Table 1 Hematologic response and survival outcome of different treatment modalities in AL amyloidosis with different genetic aberrations
Treatment modalities | Arm 1 | Arm 2 | Patient number (n1/n2) | Hematologic response | Survival outcome | ||||
|---|---|---|---|---|---|---|---|---|---|
End point 1 | Arm 1/Arm 2 | P valuec | End point 2 | Arm 1/Arm 2 | P value | ||||
Bortezomib-based [34] | t(11;14) | Non-(11;14) | 44/91 | ≥ VGPR | 41%/66% | < 0.01 | 5-year OS | 46%/72% | 0.026 |
Bortezomib-based [33] | t(11;14) | Non-(11;14) | 64/37 | ≥ VGPR | 23%/47% | 0.02 | Median OS | 8.7 months/40.7 months | 0.05 |
Bortezomib-based [18] | t(11;14) | Non-(11;14) | 82/89 | ≥ VGPR | 52%/77% | 0.004 | Median OS | 15.0 months/27.0 months | 0.05 |
Bortezomib-based [18] | Trisomies | Non trisomies | 45/125 | ≥ VGPR | 72%/65% | 0.46 | Median OS | 14.0 months/38.0 months | 0.08 |
Bortezomib-based [33] | High risk aberrationsa | Non-high risk aberrations | 13/85 | ≥ VGPR | 67%/26% | 0.008 | Median OS | NR/10.6 months | 0.04 |
MD [35] | t(11;14) | Non-(11;14) | 61/42 | ≥ VGPR | 18%/22% | 0.60 | Median OS | 38.2 months/17.5 months | 0.21 |
MD [35] | Gain of 1q21 | Non gain of 1q21 | 23/77 | ≥ VGPR | 5%/25% | 0.06 | Median OS | 12.5 months/38.2 months | 0.002 |
MD [35] | Del13q14 | Non del13q14 | 36/66 | ≥ VGPR | 23%/18% | 0.78 | 5-year OS | 36%/33% | 0.70 |
MD [35] | Hyperdiploidyb | Non-hyperdiploidy | 15/84 | ≥ VGPR | 10%/22% | 1 | 5-year OS | 40%/36% | 0.70 |
Melphalan-based [18] | t(11;14) | Non-(11;14) | 96/95 | ≥ VGPR | 41%/54% | 0.13 | Median OS | 23.0 months/26.0 months | 0.94 |
Melphalan-based [18] | Trisomies | Non trisomies | 49/139 | ≥ VGPR | 39%/52% | 0.2 | Median OS | 15.0 months/32.0 months | 0.02 |
HDM + ASCT [36] | t(11;14) | Non-(11;14) | 72/51 | CR | 41%/20% | 0.02 | Median OS | NR/93.7 months | 0.07 |
HDM + ASCT [36] | Gain of 1q21 | Non gain of 1q21 | 25/91 | CR | 22%/35% | 0.32 | Median OS | NR/128.8 months | 0.93 |
HDM + ASCT [36] | Del13q14 | Non del13q14 | 36/87 | CR | 21%/37% | 0.13 | Median OS | 128.8 months/NR | 0.10 |
HDM + ASCT [36] | Hyperdiploidyb | Non-hyperdiploidy | 16/95 | CR | 27%/33% | 0.77 | Median OS | 90.6 months/128.8 months | 0.84 |
HDM + ASCT [36] | High risk aberrationsa | Non high risk aberrations | 9/113 | CR | 0%/35.2% | 0.03 | Median OS | 47.4 months/NR | 0.06 |
ASCT [18] | t(11;14) | Non-(11;14) | 134/113 | ≥ VGPR | 70%/78% | 0.15 | Median OS | NR/NR | 0.51 |
ASCT [18] | Trisomies | Non trisomies | 56/186 | ≥ VGPR | 80%/71% | 0.17 | Median OS | NR/NR | 0.98 |
DD [30] | t(11;14) | Non-(11;14) | 53/32 | Median hemEFS | 24.3 months/5.5 months | < 0.01 | Median OS | NR/19.3 months | 0.07 |
DD [30] | Gain of 1q21 | Non gain of 1q21 | 25/58 | Median hemEFS | 5.8 months/21.6 months | 0.03 | Median OS | 14.8 months/NR | 0.02 |
DVD [30] | t(11;14) | Non-(11;14) | 23/20 | Median hemEFS | 19.0 months/10.0 months | 0.69 | Median OS | NR/NR | 0.62 |
DVD [30] | Gain of 1q21 | Non gain of 1q21 | 10/30 | Median hemEFS | 6.8 months/ 22.1 months | 0.11 | Median OS | 9.5 months/NR | 0.02 |
DRD [66] | t(11;14) | Non-(11;14) | 16/15 | Median hemEFS | 17.3 months/22.6 months | > 0.05 | – | ||
DRD [66] | Gain of 1q21 | Non gain of 1q21 | 16/15 | Median hemEFS | 10.9 months/NR | < 0.01 | – | ||
IMiD-based [18] | t(11;14) | Non-(11;14) | 8/15 | ≥ VGPR | 13%/54% | 0.04 | Median OS | 12 months/32 months | 0.05 |
IMiD-based [18] | Trisomies | Non trisomies | 7/17 | ≥ VGPR | 40%/38% | 0.92 | Median OS | 17 months/23 months | 0.93 |
Venetoclax [44] | t(11;14) | Non-(11;14) | 31/11 | ≥ VGPR | 78%/30% | 0.02 | Median OS | NR/NR | 0.14 |