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Journal Articles

Primary results from VAYHIT2, a randomized, double-blind, phase 3 trial of ianalumab plus eltrombopag versus placebo plus eltrombopag in patients with primary immune thrombocytopenia (ITP) who failed first-line corticosteroid treatment

Blood (2025) 146 (Supplement 2): LBA-2.

https://doi.org/10.1182/blood-2025-LBA-2

Published: 2025

Journal Articles

Reduction in corticosteroid use with rilzabrutinib and sustained response in adults with persistent/chronic immune thrombocytopenia in the long-term extension period of the Phase 3 LUNA3 study

David Kuter, Lei Zhang, Waleed Ghanima, Yoshitaka Miyakawa, Yu Hu, David Gomez-Almaguer, Matias Cordoba, Marek Wardecki, Larissa Mege, Meredith Foster, Ahmed Daak, Howard Liebman

Blood (2025) 146 (Supplement 1): 1260.

https://doi.org/10.1182/blood-2025-1260

Published: 2025

Journal Articles

Impact of treatment with fostamatinib on coagulation profile of patients with immune thrombocytopenia

Ana Mendoza Martínez, Elena Monzon Manzano, Paula Acuña, Elena G Arias-Salgado, Jose Manuel Martín de Bustamante Gonzalez-Iglesias, Maria Isabel Rivas Pollmar, Eduardo Garcia Perez, Monica Martin Salces, Mar Gutierrez Alvariño, Víctor Jiménez-Yuste, Waleed Ghanima, Lamya Garabet, Nora Butta, María Teresa Alvarez-Román

Blood (2025) 146 (Supplement 1): 1242.

https://doi.org/10.1182/blood-2025-1242

Published: 2025

Journal Articles

Improved health-related quality of life (HRQoL) and bleeding scores with oral bruton tyrosine kinase (BTK) inhibitor rilzabrutinib in the open-label (OL) period of the multicenter Phase 3 LUNA3 study in adults with immune thrombocytopenia (ITP)

Nicola Cooper, Waleed Ghanima, Francesco Zaja, Lei Zhang, Yoshitaka Miyakawa, Marie Luise Hütter-Krönke, Matias Cordoba, Mattea Orsini, Imene Gouia, Ahmed Daak, Howard Liebman

Blood (2025) 146 (Supplement 1): 1254.

https://doi.org/10.1182/blood-2025-1254

Published: 2025

Journal Articles

Trial in progress: Study design of a randomized, phase 3 trial evaluating the efficacy and safety of mezagitamab (TAK-079) compared with placebo in adults with chronic primary immune thrombocytopenia

David Kuter, Chen Yunfei, Nicola Cooper, Waleed Ghanima, Hisashi Kato, Yoshiaki Tomiyama, Raymond Wong, Renchi Yang, Cheryl Li, Emily Skelton, Scarlett Wang, Donald Yee, Parth Patwari

Blood (2025) 146 (Supplement 1): 3049.

https://doi.org/10.1182/blood-2025-3049

Published: 2025

Journal Articles

The holistic trial: A comprehensive evaluation of two second-line therapeutic approaches for immune thrombocytopenia (ITP) – a pragmatic randomized controlled trial

Eirik Tjønnfjord, Inge Christoffer Olsen, Lamya Garabet, Hoa Tran, Doaa El Demerdash, Salam AlKindy, Geir Tjønnfjord, Waleed Ghanima

Blood (2025) 146 (Supplement 1): 8226.

https://doi.org/10.1182/blood-2025-8226

Published: 2025

Journal Articles

Early multi-immune modulation with rilzabrutinib in patients with primary ITP after first-line treatment failure: A phase 3b study (LUNA 4)

David Kuter, Waleed Ghanima, Nicola Cooper, Marie Luise Hütter-Krönke, Guillaume Moulis, Francesco Zaja, Maria Cristina Pascual Izquierdo, Howard Liebman, Sandhya Panch, Hisashi Kato, Matias Cordoba, Ahmed Daak, Maria Belen Rodriguez

Blood (2025) 146 (Supplement 1): 4827.

https://doi.org/10.1182/blood-2025-4827

Published: 2025

Journal Articles

The safety and efficacy of pirtobrutinib in adults with immune thrombocytopenia: A phase 1/2 dose-finding study in progress

Catherine Broome, Tomas Jose Gonzalez-Lopez, Ananya Guntur, Chunxiao Wang, Sasha Amdur, James Pauff, Waleed Ghanima

