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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

Assessing Patient and Provider Perspectives, Clinical Practice, Behaviors, and Knowledge on Hemophilia A Care

Tariqa A Ackbarali, PhDc), Stacy E. Croteau, MD, Steven W. Pipe, MD, Kelly Tickle, MSN, APN, CWON, PCNS-BS, PPCNP-BC, Lori Long, Sonji Wilkes, BS, Kate Nammacher, MPH, Neil Frick, Katie Kowalski, Wendy Turell

Blood (2020) 136 (Supplement 1): 24–25.

https://doi.org/10.1182/blood-2020-141657

Published: 2020

Journal Articles

Long-Term Durability, Safety and Efficacy of Fitusiran Prophylaxis in People with Hemophilia a or B, with or without Inhibitors - Results from the Phase II Study

Steven W. Pipe, MD, John Pasi, MB, ChB, PhD FRCP, FRCPath, FRCPCH, Toshko Lissitchkov, Margaret V. Ragni, MDMPH, Claude Négrier, Qifeng Yu, Stacey Poloskey, Baisong Mei, Shauna R. Andersson

Blood (2020) 136 (Supplement 1): 3–4.

https://doi.org/10.1182/blood-2020-136501

Published: 2020

Journal Articles

Safety and Efficacy of Emicizumab in Persons with Hemophilia a with or without FVIII Inhibitors: Pooled Data from Four Phase III Studies (HAVEN 1-4)

Blood (2020) 136 (Supplement 1): 3–5.

https://doi.org/10.1182/blood-2020-137438

Published: 2020

Journal Articles

Inhibit Clinical Trials Platform to Prevent and Eradicate Inhibitors: Feasibility Survey of Current Prophylaxis and Immune Tolerance Practices

Blood (2020) 136 (Supplement 1): 14–15.

https://doi.org/10.1182/blood-2020-136814

Published: 2020

Journal Articles

First-in-Human Gene Therapy Study of AAVhu37 Capsid Vector Technology in Severe Hemophilia A - BAY 2599023 has Broad Patient Eligibility and Stable and Sustained Long-Term Expression of FVIII

Steven W. Pipe, MD, Francesca Ferrante, MD, Muriel Reis, Sara Wiegmann, Claudia Lange, Manuela Braun, Lisa A Michaels

Blood (2020) 136 (Supplement 1): 44–45.

https://doi.org/10.1182/blood-2020-139803

Published: 2020

Journal Articles

Longitudinal Assessment of Thrombin Generation in Patients with Hemophilia Receiving Fitusiran Prophylaxis: Phase II Study Results

Claude Négrier, Margaret V. Ragni, MDMPH, John Pasi, MB, ChB, PhD FRCP, FRCPath, FRCPCH, Steven W. Pipe, MD, Gili Kenet, Savita Rangarajan, MBBS,MDFRCP,FRCPath, Salim Kichou, Baisong Mei, Shauna R. Andersson

Blood (2020) 136 (Supplement 1): 36–37.

https://doi.org/10.1182/blood-2020-136536

Published: 2020

Journal Articles

In Vitro Evaluation of Thrombin Generation of Eptacog Beta (Factor VIIa, Recombinant) and Emicizumab in Congenital Hemophilia Α Plasma with and without Inhibitors

Steven W. Pipe, MD, Michael Recht, MD PhD, Michael U. Callaghan, MD, Robert F. Sidonio, Jr., MDMSc, Jerry Grandoni, PhD, Veronique Duretz, Daniel Bonzo, PhD, Jean Luc Plantier, PhD, Sean Evans, PhD RPh, Ian S Mitchell, PhD, Cedric Hermans, MDMRCP,PhD

Blood (2020) 136 (Supplement 1): 41–42.

https://doi.org/10.1182/blood-2020-136430

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

Modelling FVIII Levels for Prediction of Zero Spontaneous-Joint Bleeding in a Cohort of Severe Hemophilia a Subjects with Target Joints Initiated on Tertiary Prophylaxis

Kathelijn Fischer, MD PhD MSc, Pratima Chowdary, FRCPath, MRCPath, MRCP, MD, Peter Collins, MD, Amy Cotterill, PhD, Barbara Konkle, MD, Victor S. Blanchette, FRCP, FRCPC, Steven W. Pipe, MD, Martin Wolfsegger, Cstat (RSS), Werner Engl, PhD Cstat, Jimena V Goldstine, PhD, Leonard A. Valentino, MD FAAP, Gerald Spotts, PhD

