Editorial Material
Medical Laboratory Technology
Lucio Luzzatto
Summary: PNH is a chronic hemolytic disorder caused by an intrinsic red cell abnormality. Prolonged administration of corticosteroids or chemotherapy has not been proven to be beneficial. However, patients with PNH can live for many years with supportive management. Complement inhibitor therapy is currently recommended in most cases, but financial barriers need to be overcome.
JOURNAL OF CLINICAL LABORATORY ANALYSIS
(2022)
Review
Medicine, Research & Experimental
Imre Bodo, Ismail Amine, Ana Boban, Horia Bumbea, Alexander Kulagin, Elena Lukina, Agnieszka Piekarska, Irena Preloznik Zupan, Juraj Sokol, Jerzy Windyga, Jaroslav Cermak
Summary: Hemolysis in PNH is complement-mediated due to the lack of complement inhibitors in the cell membranes. To manage PNH, complement inhibition is the best approach. Three complement inhibitors – eculizumab, ravulizumab, and pegcetacoplan – are approved as targeted therapy for PNH. However, current guidelines do not consider the latest clinical trial evidence. Expert recommendations were created to identify specific populations who may benefit from switching to proximal C3 inhibition.
ADVANCES IN THERAPY
(2023)
Article
Pediatrics
Laura Gomez-Ganda, Maria Isabel Benitez-Carabante, Aurora Fernandez-Polo, Marina Munoz-Lopez, Berta Renedo-Miro, Gema Ariceta, Cristina Diaz De Heredia
Summary: In pediatric patients diagnosed with high-risk TA-TMA after HSCT, treatment with eculizumab led to improvement in renal function, proteinuria, and hypertension. The patients achieved resolution of TA-TMA after treatment with eculizumab for varying durations, and no increased risk of infection was observed with proper vaccine and antibiotic prophylaxis.
FRONTIERS IN PEDIATRICS
(2021)
Article
Urology & Nephrology
Aishwarya Ravindran, Lilian Monteiro Pereira Palma, Fernando C. Fervenza, Sanjeev Sethi
Summary: This study describes cases of patients with both C3 glomerulopathy and thrombotic microangiopathy. The overlap of these two diseases is rare and can present as a C3 glomerulopathy-predominant or a thrombotic microangiopathy-predominant phenotype.
KIDNEY INTERNATIONAL REPORTS
(2023)
Article
Hematology
Sufana Shikdar, Azra Borogovac, Elabdallah Mohamad, Mohamad Khawandanah
Summary: This case may demonstrate the beneficial effects of ravulizumab on complement mediated inflammatory damage linked with COVID19 infection especially in PNH patients.
THROMBOSIS JOURNAL
(2021)
Editorial Material
Biotechnology & Applied Microbiology
Robert T. Lawrence, Peter D. Senter
Summary: Logic-gated monoclonal antibodies have been developed to enhance safety and effectiveness.
NATURE BIOTECHNOLOGY
(2022)
Article
Immunology
Hui Yang, Xingxing Chai, Yuemin Gong, Xinyu Zhang, Lingling Wang, Xin Zhou, Xiaoyu Chen, Jinge Xu, Dan Xu, Guangsheng He, Jianyong Li
Summary: This study collected demographic and clinical data of 20 PNH patients with SARS-CoV-2 Omicron infection. The results showed that these patients had high disease activity, with LDH levels exceeding previous reports, and elevated D-dimer levels in 10 patients. Two patients developed mild pulmonary artery hypertension, while glomerular filtration rate decreased in 5 patients and 1 patient developed acute renal failure requiring hemodialysis. Anemia and hemolysis improved in 5 patients treated with eculizumab. Therefore, hemolytic exacerbation of PNH with COVID-19 is severe and eculizumab may be an effective treatment.
IMMUNITY INFLAMMATION AND DISEASE
(2023)
Article
Education, Scientific Disciplines
Daria Babushok
Summary: Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired blood disease caused by somatic mutations in the PIGA gene, manifesting as either hemolysis or bone marrow failure. Clinical presentations are influenced by clone size, and only classical PNH patients benefit from complement inhibitors.
HEMATOLOGY-AMERICAN SOCIETY OF HEMATOLOGY EDUCATION PROGRAM
(2021)
Article
Immunology
Sutian Jiang, Yifan Zhang, Xiaojing Zhang, Bing Lu, Pingping Sun, Qianqian Wu, Xuzhong Ding, Jianfei Huang
Summary: Recognizing the crucial role of the immune microenvironment in tumor progression is key to identifying immunotherapeutic targets. This study explores the potential impact of GARP on regulating survival and tumor-infiltrating T-cells in gastric cancer. The upregulation of GARP was associated with increased Treg and CD4+ T cell infiltration, as well as positive relationships with immune checkpoint expression in the TME of gastric cancer. Cox regression analysis highlighted the predictive value of GARP or GARP+CD4+ T cell on overall survival probability.
