Thromboembolic Risks of Non-Factor Replacement Therapies in Hemophilia

 

 

MissingLinkText

• References

• 1 Oldenburg J, Mahlangu JN, Kim B, Schmitt C, Callaghan MU, Young G, Santagostino E, Kruse-Jarres R, Negrier C, Kessler C, Valente N, Asikanius E, Levy GG, Windyga J, Shima M. Emicizumab Prophylaxis in Hemophilia A with Inhibitors. N Engl J Med 2017; 377: 809-818.
• 2 Pasi KJ, Rangarajan S, Georgiev P, Mant T, Creagh MD, Lissitchkov T, Bevan D, Austin S, Hay CR, Hegemann I, Kazmi R, Chowdary P, Gercheva-Kyuchukova L, Mamonov V, Timofeeva M, Soh CH, Garg P, Vaishnaw A, Akinc A, Sorensen B, Ragni MV. Targeting of Antithrombin in Hemophilia A or B with RNAi Therapy. N Engl J Med 2017; 377: 819-828.
• 3 Astermark J. FVIII inhibitors: pathogenesis and avoidance. Blood 2015; 125: 2045-2051.
• 4 Scharf RE. Hot topic: Alloantibodies to therapeutic FVIII and the use of emicizumab in hemophilia A. Hämostaseologie 2016; 36: 195-198.
• 5 Holstein K, Batorova A, Carvalho M, Fijnvandraat K, Holme P, Kavakli K, Lambert T, Rocino A, Jimenez-Yuste V, Astermark J. European Haemophilia Therapy Strategy B. Current view and outcome of ITI therapy A change over time?. Thromb Res 2016; 148: 38-44.
• 6 Oldenburg J, Schwaab R, Brackmann HH. Induction of immune tolerance in haemophilia A inhibitor patients by the “Bonn Protocol’: predictive parameter for therapy duration and outcome. Vox Sang 1999; 77 (Suppl. 01) 49-54.
• 7 Hay CR, DiMichele DM. The principal results of the International Immune Tolerance Study: a randomized dose comparison. Blood 2012; 119: 1335-1344.
• 8 Rodriguez V, Mancuso ME, Warad D, Hay CR, DiMichele DM, Valentino L, Kenet G, Kulkarni R. Central venous access device (CVAD) complications in Haemophilia with inhibitors undergoing immune tolerance induction: Lessons from the international immune tolerance study. Haemophilia 2015; 21: e369-e374.
• 9 Morfini M, Haya S, Tagariello G, Pollmann H, Quintana M, Siegmund B, Stieltjes N, Dolan G, Tusell J. European study on orthopaedic status of haemophilia patients with inhibitors. Haemophilia 2007; 13: 606-612.
• 10 Gringeri A, Mantovani LG, Scalone L, Mannucci PM, Group CS. Cost of care and quality of life for patients with hemophilia complicated by inhibitors: the COCIS Study Group. Blood 2003; 102: 2358-2363.
• 11 Scalone L, Mantovani LG, Mannucci PM, Gringeri A, Investigators CS. Quality of life is associated to the orthopaedic status in haemophilic patients with inhibitors. Haemophilia 2006; 12: 154-162.
• 12 Holstein K, von Mackensen S, Bokemeyer C, Langer F. The impact of bleeding disorders on the socioeconomic status of adult patients. Results of a comparative single centre cohort study. Hämostaseologie. 2017 99.
• 13 Astermark J, Donfield SM, DiMichele DM, Gringeri A, Gilbert SA, Waters J, Berntorp E, Group FS. A randomized comparison of bypassing agents in hemophilia complicated by an inhibitor: the FEIBA NovoSeven Comparative (FENOC) Study. Blood 2007; 109: 546-551.
