期刊
BRITISH JOURNAL OF HAEMATOLOGY
卷 164, 期 1, 页码 83-93出版社
WILEY
DOI: 10.1111/bjh.12607
关键词
polycythaemia vera; essential thrombocythaemia; JAK2-V617F mutation; lestaurtinib; CEP-701
类别
资金
- Teva Branded Pharmaceutical Products R&D, Inc, Frazer, PA
- Teva Branded Pharmaceutical Products RD, Inc.
JAK2-V617F is central to the pathogenesis of myeloproliferative neoplasms. We examined whether lestaurtinib decreased JAK2-V617F allele burden and evaluated its clinical benefits and tolerability in patients with polycythaemia vera (PV) and essential thrombocythaemia (ET). This phase 2, open-label, multicentre study was designed to detect 15% reduction in JAK2-V617F allele burden in 15% of patients. Eligible patients received lestaurtinib 80mg twice daily for 18weeks and could participate in a 1-year extension phase of treatment. Of 39 enrolled patients, 27 (69%) had PV; 12 (31%) had ET. While the pre-specified responder rate of 15% was not met, lestaurtinib modestly reduced JAK2-V617F allele burden and reduced spleen size in a subset of patients. Of 37 patients in the full efficacy analysis, 5 (14%) responded clinically. Every patient had 1 adverse event, most commonly gastrointestinal (95%). Fifteen patients (38%) experienced serious adverse events; 23 (59%) withdrew due to adverse events. This is the first reported study of JAK2-inhibitor treatment in patients with PV/ET and highlights both the need for further studies to assess the role of JAK2 inhibition in treatment of PV/ET and the use of JAK2-V617F as a biomarker for response. This trial was registered at www.clinicaltrials.gov as NCT00586651.
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