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Articles

Re-personalization and stratification of hemophilia care in an evolving treatment landscape

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References

  • Collins PW, Blanchette VS, Fischer K, et al. Ewenstein B; rAHF-PFM Study Group. Break-through bleeding in relation to predicted factor VIII levels in patients receiving prophylactic treatment for severe hemophilia A. J Thromb Haemost. 2009 Mar;7(3):413–420. doi: 10.1111/j.1538-7836.2008.03270.x
  • Iorio A, Edginton AN, Blanchette V, et al. Performing and interpreting individual pharmacokinetic profiles in patients with hemophilia A or B: Rationale and general considerations. Res Pract Thromb Haemost. 2018 May 20;2(3):535–548. doi: 10.1002/rth2.12106
  • Ragni MV, Croteau SE, Morfini M, et al. Subcommittee on factor VIII, factor IX, and rare bleeding disorders. Pharmacokinetics and the transition to extended half-life factor concentrates: communication from the SSC of the ISTH. J Thromb Haemost. 2018 Jul;16(7):1437–1441. doi: 10.1111/jth.14153
  • Valentino LA, Mamonov V, Hellmann A, et al. Prophylaxis Study Group. A randomized comparison of two prophylaxis regimens and a paired comparison of on-demand and prophylaxis treatments in hemophilia A management. J Thromb Haemost. 2012 Mar;10(3):359–367. doi: 10.1111/j.1538-7836.2011.04611.x
  • Lissitchkov T, Rusen L, Georgiev P, et al. PK-guided personalized prophylaxis with Nuwiq® (human-cl rhFVIII) in adults with severe haemophilia A. Haemophilia. 2017 Sep;23(5):697–704. doi: 10.1111/hae.13251
  • 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 Aug 9;357(6):535–544. doi: 10.1056/NEJMoa067659
  • Manco-Johnson MJ, Kempton CL, Reding MT, et al. Randomized, controlled, parallel-group trial of routine prophylaxis vs. on-demand treatment with sucrose-formulated recombinant factor VIII in adults with severe hemophilia A (SPINART). J Thromb Haemost. 2013 Jun;11(6):1119–1127. Erratum in: J Thromb Haemost. 2014 Jan;12. doi: 10.1111/jth.12202
  • Schwaab R, Brackmann HH, Meyer C, et al. Oldenburg J Haemophilia A: mutation type determines risk of inhibitor formation. Thromb Haemost. 1995 Dec;74(6):1402–1406. doi: 10.1055/s-0038-1649954
  • 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 May 26;374(21):2054–2064. doi: 10.1056/NEJMoa1516437
  • Spena S, Garagiola I, Cannavò A, et al. Peyvandi F; SIPPET Study Group Prediction of factor VIII inhibitor development in the SIPPET cohort by mutational analysis and factor VIII antigen measurement. J Thromb Haemost. 2018 Apr;16(4):778–790. doi: 10.1111/jth.13961
  • Whelan SF, Hofbauer CJ, Horling FM, et al. Reipert BM Distinct characteristics of antibody responses against factor VIII in healthy individuals and in different cohorts of hemophilia A patients. Blood. 2013 Feb 7;121(6):1039–1048. doi: 10.1182/blood-2012-07-444877
  • Astermark J, Oldenburg J, Pavlova A, et al. MIBS study Group. Polymorphisms in the IL10 but not in the IL1beta and IL4 genes are associated with inhibitor development in patients with hemophilia A. Blood. 2006 Apr 15;107(8):3167–3172. doi: 10.1182/blood-2005-09-3918
  • Astermark J, Oldenburg J, Carlson J, et al. Polymorphisms in the TNFA gene and the risk of inhibitor development in patients with hemophilia A. Blood. 2006 Dec 1;108(12):3739–3745. doi: 10.1182/blood-2006-05-024711
  • Pavlova A, Delev D, Lacroix-Desmazes S, et al. Impact of polymorphisms of the major histocompatibility complex class II, interleukin-10, tumor necrosis factor-alpha and cytotoxic T-lymphocyte antigen-4 genes on inhibitor development in severe hemophilia A. J Thromb Haemost. 2009 Dec;7(12):2006–2015. doi: 10.1111/j.1538-7836.2009.03636.x
  • Astermark J, Donfield SM, Gomperts ED, et al. Berntorp E; Hemophilia Inhibitor Genetics Study (HIGS) Combined Cohort. The polygenic nature of inhibitors in hemophilia A: results from the Hemophilia Inhibitor Genetics Study (HIGS) Combined Cohort. Blood. 2013 Feb 21;121(8):1446–1454. doi: 10.1182/blood-2012-06-434803
