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Journal Articles

Clinical Breakthrough Hemolysis (BTH) during Monotherapy with the Oral Factor B Inhibitor Iptacopan Is Generally Not Severe and Managed without Treatment Discontinuation: 48-Week Data from the Phase III Apply-PNH and Appoint-PNH Trials in Paroxysmal Nocturnal Hemoglobinuria (PNH)

Regis Peffault De Latour, Austin Kulasekararaj, Phillip Scheinberg, Philippe Ferber, Tomasz Lawniczek, Christine Thorburn, Marion Dahlke, Antonio M Risitano

Blood (2023) 142 (Supplement 1): 1338.

https://doi.org/10.1182/blood-2023-179377

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

Eltrombopag in Pediatric Patients with Previously Untreated or Refractory/Relapsed Severe Aplastic Anemia: The Phase II Escalate Trial

Akiko Shimamura, Alexey Maschan, Carolyn Bennett, Jason E Farrar, Sujith Samarasinghe, Brigitte Strahm, Winfred C. Wang, Adrianna Vlachos, Charlotte M. Niemeyer, Timothy S. Olson, Denise D'Alessio, Elise Burmeister Getz, Tomasz Lawniczek, Yunnan Xu, David A. Williams

Blood (2022) 140 (Supplement 1): 705–707.

https://doi.org/10.1182/blood-2022-158214

Published: 2022

Journal Articles

Correlation of Bridging and Lymphodepleting Chemotherapy with Clinical Outcomes in Patients with Relapsed/Refractory Diffuse Large B-Cell Lymphoma Treated with Tisagenlecleucel

Blood (2019) 134 (Supplement_1): 2883.

https://doi.org/10.1182/blood-2019-124492

Published: 2019

Journal Articles

A 1-year randomized controlled trial of deferasirox vs deferoxamine for myocardial iron removal in β-thalassemia major (CORDELIA)

Dudley J. Pennell, John B. Porter, Antonio Piga, Yongrong Lai, Amal El-Beshlawy, Khawla M. Belhoul, Mohsen Elalfy, Akif Yesilipek, Yurdanur Kilinç, Tomasz Lawniczek, Dany Habr, Marianne Weisskopf, Yiyun Zhang, Yesim Aydinok

Blood (2014) 123 (10): 1447–1454.

https://doi.org/10.1182/blood-2013-04-497842

Published: 2014

Includes: Supplemental data

Journal Articles

Baseline Characteristics and Symptom Burden in RESPONSE: A Randomized, Open-Label, Phase 3 Study of Ruxolitinib In Polycythemia Vera Patients Resistant to or Intolerant of Hydroxyurea

Alessandro M. Vannucchi, MD, Jean-Jacques Kiladjian, MD, PhD, Martin Griesshammer, Prof. Dr. med., Tamás Masszi, MD, PhD, Simon Durrant, MBBS, FRCP, FRCPath, Pierre Zachee, MD, PhD, Fabrizio Pane, MD, Claire N Harrison, DM, FRCP, FRCPath, Shui He, PhD, Lance Leopold, MD, Jingjin Li, Ulrich Pirron, PhD, Tomasz Lawniczek, MD, PhD, Srdan Verstovsek, MD, PhD

Blood (2013) 122 (21): 4071.

https://doi.org/10.1182/blood.V122.21.4071.4071

Published: 2013

Journal Articles

Deferasirox Continues to Reduce Iron Overload in Non-Transfusion-Dependent Thalassemia: A One-Year, Open-Label Extension to a One-Year, Randomized, Double-Blind, Placebo-Controlled Study (THALASSA)

Ali T Taher, MD, John B Porter, MD, Vip Viprakasit, MD, DPhil, Antonis Kattamis, MD, Suporn Chuncharunee, MD, Pranee Sutcharitchan, MD, Noppadol Siritanaratkul, MD, Renzo Galanello, MD, Zeynep Karakas, MD, Tomasz Lawniczek, MD, Dany Habr, MD, Jacqueline Ros, PhD, Zewen Zhu, M Domenica Cappellini, MD

Blood (2012) 120 (21): 3258.

https://doi.org/10.1182/blood.V120.21.3258.3258

Published: 2012

Journal Articles

A Multicenter, Randomized, Open-Label Trial Evaluating Deferasirox Compared with Deferoxamine for the Removal of Cardiac Iron in Patients with β-Thalassemia Major and Iron Overload (CORDELIA).

Dudley J Pennell, MD, John B Porter, MD, Antonio Piga, MD, Yongrong Lai, MD, Amal El-Beshlawy, MD, Khawla Beloul, MD, Mohsen Elalfy, MD, Akif Yesilipek, MD, Yurdanur Kilinç, MD, Tomasz Lawniczek, MD, Dany Habr, MD, Marianne Weisskopf, PhD, Yiyun Zhang, PhD, Yesim Aydinok, MD

Blood (2012) 120 (21): 2124.

https://doi.org/10.1182/blood.V120.21.2124.2124

Published: 2012

Journal Articles

Deferasirox reduces iron overload significantly in nontransfusion-dependent thalassemia: 1-year results from a prospective, randomized, double-blind, placebo-controlled study

