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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

Characterization and Quantification of Von Willebrand Factor Proteoforms in Von Willebrand Disease Patients and Healthy Controls: Insights from the Win-Study

Iris charlotte Kreft, Tirsa T van Duijl, Calvin Van Kwawegen, Winny Phan, Margo Schuller, Carmen van Der Zwaan, Alexander Meijer, Ruben Bierings, Frank W.G. Leebeek, Maartje Van Den Biggelaar

Blood (2023) 142 (Supplement 1): 2606.

https://doi.org/10.1182/blood-2023-185987

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

Predicting Platelet Age Using Artificial Intelligence Techniques

Johan Slotman, Maurice Swinkels, Petra E. Burgisser, Joyce Bestebroer, Thomas R.L. Klei, Adriaan B. Houtsmuller, Frank W.G. Leebeek, Ruben Bierings, A.J. Gerard Jansen

Blood (2022) 140 (Supplement 1): 2656–2657.

https://doi.org/10.1182/blood-2022-159033

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

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

Efficacy and Safety of Recombinant Factor IX-FIAV and Bypassing Agents in Thrombin Generation Analyses in Hemophilia A Patient Plasma: The FIVITAS Study

Lorenzo G.R. Romano, Viola J.F. Strijbis, Ka Lei Cheung, Ying Poi Liu, Andrew C. McCreary, Frank W.G. Leebeek, Mettine H.A. Bos

Blood (2021) 138 (Supplement 1): 3192.

https://doi.org/10.1182/blood-2021-150492

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

Low Von Willebrand Factor: Cut-Off Value for Diagnosis and Risk Score to Predict Bleeding Incidence

Ferdows Atiq, Esmee Wuijster, BSc, Moniek P.M. de Maat, PhD, Marieke J.H.A. Kruip, MD PhD, Marjon H. Cnossen, MD PhD, Frank W.G. Leebeek, MD PhD

Blood (2020) 136 (Supplement 1): 18–19.

https://doi.org/10.1182/blood-2020-142375

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

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

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

Super-Resolution Immunofluorescence Imaging of Platelet Granules

Maurice Swinkels, MSc, BSc, Johan A. Slotman, PhD, Frank W.G. Leebeek, MD PhD, Jan Voorberg, PhD, Ruben Bierings, PhD, Gerard Jansen, MD PhD

Blood (2019) 134 (Supplement_1): 3613.

https://doi.org/10.1182/blood-2019-124353

Published: 2019

Journal Articles

VWF and FVIII Levels after Desmopressin Are Associated with the Bleeding Phenotype in Type 1 VWD

Ferdows Atiq, Lisette M. Schütte, MD, Agnes E.M. Looijen, Bsc, Johan Boender, MD, Moniek P.M. de Maat, PhD, Marieke J.H.A. Kruip, MD PhD, Frank W.G. Leebeek, MD PhD

Blood (2019) 134 (Supplement_1): 1116.

https://doi.org/10.1182/blood-2019-123634

Published: 2019

Journal Articles

Clustering of Bleeding Symptoms in Patients Previously Diagnosed As Type 3 Von Willebrand Disease: Results from a Large Cohort of Type 3 Von Willebrand Disease (the 3Winters-Ips Project)

Blood (2018) 132 (Supplement 1): 2465.

https://doi.org/10.1182/blood-2018-99-113996

Published: 2018

Journal Articles

Profile of Mutations Identified in the 3WINTERS-IPS Project on European & Iranian Patients with Previously Diagnosed Type 3 Von Willebrand Disease.

Luciano Baronciani, PhD, Flora Peyvandi, MD PhD, Anne Goodeve, PhD, Reinhard Schneppenheim, MDPhD, Zahra Badiee, Mohammad-Reza Baghaipour, Javier Battle, Erik Berntorp, MD PhD, Imre Bodó, MD PhD, Ulrich Budde, MD, Andrea Cairo, PhD, Giancarlo Castaman, Laura Crookes, Jeroen C.J. Eikenboom, MD, Peyman Eshghi, MD, Cosimo Ettorre, MD, Jenny Goudemand, MD PhD, Wolf Hassenpflug, Charles Richard Morris Hay, MDFRCP,FRCPath, Hamid Hoorfar, MD, Mehran Karimi, MD, Bijan Keikhaei, Riitta Lassila, MD PR, Frank W.G. Leebeek, MD PhD, Maria Fernanda Lopez Fernandez, Pier Mannuccio Mannucci, MD, Maria Gabriella Mazzucconi, MD, Massimo Morfini, MD, Nikolas Nikšić, Johannes Oldenburg, Florian Oyen, Rafael Parra Lòpez, Ian Peake, PhD, Gholamreza Toogeh, Alberto Tosetto, Marc Trossaert, Sayed Omid Reza Zekavat, MD, Eva M.K. Zetterberg, MD PhD, Augusto B Federici, MD

Blood (2018) 132 (Supplement 1): 1184.

https://doi.org/10.1182/blood-2018-99-114702

Published: 2018

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

Characterization and Quantification of Von Willebrand Factor Proteoforms in Von Willebrand Disease Patients and Healthy Controls: Insights from the Win-Study

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

Predicting Platelet Age Using Artificial Intelligence Techniques

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

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

Efficacy and Safety of Recombinant Factor IX-FIAV and Bypassing Agents in Thrombin Generation Analyses in Hemophilia A Patient Plasma: The FIVITAS Study

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

Low Von Willebrand Factor: Cut-Off Value for Diagnosis and Risk Score to Predict Bleeding Incidence

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

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

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

Super-Resolution Immunofluorescence Imaging of Platelet Granules

VWF and FVIII Levels after Desmopressin Are Associated with the Bleeding Phenotype in Type 1 VWD

Clustering of Bleeding Symptoms in Patients Previously Diagnosed As Type 3 Von Willebrand Disease: Results from a Large Cohort of Type 3 Von Willebrand Disease (the 3Winters-Ips Project)

Profile of Mutations Identified in the 3WINTERS-IPS Project on European & Iranian Patients with Previously Diagnosed Type 3 Von Willebrand Disease.

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American Society of Hematology

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ASH Publications

American Society of Hematology

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