanchorawala et al14 | Lenalidomide 25 mg/d, 21 of 28 days; Dex was added at 10-20 mg/d from days 1-4, 9-12, and 17-20 every other cycle, if no response after 3 cycles (lenalidomide dose was reduced to 15 mg after 8 patients) | Phase 2, mixed | 34 | 47 | 21 | 18 | NA | NA |
| Moreau et al17 | Mel 0.18 mg/kg/d, days 1-4 of 28; Dex 40 mg/d, days 1-4 of 28; lenalidomide 5, 10, or 15 mg, days 1-21 of 28 (MTD was 15 mg) | Phase 1/2 | 26 | 58 | 23 | 50 | NA | 54% at 2 y |
| Palladini et al16 | Lenalidomide 15 mg, days 1-21 of 28; Dex 20 mg on days 1, 8, 15, and 22 | Phase 2 (Btz, Mel refractory) | 24 | 38 | 0 | 4 | 50 | 14 mo |
| Dietrich et al28 | Mel 16 mg/m2 IV day 1; Dex 40 mg PO, days 1-4 of 28 | Retrospective, SCT ineligible | 61 | 44 | 11 | 25 | NA | 17.5 mo |
| Wechalekar12 | Cyclophosphamide 500 mg weekly, thalidomide 200 mg/d (start 100 mg/d, increased if tolerated) continuously; Dex 40 mg, days 1-4, 9-12, of 21 days (in elderly, 28 days cycle with Dex 20 mg, and cyclophosphamide 3 of 4 weeks) | Phase 2, mixed | 75 | 64 | 19 | 21 | NA | 41 mo |
| Kastritis18 | Btz 1.3 mg/m2 days 1, 4, 8, and 11; Dex dose was variable | Retrospective, mixed | 94 | 72 | 25 | 30 | NA | 76% at 1 y |
| Reece19 | Btz 1.3 mg/m2 days 1, 4, 8, and 11 (21-day cycle) | Phase 1/2, mixed | 34 | 67 | 24 | 27 | 79 | 84% at 1 y |
| Btz 1.6 mg/m2 days 1, 8, 15, 22 (35 day cycle) | 18 | 69 | 38 | 67 | 50 | 94% at 1 y | ||
| Wechalekar29 | Btz 1.3 mg/m2 days 1, 4, 8, and 11 (9 patients received variable doses of Dex) | Retrospective | 20 | 80 | 15 | 30 | 45 | NA |
| Reference . | Treatment regimen . | Newly diagnosed vs previously treated . | No. of patients . | Heme PR, % . | Heme CR, % . | Organ response, % . | Adverse events, ≥ grade 3, % . | OS from study entry . |
|---|---|---|---|---|---|---|---|---|
| Palladini et al6 | Mel 0.22 mg/d, Dex 40 mg/d, days 1-4 of 28 | Newly diagnosed, SCT ineligible | 46 | 67 | 33 | 48 | 11 | NA |
| Dispenzieri et al13 | Lenalidomide 25 mg/d, 21 of 28 days; Dex 40 mg, days 1-4, 15-18 added if no response after 3 cycles | Phase 2, mixed | 23 | 41 | NA | 23 | 86 | NA |
| Sanchorawala et al14 | Lenalidomide 25 mg/d, 21 of 28 days; Dex was added at 10-20 mg/d from days 1-4, 9-12, and 17-20 every other cycle, if no response after 3 cycles (lenalidomide dose was reduced to 15 mg after 8 patients) | Phase 2, mixed | 34 | 47 | 21 | 18 | NA | NA |
| Moreau et al17 | Mel 0.18 mg/kg/d, days 1-4 of 28; Dex 40 mg/d, days 1-4 of 28; lenalidomide 5, 10, or 15 mg, days 1-21 of 28 (MTD was 15 mg) | Phase 1/2 | 26 | 58 | 23 | 50 | NA | 54% at 2 y |
| Palladini et al16 | Lenalidomide 15 mg, days 1-21 of 28; Dex 20 mg on days 1, 8, 15, and 22 | Phase 2 (Btz, Mel refractory) | 24 | 38 | 0 | 4 | 50 | 14 mo |
| Dietrich et al28 | Mel 16 mg/m2 IV day 1; Dex 40 mg PO, days 1-4 of 28 | Retrospective, SCT ineligible | 61 | 44 | 11 | 25 | NA | 17.5 mo |
| Wechalekar12 | Cyclophosphamide 500 mg weekly, thalidomide 200 mg/d (start 100 mg/d, increased if tolerated) continuously; Dex 40 mg, days 1-4, 9-12, of 21 days (in elderly, 28 days cycle with Dex 20 mg, and cyclophosphamide 3 of 4 weeks) | Phase 2, mixed | 75 | 64 | 19 | 21 | NA | 41 mo |
| Kastritis18 | Btz 1.3 mg/m2 days 1, 4, 8, and 11; Dex dose was variable | Retrospective, mixed | 94 | 72 | 25 | 30 | NA | 76% at 1 y |
| Reece19 | Btz 1.3 mg/m2 days 1, 4, 8, and 11 (21-day cycle) | Phase 1/2, mixed | 34 | 67 | 24 | 27 | 79 | 84% at 1 y |
| Btz 1.6 mg/m2 days 1, 8, 15, 22 (35 day cycle) | 18 | 69 | 38 | 67 | 50 | 94% at 1 y | ||
| Wechalekar29 | Btz 1.3 mg/m2 days 1, 4, 8, and 11 (9 patients received variable doses of Dex) | Retrospective | 20 | 80 | 15 | 30 | 45 | NA |
PR indicates partial response; CR, complete response; OS, overall survival; PO, oral; NA, not available; Btz, bortezomib; Mel, melphalan; Dex, dexamethasone; SCT, autologous stem cell transplantation; and MTD, maximum tolerated dose.