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ORIGINAL RESEARCH

A Post-Authorization Safety Surveillance Study to Report Clinical Experience with Purified Factor IX Concentrate in Pediatric Patients with Hemophilia B

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Pages 113-122 | Received 21 Jul 2023, Accepted 29 Jan 2024, Published online: 05 Mar 2024

References

  • Berntorp E, Fischer K, Hart DP, et al. Haemophilia. Nat Rev Dis Primers. 2021;7(1):45. doi:10.1038/s41572-021-00278-x
  • Srivastava A, Santagostino E, Dougall A, et al. WFH Guidelines for the Management of Hemophilia panelists and co-authors. WFH guidelines for the management of hemophilia, 3rd edition. Haemophilia. 2020;26(Suppl 6):1–158. doi:10.1111/hae.14046
  • Castaman G. The benefits of prophylaxis in patients with hemophilia B. Expert Rev Hematol. 2018;11(8):673–683. doi:10.1080/17474086.2018.1489719
  • Turecek PL, Abbühl B, Tangada SD, et al. Nonacog gamma, a novel recombinant factor IX with low factor IXa content for treatment and prophylaxis of bleeding episodes. Expert Rev Clin Pharmacol. 2015;8(2):163–177. doi:10.1586/17512433.2015.1011126
  • Thomas KB, Urbancik W, Turecek PL, et al. Continuous infusion of FVIII and FIX concentrates: in vitro analysis of clinically relevant parameters. Haemophilia. 1999;5(1):17–25. doi:10.1046/j.1365-2516.1999.00210.x
  • Kavakli K, Smith L, Kuliczkowski K, et al. Once-weekly prophylactic treatment vs. on-demand treatment with nonacog alfa in patients with moderately severe to severe haemophilia B. Haemophilia. 2016;22(3):381–388. doi:10.1111/hae.12878
  • Windyga J, Lissitchkov T, Stasyshyn O, et al. Pharmacokinetics, efficacy and safety of BAX326, a novel recombinant factor IX: a prospective, controlled, multicentre Phase I/III study in previously treated patients with severe (FIX level <1%) or moderately severe (FIX level ≤2%) haemophilia B. Haemophilia. 2014;20(1):15–24. doi:10.1111/hae.12228
  • Collins PW, Quon DVK, Makris M, et al. Pharmacokinetics, safety and efficacy of a recombinant factor IX product, trenonacog alfa in previously treated haemophilia B patients. Haemophilia. 2018;24(1):104–112. doi:10.1111/hae.13324
  • Valentino LA, Rusen L, Elezovic I, et al. Multicentre, randomized, open-label study of on-demand treatment with two prophylaxis regimens of recombinant coagulation factor IX in haemophilia B subjects. Haemophilia. 2014;20(3):398–406. doi:10.1111/hae.12344
  • Takeda Canada Inc. IMMUNINE® VH: factor IX concentrate (Human). Product Monograph; 2021. Available from: https://assets-dam.takeda.com/raw/upload/v1662721792/legacy-dotcom/siteassets/en-ca/home/what-we-do/our-medicines/product-monographs/immunine-vh/immunine-vh-pm-en.pdf. Accessed September 30, 2022.
  • Takeda Manufacturing Austria AG. IMMUNINE 1200 IU: powder and solvent for solution for injection or infusion. Human blood coagulation factor IX. Package Leaflet; 2020. Available from: https://file.wuxuwang.com/hma/AT_H_0177_002_FinalPL_1of3.pdf. Accessed September 30, 2022.
  • Blonde L, Khunti K, Harris SB, Meizinger C, Skolnik NS. Interpretation and impact of real-world clinical data for the practicing clinician. Adv Ther. 2018;35(11):1763–1774. doi:10.1007/s12325-018-0805-y
  • Negrier C, Voisin S, Baghaei F, et al. Global post-authorization safety surveillance study: real-world data on prophylaxis and on-demand treatment using FEIBA (an activated prothrombin complex concentrate). Blood Coagul Fibrinolysis. 2016;27(5):551–556. doi:10.1097/mbc.0000000000000525
  • Gascoigne EW, Dash CH, Harman C, Wilmot D. A retrospective survey on the safety of Replenate, a high-purity factor VIII concentrate. Pharmacoepidemiol Drug Saf. 2004;13(4):243–252. doi:10.1002/pds.956
  • Gascoigne EW, Dash CH, Harman C, Wilmot D. A retrospective survey on the safety of replenine, a high-purity factor IX concentrate. Pharmacoepidemiol Drug Saf. 2004;13(3):187–195. doi:10.1002/pds.911
  • Srivastava A, Brewer AK, Mauser-Bunschoten EP, et al. Treatment Guidelines Working Group on behalf of the World Federation of Hemophilia. Guidelines for the management of hemophilia. Haemophilia. 2013;19(1):e1–e47. doi:10.1111/j.1365-2516.2012.02909.x
  • Mancuso ME, Mahlangu JN, Pipe SW. The changing treatment landscape in haemophilia: from standard half-life clotting factor concentrates to gene editing. Lancet. 2021;397(10274):630–640. doi:10.1016/s0140-6736(20)32722-7
  • Collins PW, Young G, Knobe K, et al; Paradigm 2 Investigators. Recombinant long-acting glycoPEGylated factor IX in hemophilia B: a multinational randomized Phase 3 trial. Blood. 2014;124(26):3880–3886. doi:10.1182/blood-2014-05-573055
  • 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
  • Santagostino E, Martinowitz U, Lissitchkov T, et al. Long-acting recombinant coagulation factor IX albumin fusion protein (rIX-FP) in hemophilia B: results of a phase 3 trial. Blood. 2016;127(14):1761–1769. doi:10.1182/blood-2015-09-669234