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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

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

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

Effect of Efgartigimod IV on Immature Platelet Count Fraction in Primary Immune Thrombocytopenia

Filip Matthijssens, Yoshitaka Miyakawa, Kaichi Nishiwaki, Jaume Ayguasanosa, Lan Lan, Anna Hultberg, Peter Ulrichts

Blood (2024) 144 (Supplement 1): 5443.

https://doi.org/10.1182/blood-2024-206889

Published: 2024

Journal Articles

Post Hoc Analysis of a Phase 2/3 Study in Japanese Patients with iTTP Treated with Caplacizumab

Kazunori Imada, Yoshitaka Miyakawa, Satoshi Ichikawa, Hitoji Uchiyama, Yasunori Ueda, Yasuhiro Hashimoto, Masashi Nishimi, Sayaka Tahara, Masanori Matsumoto

Blood (2023) 142 (Supplement 1): 5428.

https://doi.org/10.1182/blood-2023-180796

Published: 2023

Journal Articles

Time to Achieve Platelet Count Response after Intravenous Efgartigimod in Adults with Primary Immune Thrombocytopenia: A Phase 3, Multicenter, Double-Blinded, Placebo-Controlled, Randomized Clinical Trial (ADVANCE IV)

Catherine M. Broome, Vickie McDonald, Yoshitaka Miyakawa, Monica Carpenedo, David J Kuter, Hanny Al-Samkari, James B. Bussel, Anna Hultberg, Filip Matthijssens, Jaume Ayguasanosa, Kristof De Beuf, Francesco Rodeghiero, Marc Michel, Adrian Newland

Blood (2023) 142 (Supplement 1): 689.

https://doi.org/10.1182/blood-2023-189125

Published: 2023

Journal Articles

Efficacy and Safety of Intravenous Efgartigimod in Adults with Primary Immune Thrombocytopenia: Results of a Phase 3, Multicenter, Double-Blinded, Placebo-Controlled, Randomized Clinical Trial (ADVANCE IV)

Catherine M. Broome, Vickie McDonald, Yoshitaka Miyakawa, Monica Carpenedo, David J. Kuter, Hanny Al-Samkari, James B. Bussel, Marie Godar, Jaume Ayguasanosa, Kristof De Beuf, Francesco Rodeghiero, Marc Michel, Adrian C. Newland

Blood (2022) 140 (Supplement 1): 6–8.

https://doi.org/10.1182/blood-2022-167838

Published: 2022

Journal Articles

The Efficacy and Safety of Caplacizumab in Japanese Patients with Immune-Mediated Thrombotic Thrombocytopenic Purpura (iTTP): An Open-Label, Phase 2/3 Study

Yoshitaka Miyakawa, Kazunori Imada, Satoshi Ichikawa, Hitoji Uchiyama, Yasunori Ueda, Akihito Yonezawa, Shigeki Fujitani, Hiroshi Handa, Tadashi Matsushita, Hidesaku Asakura, Kenji Nishio, Kodai Suzuki, Yasuhiro Hashimoto, Shinobu Ohshima, Sayaka Tahara, Tomoyuki Tanaka, Masanori Matsumoto

Blood (2021) 138 (Supplement 1): 1009.

https://doi.org/10.1182/blood-2021-148427

Published: 2021

Journal Articles

Two-Year Efficacy and Safety of Ravulizumab in Adults and Children with Atypical Hemolytic Uremic Syndrome (aHUS): Analysis of Two Phase 3 Studies

Bradley P. Dixon, Alvaro Domingo Madris-Aris, Brigitte Adams, David Kavanagh, Hee Gyung Kang, Jimmy Wang, Katherine Garlo, Kazuki Tanaka, Laurence Greenbaum, Masayo Ogawa, Nils Heyne, Seong Heon Kim, Spero Cataland, Sung-Soo Yoon, Yoshitaka Miyakawa, Yosu Luque, Melissa Muff-Luett

Blood (2021) 138 (Supplement 1): 769.

https://doi.org/10.1182/blood-2021-145040

Published: 2021

Journal Articles

Pharmacokinetic / Pharmacodynamic (PK/PD) Simulations Guide Selection of the Dose for Administration of Efgartigimod Subcutaneously in a Phase 3 Clinical Trial in Patients with Primary Immune Thrombocytopenia

Waleed Ghanima, Vickie McDonald, Shivi Jain, Monica Carpenedo, Esther N. Oliva, Anna Hultberg, Domenica Gandini, Erik Hofman, Tonke Van Bragt, Wim Parys, Benjamin van Hoorick, Yoshitaka Miyakawa, Catherine M. Broome

Blood (2021) 138 (Supplement 1): 3165.

https://doi.org/10.1182/blood-2021-153261

Published: 2021

Journal Articles

Inhibition of Complement C1s with Sutimlimab in Patients with Cold Agglutinin Disease (CAD): Interim Results of the Phase 3 Cardinal Study Long-Term Follow-up

