Page 1 | Search Results | Blood | American Society of Hematology

1-20 of 27

Sort by

Sort Order Select

Journal Articles

Elevation of liver health biomarkers before and after etranacogene dezaparvovec gene therapy in hemophilia B: Associations with long-term clinical outcomes

Wolfgang Miesbach, Steven Pipe, Cedric Hermans, Michael Recht, Miguel Escobar, Vaibhav Mahajan, Nathalie Jansen, Nicholas Galante, Rachel Church, Paul Monahan

Blood (2025) 146 (Supplement 1): 6093.

https://doi.org/10.1182/blood-2025-6093

Published: 2025

Journal Articles

Experience with minor surgeries in people with hemophilia A or B with and without inhibitors receiving fitusiran

Steven Pipe, Tienan Zhu, Kaan Kavakli, Stephanie P'ng, Chur-Woo You, Inga Hegemann, Gili Kenet, Teh-Liane Khoo, Margarita Timofeeva, Guy Young, Laurel Menapace, Abhimanyu Yarramaneni, Yuqian Shen, Umer Khan, Sandra Casiano, Arta Subagiarta, Julian Zorrilla

Blood (2025) 146 (Supplement 1): 4850.

https://doi.org/10.1182/blood-2025-4850

Published: 2025

Journal Articles

High treatment compliance and tolerability with fitusiran antithrombin-based dose regimen in people with hemophilia A or B, with or without inhibitors: ATLAS-OLE study

Mindy Simpson, Chia-Yau Chang, Kaan Kavakli, Steven Pipe, Jing Sun, Chur-Woo You, Lila-Sabrina Fetita, Abhimanyu Yarramaneni, Chuanwu Zhang, Sandra Casiano, Arta Subagiarta, Erin Cockrell

Blood (2025) 146 (Supplement 1): 1298.

https://doi.org/10.1182/blood-2025-1298

Published: 2025

Journal Articles

New fitusiran 10 mg dose increases proportion of people with hemophilia within target antithrombin range of 15–35%

Guy Young, Alok Srivastava, Marek Demissie, Malidi Ahamadi, Abhimanyu Yarramaneni, Yuqian Shen, Steven Pipe

Blood (2025) 146 (Supplement 1): 6615.

https://doi.org/10.1182/blood-2025-6615

Published: 2025

Journal Articles

Bleeding rates in patients with congenital hemophilia B without inhibitors: A systematic review and meta-analysis

Kristina Lindsley, Arianna Nevo, Carmine Colavecchia, Lisa Wilcox, Priya Patel, Carlos Estevez, Cynthia Khanji, Jennifer Uyei, Steven Pipe, Guy Young

Blood (2025) 146 (Supplement 1): 6613.

https://doi.org/10.1182/blood-2025-6613

Published: 2025

Journal Articles

End-of-study analysis of the HOPE-B trial confirms the durable efficacy and safety of etranacogene dezaparvovec hemophilia b gene therapy over 5 years

Steven Pipe, Wolfgang Miesbach, Michael Recht, Frank Leebeek, Nigel Key, Giancarlo Castaman, Susan Lattimore, Michiel Coppens, Sandra LeQuellec, Sean Gill, Vaibhav Mahajan, Douglass Drelich, Paul Monahan

Blood (2025) 146 (Supplement 1): 538.

https://doi.org/10.1182/blood-2025-538

Published: 2025

Journal Articles

Low Rebleed Incidence with Eptacog Beta Treatment through 24 and 48 Hours in Children (<12 years) with Hemophilia a or B with Inhibitors

Blood (2023) 142 (Supplement 1): 5480.

https://doi.org/10.1182/blood-2023-181299

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

HIV Comorbid Infection and Liver-Directed AAV-Based Gene Therapy in Adults with Severe and Moderately Severe Hemophilia B: Efficacy and Safety Results from Phase 2b and the Pivotal Phase 3 HOPE-B Trials 3 Years after Administration of a Single Dose of Etranacogene Dezaparvovec

Steven Pipe, Esteban Gomez, Cedric R. Hermans, Adam Giermasz, Peter Kampmann, Richard Lemons, Nicholas Galante, Sandra LeQuellec, Paul Monahan

Blood (2023) 142 (Supplement 1): 2256.

https://doi.org/10.1182/blood-2023-187059

Published: 2023

Journal Articles

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

Steven 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, Tiffany Chang, Michaela Lehle, Markus Niggli, Karin Fijnvandraat

