77
Views
0
CrossRef citations to date
0
Altmetric
Review

Inhibitor eradication and treatment for acquired hemophilia A

&
Pages 233-240 | Received 08 Jan 2024, Accepted 03 May 2024, Published online: 09 May 2024

References

  • Delgado J, Jimenez-Yuste V, Hernandez-Navarro F, et al. Acquired haemophilia: review and meta-analysis focused on therapy and prognostic factors. Br J Haematol. 2003;121(1):21–35. doi: 10.1046/j.1365-2141.2003.04162.x
  • Cohen AJ, Kessler CM. Acquired inhibitors. Baillière Clinic Haematol. 1996;9(2):331–354. doi: 10.1016/S0950-3536(96)80067-9
  • Franchini M, Marano G, Cruciani M, et al. Advances in managing rare, acquired bleeding disorders. Expert Rev Hematol. 2020;13(6):599–606. doi: 10.1080/17474086.2020.1756259
  • Franchini M, Vaglio S, Marano G, et al. Acquired hemophilia A: a review of recent data and new therapeutic options. Hematology. 2017;25:1–7.
  • Franchini M, Mannucci PM. Acquired haemophilia A: a 2013 update. Thromb Haemost. 2013;110(12):1114–1120. doi: 10.1160/TH13-05-0363
  • Franchini M, Lippi G. Acquired factor VIII inhibitors. Blood. 2008;112:250–255. doi: 10.1182/blood-2008-03-143586
  • Franchini M, Gandini G, Di Paolantonio T, et al. Acquired hemophilia A: a concise review. Am J Hematol. 2005;80(1):55–63. doi: 10.1002/ajh.20390
  • Coppola A, Favaloro EJ, Tufano A, et al. Acquired inhibitors of coagulation factors: part I—acquired hemophilia a. Semin Thromb Hemost. 2012;38(5):433–446. doi: 10.1055/s-0032-1315757
  • Collins PW, Hirsch S, Baglin TP, et al. For the UK haemophilia centre doctors’ organisation. Acquired haemophilia a in the United Kingdom: a 2-year national surveillance study by the United Kingdom haemophilia centre doctors’ organisation. Blood. 2007;109(5):1870–1877. doi: 10.1182/blood-2006-06-029850
  • Knoebl P, Marco P, Baudo F, et al. On behalf of the EACH2 registry contributors. Demographic and clinical data in acquired hemophilia A: results from the European acquired haemophilia registry (EACH2). J Thromb Haemost. 2012;10(4):622–631. doi: 10.1111/j.1538-7836.2012.04654.x
  • Kessler CM, Ma AD, Al-Mondhiry HAB, et al. Assessment of acquired hemophilia patient demographics in the United States: the hemostasis and thrombosis research society registry. Blood Coagul Fibrinolysis. 2016;27(7):761–769. doi: 10.1097/MBC.0000000000000582
  • Sun B, Xue F, Feng Y, et al. Outcome of CARE: a 6-year national registry of acquired haemophilia a in China. Br J Haematol. 2019;187(5):653–665. doi: 10.1111/bjh.16128
  • Solymoss S. Postpartum acquired factor VIII inhibitors: results of a survey. Am J Haematol. 1998;59(1):1–4. doi: 10.1002/(SICI)1096-8652(199809)59:1<1:AID-AJH1>3.0.CO;2-T
  • Franchini M. Postpartum acquired factor VIII inhibitors. Am J Hematol. 2006;81(10):768–773. doi: 10.1002/ajh.20702
  • Tengborn L, Baudo F, Huth-Kühne A, et al. On behalf of the EACH2 registry contributors. Pregnancy-associated acquired haemophilia A: results from the European acquired haemophilia (EACH2) registry. BJOG. 2012;119(12):1529–1537. doi: 10.1111/j.1471-0528.2012.03469.x
  • Tiede A, Klamroth R, Scharf RE, et al. Prognostic factors for remission of and survival in acquired hemophilia a (AHA): results from the GTH-AH 01/2010 study. Blood. 2015;125(7):1091–1097. doi: 10.1182/blood-2014-07-587089
  • Franchini M, Capra F, Nicolini N, et al. Drug-induced anti-factor VIII antibodies: a systematic review. Med Sci Monit. 2007;13(4):RA55–61.
