Clinical trials results for JAK inhibitors in MF
| Drug . | Current development stage . | Constitutional symptoms response, % . | Palpable/MRI spleen response, % . | Anemia response, % . | Leukocytosis response, % . | Thrombocytosis response, % . | JAK2V617F allele burden response, % . | Side effects . | Annual cost, $ (US) . |
|---|---|---|---|---|---|---|---|---|---|
| Ruxolitinib58,71,73 | FDA-approved; phase 3 (n = 528); phase 1/2 (n = 153) | > 50 | 29-42 (by MRI) | 14 | * | 44 | Unremarkable | Anemia; thrombocytopenia; “ruxolitinib withdrawal syndrome”† | > 80 000 |
| SAR30250359 | Phase 3; phase 1/2 (n = 59) | > 50 | 39 | 0 | 72 | 90 | 45 | Nausea/diarrhea; anemia; thrombocytopenia; transaminasemia; hyperlipase/amylasemia | NA |
| Lestaurtinib60 | Phase 2 (n = 22) | NR | > 18 | 25‡ | NR | NR | Unremarkable | Nausea/diarrhea; anemia; thrombocytopenia | NA |
| CYT38782,83 | Phase 2 (n = 163) | > 50 | 45 | 50 | NR | NR | Unremarkable | Thrombocytopenia; headaches, first dose effect§; peripheral neuropathy; transaminasemia; hyperlipase/amylasemia | NA |
| SB151887 | Phase 2 (n = 34) | > 50 | 32 (by MRI) | ‖ | NR | NR | NR | Nausea/diarrhea | NA |
| LY278454488 | Phase 1/2 (n = 19) | NR | > 22 | NR | NR | NR | Unremarkable | Nausea/diarrhea; anemia electrolyte abnormalities/TLS?; increases in serum creatinine | NA |
| XL01989 | Halted (n = 21) | > 50 | 33 | NR | ¶ | NR | NR | Peripheral neuropathy | NA |
| AZD1480 | Phase 1/2 | No information available | NA | NA | NA | NA | NA | NA | NA |
| BMS911543 | Phase 1/2 | No information available | NA | NA | NA | NA | NA | NA | NA |
| NS-018 | Phase 1/2 | No information available | NA | NA | NA | NA | NA | NA | NA |
| Drug . | Current development stage . | Constitutional symptoms response, % . | Palpable/MRI spleen response, % . | Anemia response, % . | Leukocytosis response, % . | Thrombocytosis response, % . | JAK2V617F allele burden response, % . | Side effects . | Annual cost, $ (US) . |
|---|---|---|---|---|---|---|---|---|---|
| Ruxolitinib58,71,73 | FDA-approved; phase 3 (n = 528); phase 1/2 (n = 153) | > 50 | 29-42 (by MRI) | 14 | * | 44 | Unremarkable | Anemia; thrombocytopenia; “ruxolitinib withdrawal syndrome”† | > 80 000 |
| SAR30250359 | Phase 3; phase 1/2 (n = 59) | > 50 | 39 | 0 | 72 | 90 | 45 | Nausea/diarrhea; anemia; thrombocytopenia; transaminasemia; hyperlipase/amylasemia | NA |
| Lestaurtinib60 | Phase 2 (n = 22) | NR | > 18 | 25‡ | NR | NR | Unremarkable | Nausea/diarrhea; anemia; thrombocytopenia | NA |
| CYT38782,83 | Phase 2 (n = 163) | > 50 | 45 | 50 | NR | NR | Unremarkable | Thrombocytopenia; headaches, first dose effect§; peripheral neuropathy; transaminasemia; hyperlipase/amylasemia | NA |
| SB151887 | Phase 2 (n = 34) | > 50 | 32 (by MRI) | ‖ | NR | NR | NR | Nausea/diarrhea | NA |
| LY278454488 | Phase 1/2 (n = 19) | NR | > 22 | NR | NR | NR | Unremarkable | Nausea/diarrhea; anemia electrolyte abnormalities/TLS?; increases in serum creatinine | NA |
| XL01989 | Halted (n = 21) | > 50 | 33 | NR | ¶ | NR | NR | Peripheral neuropathy | NA |
| AZD1480 | Phase 1/2 | No information available | NA | NA | NA | NA | NA | NA | NA |
| BMS911543 | Phase 1/2 | No information available | NA | NA | NA | NA | NA | NA | NA |
| NS-018 | Phase 1/2 | No information available | NA | NA | NA | NA | NA | NA | NA |
NA indicates not available; NR, not clearly reported; and TLS, tumor lysis syndrome.
Mean leukocyte count after 3 months of treatment decreased from 29.8 × 109/L to 16.0 × 109/L.
Characterized by hyperacute relapse of symptoms, painful enlargement of spleen, and cardiopulmonary distress.
Two of 8 transfusion-dependent patients became transfusion-independent.
Characterized by transient lightheadedness and hypotension occurring only during the first dose.
Two patients were reported to obtain anemia response but no denominator given.
Responses in leukocyte counts mentioned but accurate figures not given.