Michael Recht, MD, PhD, MBA
he/him/his
Professor of Clinical Pediatrics (Hematology/Oncology)Cards
About
Research
Publications
2025
Final Analysis of a Study of Etranacogene Dezaparvovec for Hemophilia B
Pipe S, Miesbach W, Recht M, Leebeek F, Key N, Castaman G, Lattimore S, Coppens M, Le Quellec S, Mahajan V, Gill S, Drelich D, Monahan P. Final Analysis of a Study of Etranacogene Dezaparvovec for Hemophilia B. The New England Journal Of Medicine 2025, 394: 463-474. PMID: 41358585, DOI: 10.1056/nejmoa2514332.Peer-Reviewed Original ResearchAdeno-associated virus serotype 5Factor IX expressionLead-in periodEtranacogene dezaparvovecIX expressionBleeding rateGene therapyBleeding eventsMonth 7Adverse eventsHemophilia BNeutralizing antibodiesTreatment of bleeding eventsFactor IX infusionsFactor IX prophylaxisYears of follow-upPhase 3 studySingle-dose treatmentFollow-up periodLow-grade severityOpen-labelRoutine prophylaxisMonth 6Prophylactic treatmentFollow-upSafety and Effectiveness of Emicizumab in People With Haemophilia A Enrolled in the ATHN 7 Haemophilia Natural History Study
Buckner T, Carpenter S, Daoud N, Kempton C, Lee L, Malec L, McLean T, Morton P, O'Neill C, Staber J, Wang M, Croteau S, Recht M. Safety and Effectiveness of Emicizumab in People With Haemophilia A Enrolled in the ATHN 7 Haemophilia Natural History Study. Haemophilia 2025, 32: 63-70. PMID: 41220279, PMCID: PMC12904170, DOI: 10.1111/hae.70151.Peer-Reviewed Original ResearchMedical record reviewProspective observational cohort studyNatural history studiesObservational cohort studyParticipant characteristicsRecord reviewAmerican ThrombosisParticipant interviewsPractice of treatmentAdverse eventsCohort studySeverity of HAMonitor useParticipantsABRATHNClinical informationHistory studiesPeopleCut-offNatural history of preexisting AAV5 antibodies in adults with hemophilia B during the lead-in of the etranacogene dezaparvovec phase 3 study
Klamroth R, Recht M, Key N, Miesbach W, Pipe S, Kaczmarek R, Drelich D, Salazar B, Le Quellec S, Monahan P, Galante N, van der Valk P, Tarrant J. Natural history of preexisting AAV5 antibodies in adults with hemophilia B during the lead-in of the etranacogene dezaparvovec phase 3 study. Molecular Therapy — Methods & Clinical Development 2025, 33: 101568. PMID: 40969675, PMCID: PMC12441693, DOI: 10.1016/j.omtm.2025.101568.Peer-Reviewed Original ResearchAAV serotype 5Adeno-associated virusLead-in periodNatural historyGene therapyAAV-based gene therapyLaboratory assessmentAnti-AAV5 antibodiesCorrelation of titersPhase 3 studyAssociated with older agePhase 3 trialNAb titersDetectable NAbsMedian titerHemophilia BNeutralizing antibodiesHemophilia B.Serotype 5HemophiliaImmunoglobulin MNAbsBinding antibodiesLead-InImmunoglobulin GEffectiveness of Octocog Alfa (BAY 81‐8973) to Treat People With Haemophilia A Enrolled in the ATHNdataset, Including Under‐Represented Subgroups
Chandler M, Moulton T, Charafi L, Charlet J, Recht M. Effectiveness of Octocog Alfa (BAY 81‐8973) to Treat People With Haemophilia A Enrolled in the ATHNdataset, Including Under‐Represented Subgroups. European Journal Of Haematology 2025, 115: 484-492. PMID: 40770897, PMCID: PMC12505839, DOI: 10.1111/ejh.70016.Peer-Reviewed Original ResearchThe Bleeding Disorders Research Collaborative
Valentino L, Santaella M, Witkop M, Stanhope R, Valadez S, Carlson S, Benasutti H, DiMichele D, Recht M. The Bleeding Disorders Research Collaborative. Blood Vessels Thrombosis & Hemostasis 2025, 2: 100099. PMID: 41070002, PMCID: PMC12506547, DOI: 10.1016/j.bvth.2025.100099.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsCompletion of phase 2b trial of etranacogene dezaparvovec gene therapy in patients with hemophilia B over 5 years
von Drygalski A, Gomez E, Giermasz A, Castaman G, Key N, Lattimore S, Leebeek F, Miesbach W, Recht M, Monahan P, Le Quellec S, Pipe S. Completion of phase 2b trial of etranacogene dezaparvovec gene therapy in patients with hemophilia B over 5 years. Blood Advances 2025, 9: 3543-3552. PMID: 40188458, PMCID: PMC12275190, DOI: 10.1182/bloodadvances.2024015291.Peer-Reviewed Original ResearchAdeno-associated virus serotype 5Etranacogene dezaparvovecHemophilia BRecombinant adeno-associated virus serotype 5FIX inhibitor developmentLiver-specific promoterPost-administrationSelf-administered infusionsSevere hemophilia BMulti-center trialYears post-administrationBleeding episodesOpen-labelSingle-doseSecondary endpointsIntravenous doseSingle-armGene therapyThrombotic complicationsYears post-treatmentSafety profileClinically significant elevationsAdverse eventsBleeding frequencyNeutralizing antibodiesPlain language summary of 4-year results of the GENEr8-1 clinical trial of valoctocogene roxaparvovec gene therapy for hemophilia A
