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Journal Articles

ENERGIZE-T: A Global, Phase 3, Double-Blind, Randomized, Placebo-Controlled Study of Mitapivat in Adults with Transfusion-Dependent Alpha- or Beta-Thalassemia

Blood (2024) 144 (Supplement 1): 409.

https://doi.org/10.1182/blood-2024-200867

Published: 2024

Journal Articles

A Phase 2B, Open-Label Multicenter Study of Tebapivat (AG-946), a Potent Pyruvate Kinase Activator, in Patients with Anemia Due to Lower-Risk Myelodysplastic Syndromes

Amer M. Zeidan, Mikkael A. Sekeres, Hanny Al-Samkari, Hetty E. Carraway, Amy E. DeZern, Moshe Mittelman, Meredith Little, Vanessa Beynon, Xiaoshu Dai, Sal Sommakia, Jenny M. Despotovic, Parija Patel, Melissa Dibacco, Bruno Fattizzo, Eytan M. Stein, David Sallman, Austin G. Kulasekararaj, Uwe Platzbecker, Pierre Fenaux

Blood (2024) 144 (Supplement 1): 6708.

https://doi.org/10.1182/blood-2024-203073

Published: 2024

Journal Articles

Characterizing the Healthcare Resource Utilization and Costs of Hereditary Hemorrhagic Telangiectasia (HHT)

Hanny Al-Samkari, Tracy Mayne, Sandra Texeira, Marianne S. Clancy, Eric Duhaime

Blood (2024) 144 (Supplement 1): 278.

https://doi.org/10.1182/blood-2024-198721

Published: 2024

Journal Articles

Intravenous Efgartigimod for the Treatment of Primary Immune Thrombocytopenia in Adults: A Planned, Phase 3, Randomized, Double-Blinded, Placebo-Controlled, Parallel-Arm Study with Open-Label Period (ADVANCE NEXT)

Hanny Al-Samkari, Vickie McDonald, Monica Carpenedo, Francesco Zaja, Nichola Cooper, Spero Cataland, Michael W Drazer, Yoshitaka Miyakawa, Marc Michel, Jaume Ayguasanosa, Janki Brahmbhatt, Lan Lan, Filip Matthijssens, Catherine M. Broome, Waleed Ghanima

Blood (2024) 144 (Supplement 1): 2570.1.

https://doi.org/10.1182/blood-2024-194816

Published: 2024

Journal Articles

Iptacopan Monotherapy in Patients with Cold Agglutinin Disease: Phase II Study Results

Alexander Röth, Wilma Barcellini, Christine Ademokun, Shripad Chitnis, Sofiya Matviykiv, Alessandra Vitaliti, Chi Chen, Vasiliki Katsanou, Raghav Chawla, Hanny Al-Samkari

Blood (2024) 144 (Supplement 1): 2457.

https://doi.org/10.1182/blood-2024-199637

Published: 2024

Journal Articles

Long-Term Safety and Effectiveness of Pomalidomide for Bleeding in Hereditary Hemorrhagic Telangiectasia

Ellen Zhang, Pamela G Hodges, Josanna Rodriguez-Lopez, Hanny Al-Samkari

Blood (2024) 144 (Supplement 1): 558.

https://doi.org/10.1182/blood-2024-202284

Published: 2024

Journal Articles

A Randomized, Placebo-Controlled, Multicenter Proof-of-Concept (POC) Study to Assess the Safety and Efficacy of the Novel Allosteric AKT Inhibitor, VAD044, in Adults with Hereditary Hemorrhagic Telangiectasia (HHT)

Hanny Al-Samkari, Josefien Hessels, Antoni Riera-Mestre, Sophie Dupuis-Girod, Thibaut Van Zele, Vincente Gómez del Olmo, Pierre Saint-Mezard, Hedvika Lazar, Damien Picard, Debra Barker, Elisabetta Buscarini, Hans-Jurgen Mager

Blood (2024) 144 (Supplement 1): 553.

https://doi.org/10.1182/blood-2024-206244

Published: 2024

Journal Articles

Results from a Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Tebapivat (AG-946) in Patients with Sickle Cell Disease

Julia Z Xu, Enrico M Novelli, Jean-Antoine Ribeil, Andreas Glenthøj, Srila Gopal, Hanny Al-Samkari, Modupe Idowu, Jenny Despotovic, Spurthi Patil, Xiaoshu Dai, Abdullah Al Masud, Michael Callaghan, Fuad El Rassi

Blood (2024) 144 (Supplement 1): 2496.

