Ruxolitinib for the treatment of myelofibrosis: its clinical potential

References

• KuterDJBainBMuftiGBaggAHasserjianRPBone marrow fibrosis: pathophysiology and clinical significance of increased bone marrow stromal fibresBr J Haematol2007139335136217910625
   PubMed Web of Science ®Google Scholar
• VardimanJWThieleJArberDAThe 2008 revision of the World Health Organization (WHO) classification of myeloid neoplasms and acute leukemia: rationale and important changesBlood2009114593795119357394
   PubMed Web of Science ®Google Scholar
• BarosiGMesaRAThieleJProposed criteria for the diagnosis of post-polycythemia vera and post-essential thrombocythemia myelofibrosis: a consensus statement from the International Working Group for Myelofibrosis Research and TreatmentLeukemia200822243743817728787
   PubMed Web of Science ®Google Scholar
• ChaiterYBrennerBAghaiETatarskyIHigh incidence of myeloproliferative disorders in Ashkenazi Jews in northern IsraelLeuk Lymphoma1992732512551477653
   PubMed Web of Science ®Google Scholar
• DouganLEMatthewsMLArmstrongBKThe effect of diagnostic review on the estimated incidence of lymphatic and hematopoietic neoplasms in Western AustraliaCancer19814838668727248915
   PubMed Web of Science ®Google Scholar
• JohanssonPKuttiJAndreassonBTrends in the incidence of chronic Philadelphia chromosome negative (Ph−) myeloproliferative disorders in the city of Goteborg, Sweden, during 1983–1999J Intern Med2004256216116515257729
   PubMed Web of Science ®Google Scholar
• KuttiJRidellBEpidemiology of the myeloproliferative disorders: essential thrombocythaemia, polycythaemia vera and idiopathic myelofibrosisPathol Biol (Paris)200149216416611317963
   PubMedGoogle Scholar
• MesaRASilversteinMNJacobsenSJWollanPCTefferiAPopulation-based incidence and survival figures in essential thrombocythemia and agnogenic myeloid metaplasia: an Olmsted County Study, 1976–1995Am J Hematol1999611101510331505
   PubMed Web of Science ®Google Scholar
• WoodliffHJDouganLMyelofibrosis in Western Australia: an epidemiological study of 29 casesMed J Aust1976115523525933939
   PubMed Web of Science ®Google Scholar
• Alvarez-LarránACervantesFBellosilloBEssential thrombocythemia in young individuals: frequency and risk factors for vascular events and evolution to myelofibrosis in 126 patientsLeukemia20072161218122317519959
   PubMed Web of Science ®Google Scholar
• NajeanYRainJDDreschCRisk of leukaemia, carcinoma, and myelofibrosis in 32P- or chemotherapy-treated patients with polycythaemia vera: a prospective analysis of 682 cases. The “French Cooperative Group for the Study of Polycythaemias.”Leuk Lymphoma199622Suppl 11111198951781
   PubMed Web of Science ®Google Scholar
• PassamontiFRumiEArcainiLPrognostic factors for thrombosis, myelofibrosis, and leukemia in essential thrombocythemia: a study of 605 patientsHaematologica200893111645165118790799
   PubMed Web of Science ®Google Scholar
• RandiMLBarboneEFabrisFVarottoLMacriCGirolamiAPost- polycythemia myeloid metaplasia: experience with a large cohort of patientsJ Med19942563633697769374
   PubMedGoogle Scholar
• WolanskyjAPSchwagerSMMcClureRFLarsonDRTefferiAEssential thrombocythemia beyond the first decade: life expectancy, long-term complication rates, and prognostic factorsMayo Clin Proc200681215916616471068
   PubMed Web of Science ®Google Scholar
• VannucchiAMBianchiLCellaiCDevelopment of myelofibrosis in mice genetically impaired for GATA-1 expression (GATA-1(low) mice)Blood200210041123113212149188
   PubMed Web of Science ®Google Scholar
