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Journal Articles

Patient Preferences and Treatment Satisfaction in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Treated with Crovalimab and Approved C5 Inhibitors in the Phase III Randomized COMMODORE 1 and COMMODORE 2 Trials

Austin Kulasekararaj, Fernando Ataulfo Gonzalez Fernandez, Phillip Scheinberg, Nicole Straetmans, Yasutaka Ueda, Brittany Gentile, Jennifer Stefani, Marianne Uguen, Alexander Roeth

Blood (2023) 142 (Supplement 1): 5628.

https://doi.org/10.1182/blood-2023-178610

Published: 2023

Journal Articles

Insights from Trajectories of PIGA -Mutated Clones in Paroxysmal Nocturnal Hemoglobinuria

Hiroyuki Takamori, Yasutaka Ueda, Yoshikazu Matsuoka, Tatsuya Fujioka, Hideki Makishima, Yoshiko Murakami, Taroh Kinoshita, Satoru Miyano, Yuzuru Kanakura, Jun-Ichi Nishimura, Naoki Hosen, Seishi Ogawa, Yasuhito Nannya

Blood (2023) 142 (Supplement 1): 2724.

https://doi.org/10.1182/blood-2023-188992

Published: 2023

Journal Articles

Combined Safety Data for Sutimlimab in Cold Agglutinin Disease: A Post-Hoc Analysis of the Phase 3 Cardinal and Cadenza Studies

Catherine M. Broome, Wilma Barcellini, Yasutaka Ueda, Umer Khan, Reena Patel, Marek Wardęcki, Katarina Kralova, Shruti Srivastava, Alexander Roeth

Blood (2023) 142 (Supplement 1): 3833.

https://doi.org/10.1182/blood-2023-188686

Published: 2023

Journal Articles

Patient-Reported Improvements in Fatigue and Health-Related Quality of Life in the Phase 3 Studies Apply-PNH and Appoint-PNH Evaluating the Use of Iptacopan in C5 Inhibitor-Treated and Treatment-Naïve Patients with Paroxysmal Nocturnal Hemoglobinuria

Antonio M Risitano, Carlos M de Castro, Bing Han, Austin Kulasekararaj, Jaroslaw P. Maciejewski, Phillip Scheinberg, Yasutaka Ueda, Susan Vallow, Georgina Bermann, Marion Dahlke, Rakesh Kumar, Regis Peffault de Latour

Blood (2023) 142 (Supplement 1): 487.

https://doi.org/10.1182/blood-2023-179035

Published: 2023

Journal Articles

Factor B Inhibition with Oral Iptacopan Monotherapy Demonstrates Sustained Long-Term Efficacy and Safety in Anti-C5-Treated Patients (pts) with Paroxysmal Nocturnal Hemoglobinuria (PNH) and Persistent Anemia: Final 48-Week Results from the Multicenter, Phase III APPLY-PNH Trial

Blood (2023) 142 (Supplement 1): 571.

https://doi.org/10.1182/blood-2023-180780

Published: 2023

Journal Articles

Cold Agglutinin Disease (CAD) Real-World Evidence (CADENCE) Registry: Update to the Study Design of the First International, Prospective CAD Registry

Alexander Roeth, Wilma Barcellini, Ulrich Jaeger, Yasutaka Ueda, Marc Michel, Quentin A. Hill, Shruti Srivastava, Ronnie Yoo, Catherine M. Broome

Blood (2023) 142 (Supplement 1): 7341.

https://doi.org/10.1182/blood-2023-180678

Published: 2023

Journal Articles

Efficacy of Sutimlimab in Improving FACIT-Fatigue Scores in Patients with Cold Agglutinin Disease: A Post-Hoc Analysis of the Phase 3 Cadenza (Part A) Study

Alexander Roeth, Ilene Weitz, Yasutaka Ueda, Sylvie Bozzi, Reena Patel, Alia Karaouni, Ronnie Yoo, David Cella, Jennifer Wang

Blood (2023) 142 (Supplement 1): 5558.

