Review
Health Care Sciences & Services
Anna Burguin, Caroline Diorio, Francine Durocher
Summary: Breast cancer, the most frequent cancer diagnosed in women worldwide, can be classified into four molecular subtypes for personalized treatment targeting receptors, but resistance and adverse effects remain challenges. Treatments for TNBC and other subgroups are still not standardized, with emerging approaches like immunotherapy and targeting other metabolic pathways gaining attention.
JOURNAL OF PERSONALIZED MEDICINE
(2021)
Article
Biochemistry & Molecular Biology
Gianfranco Piccirillo, Federica Moscucci, Andrea Corrao, Myriam Carnovale, Ilaria Di Diego, Ilaria Lospinuso, Cristina Caltabiano, Martina Mezzadri, Pietro Rossi, Damiano Magri
Summary: This study used bio-impedance and ECG data to compare HFrEF and HFpEF patients, and analyzed responders and non-responders to optimal medical therapy. The study found that these non-invasive parameters were able to highlight the differences between different types of heart failure patients and their response to medication.
Review
Biotechnology & Applied Microbiology
Kenneth Cornetta, Martin Bonamino, Johnny Mahlangu, Federico Mingozzi, Savita Rangarajan, Jayandharan Rao
Summary: This article reviews the current status of gene and cell therapies in low- and middle-income countries and discusses the state of product development in Brazil, South Africa, and India, as well as the need and barriers to access.
Review
Chemistry, Analytical
Francesca De Tommasi, Carlo Massaroni, Rosario Francesco Grasso, Massimiliano Carassiti, Emiliano Schena
Summary: Pain relief is crucial for patients with bone metastases and osteoid osteoma, and new hyperthermia treatments are emerging to alleviate the associated pain. Temperature monitoring during hyperthermia treatments is essential for improving clinical outcomes, especially in bone tumors. Research is ongoing to explore the use of different thermometric techniques in this scenario.
Review
Biochemistry & Molecular Biology
Claire Dunois
Summary: Direct oral anticoagulants (DOACs) provide a safe and effective alternative to previous anticoagulant therapies by directly, selectively, and reversibly inhibiting factors IIa or Xa. They have predictable pharmacokinetic and pharmacodynamic responses at a fixed dose, eliminating the need for monitoring. Testing may be helpful for patient management in specific clinical situations and for particular patient populations.
Review
Biochemistry & Molecular Biology
Lucia Cernakova, Maryam Roudbary, Susana Bras, Silva Tafaj, Celia F. Rodrigues
Summary: Candida auris is a novel and major fungal pathogen that has caused outbreaks in the last decade. Due to its high rate of multidrug resistance, difficulty in identification, and ability to form biofilms, treating C. auris infections has become highly challenging. Further research is needed to explore identification, treatment, and drug development for C. auris infections.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2021)
Review
Hematology
Marieke J. A. Verhagen, Lars L. F. G. Valke, Saskia E. M. Schols
Summary: Severe hemophilia A patients are at increased risk of bleeding, but using FVIII activity level to monitor treatment may not accurately predict the bleeding risk. Thrombin generation assays offer a better tool for evaluating treatment and monitoring bypassing agents and non-factor replacement therapy.
JOURNAL OF THROMBOSIS AND HAEMOSTASIS
(2022)
Article
Biochemistry & Molecular Biology
Carolina Seybert, Goncalo Cotovio, Luis Madeira, Miguel Ricou, Ana Matos Pires, Albino J. Oliveira-Maia
Summary: Enhanced informed consent procedures are necessary for patients undergoing psychedelic treatments, like psilocybin and MDMA, as they can induce altered states of consciousness and susceptibility to suggestion.
Review
Biochemistry & Molecular Biology
Armando Tripodi
Summary: Lupus anticoagulant (LA) is a crucial laboratory parameter for the diagnosis and management of antiphospholipid syndrome (APS). Proper detection of LA is essential for long-term treatment of patients with thrombotic events, requiring adherence to guidelines and participation in quality assessment schemes.
