Featured Publications
Cost 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 agents
2023
Cost-Effectiveness of Long-Term Prophylaxis with Plasma-Derived Vs. Recombinant Von Willebrand Factor in Severe Von Willebrand Disease
Waldron C, Ito S, Wang D, Allen C, Viswanathan G, Bona R, Cuker A, Goshua G. Cost-Effectiveness of Long-Term Prophylaxis with Plasma-Derived Vs. Recombinant Von Willebrand Factor in Severe Von Willebrand Disease. Blood 2023, 142: 3698. DOI: 10.1182/blood-2023-188018.Peer-Reviewed Original ResearchSevere von Willebrand diseaseLong-term prophylaxisIncremental net monetary benefitVon Willebrand diseasePlasma-derived von Willebrand factorIncremental cost-effectiveness ratioNet monetary benefitVon Willebrand factorProbabilistic sensitivity analysesDeterministic sensitivity analysesSevere haemophiliaWillebrand factorRecombinant von Willebrand factorWillebrand diseasePhase 3 clinical trialsHealth resource utilizationFactor replacement therapyFrequency of infusionsSubset of patientsCost of prophylaxisType 3 von Willebrand diseaseFactor replacement productsSimilar significant improvementsSevere phenotypeNon-significant reduction