[Cost-minimization analysis of replacement therapy in the treatment of von Willebrand disease]
DOI:
https://doi.org/10.7175/fe.v17i2.1249Keywords:
von Willebrand disease, von Willebrand factor, Factor VIIIctor, Factor VIIIAbstract
BACKGROUND: Replacement therapy with von Willebrand factor (VWF)/factor VIII (FVIII) concentrates represents an effective approach for patients with von Willebrand disease (VWD) who are unresponsive to desmopressin. However, various concentrates are available, with heterogeneous VWF content and VWF/FVIII ratio.
AIM: To compare the costs associated to the replacement therapy with VWF/FVIII concentrates in Italy.
METHODS: A cost-minimization analysis was performed to compare the pharmaceutical costs per patient of alternatives available for replacement therapy of VWD in the prospective of the Italian National Health Service. For each alternative the analysis calculated the number of vials, and relative costs, required to reach the target levels of VWF:RCo in patients who undergone to major surgery, minor surgery, spontaneous bleeding and prophylaxis.
RESULTS: Haemate P® is associated with the lowest FVIII dosage, numbers of vials used and costs in all the clinical situations and at all the dosages considered. With Haemate P® the average costs in major surgery, minor surgery, spontaneous bleeding, and prophylaxis was € 710.94, € 592.45, € 473.96, and € 592.45, respectively. While the costs associated to Fanhdi®, Wilate®, and Wilfactin® was: € 1,309.28, € 1,071.23, € 952.20, and € 1,190.25; € 1,512.45, € 1,344.40, € 1,176.35, and € 1,344.40; € 3,814.09, € 3,269.22, € 3,269.22, and € 3,814.09.
CONCLUSIONS: Treatment with Haemate P®, which presents a low FVIII content, allows to reach the target level of VWF:RCo with a lower number of vials and lower costs for the NHS.
[Article in Italian]
References
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: 667-74; http://dx.doi.org/10.3324/haematol.2012.077263
Istituto Superiore di Sanità. Registro Nazionale Coagulopatie Congenite. Rapporto 2013. Rapporto ISTISAN 15/14; 2015
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: 2038-46; http://dx.doi.org/10.1182/blood-2015-02-629873
Raquet E, Stockschlaeder M, Mueller-Cohrs J, et al. Utility of a high VWF: FVIII ratio in preventing FVIII accumulation: a study in VWF-deficient mice. Blood Coagul Fibrinolysis 2015; 26: 515-21; http://dx.doi.org/10.1097/MBC.0000000000000269
Mannucci PM, Franchini M, Castaman G, et al.; Italian Association of Hemophilia Centers. Evidence-based recommendations on the treatment of von Willebrand disease in Italy. Blood Transfus 2009; 7: 117-26; http://dx.doi.org/10.2450/2008.0052-08
Rodeghiero F, Castaman G, Tosetto A, et al. The discriminant power of bleeding history for the diagnosis of type 1 von Willebrand disease: an international, multicenter study. J Thromb Haemost 2005; 3: 2619-26; http://dx.doi.org/10.1111/j.1538-7836.2005.01663.x
Tosetto A, Castaman G. How I treat type 2 variant forms of von Willebrand disease. Blood 2015; 125: 907-14; http://dx.doi.org/10.1182/blood-2014-08-551960
Castaman G. Treatment of von Willebrand disease with FVIII/VWF concentrates. Blood Transfus 2011; 9 Suppl 2: s9-13; http://dx.doi.org/10.2450/2011.003S
Rodeghiero F, Castaman G, Tosetto A. How I treat von Willebrand disease. Blood 2009; 114: 1158-65; http://dx.doi.org/10.1182/blood-2009-01-153296
Lethagen S, Carlson M, Hillarp A. A comparative in vitro evaluation of six von Willebrand factor concentrates. Haemophilia 2004; 10: 243-9; http://dx.doi.org/10.1111/j.1365-2516.2004.00893.x
Haemate P® – Riassunto delle Caratteristiche di Prodotto
Wilfactin®– Riassunto delle Caratteristiche di Prodotto
Goudemand J, Scharrer I, Berntorp E, et al. Pharmacokinetic studies on Wilfactin, a von Willebrand factor concentrate with a low factor VIII content treated with three virus-inactivation/removal methods. J Thromb Haemost 2005; 3: 2219-27; http://dx.doi.org/10.1111/j.1538-7836.2005.01435.x
Borel-Derlon A, Federici AB, Roussel-Robert V, et al. Treatment of severe von Willebrand disease with a high-purity von Willebrand factor concentrate (Wilfactin): a prospective study of 50 patients. J Thromb Haemost 2007; 5: 1115-24; http://dx.doi.org/10.1111/j.1538-7836.2007.02562.x
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: 626-35; http://dx.doi.org/10.1055/s-2006-949668
Berntorp E, Archey W, Auerswald G, et al. A systematic overview of the first pasteurised VWF/FVIII medicinal product, Haemate P/ Humate -P: history and clinical performance. Eur J Haematol Suppl 2008; 70: 3-35; http://dx.doi.org/10.1111/j.1600-0609.2008.01049.x
Eandi M, Pradelli L, Zaniolo O. Farmacoeconomia: principi di base. Torino: SEEd, 2006
Bello IF, Yuste VJ, Molina MQ, et al. Fanhdi, efficacy and safety in von Willebrand’s disease: prospective international study results. Haemophilia 2007; 13 Suppl 5: 25-32; http://dx.doi.org/10.1111/j.1365-2516.2007.01570.x
Lethagen S, Kyrle PA, Castaman G, et al.; 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: 1420-30; http://dx.doi.org/10.1111/j.1538-7836.2007.02588.x
Berntorp E, Windyga J; European Wilate Study Group. Treatment and prevention of acute bleedings in von Willebrand disease--efficacy and safety of Wilate, a new generation von Willebrand factor/factor VIII concentrate. Haemophilia 2009; 15: 122-30; http://dx.doi.org/10.1111/j.1365-2516.2008.01901.x
Codifa. L’informatore Farmaceutico. Ultimo accesso febbraio 2016
Regione Lombardia. ASL Milano. Deliberazione n.1366 del 30/09/2015. Disponibile su http://www.asl.milano.it/user/download.aspx?FILE=OBJ13165.PDF&TIPO=FLE&NOME=1366_2015.pdf (ultimo accesso Febbraio 2016)
Fanhdi® – Riassunto delle Caratteristiche di Prodotto
Wilate® – Riassunto delle Caratteristiche di Prodotto
Published
How to Cite
Issue
Section
License
Authors who publish with this journal agree to the following terms:
- Authors retain copyright and grant the journal right of first publication with the work simultaneously licensed under a Creative Commons Attribution-NonCommercial 4.0 License that allows others to share the work with an acknowledgement of the work's authorship and initial publication in this journal.
- Authors are able to enter into separate, additional contractual arrangements for the non-exclusive distribution of the journal's published version of the work (e.g., post it to an institutional repository or publish it in a book), with an acknowledgement of its initial publication in this journal. The Publication Agreement can be downloaded here, and should be signed by the Authors and sent to the Publisher when the article has been accepted for publication in this journal.
- Authors are permitted and encouraged to post their work online (e.g., in institutional repositories or on their website) prior to and during the submission process, as it can lead to productive exchanges, as well as earlier and greater citation of published work (see The Effect of Open Access).
- Authors are permitted to post their work online after publication (the article must link to publisher version, in html format)
