Emicizumab for the treatment of acquired hemophilia A

Blood

Volumn 137, Issue 3, 2021, Pages 410-419

  Knoebl, Paul ^a   Thaler, Johannes ^a   Jilma, Petra ^a   Quehenberger, Peter ^a   Gleixner, Karoline ^a   Sperr, Wolfgang R ^a  

^a MEDICAL UNIVERSITY OF VIENNA   (Austria)

Author keywords

[No Author keywords available]

Indexed keywords

ACTIVATED PROTHROMBIN COMPLEX; BLOOD CLOTTING FACTOR 8; CYCLOPHOSPHAMIDE; EMICIZUMAB; FILGRASTIM; HEMOGLOBIN; PREDNISONE; RECOMBINANT BLOOD CLOTTING FACTOR 7A; RITUXIMAB; STEROID; BISPECIFIC ANTIBODY; F8 PROTEIN, HUMAN; HEMOSTATIC AGENT; IMMUNOSUPPRESSIVE AGENT; MONOCLONAL ANTIBODY;

ABDOMINAL INJURY; ACTIVATED PARTIAL THROMBOPLASTIN TIME; ADULT; ADVERSE EVENT; AGED; ARTERIOSCLEROSIS; ARTERY BYPASS; ARTICLE; ATRIAL FIBRILLATION; CASE REPORT; CEREBROVASCULAR ACCIDENT; CLINICAL ARTICLE; COHORT ANALYSIS; COMORBIDITY; DETERIORATION; DIABETES MELLITUS; DISEASE DURATION; DISEASE EXACERBATION; DISEASE SEVERITY; DRUG BLOOD LEVEL; DRUG EFFICACY; DRUG HALF LIFE; DRUG SUBSTITUTION; DRUG WITHDRAWAL; ERYTHROCYTE TRANSFUSION; FEMALE; GASTROINTESTINAL HEMORRHAGE; GENERAL ANESTHESIA; HEMARTHROSIS; HEMATURIA; HEMOGLOBIN BLOOD LEVEL; HEMOPHILIA A; HEMOSTASIS; HOSPITAL ADMISSION; HOSPITAL DISCHARGE; HUMAN; HYPERTENSION; IMMUNOSUPPRESSIVE TREATMENT; INTESTINE PERFORATION; ISCHEMIC HEART DISEASE; LUNG TUBERCULOSIS; MALE; METABOLIC SYNDROME X; MUCOSAL BLEEDING; MUSCLE HEMATOMA; NEUTROPENIA; NON ST SEGMENT ELEVATION MYOCARDIAL INFARCTION; PERITONITIS; PRIORITY JOURNAL; PROTEIN BLOOD LEVEL; REMISSION; REPEATED DRUG DOSE; SEPSIS; SINGLE DRUG DOSE; SKIN BLEEDING; THROMBOEMBOLISM; TREATMENT DURATION; TREATMENT RESPONSE; VACUUM ASSISTED CLOSURE; VERY ELDERLY; BLOOD; DOSE RESPONSE; METABOLISM; MIDDLE AGED; TREATMENT OUTCOME;

AGED; ANTIBODIES, BISPECIFIC; ANTIBODIES, MONOCLONAL, HUMANIZED; DOSE-RESPONSE RELATIONSHIP, DRUG; FACTOR VIII; FEMALE; HEMOPHILIA A; HEMOSTATICS; HUMANS; IMMUNOSUPPRESSIVE AGENTS; MALE; MIDDLE AGED; REMISSION INDUCTION; TREATMENT OUTCOME;

EID: 85099905433     PISSN: 00064971     EISSN: 15280020     Source Type: Journal    
DOI: 10.1182/blood.2020006315     Document Type: Article

References (37)

