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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

Fourth Interim Analysis of the Hem Powr Study: A Post Hoc Analysis of Real-World Effectiveness and Safety of Damoctocog Alfa Pegol in Adolescent and Older Patients with Hemophilia a

Mark T Reding, María Teresa Alvarez-Román, Giancarlo Castaman, Maissaa Janbain, Tadashi Matsushita, Karina Meijer, Kathrin Schmidt, Johannes Oldenburg

Blood (2024) 144 (Supplement 1): 3968.

https://doi.org/10.1182/blood-2024-205121

Published: 2024

Journal Articles

Type 1 VWD classification revisited: novel insights from combined analysis of the LoVIC and WiN studies

Blood (2024) 143 (14): 1414–1424.

https://doi.org/10.1182/blood.2023022457

Published: 2024

Includes: Supplemental data

Journal Articles

Adult Patients with Hemophilia B and with a History of Chronic HCV/HBV Infection Receiving Liver-Directed Gene Therapy Demonstrated Long-Term Bleeding Protection and Sustained FIX Activity: Efficacy and Safety Results from the HOPE-B Trial 3 Years after Administration of a Single Dose of Etranacogene Dezaparvovec

Annette von Drygalski, Niamh O'Connell, Peter Verhamme, Karina Meijer, Paul van der Valk, Rashid Saeed Kazmi, Priyanka Raheja, Nicholas Galante, Sandra LeQuellec, Rachel Church, Samantha Lucas, Giancarlo Castaman, Paul Monahan

Blood (2023) 142 (Supplement 1): 2258.

https://doi.org/10.1182/blood-2023-188921

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

Age Regulates Desmopressin Responses in Patients with Low Von Willebrand Factor and Type 1 Von Willebrand Disease in the Lovic and Win Studies

Blood (2023) 142 (Supplement 1): 510.

https://doi.org/10.1182/blood-2023-184734

Published: 2023

Journal Articles

The Relationship between Low Von Willebrand Factor, Type 1 Von Willebrand Disease and Ageing - Novel Insights from the Lovic and Win Cohort Studies

Blood (2023) 142 (Supplement 1): 509.

https://doi.org/10.1182/blood-2023-181603

Published: 2023

Journal Articles

Interim Subgroup Analysis of the Effectiveness and Safety of Damoctocog Alfa Pegol Prophylaxis in Previously Treated Patients with Hemophilia A Treated Every 5 or Every 7 Days: Results from the Real-World Observational HEM-POWR Study

Mark T Reding, María Teresa Alvarez Román, Martin Sanabria, Giancarlo Castaman, Maissaa Janbain, Tadashi Matsushita, Karina Meijer, Kathrin Schmidt, Johannes Oldenburg

Blood (2022) 140 (Supplement 1): 5612–5614.

https://doi.org/10.1182/blood-2022-169159

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

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

Semiautomatic VWF Multimer Densitometric Analysis: Validation of the Clinical Accuracy and Clinical Implications in Von Willebrand Disease

Ferdows Atiq, Johan Boender, MD, Marjon H. Cnossen, MD PhD, Johanna G van der Bom, MD PhD, Karin Fijnvandraat, MD PhD, Joke de Meris, Moniek P.M. de Maat, PhD, Karin P.M. Van Galen, MD MSc, PhD, Britta A.P. Laros-Van Gorkom, MD PhD, Karina Meijer, MD PhD, Jeroen C.J. Eikenboom, MD PhD, Frank W.G. Leebeek, MD PhD

Blood (2020) 136 (Supplement 1): 15–16.

https://doi.org/10.1182/blood-2020-142287

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

Real-World Effectiveness and Safety of BAY 94-9027 (Damoctocog Alfa Pegol) in Previously Treated Patients with Hemophilia A (HEM-POWR): Online Patient Portal and LIFE-ACTIVE Sub-Study

Johannes Oldenburg, María Teresa Alvarez Román, Giancarlo Castaman, Maissaa Janbain, MD, Tadashi Matsushita, Karina Meijer, Sabine Friedl, Martin Sanabria, Mark Reding

Blood (2019) 134 (Supplement_1): 4943.

https://doi.org/10.1182/blood-2019-128140

Published: 2019

Journal Articles

Reduced incidence of vein occlusion and postthrombotic syndrome after immediate compression for deep vein thrombosis

