Featured Publications
Cost‐effectiveness of sutimlimab in cold agglutinin disease
Ito S, Wang D, Purcell A, Chetlapalli K, Lee A, Cuker A, Goshua G. Cost‐effectiveness of sutimlimab in cold agglutinin disease. American Journal Of Hematology 2024, 99: 1475-1484. PMID: 38733355, DOI: 10.1002/ajh.27358.Peer-Reviewed Original ResearchPrimary cold agglutinin diseaseCold agglutinin diseaseStandard-of-careIncremental cost-effectiveness ratioUS willingness-to-pay thresholdRare autoimmune hemolytic anemiaPhase 3 studyWillingness-to-pay thresholdsAutoimmune hemolytic anemiaTime-limited treatmentHuman monoclonal antibodyCold-reactive antibodiesBinding to red blood cellsFollow-up dataCost-effectiveness ratioComplement-mediated hemolysisDeterministic sensitivity analysisHealth resource utilizationProbabilistic sensitivity analysesTransfusion-dependentClinical remissionTransfusion supportImprove quality of lifeRed blood cellsHemolytic anemiaCancer research is not correlated with driver gene mutation burdens
Mendiratta G, Liarakos D, Tong M, Ito S, Ke E, Goshua G, Stites E. Cancer research is not correlated with driver gene mutation burdens. Med 2024, 5: 832-838.e4. PMID: 38908369, DOI: 10.1016/j.medj.2024.05.013.Peer-Reviewed Original ResearchCancer patient populationCancer researchCancer research effortsResearch allocation decisionsNational InstitutePatient populationResearch fundingBurdenBurden of mutationsFunding decisionsCancerGenetic driversGene mutation burdenFunding amountFundingGenetic drivers of cancerAllocation decisionsCancer-associated genesEpidemiologyDrivers of cancerMutational burdenBaselineEffortsFactorsBalance of prioritiesDecreasing alloimmunization‐specific mortality in sickle cell disease in the United States: Cost‐effectiveness of a shared transfusion resource
Ito S, Pandya A, Hauser R, Krishnamurti L, Stites E, Tormey C, Krumholz H, Hendrickson J, Goshua G. Decreasing alloimmunization‐specific mortality in sickle cell disease in the United States: Cost‐effectiveness of a shared transfusion resource. American Journal Of Hematology 2024, 99: 570-576. PMID: 38279581, DOI: 10.1002/ajh.27211.Peer-Reviewed Original ResearchSickle cell diseaseDelayed hemolytic transfusion reactionQuality-adjusted life expectancyAlloimmunized patientsPatient populationRed blood cell alloimmunizationCell diseaseCost-effective interventionMedical expenditure of patientsHealth system perspectiveExpenditure of patientsIncremental cost-effectiveness ratioHemolytic transfusion reactionsUnited StatesMarkov cohort simulationCost-effectiveAverage patient populationCost-effectiveness ratioBirth cohortAnalytical time horizonAntibody historyCohort simulationTransfusionTransfusion reactionsLife expectancyDistributional Cost-Effectiveness of Equity-Enhancing Gene Therapy in Sickle Cell Disease in the United States.
