Clinical experience of switching patients with severe hemophilia to rVIII-SingleChain or rIX-FP

References

• Srivastava A, Santagostino E, Dougall A, et al. WFH guidelines for the management of hemophilia, 3rd edition. Haemophilia. 2020;26(Suppl 6):1–158.
   PubMedGoogle Scholar
• Peyvandi F, Berger K, Seitz R, et al. Kreuth V initiative: European consensus proposals for treatment of hemophilia using standard products, extended half-life coagulation factor concentrates and non-replacement therapies. Haematologica. 2020;105(8):2038–2043.
   PubMed Web of Science ®Google Scholar
• Oldenburg J. Optimal treatment strategies for hemophilia: achievements and limitations of current prophylactic regimens. Blood. 2015;125(13):2038–2044.
   PubMed Web of Science ®Google Scholar
• Collins PW, Blanchette VS, Fischer K, et al. Break-through bleeding in relation to predicted factor VIII levels in patients receiving prophylactic treatment for severe hemophilia A. J Thromb Haemost. 2009;7(3):413–420.
   PubMed Web of Science ®Google Scholar
• Manco-Johnson M, Abshire TC, Shapiro AD, et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med. 2007;357(6):535–544.
   PubMed Web of Science ®Google Scholar
• Hacker MR, Geraghty S, Manco-Johnson M. Barriers to compliance with prophylaxis therapy in haemophilia. Haemophilia. 2001;7:392–396.
   PubMed Web of Science ®Google Scholar
• Mahlangu J, Kuliczkowski K, Karim FA, et al. Efficacy and safety of rVIII-SingleChain: results of a phase 1/3 multicenter clinical trial in severe hemophilia A. Blood. 2016;128(5):630–637.
   PubMed Web of Science ®Google Scholar
• 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.
   PubMed Web of Science ®Google Scholar
• Escobar M, Santagostino E, Mancuso ME, et al. Switching patients in the age of long-acting recombinant products? Expert Rev Hematol. 2019;12(sup1):1–13.
   PubMed Web of Science ®Google Scholar
• Zollner S, Raquet E, Claar P, et al. Non-clinical pharmacokinetics and pharmacodynamics of rVIII-SingleChain, a novel recombinant single-chain factor VIII. Thromb Res. 2014;134(1):125–131.
   PubMed Web of Science ®Google Scholar
• Schmidbauer S, Witzel R, Robbel L, et al. Physicochemical characterisation of rVIII-SingleChain, a novel recombinant single-chain factor VIII. Thromb Res. 2015;136(2):388–395.
   PubMed Web of Science ®Google Scholar
• Klamroth R, Simpson M, von Depka-Prondzinski M, et al. Comparative pharmacokinetics of rVIII-SingleChain and octocog alfa (advate((R))) in patients with severe haemophilia A. Haemophilia. 2016;22(5):730–738.
   PubMed Web of Science ®Google Scholar
• Stasyshyn O, Djambas Khayat C, Iosava G, et al. Safety, efficacy and pharmacokinetics of rVIII-SingleChain in children with severe hemophilia A: results of a multicenter clinical trial. J Thromb Haemost. 2017;15(4):636–644.
   PubMed Web of Science ®Google Scholar
• Hajducek D, Chelle P, Hermans C, et al. Development and evaluation of the population pharmacokinetic models for FVIII and FIX concentrates of the WAPPS-Hemo project. Haemophilia. 2020;26(3):384–400.
   PubMed Web of Science ®Google Scholar
• Bowyer A, Key N, Dalton D, et al. The coagulation laboratory monitoring of afstyla single-chain FVIII concentrate. Haemophilia. 2017;23(5):e469–e470.
   PubMed Web of Science ®Google Scholar
• Mahlangu J, Oldenburg J, Paz-Priel I, et al. Emicizumab prophylaxis in patients who have hemophilia a without inhibitors. N Engl J Med. 2018;379(9):811–822.
   PubMed Web of Science ®Google Scholar
• Oldenburg J, Mahlangu JN, Kim B, et al. Emicizumab prophylaxis in hemophilia a with inhibitors. N Engl J Med. 2017;377(9):809–818.
   PubMed Web of Science ®Google Scholar
• Pipe SW, Shima M, Lehle M, et al. Efficacy, safety, and pharmacokinetics of emicizumab prophylaxis given every 4 weeks in people with haemophilia A (HAVEN 4): a multicentre, open-label, non-randomised phase 3 study. Lancet Haematol. 2019;6(6):e295–e305.
