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Journal Articles

Pain Reduction Following Treatment of Bleeding Episodes with Eptacog Beta in Adults and Adolescents with Hemophilia A or B Complicated By Inhibitors

Tyler W. Buckner, Craig Kessler, Giancarlo Castaman, Cédric Hermans, Victor Jimenez-Yuste, Johnny Mahlangu, Wolfgang A. Miesbach, Johannes Oldenburg, Michael Recht, Leonard A. Valentino, Allison P Wheeler, Steven W. Pipe

Blood (2024) 144 (Supplement 1): 3972.

https://doi.org/10.1182/blood-2024-194935

Published: 2024

Journal Articles

Sustained Improvement of Survival in Patients Receiving Emicizumab Prophylaxis Instead of Immunosuppression during Early Management of Acquired Hemophilia a (AHA)

Inga M. Schimansky, Andreas Tiede, Christiane Dobbelstein, Dr. Robert Klamroth, Christina Hart, Ulrich J. Sachs, Richard Greil, Paul N. Knöbl, Johannes Oldenburg, Christian Pfrepper, Karolin Trautmann-Grill, Patrick Moehnle, Katharina Holstein, Wolfgang A. Miesbach

Blood (2024) 144 (Supplement 1): 555.

https://doi.org/10.1182/blood-2024-205490

Published: 2024

Journal Articles

Durability of Factor IX Activity and Bleeding Rate in People with Severe or Moderately Severe Hemophilia B after 5 Years of Follow-up in the Phase 1/2 Study of AMT-060, and after 3 Years of Follow-up in the Phase 2b and 2 Years of Follow-up in the Phase 3 Studies of Etranacogene Dezaparvovec (AMT-061)

Wolfgang A. Miesbach, Michael Recht, Nigel S. Key, Krupa Sivamurthy, Paul E. Monahan, Steven W. Pipe

Blood (2022) 140 (Supplement 1): 4913–4914.

https://doi.org/10.1182/blood-2022-166810

Published: 2022

Journal Articles

Adults with Severe or Moderately Severe Hemophilia B Receiving Etranacogene Dezaparvovec in the HOPE-B Phase 3 Clinical Trial Continue to Experience a Stable Increase in Mean Factor IX Activity Levels and Durable Hemostatic Protection after 24 Months’ Follow-up

Steven W. Pipe, Frank W.G. Leebeek, Michael Recht, Nigel S. Key, Susan Lattimore, Giancarlo Castaman, Michiel Coppens, David Cooper, Robert Gut, Sergio Slawka, Stephanie Verweij, Ricardo Dolmetsch, Yanyan Li, Paul E. Monahan, Wolfgang A. Miesbach

Blood (2022) 140 (Supplement 1): 4910–4912.

https://doi.org/10.1182/blood-2022-166135

Published: 2022

Journal Articles

Durability of Bleeding Protection and Factor IX Activity Levels Are Demonstrated in Individuals with and without Adeno-Associated Virus Serotype 5 Neutralizing Antibodies (Titers <1:700) with Comparable Safety in the Phase 3 HOPE-B Clinical Trial of Etranacogene Dezaparvovec Gene Therapy for Hemophilia B

Steven W. Pipe, Frank W.G. Leebeek, Michael Recht, Nigel S. Key, Susan Lattimore, Giancarlo Castaman, David Cooper, Stephanie Verweij, Ricardo Dolmetsch, Jacqueline Tarrant, Yanyan Li, Paul E. Monahan, Wolfgang A. Miesbach

Blood (2022) 140 (Supplement 1): 4904–4906.

https://doi.org/10.1182/blood-2022-166745

Published: 2022

Journal Articles

Eptacog Beta (rFVIIa) Has a Low Incidence of Spontaneous Rebleeding through 24 and 48 Hours in Adult and Adolescent Patients with Hemophilia A or B with Inhibitors

Amy L. Dunn, Yesim Dargaud, Yasmina L. Abajas, Manuel Carcao, Giancarlo Castaman, Adam Giermasz, Cédric Hermans, Magdalena D. Lewandowska, Johnny Mahlangu, Shannon L. Meeks, Wolfgang A. Miesbach, Michael Recht, Vanessa Salinas, Tammuella Chrisentery-Singleton, Hongying Wang, Ian S Mitchell, Guy Young

