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Journal Articles

Pharmacokinetic-Guided Reduction of Emicizumab in Patients with Congenital Hemophilia a in the Netherlands: Interim Analysis from the Dosemi Study

Konrad Van Der Zwet, Marjon H. Cnossen, Floor CJI Moenen, Saskia EM Schols, Paula F Ypma, Paul L. den Exter, Louise Hooimeijer, Michiel Coppens, Ron A.A. Mathôt, Anouk A.M.T. Donners, Alexander Janssen, Ilmar Kruis, Eduard J. Van Beers, Corien L. Eckhardt, Rolf T. Urbanus, Lize Fie Ditteke Van Vulpen, Roger EG Schutgens, Kathelijn Fischer

Blood (2024) 144 (Supplement 1): 127.

https://doi.org/10.1182/blood-2024-193995

Published: 2024

Journal Articles

Postpartum Hemorrhage in Hemophilia a and B Carriers after Enhanced Prophylactic Clotting Factor Suppletion: The Pregnancy and Inherited Bleeding Disorders Study (PRIDES)

Anne de Vaan, Marieke JHA Kruip, Jeroen Eikenboom, Marieke C Punt, Michiel Coppens, Laurens Nieuwenhuizen, Saskia EM Schols, Anja BU Mäkelburg, Floor CJI Moenen, Hans JJ Duvekot, Marjolein Peters, Annemieke JM Middeldorp, Kitty WM Bloemenkamp, Roger EG Schutgens, Titia AT Lely, Karin PM van Galen

Blood (2024) 144 (Supplement 1): 129.

https://doi.org/10.1182/blood-2024-207022

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

The Influence of Postpartum Hemorrhage on Patient Reported Outcomes Among Women with Von Willebrand Disease: The Pregnancy and Inherited Bleeding Disorders Study (PRIDES)

Anne de Vaan, Jeroen Eikenboom, Marieke JHA Kruip, Elke A Doeff, Marieke C Punt, Michiel Coppens, Laurens Nieuwenhuizen, Saskia EM Schols, Anja BU Mäkelburg, Floor CJI Moenen, Hans JJ Duvekot, Marjolein Peters, Annemieke JM Middeldorp, Kitty WM Bloemenkamp, Roger EG Schutgens, Karin PM van Galen

Blood (2024) 144 (Supplement 1): 2601.

https://doi.org/10.1182/blood-2024-207071

Published: 2024

Journal Articles

Postpartum Hemorrhage in Women with Von Willebrand Disease after Enhanced Prophylactic Clotting Factor Suppletion: The Pregnancy and Inherited Bleeding Disorders Study (PRIDES)

Anne De Vaan, Jeroen Eikenboom, Marieke JHA Kruip, Marieke C Punt, Michiel Coppens, Laurens Nieuwenhuizen, Saskia EM Schols, Anja BU Mäkelburg, Floor CJI Moenen, Hans JJ Duvekot, Marjolein Peters, Annemieke JM Middeldorp, Kitty WM Bloemenkamp, Roger EG Schutgens, Titia AT Lely, Karin PM Van Galen

Blood (2024) 144 (Supplement 1): 2595.

https://doi.org/10.1182/blood-2024-193986

Published: 2024

Journal Articles

The Influence of Postpartum Hemorrhage on the Quality of Life Among Pregnant Hemophilia a and B Carriers: The Pregnancy and Inherited Bleeding Disorders Study (PRIDES)

Anne de Vaan, Marieke JHA Kruip, Jeroen Eikenboom, Elke A Doeff, Marieke C Punt, Michiel Coppens, Laurens Nieuwenhuizen, Saskia EM Schols, Anja BU Mäkelburg, Floor CJI Moenen, Hans JJ Duvekot, Annemieke JM Middeldorp, Kitty WM Bloemenkamp, Roger EG Schutgens, Karin PM van Galen

Blood (2024) 144 (Supplement 1): 1208.

https://doi.org/10.1182/blood-2024-207237

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

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

Colorectal Cancer Screening in Patients with Inherited Bleeding Disorders: High Cancer Detection Rate in Hemophilia Patients

Eva Kempers, Calvin B. van Kwawegen, Joke de Meris, Saskia E.M. Schols, Paula F. Ypma, Floor C.J.I. Moenen, Lize F.D. Van Vulpen, Michiel Coppens, Johanna G. van der Bom, Karin Fijnvandraat, Karina Meijer, Jeroen C.J. Eikenboom, Samantha C. Gouw, Frank W.G. Leebeek, Marieke J.H.A. Kruip

Blood (2022) 140 (Supplement 1): 1416–1417.

https://doi.org/10.1182/blood-2022-157537

Published: 2022

Journal Articles

Incidence and mortality rates of intracranial hemorrhage in hemophilia: a systematic review and meta-analysis

