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Journal Articles

OMS906, a Novel Alternative Pathway MASP-3 Inhibitor, Normalizes Hemoglobin Levels and Increases Clone Size in Treatment-Naïve PNH Patients

Oksana Karnabeda, Valentyn Moskalenko, Zoreslava Lysak, Narinder Nangia, Charlotte A. Osborne, J. Steve Whitaker, Eleni Gavriilaki, Jens Panse

Blood (2023) 142 (Supplement 1): 573.

https://doi.org/10.1182/blood-2023-177921

Published: 2023

Journal Articles

Patient-Reported Outcomes: Danicopan As Add-on Therapy to Ravulizumab or Eculizumab Versus Placebo in Patients with Paroxysmal Nocturnal Hemoglobinuria and Clinically Significant Extravascular Hemolysis

Caroline I Piatek, Jong-Wook Lee, Morag Griffin, Jun-Ichi Nishimura, Christopher J. Patriquin, Hubert Schrezenmeier, Wilma Barcellini, Jamile Shammo, Anna Gaya, Yogesh Patel, Peng Liu, Karl-Johan Myrén, Flore Sicre De Fontbrune, Jens Panse, Austin Kulasekararaj

Blood (2023) 142 (Supplement 1): 1346.

https://doi.org/10.1182/blood-2023-190015

Published: 2023

Journal Articles

Allogeneic Hematopoietic Stem Cell Transplantation in Advanced Systemic Mastocytosis

Blood (2023) 142 (Supplement 1): 3605.

https://doi.org/10.1182/blood-2023-178304

Published: 2023

Journal Articles

Elenestinib, an Investigational, Next Generation KIT D816V Inhibitor, Reduces Mast Cell Burden, Improves Symptoms, and Has a Favorable Safety Profile in Patients with Indolent Systemic Mastocytosis: Analysis of the Harbor Trial

Blood (2023) 142 (Supplement 1): 76.

https://doi.org/10.1182/blood-2023-188904

Published: 2023

Journal Articles

CNS Irradiation in Adult De Novo B-Precursor ALL / Lbl: Results of the Randomized GMALL T rial 08/2013 Indicate Potential Antileukemic Efficacy

Blood (2023) 142 (Supplement 1): 828.

https://doi.org/10.1182/blood-2023-179442

Published: 2023

Journal Articles

Analysis of the Impact of Body Mass Index (BMI) on the Durability of Response in Patients with Aplastic Anemia Treated with Weight-Adjusted Horse Anti-Thymocyte Globulin (hATG)

Jeanette Walter, Benjamin Rolles, Yannic Schumacher, Frauke Theis, Leo Hansmann, Daniel Heudobler, Markus Radsak, Michael Heuser, Julia-Annabell Georgi, Jörg Chromik, Beatrice Drexler, Jörg Schubert, Kim Kricheldorf, Susanne Isfort, Jens Panse, Tim H. Brümmendorf, Fabian Beier

Blood (2023) 142 (Supplement 1): 5641.

https://doi.org/10.1182/blood-2023-183075

Published: 2023

Journal Articles

Real-Life Data of Cyclosporine (CsA) Tapering in Patients with Very/ Severe Aplastic Anemia (v/sAA) Treated with Horse Anti-Thymocyte Globulin (hATG/CsA) Suggest Higher Risk of AA Relapse at Cyclosporine Plasma Levels below 100 Nanogram per Milliliter

Jeanette Walter, Kim Kricheldorf, Yannic Schumacher, Susanne Isfort, Jörg Chromik, Beatrice Drexler, Frauke Theis, Jens Panse, Tim H. Brümmendorf, Fabian Beier

Blood (2023) 142 (Supplement 1): 5636.

https://doi.org/10.1182/blood-2023-183046

Published: 2023

Journal Articles

Patient-Reported Outcomes (PROs) in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Treated with Crovalimab and Eculizumab: Results from the Phase III Randomized COMMODORE 2 and COMMODORE 1 Trials

Jens Panse, Jaroslav Cermak, Olena Kyselova, Akihiko Gotoh, Fahri Sahin, Hubert Schrezenmeier, Alice C. Chang, Brittany Gentile, Marianne Uguen, Bing Han

Blood (2023) 142 (Supplement 1): 4090.