Blood (2016) 128 (22): 2576.

https://doi.org/10.1182/blood.V128.22.2576.2576

Published: 2016

Journal Articles

Modeling Minimally-Effective FVIII Trough Levels in Hemophilia a Patients on PK-Guided Prophylaxis

Gerald Spotts, PhD, Steven W. Pipe, MD, Erik Berntorp, MD PhD, Peter W. Collins, MD, Victor S. Blanchette, MD FRCPC, Kathelijn Fischer, MD PhD MSC, MyungShin Oh, Leonard A. Valentino, MD FAAP

Blood (2014) 124 (21): 689.

https://doi.org/10.1182/blood.V124.21.689.689

Published: 2014

Journal Articles

Understanding Ectopically Expressed Factor VIII (F8) In Megakaryocytes: Implications for Optimum Platelet-Delivered F8 Activity for Gene Therapy

Teshell K Greene, B.S, Denise E. Sabatino, PhD, Nicholas P Iacobelli, B.S, Li Zhai, Steven W. Pipe, MD, M. Anna Kowalska, PhD, Rodney M. Camire, PhD, Valder Arruda, MD, PhD

Blood (2010) 116 (21): 2205.

https://doi.org/10.1182/blood.V116.21.2205.2205

Published: 2010

Journal Articles

Asparagine-Linked Glycosylation at the NH 2 -Terminus Does Not Influence Secretion or Function of Coagulation Factors V and VIII

Sundar Rajan Selvaraj, PhD, Steven W Pipe, MD

Blood (2010) 116 (21): 2220.

https://doi.org/10.1182/blood.V116.21.2220.2220

Published: 2010

Journal Articles

The CDC Hemostasis and Thrombosis Centers (HTC) Pilot Sites: Data From the Pediatric Registry.

Lisa A Michaels, MD, Michele Beckman, MPH, Courtney Thornburg, MD, MS, Kristy Marquez, MPH, Roshni Kulkarni, MD, Steven W. Pipe, MD, Stephan Moll, MD, Marilyn Manco-Johnson, MD

Blood (2009) 114 (22): 2990.

https://doi.org/10.1182/blood.V114.22.2990.2990

Published: 2009

Journal Articles

Strategies to Enhance the Efficacy of Platelet-Derived Factor (F) VIII: Studies with Inactivation Resistant FVIII (IR8) and Canine FVIII in Hemophilia A Mice.

Teshell K. Greene, BS, Cheng Wang, Jessica Hirsch, BS, Hongzhi Miao, Steven W. Pipe, MD, M. Anna Kowalska, PhD, Mortimer Poncz, MD

Blood (2009) 114 (22): 3496.

https://doi.org/10.1182/blood.V114.22.3496.3496

Published: 2009

Journal Articles

Risk for Post Thrombotic Syndrome (PTS) Development in Children with Extremity Deep Venous Thrombosis (DVT): Results of the US Centers for Disease Control and Prevention (CDC) Pediatric Thrombosis and Hemostasis Centers.

Marilyn J. Manco-Johnson, Michele G. Beckman, MPH, Neil A. Goldenberg, MD, PhD, Courtney Thornburg, MD, MS, Lisa A. Michaels, MD, Steven W. Pipe, MD, Stephan Moll, MD, Vilmarie Rodriquez, MD, Roshni Kulkarni

Blood (2009) 114 (22): 4000.

https://doi.org/10.1182/blood.V114.22.4000.4000

Published: 2009

Journal Articles

Strategies for Securing and Maintaining Predictable Efficacy with Recombinant Activated Factor VII in On-Demand Treatment of Haemophilia Patients with Inhibitors.

Benny Sørensen, MD, Ph.D., Yesim Dargaud, Gili Kenet, MD, PhD, Jeanne M. Lusher, MD, Andrew Mumford, Steven W. Pipe, MD, Andreas Tiede, MD, PhD

Blood (2009) 114 (22): 4443.

https://doi.org/10.1182/blood.V114.22.4443.4443

Published: 2009

Journal Articles

Membrane-Interactive Amino Acids in the Factor VIII C1 Domain Are Cooperative with C2 Domain Epitopes for Membrane Binding and Cofactor Function.

