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Review

Critical appraisal of the role of ruxolitinib in myeloproliferative neoplasm-associated myelofibrosis

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Pages 1091-1102 | Published online: 18 May 2015

References

  • MesaRAGreenABarosiGVerstovsekSVardimanJGaleRPMPN-associated myelofibrosis (MPN-MF)Leuk Res2011351121320684988
  • TefferiAPrimary myelofibrosis: 2014 update on diagnosis, risk-stratification, and managementAm J Hematol201489991592525124313
  • KralovicsRPassamontiFBuserASA gain-of-function mutation of JAK2 in myeloproliferative disordersN Engl J Med2005352171779179015858187
  • BaxterEJScottLMCampbellPJAcquired mutation of the tyrosine kinase JAK2 in human myeloproliferative disordersLancet200536594641054106115781101
  • JamesCUgoVLe CouédicJPA unique clonal JAK2 mutation leading to constitutive signalling causes polycythaemia veraNature200543470371144114815793561
  • LevineRLWadleighMCoolsJActivating mutation in the tyrosine kinase JAK2 in polycythemia vera, essential thrombocythemia, and myeloid metaplasia with myelofibrosisCancer Cell20057438739715837627
  • PikmanYLeeBHMercherTMPLW515L is a novel somatic activating mutation in myelofibrosis with myeloid metaplasiaPLoS Med200637e27016834459
  • NangaliaJMassieCEBaxterEJSomatic CALR mutations in myeloproliferative neoplasms with nonmutated JAK2N Engl J Med2013369252391240524325359
  • KlampflTGisslingerHHarutyunyanASSomatic mutations of calreticulin in myeloproliferative neoplasmsN Engl J Med2013369252379239024325356
  • CervantesFDupriezBPereiraANew prognostic scoring system for primary myelofibrosis based on a study of the International Working Group for Myelofibrosis Research and TreatmentBlood2009113132895290118988864
  • PassamontiFCervantesFVannucchiAMA dynamic prognostic model to predict survival in primary myelofibrosis: a study by the IWG-MRT (International Working Group for Myeloproliferative Neoplasms Research and Treatment)Blood201011591703170820008785
  • GangatNCaramazzaDVaidyaRDIPSS plus: a refined Dynamic International Prognostic Scoring System for primary myelofibrosis that incorporates prognostic information from karyotype, platelet count, and transfusion statusJ Clin Oncol201129439239721149668
  • GuglielmelliPLashoTLRotunnoGThe number of prognostically detrimental mutations and prognosis in primary myelofibrosis: an international study of 797 patientsLeukemia20142891804181024549259
  • TefferiAGuglielmelliPLashoTLCALR and ASXL1 mutations-based molecular prognostication in primary myelofibrosis: an international study of 570 patientsLeukemia20142871494150024496303
  • BarbuiTBarosiGBirgegardGPhiladelphia-negative classical myeloproliferative neoplasms: critical concepts and management recommendations from European LeukemiaNetJ Clin Oncol201129676177021205761
  • TefferiACervantesFMesaRRevised response criteria for myelofibrosis: International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) and European LeukemiaNet (ELN) consensus reportBlood201312281395139823838352
  • EmanuelRMDueckACGeyerHLMyeloproliferative neoplasm (MPN) symptom assessment form total symptom score: prospective international assessment of an abbreviated symptom burden scoring system among patients with MPNsJ Clin Oncol201230334098410323071245
  • Quintás-CardamaAVaddiKLiuPPreclinical characterization of the selective JAK1/2 inhibitor INCB018424: therapeutic implications for the treatment of myeloproliferative neoplasmsBlood2010115153109311720130243
  • HarrisonCKiladjianJJAl-AliHKJAK inhibition with ruxolitinib versus best available therapy for myelofibrosisN Engl J Med2012366978779822375970
  • DeisserothAKaminskasEGrilloJU.S. Food and Drug Administration approval: ruxolitinib for the treatment of patients with intermediate and high-risk myelofibrosisClin Cancer Res201218123212321722544377
