ReviewBlood advances2019
New therapies for von Willebrand disease.
Review in Blood advances, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers.
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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.
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Who cites it
17 citing papers in PubMed, 48 citations in OpenAlex.
- A Rare Combination of von Willebrand Disease Type 2A and 2M: Diagnostic and Therapeutic Challenges - A case report.Research and practice in thrombosis and haemostasis · 2026Article
- HMB-002: a monovalent antibody that elevates circulating VWF and FVIII levels for treatment of von Willebrand disease.Blood advances · 2026Article
- Acute Thyroid Swelling After Fine-Needle Aspiration in von Willebrand Disease: Case Report and Literature Review.Cureus · 2025Article
- Amelioration of a von Willebrand disease type 2B phenotype in vivo upon treatment with allele-selective siRNAs.Blood advances · 2025Article
- Iatrogenic hemorrhage and extensive venous thromboembolism during iTTP treatment with caplacizumab-A case report.EJHaem · 2024Article
- New and emerging therapies for women, girls, and people with the potential to menstruate with VWD.Blood advances · 2023Article
- Development of a dual hybrid AAV vector for endothelial-targeted expression of von Willebrand factor.Gene therapy · 2023Article
- Treatment of Acquired von Willebrand Disease due to Extracorporeal Membrane Oxygenation in a Pediatric COVID-19 Patient with Vonicog Alfa: A Case Report and Literature Review.TH open : companion journal to thrombosis and haemostasis · 2023Article
- O-glycosylation and its role in therapeutic proteins.Bioscience reports · 2022Review
- The VWF binding aptamer rondoraptivon pegol increases platelet counts and VWF/FVIII in type 2B von Willebrand disease.Blood advances · 2022Article
- Structure and Function of Recombinant versus Plasma-Derived von Willebrand Factor and Impact on Multimer Pharmacokinetics in von Willebrand Disease.Journal of blood medicine · 2022Article
- Etiology and Management of Bleeding during ECMO in a COVID-19 Patient.Journal of atherosclerosis and thrombosis · 2021Article
- Prophylactic management of patients with von Willebrand disease.Therapeutic advances in hematology · 2021Review
- New developments in von Willebrand disease.British journal of haematology · 2020Review
- Acquired von Willebrand syndrome: focused for hematologists.Haematologica · 2020Review
- Management of von Willebrand disease with a factor VIII-poor von Willebrand factor concentrate: Results from a prospective observational post-marketing study.Journal of thrombosis and haemostasis : JTH · 2020Observational
- Hemophilia therapy: the future has begun.Haematologica · 2020Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
1 author.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The management of von Willebrand disease (VWD) is based upon the dual correction of the primary hemostasis defect, due to the inherited deficiency of von Willebrand factor (VWF), and of the secondary defect of factor VIII coagulant activity (FVIII:C), due to the loss of binding and stabilization by VWF of this intrinsic coagulation factor in flowing blood. The traditional therapeutic weapons (the synthetic derivative of the antidiuretic hormone desmopressin and plasma-derived VWF/FVIII concentrates) are able to transiently correct both the defects. With the goal of tackling the primary deficiency in the disease, that is, VWF, but at the same time exploiting the normal capacity of patients to produce FVIII, the novel approach of replacing only VWF was implemented in the last 10 years. Following the manufacturing of a concentrate fractionated from human plasma and of one obtained by recombinant DNA technology, clinical studies have shown that VWF-only products correct not only the primary VWF deficiency but also the secondary FVIII:C deficiency. The demonstrated efficacy of these products in various clinical situations and, ultimately, in such a hemostasis-challenging context as surgery testifies to the effectiveness and safety of this approach. It remains to be seen whether VWF-only products are efficacious and safe in still-unexplored situations, such as use in children; the long-term use for prophylaxis; and in recurrent gastrointestinal (GI) bleeding due to angiodysplasia, a major therapeutic problem in VWD.
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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.