Goshua G, Calhoun C, Ito S, James L, Luviano A, Krishnamurti L, Pandya A. Distributional Cost-Effectiveness of Equity-Enhancing Gene Therapy in Sickle Cell Disease in the United States. Annals Of Internal Medicine 2023, 176: 779-787. PMID: 37247420, PMCID: PMC10370480, DOI: 10.7326/m22-3272.Peer-Reviewed Original ResearchCost-effectiveness of prophylaxis with recombinant vs plasma-derived VWF in severe von Willebrand disease in the United States
Waldron C, Ito S, Wang D, Allen C, Viswanathan G, Bona R, Cuker A, Goshua G. Cost-effectiveness of prophylaxis with recombinant vs plasma-derived VWF in severe von Willebrand disease in the United States. Blood 2024, 143: 2332-2335. PMID: 38635764, DOI: 10.1182/blood.2024024209.Peer-Reviewed Original ResearchCost-effectiveness of bevacizumab therapy in the care of patients with hereditary hemorrhagic telangiectasia
Wang D, Ito S, Waldron C, Butt A, Zhang E, Krumholz H, Al-Samkari H, Goshua G. Cost-effectiveness of bevacizumab therapy in the care of patients with hereditary hemorrhagic telangiectasia. Blood Advances 2024, 8: 2835-2845. PMID: 38537061, PMCID: PMC11176968, DOI: 10.1182/bloodadvances.2024012589.Peer-Reviewed Original ResearchIncremental net monetary benefitHereditary hemorrhagic telangiectasiaStandard-of-careBevacizumab therapyWillingness-to-payNet monetary benefitHemorrhagic telangiectasiaWillingness-to-pay thresholdsStandard-of-care strategiesCare of patientsCost-effectiveness analysisAnti-VEGF bevacizumabSystemic antiangiogenic therapyCurrent standard-of-careMonetary benefitsTime horizonFormulary placementHealthcare resource utilizationStandard of careLifetime time horizonPatient quality-of-lifeScenario analysisProbabilistic sensitivity analysesIV bevacizumabAnti-VEGFCost-effectiveness of rapid vs in-house vs send-out ADAMTS13 testing for immune thrombotic thrombocytopenic purpura
Allen C, Ito S, Butt A, Purcell A, Richmond R, Tormey C, Krumholz H, Cuker A, Goshua G. Cost-effectiveness of rapid vs in-house vs send-out ADAMTS13 testing for immune thrombotic thrombocytopenic purpura. Blood Advances 2024, 8: 2279-2289. PMID: 38502197, PMCID: PMC11116991, DOI: 10.1182/bloodadvances.2024012608.Peer-Reviewed Original ResearchImmune thrombotic thrombocytopenic purpuraPLASMIC scoreThrombotic thrombocytopenic purpuraThrombocytopenic purpuraADAMTS13 testingIncremental net monetary benefitPer-patient cost savingsTherapeutic plasma exchangeBase-case analysisMarkov cohort simulationProbabilistic sensitivity analysesAmount of QALYEmpirical therapyADAMTS13 assaysPlasma exchangeEmpirical treatmentCaplacizumabFRET-based assayPrimary outcomePatientsNet monetary benefitCohort simulationCost-effectiveness evaluationPurpuraTesting strategiesNapkin math can change the world
Goshua G. Napkin math can change the world. Science 2023, 382: 657-657. PMID: 37943923, DOI: 10.1126/science.adk8633.Peer-Reviewed Original ResearchCost‐effectiveness of prophylactic emicizumab versus prophylactic recombinant factor VIII in patients with moderate or mild hemophilia A without inhibitors in the United States
Potnis K, Viswanathan G, Bona R, Ito S, Kempton C, Pandya A, Krumholz H, Goshua G. Cost‐effectiveness of prophylactic emicizumab versus prophylactic recombinant factor VIII in patients with moderate or mild hemophilia A without inhibitors in the United States. American Journal Of Hematology 2023, 98: e247-e250. PMID: 37401660, DOI: 10.1002/ajh.27014.Peer-Reviewed Original ResearchApixaban at Apex? Aligning Drug Pricing With Value in Cancer-Associated Thrombosis.
Ito S, Goshua G. Apixaban at Apex? Aligning Drug Pricing With Value in Cancer-Associated Thrombosis. Annals Of Internal Medicine 2022, 176: 125-126. PMID: 36571840, DOI: 10.7326/m22-3404.Peer-Reviewed Original ResearchCost‐effectiveness of second‐line therapies in adults with chronic immune thrombocytopenia