Blood (2022) 140 (Supplement 1): 8485–8488.

https://doi.org/10.1182/blood-2022-157343

Published: 2022

Journal Articles

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

Steven W. Pipe, MD, Michael Recht, MD PhD, Nigel S. Key, MD, Frank W.G. Leebeek, MD PhD, Giancarlo Castaman, MD, Susan U. Lattimore, R.N., Paul Van Der Valk, Kathelijne Peerlinck, MD PhD, Michiel Coppens, MD, Niamh O'Connell, MD PhD, John Pasi, MB, ChB, PhD FRCP, FRCPath, FRCPCH, Peter Kampmann, MD, Karina Meijer, MD PhD, Annette von Drygalski, MD Pharm D, Guy Young, MD, Cedric Hermans, MDMRCP,PhD, Jan Astermark, MD PhD, Robert Klamroth, MD PhD, Richard S. Lemons, MD, Nathan Visweshwar, MD, Shelley Crary, MD MS, Rashid Kazmi, MBBS, Emily Symington, Miguel A. Escobar, MD, Esteban Gomez, MD, Rebecca Kruse-Jarres, MD, Adam Kotowski, MD, Doris Quon, MD PhD, Michael Wang, MD, Allison P. Wheeler, MD, Eileen K Sawyer, PhD, Stephanie Verweij, BSc, Valerie Colletta, MSc, Naghmana Bajma, MD, Robert Gut, MD PhD, Wolfgang A. Miesbach, MD PhD

Blood (2020) 136 (Supplement_2): LBA-6.

https://doi.org/10.1182/blood-2020-143560

Published: 2020

Journal Articles

AMT-060 Gene Therapy in Adults with Severe or Moderate-Severe Hemophilia B Confirm Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for up to 5 Years

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

Blood (2020) 136 (Supplement 1): 26.

https://doi.org/10.1182/blood-2020-139225

Published: 2020

Journal Articles

First-in-Human Phase 1/2 Clinical Trial of SIG-001, an Innovative Shielded Cell Therapy Platform, for Hemophilia Α

Amy D Shapiro, MD, Barbara A. Konkle, MD, Stacy E. Croteau, MD, Wolfgang A. Miesbach, MD, Charles Richard Morris Hay, MDFRCP,FRCPath, Rashid Kazmi, MBBS, Marina Mihova, Savita Rangarajan, MD MBBS, John Pasi, MB, ChB, PhD FRCP, FRCPath, FRCPCH

Blood (2020) 136 (Supplement 1): 8.

https://doi.org/10.1182/blood-2020-141381

Published: 2020

Journal Articles

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

Annette von Drygalski, Adam Giermasz, MD PhD, Giancarlo Castaman, MD, Nigel S. Key, MD, Susan U. Lattimore, Frank W.G. Leebeek, MD PhD, Wolfgang A. Miesbach, MD, Michael Recht, MD PhD, Esteban Gomez, MD, Robert Gut, Steven W. Pipe, MD

Blood (2020) 136 (Supplement 1): 13.

https://doi.org/10.1182/blood-2020-139295

Published: 2020

Journal Articles

Efficacy and Safety of BAY 94-9027 (Damoctocog Alfa Pegol) Prophylaxis in Patients with Severe Hemophilia a and Comorbidities: A Post Hoc Analysis of PROTECT VIII Data

Wolfgang A. Miesbach, Giovanni Di Minno, Elena Santagostino, Dr. Klamroth, Inga Bayh, Amaryllis Soto, Cedric Hermans

Blood (2019) 134 (Supplement_1): 1117.

https://doi.org/10.1182/blood-2019-128486

Published: 2019

Journal Articles

Stable Elevations in FIX Activity and Reductions in Annualized Bleeding Rate over up to 2 Years of Follow-up of Adults with Severe or Moderate-Severe Hemophilia B Treated with AMT-060 (AAV5-hFIX) Gene Therapy

Frank W.G. Leebeek, MD PhD, K Meijer, Michiel Coppens, MD PhD, Peter Kampmann, R Klamroth, Roger Schutgens, MD PhD, Giancarlo Castaman, Erhard Seifried, MD PhD, J Schwaeble, Halvard Bonig, MD MA, Eileen K Sawyer, Wolfgang A. Miesbach, MD PhD

