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. 2020 Dec 29;5(1):e516. doi: 10.1097/HS9.0000000000000516

Table 1.

Selected Clinical Studies With JAK Inhibitors in MPN.

JAK Inhibitors Clinical Trial Design Clinical Impact Main Side Effects
Ruxolitinib MF: COMFORT-I phase 324,27 vs Placebo Spleen volume reduction Reduction of constitutional symptoms Survival benefit Anemia, thrombocytopenia
MF: COMFORT-II phase 335,37 vs BAT
PV: RESPONSE phase 325,36 vs BAT Spleen volume reduction Hematocrit control Reduction of constitutional symptoms
PV: RESPONSE-2 phase 338 vs BAT
ET: MAJIC-ET phase 226 vs BAT Nonsignificant benefit (complete hematologic response)
Fedratinib MF: JAKARTA phase 339 vs Placebo Spleen volume reduction Anemia, gastrointestinal, encephalopathy (black box warning!)
Rux naive
MF: JAKARTA-2 phase 240 vs Placebo Reduction of constitutional symptoms
Rux refractory
Momelotinib MF: SIMPLIFY-1 phase 341 vs Rux Spleen volume reduction (n.s.) Reduction of constitutional symptoms (n.s.) Decreased transfusion dependence Peripheral neuropathy, anemia, thrombocytopenia
Rux naive
MF: SIMPLIFY-2 phase 242 vs BAT
Rux refractory
Pacritinib MF: PERSIST-1 phase 343 vs BAT Spleen volume reduction Reduction of constitutional symptoms Decreased transfusion dependence Anemia, thrombocytopenia, gastrointestinal
MF: PERSIST-2 phase 344 vs BAT, in thrombocytopenia

Key studies on JAK2 inhibitors in clinical development are indicated including design, main clinical impact, and most important side effects.

BAT = best available therapy; ET = essential thrombocythemia; JAK = Janus kinase; MF = myelofibrosis; MPNs = myeloproliferative neoplasms; n.s. = non significant; PV = polycythemia vera; Rux = ruxolitinib.