Blood (2023) 142 (Supplement 1): 505.

https://doi.org/10.1182/blood-2023-177963

Published: 2023

Journal Articles

Pharmacodynamic Biomarkers in Infants with Hemophilia A Receiving Emicizumab in HAVEN 7

Anna Kiialainen, Steven Pipe, Karin Fijnvandraat, Gili Kenet, Christophe Schmitt, Muriel Buri, Víctor Jiménez-Yuste, Johannes Oldenburg, Maria Elisa Mancuso, Christophe Dhalluin, Michaela Lehle, Peter Collins

Blood (2023) 142 (Supplement 1): 1238.

https://doi.org/10.1182/blood-2023-173015

Published: 2023

Journal Articles

One Year Data from a Phase 2b Trial of AMT-061 (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B

Steven Pipe, MD, Adam Giermasz, MD PhD, Giancarlo Castaman, MD, Nigel S. Key, MBChB, FRCP, Susan U Lattimore, Frank Leebeek, MD PhD, Wolfgang Miesbach, MD PhD, Michael Recht, MD PhD, Esteban Gomez, MD, Alison Long, Robert Gut, Annette von Drygalski, MD PharmD

Blood (2019) 134 (Supplement_1): 3348.

https://doi.org/10.1182/blood-2019-128765

Published: 2019

Journal Articles

First-in-human Gene Therapy Study of AAVhu37 Capsid Vector Technology in Severe Hemophilia A

Steven Pipe, MD, Michael Becka, Elke Detering, Konstantina Vanevski, Toshko Lissitchkov

Blood (2019) 134 (Supplement_1): 4630.

https://doi.org/10.1182/blood-2019-125764

Published: 2019

Journal Articles

Fitusiran, an RNAi Therapeutic Targeting Antithrombin to Restore Hemostatic Balance in Patients with Hemophilia a or B with or without Inhibitors: Management of Acute Bleeding Events

Steven Pipe, MD, Margaret V. Ragni, MD MPH, Claude Négrier, MD PhD, Qifeng Yu, PhD, Naghmana Bajwa, MD, John Caminis, MD, Baisong Mei, MD PhD, Shauna R. Andersson, MD PhD

Blood (2019) 134 (Supplement_1): 1138.

https://doi.org/10.1182/blood-2019-124108

Published: 2019

Journal Articles

The Irish Personalized Approach to the Treatment of Haemophilia (iPATH) - Determinants of Inter-Individual Variation in FVIII Pharmacokinetics

Michelle Lavin, MB, PhD FRCPath, Niamh Larkin, Anjali Patel, Evelyn Singleton, Mary Byrne, Kevin Ryan, MB, FRCPath, Niamh M O'Connell, MB, PhD FRCPath, Birgit M Reipert, Steven Pipe, Peter L Turecek, PhD, James S O'Donnell, MD PhD

Blood (2018) 132 (Supplement 1): 1190.

https://doi.org/10.1182/blood-2018-99-112850

Published: 2018

Journal Articles

Real-World Data of Immune Tolerance Induction Using rFVIIIFc in Subjects With Severe Hemophilia A With Inhibitors at High Risk for ITI Failure

Manuel Carcao, Amy Shapiro, Nina Hwang, Steven Pipe, Sanjay Ahuja, Kenneth Lieuw, MD PhD, Janice Staber, Haowei Linda Sun, Hilda Ding, Michael Wang, Macgregor Steele, Elisa Tsao, Jing Feng, Zahra Al-Khateeb, Jennifer Dumont, Nisha Jain

Blood (2018) 132 (Supplement 1): 2500.

https://doi.org/10.1182/blood-2018-99-110467

Published: 2018

Journal Articles

Preference for Emicizumab over Prior Factor Treatments: Results from the HAVEN 3 and HAVEN 4 Studies

Victor Jimenez-Yuste, Midori Shima, MD PhD, Ido Paz-Priel, Aric Parnes, Michaela Lehle, Adam Giermasz, Avrita Campinha-Bacote, Markus Niggli, Jerzy Windyga, Sammy Chebon, Peter Trask, Johnny Mahlangu, Steven Pipe, Johannes Oldenburg

Blood (2018) 132 (Supplement 1): 1187.

https://doi.org/10.1182/blood-2018-99-118132

Published: 2018

Journal Articles

101HEMB01 Is a Phase 1/2 Open-Label, Single Ascending Dose-Finding Trial of DTX101 (AAVrh10FIX) in Patients with Moderate/Severe Hemophilia B That Demonstrated Meaningful but Transient Expression of Human Factor IX (hFIX)

