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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

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

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

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

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

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

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

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

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

Journal Articles

Factor VIII Inhibitors in Haemophilia A Patients Who Are Considered as Previously Treated Patients.

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

Blood (2005) 106 (11): 4105.

https://doi.org/10.1182/blood.V106.11.4105.4105

Published: 2005

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

Acquired Hemophilia Caused by Non-Hemophilic Factor VIII Gene Variants.

Andreas Tiede, MD, PhD, Roswith Eisert, MD, Andreas Czwalinna, PhD, Wolfgang Miesbach, MD, Inge Scharrer, MD, Arnold Ganser, MD, Mario von Depka, MD

Blood (2004) 104 (11): 3083.

https://doi.org/10.1182/blood.V104.11.3083.3083

Published: 2004

Journal Articles

The Frequency and the Significance of ADAMTS-13 Neutralising Inhibitors in 62 Patients with Non-Familial Thrombotic Thrombocytopenic Purpura.

Martina Böhm, PhD, Manuela Krause, MD, Charis Von Auer, MD, Wolfgang Miesbach, MD, Inge Scharrer, MD

Blood (2004) 104 (11): 3945.

https://doi.org/10.1182/blood.V104.11.3945.3945

Published: 2004

Journal Articles

Effect of FV-Leiden Mutation and Antiphospholipid Syndrome on Thethrombin GenerationTest.

Ferial Peyvandi, MD, Wolfgang Miesbach, MD, Wolfgang Wegert, PhD, Inge Scharrer, MD

Blood (2004) 104 (11): 3506.

https://doi.org/10.1182/blood.V104.11.3506.3506

Published: 2004

NARROW

Use of Avatrombopag in Patients with 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

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

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

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

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

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

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.

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

Factor VIII Inhibitors in Haemophilia A Patients Who Are Considered as Previously Treated Patients.

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

Acquired Hemophilia Caused by Non-Hemophilic Factor VIII Gene Variants.

The Frequency and the Significance of ADAMTS-13 Neutralising Inhibitors in 62 Patients with Non-Familial Thrombotic Thrombocytopenic Purpura.

Effect of FV-Leiden Mutation and Antiphospholipid Syndrome on Thethrombin GenerationTest.

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American Society of Hematology

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ASH Publications

American Society of Hematology

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