Elham E. Amin, Ingrid M. Bistervels, Karina Meijer, Lidwine W. Tick, Saskia Middeldorp, Guy Mostard, Marlène van de Poel, Erik H. Serné, Hans M. Otten, Edith M. Klappe, Manuela A. Joore, Hugo ten Cate, Marije ten Wolde, Arina J. ten Cate-Hoek

Blood (2018) 132 (21): 2298–2304.

https://doi.org/10.1182/blood-2018-03-836783

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

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

No Association Between Normalization of VWF Levels and Bleeding Phenotype in Patients with Type 1 VWD - from the Win Study

Johan Boender, MD, Jeroen Eikenboom, MD PhD, Karin Fijnvandraat, MD PhD, Waander van Heerde, PhD, Karina Meijer, MD PhD, Evelien Mauser-Bunschoten, MD PhD, Marjon Cnossen, MD PhD, Joke de Meris, Britta Laros-van Gorkom, MD PhD, Anske van der Bom, MD PhD, Frank W.G. Leebeek, MD PhD

Blood (2016) 128 (22): 2577.

https://doi.org/10.1182/blood.V128.22.2577.2577

Published: 2016

Journal Articles

Incidence and Risk Factors for Venous Thrombosis in Human Immunodeficiency Virus (HIV) Infection, Data from the Dutch Athena Cohort Study

James Francis Borjas-Howard, MD MSc, Casper Rokx, MD PhD, Colette Smit, PhD, Ferdinand Wit, MD PhD, Elise Pieterman, MD MSc, Karina Meijer, MD PhD, Bart Rijnders, MD PhD, Wouter Bierman, MD PhD, Vladimir Tichelaar, MD PhD

Blood (2016) 128 (22): 271.

https://doi.org/10.1182/blood.V128.22.271.271

Published: 2016

Journal Articles

The Bleeding Risk of Vitamin K Antagonists Hardly Increases after the Age of 80 Years: Data from a Large Real-Life Cohort Study

Hilde Kooistra, MD, Agneta Calf, MD, Margriet Piersma-Wichers, MD, Gerbrand Izaks, MD, Nic Veeger, MSc, Karina Meijer, MD PhD

Blood (2015) 126 (23): 891.

https://doi.org/10.1182/blood.V126.23.891.891

Published: 2015

NARROW

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

Fourth Interim Analysis of the Hem Powr Study: A Post Hoc Analysis of Real-World Effectiveness and Safety of Damoctocog Alfa Pegol in Adolescent and Older Patients with Hemophilia a

Type 1 VWD classification revisited: novel insights from combined analysis of the LoVIC and WiN studies

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

Age Regulates Desmopressin Responses in Patients with Low Von Willebrand Factor and Type 1 Von Willebrand Disease in the Lovic and Win Studies

The Relationship between Low Von Willebrand Factor, Type 1 Von Willebrand Disease and Ageing - Novel Insights from the Lovic and Win Cohort Studies

Interim Subgroup Analysis of the Effectiveness and Safety of Damoctocog Alfa Pegol Prophylaxis in Previously Treated Patients with Hemophilia A Treated Every 5 or Every 7 Days: Results from the Real-World Observational HEM-POWR Study

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

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

Semiautomatic VWF Multimer Densitometric Analysis: Validation of the Clinical Accuracy and Clinical Implications in Von Willebrand Disease

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

Real-World Effectiveness and Safety of BAY 94-9027 (Damoctocog Alfa Pegol) in Previously Treated Patients with Hemophilia A (HEM-POWR): Online Patient Portal and LIFE-ACTIVE Sub-Study

Reduced incidence of vein occlusion and postthrombotic syndrome after immediate compression for deep vein thrombosis

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

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

No Association Between Normalization of VWF Levels and Bleeding Phenotype in Patients with Type 1 VWD - from the Win Study

Incidence and Risk Factors for Venous Thrombosis in Human Immunodeficiency Virus (HIV) Infection, Data from the Dutch Athena Cohort Study

The Bleeding Risk of Vitamin K Antagonists Hardly Increases after the Age of 80 Years: Data from a Large Real-Life Cohort Study

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American Society of Hematology

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American Society of Hematology

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