  • Franchini M, Glingani C, De Donno G, et al. The first case of acquired hemophilia a associated with SARS-CoV-2 infection. Am J Hemartol. 2020;95(8):E197–8. doi: 10.1002/ajh.25865
  • Franchini M, Focosi D. Association between SARS-CoV-2 infection or vaccination and acquired hemophilia A: a case report and literature update. Thromb Res. 2023;222:7–11. doi:10.1016/j.thromres.2022.12.010
  • Coppola A, Franchini M, Tripodi A, et al.; ad hoc Working Group. Acquired haemophilia A: Italian consensus recommendations on diagnosis, general management and treatment of bleeding. Blood Transfus. 2022;20(3):245–262. doi: 10.2450/2022.0238-21
  • Marino R. Acquired hemophilia A: bleeding pattern and hemostatic therapeutic strategies. Medicina (Kaunas). 2023;59(10):1739. doi:10.3390/medicina59101739
  • Green D, Lechner K. A survey of 215 non-hemophilic patients with inhibitors to factor VIII. Thromb Haemost. 1981;45(3):200–203. doi: 10.1055/s-0038-1650169
  • Lottenberg R, Kentro TB, Kitchens CS. Acquired hemophilia: a natural history study of 16 patients with factor VIII inhibitors receiving little or no therapy. Arch Intern Med. 1987;147(6):1077–1081. doi: 10.1001/archinte.1987.00370060073014
  • Baudo F, Collins P, Huth-Kühne A, et al. On behalf of the EACH2 registry contributors. Management of bleeding in acquired hemophilia A: results from the European acquired haemophilia (EACH2) registry. Blood. 2012;120(1):39–46. doi: 10.1182/blood-2012-02-408930
  • Huth-Kühne A, Baudo F, Collins P, et al. International recommendations on the diagnosis and treatment of patients with acquired hemophilia A. Haematologica. 2009;94(4):566–575. doi: 10.3324/haematol.2008.001743
  • Collins PW, Chalmers E, Hart D, et al. Diagnosis and management of acquired coagulation inhibitors: a guideline from UKHCDO. Br J Haematol. 2013;162(6):758–773. doi: 10.1111/bjh.12463
  • Tiede A, Collins P, Knoebl P, et al. International recommendations on the diagnosis and treatment of acquired hemophilia a. Haematologica. 2020;105(7):1791–1801. doi: 10.3324/haematol.2019.230771
  • Kershaw G, Jayakodi D, Dunkley S. Laboratory identification of factor inhibitors: the perspective of a large tertiary hemophilia center. Semin Thromb Hemost. 2009;35(8):760–768. doi: 10.1055/s-0029-1245108
  • Tiede A, Werwitzke S, Scharf RE. Laboratory diagnosis of acquired hemophilia A: limitations, consequences and challenges. Semin Thromb Hemost. 2014;40(7):803–811. doi: 10.1055/s-0034-1390004
  • Verbruggen B, Novakova I, Wessels H, et al. The Nijmegen modification of the Bethesda assay for factor VIII: C inhibitors: improved specificity and reliability. Thromb Haemost. 1995;73:247–251. doi: 10.1055/s-0038-1653759
  • Kruse-Jarres R, Kempton CL, Baudo F, et al. Acquired hemophilia A: updated review of evidence and treatment guidance. Am J Hematol. 2017;92(7):695–705. doi: 10.1002/ajh.24777
  • Tiede A, Giangrande P, Teitel J, et al. Clinical evaluation of bleeds and response to haemostatic treatment in patients with acquired haemophilia: a global expert consensus statement. Haemophilia. 2019;25(6):969–978. doi: 10.1111/hae.13844
  • Franchini M, Schiavulli M, Liumbruno GM. Hemostatic therapy as a management strategy for acquired hemophilia: what does the future hold? Expert Rev Hematol. 2021;14(3):263–270. doi:10.1080/17474086.2021.1892483
