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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

Single 225μg/Kg Dose Treatment with Eptacog Beta (Factor VIIa, Recombinant) Results in Rapid Hemostasis in Joint Bleeds for Persons with Hemophilia Α or Β with Inhibitors: A PERSEPT1 Subset Analysis

Janna M. Journeycake, MD, Cedric Hermans, MDMRCP,PhD, Jonathan M. Ducore, MD MPH, Miguel A. Escobar, MD, Guy Young, MD, Michael Wang, MD, Doris Quon, MD PhD, W Allan Alexander, MD, Ian S Mitchell, PhD, Ahmad Al-Sabbagh, MD, Daniel Bonzo, PhD

Blood (2020) 136 (Supplement 1): 2–3.

https://doi.org/10.1182/blood-2020-136443

Published: 2020

Journal Articles

N-Linked Glycans on Therapeutic Factor VIII (FVIII) Proteins Attenuate Immunogenicity Potential: Evidence from Independent HLA-Class-II/FVIII (HLAcII/FVIII) Peptidomes

Vincent P. Diego, PhD, Bernadette W. Luu, B.S., M.T. (CLS), Hari Movva, B.S., Marco Hofmann, M.S., Marcio A. Almeida, PhD, Long V. Dinh, PhD, Jerry S. Powell, MD, Raja Rajalingam, PhD, Juan M Peralta, M.S., Henry Mead, M.B.A., M.P.H., Satish Kumar, PhD, Joanne E. Curran, PhD, Miguel A. Escobar, MD, Sarah Williams-Blangero, PhD, Eugene Maraskovsky, PhD, John Blangero, PhD, Tom E. Howard, MD, PhD

Blood (2020) 136 (Supplement 1): 29–30.

https://doi.org/10.1182/blood-2020-142493

Published: 2020

Journal Articles

PERSEPT3: A Phase 3 Clinical Trial to Evaluate the Hemostatic Efficacy of Eptacog Beta (Recombinant Human FVIIa) in Perioperative Care in Subjects with Hemophilia Α or B with Inhibitors

Miguel A. Escobar, MD, Doris Quon, MD PhD, Cindy A. Leissinger, MD, James V Luck, Jr., MD, Ahmad Al-Sabbagh, MD, Daniel Bonzo, PhD, Ian S Mitchell, PhD, W Allan Alexander, MD, Cedric Hermans, MDMRCP,PhD

Blood (2020) 136 (Supplement 1): 34–36.

https://doi.org/10.1182/blood-2020-136448

Published: 2020

Journal Articles

A Phase IV, Multicenter, Open-Label Study of Emicizumab Prophylaxis in Persons with Hemophilia a with or without FVIII Inhibitors Undergoing Minor Surgical Procedures

Miguel A. Escobar, MD, Amy Dunn, Doris Quon, MD PhD, Ben Trzaskoma, Lucy Lee, Richard H Ko, Shannon L Carpenter, MD

Blood (2020) 136 (Supplement 1): 30–31.

https://doi.org/10.1182/blood-2020-134906

Published: 2020

Journal Articles

Specific Correction of the Intron-22 Inverted Factor VIII Gene in Autologous Blood Outgrowth Endothelial Cells from Patients with Severe Hemophilia A

Long V. Dinh, PhD, Eli Fine, PhD, Afshin Ameri, MD, Bernadette W. Luu, B.S., M.T. (CLS), Satish Kumar, PhD, Vincent P. Diego, PhD, Joanne E. Curran, PhD, Juan M Peralta, M.S., Henry Mead, M.B.A., M.P.H., Miguel A. Escobar, MD, Sarah Williams-Blangero, PhD, John Blangero, PhD, Jerry S. Powell, MD, Timothy C. Nichols, MD, Robert P. Hebbel, MD, Tom E. Howard, MD, PhD

Blood (2020) 136 (Supplement 1): 30–31.

https://doi.org/10.1182/blood-2020-142679

Published: 2020

Journal Articles

Retrospective Chart Review of Gastrointestinal Bleeding in Patients with Von Willebrand Disease