Article
Construction & Building Technology
Silje Asphaug, Ingrid Hjermann, Berit Time, Tore Kvande
Summary: Basements used for habitation pose challenges in terms of moisture safety design, as they have limited ability for outward drying. The effect of using exterior vapour-permeable thermal insulation combined with a dimpled membrane is not well documented. A laboratory experiment was conducted to investigate the outward drying of concrete walls. The results suggest that the ability of concrete to transfer moisture to the drying surface limits outward drying.
BUILDING AND ENVIRONMENT
(2022)
Article
Immunology
Hiroki Hosoi, Misato Tane, Hideki Kosako, Masaki Ibe, Masahiro Takeyama, Shogo Murata, Toshiki Mushino, Takashi Sonoki
Summary: A case of acquired hemophilia A (AHA) in a young patient after mRNA COVID-19 vaccine administration is reported. The patient's condition improved rapidly and the factor VIII inhibitor levels decreased significantly. This case suggests that AHA occurring after COVID-19 vaccination in young patients may resolve spontaneously and exhibit a more rapid decline in FVIII inhibitors compared to idiopathic AHA.
JOURNAL OF AUTOIMMUNITY
(2022)
Article
Hematology
Jan Astermark, Cihan Ay, Manuela Carvalho, Roseline D'Oiron, Philippe De Moerloose, Gerard Dolan, Pierre Fontana, Cedric Hermans, Pal Andre Holme, Olga Katsarou, Gili Kenet, Robert Klamroth, Maria Elisa Mancuso, Natascha Marquardt, Ramiro Nunez, Ingrid Pabinger, Robert Tait, Paul van der Valk
Summary: An investigation into late-onset inhibitors in patients with hemophilia A and B aged >= 40 years found that prophylaxis may be beneficial in older patients with severe HA to maintain tolerance at a higher age. The study did not identify a second peak of inhibitor development in older patients with hemophilia.
THROMBOSIS AND HAEMOSTASIS
(2022)
Article
Biochemistry & Molecular Biology
Junzheng Wu, Hang Zhang, Tong Lian, Yaling Ding, Chunlei Song, Dekuan Li, Liheng Wu, Tao Lei, Hong Liang
Summary: A novel B-domain-deleted recombinant FVIII was developed in this study without the use of animal or human serum-derived components. rFVIII promoted the generation of activated factor X and downstream thrombin, and exhibited ideal binding affinity to human von Willebrand factor. In vitro and in vivo experiments demonstrated the satisfactory efficacy and potency of rFVIII, suggesting its potential as an effective treatment strategy for FVIII deficiency.
BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS
(2023)
Review
Multidisciplinary Sciences
Miroslav Kocifaj, Stefan Wallner, John C. Barentine
Summary: Understanding and mitigating light pollution requires measuring and monitoring artificial light at night. This review explores various techniques for measuring artificial light, including ground measurements and satellite remote sensing. Spectroscopic differences and atmospheric variability pose challenges in interpreting data. Theoretical models provide additional information for calibration and interpretation of results. Shortcomings and challenges in current approaches are identified, and suggestions for improvement are proposed.
Letter
Genetics & Heredity
Chatphatai Moonla, Darintr Sosothikul, Bunchoo Pongtanakul, Bundarika Suwanawiboon, Chanchai Traivaree, Rungrote Natesirinilkul, Nongnuch Sirachainan, Pantep Angchaisuksiri
Summary: Hemophilia is an inherited bleeding disorder caused by deficiency of specific coagulation factors. Thailand has a low number of registered hemophiliacs due to underdiagnosis, which is a major concern in Asian countries. Despite challenges, Thailand has made substantial progress in providing hemophilia care through knowledge acquisition, collaboration, and overcoming infrastructure and cost barriers. The authors summarize the practices and challenges of hemophilia management in Thailand in this letter-to-the-editor.
ORPHANET JOURNAL OF RARE DISEASES
(2023)
Article
Biochemistry & Molecular Biology
Elena Barbon, Charlotte Kawecki, Solenne Marmier, Aboud Sakkal, Fanny Collaud, Severine Charles, Giuseppe Ronzitti, Caterina Casari, Olivier D. Christophe, Cecile V. Denis, Peter J. Lenting, Federico Mingozzi
Summary: Von Willebrand disease (VWD), the most common inherited bleeding disorder, has limited efficacy with current liver-directed gene therapy. This study developed a gene therapy strategy using an optimized AAV vector and endothelial-targeting peptide to deliver the VWF gene into endothelial cells, achieving long-term expression.
