Open-label study of oral CEP-701 (lestaurtinib) in patients with polycythaemia vera or essential thrombocythaemia withJAK2-V617F mutation
In: British Journal of Haematology, Jg. 164 (2013-10-28), S. 83-93
Online
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Zugriff:
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 80 mg twice daily for 18 weeks 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.
Titel: |
Open-label study of oral CEP-701 (lestaurtinib) in patients with polycythaemia vera or essential thrombocythaemia withJAK2-V617F mutation
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Autor/in / Beteiligte Person: | Moliterno, Alison R. ; Mascarenhas, John ; Hexner, Elizabeth O. ; Hoffman, Ron ; Luger, Selina M. ; Carroll, Martin ; Clementi, Regina ; Roboz, Gail J. ; Bensen-Kennedy, Debra M. |
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Zeitschrift: | British Journal of Haematology, Jg. 164 (2013-10-28), S. 83-93 |
Veröffentlichung: | Wiley, 2013 |
Medientyp: | unknown |
ISSN: | 0007-1048 (print) |
DOI: | 10.1111/bjh.12607 |
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