https://doi.org/10.1182/blood-2023-181563

Published: 2023

Journal Articles

Categorization of Hematological Responses to Oral Iptacopan Monotherapy in Anti-C5-Treated Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) and Persistent Anemia in the Phase III APPLY-PNH Trial and Complement Inhibitor-Naïve Patients in the Phase III APPOINT-PNH Trial

Antonio M Risitano, Bing Han, Austin Kulasekararaj, Yasutaka Ueda, Phillip Scheinberg, Carlos de Castro, Jaroslaw P. Maciejewski, Josefin Snellman, Ranjan Tiwari, Marion Dahlke, Régis Peffault de Latour

Blood (2023) 142 (Supplement 1): 4084.

https://doi.org/10.1182/blood-2023-180866

Published: 2023

Journal Articles

Oral Monotherapy with Iptacopan, a Proximal Complement Inhibitor of Factor B, Has Superior Efficacy to Intravenous Terminal Complement Inhibition with Standard of Care Eculizumab or Ravulizumab and Favorable Safety in Patients with Paroxysmal Nocturnal Hemoglobinuria and Residual Anemia: Results from the Randomized, Active-Comparator-Controlled, Open-Label, Multicenter, Phase III Apply-PNH Study

Blood (2022) 140 (Supplement 2): LBA-2.

https://doi.org/10.1182/blood-2022-171469

Published: 2022

Journal Articles

Trial in Progress: Randomized, Double-Blind, Placebo-Controlled Multicenter Phase 3 Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients with Cold Agglutinin Disease (CASCADE)

Bernd Jilma, Eloy Roman, Yasutaka Ueda, Silvia Mappa, Johan Szamosi, Jessica Savage, Alexander Roeth

Blood (2022) 140 (Supplement 1): 5326–5327.

https://doi.org/10.1182/blood-2022-155989

Published: 2022

Journal Articles

Clinically Important Difference for the FACIT-Fatigue Scale in Paroxysmal Nocturnal Hemoglobinuria: A Derivation from International PNH Registry Patient Data

David Cella, Peter Johansson, Yasutaka Ueda, Ioannis Tomazos, Philippe Gustovic, Alice Wang, Ami S. Patel, Hubert Schrezenmeier

Blood (2021) 138 (Supplement 1): 1952.

https://doi.org/10.1182/blood-2021-153127

Published: 2021

Journal Articles

Clinical Significance of Small PNH-Type Cell Populations in Bone Marrow Failure Syndromes - an Interim Analysis of Japanese Multicentrer Prospective Study -

Yasutaka Ueda, Kohei Hosokawa, Ken Ishiyama, Hiroyuki Takamori, Yuji Yonemura, Naoshi Obara, Hideyoshi Noji, Kiyoshi Ando, Tsutomu Shichishima, Takayuki Ikezoe, Shigeru Chiba, Haruhiko Ninomiya, Tatsuya Kawaguchi, Jun-ichi Nishimura, Yuzuru Kanakura, Shinji Nakao

Blood (2021) 138 (Supplement 1): 1122.

https://doi.org/10.1182/blood-2021-146353

Published: 2021

Journal Articles

An Optimized Crovalimab Dose and Regimen Reduced the Formation of Drug-Target-Drug Complexes in Patients with Paroxysmal Nocturnal Hemoglobinuria from the Phase I/II COMPOSER Trial

Blood (2020) 136 (Supplement 1): 2–3.

https://doi.org/10.1182/blood-2020-136265

Published: 2020

Journal Articles

Inflammation and Fatigue in Patients with Cold Agglutinin Disease (CAD): Analysis from the Phase 3 Cardinal Study

Ilene C. Weitz, Yasutaka Ueda, Frank Shafer, Jun Su, Meredith Foster, Michael Storek, Parija Patel

Blood (2020) 136 (Supplement 1): 7–8.

https://doi.org/10.1182/blood-2020-140335

Published: 2020

Journal Articles

Current Status and Optimal Management of Eculizumab Poor-Responders Due to C5 Polymorphisms

Yasutaka Ueda, MD PhD, Hiroyuki Takamori, MD, Jun-Ho Jang, MD PhD, Chezi Ganzel, MD, Saskia Langemeijer, MD PhD, Makiko Osato, MS, Petra Muus, MD PhD, Jong-Wook Lee, MD PhD, Jun-Ichi Nishimura, MD PhD, Yuzuru Kanakura, MD PhD