https://doi.org/10.1182/blood-2023-177584

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

Quality of Life Analysis in Patients with AA Using the QLQ-AA/PNH-54 at Initial Diagnosis of AA - a Registry Based Analysis

Jeanette Walter, Andrea Petermann-Meyer, Hatice Soysal, Anna Doleschall, Deborah Christen, Kim Kricheldorf, Susanne Isfort, Tim H. Brümmendorf, Fabian Beier, Jens Panse

Blood (2023) 142 (Supplement 1): 5635.

https://doi.org/10.1182/blood-2023-182810

Published: 2023

Journal Articles

Effects on Survival of Non-Myeloablative Chemoimmunotherapy Compared to High-Dose Chemotherapy Followed By Autologous Stem Cell Transplantation (HDC-ASCT) As Consolidation Therapy in Patients with Primary CNS Lymphoma - Results of an International Randomized Phase III Trial (MATRix/IELSG43)

Blood (2022) 140 (Supplement 2): LBA-3.

https://doi.org/10.1182/blood-2022-171733

Published: 2022

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

Germline Variants in MDM4 Cause a Disorder of p53 Dysregulation and Insufficient Telomere Maintenance

Robert Meyer, Matthias Begemann, Marcus Jakob, Claudia Khurana, Daniela Dey, Tarek Hanafee Alali, Heinz Gabriel, Kim Kricheldorf, Margherita Vieri, Susanne Isfort, Martin Kirschner, Jens Panse, Udo Kontny, Selim Corbacioglu, Tim H. Brümmendorf, Ingo Kurth, Fabian Beier

Blood (2022) 140 (Supplement 1): 2951–2952.

https://doi.org/10.1182/blood-2022-168631

Published: 2022

Journal Articles

Long-Term Safety and Efficacy of Pegcetacoplan Treatment in Adults with Paroxysmal Nocturnal Hemoglobinuria

Christopher J. Patriquin, Andrija Bogdanovic, Morag Griffin, Richard Kelly, Jaroslaw P. Maciejewski, Brian Mulherin, Régis Peffault de Latour, Alexander Roeth, Veena Selvaratnam, Jeffrey Szer, Jessica Savage, Regina Horneff, Lisa Tan, Michael Yeh, Jens Panse

Blood (2022) 140 (Supplement 1): 2921–2923.

https://doi.org/10.1182/blood-2022-163625

Published: 2022

Journal Articles

Clinical Presentation of Patients with Adult Late-Onset Telomere Biology Disorders - Results from the Aachen Telomeropathy Registry

Blood (2021) 138 (Supplement 1): 1130.

https://doi.org/10.1182/blood-2021-153630

Published: 2021

Journal Articles

Post Hoc Analysis of the Effect of Pegcetacoplan Treatment of Patients with Paroxysmal Nocturnal Hemoglobinuria and Baseline Hemoglobin Levels Greater Than 10 Grams per Deciliter

Jens Panse, Nicolas Daguindau, Sonia Okuyama Sasaki, Regis Peffault De Latour, Philippe Schafhausen, Nicole Straetmans, Mohammed Al-Adhami, Temitayo Ajayi, Crystal Chen, Michael Yeh, Raymond S Wong

Blood (2021) 138 (Supplement 1): 2194.

https://doi.org/10.1182/blood-2021-146962

Published: 2021

Journal Articles

Nintedanib targets KIT D816V neoplastic cells derived from induced pluripotent stem cells of systemic mastocytosis

Blood (2021) 137 (15): 2070–2084.

https://doi.org/10.1182/blood.2019004509

Published: 2021

Includes: Supplemental data

Journal Articles

Prevalence of Inherited Predisposition Syndromes in Young Patients with Acute Myeloid Leukemia and Aberrant Karyotype

Martin Kirschner, Benjamin Rolles, MD, Martina Crysandt, Christoph Röllig, Prof., MD, Friedrich Stolzel, MD, Martin Bornhäuser, MD, Carsten Müller-Tidow, MD, Kim Kricheldorf, Margherita Vieri, PhD, Matthias Begemann, Angela Maurer, Jens Panse, MD, Susanne Isfort, Marcin Wlodarski, MD PhD, Sushree Sangita Sahoo, PhD, Tim H Brümmendorf, MD, Edgar Jost, Fabian Beier, MD

