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Journal Articles

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

Steven Pipe, Wolfgang Miesbach, Michael Recht, Frank Leebeek, Nigel Key, Giancarlo Castaman, Susan Lattimore, Michiel Coppens, Sandra LeQuellec, Sean Gill, Vaibhav Mahajan, Douglass Drelich, Paul Monahan

Blood (2025) 146 (Supplement 1): 538.

https://doi.org/10.1182/blood-2025-538

Published: 2025

Journal Articles

Using FXIa-triggered thrombin generation to assess the risk of breakthrough bleeding in patients receiving emicizumab for acquired Hemophilia A

Fabius Pelzer, Ella Ertekin, Olga Oleshko, Annika Klingberg, Paul Knoebl, Christian Pfrepper, Richard Greil, Johannes Oldenburg, Ulrich Sachs, Wolfgang Miesbach, Karolin Trautmann-Grill, Katharina Holstein, Hermann Eichler, Patrick Moehnle, Christina Hart, Robert Klamroth, Andreas Tiede, Sonja Werwitzke

Blood (2025) 146 (Supplement 1): 310.

https://doi.org/10.1182/blood-2025-310

Published: 2025

Journal Articles

Elevation of liver health biomarkers before and after etranacogene dezaparvovec gene therapy in hemophilia B: Associations with long-term clinical outcomes

Wolfgang Miesbach, Steven Pipe, Cedric Hermans, Michael Recht, Miguel Escobar, Vaibhav Mahajan, Nathalie Jansen, Nicholas Galante, Rachel Church, Paul Monahan

Blood (2025) 146 (Supplement 1): 6093.

https://doi.org/10.1182/blood-2025-6093

Published: 2025

Journal Articles

Patient-reported outcomes of avatrombopag for chronic immune thrombocytopenia: Interim analysis of the Phase 4 adopt study

Maria Eva Mingot, María Teresa Alvarez-Román, Maria Lozano, Wolfgang Miesbach, Hafiz Qureshi, Vickie McDonald, Jessica Zhang, Viridiana Cano Garcia, Nina Skuban

Blood (2025) 146 (Supplement 1): 6237.

https://doi.org/10.1182/blood-2025-6237

Published: 2025

Journal Articles

Stable Factor IX Expression and Sustained Reductions in Factor IX Use 8 Years after Gene Therapy with CSL220 (Formerly AMT-060) in Adults with Hemophilia B

Frank W.G. Leebeek, Karina Meijer, Michiel Coppens, Peter Kampmann, Dr. Klamroth, Paul van der Valk, Paul E. Monahan, Karen Pinachyan, Sandra LeQuellec, Wolfgang Miesbach

Blood (2024) 144 (Supplement 1): 3578.

https://doi.org/10.1182/blood-2024-198379

Published: 2024

Journal Articles

Use of Avatrombopag in Patients with Immune Thrombocytopenia: Interim Analysis of the Phase 4 Adopt Study

María Eva Mingot-Castellano, María Teresa Alvarez-Román, María Luisa Lozano, Wolfgang Miesbach, Hafiz Qureshi, Vickie McDonald, Milica Putnik, Jessica Zhang, Nina Skuban, Viridiana Cano Garcia, Stefan Lethagen

Blood (2024) 144 (Supplement 1): 714.

https://doi.org/10.1182/blood-2024-203491

Published: 2024

Journal Articles

Low Rebleed Incidence with Eptacog Beta Treatment through 24 and 48 Hours in Children (<12 years) with Hemophilia a or B with Inhibitors

Blood (2023) 142 (Supplement 1): 5480.

https://doi.org/10.1182/blood-2023-181299

Published: 2023

Journal Articles

Long-Term Bleeding Protection, Sustained FIX Activity, Reduction of FIX Consumption and Safety of Hemophilia B Gene Therapy: Results from the HOPE-B Trial 3 Years after Administration of a Single Dose of Etranacogene Dezaparvovec in Adult Patients with Severe or Moderately Severe Hemophilia B

Blood (2023) 142 (Supplement 1): 1055.