Blood (2025) 146 (Supplement 1): 8231.

https://doi.org/10.1182/blood-2025-8231

Published: 2025

Journal Articles

Differential expression of coagulation and inflammation genes in ITP patients before and after treatment with thrombopoietin receptor agonists

Lamya Garabet, Mark Burton, Mads Thomassen, Anna Eriksson, Hans Hasselbalch, Vibe Skov, Christine Jonassen, James Bussel, Waleed Ghanima

Blood (2025) 146 (Supplement 1): 3018.

https://doi.org/10.1182/blood-2025-3018

Published: 2025

Journal Articles

The prolong trial: A two Phase randomized placebo-controlled trial to optimize rituximab response with dexamethasone and maintenance therapy with low dose rituximab in immune thrombocytopenia (ITP) patients

Waleed Ghanima, Doaa El Demerdash, Henrik Frederiksen, James Bussel, Galina Tsykunova, Marc Michel, Hoa Tran, Carina Nassar, Bernadette Darne, Eirik Tjønnfjord, Tor Henrik Tvedt, Fehmi M'Sadek, Mervat Mattar, Abderraheem Khelif, Pål Holme

Blood (2025) 146 (Supplement 1): 736.

https://doi.org/10.1182/blood-2025-736

Published: 2025

Journal Articles

Safety and efficacy of rilzabrutinib vs placebo in adults with immune thrombocytopenia: the phase 3 LUNA3 study

Blood (2025) 145 (24): 2914–2926.

https://doi.org/10.1182/blood.2024027336

Published: 2025

Includes: Supplemental data

Journal Articles

Characterizing Diagnostic Investigations for Suspected Pulmonary Embolism in Postpartum Individuals

Sophie Greutert, Gurjeet Bhangu, Helia Robert-Ebadi, Jeffrey Kline, Marc Righini, Andrea Penaloza, Arnaud Perrier, Waleed Ghanima, Ranjeeta Mallick, Toshihiko Takada, Nick Van Es, Frederikus A Klok, Geert-Jan Geersing, Grégoire Le Gal, Leslie Skeith

Blood (2024) 144 (Supplement 1): 1249.

https://doi.org/10.1182/blood-2024-203216

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

Efficacy and Safety of Oral Bruton Tyrosine Kinase Inhibitor (BTKi) Rilzabrutinib in Adults with Previously Treated Immune Thrombocytopenia (ITP): A Phase 3, Placebo-Controlled, Parallel-Group, Multicenter Study (LUNA 3)

Blood (2024) 144 (Supplement 1): 5.

https://doi.org/10.1182/blood-2024-199718

Published: 2024

Journal Articles

Patients with Immune Thrombocytopenia Have Higher Proportion of Ultra Large VWF Multimers and Lower ADAMTS13 Activity Than Healthy Controls

Lamya Garabet, Waleed Ghanima, Åse-Berit Mathisen, Stine Jeanette Lae, Marie Skogstad Le, James B. Bussel, Andreas Hillarp, Carola Henriksson

Blood (2024) 144 (Supplement 1): 2556.

https://doi.org/10.1182/blood-2024-202331

Published: 2024

Journal Articles

Improved Health-Related quality of Life (HRQoL) with Oral Bruton Tyrosine Kinase Inhibitor (BTKi) Rilzabrutinib Vs Placebo in Adults with Previously Treated Immune Thrombocytopenia (ITP): Phase 3 Luna 3 Multicenter Study

Waleed Ghanima, Howard A. Liebman, Yu Hu, Yoshitaka Miyakawa, Nichola Cooper, Güray Saydam, Marie Luise Hütter-Krönke, Sylvain Audia, Mengjie Yao, Ahmed Daak, Imene Gouia, Matias Cordoba, David J Kuter

Blood (2024) 144 (Supplement 1): 2552.

https://doi.org/10.1182/blood-2024-200279

Published: 2024

Journal Articles

The Lack of Tolerable Treatments Options That Can Induce Durable Responses without Fear of Relapse after Discontinuation Represents a Significant Unmet Need for Patients (Pts) with Immune Thrombocytopenia (ITP): Results from the ITP World Impact Survey (I-WISh) 2.0

James B. Bussel, Nichola Cooper, Waleed Ghanima, Drew Provan, Yoshiaki Tomiyama, Ming Hou, Donald Arnold, Cristina Santoro, Francesco Zaja, Barbara Lovrencic, Mervyn Morgan, Leron Lahav, Michal Winograd, Jennifer MacWhirter - DiRaimo, Tom Bailey, Moyra Aziz, Roberto Abi Rached, Caroline Kruse

