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Journal Articles

Characterizing a Cohort of Patients with Hemophilia B Treated with Fidanacogene Elaparvovec from the Phase 3 Benegene-2 Study Who Returned to Factor IX Prophylaxis

Laurent Frenzel, Kaan Kavakli, Robert Klamroth, Shyh-Shin Chiou, Amy D. Shapiro, Pengling Sun, Joanne Fuiman, John McKay, Annie F. Fang, Francesca Biondo, Frank Plonski, Jeremy Rupon

Blood (2023) 142 (Supplement 1): 2257.

https://doi.org/10.1182/blood-2023-181223

Published: 2023

Journal Articles

Factor VIII Use in the Treatment of Breakthrough Bleeds in People with Moderate or Mild Hemophilia A without Factor VIII Inhibitors Receiving Emicizumab Prophylaxis: The Phase III HAVEN 6 Experience

Víctor Jiménez-Yuste, Elise Lim, Ketna Mistry, Giuliana Ventriglia, Amy D. Shapiro

Blood (2023) 142 (Supplement 1): 3999.

https://doi.org/10.1182/blood-2023-173081

Published: 2023

Journal Articles

Treatment-Related Clinical and Humanistic Unmet Needs in Hemophilia A without Inhibitors

Allison P Wheeler, John Waller, Sanket Shah, Mohd Nawi bin Wahid, Jennifer Dusendang, Amy D. Shapiro

Blood (2023) 142 (Supplement 1): 508.

https://doi.org/10.1182/blood-2023-179128

Published: 2023

Journal Articles

Adherence to Clotting Factor Treatment and Association with Bleeding and Health-Related Quality of Life in Persons with Hemophilia

Duc Quang Tran Jr., Megan M. Ullman, Brenda Riske, Cathy Buranahirun-Burns, Amy D. Shapiro, Margaret V. Ragni, Barbara A. Konkle, Nicole Crook, Jonathan C. Roberts, Randall Curtis, Joanne Wu, Michael B. Nichol

Blood (2023) 142 (Supplement 1): 7334.

https://doi.org/10.1182/blood-2023-187387

Published: 2023

Journal Articles

Emicizumab Prophylaxis in People with Moderate or Mild Hemophilia A Aged ≥40 Years, with and without Comorbidities - Findings from the Phase III HAVEN 6 Study

Víctor Jiménez-Yuste, Eunice Tzeng, Elise Lim, Giuliana Ventriglia, Amy D. Shapiro, Johannes Oldenburg, Johnny Mahlangu

Blood (2023) 142 (Supplement 1): 5495.

https://doi.org/10.1182/blood-2023-173016

Published: 2023

Journal Articles

Assessment of Emicizumab Adherence: Psychometrics and Applications of Novel Self-Report Measure Veritas-Nexgen

Amanda Okolo, William K. Kronenberger, Chris Roberson, Isaac A. Janson, Mackenzie Nolte, Lisa Littner, James Okungade, Amy D. Shapiro

Blood (2022) 140 (Supplement 1): 10767–10768.

https://doi.org/10.1182/blood-2022-169861

Published: 2022

Journal Articles

Health-Related Quality of Life in Patients with Hemophilia A or B with Inhibitors on Concizumab Prophylaxis: Results from the Phase 3 explorer7 Study

Amy D. Shapiro, Aby Abraham, Silvia Linari, Jesper Skov Neergaard, Jan Odgaard-Jensen, Jay Jay Thaung Zaw, Huyen Tran

Blood (2022) 140 (Supplement 1): 2732–2734.

https://doi.org/10.1182/blood-2022-166306

Published: 2022

Journal Articles

Performance of the Comprehensive Assessment Tool for Challenges in Hemophilia (CATCH) 2.0

Marquita Decker-Palmer, Susan Mathias, Hilary Colwell, Ross Crosby, Brittany Gentile, Janet Chupka, Robert F. Sidonio, Jr., Michael Recht, Amy D. Shapiro, James Zazzali

Blood (2022) 140 (Supplement 1): 11328–11329.

