Review
Medicine, General & Internal
Juan Carlos Garcia-Pagan, Dominique-Charles Valla
Summary: Primary Budd-Chiari Syndrome (BCS) is a life-threatening disease caused by obstruction of the hepatic venous outflow tract due to thrombosis or a primary venous wall disease. Recent advancements have enhanced our knowledge of BCS and its outcomes.
NEW ENGLAND JOURNAL OF MEDICINE
(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)
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
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)
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
Medicine, General & Internal
Runsen Du, Lihua Zheng, Peng Liu, Yaheng Zhao, Yan Yang, Lei Zhang, Zengren Zhao
Summary: We report a case of a female who was cured of hemorrhagic esophageal varices caused by paroxysmal nocturnal hemoglobinuria (PNH) through transjugular intrahepatic portosystemic shunt (TIPS) treatment. PNH complicated by portal vein and visceral veins thrombosis without hepatic veins is extremely rare, and it can be easily misdiagnosed due to lack of awareness. Hemorrhagic esophageal varices due to PNH with portal vein thrombosis have only been reported in one previous case in 1974, and this report presents the second case.
FRONTIERS IN MEDICINE
(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
Aurelie Plessier, Marina Esposito-Farese, Anna Baiges, Akash Shukla, Juan Carlos Garcia Pagan, Emmanuelle De Raucourt, Isabelle Ollivier-Hourmand, Jean-Paul Cervoni, Victor De Ledinghen, Zoubida Tazi, Jean-Baptiste Nousbaum, Rene Bun, Christophe Bureau, Christine Silvain, Olivier Tournilhac, Mathieu Gerfaud-Valentin, Francois Durand, Odile Goria, Luis Tellez, Agustin Albillos, Stefania Gioia, Oliviero Riggio, Andrea De Gottardi, Audrey Payance, Pierre-Emmanuel Rautou, Louis Terriou, Aude Charbonnier, Laure Elkrief, Regis Peffault de la Tour, Dominique-Charles Valla, Nathalie Gault, Flore Sicre de Fontbrune
Summary: The study aimed to assess the impact of eculizumab on the outcome of patients with paroxysmal nocturnal hemoglobinuria (PNH) and vascular liver disease (VLD). The results showed that eculizumab was significantly associated with lower mortality and less thrombotic recurrence in these patients, while other secondary outcomes did not reach statistical significance.
AMERICAN JOURNAL OF HEMATOLOGY
(2022)
Article
Medicine, General & Internal
Juraj Sokol, Frantisek Nehaj, Michal Mokan, Lenka Lisa, Jan Stasko
Summary: Paroxysmal nocturnal hemoglobinuria (PNH) patients with COVID-19 infection showed severe symptoms, but were successfully treated with anticoagulants, steroids, and cefuroxime, and discharged after symptom relief. Research indicated that COVID-19 infection activates the complement system, leading to acute lung injury and increased prothrombotic state, with complement inhibitors showing potential as a therapeutic approach.
Article
Hematology
Deniz Goren Sahin, Olga Meltem Akay, Muzaffer Keklik, Vahap Okan, Abdullah Karakus, Cengiz Demir, Mehmet Ali Erkurt, Kadir Ilkkilic, Rahsan Yildirim, Gulsum Akgun Cagliyan, Salih Aksu, Mehmet Hilmi Dogu, Mehmet Sinan Dal, Volkan Karakus, Ali Ihsan Gemici, Hatice Terzi, Engin Kelkitli, Serdar Sivgin, Ali Unal, Mehmet Yilmaz, Orhan Ayyildiz, Serdal Korkmaz, Bulent Eser, Fevzi Altuntas
Summary: This study aimed to collect PNH patient data from hematology centers across Turkey to identify clinical features and management. After evaluating patients from 19 different institutions, it was found that fatigue and abdominal pain were the most frequent presenting symptoms. This study provided valuable information for understanding the disease and differences between patients in Turkey and existing literature.
ANNALS OF HEMATOLOGY
(2021)
Article
Obstetrics & Gynecology
H. M. G. Wiegers, E. N. Hamulyak, S. E. Damhuis, J. R. van Duuren, S. Darwish Murad, L. J. J. Scheres, S. J. Gordijn, J. Leentjens, J. J. Duvekot, M. N. Lauw, B. A. Hutten, S. Middeldorp, W. Ganzevoort
Summary: Women previously diagnosed with Budd-Chiari syndrome and/or portal vein thrombosis, with or without concomitant portal hypertension, can achieve relatively good pregnancy outcomes, with a high rate of term live births and lower risk of maternal and neonatal complications. Therefore, these conditions should not be considered as absolute contraindications for pregnancy, and individualized counseling and multidisciplinary pregnancy surveillance are essential.