  • Mazzucconi MG, Baldacci E, Ferretti A, et al. Acquired haemophilia A: an intriguing disease. Mediterr J Hematol Infect Dis. 2020;12(1):e2020045. doi:10.4084/mjhid.2020.045
  • Sallah S. Treatment of acquired haemophilia with factor eight inhibitor bypassing activity. Haemophilia. 2004;10(2):169–173. doi: 10.1046/j.1365-2516.2003.00856.x
  • Ehrlich HJ, Henzl MJ, Gomperts ED. Safety of factor VIII inhibitor bypass activity (FEIBA ®): 10-year compilation of thrombotic adverse events. Haemophilia. 2002;8(2):83–90. doi: 10.1046/j.1365-2516.2002.00532.x
  • Zanon E, Pasca S, Santoro C, et al. Activated prothrombin complex concentrate (FEIBA ®) in acquired haemophilia A: a large multicentre Italian study – the FAIR registry. Br J Haematol. 2019;184(5):853–855. doi: 10.1111/bjh.15175
  • Franchini M, Lippi G. Recombinant activated factor VII: mechanisms of action and current indications. Semin Thromb Hemost. 2010;36(5):485–492. doi: 10.1055/s-0030-1255442
  • Franchini M, Coppola A, Tagliaferri A, et al. FEIBA versus NovoSeven in hemophilia patients with inhibitors. Semin Thromb Hemost. 2013;39(7):772–778. doi: 10.1055/s-0033-1354425
  • Hay CRM, Negrier C, Ludlam CA. The treatment of bleeding in acquired hemophilia with recombinant factor VIIa: a multicenter study. Thromb Haemost. 1997;78(6):1463–1467. doi: 10.1055/s-0038-1665434
  • Sumner MJ, Geldziler BD, Pedersen M, et al. Treatment of acquired haemophilia with recombinant activated FVII: a critical appraisal. Haemophilia. 2007;13(5):451–461. doi: 10.1111/j.1365-2516.2007.01474.x
  • Mannucci PM, Franchini M. Porcine recombinant factor VIII: an additional weapon to handle anti-factor VIII antibodies. Blood Transfus. 2017;15(4):365–368. doi: 10.2450/2016.0030-16
  • Kruse-Jarres R, St-Louis J, Greist A, et al. Efficacy and safety of OBI-1, an antihaemophilic factor VIII (recombinant), porcine sequence, in subjects with acquired haemophilia A. Haemophilia. 2015;21(2):162–170. doi: 10.1111/hae.12627
  • Tarantino MD, Cuker A, Hardesty B, et al. Recombinant porcine sequence factor VIII (rpFVIII) for acquired haemophilia A: practical clinical experience of its use in seven patients. Haemophilia. 2017;23(1):25–32. doi: 10.1111/hae.13040
  • Zanon E, Pasca S, Borchiellini A, et al. Susoctocog-alfa (Obizur®) in the treatment of nine elderly patients with acquired haemophilia A: an Italian multicentre real world experience. Blood Transfus. 2020;18(4):312–321. doi: 10.2450/2020.00006-20
  • Campbell S, Mason J, Prasad R, et al. Acquired haemophilia and haemostatic control with recombinant porcine factor VIII: case series. Int Med J. 2021;51(2):215–219. doi: 10.1111/imj.14773
  • Türkantoz H, Königs C, Knöbl P, et al. Cross-reacting inhibitors against recombinant porcine factor VIII in acquired hemophilia A: data from the GTH-AH 01/2010 study. J Thromb Haemost. 2020;18(1):36–43. doi: 10.1111/jth.14618
  • Franchini M, Marano G, Pati I, et al. Investigational drugs to treat hemophilia. Expert Opin Investig Drugs. 2020;29(3):295–301. doi: 10.1080/13543784.2020.1722999
  • Franchini M, Marano G, Pati I, et al. Emicizumab for the treatment of haemophilia A: a narrative review. Blood Transfus. 2019;17(3):223–228. doi: 10.2450/2019.0026-19
