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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

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

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

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

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

Elevated Levels of Circulating Nucleosomes Are Not Associated with Venous Thrombosis or Neutrophil Activation in Patients with Multiple Myeloma

Mandy N. Lauw, MD PhD, Frank W.G. Leebeek, MD PhD, Moniek P.M. de Maat, PhD, Gerard J. van Mierlo, Saskia Middeldorp, MD PhD, Sacha S. Zeerleder, MD PhD

Blood (2016) 128 (22): 274.

https://doi.org/10.1182/blood.V128.22.274.274

Published: 2016

Journal Articles

Label Free Quantitative Mass Spectrometry Identifies Processes Linked to Platelet Degranulation As Early Events during Platelet Storage

Maaike Rijkers, Floris P. van Alphen, BSc, Pieter F. van der Meer, PhD, Dirk de Korte, PhD, Frank W.G. Leebeek, MD PhD, Alexander B. Meijer, PhD, Jan Voorberg, PhD, Gerard AJG Jansen, MD PhD

Blood (2016) 128 (22): 2638.

https://doi.org/10.1182/blood.V128.22.2638.2638

Published: 2016

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

Clinical Joint Outcome after Joint Bleeds in Patients with Von Willebrand Disease Is Comparable to Moderate and Severe Hemophilia A Despite Fewer Joint Bleeds

Karin PM van Galen, MD, Merel Timmer, Mrs., Piet de Kleijn, Mr., Frank W.G. Leebeek, MD PhD, Roger E.G. Schutgens, MD PhD, Kathelijn Fischer, Eveline Mauser-Bunschoten

Blood (2016) 128 (22): 3789.

https://doi.org/10.1182/blood.V128.22.3789.3789

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

Perioperative FVIII Concentrate Treatment in Mild Hemophilia a Patients Shows a High Rate of Overdosing - David/Opti-Clot Studies

Lisette M. Schütte, MD MSc, Nils de Rooij, BSc, Hendrika C.A.M. Hazendonk, MD MSc, Ron A.A. Mathôt, PharmD, PhD, Reinier M. van Hest, PharmD, PhD, Frank W.G. Leebeek, MD PhD, Marjon H. Cnossen, MD PhD, Marieke J.H.A. Kruip, MD PhD

Blood (2015) 126 (23): 3510.

https://doi.org/10.1182/blood.V126.23.3510.3510

Published: 2015

Journal Articles

Targeting Clotting Factor VIII Plasma Values in the Perioperative Setting in Hemophilia a: “Tackling a Moving Target” (“OPTI-CLOT” Studies)

Janske Lock, MD PhDstudent, Hendrika C.A.M. Hazendonk, MD PhDstudent, Ron A.A. Mathôt, Prof. Dr., Karina Meijer, MD PhD, Marjolein Peters, MD PhD, Paul Brons, Felix J.M. Van der Meer, MD PhD, Hannan Bouzariouh, MD, Mariette H.E. Driessens, PhD, Karin Fijnvandraat, MD PhD, Frank W.G. Leebeek, MD PhD, Marjon H. Cnossen, MD PhD

Blood (2014) 124 (21): 5031.

https://doi.org/10.1182/blood.V124.21.5031.5031

Published: 2014

Journal Articles

Towards Successful Implementation of Pharmacokinetic-Guided Prophylactic Dosing of Clotting Factor Concentrate in Hemophilia; The Do’s and Don’ts after Discrete Choice Experiment Analysis

Janske Lock, MD PhDstudent, Esther W. de Bekker-Grob, PhD, Gamze Urhan, MD, Saskia Middeldorp, MD PhD, Marjolein Peters, MD PhD, Karina Meijer, MD PhD, Rienk Y.J. Tamminga, MD PhD, Britta Laros-van Gorkum, MD PhD, Paul Brons, Felix J.M. Van der Meer, MD PhD, Mariette H.E. Driessens, PhD, Peter W. Collins, MD, Karin Fijnvandraat, MD PhD, Frank W.G. Leebeek, MD PhD, Marjon H. Cnossen, MD PhD

Blood (2014) 124 (21): 5038.

https://doi.org/10.1182/blood.V124.21.5038.5038

Published: 2014

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

NARROW

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

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

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

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.

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

Elevated Levels of Circulating Nucleosomes Are Not Associated with Venous Thrombosis or Neutrophil Activation in Patients with Multiple Myeloma

Label Free Quantitative Mass Spectrometry Identifies Processes Linked to Platelet Degranulation As Early Events during Platelet Storage

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

Clinical Joint Outcome after Joint Bleeds in Patients with Von Willebrand Disease Is Comparable to Moderate and Severe Hemophilia A Despite Fewer Joint Bleeds

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

Perioperative FVIII Concentrate Treatment in Mild Hemophilia a Patients Shows a High Rate of Overdosing - David/Opti-Clot Studies

Targeting Clotting Factor VIII Plasma Values in the Perioperative Setting in Hemophilia a: “Tackling a Moving Target” (“OPTI-CLOT” Studies)

Towards Successful Implementation of Pharmacokinetic-Guided Prophylactic Dosing of Clotting Factor Concentrate in Hemophilia; The Do’s and Don’ts after Discrete Choice Experiment Analysis

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

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American Society of Hematology

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American Society of Hematology

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