References
- Franchini M. Current management of hemophilia B: recommendations, complications and emerging issues. Expert Rev Hematol. 2014;7(5):573–581. doi:10.1586/17474086.2014.947955
- World Federation of Hemophilia. Report on the annual global survey 2021; 2022; Available from: https://www1.wfh.org/publications/files/pdf-2324.pdf. Accessed November 20, 2023.
- Mancuso ME, Santagostino E. Outcome of clinical trials with new extended half-life FVIII/IX concentrates. J Clin Med. 2017;6(4):E39. doi:10.3390/jcm6040039
- Fukutake K, Taki M, Matsushita T, et al. Postmarketing safety and effectiveness of recombinant factor IX (nonacog alfa) in Japanese patients with haemophilia B. Haemophilia. 2019;25(4):e247–e256. doi:10.1111/hae.13783
- Pharmaceuticals and Medical Devices Agency. Refixia (nonacog beta pegol) Japanese regulatory review: report on the deliberation results; 2018; Available from: https://www.pmda.go.jp/files/000238159.pdf. Accessed February 8, 2023.
- Pharmaceuticals and Medical Devices Agency. Idelvion (albutrepenonacog alfa) Japanese regulatory review: report on deliberation results; 2016; Available from: https://www.pmda.go.jp/files/000221483.pdf. Accessed February 8, 2023.
- Biogen. Alprolix (eftrenonacog alfa); 2016; Available from: https://www.pharmacodia.com/yaodu/html/v1/biologics/1e5e1435c95e420a1cd34d3202769c18.html. Accessed February 8, 2023.
- Pelland-Marcotte MC, Carcao MD. Hemophilia in a changing treatment landscape. Hematol Oncol Clin North Am. 2019;33(3):409–423. doi:10.1016/j.hoc.2019.01.007
- Mannucci PM. Hemophilia therapy: the future has begun. Haematologica. 2020;105(3):545–553. doi:10.3324/haematol.2019.232132
- Collins PW. Personalized prophylaxis. Haemophilia. 2012;18(suppl 4):131–135. doi:10.1111/j.1365-2516.2012.02838.x
- Kavakli K, Smith L, Kuliczkowski K, et al. Once-weekly prophylactic treatment versus on-demand treatment with nonacog alfa in patients with moderately severe to severe hemophilia B. Haemophilia. 2016;22(3):381–388. doi:10.1111/hae.12878
- Powell JS, Pasi KJ, Ragni MV, et al. Phase 3 study of recombinant factor IX Fc fusion protein in hemophilia B. N Engl J Med. 2013;369(24):2313–2323. doi:10.1056/NEJMoa1305074
- Mancuso ME, Lubetsky A, Pan-Petesch B, et al. Long-term safety and efficacy of rIX-FP prophylaxis with extended dosing intervals up to 21 days in adults/adolescents with hemophilia B. J Thromb Haemost. 2020;18(5):1065–1074. doi:10.1111/jth.14778
- Japanese Society for Thrombosis and Hemostasis. Hemostasis treatment guidelines for hemophilia patients without inhibitors; 2013; Available from: https://www.jsth.org/wordpress/wp-content/uploads/2015/04/03_inhibitor_H1_B.pdf. Accessed January 18, 2022.
- Tortella BJ, Alvir J, McDonald M, et al. Real-world analysis of dispensed IUs of coagulation factor IX and resultant expenditures in hemophilia B patients receiving standard half-life versus extended half-life products and those switching from standard half-life to extended half-life products. J Manag Care Spec Pharm. 2018;24(7):643–653. doi:10.18553/jmcp.2018.17212
- Cortesi PA, D’Angiolella LS, Lafranconi A, et al. Modern treatments of haemophilia: review of cost-effectiveness analyses and future directions. Pharmacoeconomics. 2018;36(3):263–284. doi:10.1007/s40273-017-0588-z
- Guh S, Grosse SD, McAlister S, Kessler CM, Soucie JM. Healthcare expenditures for males with haemophilia and employer-sponsored insurance in the United States, 2008. Haemophilia. 2012;18(2):268–275. doi:10.1111/j.1365-2516.2011.02692.x
- Machin N, Ragni MV, Smith KJ. Gene therapy in hemophilia A: a cost-effectiveness analysis. Blood Adv. 2018;2(14):1792–1798. doi:10.1182/bloodadvances.2018021345
- Tortella BJ, Fogarty PF, Alvir J, et al. Real world data analysis of coagulation factor IX costs in specific patients with hemophilia B switching from standard half-life to extended half-life products [abstract]. Blood. 2016;128:4963.
- Peyvandi F, Garagiola I, Young G. The past and future of haemophilia: diagnosis, treatments, and its complications. Lancet. 2016;388(10040):187–197. doi:10.1016/s0140-6736(15)01123-x
- Chhabra A, Spurden D, Fogarty PF, et al. Real-world outcomes associated with standard half-life and extended half-life factor replacement products for treatment of haemophilia A and B. Blood Coagul Fibrinolysis. 2020;31(3):186–192. doi:10.1097/mbc.0000000000000885
- The Japanese society for pediatric endocrinology. Available from: http://jspe.umin.jp/medical/taikaku.html. Accessed July 19, 2023.
- General Counter of Official Statistics (e-Stat). National health and nutrition survey: average and standard deviation of height and weight. Available from: https://www.e-stat.go.jp/dbview?sid=0003224177. Accessed July 19, 2023.
- Ministry of Health Labor and Welfare. Various information of medical fee. Available from: https://shinryohoshu.mhlw.go.jp/shinryohoshu/. Accessed July 20, 2023.
- Bank of Japan. Transactions on the Tokyo foreign exchange market (in 2019); 2020; Available from: https://www.boj.or.jp/statistics/market/forex/fxdaily/ex2019.pdf. Accessed July 20, 2023.
- Shirahata A. Treatment for hemophilia from the economic points of view. Jpn J Thromb Hemost. 2021;32(1):55–63. doi:10.2491/jjsth.32.55
- Buckner TW, Bocharova I, Hagan K, et al. Health care resource utilization and cost burden of hemophilia B in the United States. Blood Adv. 2021;5(7):1954–1962. doi:10.1182/bloodadvances.2020003424
- Nagao A, Bingo M, Yamaguchi T, Fukutake K. Real-world use of albutrepenonacog alfa, A recombinant coagulation factor IX albumin fusion protein, for personalized prophylaxis in Japanese individuals with hemophilia B: a case series. Cureus. 2023;15(1):e33573. doi:10.7759/cureus.33573
- Japan Foundation for AIDS Prevention. National survey of blood coagulation disorders; 2021; Available from: https://api-net.jfap.or.jp/library/project.html. Accessed February 8, 2023.