PDF herunterladen
CC BY-NC-ND 4.0 · Thromb Haemost 2024; 124(09): 828-841
DOI: 10.1055/a-2253-9701
Coagulation and Fibrinolysis

A Systematic Review of Efficacy and Safety of Plasma-Derived von Willebrand Factor/Factor VIII Concentrate (Voncento) in von Willebrand Disease

Lucia Rugeri 
1   Unite d'Hemostase Clinique, Hôpital Cardiologique, Hospices Civils de Lyon, Lyon, France
,
Will Thomas 
2   Cambridge University Hospitals NHS Foundation Trust, Cambridge Biomedical Campus, Cambridge, United Kingdom
,
Kathrin Schirner 
3   CSL Behring Innovation GmbH, Marburg, Germany
,
Lisa Heyder 
3   CSL Behring Innovation GmbH, Marburg, Germany
,
Günter Auerswald 
4   Coagulation Centre, Bremen Central Clinic, GeNo Ltd., Parent-Child-Centre Prof. Hess, Bremen, Germany
› Institutsangaben
› Weitere Informationen
Auch verfügbar auf
   Lizenzen und Reprints


Abstract

Background For the treatment of von Willebrand disease (VWD), von Willebrand factor (VWF) concentrates can be used in on-demand, long-term prophylaxis, and surgical prophylaxis regimens.

Methods This systematic literature review was conducted to evaluate the efficacy, consumption, and safety of plasma-derived human coagulation FVIII/human VWF (pdVWF/FVIII; Voncento/Biostate) for the treatment of patients with any inherited VWD type. An electronic search was conducted in MEDLINE and Cochrane Library databases on VWD therapies. All retrieved publications were assessed against predefined inclusion/exclusion criteria following the Cochrane group recommendations. Associated pharmacovigilance data were collected across the same time period.

Results Eleven publications from eight study cohorts were identified for data retrieval. All were from multicenter studies and included both pediatric and adult patients. Eight publications included evaluations of the efficacy of pdVWF/FVIII for on-demand treatment, eight included long-term prophylactic treatment, and eight included surgical prophylaxis. Treatment protocols and VWF administration methods differed between studies, as did safety evaluations. The clinical response was rated as excellent/good for on-demand treatment in 66 to 100% of nonsurgical bleeds, 89 to 100% in the treatment of breakthrough bleeds during long-term prophylaxis treatment, and hemostatic efficacy in surgical procedures was 75 to 100%. Pharmacovigilance data confirmed a low incidence of adverse events in treated patients.

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.

Keywords

blood products - factor VIII - systematic review - von Willebrand disease - von Willebrand factor

Authors' Contribution

L.R., W.T., and G.A. contributed to the concept and design of the systematic literature review, and provided critical appraisal and interpretation of the data presented. L.H. and K.S. performed analysis and interpretation of the pharmacovigilance data. All authors provided critical appraisal and revisions of this document during its creation. All authors reviewed and approved the final version of this manuscript prior to submission. Medical writing assistance was provided by Lucy Craggs and Claire Crouchley of Meridian HealthComms, Plumley, UK under the guidance of all authors.


Supplementary Material



Publikationsverlauf

Eingereicht: 21. August 2023

Angenommen: 18. Januar 2024

Accepted Manuscript online:
25. Januar 2024

Artikel online veröffentlicht:
29. April 2024

© 2024. The Author(s). This is an open access article published by Thieme under the terms of the Creative Commons Attribution-NonDerivative-NonCommercial License, permitting copying and reproduction so long as the original work is given appropriate credit. Contents may not be used for commercial purposes, or adapted, remixed, transformed or built upon. (https://creativecommons.org/licenses/by-nc-nd/4.0/)

Georg Thieme Verlag KG
Rüdigerstraße 14, 70469 Stuttgart, Germany

 