• 14 Antunes SV, Tangada S, Stasyshyn O, Mamonov V, Phillips J, Guzman-Becerra N, Grigorian A, Ewenstein B, Wong WY. Randomized comparison of prophylaxis and on-demand regimens with FEIBA NF in the treatment of haemophilia A and B with inhibitors. Haemophilia 2014; 20: 65-72.
• 15 Leissinger C, Gringeri A, Antmen B, Berntorp E, Biasoli C, Carpenter S, Cortesi P, Jo H, Kavakli K, Lassila R, Morfini M, Negrier C, Rocino A, Schramm W, Serban M, Uscatescu MV, Windyga J, Zulfikar B, Mantovani L. Anti-inhibitor coagulant complex prophylaxis in hemophilia with inhibitors. N Engl J Med 2011; 365: 1684-1692.
• 16 Konkle BA, Ebbesen LS, Erhardtsen E, Bianco RP, Lissitchkov T, Rusen L, Serban MA. Randomized, prospective clinical trial of recombinant factor VIIa for secondary prophylaxis in hemophilia patients with inhibitors. J Thromb Haemost 2007; 05: 1904-1913.
• 17 Klamroth R. A new era of treatment for patients with haemophilia A?. Hämostaseologie 2017; 37: 216-218.
• 18 Shima M, Hanabusa H, Taki M, Matsushita T, Sato T, Fukutake K, Fukazawa N, Yoneyama K, Yoshida H, Nogami K. Factor VIII-Mimetic Function of Humanized Bispecific Antibody in Hemophilia A. N Engl J Med 2016; 374: 2044-2053.
• 19 Roche press release of 26th June 2017. http://www.roche.com/media/store/releases/med-cor-2017–06–26.htm Accessed on 21st September 2017
• 20 Luu H, Ewenstein B. FEIBA safety profile in multiple modes of clinical and home-therapy application. Haemophilia 2004; 10 (Suppl. 02) 10-16.
• 21 Neufeld EJ, Negrier C, Arkhammar P, Benchikh Sel Fegoun, Simonsen MD, Rosholm A, Seremetis S. Safety update on the use of recombinant activated factor VII in approved indications. Blood Rev 2015; 29 (Suppl. 01) S34-S41.
• 22 Tiede A, Amano K, Ma A, Arkhammar P, El Fegoun SB, Rosholm A, Seremetis S, Baudo F. The use of recombinant activated factor VII in patients with acquired haemophilia. Blood Rev 2015; 29 (Suppl. 01) S19-S25.
• 23 Kitazawa T, Esaki K, Tachibana T, Ishii S, Soeda T, Muto A, Kawabe Y, Igawa T, Tsunoda H, Nogami K, Shima M, Hattori K. Factor VIIIa-mimetic cofactor activity of a bispecific antibody to factors IX/IXa and X/Xa, emicizumab, depends on its ability to bridge the antigens. Thromb Haemost 2017; 117: 1348-1357.
• 24 Alhenc-Gelas M, Canonico M, Picard V. Influence of natural SERPINC1 mutations on ex vivo thrombin generation. J Thromb Haemost 2010; 08: 845-848.
• 25 Shetty S, Vora S, Kulkarni B, Mota L, Vijapurkar M, Quadros L, Ghosh K. Contribution of natural anticoagulant and fibrinolytic factors in modulating the clinical severity of haemophilia patients. Br J Haematol 2007; 138: 541-544.
• 26 Bolliger D, Szlam F, Suzuki N, Matsushita T, Tanaka KA. Heterozygous antithrombin deficiency improves in vivo haemostasis in factor VIII-deficient mice. Thromb Haemost 2010; 103: 1233-1238.
• 27 Alnylam press release of 10th July 2017. http://in-vestors.alnylam.com/releasedetail.cfm?ReleaseID=1032569 Accessed on 21st September 2017.
• 28 Alnylam press release of 7th September 2017. http://investors.alnylam.com/releasede-tail.cfm?ReleaseID=1039464 Accessed on 21st September 2017.