References
- Srivastava A, Santagostino E, Dougall A, the WFH Guidelines for the Management of Hemophilia panelists and co-authors, et al. WFH guidelines for the management of hemophilia. Haemophilia. 2020;26(S6):1–158.
- Abbonizio F, Arcieri R, Associazione Italiana Centri Emofilia (AICE), et al. Registro Nazionale delle Coagulopatie Congenite (Rapporto 2018. (Rapporti ISTISAN 20/14). Roma: istituto Superiore di Sanità; 2020. p. 53.
- Berntorp E, Fischer K, Hart DP, et al. Haemophilia. Nat Rev Dis Primers. 2021;7(1):45.
- Franchini M, Mannucci PM. Hemophilia a in the third millennium. Blood Rev. 2013;27(4):179–184.
- Manco-Johnson M, Abshire T, Shapiro A, et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. NJEM. 2007;9(357):535–544.
- Kodra Y, Cavazza M, Schieppati A, et al. The social burden and quality of life of patients with haemophilia in Italy. Blood Transfus. 2014;12(Suppl 3):S567–S75.
- O’Hara J, Hughes D, Camp C, et al. The cost of severe haemophilia in Europe: the CHESS study. Orphanet J Rare Dis. 2017;12(1):106.
- Zollner SB, Raquet E, Muller-Cohrs J, et al. Preclinical efficacy and safety of rVIII-SingleChain (CSL627), a novel recombinant single-chain factor VIII. Thromb Res. 2013;132(2):280–287.
- Raso S, Hermans C. Lonoctocog alfa (rVIII-SingleChain) for the treatment of haemophilia A. Expert Opin Biol Ther. 2018;18(1):87–94.
- Mahlangu J, Kuliczkowski K, Karim FA, AFFINITY Investigators, et al. Efficacy and safety of rVIII-SingleChain: results of a phase 1/3 multicenter clinical trial in severe hemophilia A. Blood. 2016;128(5):630–637.
- Santagostino E, Fischer K, Koenigs C, et al. Interim analysis of the extension study with rVIII-SingleChain in previously untreated patients (PUPs) with severe hemophilia A (CSL627-3001). Blood. 2019;134(Supplement_1):162–162.
- Stasyshyn O, Djambas Khayat C, Iosava G, et al. Safety, efficacy and pharmacokinetics of rVIII-SingleChain in children with severe hemophilia A: results of a multicenter clinical trial. J Thromb Haemost. 2017;15(4):636–644.
- Hassoun AA. Real-World experience of rVIII-SingleChain treatment in a French single center. Res Pract Thromb Haemost. 2020;4(Suppl 1):PB0980.
- Simpson ML, Desai V, Maro GS, et al. Comparing factor use and bleed rates in U.S. Hemophilia a patients receiving prophylaxis with 3 different long-acting recombinant factor VIII products. J Manag Care Spec Pharm. 2020;26(4):504–512.
- Yan S, Maro GS, Desai V, et al. A real-world analysis of commonly prescribed FVIII products based on US medical charts: consumption and bleeding outcomes in hemophilia A patients. J Manag Care Spec Pharm. 2020;26(10):1258–1265.
- Olivieri M, Simpson M, Yan S, et al. Analysis of pooled real-world data from Germany, Italy, and the United States of rVIII-SingleChain compared with standard- and long-acting FVIII products for prophylaxis of hemophilia A. Curr Med Res Opin. 2022;38(7):1133–1139.
- [online] BIA. York; York Health Economics Consortium 2016 [cited 2022 April]. Available from: https://yhec.co.uk/glossary/budget-impact-analysis/.
- Abbonizio F, Giampaolo A, Arcieri R, et al. Registro Nazionale delle Coagulopatie Congenite. Rapporto 2013. (Rapporti ISTISAN 15/14). Roma: Istituto Superiore di Sanità; 2015.
- Abbonizio F, Giampaolo A, Arcieri R, et al. Registro Nazionale delle Coagulopatie Congenite. Rapporto 2014. (Rapporti ISTISAN 16/20). Roma: Istituto Superiore di Sanità; 2016.
- Abbonizio F, Giampaolo A, Arcieri R, et al. Registro Nazionale delle Coagulopatie Congenite. Rapporto 2015. (Rapporti ISTISAN 17/14). Istituto Superiore di Sanità; 2017.
- Abbonizio F, Giampaolo A, Riccioni R, et al. Registro Nazionale delle Coagulopatie Congenite. Rapporto 2016. (Rapporti ISTISAN 17/44). Istituto Superiore di Sanità; 2017.
- Abbonizio F, Hassan H, Riccioni R, et al. Registro Nazionale delle Coagulopatie Congenite. Rapporto 2017. (Rapporti ISTISAN 19/8). Istituto Superiore di Sanità; 2019.
- Berntorp E, Dolan G, Hay C, et al. European retrospective study of real-life haemophilia treatment. Haemophilia. 2017;23(1):105–114.
- McMullen S, Buckley B, Hall E, 2nd, et al. Budget impact analysis of prolonged Half-Life recombinant FVIII therapy for hemophilia in the United States. Value Health. 2017;20(1):93–99. Jan
- Fujii T, Kidoguchi Y, Takahashi N, et al. Budget impact analysis of jivi (Damoctocog alfa pegol, Bay 94-9027) in severe hemophilia a in Japan. J Med Econ. 2021;24(1):218–225.
