8. FEJEZET | HIVATKOZÁSOK
- Blanchette VS, Key NS, Ljung LR, et al. Definitions in hemophilia: communication from the SSC of the ISTH. J Thromb Haemost. 2014;12(11):1935-1939.
- Meijer P, Verbruggen B. The between-laboratory variation of factor VIII inhibitor testing: the experience of the external quality assessment program of the ECAT foundation. Semin Thromb Hemost. 2009;35(8):786-793.
- Verbruggen B, van Heerde WL, Laros-van Gorkom BA. Improvements in factor VIII inhibitor detection: from Bethesda to Nijmegen. Semin Thromb Hemost. 2009;35(8):752-759.
- Miller CH. Laboratory testing for factor VIII and IX inhibitors in haemophilia: a review. Haemophilia. 2018;24(2):186-197.
- Miller CH, Platt SJ, Rice AS, Kelly F, Soucie JM, Hemophilia Inhibitor Research Study Investigators. Validation of Nijmegen-Bethesda assay modifications to allow inhibitor measurement during replacement therapy and facilitate inhibitor surveillance. J Thromb Haemost. 2012;10(6):1055-1061.
- Peyvandi F, Mannucci PM, Garagiola I, et al. A randomized trial of factor VIII and neutralizing antibodies in hemophilia A. N Engl J Med. 2016;374(21):2054-2064.
- Hay CR, Brown S, Collins PW, Keeling DM, Liesner R. The diagnosis and management of factor VIII and IX inhibitors: a guideline from the United Kingdom Haemophilia Centre Doctors Organisation. Br J Haematol. 2006;133(6):591-605.
- de Moerloose P, Fischer K, Lambert T, et al. Recommendations for assessment, monitoring and follow-up of patients with haemophilia. Haemophilia. 2012;18(3):319-325.
- Ragni MV, Ojeifo O, Feng J, et al. Risk factors for inhibitor formation in haemophilia: a prevalent case-control study. Haemophilia. 2009;15(5):1074-1082.
- Astermark J, Altisent C, Batorova A, et al. Non-genetic risk factors and the development of inhibitors in haemophilia: a comprehensive review and consensus report. Haemophilia. 2010;16(5):747-766.
- Teitel JM, Carcao M, Lillicrap D, et al. Orthopaedic surgery in haemophilia patients with inhibitors: a practical guide to haemostatic, surgical and rehabilitative care. H aemophilia. 2009;15(1):227-239.
- Kempton CL, Soucie JM, Miller CH, et al. In non-severe hemophilia A the risk of inhibitor after intensive factor treatment is greater in older patients: a case-control study. J Thromb Haemost. 2010;8(10):2224-2231.
- Berntorp E, Collins P, D’Oiron R, et al. I dentifying non-responsive bleeding episodes in patients with haemophilia and inhibitors: a consensus definition. Haemophilia. 2011;17(1):e202-e210.
- McMillan CW, Shapiro SS, Whitehurst D, Hoyer LW, Rao AV, Lazerson J. The natural history of factor VIII: C inhibitors in patients with hemophilia A: a national cooperative study, II: observations on the initial development of factor VIII: C inhibitors. Blood. 1988;71(2):344- 348.
- Sharathkumar A, Lillicrap D, Blanchette VS, et al. I ntensive exposure to factor VIII is a risk factor for inhibitor development in mild hemophilia A. J Thromb Haemost. 2003;1(6):1228-1236.
- Gouw SC, van den Berg HM, Fischer K, et al. Intensity of factor VIII treatment and inhibitor development in children with severe hemophilia A: the RODIN study. Blood. 2013;121(20):4046-4055.
- Castaman G, Fijnvandraat K. Molecular and clinical predictors of inhibitor risk and its prevention and treatment in mild hemophilia A. Blood. 2014;124(15):2333-2336.