• Le Bousse-KerdilèsMCMartyréMCFrench INSERM research network on idiopathic myelofibrosis. Involvement of the fibrogenic cytokines, TGF-beta and bFGF, in the pathogenesis of idiopathic myelofibrosisPathol Biol (Paris)200149215315711317961
   PubMedGoogle Scholar
• ReillyJTPathogenesis of idiopathic myelofibrosis: role of growth factorsJ Clin Pathol19924564614641624590
   PubMed Web of Science ®Google Scholar
• ChagraouiHKomuraETulliezMGiraudierSVainchenkerWWendlingFProminent role of TGF-beta 1 in thrombopoietin-induced myelofibrosis in miceBlood2002100103495350312393681
   PubMed Web of Science ®Google Scholar
• JacobsonRJSaloAFialkowPJAgnogenic myeloid metaplasia: a clonal proliferation of hematopoietic stem cells with secondary myelofibrosisBlood1978512189194620081
   PubMed Web of Science ®Google Scholar
• TefferiAMyelofibrosis with myeloid metaplasiaN Engl J Med2000342171255126510781623
   PubMed Web of Science ®Google Scholar
• ReederTLBaileyRJDewaldGWTefferiABoth B and T lymphocytes may be clonally involved in myelofibrosis with myeloid metaplasiaBlood200310151981198312406879
   PubMed Web of Science ®Google Scholar
• IhleJNKerrIMJaks and Stats in signaling by the cytokine receptor superfamilyTrends Genet199511269747716810
   PubMed Web of Science ®Google Scholar
• RawlingsJSRoslerKMHarrisonDAThe JAK/STAT signaling pathwayJ Cell Sci2004117Pt 81281128315020666
   PubMed Web of Science ®Google Scholar
• DrachmanJGGriffinJDKaushanskyKThe c-Mpl ligand (thrombopoietin) stimulates tyrosine phosphorylation of Jak2, Shc, and c-MplJ Biol Chem199527010497949827534285
   PubMed Web of Science ®Google Scholar
• NeubauerHCumanoAMullerMWuHHuffstadtUPfefferKJak2 deficiency defines an essential developmental checkpoint in definitive hematopoiesisCell19989333974099590174
   PubMed Web of Science ®Google Scholar
• ParganasEWangDStravopodisDJak2 is essential for signaling through a variety of cytokine receptorsCell19989333853959590173
   PubMed Web of Science ®Google Scholar
• RadosevicNWintersteinDKellerJRNeubauerHPfefferKLinnekinDJAK2 contributes to the intrinsic capacity of primary hematopoietic cells to respond to stem cell factorExp Hematol200432214915615102475
   PubMed Web of Science ®Google Scholar
• SilvennoinenOWitthuhnBAQuelleFWClevelandJLYiTIhleJNStructure of the murine Jak2 protein-tyrosine kinase and its role in interleukin 3 signal transductionProc Natl Acad Sci U S A19939018842984338378315
   PubMed Web of Science ®Google Scholar
• WitthuhnBAQuelleFWSilvennoinenOJAK2 associates with the erythropoietin receptor and is tyrosine phosphorylated and activated following stimulation with erythropoietinCell19937422272368343951
   PubMed Web of Science ®Google Scholar
• Quintás-CardamaAVaddiKLiuPPreclinical characterization of the selective JAK1/2 inhibitor INCB018424: therapeutic implications for the treatment of myeloproliferative neoplasmsBlood2010115153109311720130243
   PubMed Web of Science ®Google Scholar
• PikmanYLeeBHMercherTMPLW515L is a novel somatic activating mutation in myelofibrosis with myeloid metaplasiaPLoS Med200637e27016834459
   PubMed Web of Science ®Google Scholar
• PardananiADLevineRLLashoTMPL515 mutations in myeloproliferative and other myeloid disorders: a study of 1182 patientsBlood2006108103472347616868251
   PubMed Web of Science ®Google Scholar
• OhSTSimondsEFJonesCNovel mutations in the inhibitory adaptor protein LNK drive JAK-STAT signaling in patients with myeloproliferative neoplasmsBlood2010116698899220404132
   PubMed Web of Science ®Google Scholar
• GrandFHHidalgo-CurtisCEErnstTFrequent CBL mutations associated with 11q acquired uniparental disomy in myeloproliferative neoplasmsBlood2009113246182619219387008
