Article
Hematology
Pierre Chelle, Alfonso Iorio, Andrea N. Edginton
Summary: This study evaluates a personalized limited sampling approach (Personalized LSA) for more accurate estimation of the terminal half-life of FVIII concentrates in patients with hemophilia A using population PK modeling.
JOURNAL OF THROMBOSIS AND HAEMOSTASIS
(2022)
Review
Oncology
Cedric Hermans, Mark T. Reding, Jan Astermark, Robert Klamroth, Maria Elisa Mancuso
Summary: This review examines the methodology of clinical studies on extended-half-life (EHL) factor VIII products and their applicability for personalized prophylaxis. The similarities and differences between the studies are compared to broaden physicians' understanding of each treatment's effectiveness and assist in treatment selection for individual patients.
CRITICAL REVIEWS IN ONCOLOGY HEMATOLOGY
(2022)
Article
Business
Shweta Gaonkar, Mahka Moeen
Summary: Employee spinouts, startups founded by former employees of an industry firm, have a critical role in firm creation and knowledge transfer. This article explores the impact of prior employment in the parent firm on an employee spinout's alliance formation. The study finds that spinouts building on their parent's technologies tend to have greater partner overlap, while product market overlap leads to fewer overlapping partners.
STRATEGIC MANAGEMENT JOURNAL
(2023)
Review
Clinical Neurology
Claude Steriade, Maarten J. Titulaer, Annamaria Vezzani, Josemir W. Sander, Roland D. Thijs
Summary: Systemic autoimmune disorders are more common in epilepsy patients than in the general population, with vascular and metabolic factors playing a key role in their relationship. Immune dysfunction can affect neuronal excitability, with implications for both traditional epilepsies and autoimmune encephalitis.
Review
Clinical Neurology
Claude Steriade, Maarten J. Titulaer, Annamaria Vezzani, Josemir W. Sander, Roland D. Thijs
Summary: Systemic autoimmune disorders are more common in patients with epilepsy, especially in systemic lupus erythematosus and type 1 diabetes mellitus. Vascular and metabolic factors play important roles in the relationship between systemic autoimmune disorders and epilepsy. Dysfunction in the immune system can affect neuronal excitability in various ways.
Article
Medicine, General & Internal
Abdullah T. Almohammadi, Osman Radhwi, Hatem AlAhwal, Ahmed Barefah, Salem Bahashwan, Ibraheem M. Ashankyty, Majed Almashjari, Rawan Ayaz, Adel Al-Marzouki, Galila F. Zaher, Hend Hussain, Abeer A. Samman, Abeer Zakariyah
Summary: Hemophilia A is an X-linked recessive disorder caused by mutations in the factor VIII gene. Most affected individuals are males, inheriting the mutation from their mothers. Females can be carriers unless they inherit the mutation from both parents. Hemophilia A carriers may exhibit similar symptoms to affected patients, such as low factor VIII levels and a risk of bleeding.
CUREUS JOURNAL OF MEDICAL SCIENCE
(2023)
Article
Medicine, General & Internal
Mar Villalon-Gonzalez, Inigo Fernandez de Luco-Santamaria, Ruben Cuesta-Barriuso, Jose Antonio Lopez-Pina, Raul Perez-Llanes
Summary: This study examined the differences in muscle strength, muscle activation, and pressure pain threshold between patients with knee arthropathy and healthy individuals. The results showed that patients with hemophilia have lower muscle strength and pressure pain thresholds. Additionally, they experience greater muscle fatigue during anaerobic exercise. Therefore, strength training for patients with hemophilia should focus on activating the vastus medialis and lateralis muscles.
JOURNAL OF CLINICAL MEDICINE
(2023)
Article
Health Care Sciences & Services
Hae Kyung Kim, Carmen Peral, Dario Rubio-Rodriguez, Carlos Rubio-Terres
Summary: According to the analysis, using standard half-life rFVIII instead of extended half-life rFVIII products in Spain could lead to an average annual savings of euro11,227 per patient. Not switching treatment from SHL to EHL rFVIII would result in greater savings per patient (euro53,078). Considering the frequency of rFVIII dispensation in the US, annual savings per patient would increase to euro16,350 in Spain.
