Editorials
Vol. 2 No. 1 (2023)
Growing weapons to fight hemophilia
Publisher's note
All claims expressed in this article are solely those of the authors and do not necessarily represent those of their affiliated organizations, or those of the publisher, the editors and the reviewers. Any product that may be evaluated in this article or claim that may be made by its manufacturer is not guaranteed or endorsed by the publisher.
All claims expressed in this article are solely those of the authors and do not necessarily represent those of their affiliated organizations, or those of the publisher, the editors and the reviewers. Any product that may be evaluated in this article or claim that may be made by its manufacturer is not guaranteed or endorsed by the publisher.
Received: 16 February 2023
Accepted: 17 February 2023
Accepted: 17 February 2023
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Very few rare diseases have witnessed the gigantic progress in patient care that took place for the hemophilias in the last 20 years. The first landmark was in the 1990s, when recombinant DNA technology afforded the industrial production, regulatory approval and commercialization of an array of factor VIII (FVIII) and factor IX (FIX) products, potentially available in unlimited quantity, efficacious and free from the risk of transmission of such bloodborne infections as HIV and the hepatitis virus B and C [...].
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Citations
White GC 2nd, McMillan CW, Kingdon HS, Shoemaker CB. Use of recombinant antihemophilic factor in the treatment of two patients with classic hemophilia. N Engl J Med 1989;320:166-70. DOI: https://doi.org/10.1056/NEJM198901193200307
Manco-Johnson MJ, 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: 535-44. DOI: https://doi.org/10.1056/NEJMoa067659
Nilsson IM, Berntorp E, Löfqvist T, Pettersson H. Twenty-five years' experience of prophylactic treatment in severe haemophilia A and B. J Intern Med 1992;232:25-32. DOI: https://doi.org/10.1111/j.1365-2796.1992.tb00546.x
den Uijl IE, Fischer K, Van Der Bom JG, et al. Analysis of low frequency bleeding data: the association of joint bleeds according to baseline FVIII activity levels. Haemophilia 2011;17:41-4. DOI: https://doi.org/10.1111/j.1365-2516.2010.02383.x
Aledort L, Mannucci PM, Schramm W, Tarantino M. Factor VIII replacement is still the standard of care in haemophilia A. Blood Transfus 2019;17:479-86.
Mannucci PM. Benefits and limitations of extended plasma half-life factor VIII products in hemophilia A. Expert Opin Investig Drugs 2020;29:303-9. DOI: https://doi.org/10.1080/13543784.2020.1723547
Chhabra ES, Liu T, Kulman J, et al. BIVV001, a new class of factor VIII replacement for hemophilia A that is independent of von Willebrand factor in primates and mice. Blood 2020;135:1484-96. DOI: https://doi.org/10.1182/blood.2019001292
Mannucci PM. Treatment of hemophilia - more amazing progress. N Engl J Med 2020;383:1068-70. DOI: https://doi.org/10.1056/NEJMe2024545
Konkle BA, Shapiro AD, Quon DV, et al. BIVV001 fusion protein as factor VIII replacement therapy for hemophilia A. N Engl J Med 2020;383:1018-27. DOI: https://doi.org/10.1056/NEJMoa2002699
von Drygalski A, Chowdary P, Kulkarni R, et al. Efanesoctocog alfa prophylaxis for patients with severe hemophilia A. N Engl J Med 2023;388:310-8. DOI: https://doi.org/10.1056/NEJMoa2209226
Leissinger C. Another victory for patients with hemophilia.N Engl J Med 2023;388:372-3. DOI: https://doi.org/10.1056/NEJMe2216176
Mahlangu J, Oldenburg J, Paz-Priel I, et al. Emicizumab prophylaxis in patients who have hemophilia A without inhibitors. N Engl J Med 2018;379:811-22. DOI: https://doi.org/10.1056/NEJMoa1803550
Ozelo MC, Mahlangu J, Pasi KJ, et al. Valoctocogene roxaparvovec gene therapy for hemophilia A. N Engl J Med2022;386:1013-25. DOI: https://doi.org/10.1056/NEJMoa2113708
Mannucci PM. Gene transfer in hemophilia A: not cogent yet.Bleeding, Thrombosis and Vascular Biology 2022; 1:32. DOI: https://doi.org/10.4081/btvb.2022.32
Pasi KJ, Rangarajan S, Georgiev P, et al. Targeting of antithrombin in hemophilia A or B with RNAi therapy. N EnglJ Med 2017;377:819-28. DOI: https://doi.org/10.1056/NEJMoa1616569
Shapiro AD, Angchaisuksiri P, Astermark J, et al. Subcutaneous concizumab prophylaxis in hemophilia A and hemophilia A/B with inhibitors: phase 2 trial results. Blood 2019;134:1973-82. DOI: https://doi.org/10.1182/blood.2019001542
How to Cite
1.
Mannucci PM. Growing weapons to fight hemophilia. Bleeding Thromb Vascul Biol [Internet]. 2023 Feb. 23 [cited 2025 Nov. 7];2(1). Available from: https://www.btvb.org/btvb/article/view/64
Copyright (c) 2023 The Author(s)
This work is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License.
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