{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,26]],"date-time":"2026-09-26T14:27:02Z","timestamp":1790432822449,"version":"4.1.0"},"reference-count":31,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2013,10,28]],"date-time":"2013-10-28T00:00:00Z","timestamp":1382918400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Br J Haematol"],"published-print":{"date-parts":[[2014,1]]},"abstract":"<jats:title>Summary<\/jats:title><jats:p><jats:italic><jats:styled-content style=\"fixed-case\">JAK<\/jats:styled-content>2\u2010<\/jats:italic>V617F is central to the pathogenesis of myeloproliferative neoplasms. We examined whether lestaurtinib decreased <jats:italic><jats:styled-content style=\"fixed-case\">JAK<\/jats:styled-content>2\u2010<\/jats:italic>V617F allele burden and evaluated its clinical benefits and tolerability in patients with polycythaemia vera (<jats:styled-content style=\"fixed-case\">PV<\/jats:styled-content>) and essential thrombocythaemia (<jats:styled-content style=\"fixed-case\">ET<\/jats:styled-content>). This phase 2, open\u2010label, multicentre study was designed to detect \u226515% reduction in <jats:italic><jats:styled-content style=\"fixed-case\">JAK<\/jats:styled-content>2\u2010<\/jats:italic>V617F allele burden in 15% of patients. Eligible patients received lestaurtinib 80\u00a0mg twice daily for 18\u00a0weeks and could participate in a 1\u2010year extension phase of treatment. Of 39 enrolled patients, 27 (69%) had <jats:styled-content style=\"fixed-case\">PV<\/jats:styled-content>; 12 (31%) had <jats:styled-content style=\"fixed-case\">ET<\/jats:styled-content>. While the pre\u2010specified responder rate of 15% was not met, lestaurtinib modestly reduced <jats:italic><jats:styled-content style=\"fixed-case\">JAK<\/jats:styled-content>2\u2010<\/jats:italic>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 \u22651 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 <jats:italic><jats:styled-content style=\"fixed-case\">JAK<\/jats:styled-content>2<\/jats:italic>\u2010inhibitor treatment in patients with <jats:styled-content style=\"fixed-case\">PV<\/jats:styled-content>\/<jats:styled-content style=\"fixed-case\">ET<\/jats:styled-content> and highlights both the need for further studies to assess the role of <jats:italic><jats:styled-content style=\"fixed-case\">JAK<\/jats:styled-content>2<\/jats:italic> inhibition in treatment of <jats:styled-content style=\"fixed-case\">PV<\/jats:styled-content>\/<jats:styled-content style=\"fixed-case\">ET<\/jats:styled-content> and the use of <jats:italic><jats:styled-content style=\"fixed-case\">JAK<\/jats:styled-content>2\u2010<\/jats:italic>V617F as a biomarker for response. This trial was registered at <jats:ext-link xmlns:xlink=\"http:\/\/www.w3.org\/1999\/xlink\" xlink:href=\"http:\/\/www.clinicaltrials.gov\">www.clinicaltrials.gov<\/jats:ext-link> as <jats:styled-content style=\"fixed-case\">NCT<\/jats:styled-content>00586651.<\/jats:p>","DOI":"10.1111\/bjh.12607","type":"journal-article","created":{"date-parts":[[2013,10,28]],"date-time":"2013-10-28T08:58:38Z","timestamp":1382950718000},"page":"83-93","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":27,"title":["Open\u2010label study of oral <scp>CEP<\/scp>\u2010701 (lestaurtinib) in patients with polycythaemia vera or essential thrombocythaemia with <i><scp>JAK<\/scp>2\u2010<\/i>V617F mutation"],"prefix":"10.1111","volume":"164","author":[{"given":"Elizabeth","family":"Hexner","sequence":"first","affiliation":[{"name":"University of Pennsylvania Philadelphia PA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gail","family":"Roboz","sequence":"additional","affiliation":[{"name":"Weill Cornell Medical College New York NY USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ron","family":"Hoffman","sequence":"additional","affiliation":[{"name":"Mount Sinai School of Medicine Tisch Cancer Institute New York NY USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Selina","family":"Luger","sequence":"additional","affiliation":[{"name":"University of Pennsylvania Philadelphia PA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John","family":"Mascarenhas","sequence":"additional","affiliation":[{"name":"Mount Sinai School of Medicine Tisch Cancer Institute New York NY USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Martin","family":"Carroll","sequence":"additional","affiliation":[{"name":"University of Pennsylvania Philadelphia PA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Regina","family":"Clementi","sequence":"additional","affiliation":[{"name":"Teva Branded Pharmaceutical Products R&amp;D, Inc. 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