  • GuptaVGotlibJRadichJPJanus kinase inhibitors and allogeneic stem cell transplantation for myelofibrosisBiol Blood Marrow Transplant20142091274128124680977
  • JaekelNBehreGBehningAAllogeneic hematopoietic cell transplantation for myelofibrosis in patients pretreated with the JAK1 and JAK2 inhibitor ruxolitinibBone Marrow Transplant201449217918424292520
  • StübigTAlchalbyHDitschkowskiMJAK inhibition with ruxolitinib as pretreatment for allogeneic stem cell transplantation in primary or post-ET/PV myelofibrosisLeukemia20142881736173824569777
  • PieriLPaoliCArenaUA phase 2 study of ruxolitinib in patients with splanchnic vein thrombosis associated with myeloproliferative neoplasm: a study from the AGIMM groupBlood2014 Abstractn.72125, ASH
  • WadeRRoseMNeilsonARRuxolitinib for the treatment of myelofibrosis: a NICE single technology appraisalPharmacoeconomics2013311084185223996108
  • GuyattGHOxmanADVistGEGRADE: an emerging consensus on rating quality of evidence and strength of recommendationsBMJ2008336765092492618436948
  • GuyattGHOxmanADKunzRGoing from evidence to recommendationsBMJ200833676521049105118467413
  • VerstovsekSMesaRAGotlibJA double-blind, placebo-controlled trial of ruxolitinib for myelofibrosisN Engl J Med2012366979980722375971
  • VerstovsekSMesaRAGotlibJEfficacy, safety and survival with ruxolitinib in patients with myelofibrosis: results of a median 2-year follow-up of COMFORT-IHaematologica201398121865187124038026
  • CervantesFVannucchiAMKiladjianJJThree-year efficacy, safety, and survival findings from COMFORT-II, a phase 3 study comparing ruxolitinib with best available therapy for myelofibrosisBlood2013122254047405324174625
  • GuglielmelliPBiamonteFRotunnoGImpact of mutational status on outcomes in myelofibrosis patients treated with ruxolitinib in the COMFORT-II studyBlood2014123142157216024458439
  • MesaRAShieldsAHareTProgressive burden of myelofibrosis in untreated patients: assessment of patient-reported outcomes in patients randomized to placebo in the COMFORT-I studyLeuk Res201337891191623684482
  • HarrisonCNMesaRAKiladjianJJHealth-related quality of life and symptoms in patients with myelofibrosis treated with ruxolitinib versus best available therapyBr J Haematol2013162222923923672349
  • VerstovsekSMesaRAGotlibJThe clinical benefit of ruxolitinib across patient subgroups: analysis of a placebo-controlled, Phase III study in patients with myelofibrosisBr J Haematol2013161450851623480528
  • MesaRAGotlibJGuptaVEffect of ruxolitinib therapy on myelofibrosis-related symptoms and other patient-reported outcomes in COMFORT-I: a randomized, double-blind, placebo-controlled trialJ Clin Oncol201331101285129223423753
  • 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
  • VerstovsekSKantarjianHMesaRASafety and efficacy of INCB018424, a JAK1 and JAK2 inhibitor, in myelofibrosisN Engl J Med2010363121117112720843246
  • TalpazMPaquetteRAfrinLInterim analysis of safety and efficacy of ruxolitinib in patients with myelofibrosis and low platelet countsJ Hematol Oncol2013618124283202
  • HigginsJAltmanDHigginsJGreenSChapter 8: assessing risk of bias in included studiesCochrane Handbook for Systematic Reviews of Interventions version 50Chichester, UKJohn Wiley & Sons2008
  • PapaconstantinouCKrischerJPAn automated patient registration and treatment randomization systemJ Med Syst19951964454568750375
  • KuznetsovaOMTymofyeyevYExpansion of the modified Zelen’s approach randomization and dynamic randomization with partial block supplies at the centers to unequal allocationContemp Clin Trials201132696297221888990
  • BoutronIGuittetLEstellatCMoherDHróbjartssonARavaudPReporting methods of blinding in randomized trials assessing nonpharmacological treatmentsPLoS Med200742e6117311468