Anne-Fleur Zwagemaker, Samantha C. Gouw, Julie S. Jansen, Caroline Vuong, Michiel Coppens, Qun Hu, Xiaoqin Feng, Soon K. Kim, Johanna G. Van der Bom, Karin Fijnvandraat

Blood (2021) 138 (26): 2853–2873.

https://doi.org/10.1182/blood.2021011849

Published: 2021

Includes: Supplemental data

Journal Articles

First-in-Human Dose-Finding Study of AAVhu37 Vector-Based Gene Therapy: BAY 2599023 Has Stable and Sustained Expression of FVIII over 2 Years

Steven W Pipe, John P Sheehan, Michiel Coppens, Hermann Eichler, Camila Linardi, Sara Wiegmann, Charles RM Hay, Toshko Lissitchkov

Blood (2021) 138 (Supplement 1): 3971.

https://doi.org/10.1182/blood-2021-148661

Published: 2021

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

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

Effects of Andexanet Alfa on Thrombin Generation in Bleeding Associated with Factor Xa Inhibitors

Michiel Coppens, MD, Lizhen Xu, PhD, Roisin Bavalia, MD, Saskia Middeldorp, MD PhD, Peter Verhamme, MD PhD, John W. Eikelboom, MBBS, MSc, FRACP, FRCPA, Mark Crowther, MD, Genmin Lu, Patrick Yue, MD, Pamela B. Conley, PhD, Stuart J. Connolly, MD MSc, FRCPC

Blood (2019) 134 (Supplement_1): 711.

https://doi.org/10.1182/blood-2019-124596

Published: 2019

Journal Articles

Perioperative Anticoagulant Use for Surgery Evaluation (PAUSE) Study: A Perioperative Management Plan for Patients with Atrial Fibrillation Who Are Receiving a Direct Oral Anticoagulant

Blood (2018) 132 (Supplement 1): LBA-5.

https://doi.org/10.1182/blood-2018-120770

Published: 2018

Journal Articles

Reduction in Annualized Bleeding and Factor IX Consumption up to 2.5 Years in Adults with Severe or Moderate-Severe Hemophilia B Treated with AMT-060 (AAV5-hFIX) Gene Therapy

Frank Leebeek, K Meijer, MD, Michiel Coppens, MD, Peter Kampmann, MD, Robert Klamroth, Roger Schutgens, MD PhD MSc, Giancarlo Castaman, Erhard Seifried, MD, J Schwaeble, MD, Halvard Bonig, MD, Eileen K Sawyer, PhD, W Miesbach

Blood (2018) 132 (Supplement 1): 3476.

https://doi.org/10.1182/blood-2018-99-109995

Published: 2018

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

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

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

NARROW

Pharmacokinetic-Guided Reduction of Emicizumab in Patients with Congenital Hemophilia a in the Netherlands: Interim Analysis from the Dosemi Study

Postpartum Hemorrhage in Hemophilia a and B Carriers after Enhanced Prophylactic Clotting Factor Suppletion: The Pregnancy and Inherited Bleeding Disorders Study (PRIDES)

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

The Influence of Postpartum Hemorrhage on Patient Reported Outcomes Among Women with Von Willebrand Disease: The Pregnancy and Inherited Bleeding Disorders Study (PRIDES)

Postpartum Hemorrhage in Women with Von Willebrand Disease after Enhanced Prophylactic Clotting Factor Suppletion: The Pregnancy and Inherited Bleeding Disorders Study (PRIDES)

The Influence of Postpartum Hemorrhage on the Quality of Life Among Pregnant Hemophilia a and B Carriers: The Pregnancy and Inherited Bleeding Disorders Study (PRIDES)

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

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

Colorectal Cancer Screening in Patients with Inherited Bleeding Disorders: High Cancer Detection Rate in Hemophilia Patients

Incidence and mortality rates of intracranial hemorrhage in hemophilia: a systematic review and meta-analysis

First-in-Human Dose-Finding Study of AAVhu37 Vector-Based Gene Therapy: BAY 2599023 Has Stable and Sustained Expression of FVIII over 2 Years

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

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

Effects of Andexanet Alfa on Thrombin Generation in Bleeding Associated with Factor Xa Inhibitors

Perioperative Anticoagulant Use for Surgery Evaluation (PAUSE) Study: A Perioperative Management Plan for Patients with Atrial Fibrillation Who Are Receiving a Direct Oral Anticoagulant

Reduction in Annualized Bleeding and Factor IX Consumption up to 2.5 Years in Adults with Severe or Moderate-Severe Hemophilia B Treated with AMT-060 (AAV5-hFIX) Gene Therapy

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

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

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

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American Society of Hematology

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American Society of Hematology

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