Anouk Donners

19 General Introduction References 1. Mannucci PM, Tuddenham EG. The hemophilias--from royal genes to gene therapy. N Engl J Med. 2001; 344: 1773-1779. 2. Stichting_HemoNED. Jaarrapportage 2021. Data en trends vanuit het Nederlands Hemofilie Register en het digitale logboek VastePrik. https://hemoned.nl/publicaties/jaarrapportages/. Accessed on 2010-2022. 3. Berntorp E, Fischer K, Hart DP, Mancuso ME, Stephensen D, Shapiro AD, Blanchette V. Haemophilia. Nat Rev Dis Primers. 2021; 7: 45. 4. Srivastava A, Santagostino E, Dougall A, Kitchen S, Sutherland M, Pipe SW, Carcao M, Mahlangu J, Ragni MV, Windyga J, Llinas A, Goddard NJ, Mohan R, Poonnoose PM, Feldman BM, Lewis SZ, van den Berg HM, Pierce GF, panelists WFHGftMoH, co a. WFH Guidelines for the Management of Hemophilia, 3rd edition. Haemophilia. 2020; 26 Suppl 6: 1-158. 5. Aledort L, Mannucci PM, Schramm W, Tarantino M. Factor VIII replacement is still the standard of care in haemophilia A. Blood Transfus. 2019; 17: 479-486. 6. Marchesini E, Morfini M, Valentino L. Recent Advances in the Treatment of Hemophilia: A Review. Biologics. 2021; 15: 221-235. 7. Farrugia A, Smit C, Buzzi A. The legacy of haemophilia: Memories and reflections from three survivors. Haemophilia. 2022; 28: 872-884. 8. O’Mahony B. Haemophilia care in Europe: Past progress and future promise. Haemophilia. 2020; 26: 752-758. 9. Mannucci PM. Hemophilia therapy: the future has begun. Haematologica. 2020; 105: 545-553. 10. Versloot O, Iserman E, Chelle P, Germini F, Edginton AN, Schutgens REG, Iorio A, Fischer K, Pharmacokinetic Expert Working Group of the International Prophylaxis Study G. Terminal half-life of FVIII and FIX according to age, blood group and concentrate type: Data from the WAPPS database. J Thromb Haemost. 2021; 19: 1896-1906. 11. Franchini M, Mannucci PM. The More Recent History of Hemophilia Treatment. Semin Thromb Hemost. 2022; 48: 904-910. 12. Graf L. Extended Half-Life Factor VIII and Factor IX Preparations. Transfus Med Hemother. 2018; 45: 86-91. 13. CBG-MEB. Geneesmiddeleninformatiebank ATC code B02BD02. https://www. geneesmiddeleninformatiebank.nl. Accessed on 20-10-2022. 14. Ay C, Perschy L, Rejto J, Kaider A, Pabinger I. Treatment patterns and bleeding outcomes in persons with severe hemophilia A and B in a real-world setting. Ann Hematol. 2020; 99: 2763-2771. 15. Den Uijl IE, Mauser Bunschoten EP, Roosendaal G, Schutgens RE, Biesma DH, Grobbee DE, Fischer K. Clinical severity of haemophilia A: does the classification of the 1950s still stand? Haemophilia. 2011; 17: 849-853. 16. Tiede A, Abdul Karim F, Jimenez-Yuste V, Klamroth R, Lejniece S, Suzuki T, Groth A, Santagostino E. Factor VIII activity and bleeding risk during prophylaxis for severe hemophilia A: a population pharmacokinetic model. Haematologica. 2021; 106: 1902-1909. 1

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