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
2018
Immunosuppressive therapy in myelodysplastic syndromes: a borrowed therapy in search of the right place
Shallis RM, Chokr N, Stahl M, Pine AB, Zeidan AM. Immunosuppressive therapy in myelodysplastic syndromes: a borrowed therapy in search of the right place. Expert Review Of Hematology 2018, 11: 715-726. PMID: 30024293, DOI: 10.1080/17474086.2018.1503049.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsConceptsImmunosuppressive therapyMyelodysplastic syndromeImmune pathwaysManagement of MDSTreatment of MDSClonal hematopoietic stem cell disordersMeaningful clinical activityAdaptive immune pathwaysHematopoietic stem cell disordersStem cell disordersImmune dysregulationDisease coursePeripheral cytopeniasClinical benefitImmune activationTherapeutic optionsAplastic anemiaClinical activityHematologic diseasesCell disordersClinical experienceContinued clarificationLeukemic progressionTherapyPatients