- Watanabe A, Huey Lee S, Chai-Adisaksopha C, et al. Budget impact of emicizumab for routine prophylaxis of bleeding episodes in patients with hemophilia a with inhibitors. Value Health Reg Issues. 2022;28:7–13.
- Lorenzoni V, Triulzi I, Turchetti G. Budget impact analysis of the use of extended half-life recombinant factor VIII (efmoroctocog alfa) for the treatment of congenital haemophilia a: the Italian National Health System perspective. BMC Health Serv Res. 2018;18(1):596.
- European Medicines Agency. Summary of product characteristics – AFSTYLA; 2019. Available from: https://www.ema.europa.eu/en/documents/product-information/afstyla-epar-product-information_en.pdf.
- European Medicine Agency. Summary of product characteristics – ELOCTA; 2020. Available from: https://www.ema.europa.eu/en/documents/product-information/elocta-epar-product-information_en.pdf.
- European Medicines Agency. Summary of product characteristics – ADYNOVI; 2019. Available from: https://www.ema.europa.eu/documents/product-information/adynovi-epar-product-information_en.pdf.
- European Medicines Agency. Summary of product characteristics – NUWIQ. 2019. Available from: https://www.ema.europa.eu/documents/product-information/nuwiq-epar-product-information_en.pdf.
- European Medicines Agency. Summary of product characteristics – REFACTO AF. 2019. Available from: https://www.ema.europa.eu/documents/product-information/refacto-af-epar-product-information_en.pdf.
- European Medicines Agency. Summary of product characteristics – JIVI; 2020. Available from: https://www.ema.europa.eu/documents/product-information/jivi-epar-product-information_en.pdf.
- European Medicines Agency. Summary of product characteristics – ESPEROCT; 2020. Available from: https://www.ema.europa.eu/documents/product-information/esperoct-epar-product-information_en.pdf.
- European Medicines Agency. Summary of product characteristics – KOVALTRY; 2020. Available from: https://www.ema.europa.eu/en/documents/product-information/kovaltry-epar-product-information_en.pdf.
- European Medicines Agency. Summary of product characteristics – ADVATE; 2020. Available from: https://www.ema.europa.eu/documents/product-information/advate-epar-product-information_en.pdf.
- European Medicines Agency. Summary of product characteristics – NOVOEIGHT; 2020. Available from: https://www.ema.europa.eu/documents/product-information/novoeight-epar-product-information_en.pdf.
- European Medicines Agency. Summary of product characteristics – HEMLIBRA; 2021. Available from: https://www.ema.europa.eu/documents/product-information/hemlibra-epar-product-information_en.pdf.
- Gazzetta Ufficiale Serie Generale. 158(191), 17-08-2017. [cited]. Available from: https://www.gazzettaufficiale.it/eli/gu/2017/08/17/191/sg/pdf.
- Gazzetta Ufficiale Serie Generale. 157(174), 27-07-2016. Available from: https://www.gazzettaufficiale.it/eli/gu/2016/07/27/174/sg/pdf.
- Gazzetta Ufficiale Serie Generale. 161(14), 18-01-2020. Available from: https://www.gazzettaufficiale.it/eli/gu/2020/01/18/14/sg/pdf.
- Gazzetta Ufficiale Serie Generale. 161(21), 27-01-2020. Available from: https://www.gazzettaufficiale.it/eli/gu/2020/01/27/21/sg/pdf.
- Gazzetta Ufficiale Serie Generale. 178(76), 16-07-2020. Available from: https://www.gazzettaufficiale.it/eli/gu/2020/07/16/178/so/24/sg/pdf.
- Gazzetta Ufficiale Serie Generale. 163(52), 03-03-2022. Available from: https://www.gazzettaufficiale.it/eli/gu/2022/03/03/52/sg/pdf.
- Gazzetta Ufficiale Serie Generale. 156(125), 01-06-2015. Available from: https://www.gazzettaufficiale.it/eli/gu/2015/06/01/125/sg/pdf.
- Gazzetta Ufficiale Serie Generale. 156(198), 27-08-2015. Available from: https://www.gazzettaufficiale.it/eli/gu/2015/08/27/198/sg/pdf.
- Gazzetta Ufficiale Serie Generale. 152(298), 23-12-2011. Available from: https://www.gazzettaufficiale.it/eli/gu/2011/12/23/298/sg/pdf.
- Gazzetta Ufficiale Serie Generale. 155(190), 18-08-2014. Available from: https://www.gazzettaufficiale.it/eli/gu/2014/08/18/190/sg/pdf.
- Gazzetta Ufficiale Serie Generale. 161(71), 18-03-2020. Available from: https://www.gazzettaufficiale.it/eli/gu/2020/03/18/71/sg/pdf.
- Biogen Canada Inc. Product monograph – ELOCTATE; 2016. Available from: https://www.biogen.ca/content/dam/corporate/en_CA/pdfs/products/ELOCTATE/ELOCTATE_PM_E.pdf.
- Novo Nordisk Canada Inc. Product monograph – ESPEROCT; 2020. Available from: https://www.novonordisk.ca/content/dam/nncorp/ca/en/products/esperoct-product-monograph.pdf.
- Octapharma Canada Inc. Product monograph – NUWIQ; 2018. Available from: https://pdf.hres.ca/dpd_pm/00044390.pdf.
- Shire Pharma Canada ULC. Product monograph – ADVATE; 2018. Available from: https://pdf.hres.ca/dpd_pm/00046596.pdf.