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Journal Articles

Evaluation of a newly formulated intranasal desmopressin versus intranasal and subcutaneous desmopressin in individuals with type 1 von Willebrand disease and mild Hemophilia A

Sanjay Ahuja, Sweta Gupta, Magdalena Lewandowska, Jennifer Maahs, Sonia Nasr, Laura Swystun, Amy Shapiro

Blood (2025) 146 (Supplement 1): 2688.

https://doi.org/10.1182/blood-2025-2688

Published: 2025

Journal Articles

NXT007 prophylaxis in people with hemophilia A with or without FVIII inhibitors: A global Phase I/II multiple-ascending-dose study

Maria Elisa Mancuso, Davide Matino, Dan Hart, Francisco-Jose Lopez-Jaime, Christophe Schmitt, Amy Shapiro, Giuliana Ventriglia, Anna Kiialainen, Laura Young, Olivier Catalani, Mark Belletrutti, Tom Chu, Jerzy Windyga, Víctor Jiménez-Yuste, Michaela Lehle, Janice Staber

Blood (2025) 146 (Supplement 1): 302.

https://doi.org/10.1182/blood-2025-302

Published: 2025

Journal Articles

Pharmacodynamic biomarkers in people with hemophilia A receiving multiple ascending doses of NXT007

Anna Kiialainen, Christophe Schmitt, Davide Matino, Janice Staber, Maria Elisa Mancuso, Víctor Jiménez-Yuste, Jerzy Windyga, Amy Shapiro, Matthew Locke, Dan Hart

Blood (2025) 146 (Supplement 1): 4841.

https://doi.org/10.1182/blood-2025-4841

Published: 2025

Journal Articles

Treatment of type 1 plasminogen deficiency in women and girls with reproductive tract lesions: Pharmacokinetics and clinical outcomes with plasminogen therapy

Karen Thibaudeau, Per Morten Sandset, Anne Flem Jacobsen, Joseph Parker, Amy Shapiro

Blood (2025) 146 (Supplement 1): 1310.

https://doi.org/10.1182/blood-2025-1310

Published: 2025

Journal Articles

Efficacy and Safety of Subcutaneous Prophylaxis with Concizumab in Patients with Hemophilia a or B with Inhibitors: Results from explorer4, a Phase 2, Randomized, Open-Label, Controlled Trial

Amy Shapiro, Giancarlo Castaman, MD, Katarina Cepo, Lone Hvitfeldt Poulsen, Christian Hollensen, Tadashi Matsushita, Guy Young, MD, Silva Zupancic-Salek, Victor Jimenez-Yuste

Blood (2019) 134 (Supplement_1): 1139.

https://doi.org/10.1182/blood-2019-122809

Published: 2019

Journal Articles

An Update on rFVIIa Use in Females with Rare Bleeding Disorders

Meera B. Chitlur, MD, Manuel Carcao, David Cooper, Shilpa Jain, Kaan Kavakli, Bryce Kerlin, Skye Peltier, Man-Chiu Poon, Hossam Saad, Amy Shapiro

Blood (2019) 134 (Supplement_1): 1119.

https://doi.org/10.1182/blood-2019-130298

Published: 2019

Journal Articles

Early Prophylaxis Provides Continued Joint Protection in Severe Hemophilia A: Results of the Joint Outcome Continuation Study

Beth Boulden Warren, MD MS, Dianne Thornhill, PhD, Jill Stein, MD, Michael Fadell, MD, Sharon Funk, DPT, Heidi D. Lane, DPT, Carolyn M. Bennett, MD MSc, Amy Shapiro, Michael Recht, MD PhD, Marilyn J Manco-Johnson, MD

Blood (2018) 132 (Supplement 1): 382.

https://doi.org/10.1182/blood-2018-99-117413

Published: 2018

Journal Articles

Data Coming out of the Human Inhibitor PUP Study (HIPS) Reveal 4 Subgroups of Patients with Distinct Antibody Signatures

Blood (2018) 132 (Supplement 1): 3774.

