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Journal Articles

Innovations in RNA therapy for hemophilia

Margaret V. Ragni, Stephen Y. Chan

Blood (2023) 142 (19): 1613–1621.

https://doi.org/10.1182/blood.2022018661

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

Neutrophil-Dependent Immune Response Promotes Joint Damage in Hemophilia

Tomasz Waldemar Kaminski, Omika Katoch, Margaret V. Ragni, Prithu Sundd

Blood (2023) 142 (Supplement 1): 5351.

https://doi.org/10.1182/blood-2023-190711

Published: 2023

Journal Articles

Predictors of Liver Disease Outcomes in People with Hemophilia and HCV Infection

Ilias Christodoulou, Scott Rothenberger, Margaret V. Ragni

Blood (2023) 142 (Supplement 1): 3984.

https://doi.org/10.1182/blood-2023-174329

Published: 2023

Journal Articles

Prevalence and Risk Factors for Bleeding in Individuals with Hereditary Hemorrhagic Telangiectasia: A National Inpatient Sample Study

Jabra Zarka, Kwonho Jeong, Jonathan G. Yabes, Margaret V. Ragni

Blood (2022) 140 (Supplement 1): 5584–5585.

https://doi.org/10.1182/blood-2022-170616

Published: 2022

Journal Articles

Prevalence of Heavy Menstrual Bleeding and Use of Hormonal Therapy Among Women Type 1 Von Willebrand Disease from the Zimmerman Program

Nicoletta C Machin, Scott Rothenberger, Veronica H Flood, Pamela A Christopherson, Robert R Montgomery, Margaret V. Ragni

Blood (2022) 140 (Supplement 1): 8447–8448.

https://doi.org/10.1182/blood-2022-168026

Published: 2022

Journal Articles

Long-Term Durable FVIII Expression with Improvements in Bleeding Rates Following AAV-Mediated FVIII Gene Transfer for Hemophilia A: Multiyear Follow-up on the Phase I/II Trial of SPK-8011

Blood (2022) 140 (Supplement 1): 1899–1901.

https://doi.org/10.1182/blood-2022-158903

Published: 2022

Journal Articles

Multicenter, Randomized Crossover Trial Comparing Recombinant Von Willebrand Factor and Tranexamic Acid for Heavy Menstrual Bleeding in Von Willebrand Disease

Blood (2022) 140 (Supplement 1): 1413–1415.

https://doi.org/10.1182/blood-2022-156921

Published: 2022

Journal Articles

Rapid Clearance of Vector Following AAV-Mediated FVIII Gene Transfer in the Phase I/II Trial of SPK-8011 in People with Hemophilia A

Huyen Tran, M. Elaine Eyster, Stacy E. Croteau, Margaret V. Ragni, Benjamin J. Samelson-Jones, Spencer Sullivan, John E.J. Rasko, Kristen Jaworski, Amy MacDougall, Savina Jaeger, Magdalena Hofer, Charlie Li, Federico Mingozzi, Tiffany Chang, Gallia Levy

Blood (2022) 140 (Supplement 1): 10662–10664.

https://doi.org/10.1182/blood-2022-159027

Published: 2022

Journal Articles

Notch Pathway Driven Delayed Dedifferentiation of Liver Sinusoidal Endothelium Impacts Liver-Directed Gene Transfer in Hemophilia-a Mice

Tirthadipa Pradhan-Sundd, Tomasz W. Kaminski, Rikesh K.K. Dubey, Shweta Gudapati, Omika Katoch, Jesús Tejero, Enrico M. Novelli, Margaret V. Ragni, Prithu Sundd

Blood (2022) 140 (Supplement 1): 10658–10659.

https://doi.org/10.1182/blood-2022-170050

Published: 2022

Journal Articles

Neutrophil Extracellular Traps Promote Joint Injury in Hemophilia

Tomasz W. Kaminski, Tomasz Brzoska, Egemen Tutuncuoglu, Margaret V. Ragni, Prithu Sundd

Blood (2021) 138 (Supplement 1): 990.

https://doi.org/10.1182/blood-2021-153645

Published: 2021

Journal Articles

Iron Ladies: Variation in the Identification and Management of Iron Deficiency in Women with Bleeding Disorders

Meghan McCormick, Margaret V. Ragni

Blood (2021) 138 (Supplement 1): 1036.

https://doi.org/10.1182/blood-2021-148076

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

The Von Willebrand Disease Aging and Bleeding Correlation (VWD ABC) Study

Craig D. Seaman, Margaret V. Ragni, Peter A. Kouides, Eric H. Kraut, Rajiv K. Pruthi, Leslie J. Raffini, Jonathan C. Roberts, Annette von Drygalski, Daniel B. Bellissimo, Maria M Brooks

Blood (2021) 138 (Supplement 1): 1044.

https://doi.org/10.1182/blood-2021-152755

Published: 2021

Journal Articles

Prevalence and Risk Factor for Hepatocellular Carcinoma in Individuals with Hemophilia in the Era of Direct-Acting Antiviral Agents: A National Inpatient Sample Study

XI Yang, Kwonho Jeong, Jonathan G. Yabes, Margaret V. Ragni

Blood (2021) 138 (Supplement 1): 593.

https://doi.org/10.1182/blood-2021-151713

Published: 2021

Journal Articles

Genotype Analysis of Adolescents with Heavy Menstrual Bleeding and Low Von Willebrand Activity - Report of a Multi-Center Study

