Article
Oncology
Bruno Fattizzo, Robin Ireland, Alan Dunlop, Deborah Yallop, Shireen Kassam, Joanna Large, Shreyans Gandhi, Petra Muus, Charles Manogaran, Katy Sanchez, Dario Consonni, Wilma Barcellini, Ghulam J. Mufti, Judith C. W. Marsh, Austin G. Kulasekararaj
Summary: In this large single-center study, a high prevalence of small and very small PNH clones was reported among patients tested. PNH positivity was associated with better response to immunosuppressive therapy and stem cell transplant, as well as a more favorable impact on disease progression, leukemic evolution and overall survival, highlighting the importance of systematic PNH testing in patients with bone marrow failures.
Article
Immunology
Jie Wang, Zheng Yang, Danhong Ren, Zhanli Shi, Kun Fang, Zhihui Li
Summary: This is a case report about a patient with AA-PNH syndrome complicated with fatal disseminated varicella zoster virus infection, highlighting the serious complications and high mortality rate associated with this infection. Clinicians need to be aware of the infection in such patients and provide prompt and effective treatment.
FRONTIERS IN CELLULAR AND INFECTION MICROBIOLOGY
(2023)
Review
Hematology
Jens Panse
Summary: In the past 20 years, therapy for paroxysmal nocturnal hemoglobinuria (PNH) mainly relied on antibody-based terminal complement inhibition. PNH is a disease characterized by a mutation that causes the absence or deficiency of complement-regulatory proteins on blood cells, leading to intravascular hemolysis and related complications. Recently, there has been a development of new drugs targeting the proximal and terminal complement cascade, with the approval of the first proximal complement inhibitor targeting C3 in 2021. This article aims to provide an overview of the progress made in PNH treatment and discuss the approved therapeutic options, as well as the potential impact and consequences of current and future treatments on patients' lives.
AMERICAN JOURNAL OF HEMATOLOGY
(2023)
Review
Immunology
Melissa A. Colden, Sushant Kumar, Bolormaa Munkhbileg, Daria V. Babushok
Summary: Paroxysmal Nocturnal Hemoglobinuria (PNH) is a disease that involves mutations in a specific gene, leading to hemolysis and abnormal clonal expansion of blood cells. The mechanisms behind this expansion are still debated, but recent advancements in research and technology offer new opportunities for understanding the disease.
FRONTIERS IN IMMUNOLOGY
(2022)
Article
Hematology
Gloria F. Gerber, Robert A. Brodsky
Summary: This article discusses the theoretical basis and clinical studies of using C3 inhibitors in the treatment of PNH, as well as provides suggestions for treatment sequencing.
Article
Oncology
Eleni Gavriilaki, Athanasios Tragiannidis, Maria Papathanasiou, Sotiria Besikli, Paraskevi Karvouni, Vassiliki Douka, Eleni Paphianou, Emmanuel Hatzipantelis, Giorgos Papaioannou, Anastasia Athanasiadou, Anastasia Marvaki, Alkistis-Kira Panteliadou, Anna Vardi, Ioannis Batsis, Antonia Syrigou, Despina Mallouri, Chrysavgi Lalayanni, Ioanna Sakellari
Summary: This study aims to investigate the efficiency and safety of therapeutic methods for aplastic anemia and paroxysmal nocturnal hemoglobinuria. The study found that novel treatments are changing the field of BMF syndromes, but further research is needed to personalize algorithms.
FRONTIERS IN ONCOLOGY
(2022)
Review
Immunology
Chenyuan Li, Xifeng Dong, Huaquan Wang, Zonghong Shao
Summary: Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired genetic mutation disease caused by defects in the PIG-A gene, leading to hemolysis of erythrocyte membranes.
FRONTIERS IN IMMUNOLOGY
(2021)
Article
Medicine, General & Internal
Peter Hillmen, Jeff Szer, Ilene Weitz, Alexander Roeth, Britta Hoechsmann, Jens Panse, Kensuke Usuki, Morag Griffin, Jean-Jacques Kiladjian, Carlos de Castro, Hisakazu Nishimori, Lisa Tan, Mohamed Hamdani, Pascal Deschatelets, Cedric Francois, Federico Grossi, Temitayo Ajayi, Antonio Risitano, Regis Peffault de la Tour
Summary: The study demonstrated that Pegcetacoplan was superior to eculizumab in improving hemoglobin and clinical and hematologic outcomes in patients with PNH by providing broad hemolysis control, including control of intravascular and extravascular hemolysis.
