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Expert Review of Hematology Volume 15, 2022 - Issue 10
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Original Research

Real-world study of rurioctocog alfa pegol and emicizumab in US clinical practice among patients with hemophilia A

Shawn X. Suna Takeda Development Center Americas, Inc, Global Evidence and Outcomes Department, Cambridge, MA, USACorrespondence[email protected]
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,
Andrew Frickb Trio Health Inc, Analytics Department, Louisville, CO, USAView further author information
,
Vinod Balasac Valley Chidren’s Hospital, Hematology Department, Madera, CA, USAView further author information
&
Jonathan C. Robertsd Bleeding & Clotting Disorders Institute, Peoria, IL, USAORCID Iconhttps://orcid.org/0000-0002-9415-9543View further author information
ORCID Icon
Pages 943-950 | Received 25 Mar 2022, Accepted 03 Jul 2022, Published online: 24 Aug 2022

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.
  • Aledort L, Mannucci PM, Schramm W, et al. Factor VIII replacement is still the standard of care in haemophilia A. Blood Transfus. 2019;17(6):479–486.
  • ADYNOVATE (Antihemophilic Factor (Recombinant)) [ package insert]. Shire Website; [ updated 2021 Jun; cited 2021 Sept 1. Available from: https://www.shirecontent.com/PI/PDFs/ADYNOVATE_USA_ENG.pdf
  • 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.
  • Mullins ES, Stasyshyn O, Alvarez-Roman MT, et al. Extended half-life pegylated, full-length recombinant factor VIII for prophylaxis in children with severe haemophilia A. Haemophilia. 2017;23(2):238–246.
  • Aledort L, Milligan S, Watt M, et al. A retrospective observational study of rurioctocog alfa pegol in clinical practice in the United States. J Manag Care Spec Pharm. 2020;26(4):492–503.
  • Journeycake J, Caicedo J, Cheng D, et al. Clinical outcomes in patients with haemophilia A after switching to rurioctocog alfa pegol prophylaxis in the ATHN 2 study. Haemophilia. 2021;27(suppl 2):ABS070.
  • HEMLIBRA (emicizumab-kxwh) injection. Prescribing information. South San Francisco, CA: Genentech, Inc; 2021.
  • 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.
  • McCary I, Guelcher C, Kuhn J, et al. Real-world use of emicizumab in patients with haemophilia A: bleeding outcomes and surgical procedures. Haemophilia. 2020;26(4):631–636.
  • Institute for Clinical and Economic Review. Valoctocogene roxaparvovec and emicizumab for hemophilia A without inhibitors: final policy recommendations; 2020 [cited 2021 Sept 20]. Available from: https://icer.org/wp-content/uploads/2020/10/ICER_Hemophilia-A_Key-Policy-Recommendations_112020.pdf
  • Kruse-Jarres R, Oldenburg J, Santagostino E, et al. Bleeding and safety outcomes in persons with haemophilia A without inhibitors: results from a prospective non-interventional study in a real-world setting. Haemophilia. 2019;25(2):213–220.
  • Reyes A, Revil C, Niggli M, et al. Efficacy of emicizumab prophylaxis versus factor VIII prophylaxis for treatment of hemophilia A without inhibitors: network meta-analysis and sub-group analyses of the intra-patient comparison of the HAVEN 3 trial. Curr Med Res Opin. 2019;35(12):2079–2087.
  • Le Quellec S. Clinical evidence and safety profile of emicizumab for the management of children with hemophilia A. Drug Des Devel Ther. 2020;14:469–481.
  • Zhou ZY, Raimundo K, Patel AM, et al. Model of short- and long-term outcomes of emicizumab prophylaxis treatment for persons with hemophilia A. J Manag Care Spec Pharm. 2020;26(9):1109–1120.

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