Jonathan C. Roberts, MD, Miguel A. Escobar, MD, Suchitra Acharya Acharya, MD, Nina Hwang, MD, Michael Wang, MD, Sarah A Hale, MS, PhD, Abiola Oladapo, PhD, Sohaib Asghar, Syed Mehdi, MD, Peter A. Kouides, MD

Blood (2020) 136 (Supplement 1): 20–21.

https://doi.org/10.1182/blood-2020-133499

Published: 2020

Journal Articles

Disentangling the Effects of HLA DRB115:01 and DQB106:02 to Establish the True HLA Risk Allele for Inhibitor Development in the Treatment of Hemophilia A

Vincent P. Diego, PhD, Bernadette W. Luu, B.S., M.T. (CLS), Alexis Garza, B.S., Marco Hofmann, M.S., Marcio A. Almeida, PhD, Jerry S. Powell, MD, Long V. Dinh, PhD, Raja Rajalingam, PhD, Juan M Peralta, M.S., Satish Kumar, PhD, Eli Fine, PhD, Joanne E. Curran, PhD, Miguel A. Escobar, MD, Sarah Williams-Blangero, PhD, Eugene Maraskovsky, PhD, John Blangero, PhD, Tom E. Howard, MD, PhD

Blood (2020) 136 (Supplement 1): 1–2.

https://doi.org/10.1182/blood-2020-142716

Published: 2020

Journal Articles

Consistency of Dosing of Turoctocog Alfa Pegol (N8-GP) throughout the Pathfinder Clinical Trials: Implications for Recommendations for Effective Prophylaxis, Bleed Resolution, and Perioperative Management

Miguel A. Escobar, MD, Allison P. Wheeler, MD, Steven R. Lentz, MD PhD, Wan Hui Ong Clausen, PhD, David Cooper

Blood (2019) 134 (Supplement_1): 3635.

https://doi.org/10.1182/blood-2019-124889

Published: 2019

Journal Articles

The Dendritic Cell HLA-Class-II/Therapeutic Factor VIII (FVIII) Peptidome Is Influenced in Unanticipated Ways By the B-Domain of FVIII and the FVIII Chaperon Protein, Von Willebrand Factor: The Outrigger and Glycosylation-Umbrella (GUMB) Hypotheses

Tom E. Howard, MD, PhD, Bernadette W. Luu, B.S., M.T. (CLS), Marco Hofmann, M.S., Marcio A. Almeida, PhD, Satish Kumar, PhD, Long V. Dinh, PhD, Henry Mead, M.B.A., M.P.H., Jerry S. Powell, MD, Miguel A. Escobar, MD, Raja Rajalingam, PhD, Sarah Williams-Blangero, PhD, John Blangero, PhD, Maraskovsky Eugene, PhD, Vincent P. Diego, PhD

Blood (2019) 134 (Supplement_1): 161.

https://doi.org/10.1182/blood-2019-131727

Published: 2019

Journal Articles

Immune Tolerance Induction (ITI) with a Plasma-Derived Factor VIII for Patients with Hemophilia a and Inhibitors: A Retrospective Study

Miguel A. Escobar, MD, Linda Shaffer, Mark Holguin, MD, Timothy McCavit, MD, Sandeep K. Rajan, MD, Novinyo Amega, MD

Blood (2019) 134 (Supplement_1): 4923.

https://doi.org/10.1182/blood-2019-122949

Published: 2019

Journal Articles

On the Role of F8 Sequence Mismatch and Class-II Human Leukocyte Antigen Binding in the Development of Neutralizing Antibodies ("Inhibitors") Directed Against Therapeutic Factor VIII Proteins (tFVIIIs): Evidence from the PATH Study

Vincent P. Diego, PhD, Bernadette W. Luu, B.S., M.T. (CLS), Afshin Ameri, MD, Meera B. Chitlur, MD, Marcio A. Almeida, PhD, Karin Haack, PhD, Satish Kumar, PhD, Long V. Dinh, PhD, Henry Mead, M.B.A., M.P.H., Jerry S. Powell, MD, Miguel A. Escobar, MD, Raja Rajalingam, PhD, Sarah Williams-Blangero, PhD, Carol K. Kasper, MD, Laura Almasy, PhD, Shelley Cole, PhD, John Blangero, PhD, Tom E. Howard, MD, PhD

