Anouk Donners

138 Chapter 7 REFERENCES 1. Callaghan MU, Negrier C, Paz-Priel I, Chang T, Chebon S, Lehle M, Mahlangu J, Young G, Kruse-Jarres R, Mancuso ME, Niggli M, Howard M, Bienz NS, Shima M, Jimenez-Yuste V, Schmitt C, Asikanius E, Levy GG, Pipe SW, Oldenburg J. Long-term outcomes with emicizumab prophylaxis for hemophilia A with or without FVIII inhibitors from the HAVEN 1-4 studies. Blood. 2021; 137: 2231-2242. 2. Jimenez-Yuste V, Auerswald G, Benson G, Dolan G, Hermans C, Lambert T, Ljung R, Morfini M, Santagostino E, Zupancic Salek S. Practical considerations for nonfactor-replacement therapies in the treatment of haemophilia with inhibitors. Haemophilia. 2021; 27: 340-350. 3. Cafuir L, Estrin A, Chen E, Hinds D, Prince P, Thorburn J, Mead H, Kempton CL. Early real-world experience with emicizumab and concomitant factor VIII replacement products in adult males with Hemophilia A without inhibitors. J Med Econ. 2022; 25: 984-992. 4. Mancuso ME, Castaman G, Pochopien M, Aballea S, Drzewiecka A, Hakimi Z, Nazir J, Fatoye F. Costminimization analysis of recombinant factor VIII Fc versus emicizumab for treating patients with hemophilia A without inhibitors in Europe. J Med Econ. 2022; 25: 1068-1075. 5. ZIN. Horizonscan - emicizumab voor routinematige profylaxe van bloedingen bij patiënten met hemofilie A zonder remmers tegen factor VIII, versie 6. geneesmiddelen/emicizumab-cardiovasculaire-aandoeningen-hemostase_bevorderende_medicatie/ versie6. Accessed on 08-12-2020. 6. European Medicines Agency. Hemlibra (emicizumab) Summary of Product Characteristics. 2018. https:// Accessed on 08-12-2020. 7. Mahlangu J, Iorio A, Kenet G. Emicizumab state-of-the-art update. Haemophilia. 2022; 28: 103-110. 8. Bukkems LH, Fischer K, Kremer-Hovinga I, Donners AAM, Fijnvandraat K, Schutgens REG, Cnossen MH, Mathot RAA. Emicizumab Dosing in Children and Adults with Hemophilia A: Simulating a User-Friendly and Cost-Efficient Regimen. Thromb Haemost. 2022; 122: 208-215. 9. Yu JK, Iorio A, Chelle P, Edginton AN. Pharmacokinetic implications of dosing emicizumab based on vial size: A simulation study. Haemophilia. 2021; 27: 358-365. 10. Srivastava A, Brewer AK, Mauser-Bunschoten EP, Key NS, Kitchen S, Llinas A, Ludlam CA, Mahlangu JN, Mulder K, Poon MC, Street A, Treatment Guidelines Working Group on Behalf of The World Federation Of H. Guidelines for the management of hemophilia. Haemophilia. 2013; 19: e1-47. 11. Amrani ME, Gerencser L, Huitema ADR, Hack CE, van Luin M, van der Elst KCM. A generic sample preparation method for the multiplex analysis of seven therapeutic monoclonal antibodies in human plasma or serum with liquid chromatography-tandem mass spectrometry. J Chromatogr A. 2021; 1655: 462489. 12. Donners A, Gerencser L, van der Elst KCM, Egberts TCG, de Maat MPM, Huisman A, Urbanus RT, El Amrani M. Quantification of emicizumab by mass spectrometry in plasma of people with hemophilia A: A method validation study. Res Pract Thromb Haemost. 2022; 6: e12725. 13. Blanchette VS, Key NS, Ljung LR, Manco-Johnson MJ, van den Berg HM, Srivastava A, Subcommittee on Factor Viii FIX, Rare Coagulation Disorders of the S, Standardization Committee of the International Society on T, Hemostasis. Definitions in hemophilia: communication from the SSC of the ISTH. J Thromb Haemost. 2014; 12: 1935-1939.