   PubMed Web of Science ®Google Scholar
• BarbuiTBarosiGBirgegardGPhiladelphia-negative classical myeloproliferative neoplasms: critical concepts and management recommendations from European LeukemiaNetJ Clin Oncol201129676177021205761
   PubMed Web of Science ®Google Scholar
• SamuelsonSSandmaierBMHeslopHEAllogeneic haematopoietic cell transplantation for myelofibrosis in 30 patients 60–78 years of ageBr J Haematol20111531768221323890
   PubMed Web of Science ®Google Scholar
• LöfvenbergEWahlinAManagement of polycythaemia vera, essential thrombocythaemia and myelofibrosis with hydroxyureaEur J Haematol19884143753813197824
   PubMed Web of Science ®Google Scholar
• Martínez-TrillosAGayaAMaffioliMEfficacy and tolerability of hydroxyurea in the treatment of the hyperproliferative manifestations of myelofibrosis: results in 40 patientsAnn Hematol201089121233123720567824
   PubMed Web of Science ®Google Scholar
• SteurerMGastlGJedrzejczakWWAnagrelide for thrombocytosis in myeloproliferative disorders: a prospective study to assess efficacy and adverse event profileCancer2004101102239224615476273
   PubMed Web of Science ®Google Scholar
• BirgegardGBjorkholmMKuttiJAdverse effects and benefits of two years of anagrelide treatment for thrombocythemia in chronic myeloproliferative disordersHaematologica200489552052715136214
   PubMed Web of Science ®Google Scholar
• SirhanSLashoTLHansonCAMesaRAPardananiATefferiAThe presence of JAK2V617F in primary myelofibrosis or its allele burden in polycythemia vera predicts chemosensitivity to hydroxyureaAm J Hematol200883536336518266209
   PubMed Web of Science ®Google Scholar
• MesaRANagorneyDSSchwagerSAllredJTefferiAPalliative goals, patient selection, and perioperative platelet management: outcomes and lessons from 3 decades of splenectomy for myelofibrosis with myeloid metaplasia at the Mayo ClinicCancer2006107236137016770787
   PubMed Web of Science ®Google Scholar
• ElliottMAChenMGSilversteinMNTefferiASplenic irradiation for symptomatic splenomegaly associated with myelofibrosis with myeloid metaplasiaBr J Haematol199810325055119827926
   PubMed Web of Science ®Google Scholar
• BesaECNowellPCGellerNLGardnerFHAnalysis of the androgen response of 23 patients with agnogenic myeloid metaplasia: the value of chromosomal studies in predicting response and survivalCancer19824923083137053830
   PubMed Web of Science ®Google Scholar
• ShimodaKShideKKamezakiKThe effect of anabolic steroids on anemia in myelofibrosis with myeloid metaplasia: retrospective analysis of 39 patients in JapanInt J Hematol200785433834317483079
   PubMed Web of Science ®Google Scholar
• CervantesFAlvarez-LarránADomingoAArellano-RodrigoEMontserratEEfficacy and tolerability of danazol as a treatment for the anaemia of myelofibrosis with myeloid metaplasia: long-term results in 30 patientsBr J Haematol2005129677177515953003
   PubMed Web of Science ®Google Scholar
• MerupMKuttiJBirgergårdGNegligible clinical effects of thalidomide in patients with myelofibrosis with myeloid metaplasiaMed Oncol2002192798612180484
   PubMed Web of Science ®Google Scholar
• BarosiGElliottMCanepaLThalidomide in myelofibrosis with myeloid metaplasia: a pooled-analysis of individual patient data from five studiesLeuk Lymphoma200243122301230712613516
   PubMed Web of Science ®Google Scholar
• MesaRAElliottMASchroederGTefferiADurable responses to thalidomide-based drug therapy for myelofibrosis with myeloid metaplasiaMayo Clin Proc200479788388915244384
   PubMed Web of Science ®Google Scholar