https://doi.org/10.1182/blood-2018-99-115979

Published: 2018

Journal Articles

Prospective Diagnosis of VWD in a Large Cohort of Patients with Bleeding Symptoms through the Zimmerman Program

Veronica H Flood, MD, Pamela A Christopherson, Joan Cox Gill, MD, Kenneth Dale Friedman, MD, Sandra L Haberichter, PhD, Jorge Di Paola, MD, Paula D. James, MD, Janna M. Journeycake, MD, Steven R. Lentz, MD PhD, Cindy A. Leissinger, MD, Margaret V. Ragni, MD MPH, Madhvi Rajpurkar, MD, Jonathan C. Roberts, MD, Amy Shapiro, Robert F. Sidonio, Robert R Montgomery, MD, Thomas C. Abshire, MD

Blood (2018) 132 (Supplement 1): 979.

https://doi.org/10.1182/blood-2018-99-110650

Published: 2018

Journal Articles

BIVV001: The First Investigational Factor VIII Therapy to Break Through the VWF Ceiling in Hemophilia A, with Potential for Extended Protection for One Week or Longer

Barbara A Konkle, Amy Shapiro, Doris Quon, Janice Staber, Takashi Suzuki, MD PhD, Stacey Poloskey, Kara Rice, Suresh Katragadda, Dan Rudin, Joachim Fruebis

Blood (2018) 132 (Supplement 1): 636.

https://doi.org/10.1182/blood-2018-99-113575

Published: 2018

Journal Articles

Surveillance of Hemophilia in Indiana: A Preliminary Report

Amanda Okolo, MPH, John M Soucie, PhD, Scott D. Grosse, PhD, Chris Roberson, JD, MPH, Isaac Janson, PhD, Martha Allen, MSN, Amy Shapiro

Blood (2018) 132 (Supplement 1): 3516.

https://doi.org/10.1182/blood-2018-99-118849

Published: 2018

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

Real-World Data on the Use of rFIXFc in Subjects With Hemophilia B for Up to 3.7 Years Demonstrates Improved Bleed Control and Adherence With Reduced Treatment Burden

Amy Shapiro, Ateefa Chaudhury, Nisha Jain, Elisa Tsao, Chris Barnowski, Jing Feng, Doris Quon

Blood (2018) 132 (Supplement 1): 2493.

https://doi.org/10.1182/blood-2018-99-116909

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

On-Demand Treatment of Bleeding Episodes in Patients with Hereditary Factor X Deficiency Using a High-Purity Factor X Concentrate: Data from 2 Clinical Trials and a Data-Collection Study

James N. Huang, MD, Chioma Akanezi, BSc, PgDip, Amy Shapiro

Blood (2018) 132 (Supplement 1): 1215.

https://doi.org/10.1182/blood-2018-99-111397

Published: 2018

Journal Articles

B-YOND Final Results Confirm Established Safety, Sustained Efficacy, and Extended Dosing Interval for Up to 4 Years of Treatment With rFIXFc in Previously Treated Subjects With Severe Hemophilia B

Margaret Ragni, Roshni Kulkarni, K. John Pasi, MB, ChB, PhD FRCP, FRCPath, FRCPCH, Kathelijn Fischer, Johnny Mahlangu, Amy Shapiro, Beatrice Nolan, Johannes Oldenburg, Tadashi Matsushita, Annemieke Willemze, Huixing Yuan, Dan Rudin

Blood (2018) 132 (Supplement 1): 1214.

https://doi.org/10.1182/blood-2018-99-110100

Published: 2018

Journal Articles

Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A

Blood (2014) 123 (3): 317–325.

https://doi.org/10.1182/blood-2013-10-529974

Published: 2014

Includes: Supplemental data

Journal Articles

SNP Analysis Of The VWF GENE Identifies Multiple Common Variants THAT Affect VWF Levels and OCCUR At Different Frequencies In Patients With TYPE 1 VWD

Jesse Hinckley, PhD, Trudy Burns, PhD, Kai Wang, PhD, Ayse Bilge Ozel, PhD, Meadow Heiman, Amy Shapiro, MD, Robert R. Montgomery, MD, Lawrence Brody, PhD, Karl C. Desch, MD, Jorge Di Paola, MD