  • References

  • 1 Castaman G, Goodeve A, Eikenboom J. European Group on von Willebrand Disease. Principles of care for the diagnosis and treatment of von Willebrand disease. Haematologica 2013; 98 (05) 667-674
    CrossrefPubMedSuche in Google Scholar
  • 2 Connell NT, Flood VH, Brignardello-Petersen R. et al. ASH ISTH NHF WFH 2021 guidelines on the management of von Willebrand disease. Blood Adv 2021; 5 (01) 301-325
    CrossrefPubMedSuche in Google Scholar
  • 3 Heijdra JM, Cnossen MH, Leebeek FWG. Current and emerging options for the management of inherited von Willebrand disease. Drugs 2017; 77 (14) 1531-1547
    CrossrefPubMedSuche in Google Scholar
  • 4 Federici AB. Prophylaxis in patients with von Willebrand disease: who, when, how?. J Thromb Haemost 2015; 13 (09) 1581-1584
    CrossrefPubMedSuche in Google Scholar
  • 5 European Medicines Agency. Summary of Products Characteristics - Voncento® . Accessed May 31, 2023 at: https://www.ema.europa.eu/en/medicines/human/EPAR/voncento
    PubMed
  • 6 Lissitchkov TJ, Buevich E, Kuliczkowski K. 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 Coagul Fibrinolysis 2017; 28 (02) 152-162
    CrossrefPubMedSuche in Google Scholar
  • 7 Gaasterland CMW, van der Weide MCJ, du Prie-Olthof MJ. et al. The patient's view on rare disease trial design - a qualitative study. Orphanet J Rare Dis 2019; 14 (01) 31
    CrossrefPubMedSuche in Google Scholar
  • 8 Auerswald G, Djambas Khayat C, Stasyshyn O. et al. Pharmacokinetics, efficacy and safety of a plasma-derived VWF/FVIII concentrate (formulation V) in pediatric patients with von Willebrand Disease (SWIFTLY-VWD study). J Blood Med 2020; 11: 213-225
    CrossrefPubMedSuche in Google Scholar
  • 9 Lissitchkov T, Klukowska A, Buevich E. et al. 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). J Blood Med 2020; 11: 345-356
    CrossrefPubMedSuche in Google Scholar
  • 10 Dunkley S, Baker RI, Pidcock M. et al. Clinical efficacy and safety of the factor VIII/von Willebrand factor concentrate BIOSTATE in patients with von Willebrand's disease: a prospective multi-centre study. Haemophilia 2010; 16 (04) 615-624
    CrossrefPubMedSuche in Google Scholar
  • 11 EU Clinical Trials Register. Identifier EUDRACT 2013–003305–25. An Open-label, Multi-centre Post-marketing Study to Assess the Efficacy and Safety of Voncento® in Subjects with Von Willebrand Disease. Accessed May 31, 2023 at: https://www.clinicaltrialsregister.eu/ctr-search/search?query=2013-003305-25
    PubMed
  • 12 d'Oiron R, Rugeri L, Harroche A. et al. 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. Haemophilia 2022; 28 (POSTER PRESENTATIONS): 25-126
    PubMedSuche in Google Scholar
  • 13 Rugeri L, d'Oiron R, Harroche A. et al. Effectiveness and safety of hFVIII/VWF concentrate (Voncento®) in patients with inherited von Willebrand disease requiring surgical procedures: the OPALE multicentre observational study. Blood Transfus 2021; 19 (02) 152-157
    PubMedSuche in Google Scholar
  • 14 Rugeri L, Harroche A, Repessé Y. et al. Effectiveness of long-term prophylaxis using pdFVIII/VWF concentrate in patients with inherited von Willebrand disease. Eur J Haematol 2022; 109 (01) 109-117
    CrossrefPubMedSuche in Google Scholar