  • Muto A, Yoshihashi K, Takeda M, et al. The anti-factor IXa/X bispecific antibody ACE910 prevents spontaneous joint bleeds in a long-term primate model of acquired hemophilia A. Blood. 2014;124(20):3165–3171. doi: 10.1182/blood-2014-07-585737
  • Knoebl P, Sperr WR, Schellongowski P, et al. Emicizumab for the treatment of acquired hemophilia A: lessons learned from 4 very different cases. Blood. 2018;132(Suppl 1):2476. doi: 10.1182/blood-2018-99-116973
  • Möhnle P, Pekrul I, Spannagl M, et al. Emicizumab in the treatment of acquired haemophilia: a case report. Transfus Med Hemother. 2019;46(2):121–123. doi: 10.1159/000497287
  • Dane KE, Lindsley JP, Streiff MB, et al. Successful use of emicizumab in a patient with refractory acquired hemophilia a and acute coronary syndrome requiring percutaneous coronary intervention. Res Pract Thromb Haemost. 2019;3(3):420–423. doi: 10.1002/rth2.12201
  • Al-Banaa K, Alhillan A, Hawa F, et al. Emicizumab use in treatment of acquired hemophilia A: a case report. Am J Case Rep. 2019;20:1046–1048. doi: 10.12659/AJCR.916783
  • Hess KJ, Patel P, Joshi AM, et al. Utilization of Emicizumab in acquired factor VIII deficiency. Am J Case Rep. 2020;21:e922326. doi:10.12659/AJCR.922326
  • Knoebl P, Thaler J, Jilma P, et al. Emicizumab for the treatment of acquired hemophilia A. Blood. 2021;137(3):410–419. doi:10.1182/blood.2020006315
  • Engelen MM, Vandesande J, De Bent J, et al. Emicizumab for acquired haemophilia A: a case series. Haemophilia. 2023;29(4):1049–1055. doi: 10.1111/hae.14809
  • Shima M, Amano K, Ogawa Y, et al. A prospective, multicenter, open-label phase III study of emicizumab prophylaxis in patients with acquired hemophilia A. J Thromb Haemost. 2023;21(3):534–545. doi: 10.1016/j.jtha.2022.10.004
  • Thomas VM, Abou-Ismail MY, Lim MY. Off-label use of emicizumab in persons with acquired haemophilia A and von Willebrand disease: a scoping review of the literature. Haemophilia. 2022;28(1):4–17. doi:10.1111/hae.14450
  • Tiede A, Kemkes-Matthes B, Knöbl P. Should emicizumab be used in patients with acquired hemophilia A? J Thromb Haemost. 2021;19(3):637–644. doi:10.1111/jth.15208
  • Poston JN, Kruse-Jarres R. Advances in acquired hemophilia a. Transfus Med Rev. 2022;36(4):215–219. doi:10.1016/j.tmrv.2022.07.001
  • Tiede A, Hart C, Knöbl P, et al. Emicizumab prophylaxis in patients with acquired haemophilia A (GTH-AHA-EMI): an open-label, single-arm, multicentre, phase 2 study. Lancet Haematol. 2023;10(11):e913–21. doi: 10.1016/S2352-3026(23)00280-6
  • Green D, Rademaker AW, Briët E. A prospective, randomized trial of prednisone and cyclophosphamide in the treatment of patients with factor VIII autoantibodies. Thromb Haemost. 1993;70(5):753–757. doi: 10.1055/s-0038-1649664
  • Bitting RL, Bent S, Li Y, et al. The prognosis and treatment of acquired hemophilia: a systematic review and meta-analysis. Blood Coagul Fibrinolysis. 2009;20(7):517–523. doi: 10.1097/MBC.0b013e32832ca388
  • Collins P, Baudo F, Knoebl P, et al. EACH2 registry collaborators. Immunosuppression for acquired hemophilia A: results from the European acquired haemophilia registry (EACH2). Blood. 2012;120(1):47–55. doi: 10.1182/blood-2012-02-409185
  • Annex 1. Summary of product characteristics. MabThera, INN-rituximab (europa.Eu). [cited 2023 Oct 1].
