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
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)
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)
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.
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.
Review
Medicine, General & Internal
Nuan-Nuan Xiu, Xiao-Dong Yang, Jia Xu, Bo Ju, Xiao-Yun Sun, Xi-Chen Zhao
Summary: Aggravated inflammatory stressors can repress normal and leukemic hematopoiesis, and relieved inflammatory stressors can facilitate penetration of neoplastic hematopoiesis.
WORLD JOURNAL OF CLINICAL CASES
(2023)
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
Oncology
Hana Votavova, Monika Belickova
Summary: The translated article discusses the differences between hypoplastic myelodysplastic syndrome (hMDS) and aplastic anemia (AA), highlighting the importance of molecular testing.
INTERNATIONAL JOURNAL OF ONCOLOGY
(2022)
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)
Editorial Material
Hematology
Daria V. Babushok
Editorial Material
Hematology
Pamela J. Sung, Daria V. Babushok
Article
Hematology
Yash B. Shah, Salvatore F. Priore, Yimei Li, Chi N. Tang, Peter Nicholas, Peter Kurre, Timothy S. Olson, Daria Babushok
Summary: Acquired aplastic anemia (AA) and inherited bone marrow failure syndromes (IBMFSs) require accurate and prompt diagnosis for optimal patient outcomes. Laboratory findings specific to the autoimmune pathogenesis of AA, including paroxysmal nocturnal hemoglobinuria (PNH) and acquired 6p CN-LOH clones, effectively distinguish AA from IBMFSs and should be used in diagnostic evaluation.
Editorial Material
Hematology
Daria Babushok
Summary: In this study, Patel et al. report the long-term outcomes of a landmark phase 2 trial investigating the combination of eltrombopag and immunosuppressive therapy for the upfront treatment of severe aplastic anemia (SAA).
Article
Hematology
Ji Zha, Lori K. Kunselman, Hongbo M. Xie, Brian Ennis, Yash B. Shah, Xia Qin, Jian-Meng Fan, Daria Babushok, Timothy S. Olson
Summary: This study reveals the significant impact of SBDS deficiency on host hematopoietic niche function, which may explain the morbidity noted in patients with SDS after HSCT. The results suggest that novel therapeutic strategies targeting host niches could improve clinical HSCT outcomes for patients with SDS.
Article
Hematology
Timothy J. Brown, Neil Barrett, Hu Meng, Emanuela Ricciotti, Ciara McDonnell, Andrew Dancis, Julianne Qualtieri, Garret A. FitzGerald, Melanie Cotter, Daria V. Babushok
Summary: Advancements in genomic diagnostics offer hope for better care of rare hematologic diseases. Researchers have found a targeted therapeutic approach for Ghosal hematodiaphyseal dysplasia, a rare autosomal recessive disease characterized by severe anemia and bone abnormalities. The study suggests that nonsteroidal anti-inflammatory drugs (NSAIDs) can improve hematologic function by reducing prostaglandin formation, offering a potential first-line treatment for this disease.
Article
Hematology
Craig W. Freyer, Daria Babushok, Noelle Frey, Saar Gill, Alison W. Loren, Selina M. Luger, Amit Maity, Mary Ellen Martin, John P. Plastaras, David L. Porter, Elizabeth O. Hexner
Summary: This study retrospectively compared the outcomes of alloHCT in MF patients who received different treatment regimens. The addition of low-dose TBI to Flu/Bu significantly reduced graft failure and improved the rates of molecular remission and full donor chimerism.
TRANSPLANTATION AND CELLULAR THERAPY
(2022)
Article
Medicine, Research & Experimental
Tammarah Sklarz, Stephanie N. Hurwitz, Natasha L. Stanley, Jane Juusola, Adam Bagg, Daria Babushok
COLD SPRING HARBOR MOLECULAR CASE STUDIES
(2020)