Blood (2018) 132 (Supplement 1): 2323.

https://doi.org/10.1182/blood-2018-99-111230

Published: 2018

Journal Articles

Baseline Assessment of Patients with Newly-Diagnosed Acquired Bone Marrow Failure Enrolled in a Prospective Observational Study, "Search for Unidentified Links between PNH Clone Size and the Related Clinical Manifestations By High Precision Flow Cytometry (SUPREMACY)"

Yuji Yonemura, MD PhD, Tatsuya Kawaguchi, MD PhD, Ken Ishiyama, MD PhD, Chiharu Sugimori, MD PhD, Kohei Hosokawa, Naoshi Obara, MD PhD, Hideyoshi Noji, MD PhD, Yukari Shirasugi, MD PhD, Yasutaka Ueda, MD PhD, Hiroyuki Takamori, MD PhD, Tsutomu Shichishima, MD PhD, Shigeru Chiba, MD PhD, Haruhiko Ninomiya, MD PhD, Kiyoshi Ando, MD PhD, Jun-Ichi Nishimura, MD PhD, Yuzuru Kanakura, MD PhD, Masanori Takahashi, Shinji Nakao, MD PhD

Blood (2017) 130 (Supplement 1): 2466.

https://doi.org/10.1182/blood.V130.Suppl_1.2466.2466

Published: 2017

Journal Articles

Elucidation of Autoinflammatory Mechanism in Pigt-PNH

Makiko Osato, MS, Yoshiko Murakami, MD PhD, Shogo Murata, MD PhD, Jun-Ichi Nishimura, MD PhD, Yasutaka Ueda, MD PhD, Taroh Kinoshita, PhD, Yuzuru Kanakura, MD PhD

Blood (2017) 130 (Supplement 1): 2201.

https://doi.org/10.1182/blood.V130.Suppl_1.2201.2201

Published: 2017

Journal Articles

Effects of Eculizumab Treatment on the Quality of Life in Patients with Paroxysmal Nocturnal Hemoglobinuria Treated in Japan

Yasutaka Ueda, MD PhD, Naoshi Obara, MD PhD, Yuji Yonemura, MD PhD, Hideyoshi Noji, MD PhD, Takahisa Matsuda, Hirozumi Akiyama, Takayuki Ikezoe, MD, PhD, Shigeru Chiba, MD PhD, Yoshinobu Kanda, MD PhD, Tatsuya Kawaguchi, MD PhD, Tsutomu Shichishima, MD PhD, Hideki Nakakuma, MD PhD, Shinichiro Okamoto, MD PhD, Jun-Ichi Nishimura, MD PhD, Yuzuru Kanakura, MD PhD, Haruhiko Ninomiya, MD PhD

Blood (2017) 130 (Supplement 1): 4749.

https://doi.org/10.1182/blood.V130.Suppl_1.4749.4749

Published: 2017

Journal Articles

The First Follow-up Data Analysis of Patients with Acquired Bone Marrow Failure Harboring a Small Population of PNH-Type Cells in the Japanese, Multicenter, Prospective Study Optima

Yasutaka Ueda, MD PhD, Jun-Ichi Nishimura, MD PhD, Chiharu Sugimori, MD PhD, Kohei Hosokawa, MDPhD, Yuji Yonemura, MD PhD, Naoshi Obara, MDPhD, Hideyoshi Noji, MD PhD, Yoshihiko Nakamura, PhD, Yukari Shirasugi, MD PhD, Kiyoshi Ando, Tsutomu Shichishima, MD PhD, Haruhiko Ninomiya, MD PhD, Shigeru Chiba, MD, PhD, Tatsuya Kawaguchi, MD PhD, Yuzuru Kanakura, MD PhD, Shinji Nakao, MD PhD

Blood (2016) 128 (22): 3896.