Goshua G, Sinha P, Kunst N, Pischel L, Lee AI, Cuker A. Cost‐effectiveness of second‐line therapies in adults with chronic immune thrombocytopenia. American Journal Of Hematology 2022, 98: 122-130. PMID: 35147241, PMCID: PMC9365880, DOI: 10.1002/ajh.26497.Peer-Reviewed Original ResearchConceptsThrombopoietin receptor agonistsChronic immune thrombocytopeniaSecond-line therapyImmune thrombocytopeniaEarly useCurrent practice trendsProbabilistic sensitivity analysesCost-effective treatmentFirst cost-effectiveness analysisCost-effectiveness analysisU.S. health systemLack of evidenceEarly splenectomyHematology (ASH) guidelinesReceptor agonistCurrent guidelinesSplenectomyClinical practicePractice trendsTherapyHealth systemCost-effective strategyRituximabThrombocytopeniaAmerican SocietyCost-effectiveness of Tocilizumab in Severe Coronavirus Disease 2019: to See or Not to See
Pischel L, Goshua G. Cost-effectiveness of Tocilizumab in Severe Coronavirus Disease 2019: to See or Not to See. Clinical Infectious Diseases 2021, 73: 2119-2120. PMID: 33999992, PMCID: PMC8194546, DOI: 10.1093/cid/ciab459.Commentaries, Editorials and LettersAccurate accounting of caplacizumab cost effectiveness
Goshua G, Prasad V, Lee AI, Bendapudi PK. Accurate accounting of caplacizumab cost effectiveness. The Lancet Haematology 2021, 8: e315. PMID: 33894166, DOI: 10.1016/s2352-3026(21)00052-1.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsCost effectiveness of caplacizumab in acquired thrombotic thrombocytopenic purpura
Goshua G, Sinha P, Hendrickson J, Tormey C, Bendapudi PK, Lee AI. Cost effectiveness of caplacizumab in acquired thrombotic thrombocytopenic purpura. Blood 2021, 137: 969-976. PMID: 33280030, PMCID: PMC7918179, DOI: 10.1182/blood.2020006052.Peer-Reviewed Original ResearchMeSH KeywordsAdolescentAdultAgedClinical Trials, Phase II as TopicClinical Trials, Phase III as TopicCombined Modality TherapyCost-Benefit AnalysisDecision TreesDrug CostsDrug Therapy, CombinationFemaleFibrinolytic AgentsHemorrhageHumansImmunosuppressive AgentsLength of StayMaleMarkov ChainsMiddle AgedModels, EconomicMulticenter Studies as TopicPlasma ExchangePurpura, Thrombotic ThrombocytopenicRecurrenceRituximabSingle-Domain AntibodiesStandard of CareUnited StatesYoung AdultConceptsIncremental cost-effectiveness ratioThrombotic thrombocytopenic purpuraTherapeutic plasma exchangeVon Willebrand factorRelapse rateThrombocytopenic purpuraClinical trialsMajor randomized clinical trialsThrombotic microangiopathy leadingEnd-organ damageWillebrand factorPlatelet count recoveryRandomized clinical trialsHealth system costsOne-way sensitivity analysesCost-effectiveness ratioLife-threatening diseaseProbabilistic sensitivity analysesCost-effectiveness analysisHospital lengthCount recoveryPlasma exchangeTPE treatmentTTP patientsImmunomodulatory agentsReduced fixed dose tocilizumab 400 mg IV compared to weight-based dosing in critically ill patients with COVID-19: A before-after cohort study
Stukas S, Goshua G, Kinkade A, Grey R, Mah G, Biggs CM, Jamal S, Thiara S, Lau TTY, Piszczek J, Partovi N, Sweet DD, Lee AYY, Wellington CL, Sekhon MS, Chen LYC. Reduced fixed dose tocilizumab 400 mg IV compared to weight-based dosing in critically ill patients with COVID-19: A before-after cohort study. The Lancet Regional Health - Americas 2022, 11: 100228. PMID: 35345649, PMCID: PMC8941850, DOI: 10.1016/j.lana.2022.100228.Peer-Reviewed Original ResearchDoses of tocilizumabCohort studyClinical outcomesIll patientsIll COVID-19 patientsCOVID-19Interleukin-6 inhibitorsSevere COVID-19Weight-based dosingCOVID-19 patientsDrug shortagesNet monetary benefitMedian CRPControl cohortTocilizumabPatientsAssumed willingnessDexamethasoneComparable reductionMortalityPhysiological responsesSignificant differencesOnly groupDoseDosesSplenectomy reappraised: Bridging the gap in immune cytopenia treatment paradigms
Waldron C, Goshua G. Splenectomy reappraised: Bridging the gap in immune cytopenia treatment paradigms. Journal Of Internal Medicine 2023, 295: 120-122. PMID: 38044059, DOI: 10.1111/joim.13756.Peer-Reviewed Original ResearchEndotheliopathy in COVID-19-associated coagulopathy: evidence from a single-centre, cross-sectional study
