Endothelial Colony Forming Cells in the spotlight, insights into the pathophysiology of von Willebrand Disease and rare bleeding disorders.

SNJ Laan, BG Lenderink, JCJ Eikenboom… - Journal of Thrombosis …, 2024 - Elsevier
Endothelial cells deliver a vital contribution to the maintenance of hemostasis by constituting
an anatomical as well as functional barrier between the blood and the rest of the body. Apart …

Bleeding Disorder of Unknown Cause (BDUC): an illustrated review on current practice, knowledge gaps and future perspectives.

ALL Monard, CMA Mussert, TT van Duijl… - Research and practice …, 2024 - Elsevier
In more than half of the individuals with a clinically relevant bleeding tendency who are
referred to hemostasis experts, no biological etiology can be found after extensive laboratory …

Psychometrics of patient-reported outcomes measurement information system in von Willebrand disease, inherited platelet function disorders, and rare bleeding …

ES van Hoorn, SPE Willems, W Al Arashi… - Research and practice …, 2024 - Elsevier
Background Patient-reported outcomes measurement information system (PROMIS)
measures can be used to measure patient-reported outcomes. PROMIS measures, including …

Automated segmentation and quantitative analysis of organelle morphology, localization and content using CellProfiler

SNJ Laan, RJ Dirven, PE Bürgisser, J Eikenboom… - PLoS …, 2023 - journals.plos.org
One of the most used and versatile methods to study number, dimensions, content and
localization of secretory organelles is confocal microscopy analysis. However, considerable …

Ethical aspects of hemophilia gene therapy: a qualitative interview study with stakeholders

L Baas, K Meijer, M Driessens, AL Bredenoord… - Research and practice …, 2023 - Elsevier
Background There are great expectations for the potential role of gene therapy in the
treatment of hemophilia. At the same time, developments in the field of hemophilia gene …

Transcriptional and functional profiling identifies inflammation and endothelial-to-mesenchymal transition as potential drivers for phenotypic heterogeneity within a …

SNJ Laan, S de Boer, RJ Dirven, I van Moort… - Journal of Thrombosis …, 2024 - Elsevier
Background Endothelial colony-forming cells (ECFCs) derived from patients can be used to
investigate pathogenic mechanisms of vascular diseases like von Willebrand disease …

DosEmi study protocol: a phase IV, multicentre, open-label, crossover study to evaluate non-inferiority of pharmacokinetic-guided reduced dosing compared with …

A Donners, K van der Zwet, ACG Egberts… - BMJ open, 2023 - bmjopen.bmj.com
Introduction Emicizumab effectively prevents bleeding in people with haemophilia A
(PwHA), but is a burden for national healthcare budgets and consequently may limit access …

PBPK modeling of recombinant factor IX Fc fusion protein (rFIXFc) and rFIX to characterize the binding to type 4 collagen in the extravascular space

ME Cloesmeijer, E Sjögren… - CPT …, 2024 - Wiley Online Library
Patients with severe and sometimes moderate hemophilia B are prophylactically treated with
factor IX concentrates to prevent bleeding. For some time now, various extended terminal …

Functional characterization of a nanobody-based glycoprotein VI-specific platelet agonist

M Zivkovic, E Pols-van Veen, V van der Vegte… - Research and Practice …, 2024 - Elsevier
Abstract Background Glycoprotein (GP) VI is a platelet-specific collagen receptor required
for platelet activation during hemostasis. Platelet reactivity toward collagen is routinely …

[HTML][HTML] Protocol: DosEmi study protocol: a phase IV, multicentre, open-label, crossover study to evaluate non-inferiority of pharmacokinetic-guided reduced dosing …

A Donners, K van der Zwet, ACG Egberts… - BMJ Open, 2023 - ncbi.nlm.nih.gov
Introduction Emicizumab effectively prevents bleeding in people with haemophilia A
(PwHA), but is a burden for national healthcare budgets and consequently may limit access …