https://doi.org/10.1182/blood-2024-203509

Published: 2024

Journal Articles

Direct Glycoprotein-Specific Platelet Autoantibodies Detected in a Majority of Patients with Chemotherapy-Induced Thrombocytopenia

Andrew B Song, Brian Curtis, Hanny Al-Samkari

Blood (2024) 144 (Supplement 1): 1180.

https://doi.org/10.1182/blood-2024-194752

Published: 2024

Journal Articles

Risk Factors for Bleeding after Recent Medical Hospitalization: The Medical Inpatient Thrombosis and Hemostasis Study (MITH)

Mansour Gergi, Katherine Wilkinson, Andrew Sparks, Nicholas S Roetker, Hanny Al-Samkari, Nicholas L Smith, Timothy B Plante, Mary Cushman, Allen B Repp, Chris E. Holmes, Karlyn A. Martin, Neil Zakai

Blood (2024) 144 (Supplement 1): 2252.

https://doi.org/10.1182/blood-2024-201581

Published: 2024

Journal Articles

How I treat bleeding in hereditary hemorrhagic telangiectasia

Hanny Al-Samkari

Blood (2024) 144 (9): 940–954.

https://doi.org/10.1182/blood.2023021765

Published: 2024

Journal Articles

PATH-HHT, a Double-Blind, Randomized, Placebo-Controlled Trial in Hereditary Hemorrhagic Telangiectasia Demonstrates That Pomalidomide Reduces Epistaxis and Improves Quality of Life

Blood (2023) 142 (Supplement 2): LBA-3.

https://doi.org/10.1182/blood-2023-191983

Published: 2023

Journal Articles

Initial Results in a Phase 1b Trial of PB-04 in Sickle Cell Disease Demonstrate Fetal Hemoglobin Induction, Additive Activity with Hydroxyurea, and Improved Red Blood Cell Sickling Parameters

Kevin H.M. Kuo, Sylvia Titi Singer, Gershwin Theophilus Blyden, Lanetta Bronté-Hall, Aidan Faller, Abdullah Kutlar, Frans A. Kuypers, Aliya U. Zaidi, Patrick C. Hines, Niren Patel, Sandra Larkin, Hanny Al-Samkari, Sujit Sheth, Seyed Mehdi Nouraie, Susan Perrine

Blood (2023) 142 (Supplement 1): 1148.

https://doi.org/10.1182/blood-2023-184627

Published: 2023

Journal Articles

Improvements in Markers of Hemolysis and Liver Iron Concentration in Mitapivat-Treated Adult Patients with a Delayed Hemoglobin Response

Eduard J. Van Beers, Bryan McGee, Rengyi Xu, Melissa Dibacco, Kevin H.M. Kuo, Hanny Al-Samkari

Blood (2023) 142 (Supplement 1): 5226.

https://doi.org/10.1182/blood-2023-186533

Published: 2023

Journal Articles

Hereditary Hemorrhagic Telangiectasia May be the Most Clinically Significant and Morbid Inherited Bleeding Disorder of Women

Ellen Zhang, Zain Virk, Josanna Rodriguez-Lopez, Hanny Al-Samkari

Blood (2023) 142 (Supplement 1): 28.

https://doi.org/10.1182/blood-2023-187772

Published: 2023

Journal Articles

A Phase III Clinical Trial Program Investigating the Efficacy and Safety of Ianalumab in Patients with Primary Immune Thrombocytopenia (VAYHIT1 and VAYHIT2) and Warm Autoimmune Hemolytic Anemia (VAYHIA)

Hanny Al-Samkari, Wilma Barcellini, Nichola Cooper, Waleed Ghanima, Marc Michel, Raymond SM Wong, Francesco Zaja, Fengkui Zhang, Patrick Urban, Alex Allepuz, Justyna Fronczek-Sokol, Jens Haenig, Pascal Edrich, Adam Cuker

Blood (2023) 142 (Supplement 1): 5456.

https://doi.org/10.1182/blood-2023-180647

Published: 2023

Journal Articles

Impact of Degree and Duration of Iron Overload on Incidence of Diabetes Mellitus, Cardiac Disease, and Death in Congenital Hemolytic Anemias

Aaron Cheng, Hanny Al-Samkari

Blood (2023) 142 (Supplement 1): 1094.

https://doi.org/10.1182/blood-2023-179640

Published: 2023

Journal Articles

Avatrombopag for Adults with Early Versus Chronic Immune Thrombocytopenia

Zain Virk, Rebecca Karp Leaf, David J Kuter, Katayoon Goodarzi, Nathan T. Connell, Jean M. Connors, Hanny Al-Samkari

