Anouk Donners

120 Chapter 6 31. Young G, Liesner R, Chang T, Sidonio R, Oldenburg J, Jiménez-Yuste V, Mahlangu J, Kruse-Jarres R, Wang M, Uguen M, Doral MY, Wright LY, Schmitt C, Levy GG, Shima M, Mancuso ME. A multicenter, open-label phase 3 study of emicizumab prophylaxis in children with hemophilia A with inhibitors. Blood. 2019; 134: 2127-2138. 32. Pipe SW, Shima M, Lehle M, Shapiro A, Chebon S, Fukutake K, Key NS, Portron A, Schmitt C, PodolakDawidziak M, Selak Bienz N, Hermans C, Campinha-Bacote A, Kiialainen A, Peerlinck K, Levy GG, JiménezYuste V. Efficacy, safety, and pharmacokinetics of emicizumab prophylaxis given every 4 weeks in people with haemophilia A (HAVEN 4): a multicenter, open-label, non-randomised phase 3 study. Lancet Haematol. 2019; 6: e295-305. 33. Hooimeijer HL, Lukens M V., Verhagen M V., Meijer K, Stein-Wit MA, Tamminga RYJ. A boy with joint pain associated with emicizumab treatment: The importance of plasma level measurement. Haemophilia. 2020: 26: e138-140. 34. Barg AA, Livnat T, Budnik I, Avishai E, Brutman-Barazani T, Tamarin I, Bashari D, Misgav M, Kenet G. Emicizumab treatment and monitoring in a paediatric cohort: real-world data. Br J Haematol. 2020; 191: 282-290. 35. Shima M, Hanabusa H, Taki M, Matsushita T, Sato T, Fukutake K, Fukazawa N, Yoneyama K, Yoshida H, Nogami K. Factor VIII-Mimetic Function of Humanized Bispecific Antibody in Hemophilia A. N Engl J Med. 2016; 374: 2044-2053. 36. Retout S, Schmitt C, Petry C, Mercier F, Frey N. Population Pharmacokinetic Analysis and Exploratory Exposure-Bleeding Rate Relationship of Emicizumab in Adult and Pediatric Persons with Hemophilia A. Clin Pharmacokinet. 2020; 59: 1611-1625. 37. Schmitt C, Adamkewicz JI, Xu J, Petry C, Catalani O, Young G, Negrier C, Callaghan MU, Levy GG. Pharmacokinetics and Pharmacodynamics of Emicizumab in Persons with Hemophilia A with Factor VIII Inhibitors: HAVEN 1 Study. Thromb Haemost. 2020; 121: 351-360. 38. Dirks NL, Meibohm B. Population pharmacokinetics of therapeutic monoclonal antibodies. Clin Pharmacokinet. 2010; 49: 633-659. 39. Kamath A V. Translational pharmacokinetics and pharmacodynamics of monoclonal antibodies. Drug Discov Today Technol. 2016; 21-22: 75-83. 40. Viola M, Sequeira J, Seiça R, Veiga F, Serra J, Santos AC, Ribeiro AJ. Subcutaneous delivery of monoclonal antibodies: How do we get there? J of Control Release. 2018; 286: 301-314. 41. Gill KL, Machavaram KK, Rose RH, Chetty M. Potential Sources of Inter-Subject Variability in Monoclonal Antibody Pharmacokinetics. Clin Pharmacokinet. 2016; 55: 789-805. 42. Jonsson F, Schmitt C, Petry C, Mercier F, Frey N, Retout S. Exposure-Bleeding Count Modeling of Emicizumab for the Prophylaxis of Bleeding in Persons with Hemophilia A with/Without Inhibitors Against Factor VIII. Clin Pharmacokinet. 2021; 60: 931-941. 43. Keipert C, Müller-Olling M, Gauly F, Arras-Reiter C, Hilger A. Annual Bleeding Rates: Pitfalls of Clinical Trial Outcomes in Hemophilia Patients. Clin Transl Sci. 2020; 13: 1127-1136. 44. Ceponis A, Wong-Sefidan I, Glass CS, von Drygalski A. Rapid musculoskeletal ultrasound for painful episodes in adult haemophilia patients. Haemophilia. 2013; 19: 790-798. 45. Kidder W, Nguyen S, Larios J, Bergstrom J, Ceponis A, von Drygalski A. Point-of-care musculoskeletal ultrasound is critical for the diagnosis of hemarthroses, inflammation and soft tissue abnormalities in adult patients with painful haemophilic arthropathy. Haemophilia. 2015; 21: 530-537.

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