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 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 PMID: 41220279, 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-offHealthcare resource utilization in people with hemophilia treated within vs. outside u.S. hemophilia treatment center network clinics: An interim analysis of the CHESS US study
Ferri Grazzi E, Lim M, Crook N, Santaella M, Recht M, Giermasz A, Roberts J, Curtis R. Healthcare resource utilization in people with hemophilia treated within vs. outside u.S. hemophilia treatment center network clinics: An interim analysis of the CHESS US study. Blood 2025, 146: 2615-2615. DOI: 10.1182/blood-2025-2615.Peer-Reviewed Original ResearchAbstract Introduction PeopleHealthcare resource utilizationHealth care providersGeneral burdenIntroduction PeopleHospital visitsHealth related outcomesHealthcare resource utilization dataClinically complex populationMedical recordsHighest prevalenceBurden-of-illness studyBurden of diseaseSelf-completion formTarget jointsCare of PWHEmergency room visitsCost of HaemophiliaChronic joint painUnited StatesCare providersClinical characteristicsCare settingsHealth outcomesSevere PWHEnd-of-study analysis of the HOPE-B trial confirms the durable efficacy and safety of etranacogene dezaparvovec hemophilia b gene therapy over 5 years
Pipe S, Miesbach W, Recht M, Leebeek F, Key N, Castaman G, Lattimore S, Coppens M, LeQuellec S, Gill S, Mahajan V, Drelich D, Monahan P. End-of-study analysis of the HOPE-B trial confirms the durable efficacy and safety of etranacogene dezaparvovec hemophilia b gene therapy over 5 years. Blood 2025, 146: 538-538. DOI: 10.1182/blood-2025-538.Peer-Reviewed Original ResearchAdeno-associated virus serotype 5Treatment-related AEsLead-in periodPhase 3 trialEtranacogene dezaparvovecGene therapySingle doseMonth 7Hemophilia BFollow-upFrequent treatment-related AEsHemophilia B gene therapyCases of hepatocellular carcinomaIncident cases of hepatocellular carcinomaDuration of follow-upEnd-of-study analysisFIX activity levelsLong-term hepatotoxicityYears of follow-upPositive benefit/risk ratioAlanine aminotransferase elevationSpontaneous bleeding eventsBleed protectionEnd-of-studyEnd-of-study dataElevation of liver health biomarkers before and after etranacogene dezaparvovec gene therapy in hemophilia B: Associations with long-term clinical outcomes
Miesbach W, Pipe S, Hermans C, Recht M, Escobar M, Mahajan V, Jansen N, Galante N, Church R, Monahan P. Elevation of liver health biomarkers before and after etranacogene dezaparvovec gene therapy in hemophilia B: Associations with long-term clinical outcomes. Blood 2025, 146: 6093. DOI: 10.1182/blood-2025-6093.Peer-Reviewed Original ResearchAlanine aminotransferase elevationAnnualized bleeding rateEtranacogene dezaparvovecGene therapySingle doseHemophilia BAlanine aminotransferasePost-doseLead-InAdeno-associated virus-mediated gene therapyBleeding rateFollow-upVirus-mediated gene therapyLiver-targeted gene therapyC (HCV) infectionElevated prior to treatmentLong-term clinical outcomesActive hepatitis BPhase 3 studyLiver-related eventsLead-in periodAdvanced liver fibrosisHistory of HIVLong-term efficacyWeeks post-dosingReal-world use of prophylaxis in people with non-severe hemophilia in the United States: Insights from the athn transcends Study - hemophilia natural history arm
Corrales-Medina F, Afari-Dwamena N, Blair A, Carpenter S, Cohen C, Janbain M, Staber J, Schwartz J, Recht M, Chrisentery-Singleton T. Real-world use of prophylaxis in people with non-severe hemophilia in the United States: Insights from the athn transcends Study - hemophilia natural history arm. Blood 2025, 146: 4472. DOI: 10.1182/blood-2025-4472.Peer-Reviewed Original ResearchNon-severe haemophiliaStandard half-lifeMild HAHemophilia AMild diseaseModerate HAModerate diseaseProphylaxis useContinuous prophylaxisProphylactic agentHemophilia B (HBEvidence of recurrent bleedingAs-needed therapySafety of prophylaxisBaseline clinical dataB (HBMild-to-moderate haemophiliaHalf-lifeNon-severe casesNon-severe diseaseRecombinant FVIII concentratesHA participantsEarly prophylaxisBleeding phenotypeProphylaxis guidelinesNatural 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 Original ResearchCompletion 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 antibodies
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
Learn More on Yale Medicine
Yale Medicine News
News
News
Get In Touch
Contacts
Appointment Number
Mailing Address
Pediatric Hematology & Oncology
333 Cedar Street
New Haven, CT 06510
United States
Locations
Patient Care Locations
Are You a Patient? View this doctor's clinical profile on the Yale Medicine website for information about the services we offer and making an appointment.