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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

Development and Validation of a Patient/Caregiver-Report Hydroxyurea Treatment Adherence Scale

Natalie Duncan, MPH, William Kronenberger, PhD, Chris Roberson, JD, MPH, Kisha Hampton, MPH, Ellen Bloom, MPH, CHES, Brandon M. Hardesty, MD, Amy D. Shapiro, MD, Emily Riehm Meier, MD MSHS

Blood (2017) 130 (Supplement 1): 3366.

https://doi.org/10.1182/blood.V130.Suppl_1.3366.3366

Published: 2017

Journal Articles

Clinical Outcomes of 14-Day Prophylaxis with rFIXFc: Longitudinal Analysis of the B-LONG and B-YOND Study

Amy D. Shapiro, MD, Margareth C Ozelo, MD PhD, K John Pasi, Jingyuan Feng, Bent Winding, Chris Barnowski, Roshni Kulkarni

Blood (2017) 130 (Supplement 1): 3667.

https://doi.org/10.1182/blood.V130.Suppl_1.3667.3667

Published: 2017

Journal Articles

Development of a Concept-Driven Disease Impact Instrument from Patients with Hemophilia (Pediatric and Adult) and Caregivers

Jessica T. Markowitz, Esprit Ma, Paul Solari, Patrick Marquis, Amy D. Shapiro, MD, Barbara Konkle, MD, Peter Trask, Edmund Pezalla, Anna Christian, Michelle Rice, Wendy E. Owens, MA, Kimberly Haugstad, Yaping Xu, Richard H. Ko, Jennifer Whiteley, Robert F. Sidonio

Blood (2017) 130 (Supplement 1): 4732.

https://doi.org/10.1182/blood.V130.Suppl_1.4732.4732

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

Clinical Outcomes of Weekly Prophylaxis with rFVIIIFc: Longitudinal Analysis of the A-LONG and ASPIRE Study Population

Amy D. Shapiro, MD, Alok Srivastava, MD, Margaret V. Ragni, MD MPH, Ingrid Pabinger, MD, Stefan Lethagen, Elisa Tsao, Desilu Glazebrook, Doris Quon

Blood (2017) 130 (Supplement 1): 2368.

https://doi.org/10.1182/blood.V130.Suppl_1.2368.2368

Published: 2017

Journal Articles

Computer Modeling Using Historical Data Demonstrates a Significant Reduction in Expected Extravascular Fibrinous Lesions Due to Congenital Plasminogen Deficiency While Receiving Intravenous Plasminogen (PLG) Replacement

Joseph M Parker, MD, Danielle Craig, Marcia Gaido, Gary Albert, Arnaud Monseur, Amy D. Shapiro, MD, John Moran, MD

Blood (2017) 130 (Supplement 1): 2371.

https://doi.org/10.1182/blood.V130.Suppl_1.2371.2371

Published: 2017

Journal Articles

Longitudinal Antibody Signatures Following FVIII Replacement Therapy in Previously Untreated Patients with Severe Hemophilia Α- New Insights from the Hemophilia Inhibitor PUP Study (HIPS)

Bagirath Gangadharan, PhD, Birgit Reipert, Fritz Scheiflinger, PhD, Joel Bowen, Elizabeth Donnachie, PhD, Karin Fijn van Draat, Ralph A. Gruppo, MD, Jenny Klintman, MD PhD, Christoph Male, MD, Catherine E. McGuinn, MD, Shannon L. Meeks, MD, Michael Recht, MD PhD, Margaret V. Ragni, MD MPH, Hassan M. Yaish, MD, Amy D. Shapiro, MD, Donald L Yee, MD, Vlad Calin Radulescu, MD, Elena Santagostino, Deborah Brown, MD

Blood (2017) 130 (Supplement 1): 88.