  • 15 Harroche A, Rugeri L, D'Oiron R. et al. Efficacy and safety of hFVIII/VWF (Voncento®) concentrate in paediatric patients with Von Willebrand disease (VWD): the French experience. Abstract. Haemophilia 2021; 27 (S2)(Virtual Congress of the European Association for Haemophilia and Allied Disorders 2021 3–5 February 2021): 67. ABS090
    PubMedSuche in Google Scholar
  • 16 Howman R, Barnes C, Curtin J. et al. The clinical efficacy and safety of the FVIII/VWF concentrate, BIOSTATE®, in children with von Willebrand disorder: a multi-centre retrospective review. Haemophilia 2011; 17 (03) 463-469
    CrossrefPubMedSuche in Google Scholar
  • 17 Shortt J, Dunkley S, Rickard K, Baker R, Street A. Efficacy and safety of a high purity, double virus inactivated factor VIII/von Willebrand factor concentrate (Biostate) in patients with von Willebrand disorder requiring invasive or surgical procedures. Haemophilia 2007; 13 (02) 144-148
    CrossrefPubMedSuche in Google Scholar
  • 18 EU Clinical Trials Register. Identifier EUDRACT 2009–017301–11. An Open-Label, Multi-Centre Extension Study to Assess the Efficacy and Safety of Biostate® in Paediatric, Adolescent, and Adult Subjects with Von Willebrand Disease who Completed Clinical Studies CSLCT-BIO-08–52 or CSLCTBIO-08–54. Accessed May 31, 2023 at: https://www.clinicaltrialsregister.eu/ctr-search/search?query=2009-017301-11
    PubMed
  • 19 Djambas Khayat C, Iosava G, Romashevskaya I. et al. 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). J Blood Med 2021; 12: 483-495
    CrossrefPubMedSuche in Google Scholar
  • 20 Skotnicki A, Lissitchkov TJ, Mamonov V. et al. Efficacy, safety and pharmacokinetic profiles of a plasma-derived VWF/FVIII concentrate (VONCENTO®) in subjects with haemophilia A (SWIFT-HA study). Thromb Res 2016; 137: 119-125
    CrossrefPubMedSuche in Google Scholar
  • 21 Kershaw G, Barbaro P, Curtin J, Favaloro E, Shoemark R, Keegan A. High titre anti-VWF detectable by inhibition of collagen-binding activity in a patient with Type 3 von Willebrand Disease. Haemophilia 2014; 20 (s3): 1-186
    PubMedSuche in Google Scholar
  • 22 Committee for Medicinal Products for Human Use. Guideline on the clinical investigation of human plasma derived von Willebrand Factor products European Medicines Agency. Accessed May 31, 2023 at: https://www.ema.europa.eu/en/clinical-investigation-human-plasma-derived-von-willebrand-factor-products
    PubMed
  • 23 James PD, Connell NT, Ameer B. et al. ASH ISTH NHF WFH 2021 guidelines on the diagnosis of von Willebrand disease. Blood Adv 2021; 5 (01) 280-300
    Suche in Google Scholar
  • 24 Windyga J, Dolan G, Altisent C, Katsarou O, López Fernández MF, Zülfikar B. ; EHTSB. Practical aspects of factor concentrate use in patients with von Willebrand disease undergoing invasive procedures: a European survey. Haemophilia 2016; 22 (05) 739-751
    CrossrefPubMedSuche in Google Scholar
  • 25 Nichols WL, Hultin MB, James AH. et al. von Willebrand disease (VWD): evidence-based diagnosis and management guidelines, the National Heart, Lung, and Blood Institute (NHLBI) Expert Panel report (USA). Haemophilia 2008; 14 (02) 171-232
    CrossrefPubMedSuche in Google Scholar
  • 26 Kouides P, Wawra-Hehenberger K, Sajan A, Mead H, Simon T. Safety of a pasteurized plasma-derived factor VIII and von Willebrand factor concentrate: analysis of 33 years of pharmacovigilance data. Transfusion 2017; 57 (10) 2390-2403