https://doi.org/10.1182/blood-2022-157932

Published: 2022

Journal Articles

Characterizing Mild and Moderate Hemophilia A Patients in the Real World: A Patient-Centric Approach

Michelle Witkop, Tao Xu, Gillian Hanson, Richard Ofori-Asenso, Richard H. Ko, Emily Cibelli, Martynas Aizenas, Mark W Skinner, Fabian Sanabria, Amy D. Shapiro

Blood (2021) 138 (Supplement 1): 2107.

https://doi.org/10.1182/blood-2021-146543

Published: 2021

Journal Articles

Emicizumab Prophylaxis in Persons with Mild or Moderate Hemophilia A: Results from the Interim Analysis of the HAVEN 6 Study

Claude Négrier, Johnny Mahlangu, Michaela Lehle, Pratima Chowdary, Olivier Catalani, Víctor Jiménez-Yuste, Benjamin M. Beckermann, Christophe Schmitt, Cedric Hermans, Giuliana Ventriglia, Jerzy Windyga, Anna Kiialainen, Roseline d'Oiron, Paul Moorehead, Vanda Teodoro, Amy D. Shapiro, Johannes Oldenburg

Blood (2021) 138 (Supplement 1): 343.

https://doi.org/10.1182/blood-2021-146009

Published: 2021

Journal Articles

Prophylaxis with rFIXFc Reduces the Frequency and Delays Time to First Spontaneous Bleed Event in Previously Untreated Patients with Hemophilia B: A Post Hoc Analysis of the PUPs B-LONG Trial

Beatrice Nolan, Michael Recht, Pablo Rendo, Aletta Falk, Meredith Foster, Sandra Casiano, Antoine Rauch, Amy D. Shapiro

Blood (2021) 138 (Supplement 1): 498.

https://doi.org/10.1182/blood-2021-148765

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

Efanesoctocog Alfa Half-Life and Clearance Are Independent of von Willebrand Factor in Severe Hemophilia A: A Post Hoc Analysis from Phase 1/2a Studies

Janice M. Staber, Toshko Lissitchkov, Barbara A. Konkle, Amy D. Shapiro, Doris V. Quon, Roshni Kulkarni, Melinda Hamilton, Ekta Seth Chhabra, Suresh Katragadda, Arman Altincatal, Annemieke Willemze, Jennifer Dumont, Margaret V. Ragni

Blood (2021) 138 (Supplement 1): 1035.

https://doi.org/10.1182/blood-2021-148534

Published: 2021

Journal Articles

Perioperative Outcomes Associated with Inhibitor Status in Patients with Hemophilia - a Retrospective Cohort Study

Omotola O. Olasupo, Charles Nakar, Alfonso Iorio, Craig Haddix, Thushara Mathew, Lynn M Malec, Davide Matino, Lawrence Mbuagbaw, Jean-Eric Tarride, Amy D. Shapiro

Blood (2021) 138 (Supplement 1): 4990.

https://doi.org/10.1182/blood-2021-147550

Published: 2021

Journal Articles

A Novel Methodology for Building Longitudinal, Patient-Centric Real World Datasets in Hemophilia A

Mark W Skinner, Gillian Hanson, Tao Xu, Richard Ofori-Asenso, Richard H. Ko, Emily Cibelli, Francis Nissen, Michelle Witkop, Fabian Sanabria, Amy D. Shapiro

Blood (2021) 138 (Supplement 1): 594.

https://doi.org/10.1182/blood-2021-146160

Published: 2021

Journal Articles

Acute Airway Obstruction in 4 Pediatric Patients with Congenital Plasminogen Deficiency (C-PLGD) Treated with Intravenous Plasminogen (Human) Replacement Therapy Under an Expanded Access Protocol

Charles Nakar, MD, Neelam Thukral, CCRC, Heather L. McDaniel, MD, Joseph M Parker, MD, Diane Trybul, BS, Amy D Shapiro, MD

Blood (2020) 136 (Supplement 1): 2.

