Biostate (factor VIII/von Willebrand factor) / CSL Behring - LARVOL DELTA

Cost-Effectiveness of Voncento Prophylaxis Versus On-Demand Treatment in von Willebrand Disease in the United Kingdom. (PubMed, Blood Adv) - "Results suggest that Voncento LTP is more effective and cost-saving compared with ODT in the UK for VWD patients with higher ABR. Prophylaxis for patients with frequent bleeds is likely to be a cost-saving and effective strategy."

Cost effectiveness • HEOR • Journal • Hemophilia

Leveraging National Real-World Evidence to Understand Differences of Economic Burden in Rare Diseases: The Case of Von Willebrand Disease in France (ISPOR-EU 2024) - "Within adult on-demand users, HRUC related to hospitalizations, in-hospital RT and FVIII, outpatient general practitioner and nurse visits, out-hospital RT and FVIII dispensings were assessed by AP and compared across RTs (Wilfactin ® , Veyvondi®, Voncento ® , Eqwilate ® ) using Generalized Estimating Equation models (GEE) accounting for intra-patient correlation and potential confounding factors. This large-scale real-world study is the first comparing costs related to the use of RT available in France. Costs were higher in AP treated with Wilfactin ® or Voncento ® than in those treated with Veyvondi ® ."

Clinical • HEOR • Real-world • Real-world evidence • Hematological Disorders • Hemophilia • Rare Diseases

OPALE: Registry of Patients With Von WilLEbrand Disease Treated With Voncento® (clinicaltrials.gov) - P=N/A | N=135 | Completed | Sponsor: CSL Behring | Active, not recruiting ➔ Completed

Trial completion • Hemophilia

A systematic review of efficacy and safety of plasma-derived von Willebrand factor/Factor VIII concentrate (Voncento®) in von Willebrand Disease. (PubMed, Thromb Haemost) - "This review provides a comprehensive summary of studies that evaluated the use of pdVWF/FVIII in VWD demonstrating the long-term effectiveness and safety of this pdVWF/FVIII across all ages, types of VWD and treatment settings."

Journal • Review • Hemophilia • Pediatrics

Management of Type 2M Von Willebrand Disease in Lower Limb Surgery (ISTH 2023) - "Tranexamic acid was also commenced and continued for 5 days. Having established the dose required to maintain her VWF:RCo level >50 IU/dl, our patient was discharged home on day 3 post-op and continued daily Voncento 1000/2400 IU, prior to daily Enoxaparin 40mg, for the duration of her time of immobilisation... Our patient had an uneventful post-operative course. She had no excess post-operative bleeding and there were no thrombotic complications.Conclusion(s): This is, to our knowledge, the first detailed report of the management of complex lower limb surgery in a patient with Type 2M VWD. We were able to navigate a safe peri- and post-operative course for this patient, which could be adapted for future similar clinical scenarios."

Surgery • Cardiovascular • Hematological Disorders • Hemophilia • Orthopedics • Thrombosis

"Et vous...qui vous permet de critiquer une biostateticienne reconnue??" (@mag_pakite)

Efficacy, safety and consumption of plasma-derived von Willebrand factor (VWF)/Factor VIII (FVIII) concentrate with 2.4:1 VWF:FVIII ratio for the treatment of von Willebrand Disease: a systematic review (#74) (GTH 2023) - "Plasma-derived human coagulation FVIII/human VWF (pdVWF/FVIII; Voncento®/Biostate®) is indicated in all age groups for prophylaxis and treatment of haemorrhage or surgical bleeding in patients with VWD when desmopressin is ineffective or contraindicated...Pharmacovigilance data showed low incidence rates for adverse events including FVIII/VWF inhibitors, thromboembolic events, hypersensitivity reactions and transmission of infectious agents. Conclusion This review provides a comprehensive summary of studies that evaluated the use of pdVWF/FVIII in VWD demonstrating the long-term effectiveness and safety of this pdVWF/FVIII across all ages, types of VWD and treatment settings."

Clinical • Review • Cardiovascular • Hemophilia • Immunology • Pediatrics

Structure and Function of Recombinant versus Plasma-Derived von Willebrand Factor and Impact on Multimer Pharmacokinetics in von Willebrand Disease. (PubMed, J Blood Med) - "Recombinant von Willebrand factor (rVWF, vonicog alfa) is a purified VWF concentrate produced from Chinese hamster ovary cells...rVWF also demonstrated higher FVIII binding capacity than Haemate P, Voncento and Wilate. Modeling provided evidence that VWF:RCo activity in patients with severe VWD treated with rVWF is associated with L/UL VWF multimers in the circulation. Findings suggest that the L and UL multimers preserved in rVWF contribute to high biological activity and might be important for providing hemostatic efficacy."

