2022
Cost‐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 Society
2021
Cost-Effectiveness of Second-Line Therapies in Adult Patients with Chronic Immune Thrombocytopenia
Goshua G, Sinha P, Pischel L, Lee A, Cuker A. Cost-Effectiveness of Second-Line Therapies in Adult Patients with Chronic Immune Thrombocytopenia. Blood 2021, 138: 751. DOI: 10.1182/blood-2021-144819.Peer-Reviewed Original ResearchIncremental cost-effectiveness ratioChronic immune thrombocytopeniaOne-way deterministic sensitivity analysisPost-splenectomy sepsisTPO-RATPO-RA therapyImmune thrombocytopeniaOverall response rateTreatment pathwaysProbabilistic sensitivity analysesDeterministic sensitivity analysesResponse rateAdverse eventsASH guidelinesStandard second-line treatment optionSecond-line treatment optionSecond-line treatment strategiesEarly useComplete response rateSecond-line therapySevere septic shockThrombopoietin receptor agonistsSeptic shock mortalityHealth system perspectiveCost of surgery
2015
Genes Influencing the Development and Severity of Chronic ITP Identified through Whole Exome Sequencing
Despotovic J, Polfus L, Flanagan J, Bennett C, Lambert M, Neunert C, Kumar M, Klaassen R, Thornburg C, Jeng M, Recht M, Kirk S, Thompson A, Nugent D, Neufeld E, Bussel J, Boerwinkle E, Grace R. Genes Influencing the Development and Severity of Chronic ITP Identified through Whole Exome Sequencing. Blood 2015, 126: 73. DOI: 10.1182/blood.v126.23.73.73.Peer-Reviewed Original ResearchChronic immune thrombocytopeniaWhole-exome sequencingEntity's Board of DirectorsPathophysiology of immune thrombocytopeniaImmune thrombocytopeniaImmune thrombocytopenia casesTGF-b signalingPhenotypic dataExome sequencingTGF-bCellular immunityGene-based testsWhole-exome sequencing analysisITP susceptibilityCases compared to controlsSecond line therapyImmune thrombocytopenia therapiesPlatelet count >T-cell abnormalitiesRegulatory T cellsT cell numbersPrimary immune thrombocytopeniaDisease severityCD34+ cellsImmune thrombocytopenia pathogenesis
2014
PETIT and PETIT 2: Treatment with Eltrombopag in 171 Children with Chronic Immune Thrombocytopenia (ITP)
Bussel J, Grainger J, de Miguel P, Despotovic J, Locatelli F, Chotsampancharoen T, Donyush E, Navarro J, Pongtanakul B, Komvilaisak P, Sosothikul D, Blanchette V, Drelichman G, Krishnamurti L, Sirachainan N, Connor P, David M, Holzhauer S, Lebedev V, Lemons R, Pospisilova D, Ramenghi U, Boayue K, Matthews D, Marcello L, Iyengar M, Chan G, Chagin K, Theodore D, Bakshi K, Bailey C. PETIT and PETIT 2: Treatment with Eltrombopag in 171 Children with Chronic Immune Thrombocytopenia (ITP). Blood 2014, 124: 1450. DOI: 10.1182/blood.v124.21.1450.1450.Peer-Reviewed Original ResearchChronic immune thrombocytopeniaImmune thrombocytopeniaThrombopoietin receptor agonistsAdverse eventsPlatelet count responsePlatelet countStudy treatmentITP medicationsSerious AEsPBO groupRandomized periodX ULNReceptor agonistRandomized phaseTreatment groupsMedian average daily doseOral thrombopoietin receptor agonistUpper respiratory tract infectionMost subjectsAdvisory CommitteeDose of eltrombopagEfficacy of eltrombopagPediatric immune thrombocytopeniaPre-existing cataractCommon adverse events
2011
Response to steroids predicts response to rituximab in pediatric chronic immune thrombocytopenia