Blood (2017) 130 (Supplement 1): 602.

https://doi.org/10.1182/blood.V130.Suppl_1.602.602

Published: 2017

Journal Articles

Factor VIII Activity and Risk of Bleeding in Acquired Hemophilia a: Results from the Gth-AH 01/2010 Study

Katharina Holstein, MD, Andrea Smith, Paul Knöbl, MD PhD, Christiane Dobbelstein, MD, Wolfgang A. Miesbach, MD PhD, Michael Spannagl, MD PhD, Jürgen Heinz, MD PhD, Ute Scholz, MD, Hermann Eichler, MD, Angela Huth-Kühne, MD, Robert Klamroth, MD PhD, Armin Koch, MD PhD, Andreas Tiede, MD

Blood (2017) 130 (Supplement 1): 22.

https://doi.org/10.1182/blood.V130.Suppl_1.22.22

Published: 2017

Journal Articles

Acquired Factor X Deficiency in Systemic Light Chain Amyloidosis: Factor X Recovery after Bleeding Complications, Splenectomy, Chemotherapy and Remission

Rosa Toenges, MD, Juliane Steinmann, MD, Annelie Siegemund, MD, Hubert Serve, Wolfgang A. Miesbach, MD PhD

Blood (2017) 130 (Supplement 1): 4892.

https://doi.org/10.1182/blood.V130.Suppl_1.4892.4892

Published: 2017

Journal Articles

Interim Results from a Dose Escalating Study of AMT-060 (AAV5-hFIX) Gene Transfer in Adult Patients with Severe Hemophilia B

Frank W.G. Leebeek, MD PhD, Marco Tangelder, MD PhD, Karina Meijer, MD PhD, Giancarlo Castaman, Federica Cattaneo, MD, Michiel Coppens, MD PhD, Peter Kampmann, MD, Robert Klamroth, Roger E.G. Schutgens, MD PhD, Erhard Seifried, MD PhD, Joachim Schwaeble, MD, Halvard Boenig, MD PhD, Maaike Hendriks, Deya Corzo, MD FAAP, Wolfgang A. Miesbach, MD PhD

Blood (2016) 128 (22): 2314.

https://doi.org/10.1182/blood.V128.22.2314.2314

Published: 2016

Journal Articles

Perception of Actual Factor Concentrates and Expectations Towards Future Treatment with New Long-Lasting Products - Results of a Survey Among Haemophilia Patients in Germany - Analysis of Open Questions

Werner Kalnins, Wolfgang A. Miesbach, MD PhD, Sylvia von Mackensen, PhD

Blood (2015) 126 (23): 3269.

https://doi.org/10.1182/blood.V126.23.3269.3269

Published: 2015

Journal Articles

Phase 1-2 Clinical Trial of a Recombinant AAV5 Vector Containing the Human FIX Gene in Patients with Severe or Moderately Severe Haemophilia B

Wolfgang A. Miesbach, MD PhD, Christian Meyer, MD PhD, Bart Nijmeijer, PhD, Florence Salmon, PhD, Nadina Grosios, PhD, Harald Petry, PhD, Frank W.G. Leebeek, MD PhD

Blood (2014) 124 (21): 5948.

https://doi.org/10.1182/blood.V124.21.5948.5948

Published: 2014

Journal Articles

Final Evaluation Of a Prospective, Non-Interventional Study On Efficacy and Tolerability Of a New Generation VWF/FVIII Concentrate In The Treatment Of Von Willebrand Disease

Sonja Alesci, MD, Mario Von Depka, Jennifer Feddern, Susan Halimeh, MD, Oliver Hegener, PhD, Janos Kadar, Wolfgang A. Miesbach, MD, PhD, Ulrike Nowak-Göttl, Inge Scharrer, MD, PhD

Blood (2013) 122 (21): 4775.

https://doi.org/10.1182/blood.V122.21.4775.4775

Published: 2013

Journal Articles

Prognostic Parameters For Remission Of and Survival In Acquired Hemophilia A: Results Of The GTH-AH 01/2010 Multicenter Study