FRONTIERS IN IMMUNOLOGY
(2021)
Review
Medicine, Research & Experimental
Seppo Meri, Donald Bunjes, Roxanne Cofiell, Sonata Jodele
Summary: Hematopoietic stem cell transplantation-associated thrombotic microangiopathy (HSCT-TMA) is a common complication following transplantation, and the complement system plays a crucial role in its pathogenesis. Various risk factors and diagnostic methods have been identified, but the condition is often overshadowed by other symptoms. Targeted complement therapies offer promise in improving patient outcomes.
ADVANCES IN THERAPY
(2022)
Article
Immunology
Sutian Jiang, Xuzhong Ding, Qianqian Wu, Tong Cheng, Manyu Xu, Jianfei Huang
Summary: A tumor-infiltrating immune cells (TIICs) model was developed for risk stratification and prognosis prediction in gastric cancer. The model was validated for its predictive power in immunotherapy and showed potential for immune cell targeting strategies. The study highlights the importance of TIICs in predicting survival outcomes and guiding treatment in gastric cancer.
FRONTIERS IN IMMUNOLOGY
(2022)
Review
Hematology
Corrado Girmenia, Wilma Barcellini, Paola Bianchi, Eros Di Bona, Anna Paola Iori, Rosario Notaro, Simona Sica, Alberto Zanella, Antonio De Vivo, Giovanni Barosi, Antonio Risitano
Summary: This article presents the results of group discussion among experts on unmet clinical needs in managing infectious risk associated with eculizumab or new terminal complement inhibitors (CIs) in paroxysmal nocturnal hemoglobinuria (PNH). The selected clinically relevant needs were optimizing infection prevention measures, developing non-pharmacological infectious risk-mitigation strategies, and improving disease management during infectious complications. Consensus opinions and proposals for advancing clinical practice were provided for each issue. The aim is to improve CI therapy and guide future studies in this field.
Review
Hematology
Shruti Chaturvedi, Evan M. Braunstein, Robert A. Brodsky
Summary: Antiphospholipid syndrome (APS) is an acquired thromboinflammatory disorder characterized by the presence of antiphospholipid antibodies and an increased risk of venous or arterial thrombosis, with a severe form known as catastrophic APS (CAPS). Complement activation via antiphospholipid antibodies can cause cellular injury and promote coagulation, leading to a potential increase in risk for development of severe thrombotic APS and CAPS in a subset of patients with germline variants in genes crucial for complement regulation. Complement inhibition may be a promising therapy to reduce morbidity and mortality in these patients.
JOURNAL OF THROMBOSIS AND HAEMOSTASIS
(2021)
Review
Immunology
Zhaoyuan Chen, Hao Zhang, Mengdi Qu, Ke Nan, Hanzhong Cao, Juan P. Cata, Wankun Chen, Changhong Miao
Summary: Patients with sepsis often have coagulation dysfunction and the formation of thrombus, with neutrophil extracellular traps (NETs) playing a key role in sepsis. Clinical evaluation of NETs function could be a valuable biomarker for early diagnosis of sepsis. The interaction of NETs with platelets, complement, and endothelium mediates the formation of immunothrombosis in sepsis, and inhibiting NETs formation is considered a potential treatment for sepsis.
FRONTIERS IN CELLULAR AND INFECTION MICROBIOLOGY
(2021)
Article
Hematology
Melania Carlisi, Salvatrice Mancuso, Gregorio Caimi, Sergio Siragusa
Summary: The complement system is a crucial component of the innate immune defense, but if overly activated, it may harm organs and tissues. Regulatory proteins such as CD55 and CD59 play important roles in modulating the complement system to prevent excessive activation.
CLINICAL HEMORHEOLOGY AND MICROCIRCULATION
(2021)
Article
Hematology
E. O. Artemenko, S. I. Obydennyi, K. S. Troyanova, G. A. Novichkova, D. Y. Nechipurenko, M. A. Panteleev
Summary: In this study, we investigated the adhesive properties of circulating plasma-derived and platelet-derived microvesicles (MVs) and their potential role in thrombus growth. The results showed that both types of MVs weakly bound fibrinogen but did not stably adhere to immobilized fibrinogen. Moreover, the presence of these MVs did not significantly affect thrombus formation. Therefore, MVs present in the plasma of healthy individuals are unlikely to directly contribute to thrombus formation under arterial flow conditions.
