Pharmacokinetic-Pharmacodynamic Comparison of Recombinant and Plasma-Derived von Willebrand Factor in Patients with von Willebrand Disease Type 3

References

• Leebeek FW, Eikenboom JC. Von Willebrand’s disease. N Engl J Med. 2016;375(21):2067–2080. doi:10.1056/NEJMra1601561
   PubMed Web of Science ®Google Scholar
• Mannucci PM. Treatment of von Willebrand’s disease. N Engl J Med. 2004;351(7):683–694. doi:10.1056/NEJMra040403
   PubMed Web of Science ®Google Scholar
• Budde U, Metzner HJ, Müller HG. Comparative analysis and classification of von Willebrand factor/factor VIII concentrates: impact on treatment of patients with von Willebrand disease. Semin Thromb Hemost. 2006;32(6):626–635. doi:10.1055/s-2006-949668
   PubMed Web of Science ®Google Scholar
• Reininger AJ. The function of ultra-large von Willebrand factor multimers in high shear flow controlled by ADAMTS13 [Die Funktion von ultragroßen von-Willebrand-Faktor-Multimeren und ihre Regulation durch ADAMTS 13 unter Flussbedingungen mit sehr hohen Scherraten]. Hamostaseologie. 2015;35(3):225–233. doi:10.5482/HAMO-14-12-0077
   PubMed Web of Science ®Google Scholar
• Baxalta US Inc. VONVENDI (von Willebrand factor [recombinant]) lyophilized powder for solution for intravenous injection. Available from: https://www.fda.gov/media/94863/download. Accessed September 26, 2022.
   Google Scholar
• Gill JC, Castaman G, Windyga J, et al. Hemostatic efficacy, safety, and pharmacokinetics of a recombinant von Willebrand factor in severe von Willebrand disease. Blood. 2015;126(17):2038–2046. doi:10.1182/blood-2015-02-629873
   PubMed Web of Science ®Google Scholar
• Peyvandi F, Mamaev A, Wang JD, et al. Phase 3 study of recombinant von Willebrand factor in patients with severe von Willebrand disease who are undergoing elective surgery. J Thromb Haemost. 2019;17(1):52–62. doi:10.1111/jth.14313
   PubMed Web of Science ®Google Scholar
• Baxalta Innovations GmbH. VEYVONDI (vonicog alfa). Summary of product characteristics. Available from: https://www.ema.europa.eu/en/documents/product-information/veyvondi-epar-product-information_en.pdf. Accessed September 26, 2022.
   Google Scholar
• Leebeek FWG, Peyvandi F, Escobar MA, et al. Recombinant von Willebrand factor prophylaxis in patients with severe von Willebrand disease: phase 3 study results. Blood. 2022;140(2):89–98. doi:10.1182/blood.2021014810
   PubMed Web of Science ®Google Scholar
• Bello IF, Yuste VJ, Molina MQ, Navarro FH. Fanhdi, efficacy and safety in von Willebrand’s disease: prospective international study results. Haemophilia. 2007;13(Suppl 5):25–32. doi:10.1111/j.1365-2516.2007.01570.x
   PubMed Web of Science ®Google Scholar
• Rivard GE, Aledort L. Alphanate surgical investigators. Efficacy of factor VIII/von Willebrand factor concentrate Alphanate in preventing excessive bleeding during surgery in subjects with von Willebrand disease. Haemophilia. 2008;14(2):271–275. doi:10.1111/j.1365-2516.2007.01616.x
   PubMed Web of Science ®Google Scholar
• Thompson AR, Gill JC, Ewenstein BM, Mueller-Velten G, Schwartz BA; Humate-P Study Group. Successful treatment for patients with von Willebrand disease undergoing urgent surgery using factor VIII/VWF concentrate (Humate-P). Haemophilia. 2004;10(1):42–51. doi:10.1046/j.1351-8216.2003.00809.x
   PubMed Web of Science ®Google Scholar
• Federici AB, Castaman G, Franchini M, et al. Clinical use of Haemate P in inherited von Willebrand’s disease: a cohort study on 100 Italian patients. Haematologica. 2007;92(7):944–951. doi:10.3324/haematol.11124
   PubMed Web of Science ®Google Scholar
• Franchini M, Rossetti G, Tagliaferri A, et al. Efficacy and safety of factor VIII/von Willebrand’s factor concentrate (Haemate-P) in preventing bleeding during surgery or invasive procedures in patients with von Willebrand disease. Haematologica. 2003;88(11):1279–1283.