https://doi.org/10.1182/blood.V130.Suppl_1.88.88

Published: 2017

Journal Articles

Pivotal Trial with Intravenous Plasminogen Replacement in Patients with Plasminogen Deficiency Demonstrates Long-Term Efficacy for Treatment and Prevention of Ligneous Lesions

Charles T Nakar, MD, Joseph M Parker, MD, Neelam Thakral, Brandon M. Hardesty, MD, Gary Albert, Pierre Laurin, MSc, BPharm, John Moran, MD, Per Morten Sandset, MD PhD, Amy D. Shapiro, MD

Blood (2017) 130 (Supplement 1): 84.

https://doi.org/10.1182/blood.V130.Suppl_1.84.84

Published: 2017

Journal Articles

Treatment of Patients with Severe Congenital Protein C Deficiency in a Registry Study of Protein C Concentrate (Human)

Marilyn Manco-Johnson, MD, Hans-Joerg Hertfelder, MD PhD, Theodosia A. Kalfa, MD PhD, Emmett H. Broxson, Jr., MD, Lisa Bomgaars, MD, Amy D. Shapiro, MD, Dyck-Jones Jacqueline, PhD, Sandra Ehrle, MD, Maureen Finnerty, David Gelmont, MD, Leman Yel, MD, Mahmoud Loghman-Adham, MD

Blood (2016) 128 (22): 2605.

https://doi.org/10.1182/blood.V128.22.2605.2605

Published: 2016

Journal Articles

Heterozygosity for Loss-of-Function Mutation in SERPINE1 (PAI-1 Gene) Linked with Longer Absolute Telomere Length

Sweta Gupta, Sadiya Khan, Sanjiv Shah, Ekaterina Klyachko, Abigail S Baldridge, Mesut Eren, Aaron T Place, Meadow Heiman, Abraham Aviv, Amy D. Shapiro, MD, Douglas E Vaughan

Blood (2016) 128 (22): 4953.

https://doi.org/10.1182/blood.V128.22.4953.4953

Published: 2016

Journal Articles

Extended-Interval Prophylaxis with rFIXFc in Adults and Adolescents with Hemophilia B: Interim Results of the B-YOND Extension Study

Johnny Mahlangu, Amy D Shapiro, MD, K John Pasi, Margaret V Ragni, Margareth Castro Ozelo, MD PhD, Johannes Oldenburg, Tadashi Matsushita, MD PhD, Ross I Baker, MD, Huixing Yuan, Alejandra Ramirez-Santiago, Glenn F Pierce, Geoffrey Allen, Baisong Mei

Blood (2015) 126 (23): 1088.

https://doi.org/10.1182/blood.V126.23.1088.1088

Published: 2015

Journal Articles

Safety & Efficacy of Human Plasma Derived Plasminogen Ophthalmic Drops for Treatment of Ligneous Conjunctivitis: Report of Phase 2/3 Clinical Trial

Charles T Nakar, MD, Roberto Caputo, MD, Francis W Price, Jr., MD, Maria Teresa Sartori, MD, Andrea Leonardi, Anna Lotti Suffredini, Neelam Thukral, Jacopo Secci, Bennie H Jeng, MD, Amy D Shapiro, MD

Blood (2015) 126 (23): 2288.

https://doi.org/10.1182/blood.V126.23.2288.2288

Published: 2015

Journal Articles

Non-Factor VIII Coagulation Proteins Fail to Predict Joint Outcome in Severe Hemophilia a

Beth Boulden Warren, MD MS, Linda Jacobsen, BS, Elizabeth Villalobos-Menuey, BS, Thomas C. Abshire, MD, Amy D Shapiro, MD, Michael Recht, MD PhD, Deborah Brown, MD, Cindy A. Leissinger, MD, Christine L. Kempton, MD MSc, Heidi Lane, PT, DPT, PCS, Diane J. Nugent, MD, Marilyn J. Manco-Johnson, MD

Blood (2015) 126 (23): 2289.