Blood (2019) 134 (Supplement_1): 2393.

https://doi.org/10.1182/blood-2019-132218

Published: 2019

Journal Articles

Chronic Kidney Disease (CKD) in the U.S. Hemophilia Population: A Cohort Study

Blood (2018) 132 (Supplement 1): 2479.

https://doi.org/10.1182/blood-2018-99-119637

Published: 2018

Journal Articles

The Role of Class II Human Leukocyte Antigens (cII-HLAs) in Determining the Immunogenic Potential of Therapeutic Factor VIII Proteins in Hemophilia Patients: The "Gate Keeper" Hypothesis

Vincent P. Diego, PhD, Bernadette W. Luu, BS, MT/CLS, Marco Hofmann, MS, Marcio A. Almeida, PhD, Jose Antonio G. Hernandez, PhD, Juan Peralta, MS, Joanne Curran, PhD, Jerry S. Powell, MD, Raja Rajalingam, PhD, Long V. Dinh, PhD, Sarah Williams-Blangero, PhD, Miguel A Escobar, MD, Eugene Maraskovsky, PhD, John Blangero, PhD, Tom E. Howard

Blood (2018) 132 (Supplement 1): 5022.

https://doi.org/10.1182/blood-2018-99-115537

Published: 2018

Journal Articles

Efficacy of an Extended Half-Life GlycoPEGylated rFVIII (N8-GP): Pooled Analysis of ABR (Results from Two Clinical Trials)

Pratima Chowdary, MD, Lone Hvitfeldt Poulsen, Miguel A Escobar, MD, Susan Kearney, Meera B. Chitlur, MD, Robert Klamroth, Mudi Misgav, Claude Negrier, Wan Hui Ong Clausen, Frank Driessler, Andrea Landorph, Elena Santagostino

Blood (2018) 132 (Supplement 1): 1177.

https://doi.org/10.1182/blood-2018-99-110308

Published: 2018

Journal Articles

Factor V Atlanta: A Novel Mutation in the F5 Gene Reveals Potential New Cis-Acting Elements Involved in Regulating FV-Short and TFPI Levels

Karen L Zimowski, MD, Michelle D. Ho, BS, Jordan E Shields, MS, Gabriela Denning, PhD, Teodolinda Petrillo, PhD, Navdeep Jhita, MD, Angel A Rivera, PhD, Miguel A Escobar, MD, Christine L Kempton, Christopher B Doering, PhD, Rodney M Camire, PhD

Blood (2017) 130 (Supplement 1): 366.

https://doi.org/10.1182/blood.V130.Suppl_1.366.366

Published: 2017

Journal Articles

Characterization of the HLA-Class-II (HLAcII)/Therapeutic Factor VIII (FVIII)-Derived Peptidome By FVIII Domain and HLAcII Isomer

Marcio Almeida, PhD, Vincent P. Diego, PhD, Bernadette W. Luu, B.S., Marco Hofmann, M.S., Jose Antonio G. Hernandez, PhD, Juan Peralta, Joanne Curran, Miguel A Escobar, MD, Rajalingam Raja, PhD, LV Dinh, Jerry S. Powell, MD, Sarah Williams-Blangero, John Blangero, Eugene Maraskovsky, Tom Howard

Blood (2017) 130 (Supplement 1): 2352.

https://doi.org/10.1182/blood.V130.Suppl_1.2352.2352

Published: 2017

Journal Articles

Chronic Kidney Disease in the U.S. Hemophilia Population: A Cohort Study

Blood (2017) 130 (Supplement 1): 2359.

https://doi.org/10.1182/blood.V130.Suppl_1.2359.2359

Published: 2017

Journal Articles

Risk Factors for Inhibitor Development in Mild and Moderate Hemophilia A: A Case-Control Study.