Goshua G, Pine AB, Meizlish ML, Chang CH, Zhang H, Bahel P, Baluha A, Bar N, Bona RD, Burns AJ, Dela Cruz CS, Dumont A, Halene S, Hwa J, Koff J, Menninger H, Neparidze N, Price C, Siner JM, Tormey C, Rinder HM, Chun HJ, Lee AI. Endotheliopathy in COVID-19-associated coagulopathy: evidence from a single-centre, cross-sectional study. The Lancet Haematology 2020, 7: e575-e582. PMID: 32619411, PMCID: PMC7326446, DOI: 10.1016/s2352-3026(20)30216-7.Peer-Reviewed Original ResearchMeSH KeywordsAdultAgedAged, 80 and overBetacoronavirusBiomarkersBlood Coagulation DisordersCoronavirus InfectionsCOVID-19Critical IllnessCross-Sectional StudiesEndothelium, VascularFemaleFollow-Up StudiesHumansIntensive Care UnitsMaleMiddle AgedPandemicsPneumonia, ViralPrognosisSARS-CoV-2Vascular DiseasesYoung AdultConceptsCOVID-19-associated coagulopathyNon-ICU patientsIntensive care unitKaplan-Meier analysisSoluble P-selectinCross-sectional studyPlatelet activationHospital dischargeICU patientsSoluble thrombomodulinEndothelial cellsVWF antigenCOVID-19P-selectinSingle-center cross-sectional studyLaboratory-confirmed COVID-19Medical intensive care unitSevere acute respiratory syndrome coronavirus 2 (SARS-CoV-2) pathogenesisVon Willebrand factor antigenSoluble thrombomodulin concentrationsVWF antigen concentrationEndothelial cell injurySoluble CD40 ligandMicrovascular complicationsAdult patients
2024
Cost-effectiveness of iptacopan in paroxysmal nocturnal hemoglobinuria
Ito S, Chetlapalli K, Wang D, Potnis K, Richmond R, Krumholz H, Lee A, Cuker A, Goshua G. Cost-effectiveness of iptacopan in paroxysmal nocturnal hemoglobinuria. Blood 2024 PMID: 39374533, DOI: 10.1182/blood.2024025176.Peer-Reviewed Original ResearchStandard-of-careParoxysmal nocturnal hemoglobinuriaIncremental net monetary benefitNocturnal hemoglobinuriaComplement-mediated hemolytic anemiaTreating paroxysmal nocturnal hemoglobinuriaComplement C5 inhibitor eculizumabPhase 3 studyQuality-adjusted life expectancyRare blood disorderComprehensive cost-effectiveness analysisProbabilistic sensitivity analysesCost-saving thresholdsC5 inhibitor eculizumabNet monetary benefitPersistent anemiaIptacopanExtravascular hemolysisIntravenous infusionMonotherapyHemolytic anemiaAnemia resolutionC5 inhibitionFDA approvalPrimary outcomeVaccine effectiveness of 3rd generation mpox vaccines against mpox and disease severity: A systematic review and meta-analysis
Pischel L, Martini B, Yu N, Cacesse D, Tracy M, Kharbanda K, Ahmed N, Patel K, Grimshaw A, Malik A, Goshua G, Omer S. Vaccine effectiveness of 3rd generation mpox vaccines against mpox and disease severity: A systematic review and meta-analysis. Vaccine 2024, 42: 126053. PMID: 38906763, DOI: 10.1016/j.vaccine.2024.06.021.Peer-Reviewed Original ResearchPost-exposure prophylaxisMVA-BNVaccine effectivenessMeta-analysisTime of administrationNewcastle-Ottawa ScaleRisk of publication biasMeasuring vaccine efficacyImmortal time biasImmunogenicity studiesNewcastle-OttawaWeb of ScienceVaccine efficacyAnimal studiesEgger's testDisease severityFunnel plotPrimary searchDosePublication biasStudy qualityVaccineSystematic reviewVaccination timingMpox outbreakSevere, Refractory Primary Warm Autoimmune Hemolytic Anemia Requiring 90 Erythrocyte Transfusions.
Namineni N, Waldron C, Tormey C, Goshua G. Severe, Refractory Primary Warm Autoimmune Hemolytic Anemia Requiring 90 Erythrocyte Transfusions. Annals Of Internal Medicine Clinical Cases 2024, 3 PMID: 38725710, PMCID: PMC11081177, DOI: 10.7326/aimcc.2023.1141.Peer-Reviewed Original ResearchWarm autoimmune hemolytic anemiaAutoimmune hemolytic anemiaHemolytic anemiaSevere warm autoimmune hemolytic anemiaMonths of follow-upHealthy 60-year-old manTherapeutic plasma exchangeHospital day 1Erythrocyte transfusionTransfusion-dependentRefractory diseasePlasma exchangeImmune globulinHospital stayFollow-upDay 1AnemiaHospitalRituximabReticulocytopeniaRemissionSplenectomyTransfusionPatientsStay