  • HróbjartssonAForfangEHaahrMTAls-NielsenBBrorsonSBlinded trials taken to the test: an analysis of randomized clinical trials that report tests for the success of blindingInternl J Epidemiol2007363654663
  • WoodLEggerMGluudLLEmpirical evidence of bias in treatment effect estimates in controlled trials with different interventions and outcomes: meta-epidemiological studyBMJ2008336764460160518316340
  • HigginsJPTAltmanDGSterneJACChapter 8: assessing risk of bias in included studiesHigginsJPTGreenSCochrane handbook for systematic reviews of interventions (Version 5.1.0)2011 Retrieved from: www.cochrane-handbook.org
  • LittleRJD’AgostinoRCohenMLThe prevention and treatment of missing data in clinical trialsN Engl J Med2012367141355136023034025
  • HernánMAHernández-DíazSRobinsJMRandomized trials analyzed as observational studiesAnn Inten Med20131598500502
  • BarosiGVannucchiAMDe StefanoVIdentifying and addressing unmet clinical needs in Ph-neg classical myeloproliferative neoplasms: a consensus-based SIE, SIES, GITMO position paperLeuk Res201438215516024378116
  • BarosiGTefferiABessesCClinical end points for drug treatment trials in BCR-ABL1-negative classic myeloproliferative neoplasms: consensus statements from European LeukemiaNET (ELN) and International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT)Leukemia2014291202625151955
  • GuyattGHOxmanADKunzRGRADE guidelines 6. Rating the quality of evidence – imprecisionJ Clin Epidemiol201164121283129321839614
  • ReillyJTMcMullinMFBeerPABritish Committee for Standards in Haematology. Guideline for the diagnosis and management of myelofibrosisBr J Haematol2012158445347122651893
  • LiuYLitièreSde VriesEGThe role of response evaluation criteria in solid tumour in anticancer treatment evaluation: results of a survey in the oncology communityEur J Cancer201450226026624239447
  • WundtWMEthik. Eine Untersuchung der Thatsachen und Gesetze des sittlischen LebensStuttgart, GermanyF. Enke1886
  • WoodAJA proposal for radical changes in the drug-approval processN Engl J Med2006355661862316899784
  • De PalmaRLiberatiACicconeGDeveloping clinical recommendations for breast, colorectal, and lung cancer adjuvant treatments using the GRADE system: a study from the Programma Ricerca e Innovazione Emilia Romagna Oncology Research GroupJ Clin Oncol20082671033103918309939
  • LeeSCFeenstraJGeorghiouPRPneumocystis jiroveci pneumonitis complicating ruxolitinib therapyBMJ Case Rep2014piibcr2014204950
  • TongLXJacksonJKerstetterJWorswickSDReactivation of herpes simplex virus infection in a patient undergoing ruxolitinib treatmentJ Am Acad Dermatol2014703e59e6024528917
  • HeineABrossartPWolfDRuxolitinib is a potent immunosuppressive compound: is it time for anti-infective prophylaxis?Blood2013122233843384424288410
  • ShenCHHwangCEChenYYChenCCHepatitis B virus reactivation associated with ruxolitinibAnn Hematol20149361075107624173089
  • MassaMRostiVCampanelliRFoisGBarosiGRapid and long-lasting decrease of T-regulatory cells in patients with myelofibrosis treated with ruxolitinibLeukemia201428244945124145312
  • GoldbergRAReichelEOshryLJBilateral toxoplasmosis retinitis associated with ruxolitinibN Engl J Med2013369768168323944322
  • CaocciGMurgiaFPoddaLSolinasAAtzeniSLa NasaGReactivation of hepatitis B virus infection following ruxolitinib treatment in a patient with myelofibrosisLeukemia201428122522723929216
  • WathesRMouleSMilojkovicDProgressive multifocal leukoencephalopathy associated with ruxolitinibN Engl J Med2013369219719823841743
  • HeineAHeldSADaeckeSNThe JAK-inhibitor ruxolitinib impairs dendritic cell function in vitro and in vivoBlood201312271192120223770777
  • WyshamNGSullivanDRAlladaGAn opportunistic infection associated with ruxolitinib, a novel janus kinase 1, 2 inhibitorChest201314351478147923648912