   PubMed Web of Science ®Google Scholar
• Castaman G, Linari S. Vonicog alfa for the treatment of von Willebrand disease. Expert Opin Orphan Drugs. 2016;4(5):549–554. doi:10.1517/21678707.2016.1171138
   Web of Science ®Google Scholar
• Castaman G. Treatment of von Willebrand disease with FVIII/VWF concentrates. Blood Transfus. 2011;9(Suppl 2):s9–s13. doi:10.2450/2011.003S
   PubMedGoogle Scholar
• Hazendonk HC, Heijdra JM, de Jager NCB, et al. Analysis of current perioperative management with Haemate® P/Humate P® in von Willebrand disease: identifying the need for personalized treatment. Haemophilia. 2018;24(03):460–470. doi:10.1111/hae.13451
   PubMedGoogle Scholar
• Lethagen S, Kyrle PA, Castaman G, Haertel S, Mannucci PM; HAEMATE P Surgical Study Group. von Willebrand factor/factor VIII concentrate (Haemate P) dosing based on pharmacokinetics: a prospective multicenter trial in elective surgery. J Thromb Haemost. 2007;5(7):1420–1430. doi:10.1111/j.1538-7836.2007.02588.x
   PubMed Web of Science ®Google Scholar
• Mannucci PM, Kempton C, Millar C, et al. Pharmacokinetics and safety of a novel recombinant human von Willebrand factor manufactured with a plasma-free method: a prospective clinical trial. Blood. 2013;122(5):648–657. doi:10.1182/blood-2013-01-479527
   PubMed Web of Science ®Google Scholar
• European Medicines Agency. Guideline on reporting the results of population pharmacokinetic analyses. CHMP/EWP/185990/06. Available from: https://www.ema.europa.eu/en/documents/scientific-guideline/guideline-reporting-results-population-pharmacokinetic-analyses_en.pdf. Accessed November 5, 2020.
   Google Scholar
• US Food and Drug Administration. Population pharmacokinetics. Guidance for industry. Available from: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/population-pharmacokinetics. Accessed September 26, 2022.
   Google Scholar
• Beal SL, Sheiner LB, Boeckmann AJ, Bauer RJ. NONMEM Users’ Guides (1989–2014). Icon Development Solutions; 2014.
   Google Scholar
• R Foundation for Statistical Computing. R: A Language and Environment for Statistical Computing. R Foundation for Statistical Computing; 2019.
   Google Scholar
• de Jager NCB, Bukkems LH, Heijdra JM, et al. One piece of the puzzle: population pharmacokinetics of FVIII during perioperative Haemate P® /Humate P® treatment in von Willebrand disease patients. J Thromb Haemost. 2020;18(2):295–305. doi:10.1111/jth.14652
   PubMed Web of Science ®Google Scholar
• European Medicines Agency. Guideline on the core SPC for human plasma derived von Willebrand factor (CPMP/BPWG/278/02). Available from: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003423.pdf. Accessed October 5, 2020.
   Google Scholar
• Srivastava A, Santagostino E, Dougall A, et al. WFH guidelines for the management of hemophilia. Haemophilia. 2020:26(Suppl 6);1–158. doi:10.1111/hae.14046
   PubMed Web of Science ®Google Scholar
• Favaloro EJ. Towards personalised therapy for von Willebrand disease: a future role for recombinant products. Blood Transfus. 2016;14(2):262–276. doi:10.2450/2016.0258-15
   PubMedGoogle Scholar
• Phua CW, Berntorp E. A personalized approach to the management of VWD. Transfus Apher Sci. 2019;58(5):590–595. doi:10.1016/j.transci.2019.08.009
   PubMed Web of Science ®Google Scholar
• 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(1):301–325. doi:10.1182/bloodadvances.2020003264
   PubMed Web of Science ®Google Scholar
• Mansoor A, Mahabadi N. Volume of Distribution. In: StatPearls. Treasure Island (FL): StatPearls Publishing; 2021. Available from: https://www.ncbi.nlm.nih.gov/books/NBK545280/. Accessed May 28, 2023.