Article
Hematology
Cecile Denis, Sophie Susen, Peter J. Lenting
Summary: Von Willebrand disease (VWD) is characterized by its heterogeneous clinical manifestation, complicating diagnosis and treatment. Despite the current effective management, studies show a higher burden on patients' quality of life, especially for women. To improve patient care, potential innovative strategies like gene therapy, protein-based approaches, and antibody-based options are proposed for future VWD treatments.
Article
Cardiac & Cardiovascular Systems
Jeremy Lagrange, Morel E. Worou, Jean-Baptiste Michel, Alexandre Raoul, Melusine Didelot, Vincent Muczynski, Paulette Legendre, Francois Plenat, Guillaume Gauchotte, Marc-Damien Lourenco-Rodrigues, Olivier D. Christophe, Peter J. Lenting, Patrick Lacolley, Cecile Denis, Veronique Regnault
Summary: VWF induces VSMC proliferation through A2 domain binding to LRP4 receptor and integrin alpha(v)beta(3) signaling. These findings provide new insights into the mechanisms that drive physiological repair and pathological hyperplasia of the arterial vessel wall.
CARDIOVASCULAR RESEARCH
(2022)
Editorial Material
Hematology
Cecile V. Denis, Peter J. Lenting
Editorial Material
Hematology
Cecile V. Denis, Peter J. Lenting, Denis Wahl
JOURNAL OF THROMBOSIS AND HAEMOSTASIS
(2022)
Article
Hematology
Nikolett Wohner, Silvie Sebastian, Vincent Muczynski, Dana Huskens, Bas de Laat, Philip G. de Groot, Peter J. Lenting
Summary: The study reveals that VWF strings secreted from endothelial cells are only partially covered with platelets, and osteoprotegerin plays a role in regulating platelet adhesion to VWF. This regulatory mechanism may be more relevant under inflammatory conditions.
JOURNAL OF THROMBOSIS AND HAEMOSTASIS
(2022)
Article
Hematology
Caterina Casari, Remi Favier, Paulette Legendre, Alexandre Kauskot, Frederic Adam, Veronique Picard, Peter J. Lenting, Cecile Denis, Valerie Proulle
Summary: This report describes the successful treatment of splenic injury in a patient with p.V1316M-associated VWD2B and chronic thrombocytopenia using nonoperative management and a combination of VWF replacement therapy and platelet transfusions relayed by TPO-RA.
THERAPEUTIC ADVANCES IN HEMATOLOGY
(2022)
Article
Hematology
Peter J. Lenting, Claire Kizlik-Manson, Caterina Casari
Summary: Deficiency or dysfunction of von Willebrand factor (VWF) is associated with von Willebrand disease (VWD), which presents with heterogeneous clinical manifestations. Current tools to control bleeding in VWD are limited, and quality-of-life studies show significant burden in VWD patients. Therefore, there is a need for innovative treatment options.
Article
Hematology
Josepha C. Sedzro, Frederic Adam, Claire Auditeau, Elsa Bianchini, Allan De Carvalho, Ivan Peyron, Sadyo Darame, Sophie Gandrille, Stella Thomassen, Tilman M. Hackeng, Olivier D. Christophe, Peter J. Lenting, Cecile Denis, Delphine Borgel, Francois Saller
Summary: The research team generated a PS-specific sdAb (PS003) that enhances the APC-cofactor activity of PS, showing an anti-thrombotic effect in a mouse model. This innovative approach might represent a promising and safe antithrombotic strategy.