Blood (2013) 122 (21): 334.

https://doi.org/10.1182/blood.V122.21.334.334

Published: 2013

Journal Articles

Dosing Long-Lasting Recombinant Factor VIII Fc Fusion Protein: Experience In The A-LONG Study

Amy Shapiro, MD, Margaret V. Ragni, MD, MPH, Roshni Kulkarni, MD, Sarah Kulke, M.D., James Potts, Ph.D, MPH, Srividya Neelakantan, Ph.D., Ivan Nestorov, Ph.D., Jennifer A Dumont, PhD, Haiyan Jiang, PhD, Aoife Brennan, MB, BCh, BAO, MMSC, Glenn F Pierce, MD, PhD

Blood (2013) 122 (21): 3598.

https://doi.org/10.1182/blood.V122.21.3598.3598

Published: 2013

Journal Articles

Association Of Bleeding Tendency With Time Under Target FIX Activity Levels In Severe Hemophilia B Patients Treated With Recombinant Factor IX Fc Fusion Protein

Amy Shapiro, MD, James Potts, Ph.D, MPH, Shuanglian Li, Ph.D., Leonard A. Valentino, MD, FAAP, Lei Diao, PhD, Ping Wang, Ph.D., Brian Robinson, M.D., Glenn F Pierce, MD, PhD, Haiyan Jiang, PhD

Blood (2013) 122 (21): 2349.

https://doi.org/10.1182/blood.V122.21.2349.2349

Published: 2013

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Evaluation of a newly formulated intranasal desmopressin versus intranasal and subcutaneous desmopressin in individuals with type 1 von Willebrand disease and mild Hemophilia A

NXT007 prophylaxis in people with hemophilia A with or without FVIII inhibitors: A global Phase I/II multiple-ascending-dose study

Pharmacodynamic biomarkers in people with hemophilia A receiving multiple ascending doses of NXT007

Treatment of type 1 plasminogen deficiency in women and girls with reproductive tract lesions: Pharmacokinetics and clinical outcomes with plasminogen therapy

Efficacy and Safety of Subcutaneous Prophylaxis with Concizumab in Patients with Hemophilia a or B with Inhibitors: Results from explorer4, a Phase 2, Randomized, Open-Label, Controlled Trial

An Update on rFVIIa Use in Females with Rare Bleeding Disorders

Early Prophylaxis Provides Continued Joint Protection in Severe Hemophilia A: Results of the Joint Outcome Continuation Study

Data Coming out of the Human Inhibitor PUP Study (HIPS) Reveal 4 Subgroups of Patients with Distinct Antibody Signatures

Prospective Diagnosis of VWD in a Large Cohort of Patients with Bleeding Symptoms through the Zimmerman Program

BIVV001: The First Investigational Factor VIII Therapy to Break Through the VWF Ceiling in Hemophilia A, with Potential for Extended Protection for One Week or Longer

Surveillance of Hemophilia in Indiana: A Preliminary Report

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

Real-World Data on the Use of rFIXFc in Subjects With Hemophilia B for Up to 3.7 Years Demonstrates Improved Bleed Control and Adherence With Reduced Treatment Burden

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

On-Demand Treatment of Bleeding Episodes in Patients with Hereditary Factor X Deficiency Using a High-Purity Factor X Concentrate: Data from 2 Clinical Trials and a Data-Collection Study

B-YOND Final Results Confirm Established Safety, Sustained Efficacy, and Extended Dosing Interval for Up to 4 Years of Treatment With rFIXFc in Previously Treated Subjects With Severe Hemophilia B

Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A

SNP Analysis Of The VWF GENE Identifies Multiple Common Variants THAT Affect VWF Levels and OCCUR At Different Frequencies In Patients With TYPE 1 VWD

Dosing Long-Lasting Recombinant Factor VIII Fc Fusion Protein: Experience In The A-LONG Study

Association Of Bleeding Tendency With Time Under Target FIX Activity Levels In Severe Hemophilia B Patients Treated With Recombinant Factor IX Fc Fusion Protein

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