2024
1896-LB: Clinical Features and C-Peptide Levels Associated with Diabetic Ketoacidosis in Patients with Type 2 Diabetes on Dapagliflozin vs. Placebo—Insights from DECLARE-TIMI 58
MI KANG Y, MELLONI G, CAHN A, RAZ I, MOURA F, BHATT D, INZUCCHI S, LEITER L, MCGUIRE D, WILDING J, GAUSE-NILSSON I, OSCARSSON J, MARSTON N, RUFF C, SABATINE M, WIVIOTT S. 1896-LB: Clinical Features and C-Peptide Levels Associated with Diabetic Ketoacidosis in Patients with Type 2 Diabetes on Dapagliflozin vs. Placebo—Insights from DECLARE-TIMI 58. Diabetes 2024, 73 DOI: 10.2337/db24-1896-lb.Peer-Reviewed Original ResearchDuke Clinical Research InstituteC-pepClinical Research InstituteDKA eventsC-peptideLower fasting C-peptideMedian Follow-UpRandomized to dapagliflozinFasting C-peptideDuration of T2DC-peptide testingRisk of DKACV outcome trialsAmerican College of CardiologyAbsolute risk differenceRate of DKAImpaired insulin secretionType 2 diabetesPlacebo armClinical featuresPopulation Health Research InstituteCSL BehringDiabetic ketoacidosisFerring PharmaceuticalsOutcome trials1895-LB: Fasting C-Peptide and Type 1 Diabetes Polygenic Score Predict Diabetic Ketoacidosis in People with Type 2 Diabetes in DECLARE-TIMI 58
MI KANG Y, MELLONI G, CAHN A, RAZ I, MOURA F, BHATT D, INZUCCHI S, LEITER L, MCGUIRE D, WILDING J, GAUSE-NILSSON I, OSCARSSON J, MARSTON N, RUFF C, SABATINE M, WIVIOTT S. 1895-LB: Fasting C-Peptide and Type 1 Diabetes Polygenic Score Predict Diabetic Ketoacidosis in People with Type 2 Diabetes in DECLARE-TIMI 58. Diabetes 2024, 73 DOI: 10.2337/db24-1895-lb.Peer-Reviewed Original ResearchRisk of DKADuke Clinical Research InstituteC-pepC-peptidePolygenic scoresClinical Research InstitutePopulation Health Research InstituteHigh-risk ptsFasting C-peptideHealth Research InstituteCV outcome trialsAmerican College of CardiologyHigher-risk groupsPredicting DKAType 2 diabetesCSL BehringDiabetic ketoacidosisFerring PharmaceuticalsBristol-Myers SquibbSt. Jude MedicalNon-HLAIncreased riskCleveland ClinicDapagliflozinAmerican College
2022
864-P: Glycemic Control across eGFR Categories with the Dual SGLT1 and 2 Inhibitor Sotagliflozin in SCORED
INZUCCHI S, BHATT D, PITT B, SZAREK M, CANNON C, LEWIS J, RIDDLE M, KOSIBOROD M, CHERNEY D, DWYER J, SCIRICA B, BAILEY C, BANKS P, DIAZ R, RAY K, UDELL J, LOPES R, LEITER L, STEG P. 864-P: Glycemic Control across eGFR Categories with the Dual SGLT1 and 2 Inhibitor Sotagliflozin in SCORED. Diabetes 2022, 71 DOI: 10.2337/db22-864-p.Peer-Reviewed Original ResearchGlucose-lowering medicationsPre-specified subgroupsNovartis Pharmaceuticals CorporationGlaxoSmithKline plcDaiichi SankyoDual SGLT1Baseline A1CBaseline eGFRPfizer Inc.Bristol-Myers Squibb CompanyLexicon PharmaceuticalsOutcome trialsCSL BehringNovo NordiskNovartis AGAdvanced chronic kidney diseaseType 2 diabetes patientsEli LillyAmgen Inc.Lower baseline eGFROverall trial populationCKD stage 4Primary composite outcomeSpeakers bureauCardiovascular outcome trials
2021
Health insurance coverage and switching among people with hemophilia A in the United States.
