Download full-text PDF |
Source |
|---|---|
| http://dx.doi.org/10.1111/hae.13705 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
Conformation-specific RNA aptamers for phenotypic distinction between normal von Willebrand factor and type 2B von Willebrand disease.
NAR Mol Med
October 2024
Division of Hematology, Department of Internal Medicine, Mayo Clinic, 200 1st St SW, Rochester, MN, USA.
The A1 domain in Von Willebrand Factor (VWF) initiates coagulation through binding to platelet glycoprotein GPIbα receptors. Von Willebrand Disease (VWD)-Mutations in A1 that either impair (type 2M) or enhance (type 2B) platelet adhesion to VWF can locally destabilize and even misfold the domain. We leveraged misfolding in the gain-of-function type 2B VWD phenotype as a target, distinct from the normal conformation.
View Article and Find Full Text PDFBackground: Chronic arterial hypertension restructures the vascular architecture of the brain, leading to a series of pathological responses that culminate in cerebral small-vessel disease. Pericytes respond dynamically to vascular challenges; however, how they manifest under the continuous strain of hypertension has not been elucidated.
Methods And Results: In this study, we characterized pericyte behavior alongside hypertensive states in the spontaneously hypertensive stroke-prone rat model, focusing on their phenotypic and metabolic transformation.
Advances in biopharmaceutical products for hemophilia.
iScience
December 2024
Chengdu Rongsheng Pharmaceuticals Co., Ltd, Chengdu 610041, China.
Hemophilia is caused by the deficiency of clotting factors due to a single genetic abnormality. Replacement therapies have evolved from plasma-derived to recombinant coagulation factor concentrates but continue to have certain limitations. Monoclonal antibodies are clinical prophylactic treatment options unaffected by inhibitors and have better compliance than coagulation factor concentrates for patients with hemophilia.
View Article and Find Full Text PDFEx vivo evaluation of the effect of plasma-derived factor VIII/von Willebrand factor in patients with severe hemophilia A on emicizumab prophylaxis.
Clin Exp Med
December 2024
Discovery Research, Scientific Innovation Office, Grifols, Palou 3, 08150, Parets del Vallès, Barcelona, Spain.
Hemophilia A (HA) patients under emicizumab prophylaxis may require the concomitant use of procoagulant factors for breakthrough bleedings or immune tolerance induction (ITI). The aim of this study is to evaluate the ex vivo procoagulant effect of plasma-derived FVIII concentrates containing von Willebrand factor (pdFVIII/VWF) in samples from patients with severe HA without inhibitors on emicizumab prophylaxis. Samples from healthy controls (HC) and HA patients were drawn in sodium citrate plus corn trypsin inhibitor tubes and spiked with increasing concentrations of pdFVIII/VWF concentrates (10-400 IU/dL) (Fanhdi/Alphanate, Grifols), activated prothrombin complex concentrate (aPCC, 0.
View Article and Find Full Text PDFCost-Effectiveness of Voncento Prophylaxis Versus On-Demand Treatment in von Willebrand Disease in the United Kingdom.
Blood Adv
December 2024
CSL Behring, King Of Prussia, Pennsylvania, United States.
von Willebrand factor (VWF) concentrates may be required for on-demand treatment (ODT) or long-term prophylaxis (LTP) in von Willebrand disease (VWD). This study assesses the cost-effectiveness of LTP compared with ODT in VWD patients treated with Voncento in the United Kingdom (UK). A Markov structure was developed to estimate quality-adjusted life years (QALYs) and costs of VWD treatment over a lifetime horizon.
View Article and Find Full Text PDFWant AI Summaries of new PubMed Abstracts delivered to your In-box?
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!