Andreas Tiede, MD, PhD, Jan-Malte Blumtritt, Robert Klamroth, MD, PhD, Saskia Gottstein, MD, Katharina Holstein, MD, Jürgen Heinz, MD, Rüdiger E. Scharf, MD, PhD, Angela Huth-Kühne, MD, PhD, Joachim F. Schenk, MD, PhD, Richard Greil, MD, Wolfgang A. Miesbach, MD, PhD, Ralf Ulrich Trappe, MD, PhD, Ute Scholz, MD, Daniela Manner, MD, Peter Neumeister, MD, Charis von Auer-Wegener, MD, Ulrich Kalus, MD, Klaus Leimkühler, MD, Paul Knoebl, MD

Blood (2013) 122 (21): 205.

https://doi.org/10.1182/blood.V122.21.205.205

Published: 2013

Journal Articles

Descriptive Evaluation of Age-Related Differences in Clinical Manifestation and Treatment of Von Willebrand's Disease in an Open-Label, Prospective, Non-Interventional Study (wilate ® -SET)

Oliver Hegener, PhD, Inge Scharrer, MD, PhD, Jennifer Feddern, Wolfgang A. Miesbach V, MD, PhD, Mario Von Depka, Janos Kadar

Blood (2012) 120 (21): 4630.

https://doi.org/10.1182/blood.V120.21.4630.4630

Published: 2012

NARROW

Pain Reduction Following Treatment of Bleeding Episodes with Eptacog Beta in Adults and Adolescents with Hemophilia A or B Complicated By Inhibitors

Sustained Improvement of Survival in Patients Receiving Emicizumab Prophylaxis Instead of Immunosuppression during Early Management of Acquired Hemophilia a (AHA)

Adults with Severe or Moderately Severe Hemophilia B Receiving Etranacogene Dezaparvovec in the HOPE-B Phase 3 Clinical Trial Continue to Experience a Stable Increase in Mean Factor IX Activity Levels and Durable Hemostatic Protection after 24 Months’ Follow-up

Eptacog Beta (rFVIIa) Has a Low Incidence of Spontaneous Rebleeding through 24 and 48 Hours in Adult and Adolescent Patients with Hemophilia A or B with Inhibitors

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

AMT-060 Gene Therapy in Adults with Severe or Moderate-Severe Hemophilia B Confirm Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for up to 5 Years

First-in-Human Phase 1/2 Clinical Trial of SIG-001, an Innovative Shielded Cell Therapy Platform, for Hemophilia Α

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

Efficacy and Safety of BAY 94-9027 (Damoctocog Alfa Pegol) Prophylaxis in Patients with Severe Hemophilia a and Comorbidities: A Post Hoc Analysis of PROTECT VIII Data

Stable Elevations in FIX Activity and Reductions in Annualized Bleeding Rate over up to 2 Years of Follow-up of Adults with Severe or Moderate-Severe Hemophilia B Treated with AMT-060 (AAV5-hFIX) Gene Therapy

Factor VIII Activity and Risk of Bleeding in Acquired Hemophilia a: Results from the Gth-AH 01/2010 Study

Acquired Factor X Deficiency in Systemic Light Chain Amyloidosis: Factor X Recovery after Bleeding Complications, Splenectomy, Chemotherapy and Remission

Interim Results from a Dose Escalating Study of AMT-060 (AAV5-hFIX) Gene Transfer in Adult Patients with Severe Hemophilia B

Perception of Actual Factor Concentrates and Expectations Towards Future Treatment with New Long-Lasting Products - Results of a Survey Among Haemophilia Patients in Germany - Analysis of Open Questions

Phase 1-2 Clinical Trial of a Recombinant AAV5 Vector Containing the Human FIX Gene in Patients with Severe or Moderately Severe Haemophilia B

Final Evaluation Of a Prospective, Non-Interventional Study On Efficacy and Tolerability Of a New Generation VWF/FVIII Concentrate In The Treatment Of Von Willebrand Disease

Prognostic Parameters For Remission Of and Survival In Acquired Hemophilia A: Results Of The GTH-AH 01/2010 Multicenter Study

Descriptive Evaluation of Age-Related Differences in Clinical Manifestation and Treatment of Von Willebrand's Disease in an Open-Label, Prospective, Non-Interventional Study (wilate ® -SET)

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American Society of Hematology

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American Society of Hematology

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