NEW ENGLAND JOURNAL OF MEDICINE
(2021)
Article
Oncology
Niki Vyrides, Vassiliki Douka, Eleni Gavriilaki, Georgios Papaioannou, Anastasia Athanasiadou, Sofia Neofytou, Yiannis Vyrides, Chrysavgi Lalayanni, Achilles Anagnostopoulos, Styliani Kokoris
Summary: PNH patients with MDS-related cytogenetic abnormalities may see these anomalies disappear, complicating the distinction between MDS and PNH. In transplant-eligible patients, clinical phenotype may drive treatment decisions in the era of complement inhibition, highlighting the need for further research on the transient presence of cytogenetic abnormalities.
Review
Hematology
Robert A. Brodsky
Summary: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare complement-mediated hemolytic anemia with diverse manifestations, requiring differentiated treatment approaches; terminal complement inhibition is effective for intravascular hemolysis treatment but not bone marrow failure; novel complement inhibitors under clinical development show promising prospects for future applications.
Article
Hematology
Fergun Yilmaz, Nur Soyer, Guldane Cengiz Seval, Sinem Civriz Bozdag, Pervin Topcuoglu, Ali Unal, Leylagul Kaynar, Gokhan Ozgur, Gulsan Sucak, Hakan Goker, Mustafa Velet, Hakan Ozdogu, Mehmet Yilmaz, Emin Kaya, Ozan Salim, Burak Deveci, Ihsan Karadogan, Guray Saydam, Fahri Sahin, Filiz Vural
Summary: Allogeneic HCT with MSDs or MUDs is a good option for selected patients with classical PNH and PNH-AA, showing high overall survival rates and graft-versus-host disease-free, failure-free survival rates. Results did not significantly differ between classical PNH and PNH-AA patients, or between transplantations with matched sibling donors and matched unrelated donors.
TURKISH JOURNAL OF HEMATOLOGY
(2021)
Review
Hematology
Yali Du, Bing Han
Summary: In countries where eculizumab is unavailable, patients with refractory AA/PNH and severe classical PNH are candidates for HSCT, with major causes of death from transplantation being GVHD, infection, and thrombotic microangiopathy. A haploidentical donor is a potential choice for patients without an HLA-matched donor. The combination of eculizumab and HSCT may help prevent GVHD.
TRANSPLANTATION AND CELLULAR THERAPY
(2021)
Review
Hematology
Austin G. Kulasekararaj, Ioanna Lazana
Summary: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematological disorder characterized by deficiency of GPI-linked complement regulators. Despite the introduction of C5 inhibitors, residual hemolysis still occurs, leading to anemia and transfusion dependency in some patients. The development of longer-acting and subcutaneous formulations of C5 inhibitors, as well as proximal complement inhibitors, have shown promising results in improving hemoglobin levels and reducing hemolysis. Combination treatments have also been explored. This review discusses the current therapeutic options and emerging approaches for PNH.
AMERICAN JOURNAL OF HEMATOLOGY
(2023)
Review
Hematology
Raymond S. M. Wong
Summary: This article reviews multiple clinical studies on the efficacy and safety of pegcetacoplan treatment in adults with PNH. The studies found that pegcetacoplan showed superior improvements in hemoglobin levels and other hematologic parameters, and effectively managed anemia and anemia-related complications.
THERAPEUTIC ADVANCES IN HEMATOLOGY
(2022)
Article
Hematology
Silas Baenziger, Kimmo Weisshaar, Reetta Arokoski, Sabine Gerull, Joerg Halter, Alicia Rovo, Mario Bargetzi, Jeroen S. Goede, Yuliya Senft, Sabine Valenta, Jakob R. Passweg, Beatrice Drexler
Summary: This study developed and tested a customized electronic patient-reported outcomes (ePRO) system for patients with aplastic anemia (AA) and paroxysmal nocturnal hemoglobinuria (PNH). The system showed high adherence, satisfaction, and performance, indicating a high potential for data collection and patient guidance. However, the integration into clinical workflows is crucial for routine use.