Grace R, Bennett C, Ritchey A, Jeng M, Thornburg C, Lambert M, Neier M, Recht M, Kumar M, Blanchette V, Klaassen R, Buchanan G, Kurth M, Nugent D, Thompson A, Stine K, Kalish L, Neufeld E. Response to steroids predicts response to rituximab in pediatric chronic immune thrombocytopenia. Pediatric Blood & Cancer 2011, 58: 221-225. PMID: 21674758, PMCID: PMC3863944, DOI: 10.1002/pbc.23130.Peer-Reviewed Original ResearchMeSH KeywordsAdolescentAntibodies, Monoclonal, Murine-DerivedAntineoplastic AgentsChildChild, PreschoolChronic DiseaseCohort StudiesFemaleFollow-Up StudiesGlucocorticoidsHumansImmunoglobulins, IntravenousImmunologic FactorsInfantInfant, NewbornLongitudinal StudiesMalePlatelet CountPrednisoneProspective StudiesRetrospective StudiesRituximabThrombocytopeniaTreatment OutcomeConceptsResponse to rituximabResponse to steroidsSecondary immune thrombocytopeniaChronic immune thrombocytopeniaImmune thrombocytopeniaPredictor of response to rituximabPediatric chronic immune thrombocytopeniaRefractory to other therapiesPositive response to steroidsPredictors of therapy responseTreated with rituximabWeeks of rituximabPediatric immune thrombocytopeniaDays of steroidsOverall response rateTreatment response dataLocal IRB approvalTherapy responseRituximabTreatment responseMultivariate predictorsIRB approvalMultivariate analysisTreatment choiceEvaluate univariate
2010
The North American Chronic Immune Thrombocytopenia Registry (NACIR): Demographics and Treatment Responses
Grace R, Neufeld E, Ritchey A, Kumar M, Jeng M, Blanchette V, Klaassen R, Buchanan G, Kurth M, Nugent D, Thompson A, Thornburg C, Lambert M, Neier M, Recht M, Stine K, Kalish L, Bennett C. The North American Chronic Immune Thrombocytopenia Registry (NACIR): Demographics and Treatment Responses. Blood 2010, 116: 2509. DOI: 10.1182/blood.v116.21.2509.2509.Peer-Reviewed Original ResearchChronic immune thrombocytopeniaDirect anti-globulin testAcute immune thrombocytopeniaImmune thrombocytopeniaSecondary immune thrombocytopeniaEvans syndromeITP diagnosisAnti-DPlatelet countClinical diagnosis of immune thrombocytopeniasMedian duration of follow-upDiagnosis of immune thrombocytopeniaPediatric chronic immune thrombocytopeniaShort course of steroidsPredictors of response to treatmentAged 6 months-18 yearsDuration of follow-upPositive direct anti-globulin testImmune Thrombocytopenia diagnosisPlatelet count responseResponse to IVIGResponse to splenectomyCourse of steroidsPrimary immune thrombocytopeniaMedian platelet countResponse to Steroids Predicts Response to Rituximab In Pediatric Chronic Immune Thrombocytopenia.
Grace R, Bennett C, Ritchey A, Jeng M, Thornburg C, Lambert M, Neier M, Recht M, Kumar M, Blanchette V, Klaassen R, Buchanan G, Kurth M, Nugent D, Thompson A, Stine K, Kalish L, Neufeld E. Response to Steroids Predicts Response to Rituximab In Pediatric Chronic Immune Thrombocytopenia. Blood 2010, 116: 3681. DOI: 10.1182/blood.v116.21.3681.3681.Peer-Reviewed Original ResearchPredictor of response to rituximabResponse to rituximabSecondary immune thrombocytopeniaResponse to steroidsChronic immune thrombocytopeniaDiagnosis of immune thrombocytopeniaImmune thrombocytopeniaEvans syndromeITP diagnosisUnivariate predictorsTreatment responseClinical diagnosis of immune thrombocytopeniasPediatric chronic immune thrombocytopeniaPredicting response to rituximabPatients treated with rituximabRefractory to other therapiesResponse to prior treatmentAged 6 months-18 yearsPredictors of therapy responseAcute immune thrombocytopeniaImmune Thrombocytopenia diagnosisWeeks of rituximabTreated with rituximabMedian platelet countPediatric immune thrombocytopenia
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