BJOG-AN INTERNATIONAL JOURNAL OF OBSTETRICS AND GYNAECOLOGY
(2022)
Review
Public, Environmental & Occupational Health
Bei Zhang, Ran Chu, Changzhen Huang, Xiao Song, Jianye Wang, Liang Li, Yintao Xu, Yuyan Ma
Summary: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hemolytic disease with thrombosis as a major cause of death. Managing PNH in pregnant patients is challenging and there is a lack of consensus on treatment. This article reviews 32 studies on PNH in pregnancy, providing guidance for obstetricians and supporting the management of PNH patients. Eculizumab has become the primary treatment choice for PNH during pregnancy, effectively controlling intravascular hemolysis and minimizing risks to the mother and fetus.
JOURNAL OF WOMENS HEALTH
(2023)
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)
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)
Article
Radiology, Nuclear Medicine & Medical Imaging
Lu-Lu Lv, Li-Li Zhu, Gao-Hong Chen, Peng Xu, Kai Xu
Summary: The study showed that recanalization of hepatic vein-type Budd-Chiari syndrome via accessory hepatic vein achieved similar technical and clinical success rates compared to main hepatic vein recanalization, but had higher long-term patency rates.
ABDOMINAL RADIOLOGY
(2021)
Article
Gastroenterology & Hepatology
Francisco Javier Perez-Hernandez, Dalia Elena Morales-Arraez, Carla Amaral-Gonzalez, Yanira Gonzalez-Mendez, Juan Adolfo Ortega-Sanchez, Raquel de la Barreda Heuser, Inmaculada Abreu Alonso, Laura Ramos Lopez, Onofre Alarcon-Fernandez, Marta Carrillo-Palau, Manuel Hernandez-Guerra
Summary: This study examines the characteristics and predictive factors of continuity of care in a care circuit between primary and specialized care based on electronic consultation (EC). It found that the communication pathway is effective in preventing significant losses of viraemic patients. However, the referral rate is very low, high in late-stage diagnoses, and low in new diagnoses, indicating a need for early detection strategies in primary care for HCV infection.
GASTROENTEROLOGIA Y HEPATOLOGIA
(2023)
Article
Medicine, General & Internal
Lorenzo Cantarelli, Marta Carrillo Palau, Manuel Hernandez Guerra, Gloria Julia Nazco Casariego, Fernando Gutierrez Nicolas
Summary: The aim of this study was to determine the predictive potential of monitoring infliximab plasma levels for sustained clinical response and evaluate its usefulness in improving treatment efficacy and symptom control in patients with inflammatory bowel disease. The results confirmed the predictive potential of monitoring infliximab plasma levels as a strategy to evaluate sustained long-term clinical response. The study highlights the importance of introducing this monitoring into routine clinical practice to identify non-responders, optimize treatment, prevent relapse, and improve long-term therapy maintenance.
Article
Gastroenterology & Hepatology
Javier Crespo, Joaquin Cabezas, Antonio Aguilera, Marina Berenguer, Maria Buti, Xavier Forns, Federico Garcia, Javier Garcia-Samaniego, Manuel Hernandez-Guerra, Francisco Jorquera, Jeffrey Lazarus, Sabela Lens, Elisa Martro, Juan Antonio Pineda, Martin Prieto, Francisco Rodriguez-Frias, Manuel Rodriguez, Miguel Angel Serra, Juan Turnes, Raquel Dominguez-Hernandez, Miguel Angel Casado, Jose Luis Calleja
Summary: The Spanish Society of Digestive Pathology (SEPD), the Spanish Association for the Study of the Liver (AEEH), the Spanish Society of Infections and Clinical Microbiology (SEIMC) and its Viral Hepatitis Study Group (GEHEP), with the endorsement of the Alliance for the Elimination of Viral Hepatitis in Spain (AEHVE), have developed a document for comprehensive diagnosis of viral hepatitis (B, C, and D) from a single blood sample. They propose an algorithm to analyze the rest of the viruses, including viral load, when a positive result for viral hepatitis or HIV is found in the blood sample. They also recommend ruling out previous hepatitis A virus infection and determining the HIV serology. Automated alerts and epidemiological monitoring should be initiated in case of a positive result for any of the viruses.