ANNALS OF HEMATOLOGY
(2023)
Article
Hematology
David H. Henry, John Glaspy, Rosemary Harrup, Moshe Mittelman, Amy Zhou, Hetty E. Carraway, Charles Bradley, Gopal Saha, Katharina Modelska, Pamela Bartels, Robert Leong, Kin-Hung P. Yu
Summary: The study confirmed that among the three starting doses of oral roxadustat, 2.5 mg/kg showed the best performance in treating anemia, facilitating transfusion independence and reducing the need for RBC transfusions. Roxadustat administered orally thrice weekly was well-tolerated, with no fatalities or progression to acute myeloid leukemia observed.
AMERICAN JOURNAL OF HEMATOLOGY
(2022)
Letter
Hematology
Meghan C. Thompson, Rosemary A. Harrup, Catherine C. Coombs, Lindsey E. Roeker, Jeffrey J. Pu, Michael Y. Choi, Paul M. Barr, John N. Allan, Martin Simkovic, Lori Leslie, Joanna Rhodes, Elise A. Chong, Manali Kamdar, Alan Skarbnik, Frederick Lansigan, Brittany McCall, Khalid Saja, Martin J. S. Dyer, Harriet S. Walter, Marcus Lefebure, Maria Thadani-Mulero, Michelle Boyer, Juliana Biondo, Kavita Sail, Beenish S. Manzoor, Richard Furman, Kurt S. Bantilan, Andre Goy, Tatyana Feldman, Dominic Labella, Stephen J. Schuster, Jae Park, Lia Palomba, Andrew Zelenetz, Toby A. Eyre, Arnon P. Kater, John F. Seymour, Anthony R. Mato
Article
Hematology
Courtney D. DiNardo, Sangeetha Venugopal, Curtis Lachowiez, Koichi Takahashi, Sanam Loghavi, Guillermo Montalban-Bravo, Xuemei Wang, Hetty Carraway, Mikkael Sekeres, Ameenah Sukkur, Danielle Hammond, Kelly Chien, Abhishek Maiti, Lucia Masarova, Koji Sasaki, Yesid Alvarado, Tapan Kadia, Nicholas J. Short, Naval Daver, Gautam Borthakur, Farhad Ravandi, Hagop M. Kantarjian, Bhumika Patel, Amy Dezern, Gail Roboz, Guillermo Garcia-Manero
Summary: The combination of enasidenib with azacitidine showed a 74% overall response rate in newly diagnosed mIDH2 MDS patients, while enasidenib monotherapy achieved a 35% response rate in patients after HMA failure. These findings demonstrate that enasidenib is an effective treatment option for mIDH2 MDS.
Letter
Hematology
Tariq Kewan, Waled Bahaj, Arda Durmaz, Mai Aly, Olisaemeka D. Ogbue, Hetty E. Carraway, Mikkael A. Sekeres, Valeria Visconte, Carmelo Gurnari, Jaroslaw P. Maciejewski
Meeting Abstract
Hematology
Fauzia Ullah, Danai Dima, Carmelo Gurnari, Suresh Kumar Balasubramanian, Olisaemeka Ogbue, Hussein Awada, Naomi Kawashima, Najiullah Omar, Swapna Thota, Hetty E. Carraway, Valeria Visconte, Jaroslaw P. Maciejewski
Meeting Abstract
Hematology
Nicholas Short, Tae Kon Kim, Rustin Lovewell, Elizabeth A. Griffiths, Hetty E. Carraway, Stephanie Zeidan, Elizabeth Knowles, Megan Nelson, Jahangheer Shaik, Chelsea Zhou, Emilia Alina Barbu, Zachary Cusumano, Subhadip Kundu, Sasan Sharee, Ron Copeland, Dallas Flies, Solomon Langermann, Han Myint, Jessica K. Altman, Farhad Ravandi
Article
Hematology
Amer M. Zeidan, Uwe Platzbecker, Jan Philipp Bewersdorf, Maximilian Stahl, Lionel Ades, Uma Borate, David Bowen, Rena Buckstein, Andrew Brunner, Hetty E. Carraway, Naval Daver, Maria Diez-Campelo, Theo de Witte, Amy E. DeZern, Fabio Efficace, Guillermo Garcia-Manero, Jacqueline S. Garcia, Ulrich Germing, Aristoteles Giagounidis, Elizabeth A. Griffiths, Robert P. Hasserjian, Eva Hellstrom-Lindberg, Marcelo Iastrebner, Rami Komrokji, Austin G. Kulasekararaj, Luca Malcovati, Yasushi Miyazaki, Olatoyosi Odenike, Valeria Santini, Guillermo Sanz, Phillip Scheinberg, Reinhard Stauder, Arjan A. van de Loosdrecht, Andrew H. Wei, Mikkael A. Sekeres, Pierre Fenaux
Summary: The initial response criteria developed by the International Working Group (IWG) in 2000 have limitations in their application to higher-risk MDS and their ability to fully capture the clinical benefits of novel investigational drugs. Therefore, an international panel of MDS experts used a modified Delphi process to develop consensus recommendations for updated response criteria that would be more reflective of patient-centered and clinically relevant outcomes. The updated criteria should lead to a better correlation between patient-centered outcomes and clinical trial results.