- Fischer K, Lassila R, Peyvandi F, et al. I nhibitor development in haemophilia according to concentrate: four-year results from the European HAemophilia Safety Surveillance (EUHASS) project. Thromb Haemost. 2015;113(5):968-975.
- Hay CR. Factor VIII inhibitors in mild and moderate-severity haemophilia A. Haemophilia. 1998;4(4):558-563.
- Chitlur M, Warrier I, Rajpurkar M, Lusher JM. Inhibitors in factor IX deficiency a report of the ISTH-SSC international FIX inhibitor registry (1997-2006). Haemophilia. 2009;15(5):1027-1031.
- Carcao M, Re W, Ewenstein B. The role of previously untreated patient studies in understanding the development of FVIII inhibitors. Haemophilia. 2016;22(1):22-31.
- van den Berg HM, Fischer K, Carcao M, et al. Timing of inhibitor development in more than 1000 previously untreated patients with severe hemophilia A. Blood. 2019;134(3):317-320.
- Eckhardt CL, Menke LA, van Ommen CH, et al. Intensive perioperative use of factor VIII and the Arg593->Cys mutation are risk factors for inhibitor development in mild/moderate hemophilia A. J Thromb Haemost. 2009;7(6):930-937.
- Hashemi SM, Fischer K, Moons KG, van den Berg HM. Improved prediction of inhibitor development in previously untreated patients with severe haemophilia A. Haemophilia. 2015;21(2):227-233.
- Eckhardt CL, van Velzen AS, Peters M, et al. Factor VIII gene (F8) mutation and risk of inhibitor development in nonsevere hemophilia A. Blood. 2013;122(11):1954-1962.
- Eckhardt CL, Loomans JI, van Velzen AS, et al. Inhibitor development and mortality in non-severe hemophilia A. J Thromb Haemost. 2015;13(7):1217-1225.
- Lj ung RCR. How I manage patients with inherited haemophilia A and B and factor inhibitors. Br J Haematol. 2018;180(4):501-510.
- Colvin BT, Astermark J, Fischer K, et al. European principles of haemophilia care. Haemophilia. 2008;14(2):361-374.
- Teitel J, Berntorp E, Collins P, et al. A systematic approach to controlling problem bleeds in patients with severe congenital haemophilia A and high-titre inhibitors. Haemophilia. 2007;13(3):256-263.
- Berntorp E, Shapiro A, Astermark J, et al. Inhibitor treatment in haemophilias A and B: summary statement for the 2006 international consensus conference. Haemophilia. 2006;12(Suppl 6):1-7.
- Kempton CL, White GC 2nd. How we treat a hemophilia A patient with a factor VIII inhibitor. Blood. 2009;113(1):11-17.
- Young G, Liesner R, Chang T, et al. A multicenter, open-label phase 3 study of emicizumab prophylaxis in children with hemophilia A with inhibitors. Blood. 2019;134(24):2127-2138.
- Oldenburg J, Mahlangu JN, Kim B, et al. Emicizumab prophylaxis in hemophilia A with inhibitors. N Engl J Med. 2017;377(9):809-818.
- Konkle BA, Ebbesen LS, Erhardtsen E, et al. Randomized, prospective clinical trial of recombinant factor VIIa for secondary prophylaxis in hemophilia patients with inhibitors. J Thromb Haemost. 2007;5(9):1904-1913.
- Leissinger C, Gringeri A, Antmen B, et al. Anti-inhibitor coagulant complex prophylaxis in hemophilia with inhibitors. N Engl J Med. 2011;365(18):1684-1692.
- Astermark J, Donfield SM, DiMichele DM, et al. A randomized comparison of bypassing agents in hemophilia complicated by an inhibitor: the FEIBA NovoSeven Comparative (FENOC) Study. Blood. 2007;109(2):546-551.
- Seaman CD, Ragni MV. Sequential bypassing agents during major orthopedic surgery: a new approach to hemostasis. Blood Adv. 2017;1(17):1309-1311.
