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Journal Articles

Fourth Interim Analysis of the Hem Powr Study: A Post Hoc Analysis of Real-World Effectiveness and Safety of Damoctocog Alfa Pegol in Adolescent and Older Patients with Hemophilia a

Mark T Reding, María Teresa Alvarez-Román, Giancarlo Castaman, Maissaa Janbain, Tadashi Matsushita, Karina Meijer, Kathrin Schmidt, Johannes Oldenburg

Blood (2024) 144 (Supplement 1): 3968.

https://doi.org/10.1182/blood-2024-205121

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

Physical and Psychological Burden on People with Hemophilia: Global Real-World Data

Giancarlo Castaman, Cléa Percier, Naveen Shridhar, Neil Reynolds, Olivera Rajkovic-Hooley, Thom Dewar, Víctor Jiménez-Yuste

Blood (2024) 144 (Supplement 1): 2318.

https://doi.org/10.1182/blood-2024-200033

Published: 2024

Journal Articles

Unmet Needs of Patients with Hemophilia a/B with or without Inhibitors: Real-World End-of-Study Results from the explorer6 Non-Interventional Study

Allison P. Wheeler, Aby Abraham, Chris Barnes, Renée Brown Frandsen, Kingsley Hampton, Francisco José López Jaime, Camila Martins Mazini Tavares, Keiji Nogami, Jerzy Windyga, Giancarlo Castaman

Blood (2024) 144 (Supplement 1): 2585.

https://doi.org/10.1182/blood-2024-209752

Published: 2024

Journal Articles

Burden of Treatment on People with Hemophilia: Global Real-World Data

Víctor Jiménez-Yuste, Cléa Percier, Naveen Shridhar, Neil Reynolds, Olivera Rajkovic-Hooley, Thom Dewar, Giancarlo Castaman

Blood (2024) 144 (Supplement 1): 5077.

https://doi.org/10.1182/blood-2024-200008

Published: 2024

Journal Articles

The Effect of Concizumab Prophylaxis on Target Joints, Resolution and Joint Bleeds in Patients With Hemophilia A or B With or Without Inhibitors in Phase 3 Clinical Trials

Giancarlo Castaman, Aby Abraham, Pantep Angchaisuksiri, Laura Villarreal Martinez, Keiji Nogami, Jameela Sathar, Chunduo Shen, Jay Jay Thaung Zaw, Guy Young

Blood (2023) 142 (Supplement 1): 284.

https://doi.org/10.1182/blood-2023-180790

Published: 2023

Journal Articles

Physical Activity, Bleedings and Quality of Life in Patients with Haemophilia a without Inhibitors - a Multicenter, Observational Italian Study with a Wearable Device

Maria Elisa Mancuso, Chiara Biasoli, Renato Marino, Andrea Buzzi, Daniele Preti, Sara Bendinelli, Rosaria Tempre, Elena Pompeo, Luigi Sannino, Giacomo Siri, Giancarlo Castaman

Blood (2023) 142 (Supplement 1): 663.

https://doi.org/10.1182/blood-2023-179089

Published: 2023

Journal Articles

Adult Patients with Hemophilia B and with a History of Chronic HCV/HBV Infection Receiving Liver-Directed Gene Therapy Demonstrated Long-Term Bleeding Protection and Sustained FIX Activity: Efficacy and Safety Results from the HOPE-B Trial 3 Years after Administration of a Single Dose of Etranacogene Dezaparvovec

Annette von Drygalski, Niamh O'Connell, Peter Verhamme, Karina Meijer, Paul van der Valk, Rashid Saeed Kazmi, Priyanka Raheja, Nicholas Galante, Sandra LeQuellec, Rachel Church, Samantha Lucas, Giancarlo Castaman, Paul Monahan

Blood (2023) 142 (Supplement 1): 2258.

https://doi.org/10.1182/blood-2023-188921

Published: 2023

Journal Articles

Interim Subgroup Analysis of the Effectiveness and Safety of Damoctocog Alfa Pegol Prophylaxis in Previously Treated Patients with Hemophilia A Treated Every 5 or Every 7 Days: Results from the Real-World Observational HEM-POWR Study

Mark T Reding, María Teresa Alvarez Román, Martin Sanabria, Giancarlo Castaman, Maissaa Janbain, Tadashi Matsushita, Karina Meijer, Kathrin Schmidt, Johannes Oldenburg

Blood (2022) 140 (Supplement 1): 5612–5614.

https://doi.org/10.1182/blood-2022-169159

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 (rFVIIa) Has a Low Incidence of Spontaneous Rebleeding through 24 and 48 Hours in Adult and Adolescent Patients with Hemophilia A or B with Inhibitors