Journal • PK/PD data • Hemophilia

"Når New Public Management går fra HOPE til Enforce, ved man, at biostaten kører frem for fuld damp." (@stefanks)

Effectiveness of long-term prophylaxis using pdFVIII/VWF concentrate in patients with inherited von Willebrand disease. (PubMed, Eur J Haematol) - "These results suggest that Voncento® is effective to prevent recurrent bleedings in patients symptomatic VWD."

Journal • Hemophilia

EFFICACY AND SAFETY OF HIGH RATIO HVWF/FVIII CONCENTRATE (VONCENTO®) FOR THE TREATMENT OF BLEEDING EPISODES IN PATIENTS WITH VON WILLEBRAND DISEASE: THE OPALE FRENCH EXPERIENCE (EAHAD 2022) - "This French cohort study suggests that hVWF/FVIII concentrate (Voncento®) is effective and well tolerated for the treatment of bleeding, independently of VWD type and age of the patient."

Clinical • Cardiovascular • Gastroenterology • Gynecology • Hematological Disorders • Hemophilia • Musculoskeletal Diseases • Orthopedics • Rare Diseases

OPALE: Registry of Patients With Von WilLEbrand Disease Treated With Voncento® (clinicaltrials.gov) - P=N/A; N=135; Active, not recruiting; Sponsor: CSL Behring; Recruiting ➔ Active, not recruiting

Clinical • Enrollment closed • Hemophilia

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). (PubMed, J Blood Med) - P3 | "Plasma-derived von Willebrand factor/factor VIII (pdVWF/FVIII; VONCENTO, CSL Behring) is a high-concentration, low-volume, high-purity concentrate, with a high level of VWF high-molecular-weight multimers and a VWF/FVIII ratio of ~2.4:1...This study demonstrated comparable PK profiles for pediatric patients aged <6 years and aged 6-12 years, and an excellent efficacy and safety profile in this population. The adverse events reported were mostly mild to moderate with inhibitor rates within the expected incidence range."

Clinical • Journal • P3 data • PK/PD data • Hematological Disorders • Hemophilia • Pediatrics • Rare Diseases

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) (Dovepress) - P3, N=35; NCT01229007; Sponsor: CSL Behring; "This study demonstrated comparable PK profiles for pediatric patients aged < 6 years and aged 6– 12 years, and an excellent efficacy and safety profile in this population. The adverse events reported were mostly mild to moderate with inhibitor rates within the expected incidence range."

P3 data • Hemophilia

[VIRTUAL] EFFICACY AND SAFETY OF HFVIII/VWF (VONCENTO®) CONCENTRATE IN PAEDIATRIC PATIENTS WITH VON WILLEBRAND DISEASE (VWD): THE FRENCH EXPERIENCE (EAHAD 2021) - "This study demonstrates that Voncento® is effective and well tolerated in the prevention and treatment of bleeding in this paediatric French cohort."

Clinical • Gastroenterology • Hematological Disorders • Hemophilia • Osteoarthritis • Pediatrics

OPALE: Registry of Patients With Von WilLEbrand Disease Treated With Voncento® (clinicaltrials.gov) - P=N/A; N=120; Recruiting; Sponsor: CSL Behring; Trial completion date: Dec 2022 ➔ Dec 2023; Trial primary completion date: Dec 2022 ➔ Dec 2023

Clinical • Trial completion date • Trial primary completion date • Hemophilia

OPALE: Registry of Patients With Von WilLEbrand Disease Treated With Voncento® (clinicaltrials.gov) - P; N=120; Recruiting; Sponsor: CSL Behring

Clinical • New trial • Hemophilia

Effectiveness and safety of hFVIII/VWF concentrate (Voncento) in patients with inherited von Willebrand disease requiring surgical procedures: the OPALE multicentre observational study. (PubMed, Blood Transfus) - "The present study suggests that Voncento is an effective and well-tolerated therapy for the peri-operative management of patients with all VWD types."