   Google Scholar
• Bukkems LH, Heijdra JM, de Jager NCB, et al. Population pharmacokinetics of the von Willebrand factor-factor VIII interaction in patients with von Willebrand disease. Blood Adv. 2021;5(5):1513–1522. doi:10.1182/bloodadvances.2020003891
   PubMed Web of Science ®Google Scholar
• European Medicines Agency. Veyvondi, summary of product characteristics and public assessment report. Available from: https://www.ema.europa.eu/en/documents/assessment-report/veyvondi-epar-public-assessment-report_en.pdf. Accessed May 28, 2023.
   Google Scholar
• Desprez D, Drillaud N, Flaujac C, et al. Efficacy and safety of a recombinant von Willebrand factor treatment in patients with inherited von Willebrand disease requiring surgical procedures. Haemophilia. 2021;27(2):270–276. doi:10.1111/hae.14242
   PubMed Web of Science ®Google Scholar
• Turecek PL, Mitterer A, Matthiessen HP, et al. Development of a plasma- and albumin-free recombinant von Willebrand factor. Hamostaseologie. 2009;29(Suppl 1):S32–S38. doi:10.1055/s-0037-1617202
   PubMed Web of Science ®Google Scholar
• Abshire T, Cox-Gill J, Kempton CL, et al. Prophylaxis escalation in severe von Willebrand disease: a prospective study from the von Willebrand disease prophylaxis network. J Thromb Haemost. 2015;13(9):1585–1589. doi:10.1111/jth.12995
   PubMed Web of Science ®Google Scholar
• Abshire TC, Federici AB, Alvárez MT, et al. Prophylaxis in severe forms of von Willebrand’s disease: results from the von Willebrand Disease Prophylaxis Network (VWD PN). Haemophilia. 2013;19(1):76–81. doi:10.1111/j.1365-2516.2012.02916.x
   PubMed Web of Science ®Google Scholar
• Berntorp E, Petrini P. Long-term prophylaxis in von Willebrand disease. Blood Coagul Fibrinolysis. 2005;16(Suppl 1):S23–S26. doi:10.1097/01.mbc.0000167659.23262.18
   PubMedGoogle Scholar
• Holm E, Abshire TC, Bowen J, et al. Changes in bleeding patterns in von Willebrand disease after institution of long-term replacement therapy: results from the von Willebrand Disease Prophylaxis Network. Blood Coagul Fibrinolysis. 2015;26(4):383–388. doi:10.1097/MBC.0000000000000257
   PubMed Web of Science ®Google Scholar
• Raquet E, Stockschläder M, Dickneite G. Repeated infusions of VWF/FVIII concentrate: impact of VWF:FVIII ratio on FVIII trough and peak levels in a rabbit model. Haemophilia. 2011;17(5):808–814. doi:10.1111/j.1365-2516.2011.02603.x
   PubMed Web of Science ®Google Scholar
• Srivastava A, Serban M, Werner S, Schwartz BA, Kessler CM. Wonders Study Investigators. Efficacy and safety of a VWF/FVIII concentrate (wilate®) in inherited von Willebrand disease patients undergoing surgical procedures. Haemophilia. 2017;23(2):264–272. doi:10.1111/hae.13106
   PubMed Web of Science ®Google Scholar
• Gill JC, Shapiro A, Valentino LA, et al. von Willebrand factor/factor VIII concentrate (Humate-P) for management of elective surgery in adults and children with von Willebrand disease. Haemophilia. 2011;17(6):895–905. doi:10.1111/j.1365-2516.2011.02534.x
   PubMed Web of Science ®Google Scholar
• Peyvandi F, Kouides P, Turecek PL, Dow E, Berntorp E. Evolution of replacement therapy for von Willebrand disease: from plasma fraction to recombinant von Willebrand factor. Blood Rev. 2019;38:100572. doi:10.1016/j.blre.2019.04.001
   PubMed Web of Science ®Google Scholar
• Castaman G, Linari S. Human von Willebrand factor/factor VIII concentrates in the management of pediatric patients with von Willebrand disease/hemophilia A. Ther Clin Risk Manag. 2016;12:1029–1037. doi:10.2147/TCRM.S87543
   PubMed Web of Science ®Google Scholar