Selected JAKi trials in PV
| Agent . | Target(s) . | Clinical trial . | Numerosity and study-specific features . | Key results . | Toxicities . | Comments . |
|---|---|---|---|---|---|---|
| RUX | JAK1/2 | RESPONSE,35 RUX vs BAT | RUX (n = 110), BAT (n = 112); HU resistant/ intolerant, in need of phlebotomies, with splenomegaly | Hematocrit control without phlebotomy at wk 32: 60% (RUX) vs 18.8% (BAT); SVR ≥35% at wk 32: 40% (RUX) vs 0.9% (BAT); composite end-point: 22.7% (RUX) vs 0.9% (BAT) | All grade anemia, 27.2 per 100 p-y; G3/4, 0.9 per 100 p-y; all-grade thrombocytopenia, 14.9 per 100 p-y; G3/4, 2.6 per 100 p-y | Led to FDA/EMA approval for HU resistant/intolerant PV |
| RESPONSE-2,36 RUX vs BAT | RUX (n = 74), BAT (n = 75); HU resistant/ intolerant, in need of phlebotomies, without splenomegaly | Hematocrit control at wk 28: 62% (RUX) vs 19% (BAT) | G1/2 anemia, 14%; G1/2 thrombocytopenia, 3%; no G3/4 anemia/ thrombocytopenia |
| Agent . | Target(s) . | Clinical trial . | Numerosity and study-specific features . | Key results . | Toxicities . | Comments . |
|---|---|---|---|---|---|---|
| RUX | JAK1/2 | RESPONSE,35 RUX vs BAT | RUX (n = 110), BAT (n = 112); HU resistant/ intolerant, in need of phlebotomies, with splenomegaly | Hematocrit control without phlebotomy at wk 32: 60% (RUX) vs 18.8% (BAT); SVR ≥35% at wk 32: 40% (RUX) vs 0.9% (BAT); composite end-point: 22.7% (RUX) vs 0.9% (BAT) | All grade anemia, 27.2 per 100 p-y; G3/4, 0.9 per 100 p-y; all-grade thrombocytopenia, 14.9 per 100 p-y; G3/4, 2.6 per 100 p-y | Led to FDA/EMA approval for HU resistant/intolerant PV |
| RESPONSE-2,36 RUX vs BAT | RUX (n = 74), BAT (n = 75); HU resistant/ intolerant, in need of phlebotomies, without splenomegaly | Hematocrit control at wk 28: 62% (RUX) vs 19% (BAT) | G1/2 anemia, 14%; G1/2 thrombocytopenia, 3%; no G3/4 anemia/ thrombocytopenia |
p-y, patient-years of exposure.