https://doi.org/10.1182/blood-2023-187624

Published: 2023

Journal Articles

Emicizumab Versus Immunosuppression for Acquired Hemophilia Α (AHA)

Christina Hart, Robert Klamroth, Ulrich J. Sachs, Richard Greil, Paul N. Knoebl, Johannes Oldenburg, Wolfgang Miesbach, Christian Pfrepper, Karolin Trautmann-Grill, Isabell Pekrul, Katharina Holstein, Hermann Eichler, Carmen Weigt, Dorothea Schipp, Sonja Werwitzke, Andreas Tiede

Blood (2023) 142 (Supplement 1): 2612.

https://doi.org/10.1182/blood-2023-174958

Published: 2023

Journal Articles

Gene therapy for hemophilia: a review on clinical benefit, limitations, and remaining issues

Frank W. G. Leebeek, Wolfgang Miesbach

Blood (2021) 138 (11): 923–931.

https://doi.org/10.1182/blood.2019003777

Published: 2021

Journal Articles

Bleeding and response to hemostatic therapy in acquired hemophilia A: results from the GTH-AH 01/2010 study

Katharina Holstein, Xiaofei Liu, Andrea Smith, Paul Knöbl, Robert Klamroth, Ulrich Geisen, Hermann Eichler, Wolfgang Miesbach, Andreas Tiede

Blood (2020) 136 (3): 279–287.

https://doi.org/10.1182/blood.2019003639

Published: 2020

Includes: Supplemental data

Journal Articles

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

Steven Pipe, MD, Adam Giermasz, MD PhD, Giancarlo Castaman, MD, Nigel S. Key, MBChB, FRCP, Susan U Lattimore, Frank Leebeek, MD PhD, Wolfgang Miesbach, MD PhD, Michael Recht, MD PhD, Esteban Gomez, MD, Alison Long, Robert Gut, Annette von Drygalski, MD PharmD

Blood (2019) 134 (Supplement_1): 3348.

https://doi.org/10.1182/blood-2019-128765

Published: 2019

Journal Articles

Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for up to 4 Years Following AMT-060 Gene Therapy in Adults with Severe or Moderate-Severe Hemophilia B

Wolfgang Miesbach, MD PhD, Karina Meijer, Michiel Coppens, MD, Peter Kampmann, MD, Dr. Klamroth, Roger Schutgens, MD PhD MSc, Giancarlo Castaman, MD, Erhard Seifried, MD PhD, Joachim Schwaeble, Halvard Bönig, Eileen K Sawyer, PhD, Frank W.G. Leebeek, MD PhD

Blood (2019) 134 (Supplement_1): 2059.

https://doi.org/10.1182/blood-2019-122535

Published: 2019

Journal Articles

Development of a Human in Vitro Cell Model for Haemophilia a

Rosa Toenges, MD, Nadin Hodroj, Nina Kurrle, PhD, Wolfgang Miesbach, MD PhD, Frank Schnuetgen, PhD, Hubert Serve, MD

Blood (2019) 134 (Supplement_1): 3634.

https://doi.org/10.1182/blood-2019-121908

Published: 2019

Journal Articles

Gene therapy with adeno-associated virus vector 5–human factor IX in adults with hemophilia B

Wolfgang Miesbach, Karina Meijer, Michiel Coppens, Peter Kampmann, Robert Klamroth, Roger Schutgens, Marco Tangelder, Giancarlo Castaman, Joachim Schwäble, Halvard Bonig, Erhard Seifried, Federica Cattaneo, Christian Meyer, Frank W. G. Leebeek

Blood (2018) 131 (9): 1022–1031.

https://doi.org/10.1182/blood-2017-09-804419

Published: 2018

Includes: Supplemental data

Journal Articles

Prognostic factors for remission of and survival in acquired hemophilia A (AHA): results from the GTH-AH 01/2010 study

Blood (2015) 125 (7): 1091–1097.