Meeting Abstract
Hematology
Julia H. Joo, Yanwen Chen, Aaron T. Gerds, Anjali Advani, Sophia R. Balderman, Hetty E. Carraway, Abhay Singh Singh, Sudipto Mukherjee
Meeting Abstract
Hematology
Tariq Kewan, Arda Durmaz, Waled Bahaj, Carmelo Gurnari, Hussein Awada, Olisaemeka Ogbue, Ramsha Ahmed, Simona Pagliuca, Hassan Awada, Yasuo Kubota, Minako Mori, Ben Ponvilawan, Bayan Al-Share, Bhumika J. Patel, Hetty E. Carraway, Jacob Scott, Suresh Kumar Balasubramanian, Taha Bat, Yazan F. Madanat, Mikkael A. Sekeres, Torsten Haferlach, Valeria Visconte, Jaroslaw P. Maciejewski
Meeting Abstract
Hematology
Teodora Kuzmanovic, Metis Hasipek, Samuel Li, Thomas Laframboise, Valeria Visconte, Sunisa Kongkiatkamon, Seth J. Corey, Sudipto Mukherjee, Anjali Advani, Aaron T. Gerds, Yogenthiran Saunthararajah, Sophia R. Balderman, Abhay Singh Singh, Hetty E. Carraway, Niroshan Nadarajah, Manja Meggendorfer, Jaroslaw P. Maciejewski, Bhumika J. Patel
Meeting Abstract
Oncology
Sudipto Mukherjee, Weichuan Dong, Aaron T. Gerds, Hetty E. Carraway, Abhay Singh, Anjali S. Advani, Siran M. Koroukian
CANCER EPIDEMIOLOGY BIOMARKERS & PREVENTION
(2023)
Meeting Abstract
Oncology
Farhad Ravandi-Kashani, Ashwin Kishtagari, Hetty Carraway, Emily Curran, Gary Schiller, Alex Cacovean, Bhagyashree Yadav, Thomas Butler, Jeffrey Lancet
Meeting Abstract
Oncology
Farhad Ravandi, Hetty Carraway, Jack Khouri, Ashwin Kishtagari, Emily Curran, Gary Schiller, Bhagyashree (Kelshikar) Yadav, Steve Morris, Alex Cacovean, Sanchita Mourya, Thomas Butler, Jeffrey Lancet
CLINICAL LYMPHOMA MYELOMA & LEUKEMIA
(2022)
Meeting Abstract
Oncology
Farhad Ravandi, Ashwin Kishtagari, Hetty E. Carraway, Gary J. Schiller, Steve Morris, Alexandru Cacovean, Bhagyashree Kelshikar Yadav, Thomas Butler, Jeffrey E. Lancet
JOURNAL OF CLINICAL ONCOLOGY
(2022)
Meeting Abstract
Oncology
Farhad Ravandi, Hetty E. Carraway, Lilia Taningco, Eric Laille, Jing Gong, Thomas Prebet, Daniel Lopes De Menezes, Andrew H. Wei
JOURNAL OF CLINICAL ONCOLOGY
(2022)