https://doi.org/10.1182/blood.V126.23.2289.2289

Published: 2015

Journal Articles

Analysis of Target Joint Bleeding with Prophylactic Use of Recombinant Factor IX Fc Fusion Protein in Patients with Severe Hemophilia B

Amy D Shapiro, MD, David J Perry, Ross I Baker, MD, Elisa Tsao, Baisong Mei

Blood (2015) 126 (23): 2294.

https://doi.org/10.1182/blood.V126.23.2294.2294

Published: 2015

Journal Articles

Low Bleeding Rates with Increase or Maintenance of Physical Activity in Patients Treated with Recombinant Factor IX Fc Fusion Protein (rFIXFc) in the B-LONG and Kids B-LONG Studies

Roshni Kulkarni, Amy D Shapiro, MD, Jerzy Windyga, Margaret V Ragni, K John Pasi, Margareth Castro Ozelo, MD PhD, Elisa Tsao, Baisong Mei

Blood (2015) 126 (23): 2307.

https://doi.org/10.1182/blood.V126.23.2307.2307

Published: 2015

Journal Articles

Pharmacokinetics of Intravenous Infusion of Glu-Plasminogen Concentrate in Patients with Hypoplasminogenemia

Brandon M. Hardesty, MD, Stacy Plum, PhD, Karen Thibaudeau, PhD, Martin Lee, PhD, Amy D. Shapiro, MD

Blood (2015) 126 (23): 3498.

https://doi.org/10.1182/blood.V126.23.3498.3498

Published: 2015

NARROW

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

Development and Validation of a Patient/Caregiver-Report Hydroxyurea Treatment Adherence Scale

Clinical Outcomes of 14-Day Prophylaxis with rFIXFc: Longitudinal Analysis of the B-LONG and B-YOND Study

Development of a Concept-Driven Disease Impact Instrument from Patients with Hemophilia (Pediatric and Adult) and Caregivers

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

Clinical Outcomes of Weekly Prophylaxis with rFVIIIFc: Longitudinal Analysis of the A-LONG and ASPIRE Study Population

Computer Modeling Using Historical Data Demonstrates a Significant Reduction in Expected Extravascular Fibrinous Lesions Due to Congenital Plasminogen Deficiency While Receiving Intravenous Plasminogen (PLG) Replacement

Longitudinal Antibody Signatures Following FVIII Replacement Therapy in Previously Untreated Patients with Severe Hemophilia Α- New Insights from the Hemophilia Inhibitor PUP Study (HIPS)

Pivotal Trial with Intravenous Plasminogen Replacement in Patients with Plasminogen Deficiency Demonstrates Long-Term Efficacy for Treatment and Prevention of Ligneous Lesions

Treatment of Patients with Severe Congenital Protein C Deficiency in a Registry Study of Protein C Concentrate (Human)

Heterozygosity for Loss-of-Function Mutation in SERPINE1 (PAI-1 Gene) Linked with Longer Absolute Telomere Length

Extended-Interval Prophylaxis with rFIXFc in Adults and Adolescents with Hemophilia B: Interim Results of the B-YOND Extension Study

Safety & Efficacy of Human Plasma Derived Plasminogen Ophthalmic Drops for Treatment of Ligneous Conjunctivitis: Report of Phase 2/3 Clinical Trial

Non-Factor VIII Coagulation Proteins Fail to Predict Joint Outcome in Severe Hemophilia a

Analysis of Target Joint Bleeding with Prophylactic Use of Recombinant Factor IX Fc Fusion Protein in Patients with Severe Hemophilia B

Low Bleeding Rates with Increase or Maintenance of Physical Activity in Patients Treated with Recombinant Factor IX Fc Fusion Protein (rFIXFc) in the B-LONG and Kids B-LONG Studies

Pharmacokinetics of Intravenous Infusion of Glu-Plasminogen Concentrate in Patients with Hypoplasminogenemia

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American Society of Hematology

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American Society of Hematology

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