Madan B, Ozelo M, Kenet G, Chou S, Dashiell-Aje E, Robinson T, Mahlangu J, Recht M, Careta F, Ruiz A, Fletcher S, Goshen D. Plain language summary of 4-year results of the GENEr8-1 clinical trial of valoctocogene roxaparvovec gene therapy for hemophilia A. Future Rare Diseases 2025, 5: 2522071. DOI: 10.1080/23995270.2025.2522071.Peer-Reviewed Original ResearchPain Reduction Following Eptacog Beta Treatment of Bleeding Episodes in Adolescents and Adults With Haemophilia A or B Complicated by Inhibitors
Buckner T, Kessler C, Castaman G, Hermans C, Jiménez‐Yuste V, Mahlangu J, Miesbach W, Oldenburg J, Recht M, Valentino L, Wheeler A, Pipe S. Pain Reduction Following Eptacog Beta Treatment of Bleeding Episodes in Adolescents and Adults With Haemophilia A or B Complicated by Inhibitors. Haemophilia 2025, 31: 713-721. PMID: 40534395, DOI: 10.1111/hae.70077.Peer-Reviewed Original ResearchInitial dosing regimensVisual analogue scaleBleeding episodesScore reductionReceiver Operating CharacteristicEptacog betaPain reductionPain levelsTreatment responseVisual analogue scale score reductionBeta treatmentTreatment outcomesMean VAS scorePain score reductionTreat bleeding episodesVAS score reductionBaseline pain levelsAssess treatment responseEvaluate treatment responseEvaluate pain reductionControl of bleedingPredicting Treatment OutcomePost hoc studyPatient-reported outcomesBleed resolutionInhibitor development and clinical characteristics in children with severe hemophilia A in the ATHN 8 US cohort study
Thornburg C, van den Berg H, Chandler M, Malec L, Manuel M, O’Neill C, Recht M, Taggart E, Carpenter S, group A, Knoll C, Malec L, Wang M, Guerrera M, Tarango C, Sidonio R, Rodriguez N, Friedman K, Shapiro A, Yaish H, Carpenter S, Janbain M, Grabowski E, Pruthi R, Haley K, Thornburg C, Ahuja S, Simpson M, Reiss U, Lucas T, Balasa V, Wheeler A. Inhibitor development and clinical characteristics in children with severe hemophilia A in the ATHN 8 US cohort study. Blood Vessels Thrombosis & Hemostasis 2025, 2: 100082. PMID: 40918739, PMCID: PMC12412393, DOI: 10.1016/j.bvth.2025.100082.Peer-Reviewed Original ResearchClotting factor concentratesSevere hemophilia AHemophilia AInhibitor developmentExposure daysCohort studyAssociated with inhibitor developmentLow-titer inhibitorsReduce inhibitor developmentHigh-titer inhibitorsCox proportional hazards modelsEvaluate risk factorsClinically significant inhibitorUS cohort studyProportional hazards modelCongenital hemophiliaUntreated patientsSevere HAClinical characteristicsPrevent bleedingNeutralizing antibodiesModerate haemophiliaNonsense variantFactor concentrationsRisk factorsCorticosteroid use to mitigate transaminitis-associated decline in FVIII levels following valoctocogene roxaparvovec gene therapy: clinical practice guidance
Konkle B, Peyvandi F, Foster G, Hermans C, La Mura V, Leavitt A, Lillicrap D, Mahlangu J, Ozelo M, Pipe S, Recht M, Srivastava A, Young G, Miesbach W. Corticosteroid use to mitigate transaminitis-associated decline in FVIII levels following valoctocogene roxaparvovec gene therapy: clinical practice guidance. Journal Of Thrombosis And Haemostasis 2025, 23: 2086-2094. PMID: 40090623, DOI: 10.1016/j.jtha.2025.02.042.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsSevere hemophilia AValoctocogene roxaparvovecFactor VIIIGene therapyHemophilia AMarkers of hepatocyte injuryFactor VIII activity levelsCourse of corticosteroidsFactor VIII levelsElevated alanine transaminaseReduced FVIII levelsClinical practice guidanceFVIII levelsCorticosteroid useAdverse eventsClinical dataClinical trialsCorticosteroidsHepatocyte injuryAlanine transaminaseTherapyPatientsActivity levelsExpert opinionAdults
Clinical Care
Overview
Michael Recht, MD, PhD, is a pediatric hematologist who focuses on treating children, adolescents, and young adults with bleeding and clotting disorders.
Dr. Recht is a professor of clinical pediatrics at Yale School of Medicine. He has been actively involved in more than 110 clinical trials, including research on gene therapy for hemophilia. Dr. Recht also serves as the chief medical and scientific officer for the National Bleeding Disorders Foundation. In this role, he helps shape the scientific direction and research strategy of the organization, working to advance the understanding and treatment of bleeding disorders. Dr. Recht received his medical and doctoral degrees from the University of Wisconsin-Madison. He completed his residency in pediatrics and fellowship in pediatric hematology-oncology at Yale New Haven Hospital.
Clinical Specialties
Pediatric Hematology & Oncology
Fact Sheets
Bleeding Disorders
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Pediatric Hematology & Oncology
333 Cedar Street
New Haven, CT 06510
United States
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