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Journal Articles

Emicizumab prophylaxis in infants with hemophilia A (HAVEN 7): primary analysis of a phase 3b open-label trial

Steven W. Pipe, Peter Collins, Christophe Dhalluin, Gili Kenet, Christophe Schmitt, Muriel Buri, Víctor Jiménez-Yuste, Flora Peyvandi, Guy Young, Johannes Oldenburg, Maria Elisa Mancuso, Kaan Kavakli, Anna Kiialainen, Sonia Deb, Markus Niggli, Tiffany Chang, Michaela Lehle, Karin Fijnvandraat

Blood (2024) 143 (14): 1355–1364.

https://doi.org/10.1182/blood.2023021832

Published: 2024

Includes: Supplemental data

Journal Articles

Lifelong gene therapy in dogs with hemophilia A

Steven W. Pipe

Blood (2022) 140 (25): 2650–2652.

https://doi.org/10.1182/blood.2022016359

Published: 2022

Journal Articles

Durability of Factor IX Activity and Bleeding Rate in People with Severe or Moderately Severe Hemophilia B after 5 Years of Follow-up in the Phase 1/2 Study of AMT-060, and after 3 Years of Follow-up in the Phase 2b and 2 Years of Follow-up in the Phase 3 Studies of Etranacogene Dezaparvovec (AMT-061)

Wolfgang A. Miesbach, Michael Recht, Nigel S. Key, Krupa Sivamurthy, Paul E. Monahan, Steven W. Pipe

Blood (2022) 140 (Supplement 1): 4913–4914.

https://doi.org/10.1182/blood-2022-166810

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

Eptacog Beta Efficacy in Treating Mild or Moderate Bleeds in Target Joints of Individuals with Hemophilia A or B and Inhibitors in PERSEPT 1

Blood (2022) 140 (Supplement 1): 5597–5599.

https://doi.org/10.1182/blood-2022-156110

Published: 2022

Journal Articles

Emicizumab Prophylaxis for the Treatment of Infants with Severe Hemophilia A without Factor VIII Inhibitors: Results from the Interim Analysis of the HAVEN 7 Study

Steven W. Pipe, Peter Collins, Christophe Dhalluin, Gili Kenet, Christophe Schmitt, Muriel Buri, Víctor Jiménez-Yuste, Flora Peyvandi, Guy Young, Johannes Oldenburg, Maria Elisa Mancuso, Anna Kiialainen, Tiffany Chang, Michaela Lehle, Karin Fijnvandraat

Blood (2022) 140 (Supplement 1): 457–459.

https://doi.org/10.1182/blood-2022-157264

Published: 2022

Journal Articles

Characterization of Bleeding and Laboratory Phenotype of Carriers of Hemophilia and Trends of Utilization of Factor Concentrates and Hemostatic Therapies: Analysis of the National Athndataset

Neeraja Swaminathan, Brian Hu, Alison Currie, Roshni Kulkarni, Steven W. Pipe, Michael Recht, Peter A. Kouides, Anjali Sharathkumar

Blood (2022) 140 (Supplement 1): 59–60.

https://doi.org/10.1182/blood-2022-158861

Published: 2022

Journal Articles

Fitusiran, an Investigational siRNA Therapeutic Targeting Antithrombin for the Treatment of Hemophilia: First Results from a Phase 3 Study to Evaluate Efficacy and Safety in People with Hemophilia a or B without Inhibitors (ATLAS-A/B)

Alok Srivastava, Savita Rangarajan, Kaan Kavakli, Robert Klamroth, Gili Kenet, Liane Khoo, Chur-Woo You, Weiqun Xu, Niel Malan, Laurent Frenzel, Catherine N. Bagot, Oleksandra Stasyshyn, Chia-Yau Chang, Stacey Poloskey, Shauna Andersson, Zhiying Qiu, Baisong Mei, Steven W. Pipe

Blood (2021) 138 (Supplement 2): LBA-3.

