Trial reportBlood2019
Genetic determinants of VWF clearance and FVIII binding modify FVIII pharmacokinetics in pediatric hemophilia A patients.
Trial report in Blood, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 papers, 2 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
20 citing papers in PubMed, 2 syntheses or guidelines pooled it, 44 citations in OpenAlex.
- The contribution of the sinusoidal endothelial cell receptors CLEC4M, stabilin-2, and SCARA5 to VWF-FVIII clearance in thrombosis and hemostasis.Journal of thrombosis and haemostasis : JTH · 2023Pooled it
- Correlation Analysis of DNA Methylation in the von Willebrand Factor Promoter Region and the Risk of Unexplained Recurrent Hemophilia: Systematic Review and Meta-Analysis.Contrast media & molecular imaging · 2022Pooled it
- The effect of F8 missense variants on desmopressin response in people with nonsevere hemophilia A investigated using machine learning.Research and practice in thrombosis and haemostasis · 2026Article
- Individual Comparative PK Evaluation of Single-dose Octocog Alfa, Rurioctocog Alfa Pegol, and Efanesoctocog Alfa in Adults with Severe Hemophilia A.TH open : companion journal to thrombosis and haemostasis · 2026Article
- The T follicular helper/T follicular helper regulatory pathway in FVIII immune responses in mice.Blood · 2025Article
- Extending health equity to people with moderate and mild hemophilia A: revisiting the HAVEN 6 trial.Research and practice in thrombosis and haemostasis · 2025Article
- Immunogenicity profile of rurioctocog alfa pegol in previously treated patients with severe congenital hemophilia A.Blood advances · 2024Article
- Comparison of different machine learning classification models for predicting deep vein thrombosis in lower extremity fractures.Scientific reports · 2024Article
- Dynamic molecular choreography induced by acute heat exposure in human males: a longitudinal multi-omics profiling study.Frontiers in public health · 2024Article
- Pharmacokinetics of recombinant factor VIII in adults with severe hemophilia A: fixed-sequence single-dose study of octocog alfa, rurioctocog alfa pegol, and efanesoctocog alfa.Research and practice in thrombosis and haemostasis · 2023Article
- Current Understanding of Inherited Modifiers of FVIII Pharmacokinetic Variation.Pharmacogenomics and personalized medicine · 2023Review
- Endothelial VWF is critical for the pathogenesis of vaso-occlusive episode in a mouse model of sickle cell disease.Proceedings of the National Academy of Sciences of the United States of America · 2022Article
- Enhanced pharmacokinetics and reduced bleeds in boys with hemophilia A after switching to Kovaltry from other standard half-life factor VIII concentrates.Research and practice in thrombosis and haemostasis · 2022Article
- Combination ofJournal of clinical medicine · 2022Article
- Screening of lncRNA-miRNA-mRNA Coexpression Regulatory Networks Involved in Acute Traumatic Coagulation Dysfunction Based on CTD, GeneCards, and PharmGKB Databases.Oxidative medicine and cellular longevity · 2022Article
- Pharmacokinetics of perioperative FVIII in adult patients with haemophilia A: An external validation and development of an alternative population pharmacokinetic model.Haemophilia : the official journal of the World Federation of Hemophilia · 2021Article
- Efficacy, Safety and Pharmacokinetic Results of a Phase III, Open-Label, Multicenter Study with a Plasma-Derived Von Willebrand Factor (VWF)/Factor VIII (FVIII) Concentrate in Pediatric Patients <12 Years of Age with Hemophilia A (SWIFTLY-HA Study).Journal of blood medicine · 2021Article
- Biological mechanisms underlying inter-individual variation in factor VIII clearance in haemophilia.Haemophilia : the official journal of the World Federation of Hemophilia · 2020Review
- Clinical Evidence and Safety Profile of Emicizumab for the Management of Children with Hemophilia A.Drug design, development and therapy · 2020Review
- Nonneutralizing Antibodies in Haemophilia A Patients: To Be Ignored or Not to Be!Haemophilia : the official journal of the World Federation of HemophiliaReview
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
12 authors at 4 institutions in 3 countries.
Funding
Abstract
Factor VIII (FVIII) pharmacokinetic (PK) properties show high interpatient variability in hemophilia A patients. Although previous studies have determined that age, body mass index, von Willebrand factor antigen (VWF:Ag) levels, and ABO blood group status can influence FVIII PK, they do not account for all observed variability. In this study, we aim to describe the genetic determinants that modify the FVIII PK profile in a population of 43 pediatric hemophilia A patients. We observed that VWF:Ag and VWF propeptide (VWFpp)/VWF:Ag, but not VWFpp, were associated with FVIII half-life. VWFpp/VWF:Ag negatively correlated with FVIII half-life in patients with non-O blood type, but no correlation was observed for type O patients, suggesting that von Willebrand factor (VWF) half-life, as modified by the ABO blood group, is a strong regulator of FVIII PK. The FVIII-binding activity of VWF positively correlated with FVIII half-life, and the rare or low-frequency nonsynonymous VWF variants p.(Arg826Lys) and p.(Arg852Glu) were identified in patients with reduced VWF:FVIIIB but not VWF:Ag. Common variants at the
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.