John Libbey Eurotext



Traitement personnalisé dans l’hémophilie Volume 26, numéro 3, Mai-Juin 2020

  • [1] Nilsson I.M., Blomback M., Ahlberg A. Our experience in Sweden with prophylaxis on hemophilia. Bibl Haematol. 1968;34:111-124.
  • [2] Ljung R.C.R. Prophylactic treatment in Sweden – overtreatment or optimal model? Haemophilia. 1998;4:409-412.
  • [3] Petrini P. Identifying and overcoming barriers to prophylaxis in the management of haemophilia. Haemophilia. 2007;13:16-22. suppl 2
  • [4] Van Den Berg M., Fischer K., Mauser-Bunschoten E.P. Long term outcome of individualized prophylactic treatment of children with severe haemophilia. Br J Kaematol. 2001;112:561-565.
  • [5] Björkman S. Pharmacokinetics of plasma derived and recombinant factor IC-implications for prophylaxis and on demand therapy. Haemophilia. 2013;19:808-813.
  • [6] Bjorkman S., Collins P. Measurement of factor VIII pharmacokinetics in routine clinical practice. J Thromb Haemost. 2013;11:180-182. for the project on factor VIII/factor IX pharmacokinetics of the factor VIII/factor IX scientific ans standardization committee of the ISTH
  • [7] Collins P.W., Bjorkman S., Fischer K. Factor VIII requirement to maintain a target plasma level in prophylactic treatment of severe hemophilia A: influences of variance in pharmacokinetics and treatment regimens. J Thromb Haemost. 2010;8:269-275.
  • [8] Collins P.W., Blanchette V.S., Fischer K. Break-through bleeding in relation to predicted factor VIII levels in patients receiving prophylactic treatment for severe hemophilia A. J Thromb Haemost. 2009;7:413-420.
  • [9] Lindvall K., Astermark J., Bjorkman S. Daily dosing prophylaxis for haemophilia: a randomized cross-over pilot study evaluating feasibility and efficacy. Haemophilia. 2012;18:855-859.
  • [10] Tuddenam E. Gene therapy for hemophilia B. Haemophilia. 2012;18:13-17. suppl 4
  • [11] Lissitchkov T., Rusen L., Georgiev P. PK-guided personalized prophylaxis with Nuwiq (human-cl rhFVIII) in adults with severe haemophilia A. Haemophilia. 2017;23:697-704. ®5
  • [12] Santagostino E. Prolong-9FP clinical development program – phase I results of reombiannt fusion protein lonking coagulation factor IX with recombinant albumin (rFIX-FP). Thromb Res. 2013;131:S7-S10.
  • [13] Santagostino E., Martinowitz U., Lissitchkov T. Long acting recombinant coagulation factor IX albumin fusion protein in hemophilia B : results of a phase 3 trial. Blood. 2016;127:1761-1769.
  • [14] Iorio A. Using pharmacokinetics to individualize hemophilia therapy. Hematology Am Soc Hematol Educ Program. 2017;2017:595-604. 1
  • [15] Iorio A., Iserman E., Blanchette V. Target plasma factor levels for personalized treatment in haemophilia: a Delphi consensus statement. Haemophilia. 2017;23:e170-e179. 3
  • [16] Stemberger M., Kallenbach F., Schmit E. Impact of adopting population pharmacokinetics for tailoringprophylaxis in haemophilia A patients: a historically controlled observational study. Thromb Haemost. 2019;119:368-376. 3
  • [17] Carlsson M., Berntorp E., Bjorkman S. Improved cost effectiveness by pharmacokinetic dosing of FVIII in prophylactic treatment of haemophilia. Haemophilia. 1997;3:96-101.
  • [18] Delavenne X., Ollier E., Lienhart A., Dargaud Y. A new paradigm for personalized prophylaxis for patients withsevere haemophilia A. Haemophilia. 2020;26:228-235. 2
  • [19] Ebbert P.T., Xavier F., Seaman C.D., Ragni M.V. Emicizumab prophylaxis in patients with haemophilia A with and without inhibitors. Haemophilia. 2020;26:41-46. 1
  • [20] Dargaud Y., Lienhart A., Negrier C. Prospective assessment of thrombin generation test for dose monitoring of bypassing therapy in hemophilia patients with inhibitors undergoing elective surgery. Blood. 2010;116:5734-5737. 25
  • [21] Young G., Sørensen B., Dargaud Y., Negrier C., Brummel-Ziedins K., Key N.S. Thrombin generation and whole blood viscoelastic assays in the management of hemophilia: current state of art and future perspectives. Blood. 2013;121:1944-1950. 11
  • [22] Oldenburg J., Mahlangu J.N., Kim B. Emicizumab prophylaxis in hemophilia A with inhibitors. N Engl J Med. 2017;377:809-818. 9
  • [23] Dargaud Y., Lienhart A., Janbain M., Le Quellec S., Enjolras N., Negrier C. Use of thrombin generation assay to personalize treatment of breakthrough bleeds in a patient with hemophilia and inhibitors receiving prophylaxis with emicizumab. Haematologica. 2018;103:e181-e183. 4
  • [24] Machin N., Ragni M.V. An investigational RNAi therapeutic targeting antithrombin for the treatment of hemophilia A and B. J Blood Med. 2018;9:135-140.
  • [25] Pasi K.J., Rangarajan S., Georgiev P. Targeting of antithrombin in hemophilia A or B with RNAi therapy. N Engl J Med. 2017;377:819-828. 9
  • [26] Spadarella G., Di Minno A., Milan G. Paradigm shift for the treatment of hereditary haemophilia: towards precision medicine. Blood Rev. 2020;39:100618.
  • [27] Santagostino E., Mancuso M.E., Tripodi A. Severe haemophilia with mild bleeding phenotype: molecular characterization and global coagulation profile. J Thromb Haemost. 2010;8:737-743.
  • [28] Young G., Liesner R., Chang T. A multicenter, open-label phase 3 study of emicizumab prophylaxis in children with hemophilia A with inhibitors. Blood. 2019;134:2127-2138. 24
  • [29] Nathwani A.C. Gene therapy for hemophilia. Am Soc Hematol Educ Program. 2019;2019:1-8. 1
  • [30] Rangarajan S., Walsh L., Lester W. AAV5-factor VIII gene transfer in severe hemophilia A. N Engl J Med. 2017;377:2519-2530. 26