• AbgrallJFGuibaudIBastieJNThalidomide versus placebo in myeloid metaplasia with myelofibrosis: a prospective, randomized, double- blind, multicenter studyHaematologica20069181027103216885042
   PubMed Web of Science ®Google Scholar
• ThomasDAGilesFJAlbitarMThalidomide therapy for myelofibrosis with myeloid metaplasiaCancer200610691974198416583431
   PubMed Web of Science ®Google Scholar
• MesaRASteensmaDPPardananiAA phase 2 trial of combination low-dose thalidomide and prednisone for the treatment of myelofibrosis with myeloid metaplasiaBlood200310172534254112517815
   PubMed Web of Science ®Google Scholar
• JabbourEThomasDKantarjianHComparison of thalidomide and lenalidomide as therapy for myelofibrosisBlood2011118489990221622644
   PubMed Web of Science ®Google Scholar
• SilversteinMNGomesMRReMineWHElvebackLRAgnogenic myeloid metaplasia. Natural history and treatmentArch Intern Med196712055465506054588
   PubMedGoogle Scholar
• BarosiGMyelofibrosis with myeloid metaplasia: diagnostic definition and prognostic classification for clinical studies and treatment guidelinesJ Clin Oncol19991792954297010561375
   PubMed Web of Science ®Google Scholar
• KralovicsRPassamontiFBuserASA gain-of-function mutation of JAK2 in myeloproliferative disordersN Engl J Med2005352171779179015858187
   PubMed Web of Science ®Google Scholar
• JamesCUgoVLe CouèdicJPA unique clonal JAK2 mutation leading to constitutive signalling causes polycythaemia veraNature200543470371144114815793561
   PubMed Web of Science ®Google Scholar
• LevineRLWadleighMCoolsJActivating mutation in the tyrosine kinase JAK2 in polycythemia vera, essential thrombocythemia, and myeloid metaplasia with myelofibrosisCancer Cell20057438739715837627
   PubMed Web of Science ®Google Scholar
• BaxterEJScottLMCampbellPJAcquired mutation of the tyrosine kinase JAK2 in human myeloproliferative disordersLancet200536594641054106115781101
   PubMed Web of Science ®Google Scholar
• Quintàs-CardamaAKantarjianHCortesJVerstovsekSJanus kinase inhibitors for the treatment of myeloproliferative neoplasias and beyondNat Rev Drug Discov201110212714021283107
   PubMed Web of Science ®Google Scholar
• HedvatMHuszarDHerrmannAThe JAK2 inhibitor AZD1480 potently blocks Stat3 signaling and oncogenesis in solid tumorsCancer Cell200916648749719962667
   PubMed Web of Science ®Google Scholar
• TynerJWBummTGDeiningerJCYT387, a novel JAK2 inhibitor, induces hematologic responses and normalizes inflammatory cytokines in murine myeloproliferative neoplasmsBlood2010115255232524020385788
   PubMed Web of Science ®Google Scholar
• VerstovsekSOdenikeOScottBPhase I dose-escalation trial of SB1518, a novel JAK2/FLT3 inhibitor, in acute and chronic myeloid diseases, including primary or post-essential thrombocythemia/polycythemia vera myelofibrosis [abstract]Blood2009114223905
   Web of Science ®Google Scholar
• US Food and Drug AdministrationSilver SpringUS Food and Drug Administration [updated November 16, 2001; cited November 18, 2011]. Available from: http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm280102.htmAccessed December 21, 2011
   Google Scholar
• ManshouriTEstrovZKnezLCreightonCKantarjianHVerstovsekSBone marrow stroma-mediated paracrine inhibition of ruxolitinib (INCB018424)-induced apoptosis of JAK2V617F mutated cells: protective effect of myeloproliferative neoplasm patient-derived but not healthy donnor-derived stroma [abstract]Haematologica2011966 Suppl 20316
   Google Scholar
• DeshpandeAReddyMMSchadeGOKinase domain mutations confer resistance to novel inhibitors targeting JAK2V617F in myeloproliferative neoplasmsLeukemiaSeptember 162011 [Epub ahead of print.]