  • Oldenburg J, Mahlangu JN, Kim B, et al. Emicizumab prophylaxis in hemophilia A with inhibitors. N Engl J Med. 2017 Aug 31;377(9):809–818. doi: 10.1056/NEJMoa1703068
  • Pasi KJ, Rangarajan S, Georgiev P, et al. Targeting of antithrombin in hemophilia A or B with RNAi therapy. N Engl J Med. 2017 Aug 31;377(9):819–828. doi: 10.1056/NEJMoa1616569
  • 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 May;13(5):743–754. doi: 10.1111/jth.12864
  • Callaghan MU, Sidonio R, Pipe SW. Novel therapeutics for hemophilia and other bleeding disorders. Blood. 2018 Jul 5;132(1):23–30. doi: 10.1182/blood-2017-09-743385
  • Ahlberg A. Haemophilia in Sweden. VII. Incidence, treatment and prophylaxis of arthropathy and other musculo-skeletal manifestations of haemophilia A and B. Acta Orthop Scand Suppl. 1965;36(Suppl 77):3–132. doi: 10.3109/ort.1965.36.suppl-77.01
  • Nilsson IM, Hedner U, Ahlberg A. Haemophilia prophylaxis in Sweden. Acta Paediatr Scand. 1976 Mar;65(2):129–135. doi: 10.1111/j.1651-2227.1976.tb16525.x
  • Den Uijl IE, Mauser Bunschoten EP, Roosendaal G, et al. Clinical severity of haemophilia A: does the classification of the 1950s still stand? Haemophilia. 2011 Nov;17(6):849–853. doi: 10.1111/j.1365-2516.2011.02539.x
  • Soucie JM, Monahan PE, Kulkarni R, et al. US hemophilia Treatment Center Network. The frequency of joint hemorrhages and procedures in nonsevere hemophilia A vs B. Blood Adv. 2018 Aug 28;2(16):2136–2144. doi: 10.1182/bloodadvances.2018020552
  • Leissinger C, Gringeri A, Antmen B, et al. Anti-inhibitor coagulant complex prophylaxis in hemophilia with inhibitors. N Engl J Med. 2011 Nov 3;365(18):1684–1692. Erratum in: N Engl J Med. 2011 Dec 22;365. doi: 10.1056/NEJMoa1104435
  • Collins PW, Liesner R, Makris M, et al. Treatment of bleeding episodes in haemophilia A complicated by a factor VIII inhibitor in patients receiving emicizumab. Interim guidance from UKHCDO Inhibitor Working Party and Executive Committee. Haemophilia. 2018 May;24(3):344–347. doi: 10.1111/hae.13495
  • Batty P, Austin SK, Khair K, et al. Treatment burden, haemostatic strategies and real world inhibitor screening practice in non-severe haemophilia A. Br J Haematol. 2017 Mar;176(5):796–804. doi: 10.1111/bjh.14543
  • Aledort LM, Ewenstein BM. Emicizumab prophylaxis in hemophilia A with inhibitors. N Engl J Med. 2017 Nov 30;377(22):2193. doi: 10.1056/NEJMc1712683
  • Aledort LM. The evolution of comprehensive haemophilia care in the United States: perspectives from the frontline. Haemophilia. 2016 Sep;22(5):676–683. doi: 10.1111/hae.12970
  • Chalmers EA, Alamelu J, Collins PW, et al. Paediatric & Rare Disorders Working Parties of the UK haemophilia Doctors Organization. intracranial haemorrhage in children with inherited bleeding disorders in the UK 2003-2015: A national cohort study. Haemophilia. 2018 Jul;24(4):641–647. doi: 10.1111/hae.13461
  • Collins PW, Chalmers E, Hart DP, et al. UK haemophilia Centre Doctors. Diagnosis and treatment of factor VIII and IX inhibitors in congenital haemophilia: (4th edition). UK Haemophilia Centre Doctors Organization. Br J Haematol. 2013 Jan;160(2):153–170. doi: 10.1111/bjh.12091
  • Kitazawa T, Esaki K, Tachibana T, et al. 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 Jun 28;117(7):1348–1357. doi: 10.1160/TH17-01-0030
  • Uchida N, Sambe T, Yoneyama K, et al. A first-in-human phase 1 study of ACE910, a novel factor VIII-mimetic bispecific antibody, in healthy subjects. Blood. 2016 Mar 31;127(13):1633–1641. 1. doi: 10.1182/blood-2015-06-650226
  • 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.
  • Rangarajan S, Walsh L, Lester W, et al. AAV5-factor VIII gene transfer in severe hemophilia A. N Engl J Med. 2017 Dec 28;377(26):2519–2530. doi: 10.1056/NEJMoa1708483
  • George LA, Sullivan SK, Giermasz A, et al. Hemophilia B gene therapy with a high-specific-activity factor IX variant. N Engl J Med. 2017 Dec 7;377(23):2215–2227. doi: 10.1056/NEJMoa1708538
  • Hay CR, DiMichele DM. International immune tolerance study. The principal results of the international immune tolerance study: a randomized dose comparison. Blood. 2012 Feb 9;119(6):1335–1344. doi: 10.1182/blood-2011-08-369132