https://doi.org/10.1182/blood-2014-07-587089

Published: 2015

Includes: Supplemental data

Journal Articles

Engineered factor IX variants bypass FVIII and correct hemophilia A phenotype in mice

Peter Milanov, Lacramioara Ivanciu, Daniela Abriss, Patricia Quade-Lyssy, Wolfgang Miesbach, Sonja Alesci, Torsten Tonn, Manuel Grez, Erhard Seifried, Jörg Schüttrumpf

Blood (2012) 119 (2): 602–611.

https://doi.org/10.1182/blood-2011-05-353672

Published: 2012

Journal Articles

Hepatocellular Carcinoma in Patients with Hemophilia and Chronic Hepatitis C Infection.

Charis von Auer, MD, Sandra Heinsdorf, Manuela Krause, MD, Wolfgang Miesbach, MD, Inge Scharrer, MD

Blood (2006) 108 (11): 4056.

https://doi.org/10.1182/blood.V108.11.4056.4056

Published: 2006

Journal Articles

Factor VIII Inhibitors in Haemophilia A Patients after Continuous Infusion of Factor VIII Concentrates.

Charis von Auer, MD, Johannes Oldenburg, MD, Manuela Krause, MD, Wolfgang Miesbach, MD, Inge Scharrer, CI-Study Group in Germany

Blood (2005) 106 (11): 3198.

https://doi.org/10.1182/blood.V106.11.3198.3198

Published: 2005

Journal Articles

Peginterferon Alfa 2a: Treatment Option for Coagulopathy Associated with Vascular Malformation?.

Charis von Auer, MD, Manuela Krause, MD, Wolfgang Miesbach, MD, Inge Scharrer

Blood (2005) 106 (11): 4104.

https://doi.org/10.1182/blood.V106.11.4104.4104

Published: 2005

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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

Using FXIa-triggered thrombin generation to assess the risk of breakthrough bleeding in patients receiving emicizumab for acquired Hemophilia A

Elevation of liver health biomarkers before and after etranacogene dezaparvovec gene therapy in hemophilia B: Associations with long-term clinical outcomes

Patient-reported outcomes of avatrombopag for chronic immune thrombocytopenia: Interim analysis of the Phase 4 adopt study

Stable Factor IX Expression and Sustained Reductions in Factor IX Use 8 Years after Gene Therapy with CSL220 (Formerly AMT-060) in Adults with Hemophilia B

Use of Avatrombopag in Patients with Immune Thrombocytopenia: Interim Analysis of the Phase 4 Adopt Study

Low Rebleed Incidence with Eptacog Beta Treatment through 24 and 48 Hours in Children (<12 years) with Hemophilia a or B with Inhibitors

Long-Term Bleeding Protection, Sustained FIX Activity, Reduction of FIX Consumption and Safety of Hemophilia B Gene Therapy: Results from the HOPE-B Trial 3 Years after Administration of a Single Dose of Etranacogene Dezaparvovec in Adult Patients with Severe or Moderately Severe Hemophilia B

Emicizumab Versus Immunosuppression for Acquired Hemophilia Α (AHA)

Gene therapy for hemophilia: a review on clinical benefit, limitations, and remaining issues

Bleeding and response to hemostatic therapy in acquired hemophilia A: results from the GTH-AH 01/2010 study

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

Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for up to 4 Years Following AMT-060 Gene Therapy in Adults with Severe or Moderate-Severe Hemophilia B

Development of a Human in Vitro Cell Model for Haemophilia a

Gene therapy with adeno-associated virus vector 5–human factor IX in adults with hemophilia B

Prognostic factors for remission of and survival in acquired hemophilia A (AHA): results from the GTH-AH 01/2010 study

Engineered factor IX variants bypass FVIII and correct hemophilia A phenotype in mice

Hepatocellular Carcinoma in Patients with Hemophilia and Chronic Hepatitis C Infection.

Factor VIII Inhibitors in Haemophilia A Patients after Continuous Infusion of Factor VIII Concentrates.

Peginterferon Alfa 2a: Treatment Option for Coagulopathy Associated with Vascular Malformation?.

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