Case report| Volume 61, ISSUE 6, 103457, December 2022

Utilization of emicizumab in acquired hemophilia A: A case report



      Acquired Hemophilia A (AHA) is a rare autoimmune disorder associated with the development of autoantibodies against factor VIII (FVIII). Although obtaining hemostatic control through the use of recombinant factor VIIa, activated prothrombin complex concentrate and recombinant porcine FVIII are cornerstones in the clinical management of AHA, these therapies have several disadvantages, including a higher risk for the development of thromboembolic events, unpredictable efficacy and short half-lives. While emicizumab has been FDA licensed for use in bleeding prophylaxis for patients with Congenital Hemophilia A (CHA) with and without inhibitors, it has not been approved for use in AHA, with only a few reports describing its use in this context.

      Case report

      We report our experience with the use of emicizumab in an 83-year old male with AHA, complicated by the onset of atrial fibrillation following admission, drug-induced thrombocytopenia, infectious complications, and the identification of a low-grade lymphoproliferative disorder, in which emicizumab prophylaxis was used for bleeding prophylaxis in the context of persistently elevated inhibitor titers without evidence of thrombotic events or thrombotic microangiopathy.


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        • Knoebl P.
        • Marco P.
        • Baudo F.
        • Collins P.
        • Huth-Kühne A.
        • Nemes L.
        • et al.
        Demographic and clinical data in acquired hemophilia A: Results from the European Acquired Haemophilia Registry (EACH2).
        J Thromb Haemost. 2012; 10: 622-631
        • Oldenburg J.
        • Mahlangu J.N.
        • Kim B.
        • Schmitt C.
        • Callaghan M.U.
        • Young G.
        • et al.
        Emicizumab Prophylaxis in Hemophilia A with Inhibitors.
        N Engl J Med. 2017; 377: 809-818
        • Young G.
        • Liesner R.
        • Chang T.
        • Sidonio R.
        • Oldenburg J.
        • Jiménez-Yuste V.
        • et al.
        A multicenter, open-label phase 3 study of emicizumab prophylaxis in children with hemophilia A with inhibitors.
        Blood. 2019; 134: 2127-2138
        • Mahlangu J.
        • Oldenburg J.
        • Paz-Priel I.
        • Negrier C.
        • Niggli M.
        • Mancuso M.E.
        • et al.
        Emicizumab Prophylaxis in Patients Who Have Hemophilia A without Inhibitors.
        N Engl J Med. 2018; 379: 811-822
        • Pipe S.W.
        • Shima M.
        • Lehle M.
        • Shapiro A.
        • Chebon S.
        • Fukutake K.
        • et al.
        Efficacy, safety, and pharmacokinetics of emicizumab prophylaxis given every 4 weeks in people with haemophilia A (HAVEN 4): a multicentre, open-label, non-randomised phase 3 study.
        Lancet Haematol. 2019; 6: e295-e305
        • Takeyama M.
        • Nogami K.
        • Matsumoto T.
        • Noguchi-Sasaki M.
        • Kitazawa T.
        • Shima M.
        An anti-factor IXa/factor X bispecific antibody, emicizumab, improves ex vivo coagulant potentials in plasma from patients with acquired hemophilia A.
        J Thromb Haemost. 2020; 18: 825-833
        • Takeyama M.
        • Furukawa S.
        • Yada K.
        • Ogiwara K.
        • Shimonishi N.
        • Nakajima Y.
        • et al.
        Ex Vivo Prediction of Comprehensive Coagulation Potential Using Simulated Blood Concentrations of Emicizumab in Patients with Acquired Hemophilia A.
        Thromb Haemost. 2021;
        • Möhnle P.
        • Pekrul I.
        • Spannagl M.
        • Sturm A.
        • Singh D.
        • Dechant C.
        Emicizumab in the Treatment of Acquired Haemophilia: A Case Report.
        Transfus Med Hemother. 2019; 46: 121-123
        • Al-Banaa K.
        • Alhillan A.
        • Hawa F.
        • Mahmood R.
        • Zaki A.
        • El Abdallah M.
        • et al.
        Emicizumab use in treatment of acquired hemophilia A: A case report.
        Am J Case Rep. 2019; 20: 1046-1048
        • Chen S.L.
        • Ellsworth P.
        • Kasthuri R.S.
        • Moll S.
        • Ma A.D.
        • Key N.S.
        Emicizumab reduces re-hospitalization for bleeding in acquired.
        haemophilia A. Haemophilia. 2021; 27: e585-e588
        • Chung S.Y.
        • Shen J.G.
        • Sticco K.L.
