A phase 1 study of the Janus kinase 2 (JAK2)V617F inhibitor, gandotinib (LY2784544), in patients with primary myelofibrosis, polycythemia vera, and essential thrombocythemia

• Published in: Leukemia research Vol. 61; pp. 89 - 95
• Main Authors: Verstovsek, Srdan, Mesa, Ruben A., Salama, Mohamed E., Li, Li, Pitou, Celine, Nunes, Fabio P., Price, Gregory L., Giles, Jennifer L., D’Souza, Deborah N., Walgren, Richard A., Prchal, Josef T.
• Format: Journal Article
• Language: English
• Published: England Elsevier Ltd 01.10.2017
• Subjects: Adult; Aged; Antineoplastic Agents - therapeutic use; Dosage; Female; Gandotinib; Humans; Imidazoles - therapeutic use; JAK-2; Janus Kinase 2 - antagonists & inhibitors; Male; Maximum Tolerated Dose; Middle Aged; Myeloproliferative; Neoplasm; Polycythemia Vera - drug therapy; Primary Myelofibrosis - drug therapy; Protein Kinase Inhibitors - therapeutic use; Pyrazoles - therapeutic use; Pyridazines - therapeutic use; Thrombocythemia, Essential - drug therapy; Neoplasm; JAK-2; Gandotinib; Myeloproliferative; Dosage

•Gandotinib shows increased potency for the JAK2V617F mutation.•The recommended phase 2 dose of 120mg was associated with clinical improvement.•Findings at the maximum-tolerated dose of 120mg supported further clinical testing.•Gandotinib demonstrated an acceptable safety and tolerability profile. M...