   Web of Science ®Google Scholar
• ShillingADNedzaFMEmmTMetabolism, excretion, and pharmacokinetics of [14C]INCB018424, a selective Janus tyrosine kinase 1/2 inhibitor, in humansDrug Metab Dispos201038112023203120699411
   PubMed Web of Science ®Google Scholar
• ShiJGChenXMcGeeRFThe pharmacokinetics, pharmacodynamics, and safety of orally dosed INCB018424 phosphate in healthy volunteersJ Clin Pharmacol201151121644165421257798
   PubMed Web of Science ®Google Scholar
• ChenXShiJMcGeeRThe effect of various degrees of hepatic dysfunction on the pharmacokinetics of INCB018424 [abstract]Clin Pharmacol Ther2010872 Suppl 1PIIIP59
   Google Scholar
• Jakafi [package insert]Wilmington DEIncyte Corporation2011
   Google Scholar
• ShiJGChenXEmmTThe effect of CYP3A4 inhibition or induction on the pharmacokinetics and pharmacodynamics of orally administered ruxolitinib (INCB018424 phosphate) in healthy volunteersJ Clin PharmacolMay 202011 [Epub ahead of print.]
   Web of Science ®Google Scholar
• OstojicAVrhovacRVerstovsekSRuxolitinib: a new JAK1/2 inhibitor that offers promising options for treatment of myelofibrosisFuture Oncol2011791035104321919691
   PubMed Web of Science ®Google Scholar
• OstojicAVrhovacRVerstovsekSRuxolitinib for the treatment of myelofibrosisDrugs Today (Barc)2011471181782922146225
   PubMed Web of Science ®Google Scholar
• VerstovsekSKantarjianHMesaRASafety and efficacy of INCB018424, a JAK1 and JAK2 inhibitor, in myelofibrosisN Engl J Med2010363121117112720843246
   PubMed Web of Science ®Google Scholar
• HarrisonCNKiladjianJAl-AliHKResults of a randomized study of the JAK inhibitor ruxolitinib (INC424) versus best available therapy (BAT) in primary myelofibrosis (PMF), post-polycythemia vera-myelofibrosis (PPV-MF) or post-essential thrombocythemia-myelofibrosis (PET-MF) [abstract]J Clin Oncol20112915 Suppl 1LBA6501
   Web of Science ®Google Scholar
• VerstovsekSMesaRGotlibJLevyRGuptaVResults of COMFORT-I, a randomized double-blind phase III trial of JAK 1/2 inhibitor INCB18424 (424) versus placebo (PB) for patients with myelofibrosis (MF) [abstract]J Clin Oncol20112915 Suppl 16500
   Web of Science ®Google Scholar
• VerstovsekSMesaRGotlibJResults of COMFORT-I, a randomized, double-blind phase III trial of the JAK1 and JAK2 inhibitor ruxolitinib (INCB018424) versus placebo for patients with myelofibrosis [abstract]Haematologica2011966 Suppl 20505
   Google Scholar
• TefferiALitzowMRPardananiALong-term outcome of treatment with ruxolitinib in myelofibrosisN Engl J Med2011365151455145721995409
   PubMed Web of Science ®Google Scholar
• TefferiAPardananiASerious adverse events during ruxolitinib treatment discontinuation in patients with myelofibrosisMayo Clin Proc201186121188119122034658
   PubMed Web of Science ®Google Scholar
• DupriezBMorelPDemoryJLPrognostic factors in agnogenic myeloid metaplasia: a report on 195 cases with a new scoring systemBlood1996883101310188704209
   PubMed Web of Science ®Google Scholar
