2024
Cost-Effectiveness of Eltrombopag Plus Immunosuppressive Therapy Versus Immunosuppressive Therapy Alone in Adults with Severe Aplastic Anemia in the United States
Potnis K, Ito S, Patel K, Shallis R, Podoltsev N, Kewan T, Stempel J, Mendez L, Huntington S, Stahl M, Zeidan A, Bewersdorf J, Goshua G. Cost-Effectiveness of Eltrombopag Plus Immunosuppressive Therapy Versus Immunosuppressive Therapy Alone in Adults with Severe Aplastic Anemia in the United States. Blood 2024, 144: 3644. DOI: 10.1182/blood-2024-204312.Peer-Reviewed Original ResearchSevere aplastic anemiaTreatment of severe aplastic anemiaQuality-adjusted life yearsAplastic anemiaIncremental net monetary benefitDeterministic sensitivity analysisProbabilistic sensitivity analysesImmunosuppressive therapyNewly diagnosed severe aplastic anemiaCost-effectiveness of eltrombopagOral thrombopoietin receptor agonistPatients treated with eltrombopagUntreated severe aplastic anemiaHematopoietic stem cell transplantationCost-effective therapeutic strategyDevelopment of adverse eventsLonger-term follow-up dataRACE trialsLater-line therapyThrombopoietin receptor agonistsPhase I/II trialSecond-line therapyStem cell transplantationU.S. payer perspectiveFirst-line treatment
2020
Novel and combination therapies for polycythemia vera and essential thrombocythemia: the dawn of a new era
Bewersdorf JP, Zeidan AM. Novel and combination therapies for polycythemia vera and essential thrombocythemia: the dawn of a new era. Expert Review Of Hematology 2020, 13: 1189-1199. PMID: 33076714, PMCID: PMC7722191, DOI: 10.1080/17474086.2020.1839887.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsMeSH KeywordsBone MarrowClinical Trials as TopicCombined Modality TherapyDNA MethylationDrugs, InvestigationalForecastingHistone CodeHumansHydroxyureaInflammationInterferon-alphaJanus Kinase 2Molecular Diagnostic TechniquesMolecular Targeted TherapyMutation, MissenseNitrilesPolycythemia VeraPyrazolesPyrimidinesSignal TransductionTherapies, InvestigationalThrombocythemia, EssentialThrombophiliaThrombosisTumor MicroenvironmentConceptsEssential thrombocythemiaPolycythemia veraNovel agentsPV patientsTreatment selectionHigh-risk essential thrombocythemiaAberrant immune regulationFirst-line treatmentDisease-modifying potentialRecent clinical trialsNegative myeloproliferative neoplasmsBone marrow microenvironmentImmune pathobiologyCombination therapyClinical trialsClinical studiesImmune regulationPV pathogenesisConference abstractsBCR-ABL1Individualized approachMolecular testingClonal proliferationMyeloproliferative neoplasmsPatients