https://doi.org/10.1182/blood-2021-155018

Published: 2021

Journal Articles

Eptacog Beta Efficacy in Children and Adolescents with Hemophilia A or B and Inhibitors: Subset Analysis Suggests Improved Caregiver Capacity to Assess Bleeding Episode Resolution with Subject Age

Blood (2021) 138 (Supplement 1): 3195.

https://doi.org/10.1182/blood-2021-147434

Published: 2021

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

Relationship between Endogenous, Transgene FVIII Expression and Bleeding Events Following Valoctocogene Roxaparvovec Gene Transfer for Severe Hemophilia A: A Post-Hoc Analysis of the GENEr8-1 Phase 3 Trial

Blood (2021) 138 (Supplement 1): 3972.

https://doi.org/10.1182/blood-2021-144508

Published: 2021

Journal Articles

Defibrotide Therapy for Sars CoV2 Acute Respiratory Distress Syndrome

Gregory A Yanik, David Frame, Gianni Bruno Scappaticci, Mary Maliarik, Steven W Pipe, Thomas Braun, Thomas Sisson, Paul G. Richardson, Daniel A Lawrence, Vibha Lama

Blood (2021) 138 (Supplement 1): 3237.

https://doi.org/10.1182/blood-2021-151417

Published: 2021

Journal Articles

Bleeding Data across Baseline FIX Expression Levels in People with Hemophilia B: An Analysis Using the 'Factor Expression Study'

Tom Burke, Anum Shaikh, Talaha Ali, Nanxin Li, Barbara A Konkle, Declan Noone, Brian O'Mahony, Steven W Pipe, Jamie O'Hara

Blood (2021) 138 (Supplement 1): 592.

https://doi.org/10.1182/blood-2021-152551

Published: 2021

Journal Articles

Long-term outcomes with emicizumab prophylaxis for hemophilia A with or without FVIII inhibitors from the HAVEN 1-4 studies

Blood (2021) 137 (16): 2231–2242.

https://doi.org/10.1182/blood.2020009217

Published: 2021

Includes: Supplemental data

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

NARROW

Emicizumab prophylaxis in infants with hemophilia A (HAVEN 7): primary analysis of a phase 3b open-label trial

Lifelong gene therapy in dogs with hemophilia A

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

Eptacog Beta Efficacy in Treating Mild or Moderate Bleeds in Target Joints of Individuals with Hemophilia A or B and Inhibitors in PERSEPT 1

Emicizumab Prophylaxis for the Treatment of Infants with Severe Hemophilia A without Factor VIII Inhibitors: Results from the Interim Analysis of the HAVEN 7 Study

Characterization of Bleeding and Laboratory Phenotype of Carriers of Hemophilia and Trends of Utilization of Factor Concentrates and Hemostatic Therapies: Analysis of the National Athndataset

Fitusiran, an Investigational siRNA Therapeutic Targeting Antithrombin for the Treatment of Hemophilia: First Results from a Phase 3 Study to Evaluate Efficacy and Safety in People with Hemophilia a or B without Inhibitors (ATLAS-A/B)

Eptacog Beta Efficacy in Children and Adolescents with Hemophilia A or B and Inhibitors: Subset Analysis Suggests Improved Caregiver Capacity to Assess Bleeding Episode Resolution with Subject Age

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

Relationship between Endogenous, Transgene FVIII Expression and Bleeding Events Following Valoctocogene Roxaparvovec Gene Transfer for Severe Hemophilia A: A Post-Hoc Analysis of the GENEr8-1 Phase 3 Trial

Defibrotide Therapy for Sars CoV2 Acute Respiratory Distress Syndrome

Bleeding Data across Baseline FIX Expression Levels in People with Hemophilia B: An Analysis Using the 'Factor Expression Study'

Long-term outcomes with emicizumab prophylaxis for hemophilia A with or without FVIII inhibitors from the HAVEN 1-4 studies

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

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American Society of Hematology

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ASH Publications

American Society of Hematology

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