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Voncento
Indication in von Willebrand disease
Prophylaxis and treatment of haemorrhage or surgical bleeding in adults and children with VWD, when desmopressin (DDAVP) treatment alone is ineffective or contraindicated.
About VWD
Von Willebrand disease (VWD) is a relatively common inherited congenital bleeding disorder in which there is a deficiency or dysfunction of Von Willebrand factor (VWF), a clotting protein.1
VWD is the most common bleeding disorder affecting around 1% of the general population.2 VWD affects both men and women with equal incidence,3 but is often more detected in women as a result of heavy or abnormal bleeding during menstruation and after childbirth.4
VWF is a multimeric glycoprotein that has two essential functions in primary haemostasis: binding to Factor VIII (FVIII) to assist in its transportation and stabilisation, and to platelets in blood vessel walls, aiding formation of a platelet plug during the clotting process.5 A deficiency or dysfunction of Von Willebrand factor causes primary haemostasis to be impaired.5
VWD is classified into types 1, 2 and 3 with type 2 further subdivided into type 2A, 2B, 2M, 2N.6 VWD is caused by either a quantitative or qualitative deficiency in VWF. Quantitative deficiency is caused by a decrease in the level of VWF, while qualitative deficiency is due to an abnormal structure or function of VWF.6
Efficacy
Voncento is approved for the treatment of VWD, secondary to DDAVP, across all age groups and provides reliable bleed control.7-9
Percentage of non-surgical bleeding (NSB) events in which efficacy was reported as excellent in adults and adolescents (> 12 years of age) receiving on-demand treatment (n=20):7
- All: 92.1 % excellent (of 407 NSB events)
- Joint: 98% excellent (of 101 NSB events)
- Muscle: 100% excellent (of 17 NSB events)
- Major: 93.6% (of 125 NSB events)
- Minor: 91.5% (of 281 NSB events)
The total number of NSB events reported from the prophylactic arm decreased from 304 to 10 bleeds (n=8), with the haemostatic efficacy rated as "excellent" for all 10 events.7
- ABr decreased from a medium of 26.5 (18-82) on-demand, to a median of 1.0 (1-6) during prophylactic therapy
The haemostatic efficacy for all non-surgical bleeds was considered excellent or good for paediatrics (<12 years of age) receiving on-demand (n=13) and prophylaxis (n=4) treatment, in both the on-demand and prophylaxis arms.8
- The total number of bleeds requiring treatment in the on-demand arm was 80 vs 73 in the prophylaxis arm
- The number of major bleeds requiring treatment in the on-demand arm was 27.1% (n=26) vs 3.3% (n=3) in the prophylaxis arm
High molecular weight multimers (HMWM)
Voncento has a high percentage (8%-12%) of high molecular weight multimers which are important for effective haemostasis.7
The multimer profile closely resembles normal human plasma.7 Voncento's relative proportion of HMWM that are 86% of that found in normal human plasma following administration.7
The HMWM achieve effective haemostasis7
- High (>35% in 60 seconds) binding capacity for collagen and platelets11
- The HMWM remain stable 6 hours post infusion for haemostatic support7
2.4:1 Ratio
Voncento provides effective haemostatic treatment with a relatively high ratio of 2.4:1 VWF to FVIII12
- Some patients can have reduced FVIII resulting from their Von Willebrand disease13
Voncento, with FVIII and VWF, provides a combined treatment - an important option when both factors are required
- VWF and FVIII work together to promote platelet aggregation, platelet adhesion and thrombin formation7
Summary
- Replaces missing or dysfunctional Von Willebrand factor9
- Provides effective haemostatic treatment with a ratio of 2.4:1 VWF/FVIII12
- Contains high molecular weight multimers that achieve effective haemostasis7
- A broad range of clinical efficacy in adults and children7,8
- Proven efficacy for surgical bleeds, non-surgical bleeds and prophylaxis in children7,8
- Two dedicated antiviral steps14
- Provides a fast infusion rate at 6ml/min9
References
1. US Department of Health and Human Services. The Diagnosis, Evaluation, and Management of Von Willebrand Disease. 2007;NIH Publication No. 08-5832. 2. Friedman KD, Rodgers GM. Inherited Coagulation Disorders. 2004;59:1619–67. 3. Centers for Disease Control and Prevention (CDC). Data and Statistics on Von Willebrand Disease. https://www.cdc.gov/ncbddd/vwd/data.html Accessed March 2022. 4. Lillicrap D. Von Willebrand disease: advances in pathogenetic understanding, diagnosis, and therapy. American Society of Hematology. 2013;122:254-60. 5. Peyvandi F, Garagiola I, Baronciani L. Role of Von Willebrand factor in haemostasis. Blood Transfus. 2011;9(Suppl 2):s3-8. 6. Sadler JE, Budde U, Eikenboom JC, et al. Update on the pathophysiology and classification of Von Willebrand disease: a report of the Subcommittee on Von Willebrand Factor. J Thromb Haemost. 2006;4(10):2103-14. 7. Lissitchkov TJ, Buevich E, et al. Pharmacokinetics, efficacy, and safety of a plasma-derived VWF/FVIII concentrate (VONCENTO) for on-demand and prophylactic treatment in patients with Von Willebrand disease (SWIFT-VWD study). Blood Coagulation and Fibrinolysis. 2017;28(2):152–162. 8. Auerswald G, Khayat CD, et al. Pharmacokinetics, Efficacy and Safety of a Plasma-Derived VWF/FVII Concentrate (Formulation V) in Pediatric Patients with Von Willebrand Disease (SWIFTLY-VWD Study). Journal of Blood Medicine. 2020;11:213-225. 9. CSL Behring UK Limited. Voncento Summary of Product Characteristics. 10. Stockschlaeder M, Schneppenheim R and Budde U. Update on Von Willebrand factor multimers: focus on high-molecular-weight multimers and their role in hemostasis. Blood Coagulation and Fibrinolysis. 2014;25:206– 216. 11. Riddell A, Vinayagam S, et al. Evaluation of Von Willebrand factor concentrates by platelet adhesion to collagen using an in vitro flow assay. Research and Practice in Thrombosis and Haemostasis. 2019;3(1):126-135. 12. Harper P, Favaloro EJ, et al. Human plasma-derived FVIII/VWD concentrate (Biostate): a review of experimental and clinical pharmacokinetic, efficacy and safety data. Drugs in Context. 2016;5:1740-4398. 13. Miesbach W, Berntorp E. Interaction between VWF and FVIII in treating VWD. European Journal of Haematology. 2015;95:449-454. 14. European Medicines Agency. Assessment report: Voncento human coagulation factor VIII/von Willebrand factor. EMA/404213/2013.
Adverse events should be reported. Reporting forms and information can be found at https://yellowcard.mhra.gov.uk. Adverse events should also be reported to CSL Behring UK Ltd. on 01444 447 405.