Brooke Sadler, PhD, Charles Minard, PhD, Gabe Haller, Christina Gurnett, Sarah H. O'Brien, MD, Allison P. Wheeler, MD, Eric S. Mullins, MD, Mukta Sharma, MD MPH, Robert F. Sidonio, Jr., MD MSc., Shilpa Jain, Ayesha Zia, MD, Margaret V. Ragni, MDMPH, Roshni Kulkarni, MD, Jennifer Dietrich, Peter A. Kouides, MD, Jorge Di Paola, Lakshmi Srivaths, MD

Blood (2020) 136 (Supplement 1): 19–20.

https://doi.org/10.1182/blood-2020-135886

Published: 2020

Journal Articles

Long-Term Durability, Safety and Efficacy of Fitusiran Prophylaxis in People with Hemophilia a or B, with or without Inhibitors - Results from the Phase II Study

Steven W. Pipe, MD, John Pasi, MB, ChB, PhD FRCP, FRCPath, FRCPCH, Toshko Lissitchkov, Margaret V. Ragni, MDMPH, Claude Négrier, Qifeng Yu, Stacey Poloskey, Baisong Mei, Shauna R. Andersson

Blood (2020) 136 (Supplement 1): 3–4.

https://doi.org/10.1182/blood-2020-136501

Published: 2020

Journal Articles

Inhibit Clinical Trials Platform to Prevent and Eradicate Inhibitors: Feasibility Survey of Current Prophylaxis and Immune Tolerance Practices

Blood (2020) 136 (Supplement 1): 14–15.

https://doi.org/10.1182/blood-2020-136814

Published: 2020

Journal Articles

Dosing, Patient Satisfaction and Other Patient-Reported Outcomes after Switching to Rurioctocog Alfa Pegol in Athn 2: A Longitudinal, Observational Study of Previously Treated Hemophilia Patients Switching Coagulation Replacement Factor Products

Janna M. Journeycake, MD, Michael Recht, MD PhD, Christine Guelcher, Hemostasis RN-BC, MS, PPCNP-BC, Ellis J. Neufeld, Margaret V. Ragni, MDMPH, Robert F. Sidonio, Jr., MDMSc, Cliff Takemoto, Michael D Tarantino, MD, Dunlei Cheng, PhD, Susan U. Lattimore, Jorge Caicedo, Michael Denne, PharmD

Blood (2020) 136 (Supplement 1): 17–18.

https://doi.org/10.1182/blood-2020-138898

Published: 2020

Journal Articles

Weight-Based Dosing in Hemophilia a: A Randomized, Controlled, Open-Label, Crossover Trial to Measure Factor VIII Recovery Following Factor VIII Concentrate Dosing Based on Total Body Weight, Ideal Body Weight, and Lean Body Mass

Craig D. Seaman, Margaret V. Ragni, MDMPH, Jonathan G. Yabes, PhD, Christina Lalama

Blood (2020) 136 (Supplement 1): 3.

https://doi.org/10.1182/blood-2020-138544

Published: 2020

NARROW

Innovations in RNA therapy for hemophilia

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

Neutrophil-Dependent Immune Response Promotes Joint Damage in Hemophilia

Predictors of Liver Disease Outcomes in People with Hemophilia and HCV Infection

Prevalence and Risk Factors for Bleeding in Individuals with Hereditary Hemorrhagic Telangiectasia: A National Inpatient Sample Study

Prevalence of Heavy Menstrual Bleeding and Use of Hormonal Therapy Among Women Type 1 Von Willebrand Disease from the Zimmerman Program

Long-Term Durable FVIII Expression with Improvements in Bleeding Rates Following AAV-Mediated FVIII Gene Transfer for Hemophilia A: Multiyear Follow-up on the Phase I/II Trial of SPK-8011

Multicenter, Randomized Crossover Trial Comparing Recombinant Von Willebrand Factor and Tranexamic Acid for Heavy Menstrual Bleeding in Von Willebrand Disease

Rapid Clearance of Vector Following AAV-Mediated FVIII Gene Transfer in the Phase I/II Trial of SPK-8011 in People with Hemophilia A

Notch Pathway Driven Delayed Dedifferentiation of Liver Sinusoidal Endothelium Impacts Liver-Directed Gene Transfer in Hemophilia-a Mice

Neutrophil Extracellular Traps Promote Joint Injury in Hemophilia

Iron Ladies: Variation in the Identification and Management of Iron Deficiency in Women with Bleeding Disorders

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

The Von Willebrand Disease Aging and Bleeding Correlation (VWD ABC) Study

Prevalence and Risk Factor for Hepatocellular Carcinoma in Individuals with Hemophilia in the Era of Direct-Acting Antiviral Agents: A National Inpatient Sample Study

Genotype Analysis of Adolescents with Heavy Menstrual Bleeding and Low Von Willebrand Activity - Report of a Multi-Center Study

Long-Term Durability, Safety and Efficacy of Fitusiran Prophylaxis in People with Hemophilia a or B, with or without Inhibitors - Results from the Phase II Study

Inhibit Clinical Trials Platform to Prevent and Eradicate Inhibitors: Feasibility Survey of Current Prophylaxis and Immune Tolerance Practices

Dosing, Patient Satisfaction and Other Patient-Reported Outcomes after Switching to Rurioctocog Alfa Pegol in Athn 2: A Longitudinal, Observational Study of Previously Treated Hemophilia Patients Switching Coagulation Replacement Factor Products

Weight-Based Dosing in Hemophilia a: A Randomized, Controlled, Open-Label, Crossover Trial to Measure Factor VIII Recovery Following Factor VIII Concentrate Dosing Based on Total Body Weight, Ideal Body Weight, and Lean Body Mass

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American Society of Hematology

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ASH Publications

American Society of Hematology

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