He C, Hinds D, Pezalla E, Cheng D, Chen E, Sammon J, Solari P, Recht M. Health insurance coverage and switching among people with hemophilia A in the United States. Journal Of Managed Care & Specialty Pharmacy 2021, 28: 232-243. PMID: 34780299, PMCID: PMC12128936, DOI: 10.18553/jmcp.2021.21311.Peer-Reviewed Original ResearchConceptsBioMarin Pharmaceutical Inc.Insurance categoriesInsurance coverageSevere HAFollow-up periodHemophilia ACSL BehringFollow-upHealth insurance coverageFactor VIIILong-term prophylactic therapyYears of coverageDuration of follow-upOregon Health & Science UniversityYears of follow-upAmerican ThrombosisCohort of patientsYears of Medicare coverageCommercially insured patientsPayers' abilityInsurance systemStock/stock optionsMedicaid coverageProphylactic therapyBleeding eventsImpact of the COVID-19 Pandemic on in-Person Visit Rates Among Patients with Hematologic Malignancies in the United States
Goyal G, Lau K, Wang X, Davidoff A, Huntington S, Jamy O, Calip G, Shah H, Stephens D, Miksad R, Parikh R, Takvorian S, Neparidze N, Seymour E. Impact of the COVID-19 Pandemic on in-Person Visit Rates Among Patients with Hematologic Malignancies in the United States. Blood 2021, 138: 1930. PMCID: PMC8701834, DOI: 10.1182/blood-2021-153625.Peer-Reviewed Original ResearchCurrent equity holderEarly pandemic monthsLines of therapyHematologic malignanciesVisit ratesTreatment typePandemic monthsAdvisory CommitteeCAR T-cell therapyPerson medical careDiagnosis of AMLDe-identified databaseCOVID-19 pandemicSignificant reductionPandemic periodCurrent employmentElectronic health recordsFlatiron HealthOral therapyActive treatmentHematopoietic transplantsInitial diagnosisCSL BehringOutpatient infusionsActive patientsRacial Disparities in Telemedicine Uptake during the COVID-19 Pandemic Among Patients with Hematologic Malignancies in the United States
Neparidze N, Lau K, Wang X, Davidoff A, Huntington S, Jamy O, Calip G, Shah H, Stephens D, Miksad R, Parikh R, Takvorian S, Goyal G, Seymour E. Racial Disparities in Telemedicine Uptake during the COVID-19 Pandemic Among Patients with Hematologic Malignancies in the United States. Blood 2021, 138: 1973. PMCID: PMC8701527, DOI: 10.1182/blood-2021-153787.Peer-Reviewed Original ResearchCurrent equity holderWhite patientsBlack patientsInpatient treatmentVisit ratesTelemedicine uptakeTreatment categoriesPerson visitsHematologic malignanciesAdvisory CommitteeTreatment typeSignificant reductionRacial disparitiesLines of therapyDiagnosis of AMLDe-identified databaseEarly pandemic monthsPotential racial disparitiesCurrent employmentElectronic health recordsFlatiron HealthHospital admissionActive treatmentInitial diagnosisCSL BehringSars-Cov-2 Vaccination in Patients with Pre-Existing Immune Thrombocytopenia
Lee E, Moreira M, Al-Samkari H, Cuker A, DiRaimo J, Gernsheimer T, Kruse A, Kessler C, Kruse C, Leavitt A, Lee A, Liebman H, Newland A, Ray A, Tarantino M, Thachil J, Kuter D, Cines D, Bussel J. Sars-Cov-2 Vaccination in Patients with Pre-Existing Immune Thrombocytopenia. Blood 2021, 138: 586. PMCID: PMC8701617, DOI: 10.1182/blood-2021-152918.Peer-Reviewed Original ResearchSARS-CoV-2 vaccinesITP exacerbationPre-vaccination baselineBristol-Myers SquibbImmune thrombocytopeniaPlatelet countRescue treatmentDose 2Dose 1Second dosePlatelet decreaseSpeakers bureauSpark TherapeuticsConsultancy feesFirst doseCSL BehringSupport associationsNovo NordiskSARS-CoV-2 vaccinationDiagnosis of ITPAdvisory CommitteeData Safety Monitoring BoardConcurrent autoimmune diseasesMore refractory diseasePrior medical treatmentReduced Dosing Frequency Following a Switch to Rix-FP for the Treatment of Hemophilia B: Results from the Athn 2 Study