- Seaman CD, Ragni MV. Emicizumab use in major orthopedic surgery. Blood Adv. 2019;3(11):1722-1724.
- Dimichele D. Inhibitors: resolving diagnostic and therapeutic dilemmas. Haemophilia. 2002;8(3):280-287.
- HEMLIBRA® (emicizumab-kxwh) injection for subcutaneous use [U.S. prescribing information]. South San Francisco, CA: Genentech; Revised 10/2018.
- 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.
- Young G. Implementing emicizumab in hemophilia inhibitor management: emicizumab should be prescribed after tolerance. B lood Adv. 2018;2(20):2780-2782.
- 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, nonrandomised phase 3 study. Lancet Haematol. 2019;6(6):e295-e305.
- Oldenburg J, Mahlangu JN, Bujan W, et al. The effect of emicizumab prophylaxis on health-related outcomes in persons with haemophilia A with inhibitors: HAVEN 1 Study. Haemophilia. 2019;25(1):33-44.
- Nogami K, Soeda T, Matsumoto T, Kawabe Y, Kitazawa T, Shima M. Routine measurements of factor VIII activity and inhibitor titer in the presence of emicizumab utilizing anti-idiotype monoclonal antibodies. J Thromb Haemost. 2018;16(7):1383-1390.
- Tripodi A, Chantarangkul V, Novembrino C, Peyvandi F. Advances in the treatment of hemophilia: implications for laboratory testing. Clin Chem. 2019;65(2):254-262.
- Gundabolu K, Goldsweig A, Bhatt VR, Koepsell SA, Harper JL. ST- segment elevation myocardial infarction (STEMI) and pulmonary embolism in a hemophilia A patient receiving emicizumab and recombinant activated factor VII. Haemophilia. 2020;26:e5-e8.
- Bar-Ilan A, Livnat T, Hoffmann M, et al. In vitro characterization of MOD-5014, a novel long-acting carboxy-terminal peptide (CTP)- modified activated FVII. Haemophilia. 2018;24(3):477-486.
- Gruppo RA, Malan D, Kapocsi J, et al. Phase 1, single-dose escalating study of marzeptacog alfa (activated), a recombinant factor VIIa variant, in patients with severe hemophilia. J Thromb Haemost. 2018;16(10):1984-1993.
- Pasi KJ, Rangarajan S, Georgiev P, et al. Targeting of antithrombin in hemophilia A or B with RNAi therapy. N Engl J Med. 2017;377(9):819- 828.
- Eichler H, Angchaisuksiri P, Kavakli K, et al. A randomized trial of safety, pharmacokinetics and pharmacodynamics of con- cizumab in people with hemophilia A. J Thromb Haemost. 2018;16(11):2184-2195.
- Coppola A, Windyga J, Tufano A, Yeung C, Di Minno MN. Treatment for preventing bleeding in people with haemophilia or other congenital bleeding disorders undergoing surgery. Cochrane Database Syst Rev. 2015;(2):CD009961.
- Coppola A, Di Minno MN, Santagostino E. Optimizing management of immune tolerance induction in patients with severe haemophilia A and inhibitors: towards evidence-based approaches. B r J Haematol. 2010;150(5):515-528.
- DiMichele DM, Hoots WK, Pipe SW, Rivard GE, Santagostino E. International workshop on immune tolerance induction: consensus recommendations. Haemophilia. 2007;13(Suppl 1):1-22.
- Nakar C, Manco-Johnson MJ, Lail A, et al. Prompt immune tolerance induction at inhibitor diagnosis regardless of titre may increase overall success in haemophilia A complicated by inhibitors: experience of two U.S. centres. Haemophilia. 2015;21(3):365-373.
- Collins P, Chalmers E, Alamelu J, et al. First-line immune tolerance induction for children with severe haemophilia A: a protocol from the UK Haemophilia Centre Doctors’ Organisation Inhibitor and Paediatric Working Parties. Haemophilia. 2017;23(5):654-659.