GASTROENTEROLOGIA Y HEPATOLOGIA
(2023)
Article
Gastroenterology & Hepatology
Marika Rudler, Virginia Hernandez-Gea, Bogdan Dumitru Procopet, Alvaro Giraldez, Lucio Amitrano, Candid Villanueva, Luis Ibanez, Gilberto Silva-Junior, Joan Genesca, Christophe Bureau, Jonel Trebicka, Rafael Banares, Aleksander Krag, Elba Llop, Wim Laleman, Jose Maria Palazon, Jose Castellote, Susana Rodrigues, Lise Lotte Gluud, Carlos Noronha Ferreira, Nouria Canete, Manuel Rodriguez, Arnulf Ferlitsch, Jose Luis Mundi, Henning Gronbaek, Manuel Hernandez-Guerra, Romano Sassatelli, Alessandra Dell'era, Marco Senzolo, Juan G. Abraldes, Manuel Romero-Gomez, Alexander Zipprich, Meritxell Casas, Helena Masnou, Helene Larrue, Massimo Primignani, Frederik Nevens, Jose Luis Calleja, Remy Schwarzer, Christian Jansen, Marie-Angele Robic, Irene Conejo, Javier Martinez Gonzalez, Maria Vega Catalina, Agustin Albillos, Edilmar Alvarado, Maria Anna Guardascione, Maxime Mallet, Simona Tripon, Georgina Casanovas, Jaume Bosch, Juan-Carlos Garcia-Pagan, Dominique Thabut
Summary: pTIPS is more effective in improving survival compared to endoscopic treatment for high-risk patients with cirrhosis and variceal bleeding. HE at admission does not affect the efficacy of pTIPS.
Article
Gastroenterology & Hepatology
Anna Baiges, Bogdan Procopet, Gilberto Silva-Junior, Elba Llop, Luis Tellez, Anna Darnell, Angeles Garcia-Criado, Fanny Turon, Oana Nicoara-Farcau, Carlos Gonzalez-Alayon, Helene Larrue, Marta Magaz, Pol Olivas, Valeria Perez-Campuzano, Jose Luis Calleja, Agustin Albillos, Juan Carlos Reverter, Christophe Bureau, Jaime Bosch, Virginia Hernandez-Gea, Juan Carlos Garcia-Pagan
Summary: The aim of this study was to describe the incidence of recurrent thrombosis (RT) in patients with non-cirrhotic splanchnic vein thrombosis (NC-SVT) and identify its risk factors. The study found that the risk of RT in NC-SVT patients without an indication for long-term anticoagulation is not low. Furthermore, factor VIII level may be a key indicator for predicting RT.
JOURNAL OF HEPATOLOGY
(2023)
Letter
Gastroenterology & Hepatology
Marika Rudler, Virginia Hernandez-Gea, Dominique Thabut, Juan-Carlos Garcia-Pagan
ALIMENTARY PHARMACOLOGY & THERAPEUTICS
(2023)
Letter
Gastroenterology & Hepatology
Anna Baiges, Joan Carles Reverter, Joan Carles Garcia Pagan
JOURNAL OF HEPATOLOGY
(2023)
Review
Endocrinology & Metabolism
Wim Laleman, Michael Praktiknjo, Mette Munk Lauridsen, Lawrence Bonne, Emma Vanderschueren, Chris Verslype, Aleksander Krag, Jonel Trebicka, Geert Maleux
Summary: Spontaneous portosystemic shunts are often overlooked as a cause of hepatic encephalopathy associated with cirrhosis. They are considered as radiological biomarkers of clinically significant portal hypertension and have a clinical impact on complications related to portal hypertension. Interventional radiology embolization is a direct method to target these shunts.
METABOLIC BRAIN DISEASE
(2023)
Article
Hematology
Marieke J. A. Verhagen, Waander L. van Heerde, Johanna G. van der Bom, Erik A. M. Beckers, Nicole M. A. Blijlevens, Michiel Coppens, Samantha C. Gouw, Joop H. Jansen, Frank W. G. Leebeek, Lize F. D. van Vulpen, Danielle Meijer, Saskia E. M. Schols
Summary: The association between clinical bleeding phenotype and thrombin generation and plasmin generation profiles in patients with hemophilia was investigated. The study found that thrombin generation and plasmin generation parameters differed between patients with hemophilia and healthy individuals, and a decreased thrombin generation profile was associated with a severe clinical bleeding phenotype.
