119 Systematic review on PK of emicizumab 16. Moher D, Liberati A, Tetzlaff J, Altman DG, Altman D, Antes G, Atkins D, Barbour V, Barrowman N, Berlin JA, Clark J, Clarke M, Cook D, D’Amico R, Deeks JJ, Devereaux PJ, Dickersin K, Egger M, Ernst E, Gøtzsche PC, et al. Preferred reporting items for systematic reviews and meta-analyses: The PRISMA statement. PLoS Medicine. 2009; 6: e1000097. 17. Ouzzani M, Hammady H, Fedorowicz Z, Elmagarmid A. Rayyan-a web and mobile app for systematic reviews. Syst Rev. 2016; 5: 210. 18. Oldenburg J, Mahlangu JN, Kim B, Schmitt C, Callaghan MU, Young G, Santagostino E, Kruse-Jarres R, Negrier C, Kessler C, Valente N, Asikanius E, Levy GG, Windyga J, Shima M. Emicizumab Prophylaxis in Hemophilia A with Inhibitors. N Engl J Med. 2017; 377: 809-818. 19. Mahlangu J, Oldenburg J, Paz-Priel I, Negrier C, Niggli M, Mancuso ME, Schmitt C, Jiménez-Yuste V, Kempton C, Dhalluin C, Callaghan MU, Bujan W, Shima M, Adamkewicz JI, Asikanius E, Levy GG, KruseJarres R. Emicizumab Prophylaxis in Patients Who Have Hemophilia A without Inhibitors. N Engl J Med. 2018; 378: 811-822. 20. Shima M, Nogami K, Nagami S, Yoshida S, Yoneyama K, Ishiguro A, Suzuki T, Taki M. A multicenter, open-label study of emicizumab given every 2 or 4 weeks in children with severe haemophilia A without inhibitors. Haemophilia. 2019; 5: 979-987. 21. Rohatgi A. Webplotdigitizer: Version 4.4. 2020. 22. Moeyaert M, Maggin D, Verkuilen J. Reliability, Validity, and Usability of Data Extraction Programs for Single-Case Research Designs. Behav Modif. 2016; 40: 874-900. 23. Higgins JPT, Thomas J, Chandler J, Cumpston M, Li T, Page MJ, Welch VA. Cochrane handbook for systematic reviews of interventions. Cochrane Handbook for Systematic Reviews of Interventions. 2019. 2nd ed. New York: Wiley. 24. Yoneyama K, Schmitt C, Kotani N, Levy GG, Kasai R, Iida S, Shima M, Kawanishi T. A Pharmacometric Approach to Substitute for a Conventional Dose-Finding Study in Rare Diseases: Example of Phase III Dose Selection for Emicizumab in Hemophilia A. Clin Pharmacokinet. 2018; 57: 1123-1134. 25. Ferrière S, Peyron I, Christophe OD, Kawecki C, Casari C, Muczynski V, Nathwani A, Kauskot A, Lenting PJ, Denis C V. A hemophilia A mouse model for the in vivo assessment of emicizumab function. Blood. 2020; 136: 740-74. 26. Lenting PJ. Laboratory monitoring of hemophilia A treatments: New challenges. Blood Adv. 2020; 12; 4: 2111-2118. 27. Uchida N, Sambe T, Yoneyama K, Fukazawa N, Kawanishi T, Kobayashi S, Shima M. A first-in-human phase 1 study of ACE910, a novel factor VIII-mimetic bispecific antibody, in healthy subjects. Blood. 2016; 127: 1633-1641. 28. Shima M, Hanabusa H, Taki M, Matsushita T, Sato T, Fukutake K, Kasai R, Yoneyama K, Yoshida H, Nogami K. Long-term safety and efficacy of emicizumab in a phase 1/2 study in patients with hemophilia A with or without inhibitors. Blood Adv. 2017; 1: 1891-1899. 29. Kotani N, Yoneyama K, Kawakami N, Shimuta T, Fukase H, Kawanishi T. Relative and Absolute Bioavailability Study of Emicizumab to Bridge Drug Products and Subcutaneous Injection Sites in Healthy Volunteers. Clin Pharmacol Drug Dev. 2019; 8: 702-712. 30. Li H, Zhang W, Petry C, Li L, Fernandez E, Kiialainen A, Feng S, Hsu W, Li L, Wei Y, Schmitt C. Evaluation of the Pharmacokinetics, Pharmacodynamics, and Safety of a Single Dose of Emicizumab in Healthy Chinese Subjects. Clin Pharmacol Drug Dev. 2021; 10: 30-38. 6
RkJQdWJsaXNoZXIy MTk4NDMw