Blood (2023) 142 (Supplement 1): 392.

https://doi.org/10.1182/blood-2023-179601

Published: 2023

Journal Articles

Ianalumab, a Novel Anti-B-Cell Activating Factor (BAFF) Receptor (BAFF-R) Monoclonal Antibody (mAb) in Development for Immune Thrombocytopenia (ITP) and Warm Autoimmune Hemolytic Anemia (wAIHA), Has Demonstrated a Favorable Safety Profile in Sjögren's Syndrome (SjS), Systemic Lupus Erythematosus (SLE) and Chronic Lymphocytic Leukemia (CLL)

Adam Cuker, Hanny Al-Samkari, Wilma Barcellini, Nichola Cooper, Waleed Ghanima, Marc Michel, Raymond SM Wong, Francesco Zaja, Fengkui Zhang, Patrick Urban, Roberto Abi Rached, David J Kuter

Blood (2023) 142 (Supplement 1): 5427.

https://doi.org/10.1182/blood-2023-180055

Published: 2023

Journal Articles

Mitapivat Treatment Reduces Levels of Interference in Work/School Activity for Adult Patients with Pyruvate Kinase Deficiency

Jennifer A Rothman, Parija Patel, Jing Zhao, Susan Morris, Junlong Li, Hanny Al-Samkari

Blood (2023) 142 (Supplement 1): 2365.

https://doi.org/10.1182/blood-2023-180697

Published: 2023

NARROW

ENERGIZE-T: A Global, Phase 3, Double-Blind, Randomized, Placebo-Controlled Study of Mitapivat in Adults with Transfusion-Dependent Alpha- or Beta-Thalassemia

A Phase 2B, Open-Label Multicenter Study of Tebapivat (AG-946), a Potent Pyruvate Kinase Activator, in Patients with Anemia Due to Lower-Risk Myelodysplastic Syndromes

Characterizing the Healthcare Resource Utilization and Costs of Hereditary Hemorrhagic Telangiectasia (HHT)

Intravenous Efgartigimod for the Treatment of Primary Immune Thrombocytopenia in Adults: A Planned, Phase 3, Randomized, Double-Blinded, Placebo-Controlled, Parallel-Arm Study with Open-Label Period (ADVANCE NEXT)

Iptacopan Monotherapy in Patients with Cold Agglutinin Disease: Phase II Study Results

Long-Term Safety and Effectiveness of Pomalidomide for Bleeding in Hereditary Hemorrhagic Telangiectasia

A Randomized, Placebo-Controlled, Multicenter Proof-of-Concept (POC) Study to Assess the Safety and Efficacy of the Novel Allosteric AKT Inhibitor, VAD044, in Adults with Hereditary Hemorrhagic Telangiectasia (HHT)

Results from a Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Tebapivat (AG-946) in Patients with Sickle Cell Disease

Direct Glycoprotein-Specific Platelet Autoantibodies Detected in a Majority of Patients with Chemotherapy-Induced Thrombocytopenia

Risk Factors for Bleeding after Recent Medical Hospitalization: The Medical Inpatient Thrombosis and Hemostasis Study (MITH)

How I treat bleeding in hereditary hemorrhagic telangiectasia

PATH-HHT, a Double-Blind, Randomized, Placebo-Controlled Trial in Hereditary Hemorrhagic Telangiectasia Demonstrates That Pomalidomide Reduces Epistaxis and Improves Quality of Life

Initial Results in a Phase 1b Trial of PB-04 in Sickle Cell Disease Demonstrate Fetal Hemoglobin Induction, Additive Activity with Hydroxyurea, and Improved Red Blood Cell Sickling Parameters

Improvements in Markers of Hemolysis and Liver Iron Concentration in Mitapivat-Treated Adult Patients with a Delayed Hemoglobin Response

Hereditary Hemorrhagic Telangiectasia May be the Most Clinically Significant and Morbid Inherited Bleeding Disorder of Women

A Phase III Clinical Trial Program Investigating the Efficacy and Safety of Ianalumab in Patients with Primary Immune Thrombocytopenia (VAYHIT1 and VAYHIT2) and Warm Autoimmune Hemolytic Anemia (VAYHIA)

Impact of Degree and Duration of Iron Overload on Incidence of Diabetes Mellitus, Cardiac Disease, and Death in Congenital Hemolytic Anemias

Avatrombopag for Adults with Early Versus Chronic Immune Thrombocytopenia

Mitapivat Treatment Reduces Levels of Interference in Work/School Activity for Adult Patients with Pyruvate Kinase Deficiency

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American Society of Hematology

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American Society of Hematology

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