Journeycake J, Cheng D, Chrisentery-Singleton T, Desai V, von Drygalski A, Fedor C, Hirsh N, Patel B, Raffini L, Recht M, Sidinio R, Wang M, Zhang X, Neufeld E. Reduced Dosing Frequency Following a Switch to Rix-FP for the Treatment of Hemophilia B: Results from the Athn 2 Study. Blood 2021, 138: 1039. DOI: 10.1182/blood-2021-144617.Peer-Reviewed Original ResearchEntity's Board of DirectorsExtended dosing intervalRIX-FPDosing intervalHemophilia treatment centersDosing frequencyCSL BehringProspective armRetrospective armOnce-weeklyHemophilia BHemophilia B.Half-life recombinant factorTreatment switchUS hemophilia treatment centersRange of therapeutic optionsFrequent dosing regimenPrescribed dosing frequencyTreatment of hemophilia BFactor replacement productsWeeks prior to enrollmentAmerican ThrombosisSevere hemophilia BBaseline demographic dataReduced dosing frequencyEptacog Beta Efficacy in Children and Adolescents with Hemophilia A or B and Inhibitors: Subset Analysis Suggests Improved Caregiver Capacity to Assess Bleeding Episode Resolution with Subject Age
Young G, Pipe S, Hermans C, Carcao M, Castaman G, Davis J, Ducore J, Dunn A, Escobar M, Journeycake J, Khan O, Mahlangu J, Meeks S, Mitha I, Négrier C, Nowak-Göttl U, Recht M, Chrisentery-Singleton T, Stasyshyn O, Vilchevska K, Martinez L, Wang M, Windyga J, Alexander W, Al-Sabbagh A, Bonzo D, Macie C, Mitchell I, Wilkinson T, Shapiro A. Eptacog Beta Efficacy in Children and Adolescents with Hemophilia A or B and Inhibitors: Subset Analysis Suggests Improved Caregiver Capacity to Assess Bleeding Episode Resolution with Subject Age. Blood 2021, 138: 3195. DOI: 10.1182/blood-2021-147434.Peer-Reviewed Original ResearchInitial dosing regimensEntity's Board of DirectorsEptacog betaBleeding episodesPhase 3 trialCSL BehringPediatric age subgroupsConfidence intervalsTreatment successHemophilia AYears subgroupIncreasing subject ageAge subgroupsSubject ageBeta infusionAdvisory CommitteeResearch fundingOregon Health & Science UniversitySuccess proportionTreatment of bleeding episodesControl of bleeding episodesModerate bleeding episodesTreat bleeding episodesNational Hemophilia FoundationOpen-label trialA Cross-Sectional Study of the American Thrombosis and Hemostasis Network Dataset for Outcomes of Dental Extractions in People with Hemophilia
Messenger H, He C, Recht M, Kulkarni R. A Cross-Sectional Study of the American Thrombosis and Hemostasis Network Dataset for Outcomes of Dental Extractions in People with Hemophilia. Blood 2021, 138: 346. DOI: 10.1182/blood-2021-148498.Peer-Reviewed Original ResearchDental extractionBleeding episodesOn-demand therapyStandard half-life productsHemostatic therapyFactor replacementHemophilia APWH ABleeding outcomesClinical characteristicsMild haemophiliaCSL BehringAmerican ThrombosisBleeding rateBlood disordersCohort of PWHPWH aged >Emergency departmentHistory of inhibitorsType of hemophiliaHemophilia A patientsPost-DLIHemophilia B patientsAnnual clinic visitsEntity's Board of DirectorsProphylaxis with rFIXFc Reduces the Frequency and Delays Time to First Spontaneous Bleed Event in Previously Untreated Patients with Hemophilia B: A Post Hoc Analysis of the PUPs B-LONG Trial