Junhong Lu, Ph.D., Steven W. Pipe, MD, Hongzhi Miao, Marc Jacquemin, Ph.D., Gary E. Gilbert, MD

Blood (2009) 114 (22): 848.

https://doi.org/10.1182/blood.V114.22.848.848

Published: 2009

Journal Articles

Incremental Improvement in Bioengineering of Coagulation Factor VIII for Efficient Expression: Elimination of a Dispensable Disulfide Loop Enhances Secretion

Sundar Rajan Selvaraj, PhD, Randal J Kaufman, PhD, Steven W. Pipe, MD

Blood (2008) 112 (11): 3073.

https://doi.org/10.1182/blood.V112.11.3073.3073

Published: 2008

Journal Articles

Reduction of Inhibitory Anti-FVIII Antibody Titer by Using a B Domain Variant FVIII/N6 cDNA for Nonviral Gene Therapy in Hemophilia a Mice

Dominika Jirovska, Peiqing Ye, Steven W. Pipe, MD, Carol H. Miao, PhD

Blood (2008) 112 (11): 3537.

https://doi.org/10.1182/blood.V112.11.3537.3537

Published: 2008

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

Assessing Patient and Provider Perspectives, Clinical Practice, Behaviors, and Knowledge on Hemophilia A Care

Long-Term Durability, Safety and Efficacy of Fitusiran Prophylaxis in People with Hemophilia a or B, with or without Inhibitors - Results from the Phase II Study

Safety and Efficacy of Emicizumab in Persons with Hemophilia a with or without FVIII Inhibitors: Pooled Data from Four Phase III Studies (HAVEN 1-4)

Inhibit Clinical Trials Platform to Prevent and Eradicate Inhibitors: Feasibility Survey of Current Prophylaxis and Immune Tolerance Practices

First-in-Human Gene Therapy Study of AAVhu37 Capsid Vector Technology in Severe Hemophilia A - BAY 2599023 has Broad Patient Eligibility and Stable and Sustained Long-Term Expression of FVIII

Longitudinal Assessment of Thrombin Generation in Patients with Hemophilia Receiving Fitusiran Prophylaxis: Phase II Study Results

In Vitro Evaluation of Thrombin Generation of Eptacog Beta (Factor VIIa, Recombinant) and Emicizumab in Congenital Hemophilia Α Plasma with and without Inhibitors

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

Modelling FVIII Levels for Prediction of Zero Spontaneous-Joint Bleeding in a Cohort of Severe Hemophilia a Subjects with Target Joints Initiated on Tertiary Prophylaxis

Modeling Minimally-Effective FVIII Trough Levels in Hemophilia a Patients on PK-Guided Prophylaxis

Understanding Ectopically Expressed Factor VIII (F8) In Megakaryocytes: Implications for Optimum Platelet-Delivered F8 Activity for Gene Therapy

Asparagine-Linked Glycosylation at the NH 2 -Terminus Does Not Influence Secretion or Function of Coagulation Factors V and VIII

The CDC Hemostasis and Thrombosis Centers (HTC) Pilot Sites: Data From the Pediatric Registry.

Strategies to Enhance the Efficacy of Platelet-Derived Factor (F) VIII: Studies with Inactivation Resistant FVIII (IR8) and Canine FVIII in Hemophilia A Mice.

Risk for Post Thrombotic Syndrome (PTS) Development in Children with Extremity Deep Venous Thrombosis (DVT): Results of the US Centers for Disease Control and Prevention (CDC) Pediatric Thrombosis and Hemostasis Centers.

Strategies for Securing and Maintaining Predictable Efficacy with Recombinant Activated Factor VII in On-Demand Treatment of Haemophilia Patients with Inhibitors.

Membrane-Interactive Amino Acids in the Factor VIII C1 Domain Are Cooperative with C2 Domain Epitopes for Membrane Binding and Cofactor Function.

Incremental Improvement in Bioengineering of Coagulation Factor VIII for Efficient Expression: Elimination of a Dispensable Disulfide Loop Enhances Secretion

Reduction of Inhibitory Anti-FVIII Antibody Titer by Using a B Domain Variant FVIII/N6 cDNA for Nonviral Gene Therapy in Hemophilia a Mice

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American Society of Hematology

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