Alexander Röth, MD, Wilma Barcellini, Shirley D'Sa, Yoshitaka Miyakawa, Catherine M Broome, Marc Michel, David J. Kuter, MD, Bernd Jilma, Tor Henrik Anderson Tvedt, Ilene C. Weitz, Parija Patel, Xiaoyu Jiang, Caroline Reuter, Jun Su, Frank Shafer, Michelle Lee, William Hobbs, Sigbjørn Berentsen

Blood (2020) 136 (Supplement 1): 24–25.

https://doi.org/10.1182/blood-2020-138909

Published: 2020

Journal Articles

Efficacy and Safety of Intravenous Efgartigimod 10 Mg/Kg in Adult Patients with Primary Immune Thrombocytopenia: Advance, a Phase 3 Clinical Trial in Progress

Adrian C. Newland, Howard A. Liebman, MD, Vickie McDonald, MD, Marc Michel, Yoshitaka Miyakawa, Wim Parys, MD, Hans De Haard, Peter Ulrichts, Marie Godar, Kristof De Beuf, Jaume Ayguasanosa, Catherine M Broome, David J. Kuter, MD

Blood (2020) 136 (Supplement 1): 6–7.

https://doi.org/10.1182/blood-2020-141161

Published: 2020

Journal Articles

Inhibition of Complement C1s with Sutimlimab in Patients with Cold Agglutinin Disease (CAD): Results from the Phase 3 Cardinal Study

Alexander Röth, Wilma Barcellini, Shirley D'Sa, Yoshitaka Miyakawa, Catherine M Broome, Marc Michel, David J. Kuter, Bernd Jilma, Tor Henrik Anderson Tvedt, Stella Lin, Xiaoyu Jiang, Caroline Reuter, William Hobbs, Sigbjørn Berentsen

Blood (2019) 134 (Supplement_2): LBA-2.

https://doi.org/10.1182/blood-2019-132490

Published: 2019

Journal Articles

Clinical Practice Pattern of Acquired Thrombotic Thrombocytopenic Purpura in Japan: A Nationwide Inpatient Database Analysis

Yuji Yamada, MD, Hiroyuki Ohbe, MD MPH, Hideo Yasunaga, MD PhD, Yoshitaka Miyakawa, MD PhD

Blood (2019) 134 (Supplement_1): 2374.

https://doi.org/10.1182/blood-2019-125170

Published: 2019

Journal Articles

Investigator-Initiated Phase 2 Trial of Rituximab in Adult Japanese Patients with Treatment-Resistant Acquired Thrombotic Thrombocytopenic Purpura

Yoshitaka Miyakawa, Kazunori Imada, Tatsuo Ichinohe, MDPhD, Yusuke Yamane, MD, Kenji Nishio, Takayuki Abe, Yoshihiro Fujimura, MDPhD, Yasunori Ueda, MD, PhD, Masanori Matsumoto, MD PhD, Shinichiro Okamoto

Blood (2015) 126 (23): 4645.

https://doi.org/10.1182/blood.V126.23.4645.4645

Published: 2015

Journal Articles

Rituximab As Second-Line Treatment For Chronic Immune Thrombocytopenia: Investigator-Initiated Clinical Trial In Japan

Yoshitaka Miyakawa, MD, PhD, Shinya Katsutani, MD, PhD, Takahiro Yano, MD, PhD, Shosaku Nomura, MD, PhD, Kaichi Nishiwaki, MD, Yoshiaki Tomiyama, MD, PhD, Masaaki Higashihara, MD, PhD, Yukari Shirasugi, MD, PhD, Masakatsu Nishikawa, MD, PhD, Katsutoshi Ozaki, MD, PhD, Kayoko Kikuchi, Takayuki Abe, PhD, Yuji Sato, MD, PhD, Yuzuru Kanakura, Kingo Fujimura, MD, PhD, Yasuo Ikeda, MD, PhD, Shinichiro Okamoto, MD, PhD

Blood (2013) 122 (21): 3554.

https://doi.org/10.1182/blood.V122.21.3554.3554

Published: 2013

Journal Articles

A Novel Factor XII Mutation, FXII Tokyo (R84P), Causing Factor XII Deficiency In a Patient with Hereditary Spastic Paraplegia

Eri Matsuki, MD, Yoshitaka Miyakawa, MD, PhD, Shinichiro Okamoto, MD, PhD

Blood (2010) 116 (21): 1404.

https://doi.org/10.1182/blood.V116.21.1404.1404

Published: 2010

Journal Articles

Establishment of a Novel Cell Line of Plasmablastic Lymphoma with Loss of p16 Tumor Suppressor Protein and Overexpression of MDR

Eri Matsuki, MD, Yoshitaka Miyakawa, MD, PhD, Shuichi Asakawa, Yuiko Tsukada, MD, Kenji Yokoyama, MD, PhD, Jun Kudoh, Shinichiro Okamoto, MD, PhD

Blood (2010) 116 (21): 4451.

https://doi.org/10.1182/blood.V116.21.4451.4451

Published: 2010

Journal Articles

Effectiveness of a Novel Humanized VB22B Minibody against Human TPO Receptor Compared with Recombinant TPO and Eltrombopag Using Primary Human CD34+ Cells and Platelets.