- Hay CR, DiMichele DM, International Immune Tolerance Study. The principal results of the International Immune Tolerance Study: a randomized dose comparison. Blood. 2012;119(6):1335-1344.
- Malec LM, Journeycake J, Ragni MV. Extended half-life factor VIII for immune tolerance induction in haemophilia. Haemophilia. 2016;22(6):e552-e554.
- Carcao M, Shapiro A, Staber JM, et al. Recombinant factor VIII Fc fusion protein for immune tolerance induction in patients with severe haemophilia A with inhibitors–a retrospective analysis. Haemophilia. 2018;24(2):245-252.
- Ragni MV, Malec LM. Design of the INHIBIT trial: preventing inhibitors by avoiding ‘danger’, prolonging half-life and promoting tolerance. Expert Rev Hematol. 2014;7(6):747-755.
- Batsuli G, Zimowski KL, Tickle K, Meeks SL, Sidonio RF Jr. Immune tolerance induction in paediatric patients with haemophilia A and inhibitors receiving emicizumab prophylaxis. Haemophilia. 2019;25(5):789-796.
- Le Quellec S, Negrier C. Emicizumab should be prescribed independent of immune tolerance induction. Blood Adv. 2018;2(20):2783- 2786.
- Carcao M, Escuriola-Ettingshausen C, Santagostino E, et al. The changing face of immune tolerance induction in haemophilia A with the advent of emicizumab. Haemophilia. 2019;25(4):676-684.
- Matino D, Lillicrap D, Astermark J, et al. Switching clotting factor concentrates: considerations in estimating the risk of immunogenicity. Haemophilia. 2014;20(2):200-206.
- Dube E, Bonnefoy A, Merlen C, et al. A prospective surveillance study of inhibitor development in haemophilia A patients following a population switch to a third-generation B-domain-deleted recombinant factor VIII. Haemophilia. 2018;24(2):236-244.
- Coppola A, Marrone E, Conca P, et al. Safety of switching factor VIII products in the era of evolving concentrates: myths and facts. Semin Thromb Hemost. 2016;42(5):563-576.
- Santoro C, Quintavalle G, Castaman G, et al. Inhibitors in hemophilia B. Semin Thromb Hemost. 2018;44(6):578-589.
- Radic CP, Rossetti LC, Abelleyro MM, et al. Assessment of the F9 genotype-specific FIX inhibitor risks and characterisation of 10 novel severe F9 defects in the first molecular series of Argentinian patients with haemophilia B. Thromb Haemost. 2013;109(1):24-33.
- Bolton-Maggs PH, Pasi KJ. Haemophilias A and B. La ncet. 2003;361(9371):1801-1809.
- Male C, Andersson NG, Rafowicz A, et al. I nhibitor incidence in an unselected cohort of previously untreated patients with severe haemophilia B: a PedNet study. Haematologica. 2020. https://doi. org/10.3324/haematol.2019.239160
- Recht M, Pollmann H, Tagliaferri A, Musso R, Janco R, Neuman WR. A retrospective study to describe the incidence of moderate to severe allergic reactions to factor IX in subjects with haemophilia B. Haemophilia. 2011;17(3):494-499.
- Leissinger CA, Singleton T, Kruse-Jarres R. How I use bypassing therapy for prophylaxis in patients with hemophilia A and inhibitors. Blood. 2015;126(2):153-159.
- Antunes SV, Tangada S, Stasyshyn O, et al. Randomized comparison of prophylaxis and on-demand regimens with FEIBA NF in the treatment of haemophilia A and B with inhibitors. Haemophilia. 2014;20(1):65-72.
- Chowdary P, Lethagen S, Friedrich U, et al. Safety and pharmacokinetics of anti-TFPI antibody (concizumab) in healthy volunteers and patients with hemophilia: a randomized first human dose trial. J Thromb Haemost. 2015;13(5):743-754.
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