  • Wiestner A, Cho HJ, Asch AS, et al. Rituximab in the treatment of acquired factor VIII inhibitors. Blood. 2002;100(9):3426–3428. doi: 10.1182/blood-2002-03-0765
  • Sperr WR, Lechner K, Pabinger I. Rituximab for the treatment of acquired antibodies to factor VIII. Haematologica. 2007;92(1):66–72. doi: 10.3324/haematol.10553
  • Franchini M, Mannucci PM. Inhibitor eradication with rituximab in haemophilia: where do we stand? Br J Haematol. 2014;165(5):600–608. doi: 10.1111/bjh.12829
  • Franchini M. Rituximab in the treatment of adult acquired hemophilia A: a systematic review. Crit Rev Oncol Hematol. 2007;63(1):47–52. doi: 10.1016/j.critrevonc.2006.11.004
  • D’arena G, Grandone E, Di Minno MN, et al. The anti-CD20 monoclonal antibody rituximab to treat acquired haemophilia a. Blood Transfus. 2016;14(2):255–261. doi: 10.2450/2015.0090-15
  • Wang P, Zhou R, Zhou H, et al. Single-dose rituximab plus glucocorticoid versus cyclophosphamide plus glucocorticoid in patients with newly diagnosed acquired hemophilia A: a multicenter, open-label, randomized non inferiority trial. Am J Hematol. 2024;99(1):28–37. doi: 10.1002/ajh.27128
  • Schep SJ, van Dijk WEM, Beckers EAM, et al. Dutch Society of haemophilia treaters, the Netherlands. Treatment of acquired hemophilia A, a balancing act: results from a 27-year Dutch cohort study. Am J Hematol. 2021;96(1):51–59. doi: 10.1002/ajh.26009
  • Dolan G, Benson G, Bowyer A, et al. Principles of care for acquired haemophilia. Eur J Hematol. 2021;106(6):762–773. doi: 10.1111/ejh.13592
  • Franchini M, Focosi D. Innovative therapies for acquired hemophilia a. Semin Thromb Hemost. 2024;(online ahead of print). doi: 10.1055/s-0044-1779737
  • Obaji S, Rayment R, Collins PW. Mycophenolate mofetil as adjunctive therapy in acquired haemophilia A. Haemophilia. 2019;25(1):e59–e65. doi:10.1111/hae.13658
  • Pai M. Acquired hemophilia A. Hematol Oncol Clin North Am. 2021;35(6):1131–1142. doi:10.1016/j.hoc.2021.07.007
  • Arruda VR, Lillicrap D, Herzog RW. Immune complications and their management in inherited and acquired bleeding disorders. Blood. 2022;140(10):1075–1085. doi:10.1182/blood.2022016530
  • Dobbelstein C, Moschovakis GL, Tiede A. Reduced-intensity, risk factor-stratified immunosuppression for acquired hemophilia A: single-center observational study. Ann Hematol. 2020;99(9):2105–2112. doi: 10.1007/s00277-020-04150-y
  • Simon B, Ceglédi A, Dolgos J, et al. Combined immunosuppression for acquired hemophilia A: CyDRi is a highly effective low-toxicity regimen. Blood. 2022;140(18):1983–1992. doi: 10.1182/blood.2022016873
  • Holstein K, Liu X, Smith A, et al. Bleeding and response to hemostatic therapy in acquired hemophilia A: results from the GTH-AH 01/2010 study. Blood. 2020;136(3):279–287. doi: 10.1182/blood.2019003639
  • Pfrepper C, Klamroth R, Oldenburg J, et al. Emicizumab for the treatment of acquired hemophilia A: consensus recommendations from the GTH-AHA working group. Hamostaseologie. 2023;(online ahead of print). doi: 10.1055/a-2197-9738
  • Tiede A. Immunotherapy of acquired hemophilia A. Hematol Am Soc Hematol Educ Program. 2023;2023(1):19–23. doi:10.1182/hematology.2023000461

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.