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Journal Articles

Reduced Doses of Factor Concentrates and Bypassing Agents to Treat Breakthrough Bleeds in Patients with Hemophilia A and B on Fitusiran Antithrombin-Based Dosing Regimen: ATLAS-OLE

Steven W. Pipe, Kaan Kavakli, Tadashi Matsushita, Huyen Tran, Bulent Zulfikar, Laurel Menapace, Marja Puurunen, Wenruo Hu, Yuqian Shen, Chanchala Kaddi, Vanessa Salinas

Blood (2024) 144 (Supplement 1): 128.

https://doi.org/10.1182/blood-2024-206946

Published: 2024

Journal Articles

Efficacy and Safety of Giroctocogene Fitelparvovec in Adults with Moderately Severe to Severe Hemophilia Α: Primary Analysis Results from the Phase 3 ΑFFINE Gene Therapy Trial

Blood (2024) 144 (Supplement 1): 1053.

https://doi.org/10.1182/blood-2024-208486

Published: 2024

Journal Articles

Pain Reduction Following Treatment of Bleeding Episodes with Eptacog Beta in Adults and Adolescents with Hemophilia A or B Complicated By Inhibitors

Tyler W. Buckner, Craig Kessler, Giancarlo Castaman, Cédric Hermans, Victor Jimenez-Yuste, Johnny Mahlangu, Wolfgang A. Miesbach, Johannes Oldenburg, Michael Recht, Leonard A. Valentino, Allison P Wheeler, Steven W. Pipe

Blood (2024) 144 (Supplement 1): 3972.

https://doi.org/10.1182/blood-2024-194935

Published: 2024

Journal Articles

Become-9: A Phase 1/2 Dose Escalation and Expansion Study of be-101 for the Treatment of Adults with Moderately Severe or Severe Hemophilia B

Steven W. Pipe, Mark T Reding, Bryan Chin, Karen Campbell, Hanlan Liu, Wing Yen Wong

Blood (2024) 144 (Supplement 1): 2593.1.

https://doi.org/10.1182/blood-2024-208733

Published: 2024

Journal Articles

Bleed Patterns in Infants, from Birth to 12 Months of Age, with Hemophilia A Treated with Emicizumab: Exploratory Analysis of the HAVEN 7 Study

Shannon L Carpenter, Dakota Rosenfelt, Eunice Tzeng, Elise Lim, Steven W. Pipe

Blood (2024) 144 (Supplement 1): 1214.

https://doi.org/10.1182/blood-2024-193615

Published: 2024

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

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

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

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

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

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

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

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

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

NARROW

Reduced Doses of Factor Concentrates and Bypassing Agents to Treat Breakthrough Bleeds in Patients with Hemophilia A and B on Fitusiran Antithrombin-Based Dosing Regimen: ATLAS-OLE

Efficacy and Safety of Giroctocogene Fitelparvovec in Adults with Moderately Severe to Severe Hemophilia Α: Primary Analysis Results from the Phase 3 ΑFFINE Gene Therapy Trial

Pain Reduction Following Treatment of Bleeding Episodes with Eptacog Beta in Adults and Adolescents with Hemophilia A or B Complicated By Inhibitors

Become-9: A Phase 1/2 Dose Escalation and Expansion Study of be-101 for the Treatment of Adults with Moderately Severe or Severe Hemophilia B

Bleed Patterns in Infants, from Birth to 12 Months of Age, with Hemophilia A Treated with Emicizumab: Exploratory Analysis of the HAVEN 7 Study

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

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

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

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

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

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)

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

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

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

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American Society of Hematology

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American Society of Hematology

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