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Journal Articles

Clinical Outcomes of Noninhibitor Patients with Hemophilia a Switching from Prophylaxis with Factor VIII to Emicizumab: A Meta-Analysis of Real-World Evidence Studies

Miguel A. Escobar, Qi Angela Fan, Ali G Mokdad, Michael D. Tarantino, Benjamin Burgess, Emma Hawe, Maria Fernandez, Michael Bullano

Blood (2023) 142 (Supplement 1): 283.

https://doi.org/10.1182/blood-2023-178007

Published: 2023

Journal Articles

Results from an Association-Scan of the Extended MHC-Class-II Region Using Novel Association-Based Statistical Methods Establish That DQ Allotypes and Race Independently Influence the Risk of Factor VIII Inhibitor Development in Hemophilia-A Patients

Paola Ibarra, Marcio A. Almeida, Vincent P. Diego, Bernadette W. Luu, Kevin R. Viel, Raja Rajalingam, Afshin Ameri, Meera Chitlur, Benjamin Kim, Grecia Castillo, Jerry S. Powell, Juan M. Peralta, Miguel A. Escobar, Sarah Williams-Blangero, John Blangero, Carol K. Kasper, Laura Almasy, Tom E. Howard

Blood (2023) 142 (Supplement 1): 1247.

https://doi.org/10.1182/blood-2023-191140

Published: 2023

Journal Articles

Gene-Centric Association Scans of Pleiotropic Immune-Mediated Disease Genes in the PATH Study Identify Novel Determinants of Factor VIII Inhibitor Risk in Hemophilia-A Patients and Confirm Race as an Independent Predictor

Ruayda Bouls, Marcio A. Almeida, Vincent P. Diego, Bernadette W. Luu, Raja Rajalingam, Afshin Ameri, Kevin R. Viel, Meera Chitlur, Benjamin Kim, Miguel A. Escobar, Sarah Williams-Blangero, Carol K. Kasper, Laura Almasy, John Blangero, Tom E. Howard

Blood (2023) 142 (Supplement 1): 1246.

https://doi.org/10.1182/blood-2023-190094

Published: 2023

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

The G505A Nonsynonymous Single-Nucleotide Polymorphism (SNP) in TAFI , the Gene Encoding Thrombin-Activatable Fibrinolysis Inhibitor (TAFI) Is Pleiotropically Associated with TAFI Antigen Levels and Coronary Heart Disease (CHD) in Mexican Americans of South Texas

Shuchita Jhaveri, Vincent P. Diego, Hoang Anh Thi Nguyen, Satish Kumar, Marcio A. Almeida, Juan M Peralta, Joanne E. Curran, Bernadette W. Luu, Donna M. Lehman, Ralph A DeFronzo, Miguel A. Escobar, Sarah Williams-Blangero, Ravi Duggirala, Jerry S. Powell, Laura Almasy, John Blangero, Tom E. Howard

Blood (2021) 138 (Supplement 1): 3217.

https://doi.org/10.1182/blood-2021-151968

Published: 2021

Journal Articles

Longitudinal Efficacy and Safety of N8-GP for Prophylaxis and Bleed Control in US Patients

Allison P. Wheeler, Miguel A. Escobar, Susan Kearney, Janice M. Staber, Adam R. Wufsus, Vlady Ostrow, Steven R. Lentz

Blood (2021) 138 (Supplement 1): 3205.

https://doi.org/10.1182/blood-2021-147814

Published: 2021

Journal Articles

Athn 16: Safety of Coagulation Factor VIIa (recombinant)-Jncw for the Treatment of Bleeding Events in Patients with Congenital Hemophilia a or B with Inhibitors with or without Prophylactic Treatment

Tammuella Chrisentery-Singleton, W Allan Alexander, Ahmad Al-Sabbagh, Daniel Bonzo, Michael U. Callaghan, Miguel A. Escobar, Adam Giermasz, Nikki Hirsh, Janna Journeycake, Sonia Nasr, Doris V. Quon, Michael Recht

Blood (2021) 138 (Supplement 1): 3201.