Ali T. Taher, John Porter, Vip Viprakasit, Antonis Kattamis, Suporn Chuncharunee, Pranee Sutcharitchan, Noppadol Siritanaratkul, Renzo Galanello, Zeynep Karakas, Tomasz Lawniczek, Jacqueline Ros, Yiyun Zhang, Dany Habr, Maria Domenica Cappellini

Blood (2012) 120 (5): 970–977.

https://doi.org/10.1182/blood-2012-02-412692

Published: 2012

Includes: Supplemental data

Journal Articles

Deferasirox Significantly Reduces Liver Iron Concentration In Non-Transfusion-Dependent Thalassemia Patients with Iron Overload: Results From the 1-Year Randomized, Double-Blind, Placebo-Controlled Phase II THALASSA Study

Ali T Taher, MD, John B. Porter, MD, Vip Viprakasit, MD; DPhil, Antonis Kattamis, MD, Suporn Chuncharunee, MD, Pranee Sutcharitchan, MD, Noppodol Siritanaratkul, MD, Renzo Galanello, MD, Zeynep Karakas, MD, Tomasz Lawniczek, MD, Jacqueline Ros, PhD, Yiyun Zhang, Dany Habr, MD, M. Domenica Cappellini, MD

Blood (2011) 118 (21): 902.

https://doi.org/10.1182/blood.V118.21.902.902

Published: 2011

Journal Articles

Safety of Deferasirox (Exjade®) in Myelodysplastic Syndromes (MDS) and Non-MDS Patients with Transfusional Iron Overload: A Pooled Analysis Focusing On Renal Function.

Mathias Schmid, M. Domenica Cappellini, John B. Porter, Peter L. Greenberg, MD, Tomasz Lawniczek, Sabine Glaser, Victor Dong, Norbert Gattermann, MD, PhD

Blood (2009) 114 (22): 1768.

https://doi.org/10.1182/blood.V114.22.1768.1768

Published: 2009

Journal Articles

A Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial of Deferasirox (Exjade®) in Patients with Low/Intermediate-1 Risk MDS and Transfusional Iron Overload.

Emanuele Angelucci, David Bowen, Silvia M.M. Magalhães, Tomasz Lawniczek, Shyanne Douma, Peter Jakobs, Guillermo Garcia-Manero

Blood (2009) 114 (22): 4854.

https://doi.org/10.1182/blood.V114.22.4854.4854

Published: 2009

Journal Articles

Randomized Phase II Study Evaluating the Efficacy and Safety of Deferasirox in Non-Transfusion-Dependent Thalassemia Patients with Iron Overload.

Ali Taher, John B. Porter, Antonis Kattamis, Vip Viprakasit, Tomasz Lawniczek, Raffaele Pereno, Oliver Schoenborn-Kellenberger, M. Domenica Cappellini

Blood (2009) 114 (22): 5111.

https://doi.org/10.1182/blood.V114.22.5111.5111

Published: 2009

NARROW

Clinical Breakthrough Hemolysis (BTH) during Monotherapy with the Oral Factor B Inhibitor Iptacopan Is Generally Not Severe and Managed without Treatment Discontinuation: 48-Week Data from the Phase III Apply-PNH and Appoint-PNH Trials in Paroxysmal Nocturnal Hemoglobinuria (PNH)

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

Eltrombopag in Pediatric Patients with Previously Untreated or Refractory/Relapsed Severe Aplastic Anemia: The Phase II Escalate Trial

Correlation of Bridging and Lymphodepleting Chemotherapy with Clinical Outcomes in Patients with Relapsed/Refractory Diffuse Large B-Cell Lymphoma Treated with Tisagenlecleucel

A 1-year randomized controlled trial of deferasirox vs deferoxamine for myocardial iron removal in β-thalassemia major (CORDELIA)

Baseline Characteristics and Symptom Burden in RESPONSE: A Randomized, Open-Label, Phase 3 Study of Ruxolitinib In Polycythemia Vera Patients Resistant to or Intolerant of Hydroxyurea

Deferasirox Continues to Reduce Iron Overload in Non-Transfusion-Dependent Thalassemia: A One-Year, Open-Label Extension to a One-Year, Randomized, Double-Blind, Placebo-Controlled Study (THALASSA)

A Multicenter, Randomized, Open-Label Trial Evaluating Deferasirox Compared with Deferoxamine for the Removal of Cardiac Iron in Patients with β-Thalassemia Major and Iron Overload (CORDELIA).

Deferasirox reduces iron overload significantly in nontransfusion-dependent thalassemia: 1-year results from a prospective, randomized, double-blind, placebo-controlled study

Deferasirox Significantly Reduces Liver Iron Concentration In Non-Transfusion-Dependent Thalassemia Patients with Iron Overload: Results From the 1-Year Randomized, Double-Blind, Placebo-Controlled Phase II THALASSA Study

Safety of Deferasirox (Exjade®) in Myelodysplastic Syndromes (MDS) and Non-MDS Patients with Transfusional Iron Overload: A Pooled Analysis Focusing On Renal Function.

A Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial of Deferasirox (Exjade®) in Patients with Low/Intermediate-1 Risk MDS and Transfusional Iron Overload.

Randomized Phase II Study Evaluating the Efficacy and Safety of Deferasirox in Non-Transfusion-Dependent Thalassemia Patients with Iron Overload.

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American Society of Hematology

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American Society of Hematology

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