    CrossrefPubMedSuche in Google Scholar
  • 27 Srivastava A, Santagostino E, Dougall A. et al; WFH Guidelines for the Management of Hemophilia panelists and co-authors. WFH Guidelines for the Management of Hemophilia, 3rd edition. Haemophilia 2020; 26 (Suppl. 06) 1-158
    CrossrefPubMedSuche in Google Scholar
  • 28 James PD, Lillicrap D, Mannucci PM. Alloantibodies in von Willebrand disease. Blood 2013; 122 (05) 636-640
    CrossrefPubMedSuche in Google Scholar
  • 29 James PD, Lillicrap D. The molecular characterization of von Willebrand disease: good in parts. Br J Haematol 2013; 161 (02) 166-176
    CrossrefPubMedSuche in Google Scholar
  • 30 Federici AB. The safety of plasma-derived von Willebrand/factor VIII concentrates in the management of inherited von Willebrand disease. Expert Opin Drug Saf 2009; 8 (02) 203-210
    CrossrefPubMedSuche in Google Scholar
  • 31 Mannucci PM. How I treat patients with von Willebrand disease. Blood 2001; 97 (07) 1915-1919
    CrossrefPubMedSuche in Google Scholar
  • 32 Coppola A, Santoro C, Tagliaferri A, Franchini M, DI Minno G. Understanding inhibitor development in haemophilia A: towards clinical prediction and prevention strategies. Haemophilia 2010; 16 (Suppl. 01) 13-19
    CrossrefPubMedSuche in Google Scholar
  • 33 Chambost H. Assessing risk factors: prevention of inhibitors in haemophilia. Haemophilia 2010; 16 (Suppl. 02) 10-15
    CrossrefPubMedSuche in Google Scholar
  • 34 Gouw SC, van den Berg HM. The multifactorial etiology of inhibitor development in hemophilia: genetics and environment. Semin Thromb Hemost 2009; 35 (08) 723-734
    Thieme ConnectPubMedSuche in Google Scholar
  • 35 Naess IA, Christiansen SC, Romundstad P, Cannegieter SC, Rosendaal FR, Hammerstrøm J. Incidence and mortality of venous thrombosis: a population-based study. J Thromb Haemost 2007; 5 (04) 692-699
    CrossrefPubMedSuche in Google Scholar
  • 36 Fowkes FJ, Price JF, Fowkes FG. Incidence of diagnosed deep vein thrombosis in the general population: systematic review. Eur J Vasc Endovasc Surg 2003; 25 (01) 1-5
    CrossrefPubMedSuche in Google Scholar
  • 37 Rosendaal FR. Venous thrombosis: prevalence and interaction of risk factors. Haemostasis 1999; 29 (Suppl S1): 1-9
    PubMedSuche in Google Scholar
  • 38 Isma N, Svensson PJ, Gottsäter A, Lindblad B. Prospective analysis of risk factors and distribution of venous thromboembolism in the population-based Malmö Thrombophilia Study (MATS). Thromb Res 2009; 124 (06) 663-666
    CrossrefPubMedSuche in Google Scholar
  • 39 Servey JT, Reamy BV, Hodge J. Clinical presentations of parvovirus B19 infection. Am Fam Physician 2007; 75 (03) 373-376
    PubMedSuche in Google Scholar
  • 40 Klamroth R, Gröner A, Simon TL. Pathogen inactivation and removal methods for plasma-derived clotting factor concentrates. Transfusion 2014; 54 (05) 1406-1417
    CrossrefPubMedSuche in Google Scholar
  • 41 Part 2: General methods for Cochrane reviews: Chapter 7. Selecting studies and collection data. In: Higgins JP, Green S. eds. Cochrane Handbook for Systematic Reviews of Interventions, version 5.1.0 (updated March. 2011 ). The Cochrane Collaboration 2011. Accessed December 2020 at: https://handbook-5-1.cochrane.org
    PubMedSuche in Google Scholar