EXPERT REVIEW OF PHARMACOECONOMICS & OUTCOMES RESEARCH
(2021)
Review
Biochemistry & Molecular Biology
Kafayat Yusuf, Venkatesh Sampath, Shahid Umar
Summary: Bacterial infections are common and can lead to various human diseases. Some infections can be resolved with antibiotics, while others can persist and increase the risk of developing cancer. This comprehensive review highlights the complex relationship between bacterial infections and the development of different cancer types.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2023)
Article
Medicine, General & Internal
Matilda Shaked Ashkenazi, Ole Linvag Huseby, Gard Kroken, Marcela Trocha, Aurora Henriksson, Hanna Jasiak, Karen Cuartas, Alessandra Loschiavo, Isabella Kuhn, Dina Stove, Hanna Grindahl, Emilia Latour, Mathias Melbo, Katrine Holstad, Sebastian Kwiatkowski
Summary: This study surveyed 2964 participants from 59 countries to understand the symptomatology of endometriosis and assess the correlation between symptom severity and disease stage. The presentation of pain varied depending on the reported stage, with pain during and around menstruation being more prevalent in stage 1 compared to stage 4. Atypical presentation of pain was most commonly reported in stage 4, and pain solely related to triggering factors was the least common.
JOURNAL OF CLINICAL MEDICINE
(2023)
Article
Psychology, Multidisciplinary
Brooke Keilholtz, Chelsea Spencer, Summer Vail, Megan Palmer
Summary: This study aimed to understand how pandemic-specific distress was related to experiencing and perpetrating intimate partner violence (IPV). The results indicated that distress related to family relationships, taking care of children, and COVID-19 conflict increased the risk of IPV victimization, while distress related to mask mandates and friendships decreased the risk. Distress related to physical health, family relationships, taking care of children, and COVID-19 conflict increased the risk of IPV perpetration, while distress related to mental health and friendships decreased the risk.
BEHAVIORAL SCIENCES
(2023)
Review
Endocrinology & Metabolism
Brandy Wicklow, Ravi Retnakaran
Summary: Gestational diabetes mellitus (GDM) is not only a complication of pregnancy but also a signal of increased risk of developing type 2 diabetes mellitus (T2DM) and cardiovascular disease. The factors contributing to this cardiovascular risk are present even before pregnancy. GDM is now seen as a chronic metabolic disorder that affects both mother and child throughout their lives.
DIABETES & METABOLISM JOURNAL
(2023)
Review
Neurosciences
Valerie J. Sydnor, Bart Larsen, Danielle S. Bassett, Aaron Alexander-Bloch, Damien A. Fair, Conor Liston, Allyson P. Mackey, Michael P. Milham, Adam Pines, David R. Roalf, Jakob Seidlitz, Ting Xu, Armin Raznahan, Theodore D. Satterthwaite
Summary: The development of the human brain cortex is a prolonged process that progresses from lower-order functions to higher-order functions, conforming to a hierarchical pattern of organization. This developmental program enhances feature variation between different regions, but also carries risks for diverse developmental psychopathologies.
Article
Psychology, Applied
Mally Shechory Bitton
Summary: This study explores the associations between IPV, psychological distress, health and financial concerns, function and support during the COVID-19 outbreak in Israel. The findings indicate that IPV, particularly verbal violence, is directly linked to higher levels of distress and lower levels of functioning, but this link can be mitigated indirectly by health and financial concerns as well as lack of support. Gender differences were observed in rates of violence, with women reporting higher levels of verbal violence. The results highlight the importance of addressing the specific needs of families and couples in designing intervention programs during times of crisis.
Review
Clinical Neurology
Melaku Desta, Peter Memiah, Bekalu Kassie, Daniel Bekele Ketema, Haile Amha, Temesgen Getaneh, Mezinew Sintayehu
Summary: The study showed that two in every ten postpartum women in Ethiopia are suffering from postpartum depression. Intimate partner violence and inadequate social support are significant factors associated with postpartum depression. Efforts to prevent intimate partner violence and substance abuse are urgently needed to reduce the prevalence of postpartum depression.
JOURNAL OF AFFECTIVE DISORDERS
(2021)
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
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
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
Kathleen P. Pratt, Devi Gunasekera, Pooja Vir, Siyuan Tan, Glenn F. Pierce, Cara Olsen, Saulius Butenas, Kenneth G. Mann
Summary: The most common complication in the treatment of hemophilia A is the development of inhibitors, which affect the coagulation ability of patients. This study found that the level of inhibitors is not related to F8 haplotype mismatches, and the deletion of the B-domain in FVIII proteins leads to a stronger antibody reaction.
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.
Editorial Material
Hematology
Kathleen P. Pratt
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.