Nolan B, Recht M, Rendo P, Falk A, Foster M, Casiano S, Rauch A, Shapiro A. Prophylaxis with rFIXFc Reduces the Frequency and Delays Time to First Spontaneous Bleed Event in Previously Untreated Patients with Hemophilia B: A Post Hoc Analysis of the PUPs B-LONG Trial. Blood 2021, 138: 498. DOI: 10.1182/blood-2021-148765.Peer-Reviewed Original ResearchSpontaneous bleeding eventsEntity's Board of DirectorsBleeding eventsPost Hoc AnalysisHemophilia BYears of agePPX treatmentCSL BehringKaplan-MeierHalf-life recombinant factorTreatment of bleeding eventsPrevention of bleeding eventsHoc AnalysisSmall patient groupsFIX replacement therapyFactor IXKaplan-Meier (KM) survival analysisStandard of careAdvisory CommitteeOD therapyBleeding patternsUntreated patientsPrimary endpointOD regimensProlonged bleeding
2020
First Data from the Phase 3 HOPE-B Gene Therapy Trial: Efficacy and Safety of Etranacogene Dezaparvovec (AAV5-Padua hFIX variant; AMT-061) in Adults with Severe or Moderate-Severe Hemophilia B Treated Irrespective of Pre-Existing Anti-Capsid Neutralizing Antibodies
Pipe S, Recht M, Key N, Leebeek F, Castaman G, Lattimore S, Van Der Valk P, Peerlinck K, Coppens M, O'Connell N, Pasi J, Kampmann P, Meijer K, von Drygalski A, Young G, Hermans C, Astermark J, Klamroth R, Lemons R, Visweshwar N, Crary S, Kazmi R, Symington E, Escobar M, Gomez E, Kruse-Jarres R, Kotowski A, Quon D, Wang M, Wheeler A, Sawyer E, Verweij S, Colletta V, Bajma N, Gut R, Miesbach W. First Data from the Phase 3 HOPE-B Gene Therapy Trial: Efficacy and Safety of Etranacogene Dezaparvovec (AAV5-Padua hFIX variant; AMT-061) in Adults with Severe or Moderate-Severe Hemophilia B Treated Irrespective of Pre-Existing Anti-Capsid Neutralizing Antibodies. Blood 2020, 136: lba-6. DOI: 10.1182/blood-2020-143560.Peer-Reviewed Original ResearchAdeno-associated virus serotype 5Entity's Board of DirectorsEtranacogene dezaparvovecInvestigational gene therapyCSL BehringHemophilia BAdverse eventsPersonal feesGene therapyFrequent treatment-related adverse eventsClinical studiesPre-existing neutralizing antibodiesTreatment-related adverse eventsLead-InGene therapy clinical studiesPre-existing NAbsAnnualized bleeding rateGene therapy trialsInfusion-related reactionsPhase 2b studyPhase 3 studyLead-in periodUse of corticosteroidsInfluenza-like illnessLiver-specific promoterHemophilia Natural History Study (ATHN 7): Baseline Characteristics, Adverse Events, and Self-Reported Health Status of Individuals with Hemophilia a and B
Buckner T, Carpenter S, Croteau S, Cuker A, Daoud N, Kempton C, Malec L, McLean T, Raffini L, Staber J, Wang M, Watson C, Zia A, Recht M. Hemophilia Natural History Study (ATHN 7): Baseline Characteristics, Adverse Events, and Self-Reported Health Status of Individuals with Hemophilia a and B. Blood 2020, 136: 2-3. DOI: 10.1182/blood-2020-137696.Peer-Reviewed Original ResearchEuropean Haemophilia Safety SurveillanceEntity's Board of DirectorsHemophilia treatment centersAdverse eventsHemophilia ACSL BehringHemophilia therapyAmerican ThrombosisCohort studySkin reactionsNatural history cohort studyCongenital hemophilia ARisk of bleedingHemostasis NetworkTreated with clotting factorMinor skin reactionsLocal institutional review boardProspective cohort studyUnrelated to treatmentBaseline medical historyAmerican Academy of PediatricsCohort of subjectsNational Hemophilia FoundationNatural history studiesMedical record reviewEtranacogene Dezaparvovec (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B: Two Year Data from a Phase 2b Trial