Blood (2023) 142 (Supplement 1): 1212.

https://doi.org/10.1182/blood-2023-188527

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

Avatrombopag Plus Fostamatinib Combination Efficacy and Safety in Patients with Immune Thrombocytopenia

Maria Eva Mingot Castellano, Begoña Pedrote Amador, Riccardo Tomasello, Waleed Ghanima

Blood (2023) 142 (Supplement 1): 2596.

https://doi.org/10.1182/blood-2023-187518

Published: 2023

Journal Articles

Real-World Use, Effectiveness and Safety of Romiplostim, Eltrombopag and Avatrombopag in Immune Thrombocytopenia: Data from the Norwegian ITP Registry

Riccardo Tomasello, Lamya Garabet, Heidi Pettersen, Eirik Tjønnfjord, Galina Tsykunova, Torkild Pedersen, Hoa Tran, Mariasanta Napolitano, Waleed Ghanima

Blood (2023) 142 (Supplement 1): 3952.

https://doi.org/10.1182/blood-2023-182848

Published: 2023

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Primary results from VAYHIT2, a randomized, double-blind, phase 3 trial of ianalumab plus eltrombopag versus placebo plus eltrombopag in patients with primary immune thrombocytopenia (ITP) who failed first-line corticosteroid treatment

Reduction in corticosteroid use with rilzabrutinib and sustained response in adults with persistent/chronic immune thrombocytopenia in the long-term extension period of the Phase 3 LUNA3 study

Impact of treatment with fostamatinib on coagulation profile of patients with immune thrombocytopenia

Improved health-related quality of life (HRQoL) and bleeding scores with oral bruton tyrosine kinase (BTK) inhibitor rilzabrutinib in the open-label (OL) period of the multicenter Phase 3 LUNA3 study in adults with immune thrombocytopenia (ITP)

Trial in progress: Study design of a randomized, phase 3 trial evaluating the efficacy and safety of mezagitamab (TAK-079) compared with placebo in adults with chronic primary immune thrombocytopenia

The holistic trial: A comprehensive evaluation of two second-line therapeutic approaches for immune thrombocytopenia (ITP) – a pragmatic randomized controlled trial

Early multi-immune modulation with rilzabrutinib in patients with primary ITP after first-line treatment failure: A phase 3b study (LUNA 4)

The safety and efficacy of pirtobrutinib in adults with immune thrombocytopenia: A phase 1/2 dose-finding study in progress

Differential expression of coagulation and inflammation genes in ITP patients before and after treatment with thrombopoietin receptor agonists

The prolong trial: A two Phase randomized placebo-controlled trial to optimize rituximab response with dexamethasone and maintenance therapy with low dose rituximab in immune thrombocytopenia (ITP) patients

Safety and efficacy of rilzabrutinib vs placebo in adults with immune thrombocytopenia: the phase 3 LUNA3 study

Characterizing Diagnostic Investigations for Suspected Pulmonary Embolism in Postpartum Individuals

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)

Efficacy and Safety of Oral Bruton Tyrosine Kinase Inhibitor (BTKi) Rilzabrutinib in Adults with Previously Treated Immune Thrombocytopenia (ITP): A Phase 3, Placebo-Controlled, Parallel-Group, Multicenter Study (LUNA 3)

Patients with Immune Thrombocytopenia Have Higher Proportion of Ultra Large VWF Multimers and Lower ADAMTS13 Activity Than Healthy Controls

Improved Health-Related quality of Life (HRQoL) with Oral Bruton Tyrosine Kinase Inhibitor (BTKi) Rilzabrutinib Vs Placebo in Adults with Previously Treated Immune Thrombocytopenia (ITP): Phase 3 Luna 3 Multicenter Study

The Lack of Tolerable Treatments Options That Can Induce Durable Responses without Fear of Relapse after Discontinuation Represents a Significant Unmet Need for Patients (Pts) with Immune Thrombocytopenia (ITP): Results from the ITP World Impact Survey (I-WISh) 2.0

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)

Avatrombopag Plus Fostamatinib Combination Efficacy and Safety in Patients with Immune Thrombocytopenia

Real-World Use, Effectiveness and Safety of Romiplostim, Eltrombopag and Avatrombopag in Immune Thrombocytopenia: Data from the Norwegian ITP Registry

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