THROMBOSIS RESEARCH
(2024)
Letter
Hematology
Giuseppe Lippi, Camilla Mattiuzzi, Emmanuel J. Favaloro
THROMBOSIS RESEARCH
(2024)
Article
Hematology
Marium Malik, Maha Al-Ghafry, Abraham Haimed, Julia Su, Maribel Lema, Linda Shore-Lessersson, Suchitra S. Acharya
Summary: This study analyzed and evaluated leukemia-associated coagulopathy in newly diagnosed pediatric leukemia patients using CCTs and ROTEM. The results showed that fibrinogen and platelets played a significant role in causing coagulopathies in this population. This suggests the utility of using CCTs and ROTEM in the pediatric population to evaluate hemostatic function and guide blood product replacement.
THROMBOSIS RESEARCH
(2024)
Review
Hematology
Mohammad M. Elahi, Alexandra N. Witt, Edward L. G. Pryzdial, Paul B. McBeth
Summary: Thrombotic disease may be underdiagnosed in microgravity conditions, and the underlying factors are still poorly understood. A case of internal jugular vein thrombosis was recently diagnosed in a low-risk female astronaut, and six out of ten additional crew members showed risk factors for jugular venous flow. Observations in space and studies on Earth suggest that microgravity affects blood flow, coagulation, and vascular function.
THROMBOSIS RESEARCH
(2024)
Review
Hematology
Stian Ingemann-Molden, Christina Krogner Caspersen, Nanna Rolving, Anette Arbjerg Hojen, Frederikus A. Klok, Erik L. Grove, Barbara Cristina Brocki, Jane Andreasen
Summary: This study found that no single PROM covered all aspects considered important by PE patients. PROMs recommended in the ICHOM core set covered most aspects, but worrisome thoughts, hypervigilance around symptoms, and uncertainty of illness were not covered.
THROMBOSIS RESEARCH
(2024)
Article
Hematology
Haomin Yan, Tsutomu Sasaki, Yasufumi Gon, Kumiko Nishiyama, Hideaki Kanki, Hideki Mochizuki
Summary: The incidence of cancer-associated stroke has increased with the prolonged survival times of cancer patients. In this study, the researchers focused on the driver gene KRAS and found that KRAS mutations exacerbated neurological deficits after stroke and promoted tumor growth. They also identified the upregulation of pro-inflammatory responses and the promotion of STAT3 phosphorylation in mice with KRAS mutations. Additionally, the researchers found that colorectal cancer patients with stroke onset within 3 months after cancer diagnosis had a worse prognosis.
THROMBOSIS RESEARCH
(2024)
Letter
Hematology
Tomoki Togashi, Satomi Nagaya, Makiko Meguro-Horike, Haruto Matsumoto, Yuta Imai, Koichi Yamaguchi, Yoshinari Fujii, Haruka Moriya, Yuika Kikuchi, Ibuki Yasuda, Shin-ichi Horike, Eriko Morishita
THROMBOSIS RESEARCH
(2024)
Article
Hematology
Marta Najarro, Winnifer Briceno, Carmen Rodriguez, Alfonso Muriel, Sara Gonzalez, Ana Castillo, Ignacio Jara, Parth Rali, Catalin Toma, Behnood Bikdeli, David Jimenez
Summary: The CPES score is a useful tool in identifying the risk of hemodynamic deterioration in normotensive patients with acute PE, with acceptable C-statistic, excellent specificity, and low positive predictive value.
THROMBOSIS RESEARCH
(2024)
Article
Hematology
Lei Liu, Huifang Xu, Jian Wang, Haiyan Wang, Saisai Ren, Qian Huang, Mingyan Zhang, Hui Zhou, Chunyan Yang, Lu Jia, Yu Huang, Hao Zhang, Yanling Tao, Ying Li, Yanan Min
Summary: This study investigated the gut microbiota metabolites and cytokines, chemokines, and growth factors in patients with ITP and found their association with treatment responsiveness. The findings suggest that imbalanced secretion of cyto(chemo)kins/growth factors and inadequate levels of TMAO are characteristic of patients with ITP.
THROMBOSIS RESEARCH
(2024)
Article
Hematology
Roxanne Mcknight, Mohammad Salameh, Lee Ann Jones, Greta Anton, Lindsay Carter
Summary: This study evaluated the safety of fixed-dose heparin titration boluses in underweight patients with VTE. The results showed that although underweight patients required more frequent holding of heparin, there was no significant increase in bleeding risk.
THROMBOSIS RESEARCH
(2024)