Christine L Kempton, MD, MSc, J. Michael Soucie, Ph.D, Connie H. Miller, PhD, Craig Hooper, PhD, Miguel A. Escobar, MD, Mark T. Reding, MD, Alice J. Cohen, MD, Nigel S. Key, MBChB, Arthur R. Thompson, M.D., Ph.D., Marilyn J. Manco-Johnson, Thomas C. Abshire, MD

Blood (2009) 114 (22): 3184.

https://doi.org/10.1182/blood.V114.22.3184.3184

Published: 2009

Journal Articles

Rituximab as a Treatment Option for Acquired Inhibitors.

Jonas A. de Souza, MD, Miguel A. Escobar, MD

Blood (2006) 108 (11): 4065.

https://doi.org/10.1182/blood.V108.11.4065.4065

Published: 2006

NARROW

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

Single 225μg/Kg Dose Treatment with Eptacog Beta (Factor VIIa, Recombinant) Results in Rapid Hemostasis in Joint Bleeds for Persons with Hemophilia Α or Β with Inhibitors: A PERSEPT1 Subset Analysis

N-Linked Glycans on Therapeutic Factor VIII (FVIII) Proteins Attenuate Immunogenicity Potential: Evidence from Independent HLA-Class-II/FVIII (HLAcII/FVIII) Peptidomes

PERSEPT3: A Phase 3 Clinical Trial to Evaluate the Hemostatic Efficacy of Eptacog Beta (Recombinant Human FVIIa) in Perioperative Care in Subjects with Hemophilia Α or B with Inhibitors

A Phase IV, Multicenter, Open-Label Study of Emicizumab Prophylaxis in Persons with Hemophilia a with or without FVIII Inhibitors Undergoing Minor Surgical Procedures

Specific Correction of the Intron-22 Inverted Factor VIII Gene in Autologous Blood Outgrowth Endothelial Cells from Patients with Severe Hemophilia A

Retrospective Chart Review of Gastrointestinal Bleeding in Patients with Von Willebrand Disease

Disentangling the Effects of HLA DRB115:01 and DQB106:02 to Establish the True HLA Risk Allele for Inhibitor Development in the Treatment of Hemophilia A

Consistency of Dosing of Turoctocog Alfa Pegol (N8-GP) throughout the Pathfinder Clinical Trials: Implications for Recommendations for Effective Prophylaxis, Bleed Resolution, and Perioperative Management

The Dendritic Cell HLA-Class-II/Therapeutic Factor VIII (FVIII) Peptidome Is Influenced in Unanticipated Ways By the B-Domain of FVIII and the FVIII Chaperon Protein, Von Willebrand Factor: The Outrigger and Glycosylation-Umbrella (GUMB) Hypotheses

Immune Tolerance Induction (ITI) with a Plasma-Derived Factor VIII for Patients with Hemophilia a and Inhibitors: A Retrospective Study

On the Role of F8 Sequence Mismatch and Class-II Human Leukocyte Antigen Binding in the Development of Neutralizing Antibodies ("Inhibitors") Directed Against Therapeutic Factor VIII Proteins (tFVIIIs): Evidence from the PATH Study

Chronic Kidney Disease (CKD) in the U.S. Hemophilia Population: A Cohort Study

The Role of Class II Human Leukocyte Antigens (cII-HLAs) in Determining the Immunogenic Potential of Therapeutic Factor VIII Proteins in Hemophilia Patients: The "Gate Keeper" Hypothesis

Efficacy of an Extended Half-Life GlycoPEGylated rFVIII (N8-GP): Pooled Analysis of ABR (Results from Two Clinical Trials)

Factor V Atlanta: A Novel Mutation in the F5 Gene Reveals Potential New Cis-Acting Elements Involved in Regulating FV-Short and TFPI Levels

Characterization of the HLA-Class-II (HLAcII)/Therapeutic Factor VIII (FVIII)-Derived Peptidome By FVIII Domain and HLAcII Isomer

Chronic Kidney Disease in the U.S. Hemophilia Population: A Cohort Study

Risk Factors for Inhibitor Development in Mild and Moderate Hemophilia A: A Case-Control Study.

Rituximab as a Treatment Option for Acquired Inhibitors.

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American Society of Hematology

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ASH Publications

American Society of Hematology

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