2023
Moving toward disease modification in polycythemia vera
Bewersdorf J, How J, Masarova L, Bose P, Pemmaraju N, Mascarenhas J, Rampal R. Moving toward disease modification in polycythemia vera. Blood 2023, 142: 1859-1870. PMID: 37729609, DOI: 10.1182/blood.2023021503.Peer-Reviewed Original ResearchConceptsAcute myeloid leukemiaProgression to myelofibrosisPolycythemia veraCytoreductive therapyBCR-ABL1-negative myeloproliferative neoplasmsLow-risk PVLow-risk diseaseHistory of thrombosisHigh-risk diseaseDisease-modifying treatment modalitiesAssociated with higher ratesEvolving treatment landscapeClinically meaningful outcome measuresDisease-related symptomsInterferon alfaMyeloproliferative neoplasmsThromboembolic eventsPatient ageMyeloid leukemiaTreatment modalitiesIFN-a2bTherapeutic phlebotomyActivating mutationsVasomotor symptomsDisease course
2022
Hitting the brakes on accelerated and blast-phase myeloproliferative neoplasms: current and emerging concepts
Bewersdorf J, Rampal R. Hitting the brakes on accelerated and blast-phase myeloproliferative neoplasms: current and emerging concepts. Hematology 2022, 2022: 218-224. PMID: 36485103, PMCID: PMC9820986, DOI: 10.1182/hematology.2022000341.Peer-Reviewed Original ResearchConceptsMyeloproliferative neoplasmsBlast-phase MPNBlast-phase myeloproliferative neoplasmsAllogeneic hematopoietic cell transplantationBCR-ABL-negative myeloproliferative neoplasmsStages of clinical developmentMedian overall survivalHigh-risk molecular featuresHematopoietic cell transplantationCurative therapeutic modalityPrognosis of patientsAcute myeloid leukemiaMinority of patientsClinical trial enrollmentMPN-BPMyeloid blastsCurative intentHypomethylating agentsOverall survivalCell transplantationPeripheral bloodMyeloid leukemiaPalliative treatmentBone marrowClinical development
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 neoplasmsPatientsFedratinib hydrochloride to treat intermediate-2 or high-risk primary or secondary myelofibrosis.
Schiffer M, Kowalski A, Zhao J, Bewersdorf JP, Lewis RS, Zeidan AM. Fedratinib hydrochloride to treat intermediate-2 or high-risk primary or secondary myelofibrosis. Drugs Of Today 2020, 56: 755-768. PMID: 33332482, DOI: 10.1358/dot.2020.56.12.3230206.Peer-Reviewed Original ResearchConceptsSecondary myelofibrosisClinical holdSelective Janus kinase 2 inhibitorNovel oral agentsCommon adverse effectsClinical trial dataFurther drug developmentGastrointestinal symptomsLiver transaminasesOral agentsSymptom burdenJanus kinase 2 inhibitorDrug AdministrationMyeloproliferative neoplasmsTrial dataU.S. FoodAdverse effectsKinase 2 inhibitorPhase IIDrug developmentMyelofibrosisSignificant reductionFurther investigationFedratinibSplenomegaly