Clinical • Journal • Observational data • Hemophilia

An Open-Label Extension Study to Assess the Long-Term Efficacy and Safety of a Plasma-Derived von Willebrand Factor (VWF)/Factor VIII (FVIII) Concentrate in Patients with von Willebrand Disease (SWIFT-VWDext Study). (PubMed, J Blood Med) - "Plasma-derived von Willebrand factor/factor VIII (pdVWF/FVIII; VONCENTO, CSL Behring) is a high-concentration, low-volume, high-purity concentrate, which contains a high level of high-molecular-weight multimers and a VWF/FVIII ratio of ~2.4:1...There were no cases of anaphylactic reactions and angioedema, development of VWF/FVIII inhibitors, thromboembolic events, or viral infections. This contemporary comprehensive development program evaluating pdVWF/FVIII across all ages demonstrates long-term safety and efficacy for treatment and prevention of bleeds in patients with severe VWD, supporting the benefit-risk profile of pdVWF/FVIII."

Clinical • Journal • Cardiovascular • Hemophilia • Infectious Disease • Pediatrics

Providing personalized care for bleeding disorders (News-Medical) - P3, N=115; PROPEL (NCT03732820); Sponsor: AstraZeneca; CONTINUATION (NCT01945593); Sponsor: Shire; "An interview with Dr. Carmen Escuriola-Ettingshausen MD...on providing personalized care for bleeding disorders such as hemophilia and von Willebrand disease....Of course, personalized prophylaxis usually aims to prevent all bleeds. But from a world-wide perspective, personalization of therapy can also be implemented with a lower amount of resources, for example in developing countries."

Interview • P3 data

[VIRTUAL] Recombinant Human von Willebrand Factor (rVWF) Differs in Structure and Function from Plasma Derived Concentrates (ISTH 2020) - "Aims: Structural and functional comparison of clinically used pd and rVWF concentrates: pdFVIII/VWF: HAEMATE P/HUMATE-P, VONCENTO (CSL Behring, King of Prussia, USA); WILATE/EQWILATE (Octapharma, Lachen, Switzerland)pdVWF: WILFACTIN/WILLFACT (LFB, Courtaboeuf, France)rVWF: VONVENDI/VEYVONDI Cross-product comparisons included VWF content by determination of VWF: Ag, and biological function assessed by VWF: RCo, VWF: CB, and VWF: GpIbM (VWF activity assay Innovance; Siemens) using routine commercial test kits. VWF products differ in their contents, multimer size and structure of functional VWF. These differences may translate into improved biological activity of rVWF in clinical settings and influence the treatment regimen of individuals with VWD."

Targeted Protein Degradation

Efficacy and safety of hFVIII/VWF concentrate in a cohort of patients with von Willebrand disease (VWD) requiring prophylaxis regimen (OPALE study) (EAHAD 2020) - "The results of this study confirmed the efficacy and tolerance of Voncento® in the prophylactic management of hemorrhagic episodes in VWD patients."

Clinical • Cardiovascular • Hematological Disorders • Thrombosis

Pharmacokinetics, Efficacy and Safety of a Plasma-Derived VWF/FVIII Concentrate (Formulation V) in Pediatric Patients with von Willebrand Disease (SWIFTLY-VWD Study). (PubMed, J Blood Med) - "Formulation V (VONCENTO) is a plasma-derived high-concentration/low-volume, high-purity von Willebrand factor (VWF)/factor VIII (FVIII) concentrate, originally indicated for von Willebrand disease (VWD) in adults and adolescents...No cases of anaphylactic reactions or angioedema, development of FVIII/VWF inhibitors, thromboembolic events, or viral infections were reported. This study provides evidence for use of Formulation V to treat and prevent bleeding in pediatric patients with severe VWD, and led to the European approval of Formulation V in children."

Clinical • Journal • PK/PD data • Cardiovascular • Pediatrics

[VIRTUAL] Can a Patient with Von Willebrand Disease Donate a Kidney? (ATC 2020) - "The donor received Voncento (Von Willebrand factor + factor VIII) and had an uneventful hand- assisted laparoscopic left donor nephrectomy... Donors with VWD should be considered for live kidney donation following peril-operative correction of Von Willebrand factor and after discussion in an MDT approach. The donors should be involved in the decision making process as to which route of treatment to choose from including the risk of transmission of BBV."

Clinical • Nephrology

Observatory of patients with von willebrand disease treated with human coagulation FVIII/VWF complex (opale study) (EAHAD 2018) - "hFVIII/VWF (Voncento) was effective and well-tolerated in the prevention and treatment of hemorrhage and surgical bleeding in a French cohort of patients with inherited VWD."

Clinical • Biosimilar • Hematological Malignancies • Venous Thromboembolism