Blood (2020) 136 (Supplement 1): 41–42.

https://doi.org/10.1182/blood-2020-142815

Published: 2020

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

Results of the Pegasus Phase 3 Randomized Trial Demonstrating Superiority of the C3 Inhibitor, Pegcetacoplan, Compared to Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria

Peter Hillmen, MBChB, PhD, Jeffrey Szer, MBBS, Ilene C. Weitz, Alexander Röth, MD, Britta Hoechsmann, Jens Panse, MD, Kensuke Usuki, MD PhD, Morag Griffin, MBChB, FRCPath, Jean-Jacques Kiladjian, MD PhD, Carlos M. de Castro, MD, Hisakazu Nishimori, MD PhD, Lisa Tan, Mohamed Hamdani, Pascal Deschatelets, PhD, Cedric Francois, MD PhD, Federico Grossi, MD, Antonio Risitano, Regis Peffault De Latour, MD PhD

Blood (2020) 136 (Supplement 1): 35–37.

https://doi.org/10.1182/blood-2020-142174

Published: 2020

NARROW

OMS906, a Novel Alternative Pathway MASP-3 Inhibitor, Normalizes Hemoglobin Levels and Increases Clone Size in Treatment-Naïve PNH Patients

Patient-Reported Outcomes: Danicopan As Add-on Therapy to Ravulizumab or Eculizumab Versus Placebo in Patients with Paroxysmal Nocturnal Hemoglobinuria and Clinically Significant Extravascular Hemolysis

Allogeneic Hematopoietic Stem Cell Transplantation in Advanced Systemic Mastocytosis

Elenestinib, an Investigational, Next Generation KIT D816V Inhibitor, Reduces Mast Cell Burden, Improves Symptoms, and Has a Favorable Safety Profile in Patients with Indolent Systemic Mastocytosis: Analysis of the Harbor Trial

CNS Irradiation in Adult De Novo B-Precursor ALL / Lbl: Results of the Randomized GMALL T rial 08/2013 Indicate Potential Antileukemic Efficacy

Analysis of the Impact of Body Mass Index (BMI) on the Durability of Response in Patients with Aplastic Anemia Treated with Weight-Adjusted Horse Anti-Thymocyte Globulin (hATG)

Real-Life Data of Cyclosporine (CsA) Tapering in Patients with Very/ Severe Aplastic Anemia (v/sAA) Treated with Horse Anti-Thymocyte Globulin (hATG/CsA) Suggest Higher Risk of AA Relapse at Cyclosporine Plasma Levels below 100 Nanogram per Milliliter

Patient-Reported Outcomes (PROs) in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Treated with Crovalimab and Eculizumab: Results from the Phase III Randomized COMMODORE 2 and COMMODORE 1 Trials

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

Quality of Life Analysis in Patients with AA Using the QLQ-AA/PNH-54 at Initial Diagnosis of AA - a Registry Based Analysis

Effects on Survival of Non-Myeloablative Chemoimmunotherapy Compared to High-Dose Chemotherapy Followed By Autologous Stem Cell Transplantation (HDC-ASCT) As Consolidation Therapy in Patients with Primary CNS Lymphoma - Results of an International Randomized Phase III Trial (MATRix/IELSG43)

Germline Variants in MDM4 Cause a Disorder of p53 Dysregulation and Insufficient Telomere Maintenance

Long-Term Safety and Efficacy of Pegcetacoplan Treatment in Adults with Paroxysmal Nocturnal Hemoglobinuria

Clinical Presentation of Patients with Adult Late-Onset Telomere Biology Disorders - Results from the Aachen Telomeropathy Registry

Post Hoc Analysis of the Effect of Pegcetacoplan Treatment of Patients with Paroxysmal Nocturnal Hemoglobinuria and Baseline Hemoglobin Levels Greater Than 10 Grams per Deciliter

Nintedanib targets KIT D816V neoplastic cells derived from induced pluripotent stem cells of systemic mastocytosis

Prevalence of Inherited Predisposition Syndromes in Young Patients with Acute Myeloid Leukemia and Aberrant Karyotype

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

Results of the Pegasus Phase 3 Randomized Trial Demonstrating Superiority of the C3 Inhibitor, Pegcetacoplan, Compared to Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria

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American Society of Hematology

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American Society of Hematology

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