RESEARCH AND PRACTICE IN THROMBOSIS AND HAEMOSTASIS
(2023)
Article
Hematology
Eleonora Camilleri, Nienke van Rein, Bart J. M. van Vlijmen, Joseph S. Biedermann, Marieke J. H. A. Kruip, Frank W. Leebeek, Felix J. van der Meer, Christa M. Cobbaert, Suzanne C. Cannegieter, Willem M. Lijfering
Summary: The study found that rosuvastatin reduces coagulation factor levels through mechanisms related to the synthesis or regulation pathways of apolipoproteins.
RESEARCH AND PRACTICE IN THROMBOSIS AND HAEMOSTASIS
(2023)
Article
Gastroenterology & Hepatology
Ian Rowe, Candid E. Villanueva, Jessica Shearer, Ferran Torres, Agustin Albillos, Joan C. Genesca, Joan Garcia-Pagan, Dhiraj C. Tripathi, Peter Hayes, Jaume G. Bosch, Juan Abraldes
Summary: This study conducted a Bayesian reanalysis of PREDESCI and found that beta-blocker treatment is highly likely to prevent decompensation and provides a substantial gain in decompensation-free life years at the population level.
Article
Virology
Felix Lehmann, Heiko Slanina, Martin Roderfeld, Elke Roeb, Jonel Trebicka, John Ziebuhr, Wolfram H. Gerlich, Christian G. Schuettler, Bernhard Schlevogt, Dieter Glebe
Summary: Chronic hepatitis B virus (HBV) infection is a global health threat. Mutations in the surface antigen of HBV (HBsAg) may alter its antigenicity, infectivity, and transmissibility. A patient positive for HBV DNA and detectable but low-level HBsAg in parallel with anti-HBs suggested the presence of immune and/or diagnostic escape variants. Secreted HBsAg was also subjected to four widely used, state-of-the-art diagnostic assays, which all failed to detect the hyperglycosylated insertion variant. Additionally, the recognition of mutant HBsAg by vaccine- and natural infection-induced anti-HBs antibodies was severely impaired.
Article
Hematology
Martijn R. Brands, Lotte Haverman, Jelmer J. Muis, Mariette H. E. Driessens, Felix J. M. van der Meer, Geertje Goedhart, Stephan Meijer, Marianne de Jong, Johanna G. van der Bom, Marjon H. Cnossen, Karin Fijnvandraat, Samantha C. Gouw
Summary: This study examined the satisfaction of hemophilia patients in the Netherlands with their care and found high levels of satisfaction, but also identified areas for improvement such as better information provision and care coordination, with the potential use of digital health tools. These findings are important for further enhancing the care of hemophilia patients.
RESEARCH AND PRACTICE IN THROMBOSIS AND HAEMOSTASIS
(2023)
Article
Pharmacology & Pharmacy
Sjoerd F. Koopman, Tine M. H. J. Goedhart, Laura H. Bukkems, Trevor M. Mulders, Frank W. G. Leebeek, Karin Fijnvandraat, Michiel Coppens, Mary Mathias, Peter W. Collins, R. Campbell Tait, Catherine N. Bagot, Nicola Curry, Jeanette Payne, Pratima Chowdary, Marjon H. Cnossen, Ron A. A. Mathot
Summary: This study evaluated the predictive performance of a population pharmacokinetic (PK) model for a recombinant factor IX Fc fusion protein (rFIX-Fc) and developed a new model using real-world data. The published model significantly underpredicted FIX activity levels, while the new model better described rFIX-Fc PK, especially for children aged <12 years.
BRITISH JOURNAL OF CLINICAL PHARMACOLOGY
(2023)
Article
Gastroenterology & Hepatology
Genis Camprecios, Aina Anton, Anna Oncins, Carla Montironi, Maria Ruart, Rosa Montanes, Hector Garcia-Caldero, Joan Carles Garcia-Pagan, Virginia Hernandez-Gea
Summary: Liver sinusoidal endothelial cells (LSEC) are key elements in regulating the liver response to injury and regeneration. Endothelial autophagy plays a crucial role in protecting cells from injury-induced oxidative stress and fibrosis. However, this study found that endothelial autophagy is not essential for liver regeneration after partial hepatectomy (PHx).
LIVER INTERNATIONAL
(2023)