   PubMed Web of Science ®Google Scholar
• Olivieri M, Sommerer P, Maro G, et al. Assessing prophylactic use and clinical outcomes in hemophilia a patients treated with rVIII-SingleChain and other common rFVIII products in Germany. Eur J Haematol. 2020;104(4):310–317.
   PubMed Web of Science ®Google Scholar
• Simpson ML, Desai V, Maro GS, et al. Comparing factor use and bleed rates in U.S. hemophilia a patients receiving prophylaxis with 3 different long-acting recombinant factor VIII products. J Manag Care Spec Pharm. 2020;26:504–512.
   PubMed Web of Science ®Google Scholar
• Yan S, Maro GS, Desai V, et al. A real-world analysis of commonly prescribed FVIII products based on US medical charts: consumption and bleeding outcomes in hemophilia a patients. J Manag Care Spec Pharm. 2020;26:1258–1265.
   PubMed Web of Science ®Google Scholar
• Konkle BA, Stasyshyn O, Chowdary P, et al. Pegylated, full-length, recombinant factor VIII for prophylactic and on-demand treatment of severe hemophilia A. Blood. 2015;126(9):1078–1085.
   PubMed Web of Science ®Google Scholar
• Reding MT, Ng HJ, Poulsen LH, et al. Safety and efficacy of Bay 94-9027, a prolonged-half-life factor VIII. J Thromb Haemost. 2017;15(3):411–419.
   PubMed Web of Science ®Google Scholar
• Zhang Y, Roberts J, Tortorici M, et al. Population pharmacokinetics of recombinant coagulation factor VIII-SingleChain in patients with severe hemophilia A. J Thromb Haemost. 2017;15(6):1106–1114.
   PubMed Web of Science ®Google Scholar
• Chia J, Louber J, Glauser I, et al. Half-life-extended recombinant coagulation factor IX-albumin fusion protein is recycled via the FcRn-mediated pathway. J Biol Chem. 2018;293(17):6363–6373.
   PubMed Web of Science ®Google Scholar
• Santagostino E, Negrier C, Klamroth R, et al. Safety and pharmacokinetics of a novel recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in hemophilia B patients. Blood. 2012;120(12):2405–2411.
   PubMed Web of Science ®Google Scholar
• Kenet G, Chambost H, Male C, et al. Long-acting recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in children. Results of a phase 3 trial. Thromb Haemost. 2016;116(10):659–668.
   PubMed Web of Science ®Google Scholar
• Martinowitz U, Lissitchkov T, Lubetsky A, et al. Results of a phase I/II open-label, safety and efficacy trial of coagulation factor IX (recombinant), albumin fusion protein in haemophilia B patients. Haemophilia. 2015;21(6):784–790.
   PubMed Web of Science ®Google Scholar
• Kenet G, Chambost H, Male C, et al. Long-term safety and efficacy of recombinant coagulation factor IX albumin fusion protein (rIX-FP) in previously treated pediatric patients with hemophilia B: results from a phase 3b extension study. Thromb Haemost. 2020;120(4):599–606.
   PubMed Web of Science ®Google Scholar
• 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.
   PubMed Web of Science ®Google Scholar
• IDELVION (albutrepenonacog alfa) summary of product characteristics. West Sussex: CSL Behring UK Limited; 2020 [cited 2020 May 13]. Available from: https://www.medicines.org.uk/emc/product/2146/smpc
   Google Scholar
• IDELVION (Coagulation Factor IX [Recombinant], Albumin Fusion Protein (rIX-FP) Highlights of Prescribing Information. Marburg: CSL Behring GmbH; 2020 [cited 2020 May 13]. Available from: https://labeling.cslbehring.com/PI/US/Idelvion/EN/Idelvion-Prescribing-Information.pdf
   Google Scholar
• Mancuso ME, Oldenburg J, Boggio L, et al. High adherence to prophylaxis regimens in haemophilia B patients receiving rIX-FP: evidence from clinical trials and real-world practice. Haemophilia. 2020;26(4):637–642.
   PubMed Web of Science ®Google Scholar
• Oldenburg J, Yan S, Maro G, et al. Assessing bleeding rates, related clinical impact and factor utilization in German hemophilia B patients treated with extended half-life rIX-FP compared to prior drug therapy. Curr Med Res Opin. 2020;36(1):9–15.
   PubMed Web of Science ®Google Scholar
• Hermans C, Marino R, Lambert C, et al. Real-World utilisation and bleed rates in patients with haemophilia B who switched to recombinant factor IX fusion protein (rIX-FP): a retrospective international analysis. Adv Ther. 2020;37(6):2988–2998.
   PubMed Web of Science ®Google Scholar