von Drygalski A, Giermasz A, Castaman G, Key N, Lattimore S, Leebeek F, Miesbach W, Recht M, Gomez E, Gut R, Pipe S. Etranacogene Dezaparvovec (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B: Two Year Data from a Phase 2b Trial. Blood 2020, 136: 13. DOI: 10.1182/blood-2020-139295.Peer-Reviewed Original ResearchEntity's Board of DirectorsEtranacogene dezaparvovecInvestigational gene therapyLiver-specific promoterAMT-061Gene therapyHemophilia BCSL BehringAdverse eventsNormal rangeWeek 6FIX activity levelsRecords of bleedingYears of follow-upPhase 2b trialAmeliorate disease severityYears of follow-up dataWeeks post-dosingFollow-up dataMulti-center trialNational Hemophilia FoundationAdvisory CommitteeHemophilia B subjectsSustained FIX activityFixing activity
2019
One Year Data from a Phase 2b Trial of AMT-061 (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B
Pipe S, Giermasz A, Castaman G, Key N, Lattimore S, Leebeek F, Miesbach W, Recht M, Gomez E, Long A, Gut R, von Drygalski A. One Year Data from a Phase 2b Trial of AMT-061 (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B. Blood 2019, 134: 3348. DOI: 10.1182/blood-2019-128765.Peer-Reviewed Original ResearchAdeno-associated virus serotype 5Entity's Board of DirectorsAMT-061CSL BehringGene therapyAdverse eventsHemophilia BFollow-upWeek 6Weeks of follow-upFIX activity levelsInvestigational gene therapyRecords of bleedingYears of follow-upPhase 2b trialPhase 3 studyLiver-specific promoterMulti-center trialAdvisory CommitteeSpeakers bureauPatient-reported outcomesOpen-labelFixing activityLaboratory parametersPrimary endpoint
2018
Data Coming out of the Human Inhibitor PUP Study (HIPS) Reveal 4 Subgroups of Patients with Distinct Antibody Signatures
Gangadharan B, Reipert B, Berg V, Scheiflinger F, Blatny J, Fijnvandraat K, Gruppo R, Klintman J, Male C, McGuinn C, Meeks S, Radulescu V, Ragni M, Recht M, Santagostino E, Shapiro A, Staber J, Yaish H, Yee D, Brown D. Data Coming out of the Human Inhibitor PUP Study (HIPS) Reveal 4 Subgroups of Patients with Distinct Antibody Signatures. Blood 2018, 132: 3774. DOI: 10.1182/blood-2018-99-115979.Peer-Reviewed Original ResearchFVIII-specific antibodiesEntity's Board of DirectorsLow titer inhibitorsFVIII inhibitorsIntron 22 inversionCSL BehringTh17 cellsT cellsAntibody signaturesHigh-affinity IgG1Study endIgG subclassesTiter inhibitorsIgG1 antibodiesPatient subgroupsPUP studiesComplication of replacement therapyExposure daysDevelopment of FVIII inhibitorsPro-inflammatory TH17 cellsProspective multicenter observational studyMissense mutationsIntron 1 inversionAnti-FVIII antibodiesClass-switched antibodies
2017
Longitudinal Antibody Signatures Following FVIII Replacement Therapy in Previously Untreated Patients with Severe Hemophilia Α- New Insights from the Hemophilia Inhibitor PUP Study (HIPS)
Gangadharan B, Reipert B, Scheiflinger F, Bowen J, Donnachie E, van Draat K, Gruppo R, Klintman J, Male C, McGuinn C, Meeks S, Recht M, Ragni M, Yaish H, Shapiro A, Yee D, Radulescu V, Santagostino E, Brown D. Longitudinal Antibody Signatures Following FVIII Replacement Therapy in Previously Untreated Patients with Severe Hemophilia Α- New Insights from the Hemophilia Inhibitor PUP Study (HIPS). Blood 2017, 130: 88. DOI: 10.1182/blood.v130.suppl_1.88.88.Peer-Reviewed Original ResearchFVIII-specific antibodiesNon-neutralizing antibodiesPro-inflammatory TH17 cellsEntity's Board of DirectorsFVIII inhibitor developmentTotal T cellsFVIII inhibitorsT cellsCirculating immune cellsNK cellsTh17 cellsIgG subclassesInhibitor patientsIgG1 antibodiesExposure daysCSL BehringImmune cellsPUP studiesTiter of FVIII inhibitorsInhibitor developmentCirculating Th17 cellsHigh-titer inhibitorsFVIII replacement therapyAnti-FVIII antibodiesSevere hemophilia ASurgical Experience in Two Multicenter, Open-Label Phase 3 Studies of Emicizumab in Persons with Hemophilia A with Inhibitors (HAVEN 1 and HAVEN 2)
