Table 2 Summary of clinical data for emerging/novel therapies for essential thrombocythemia
From: Emerging agents and regimens for polycythemia vera and essential thrombocythemia
Agent | Mechanism of action | Clinical data | Reference |
|---|---|---|---|
Gandotinib | Pan-JAK inhibition | Phase 1 trial [31] N = 1 Results: single included patients with ET had a PR Phase 2 trial [32] N = 21 (median age 60 years) Results: 3 patients (14%) had CR, 16 (76%) had PR, thus ORR of 90% | |
Imetelstat | Telomerase activity inhibition | Phase 2 trial N = 18 (44% age > 60 years, 44% with JAK2 mutation; 94 and 72% with prior hydroxyurea and anagrelide exposure, respectively) Results: 100% hematologic response rate with 89% CHR rate | [40] |