• MesaRKantarjianHTefferiAFunctional assessment of performance status in patients with myelofibrosis (MF): Utility and feasibility of the 6-minute walk test (6MWT) [abstract]J Clin Oncol200996Suppl 15s7083
   Google Scholar
• VerstovsekSEstrovZCortesJThe MD AndersonCancer Center (MDACC) experience with ruxolitinib, an oral JAK1 and JAK2 inhibitor, in myelofibrosis: long-term follow-up outcomes of 107 patients from a phase I/II study [abstract]Blood2011118213851
   Web of Science ®Google Scholar
• VerstovsekSKantarjianHEstrovZComparison of outcomes of advanced myelofibrosis patients treated with ruxolitinib (INCB018424) to those of a historical control group: survival advantage of ruxolitinib therapy [abstract]Blood201111821793
   Web of Science ®Google Scholar
• MesaRAKantarjianHTefferiAEvaluating the serial use of the Myelofibrosis Symptom Assessment Form for measuring symptomatic improvement: performance in 87 myelofibrosis patients on a JAK1 and JAK2 inhibitor (INCB018424) clinical trialCancer2011117214869487721480207
   PubMed Web of Science ®Google Scholar
• VerstovsekSMesaRGotlibJConsistent benefit of ruxolitinib over placebo in spleen volume reduction and symptom improvement across subgroups and overall survival advantage: results from COMFORT-I [abstract]Blood201111821278
   Web of Science ®Google Scholar
• AaronsonNKAhmedzaiSBergmanBThe European Organization for Research and Treatment of Cancer QLQ-C30: a quality-of-life instrument for use in international clinical trials in oncologyJ Natl Cancer Inst19938553653768433390
   PubMed Web of Science ®Google Scholar
• MesaRKantarjianHShieldsAResults using the Modified Myelofibrosis Symptom Assessment Form (MFSAF v2.0) in COMFORT- I: a randomized, double-blind phase III trial of JAK1/2 inhibitor ruxolitinib vs placebo in myelofibrosis (MF) [abstract]Haematologica2011966 Suppl 20912
   Google Scholar
• MesaRGotlibJGuptaVAssociations between improvements in myelofibrosis (MF) symptoms and quality of life measures with splenomegaly reduction in COMFORT-I: a randomized, double-blind, phase III trial of the JAK1 and JAK2 inhibitor ruxolitinib versus placebo in patients with MF [abstract]Blood201111821384221828130
   PubMed Web of Science ®Google Scholar
• HarrisonCKiladjianJGisslingerHRuxolitinib provides reductions in splenomegaly across subgroups: an analysis of spleen response in the COMFORT-II study [abstract]Blood201111821279
   Web of Science ®Google Scholar
• HarrisonCKiladjianJKathrin Al-AliHHealth-related quality of life and symptoms in myelofibrosis patients treated with ruxolitinib versus best available therapy [abstract]Blood20111182179521642596
   PubMed Web of Science ®Google Scholar
• CellaDWebsterKCashyJDevelopment of a measure of health-related quality of life for non-Hodgkin’s lymphoma clinical research: the Functional Assessment of Cancer Therapy – Lymphoma (FACT-Lym) [abstract]Blood200510611750
   Web of Science ®Google Scholar
• MesaRVerstovsekSCervantesFComparison of the efficacy of placebo and best available therapy for the treatment of myelofibrosis in the COMFORT studies [abstract]Blood2011118211753
   Web of Science ®Google Scholar