Amy L. Dunn, Yesim Dargaud, Yasmina L. Abajas, Manuel Carcao, Giancarlo Castaman, Adam Giermasz, Cédric Hermans, Magdalena D. Lewandowska, Johnny Mahlangu, Shannon L. Meeks, Wolfgang A. Miesbach, Michael Recht, Vanessa Salinas, Tammuella Chrisentery-Singleton, Hongying Wang, Ian S Mitchell, Guy Young

Blood (2022) 140 (Supplement 1): 8485–8488.

https://doi.org/10.1182/blood-2022-157343

Published: 2022

Journal Articles

Recombinant von Willebrand factor prophylaxis in patients with severe von Willebrand disease: phase 3 study results

Frank W. G. Leebeek, Flora Peyvandi, Miguel Escobar, Andreas Tiede, Giancarlo Castaman, Michael Wang, Tung Wynn, Jovanna Baptista, Yi Wang, Jingmei Zhang, Björn Mellgård, Gülden Özen

Blood (2022) 140 (2): 89–98.

https://doi.org/10.1182/blood.2021014810

Published: 2022

Includes: Supplemental data

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

Surgical Experience from the Phase III STASEY Trial of Emicizumab Prophylaxis in Persons with Hemophilia A with FVIII Inhibitors: Final Analysis

Giancarlo Castaman, Jerzy Windyga, Hazza Alzahrani, Susan Robson, Fabian Sanabria, Monet Howard, Víctor Jiménez-Yuste

Blood (2021) 138 (Supplement 1): 344.

https://doi.org/10.1182/blood-2021-145890

Published: 2021

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

Safety and Longer-Term Efficacy of Concizumab Prophylaxis in Patients with Hemophilia a or b with Inhibitors: Results from the Extension Part of the Phase 2 explorer4 Trial

Amy D Shapiro, MD, Giancarlo Castaman, MD, Katarina Cepo, MD, Sidsel Marie Tønder, MSc, Tadashi Matsushita, MD PhD, Lone Hvitfeldt Poulsen, Guy Young, MD, Silva Zupancic-Šalek, MD, Victor Jimenez Yuste, MD PhD

Blood (2020) 136 (Supplement 1): 40–41.

https://doi.org/10.1182/blood-2020-139910

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

Subcutaneous concizumab prophylaxis in hemophilia A and hemophilia A/B with inhibitors: phase 2 trial results

Amy D. Shapiro, Pantep Angchaisuksiri, Jan Astermark, Gary Benson, Giancarlo Castaman, Pratima Chowdary, Hermann Eichler, Victor Jiménez-Yuste, Kaan Kavakli, Tadashi Matsushita, Lone Hvitfeldt Poulsen, Allison P. Wheeler, Guy Young, Silva Zupancic-Salek, Johannes Oldenburg

Blood (2019) 134 (22): 1973–1982.

https://doi.org/10.1182/blood.2019001542

Published: 2019

Includes: Multimedia, Supplemental data

NARROW

Fourth Interim Analysis of the Hem Powr Study: A Post Hoc Analysis of Real-World Effectiveness and Safety of Damoctocog Alfa Pegol in Adolescent and Older Patients with Hemophilia a

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

Physical and Psychological Burden on People with Hemophilia: Global Real-World Data

Unmet Needs of Patients with Hemophilia a/B with or without Inhibitors: Real-World End-of-Study Results from the explorer6 Non-Interventional Study

Burden of Treatment on People with Hemophilia: Global Real-World Data

The Effect of Concizumab Prophylaxis on Target Joints, Resolution and Joint Bleeds in Patients With Hemophilia A or B With or Without Inhibitors in Phase 3 Clinical Trials

Physical Activity, Bleedings and Quality of Life in Patients with Haemophilia a without Inhibitors - a Multicenter, Observational Italian Study with a Wearable Device

Interim Subgroup Analysis of the Effectiveness and Safety of Damoctocog Alfa Pegol Prophylaxis in Previously Treated Patients with Hemophilia A Treated Every 5 or Every 7 Days: Results from the Real-World Observational HEM-POWR Study

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 (rFVIIa) Has a Low Incidence of Spontaneous Rebleeding through 24 and 48 Hours in Adult and Adolescent Patients with Hemophilia A or B with Inhibitors

Recombinant von Willebrand factor prophylaxis in patients with severe von Willebrand disease: phase 3 study results

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

Surgical Experience from the Phase III STASEY Trial of Emicizumab Prophylaxis in Persons with Hemophilia A with FVIII Inhibitors: Final Analysis

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

Safety and Longer-Term Efficacy of Concizumab Prophylaxis in Patients with Hemophilia a or b with Inhibitors: Results from the Extension Part of the Phase 2 explorer4 Trial

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

Subcutaneous concizumab prophylaxis in hemophilia A and hemophilia A/B with inhibitors: phase 2 trial results

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