https://doi.org/10.1182/blood.V128.22.3896.3896

Published: 2016

Journal Articles

Paroxysmal Nocturnal Hemoglobinuria Caused By Pigt Mutations; Atypical PNH

Yoshiko Murakami, MDPhD, Michi Kawamoto, MD, Norimitsu Inoue, MD PhD, Makiko Osato, DS, Shogo Murata, MD PhD, Sho Murase, MD, Hajime Yoshimura, MD, Yasutaka Ueda, MD PhD, Jun-Ichi Nishimura, MD PhD, Yuzuru Kanakura, MD PhD, Nobuo Kohara, MD, Taroh Kinoshita, PhD

Blood (2016) 128 (22): 2450.

https://doi.org/10.1182/blood.V128.22.2450.2450

Published: 2016

NARROW

Patient Preferences and Treatment Satisfaction in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Treated with Crovalimab and Approved C5 Inhibitors in the Phase III Randomized COMMODORE 1 and COMMODORE 2 Trials

Insights from Trajectories of PIGA -Mutated Clones in Paroxysmal Nocturnal Hemoglobinuria

Combined Safety Data for Sutimlimab in Cold Agglutinin Disease: A Post-Hoc Analysis of the Phase 3 Cardinal and Cadenza Studies

Patient-Reported Improvements in Fatigue and Health-Related Quality of Life in the Phase 3 Studies Apply-PNH and Appoint-PNH Evaluating the Use of Iptacopan in C5 Inhibitor-Treated and Treatment-Naïve Patients with Paroxysmal Nocturnal Hemoglobinuria

Factor B Inhibition with Oral Iptacopan Monotherapy Demonstrates Sustained Long-Term Efficacy and Safety in Anti-C5-Treated Patients (pts) with Paroxysmal Nocturnal Hemoglobinuria (PNH) and Persistent Anemia: Final 48-Week Results from the Multicenter, Phase III APPLY-PNH Trial

Cold Agglutinin Disease (CAD) Real-World Evidence (CADENCE) Registry: Update to the Study Design of the First International, Prospective CAD Registry

Efficacy of Sutimlimab in Improving FACIT-Fatigue Scores in Patients with Cold Agglutinin Disease: A Post-Hoc Analysis of the Phase 3 Cadenza (Part A) Study

Categorization of Hematological Responses to Oral Iptacopan Monotherapy in Anti-C5-Treated Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) and Persistent Anemia in the Phase III APPLY-PNH Trial and Complement Inhibitor-Naïve Patients in the Phase III APPOINT-PNH Trial

Trial in Progress: Randomized, Double-Blind, Placebo-Controlled Multicenter Phase 3 Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients with Cold Agglutinin Disease (CASCADE)

Clinically Important Difference for the FACIT-Fatigue Scale in Paroxysmal Nocturnal Hemoglobinuria: A Derivation from International PNH Registry Patient Data

Clinical Significance of Small PNH-Type Cell Populations in Bone Marrow Failure Syndromes - an Interim Analysis of Japanese Multicentrer Prospective Study -

An Optimized Crovalimab Dose and Regimen Reduced the Formation of Drug-Target-Drug Complexes in Patients with Paroxysmal Nocturnal Hemoglobinuria from the Phase I/II COMPOSER Trial

Inflammation and Fatigue in Patients with Cold Agglutinin Disease (CAD): Analysis from the Phase 3 Cardinal Study

Current Status and Optimal Management of Eculizumab Poor-Responders Due to C5 Polymorphisms

Baseline Assessment of Patients with Newly-Diagnosed Acquired Bone Marrow Failure Enrolled in a Prospective Observational Study, "Search for Unidentified Links between PNH Clone Size and the Related Clinical Manifestations By High Precision Flow Cytometry (SUPREMACY)"

Elucidation of Autoinflammatory Mechanism in Pigt-PNH

Effects of Eculizumab Treatment on the Quality of Life in Patients with Paroxysmal Nocturnal Hemoglobinuria Treated in Japan

The First Follow-up Data Analysis of Patients with Acquired Bone Marrow Failure Harboring a Small Population of PNH-Type Cells in the Japanese, Multicenter, Prospective Study Optima

Paroxysmal Nocturnal Hemoglobinuria Caused By Pigt Mutations; Atypical PNH

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American Society of Hematology

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American Society of Hematology

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