https://doi.org/10.1182/blood-2020-137177

Published: 2020

Journal Articles

Plasminogen Eye Drops Are Effective in Preventing Recurrence of Pseudomembranes in Ligneous Conjunctivitis: Results from the Phase 2/3 KB046 Trial

Roberto Caputo, MD, Maria Teresa Sartori, MD, Andrea Leonardi, MD, Anna Lotti Suffredini, BSc (Bio), Mirella Calcinai, MD, Roberto Crea, DVM, MSc, Prasad Mathew, MD, Charles Nakar, MD, Neelam Thukral, CCRC, Amy D Shapiro, MD

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

https://doi.org/10.1182/blood-2020-137538

Published: 2020

Journal Articles

First-in-Human Phase 1/2 Clinical Trial of SIG-001, an Innovative Shielded Cell Therapy Platform, for Hemophilia Α

Amy D Shapiro, MD, Barbara A. Konkle, MD, Stacy E. Croteau, MD, Wolfgang A. Miesbach, MD, Charles Richard Morris Hay, MDFRCP,FRCPath, Rashid Kazmi, MBBS, Marina Mihova, Savita Rangarajan, MD MBBS, John Pasi, MB, ChB, PhD FRCP, FRCPath, FRCPCH

Blood (2020) 136 (Supplement 1): 8.

https://doi.org/10.1182/blood-2020-141381

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

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

Characterizing a Cohort of Patients with Hemophilia B Treated with Fidanacogene Elaparvovec from the Phase 3 Benegene-2 Study Who Returned to Factor IX Prophylaxis

Factor VIII Use in the Treatment of Breakthrough Bleeds in People with Moderate or Mild Hemophilia A without Factor VIII Inhibitors Receiving Emicizumab Prophylaxis: The Phase III HAVEN 6 Experience

Treatment-Related Clinical and Humanistic Unmet Needs in Hemophilia A without Inhibitors

Adherence to Clotting Factor Treatment and Association with Bleeding and Health-Related Quality of Life in Persons with Hemophilia

Emicizumab Prophylaxis in People with Moderate or Mild Hemophilia A Aged ≥40 Years, with and without Comorbidities - Findings from the Phase III HAVEN 6 Study

Assessment of Emicizumab Adherence: Psychometrics and Applications of Novel Self-Report Measure Veritas-Nexgen

Health-Related Quality of Life in Patients with Hemophilia A or B with Inhibitors on Concizumab Prophylaxis: Results from the Phase 3 explorer7 Study

Performance of the Comprehensive Assessment Tool for Challenges in Hemophilia (CATCH) 2.0

Characterizing Mild and Moderate Hemophilia A Patients in the Real World: A Patient-Centric Approach

Emicizumab Prophylaxis in Persons with Mild or Moderate Hemophilia A: Results from the Interim Analysis of the HAVEN 6 Study

Prophylaxis with rFIXFc Reduces the Frequency and Delays Time to First Spontaneous Bleed Event in Previously Untreated Patients with Hemophilia B: A Post Hoc Analysis of the PUPs B-LONG 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

Efanesoctocog Alfa Half-Life and Clearance Are Independent of von Willebrand Factor in Severe Hemophilia A: A Post Hoc Analysis from Phase 1/2a Studies

Perioperative Outcomes Associated with Inhibitor Status in Patients with Hemophilia - a Retrospective Cohort Study

A Novel Methodology for Building Longitudinal, Patient-Centric Real World Datasets in Hemophilia A

Acute Airway Obstruction in 4 Pediatric Patients with Congenital Plasminogen Deficiency (C-PLGD) Treated with Intravenous Plasminogen (Human) Replacement Therapy Under an Expanded Access Protocol

Plasminogen Eye Drops Are Effective in Preventing Recurrence of Pseudomembranes in Ligneous Conjunctivitis: Results from the Phase 2/3 KB046 Trial

First-in-Human Phase 1/2 Clinical Trial of SIG-001, an Innovative Shielded Cell Therapy Platform, for Hemophilia Α

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

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

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