Table 2.
Treatment data of ruxolitinib-based combinations
| Median Hgb | Median Hgb | Median PLT | Median PLT | Transfusion dependence N(%) | Transfusion dependence N(%) | AR N(%) | ≥ 3 grade Hematologic AEs N(%) |
Survival | BM fibrosis | Symptoms response | IWG-MRT response criteria | Spleen reduction N(%) | AML N(%) | Median follow-up (months) | Reference | |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Combination drug | Start ruxolitinib | After combination | Start ruxolitinib | After combination | Start ruxolitinib | After combination | ||||||||||
| Danazol | 9.0 g/dL (8.3–12.4) |
Maximum change > 1.0 g/dL: N = 5(36) |
157 × 109/L (54–441) |
Maximum change > 50 × 109/L: N = 5(36) |
N = 5(36) | -- | N = 13/14(93) | N = 10(71) | -- | -- |
MPN-TSS: Improvement ≥ 50% N = 4 |
SD: N = 9(64); CI: N = 3(21); PR: N = 1(7); PD: N = 1(7) |
Spleen responses: N = 12(86) | -- | -- | [26] |
|
Thalidomide Prednisone Danazol |
8.7g/dL (5.7–14.4) | Increase: 3 g/dL (1.8–5.4) | 123 × 109/L (74–500) |
Increase: 116 × 109/L (13–369) |
N = 2(29) | N = 0 | -- | N = 0(0) | -- | -- | Median MPN-SAF-TSS decrease: 16 score | -- |
Spleen responses: N = 6(86) |
-- | -- | [27] |
| Lenalidomide | 11 g/dL (8.9–17.5) | – | 250 × 109/L (27–1898) | -- | -- | -- | -- | N = 16 | -- |
Reduction: N = 2/17(17) MF-2 to MF-1: N = 1 MF-2 to MF-0: N = 1 |
-- | -- |
100% spleen reduction: N = 7(23) 50% spleen reduction-: N = 10(32) |
-- | 28 (12–35+) | [28] |
| Panobinostat | 9.8g/dL (8.3–12.8) | -- | 347 × 109/L (95–672) | -- | N = 2(13) | -- | -- | N = 6(43) | -- |
Reduction: N = 7 (53) MF-3 to MF-2: N = 5 (33) MF-3 to MF-1: N = 1 (7) MF-2 to MF-1: N = 1 (7) |
MPN-SAF-TSS response: N = 3/12(25) | SD: N = 8 (57); CI: N = 6 (43) | 35% spleen volume response: N = 5(46) | -- | -- | [29] |
| Pracinostat | 10.9 g/dL (7.4–16.2) | -- | 253 × 109/L (107–698) | -- | -- | N = 4(20) | -- | N = 12 (60) | Median OS: 33.8 months |
MF-3 to MF-1: N = 2 MF-3 to MF-2: N = 1 |
MPN-SAF TSS response: N = 12/15(80) | N = 5 | Spleen response (by palpation): N = 14(74) | N = 1(5) | 21.4 (12.5–39.1) | [30] |
| Azacytidine | 10.1 g/dL (6.8–16.2) | -- | 271 × 109/L (126–835) | -- | -- | -- | -- |
New onset anemia: N = 25(64); Thrombocytopenia: N = 11(28) |
Median OS: 38.7+ months | EUMNET fibrosis score reduction: N = 11(28) | -- |
N = 27/39(69) CI: N = 25(64); PR: N = 2(5) |
> 50% palpable spleen reduction: N = 23(56) | N = 4(10) | 20.4+ (0.5–37.3+) | [31] |
| Azacytidine | 10.1 g/dL (6.8–16.2) | -- | 271 × 109/L (125–1070) | -- | N = 5(11) | N = 15(33) | N = 4/41(10) | -- |
DIPSS-high: Median OS: 28 (1–62) months |
BM reticulin fibrosis improved: N = 8/14(57) BM collagen improved: N = 7/14(50) |
MPN-SAF TSS: N = 25/46(54) | N = 7/33(21) | Spleen response: N = 21(62) | N = 6(13) | 22 (5–50) | [32] |
| Hydroxyurea | 10 g/dL (6.7–16.4) |
12 weeks: 9.5 g/dL (6.8–14.6) 24 weeks: 10.3 g/dL (7–13) 48 weeks: 11.4 g/dL (7.8–14) |
186 × 109/L (60–1110) |
12 weeks: 113 × 109/L (30–652) 24 weeks: 135 × 109/L (35–545) 48 weeks: 158 × 109/L (78–523) |
-- | -- | -- | N = 7(12) | -- | -- | -- | 48 weeks: 75% patients achieved a symptoms response |
Spleen responses: 12 weeks: 14/50(28) 24 weeks: 15/40(38) 48 weeks: 15/33(45) |
N = 4(8) | 28 (4–50+) | [33] |
| ESAs | 8.7 g/dL(6–10) | -- | -- | -- | N = 31(59) | -- | N = 32/59(54) | -- |
2 years OS: 78% 4 years OS: 62% |
-- | -- | -- | -- | -- | 48 | [34] |
| HSCT | -- | -- | -- | -- | -- | -- | -- | -- |
1 year OS: 81% 1 year DFS:76% |
-- | -- | -- |
Before HSCT Spleen size response: 45% |
N = 1(5) | 12 (6–13) | [35] |
| HSCT | -- | -- | -- | -- | -- | -- | -- |
During JAK1/2 inhibitor therapy: N = 49(49) |
2 years OS: 55% | -- | -- | -- | -- | -- | After HSCT: 17 (3–53) | [36] |
| HSCT | 11.1 g/dL(8.4–16.4) | -- | -- | -- | -- | -- | -- | -- | 1 year OS: 50% | -- | -- | -- | -- | N = 2(14) | After HSCT: 9 (4–43) | [37] |
Hgb, hemoglobin; PLT, platelet; AR, anemia response; SD, stable disease; CI, clinical improvement; PR, partial response; MPN-SAF TSS, MPN Symptom Assessment Form Total Symptom Score; EUMNET, the European Myelofibrosis Network