Anouk Donners

96 Chapter 5 REFERENCES 1. Mannucci PM, Tuddenham EG. The hemophilias--from royal genes to gene therapy. N Engl J Med. 2001; 344: 1773-1779. 2. 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. 3. Ljung R, Gretenkort Andersson N. The current status of prophylactic replacement therapy in children and adults with haemophilia. Br J Haematol. 2015; 169: 777-786. 4. Beeton K, Neal D, Watson T, Lee CA. Parents of children with haemophilia--a transforming experience. Haemophilia. 2007; 13: 570-579. 5. Hassan S, Monahan RC, Mauser-Bunschoten EP, van Vulpen LFD, Eikenboom J, Beckers EAM, Hooimeijer L, Ypma PF, Nieuwenhuizen L, Coppens M, Schols SEM, Leebeek FWG, Smit C, Driessens MH, le Cessie S, van Balen EC, Rosendaal FR, van der Bom JG, Gouw SC. Mortality, life expectancy, and causes of death of persons with hemophilia in the Netherlands 2001-2018. J Thromb Haemost. 2021; 19: 645-653. 6. Zimmerman B, Valentino LA. Hemophilia: in review. Pediatr Rev. 2013; 34: 289-294. 7. Franchini M, Mannucci PM. Non-factor replacement therapy for haemophilia: a current update. Blood Transfus. 2018; 16: 457-461. 8. 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. 9. Kitazawa T, Igawa T, Sampei Z, Muto A, Kojima T, Soeda T, Yoshihashi K, Okuyama-Nishida Y, Saito H, Tsunoda H, Suzuki T, Adachi H, Miyazaki T, Ishii S, Kamata-Sakurai M, Iida T, Harada A, Esaki K, Funaki M, Moriyama C, Tanaka E, Kikuchi Y, Wakabayashi T, Wada M, Goto M, Toyoda T, Ueyama A, Suzuki S, Haraya K, Tachibana T, Kawabe Y, Shima M, Yoshioka A, Hattori K. A bispecific antibody to factors IXa and X restores factor VIII hemostatic activity in a hemophilia A model. Nat Med. 2012; 18: 1570-1574. 10. Rodriguez-Merchan EC, Valentino LA. Emicizumab: Review of the literature and critical appraisal. Haemophilia. 2019; 25: 11-20. 11. European Medicines Agency. Hemlibra (emicizumab) Summary of Product Characteristics. 2018. https:// www.ema.europa.eu/en/documents/product-information/hemlibra-epar-product-information_en.pdf. Accessed on 08-10-2021. 12. Tripodi A, Santoro RC, Testa S, Molinari AC, Bernardini S, Golato M, Lippi G, Ageno W, Santagostino E. Position paper on laboratory testing for patients with haemophilia. A consensus document from SISET, AICE, SIBioC and SIPMeL. Blood Transfus. 2019; 17: 229-236. 13. Jenkins PV, Bowyer A, Burgess C, Gray E, Kitchen S, Murphy P, Platton S, Riddell A, Chowdary P, Lester W. Laboratory coagulation tests and emicizumab treatment A United Kingdom Haemophilia Center Doctors’ Organisation guideline. Haemophilia. 2020; 26: 151-155. 14. Holstein K, Albisetti M, Bidlingmaier C, Halimeh S, Heine S, Klamroth R, Konigs C, Kurnik K, Male C, Oldenburg J, Streif W, Wermes C, Escuriola-Ettingshausen C, Standige Kommission Hamophilie’ of the German SASfTHR. Practical Guidance of the GTH Haemophilia Board on the Use of Emicizumab in Patients with Haemophilia A. Hamostaseologie. 2020; 40: 561-571.

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