Kruse-Jarres R, Callaghan M, Croteau S, Jimenez-Yuste V, Khoo L, Liesner R, Matsushita T, Recht M, Young G, Chang T, Dhalluin C, Mu Y, Xu J, Devenport J, Ko R, Solari P, Oldenburg J. Surgical Experience in Two Multicenter, Open-Label Phase 3 Studies of Emicizumab in Persons with Hemophilia A with Inhibitors (HAVEN 1 and HAVEN 2). Blood 2017, 130: 89. DOI: 10.1182/blood.v130.suppl_1.89.89.Peer-Reviewed Original ResearchPost-operative bleeding eventsPost-operative bleedingEntity's Board of DirectorsOpen-label phase 3 studyTooth extractionBleeding eventsHAVEN 1Surgical proceduresCSL BehringTreated with BPAInterim analysisHAVEN 2Hemophilia AFrequency of post-operative bleedingSurgical experienceClinical studiesHemophilia A with inhibitorsKnee arthroscopyInvestigational clinical studyPfizer Inc.Treated with emicizumabPre-operative treatmentPhase 3 studyPrevention of bleedingAnalysis of patients
2016
Identification of Qualitative Platelet Disorders in Adolescent Women with Heavy Menstrual Bleeding
Haley K, Lattimore S, McDavitt C, Khader A, Boehnlein C, Baker-Groberg S, Ngo A, McCarty O, Recht M. Identification of Qualitative Platelet Disorders in Adolescent Women with Heavy Menstrual Bleeding. Blood 2016, 128: 4922. DOI: 10.1182/blood.v128.22.4922.4922.Peer-Reviewed Original ResearchHeavy menstrual bleedingBleeding disorder diagnosisQualitative platelet disordersEntity's Board of DirectorsPlatelet function assaysPictorial Bleeding Assessment ChartPlatelet disordersPlatelet functionAdolescent womenBleeding phenotypeMenstrual bleedingPlatelet aggregationQuality of lifeBleeding disordersCSL BehringEvaluation of heavy menstrual bleedingAbnormal platelet function testsISTH bleeding assessment toolEtiology of heavy menstrual bleedingFunctional assaysMedian PBAC scoreResponse to agonist stimulationPlatelet function abnormalitiesBleeding assessment toolAssessment of platelet activationLinear and Logistic Regression Models of Patient-Reported Outcomes and Patient Characteristics in US Adults with Hemophilia from the Pain, Functional Impairment, and Quality of Life (P-FiQ) Study
Batt K, Recht M, Wang M, Quon D, Boggio L, Kessler C, Buckner T, Neff A, Iyer N, Cooper D, Kempton C. Linear and Logistic Regression Models of Patient-Reported Outcomes and Patient Characteristics in US Adults with Hemophilia from the Pain, Functional Impairment, and Quality of Life (P-FiQ) Study. Blood 2016, 128: 252. DOI: 10.1182/blood.v128.22.252.252.Peer-Reviewed Original ResearchBrief Pain Inventory v2 Short FormLack of college educationInternational Physical Activity QuestionnaireHaemophilia Activities ListEntity's Board of DirectorsPatient-reported outcomesVisual analog scalePatient-reported outcome instrumentsSevere haemophiliaQuality of lifePatient characteristicsPhysical activityCollege educationCSL BehringFunctional impairmentUS adultsOlder ageHistory of joint painMeasure patient-reported outcomesPhysical Activity QuestionnaireGreater physical activityAdvisory CommitteeAdministration of patient-reported outcomesResearch fundingSubject characteristics
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