https://doi.org/10.1182/blood-2021-146247

Published: 2021

Journal Articles

The Factor II (FII) Expression Quantitative Trait Locus (eQTL) Prothrombin G20210A Is Pleiotropically Associated with Plasma Fibrinogen Levels and Has a Profound Effect on Obesity in Mexican Americans of South Texas

Hoang Anh Thi Nguyen, Vincent P. Diego, Shuchita Jhaveri, Marcio A. Almeida, Satish Kumar, Juan M Peralta, Joanne E. Curran, Bernadette W. Luu, Donna M. Lehman, Ralph A DeFronzo, Miguel A. Escobar, Sarah Williams-Blangero, Ravi Duggirala, Jerry S. Powell, Laura Almasy, John Blangero, Tom E. Howard

Blood (2021) 138 (Supplement 1): 1059.

https://doi.org/10.1182/blood-2021-154338

Published: 2021

Journal Articles

Prophylactic Treatment with Rurioctocog Alfa Pegol Results in a Dose-Dependent Normalization of Biomarkers of Joint Health in Severe Hemophilia a: An Exploratory Analysis from the Propel Study

Tina Manon-Jensen, Srilatha Tangada, Cecilie Bager, Pratima Chowdary, Robert Klamroth, Annette von Drygalski, Jerzy Windyga, Miguel A. Escobar, Peder Frederiksen, Werner Engl, Bruce Ewenstein, Morten Karsdal

Blood (2021) 138 (Supplement 1): 2102.

https://doi.org/10.1182/blood-2021-150139

Published: 2021

Journal Articles

BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression

Barbara A. Konkle, Christopher E. Walsh, Miguel A. Escobar, Neil C. Josephson, Guy Young, Annette von Drygalski, Scott W. J. McPhee, R. Jude Samulski, Ivan Bilic, Maurus de la Rosa, Birgit M. Reipert, Hanspeter Rottensteiner, Friedrich Scheiflinger, John C. Chapin, Bruce Ewenstein, Paul E. Monahan

Blood (2021) 137 (6): 763–774.

https://doi.org/10.1182/blood.2019004625

Published: 2021

Includes: Supplemental data

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

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

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

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

NARROW

Clinical Outcomes of Noninhibitor Patients with Hemophilia a Switching from Prophylaxis with Factor VIII to Emicizumab: A Meta-Analysis of Real-World Evidence Studies

Results from an Association-Scan of the Extended MHC-Class-II Region Using Novel Association-Based Statistical Methods Establish That DQ Allotypes and Race Independently Influence the Risk of Factor VIII Inhibitor Development in Hemophilia-A Patients

Gene-Centric Association Scans of Pleiotropic Immune-Mediated Disease Genes in the PATH Study Identify Novel Determinants of Factor VIII Inhibitor Risk in Hemophilia-A Patients and Confirm Race as an Independent Predictor

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

The G505A Nonsynonymous Single-Nucleotide Polymorphism (SNP) in TAFI , the Gene Encoding Thrombin-Activatable Fibrinolysis Inhibitor (TAFI) Is Pleiotropically Associated with TAFI Antigen Levels and Coronary Heart Disease (CHD) in Mexican Americans of South Texas

Longitudinal Efficacy and Safety of N8-GP for Prophylaxis and Bleed Control in US Patients

Athn 16: Safety of Coagulation Factor VIIa (recombinant)-Jncw for the Treatment of Bleeding Events in Patients with Congenital Hemophilia a or B with Inhibitors with or without Prophylactic Treatment

The Factor II (FII) Expression Quantitative Trait Locus (eQTL) Prothrombin G20210A Is Pleiotropically Associated with Plasma Fibrinogen Levels and Has a Profound Effect on Obesity in Mexican Americans of South Texas

Prophylactic Treatment with Rurioctocog Alfa Pegol Results in a Dose-Dependent Normalization of Biomarkers of Joint Health in Severe Hemophilia a: An Exploratory Analysis from the Propel Study

BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression

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

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

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

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

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American Society of Hematology

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