Steven Pipe, MD, Kimo Stine, MD, Anita Rajasekhar, MD, Tamara Everington, MD, Allen Poma, MD, Eric Crombez, MD, Charles RM Hay, MD

Blood (2017) 130 (Supplement 1): 3331.

https://doi.org/10.1182/blood.V130.Suppl_1.3331.3331

Published: 2017

Journal Articles

Improving Hematologist/Oncologist Knowledge and Confidence in Managing Hemophilia through Online Educational Interventions

Emily S Van Laar, MS, Charlotte Warren, John Maeglin, Mila Kostic, Michael Schoen, PhD, Neil Frick, MS, Steven Pipe, MD

Blood (2015) 126 (23): 5593.

https://doi.org/10.1182/blood.V126.23.5593.5593

Published: 2015

Journal Articles

Safety and Efficacy of Recombinant Factor VIIa (rFVIIa) in Congenital Hemophilia with Inhibitors (CHwI) in the Home Treatment Setting: Systematic Review of Clinical Studies and Registries

Guy Young, MD, Miguel Escobar, Steven Pipe, MD, David L. Cooper, MD MBA

Blood (2015) 126 (23): 2302.

https://doi.org/10.1182/blood.V126.23.2302.2302

Published: 2015

Update search

NARROW

Publications

Format

Article Type

Topics

Date

Availability

Elevation of liver health biomarkers before and after etranacogene dezaparvovec gene therapy in hemophilia B: Associations with long-term clinical outcomes

Experience with minor surgeries in people with hemophilia A or B with and without inhibitors receiving fitusiran

High treatment compliance and tolerability with fitusiran antithrombin-based dose regimen in people with hemophilia A or B, with or without inhibitors: ATLAS-OLE study

New fitusiran 10 mg dose increases proportion of people with hemophilia within target antithrombin range of 15–35%

Bleeding rates in patients with congenital hemophilia B without inhibitors: A systematic review and meta-analysis

End-of-study analysis of the HOPE-B trial confirms the durable efficacy and safety of etranacogene dezaparvovec hemophilia b gene therapy over 5 years

Low Rebleed Incidence with Eptacog Beta Treatment through 24 and 48 Hours in Children (<12 years) with Hemophilia a or B with Inhibitors

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

HIV Comorbid Infection and Liver-Directed AAV-Based Gene Therapy in Adults with Severe and Moderately Severe Hemophilia B: Efficacy and Safety Results from Phase 2b and the Pivotal Phase 3 HOPE-B Trials 3 Years after Administration of a Single Dose of Etranacogene Dezaparvovec

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

Pharmacodynamic Biomarkers in Infants with Hemophilia A Receiving Emicizumab in HAVEN 7

One Year Data from a Phase 2b Trial of AMT-061 (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B

First-in-human Gene Therapy Study of AAVhu37 Capsid Vector Technology in Severe Hemophilia A

Fitusiran, an RNAi Therapeutic Targeting Antithrombin to Restore Hemostatic Balance in Patients with Hemophilia a or B with or without Inhibitors: Management of Acute Bleeding Events

The Irish Personalized Approach to the Treatment of Haemophilia (iPATH) - Determinants of Inter-Individual Variation in FVIII Pharmacokinetics

Real-World Data of Immune Tolerance Induction Using rFVIIIFc in Subjects With Severe Hemophilia A With Inhibitors at High Risk for ITI Failure

Preference for Emicizumab over Prior Factor Treatments: Results from the HAVEN 3 and HAVEN 4 Studies

101HEMB01 Is a Phase 1/2 Open-Label, Single Ascending Dose-Finding Trial of DTX101 (AAVrh10FIX) in Patients with Moderate/Severe Hemophilia B That Demonstrated Meaningful but Transient Expression of Human Factor IX (hFIX)

Improving Hematologist/Oncologist Knowledge and Confidence in Managing Hemophilia through Online Educational Interventions

Safety and Efficacy of Recombinant Factor VIIa (rFVIIa) in Congenital Hemophilia with Inhibitors (CHwI) in the Home Treatment Setting: Systematic Review of Clinical Studies and Registries

Email alerts

ASH Publications

American Society of Hematology

Update search

Email alerts

ASH Publications

American Society of Hematology

This Feature Is Available To Subscribers Only