Eri Matsuki, MD, Akiko Yamane, MD, PhD, Shinichiro Okamoto, MD, PhD, Yoshitaka Miyakawa, MD, PhD

Blood (2009) 114 (22): 4559.

https://doi.org/10.1182/blood.V114.22.4559.4559

Published: 2009

Journal Articles

Six Month Treatment of Low Dose Eltrombopag Is Efficacious in Japanese Patients with Refractory Chronic Immune Thrombocytopenic Purpura (ITP).

Yoshiaki Tomiyama, MD, Yoshitaka Miyakawa, MD, PhD, Shinichiro Okamoto, MD, PhD, Shinya Katsutani, MD, Akiro Kimura, MD, Yasushi Okoshi, MD, PhD, Haruhiko Ninomiya, MD, PhD, Hiroshi Kosugi, MD, PhD, Shosaku Nomura, MD, Katsutoshi Ozaki, MD, Yasuo Ikeda, MD, Nobuyuki Koh, Koichi Katsura, MD, PhD, Yuzuru Kanakura, MD, PhD

Blood (2009) 114 (22): 1324.

https://doi.org/10.1182/blood.V114.22.1324.1324

Published: 2009

NARROW

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)

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

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)

Effect of Efgartigimod IV on Immature Platelet Count Fraction in Primary Immune Thrombocytopenia

Post Hoc Analysis of a Phase 2/3 Study in Japanese Patients with iTTP Treated with Caplacizumab

Time to Achieve Platelet Count Response after Intravenous Efgartigimod in Adults with Primary Immune Thrombocytopenia: A Phase 3, Multicenter, Double-Blinded, Placebo-Controlled, Randomized Clinical Trial (ADVANCE IV)

Efficacy and Safety of Intravenous Efgartigimod in Adults with Primary Immune Thrombocytopenia: Results of a Phase 3, Multicenter, Double-Blinded, Placebo-Controlled, Randomized Clinical Trial (ADVANCE IV)

The Efficacy and Safety of Caplacizumab in Japanese Patients with Immune-Mediated Thrombotic Thrombocytopenic Purpura (iTTP): An Open-Label, Phase 2/3 Study

Two-Year Efficacy and Safety of Ravulizumab in Adults and Children with Atypical Hemolytic Uremic Syndrome (aHUS): Analysis of Two Phase 3 Studies

Pharmacokinetic / Pharmacodynamic (PK/PD) Simulations Guide Selection of the Dose for Administration of Efgartigimod Subcutaneously in a Phase 3 Clinical Trial in Patients with Primary Immune Thrombocytopenia

Inhibition of Complement C1s with Sutimlimab in Patients with Cold Agglutinin Disease (CAD): Interim Results of the Phase 3 Cardinal Study Long-Term Follow-up

Efficacy and Safety of Intravenous Efgartigimod 10 Mg/Kg in Adult Patients with Primary Immune Thrombocytopenia: Advance, a Phase 3 Clinical Trial in Progress

Inhibition of Complement C1s with Sutimlimab in Patients with Cold Agglutinin Disease (CAD): Results from the Phase 3 Cardinal Study

Clinical Practice Pattern of Acquired Thrombotic Thrombocytopenic Purpura in Japan: A Nationwide Inpatient Database Analysis

Investigator-Initiated Phase 2 Trial of Rituximab in Adult Japanese Patients with Treatment-Resistant Acquired Thrombotic Thrombocytopenic Purpura

Rituximab As Second-Line Treatment For Chronic Immune Thrombocytopenia: Investigator-Initiated Clinical Trial In Japan

A Novel Factor XII Mutation, FXII Tokyo (R84P), Causing Factor XII Deficiency In a Patient with Hereditary Spastic Paraplegia

Establishment of a Novel Cell Line of Plasmablastic Lymphoma with Loss of p16 Tumor Suppressor Protein and Overexpression of MDR

Effectiveness of a Novel Humanized VB22B Minibody against Human TPO Receptor Compared with Recombinant TPO and Eltrombopag Using Primary Human CD34+ Cells and Platelets.

Six Month Treatment of Low Dose Eltrombopag Is Efficacious in Japanese Patients with Refractory Chronic Immune Thrombocytopenic Purpura (ITP).

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American Society of Hematology

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American Society of Hematology

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