        Acquired haemophilia A: Successful treatment of a patient using upfront immunosuppressive therapy and haemostatic agents.
        BMJ Case Rep. 2021; 14: 1-4
        • Dane K.E.
        • Lindsley J.P.
        • Streiff M.B.
        • Moliterno A.R.
        • Khalid M.K.
        • Shanbhag S.
        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: 420-423
        • Ganslmeier M.
        • Pekrul I.
        • Heinrich D.A.
        • Angstwurm M.
        • Spannagl M.
        • Möhnle P.
        Persistent inhibitor in acquired haemophilia A: A case for emicizumab?.
        Haemophilia. 2021; 27: e502-e505
        • Hansenne A.
        • Hermans C.
        Emicizumab in acquired haemophilia A: about two clinical cases and literature review.
        Ther Adv Hematol. 2021; 12 (204062072110381)
        • Hess K.J.
        • Patel P.
        • Joshi A.M.
        • Kotkiewicz A.
        Utilization of emicizumab in acquired factor VIII deficiency.
        Am J Case Rep. 2020; 21: 1-3
        • Knoebl P.
        • Thaler J.
        • Jilma P.
        • Quehenberger P.
        • Gleixner K.
        • Sperr W.R.
        Emicizumab for the treatment of acquired hemophilia A.
        Blood. 2021; 137: 410-419
        • Makris M.
        • Iorio A.
        • Lenting P.J.
        Emicizumab and thrombosis: The story so far.
        J Thromb Haemost. 2019; 17: 1269-1272
        • Baudo F.
        • Collins P.
        • Huth-Kühne A.
        • Lévesque H.
        • Marco P.
        • Nemes L.
        • et al.
        Management of bleeding in acquired hemophilia A: Results from the European Acquired Haemophilia (EACH2) registry.
        Blood. 2012; 120: 39-46
        • Knoebl P.
        • Marco P.
        • Baudo F.
        • Collins P.
        • Huth‐Kühne A.
        • Nemes L.
        • et al.
        Demographic and clinical data in acquired hemophilia A: results from the European Acquired Haemophilia Registry (EACH2).
        J Thromb Haemost. 2012; 10: 622-631
        • Tiede A.
        • Worster A.
        Lessons from a systematic literature review of the effectiveness of recombinant factor VIIa in acquired haemophilia.
        Ann Hematol. 2018; 97: 1889-1901
        • Sun B.
        • Xue F.
        • Feng Y.
        • Sun J.
        • Yu Z.
        • Hou M.
        • et al.
        Outcome of CARE: a 6–year national registry of acquired haemophilia A in China.
        Br J Haematol. 2019; 187: 653-665
        • Tiede A.
        • Klamroth R.
        • Scharf R.E.
        • Trappe R.U.
        • Holstein K.
        • Huth-Kühne A.
        • et al.
        Prognostic factors for remission of and survival in acquired hemophilia A (AHA): results from the GTH-AH 01/2010 study.
        Blood, J Am Soc Hematol. 2015; 125: 1091-1097
        • Shima M.
        • Hanabusa H.
        • Taki M.
        • Matsushita T.
        • Sato T.
        • Fukutake K.
        • et al.
        Factor VIII–mimetic function of humanized bispecific antibody in hemophilia A.
        N Engl J Med. 2016; 374: 2044-2053
        • Shima M.
        • Hanabusa H.
        • Taki M.
        • Matsushita T.
        • Sato T.
        • Fukutake K.
        • et al.
        Long-term safety and efficacy of emicizumab in a phase 1/2 study in patients with hemophilia A with or without inhibitors.
        Blood Adv. 2017; 1: 1891-1899
        • Shima M.
        • Nagami S.
        • Yoneyama K.
        • Nomura A.
        • Ogawa Y.
        • Amano K.
        • et al.
        An Investigational Dosing Algorithm of Emicizumab for Prophylaxis in Acquired Hemophilia a.
        Blood. 2020; 136: 26-27
        • Holstein K.
        • Liu X.
        • Smith A.
        • Knöbl P.
        • Klamroth R.
        • Geisen U.
        • et al.
        Bleeding and response to hemostatic therapy in acquired hemophilia A: Results from the GTH-AH 01/2010 study.
        Blood. 2020; 136: 279-287
        • Lottenberg R.
        • Kentro T.B.
        • Kitchens C.S.
        Acquired Hemophilia: A Natural History Study of 16 Patients With Factor VIII Inhibitors Receiving Little or No Therapy.
        Arch Intern Med. 1987; 147: 1077-1081
        • Bonnefoy A.
        • Merlen C.
        • Dubé E.
        • Claus‐Desbonnet H.
        • Rivard G.
        • St‐Louis J.
        Predictive significance of anti‐FVIII immunoglobulin patterns on bleeding phenotype and outcomes in acquired hemophilia A: Results from the Quebec Reference Center for Inhibitors.
        J Thromb Haemost. 2021;