JOURNAL OF THROMBOSIS AND HAEMOSTASIS
(2022)
Article
Cardiac & Cardiovascular Systems
Eric Van Belle, Nicolas Debry, Flavien Vincent, Gregory Kuchcinski, Charlotte Cordonnier, Antoine Rauch, Emmanuel Robin, Fanny Lassalle, Francois Pontana, Cedric Delhaye, Guillaume Schurtz, Emmanuelle JeanPierre, Natacha Rousse, Caterina Casari, Hugues Spillemaeker, Sina Porouchani, Thibault Pamart, Tom Denimal, Xavier Neiger, Basile Verdier, Laurent Puy, Alessandro Cosenza, Francis Juthier, Marjorie Richardson, Martin Bretzner, Jean Dallongeville, Julien Labreuche, Mikael Mazighi, Annabelle Dupont-Prado, Bart Staels, Peter J. Lenting, Sophie Susen
Summary: This study found that one-fourth of patients undergoing TAVR had pre-existing CMBs, and one-fourth developed new CMBs after the procedure. The occurrence of new CMBs was associated with procedural factors, antithrombotic management, and persistence of acquired von Willebrand factor defect.
Article
Hematology
Claire Kizlik-Masson, Ivan Peyron, Stephane Gangnard, Gaelle Le Goff, Solen M. Lenoir, Sandra Damodaran, Marie Clavel, Stephanie Roullet, Veronique Regnault, Antoine Rauch, Flavien Vincent, Emmanuelle Jeanpierre, Annabelle Dupont, Catherine Ternisien, Thibault Donnet, Olivier D. Christophe, Eric van Belle, Cecile Denis, Caterina Casari, Sophie Susen, Peter J. Lenting
Summary: A nanobody (KB-VWF-D3.1) is identified to distinguish proteolyzed and non-proteolyzed VWF and monitor VWF degradation levels. Reduced intact-VWF levels are observed in patients with von Willebrand disease, aortic stenosis, and patients receiving mechanical circulatory support.
Meeting Abstract
Hematology
Vincent Muczynski, Angel Smith, Thibaud Sefiane, Olivier D. Christophe, Peter J. Lenting, David Granger, Kerry Chester, Amit C. Nathwani
Article
Hematology
Stephanie Roullet, Norman Luc, Julie Rayes, Jean Solarz, Dante Disharoon, Andrew Ditto, Emily Gahagan, Christa Pawlowski, Thibaud Sefiane, Frederic Adam, Caterina Casari, Olivier D. Christophe, Michael Bruckman, Peter J. Lenting, Anirban Sen Gupta, Cecile Denis
Summary: The lack of innovation in von Willebrand disease (VWD) is due to the complexity and heterogeneity of the disease and a lack of recognition of the impact of bleeding symptoms experienced by patients. This study proposes a new approach using synthetic platelet (SP) nanoparticles for the treatment of VWD-2B and severe VWD. The results show that SP can improve thrombus formation and reduce blood loss in VWD-2B and VWF-KO mice.
Article
Hematology
Marie-Daniela Dubois, Ivan Peyron, Olivier-Nicolas Pierre-Louis, Serge Pierre-Louis, Johalene Rabout, Pierre Boisseau, Annika de Jong, Sophie Susen, Jenny Goudemand, Remi Neviere, Pascal Fuseau, Olivier D. Christophe, Peter J. Lenting, Cecile Denis, Caterina Casari
Summary: This study reports two mutations in the D4 domain of VWF that induce combined qualitative and quantitative defects. One mutation leads to almost complete intracellular retention of the protein, while the other mutation results in reduced binding to collagen and platelet receptors.
RESEARCH AND PRACTICE IN THROMBOSIS AND HAEMOSTASIS
(2022)
Article
Hematology
Steven de Maat, Chantal C. Clark, Arjan D. Barendrecht, Simone Smits, Nadine D. van Kleef, Hinde El Otmani, Manon Waning, Marc van Moorsel, Michael Szardenings, Nicolas Delaroque, Kristof Vercruysse, Rolf T. Urbanus, Silvie Sebastian, Peter J. Lenting, Christoph Hagemeyer, Thomas Renne, Karen Vanhoorelbeke, Claudia Tersteeg, Coen Maas
Summary: Thrombotic microangiopathies are characterized by attacks of disseminated microvascular thrombosis. This study developed a fusion protein called Microlyse that can target and activate plasminogen on microthrombi, showing greater efficacy for the treatment of TTP compared to current methods.
