Anouk Donners

20 Chapter 1 17. Fischer K, Steen Carlsson K, Petrini P, Holmstrom M, Ljung R, van den Berg HM, Berntorp E. Intermediatedose versus high-dose prophylaxis for severe hemophilia: comparing outcome and costs since the 1970s. Blood. 2013; 122: 1129-1136. 18. Pierce GF, Adediran M, Diop S, Dunn AL, El Ekiaby M, Kaczmarek R, Konkle BA, Pipe SW, Skinner MW, Valentino LA, Robinson F, Ampartzidis G, Martin J, Haffar A. Achieving access to haemophilia care in low-income and lower-middle-income countries: expanded Humanitarian Aid Program of the World Federation of Hemophilia after 5 years. Lancet Haematol. 2022; 9: e689-e697. 19. Kizilocak H, Marquez-Casas E, Malvar J, Carmona R, Young G. Determining the approximate factor VIII level of patients with severe haemophilia A on emicizumab using in vivo global haemostasis assays. Haemophilia. 2021; 27: 730-735. 20. Lenting PJ. Laboratory monitoring of hemophilia A treatments: new challenges. Blood Adv. 2020; 4: 2111-2118. 21. Ferriere S, Peyron I, Christophe OD, Kawecki C, Casari C, Muczynski V, Nathwani A, Kauskot A, Lenting PJ, Denis CV. A hemophilia A mouse model for the in vivo assessment of emicizumab function. Blood. 2020; 136: 740-748. 22. Kizilocak H, Marquez-Casas E, Malvar J, Young G. Safety of FEIBA and emicizumab (SAFE): Dose escalation study evaluating the safety of in vivo administration of activated prothrombin complex concentrate in haemophilia A patients on emicizumab. Haemophilia. 2023; 29: 100-105. 23. Schmitt C, Emrich T, Chebon S, Fernandez E, Petry C, Yoneyama K, Kiialainen A, Howard M, Niggli M, Paz-Priel I, Chang T. Low immunogenicity of emicizumab in persons with haemophilia A. Haemophilia. 2021; 27: 984-992. 24. 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 20-10-2022. 25. ZIN. Horizonscan - emicizumab for routine prophylaxis of bleeding episodes in patients with haemophilia A (congenital factor VIII deficiency): mild or moderate disease for whom prophylaxis is clinically indicated, version 2. Accessed on 10-01-2023. 2022; online ahead of print. 26. Oka G, Pieragostini R, Roussel-Robert V, Paubel P, Degrassat-Theas A, Lopez I. [Assessment of the budgetary impact of an emicizumab therapy introduction for patients with severe haemophilia A without inhibitor]. Ann Pharm Fr. 2022; online ahead of print. 27. Swan D, Mahlangu J, Thachil J. Non-factor therapies for bleeding disorders: A primer for the general haematologist. EJHaem. 2022; 3: 584-595. 28. Gualtierotti R, Pasca S, Ciavarella A, Arcudi S, Giachi A, Garagiola I, Suffritti C, Siboni SM, Peyvandi F. Updates on Novel Non-Replacement Drugs for Hemophilia. Pharmaceuticals (Basel). 2022; 15. 29. RIVM. De ziekten die de hielprik opspoort. Accessed on 01-06-2022. 30. Spadarella G, Di Minno A, Brunetti-Pierri N, Mahlangu J, Di Minno G. The evolving landscape of gene therapy for congenital haemophilia: An unprecedented, problematic but promising opportunity for worldwide clinical studies. Blood Rev. 2021; 46: 100737.