1. Knöbl P. Prevention and management of bleeding episodes in patients with acquired hemophilia A. Drugs. 2018;78(18):1861-1872.
2. Franchini M, Castaman G, Coppola A, et al. Acquired inhibitors of clotting factors: AICE recommendations for diagnosis and management. Blood Transfus. 2015;13(3): 498-513.
3. Tiede A, Scharf RE, Dobbelstein C, Werwitzke S. Management of acquired haemophilia A. Hamostaseologie. 2015;35(4):311-318.
4. Franchini M, Vaglio S, Marano G, et al. Acquired hemophilia A: a review of recent data and new therapeutic options. Hematology. 2017;22(9):514-520.
5. 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.
6. Knoebl P, Marco P, Baudo F, et al; 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.
7. Baudo F, Collins P, Huth-Kü hne A, et al; EACH2 registry contributors. Management of bleeding in acquired hemophilia A: results from the European Acquired Haemophilia (EACH2) Registry. Blood. 2012;120(1):39-46.
8. 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.
9. Tiede A, Amano K, Ma A, et al. The use of recombinant activated factor VII in patients with acquired haemophilia. Blood Rev. 2015; 29(suppl 1):S19-S25.
10. Tiede A, Worster A. Lessons from a systematic literature review of the effectiveness of recombinant factor VIIa in acquired haemophilia [published correction appears in Ann Hematol. 2018;97(12):2531]. Ann Hematol. 2018;97(10):1889-1901.
11. 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.
12. Tü rkantoz H, Königs C, Knöbl P, et al. Crossreacting 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.
13. 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.
14. Zanon E, Pasca S, Siragusa S, et al; FAIR Study Group. Low dose of aPCC after the initial treatment in acquired haemophilia A is useful to reduce bleeding relapses: Data from the FAIR registry. Thromb Res. 2019;174:24-26.
15. 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.
16. 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.
17. Tiede A, Hofbauer CJ, Werwitzke S, et al. Antifactor VIII IgA as a potential marker of poor prognosis in acquired hemophilia A: results from the GTH-AH 01/2010 study. Blood. 2016;127(19):2289-2297.
18. Tiede A, Huth-Kü hne A, Oldenburg J, et al. Immunosuppressive treatment for acquired haemophilia: current practice and future directions in Germany, Austria and Switzerland. Ann Hematol. 2009;88(4):365-370.
19. Wiestner A, Cho HJ, Asch AS, et al. Rituximab in the treatment of acquired factor VIII inhibitors. Blood. 2002;100(9):3426-3428.
20. Sperr WR, Lechner K, Pabinger I. Rituximab for the treatment of acquired antibodies to factor VIII. Haematologica. 2007;92(1):66-71.
21. Franchini M, Mannucci PM. Inhibitor eradication with rituximab in haemophilia: where do we stand? Br J Haematol. 2014;165(5): 600-608.
22. Werwitzke S, Geisen U, Nowak-Göttl U, et al. Diagnostic and prognostic value of factor VIII binding antibodies in acquired hemophilia A: data from the GTH-AH 01/2010 study. J Thromb Haemost. 2016;14(5):940-947.
23. 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.
24. 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.
25. Reyes A, Ré vil C, Niggli M, et al. Efficacy of emicizumab prophylaxis versus factor VIII prophylaxis for treatment of hemophilia A without inhibitors: network meta-analysis and sub-group analyses of the intra-patient comparison of the HAVEN 3 trial. Curr Med Res Opin. 2019;35(12):2079-2087.
26. 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, non-randomised phase 3 study. Lancet Haematol. 2019;6(6):e295-e305.
27. Oldenburg J, Mahlangu JN, Kim B, et al. Emicizumab prophylaxis in hemophilia A with inhibitors. N Engl J Med. 2017;377(9): 809-818.
28. 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(2): 121-123.
29. Dane KE, Lindsley JP, Streiff MB, Moliterno AR, Khalid MK, 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(3):420-423.
30. 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.
31. Adamkewicz JI, Chen DC, Paz-Priel I. Effects and interferences of emicizumab, a humanised bispecific antibody mimicking activated factor VIII cofactor function, on coagulation assays. Thromb Haemost. 2019;119(7): 1084-1093.
32. Peyvandi F, Kenet G, Pekrul I, Pruthi RK, Ramge P, Spannagl M. Laboratory testing in hemophilia: impact of factor and non-factor replacement therapy on coagulation assays. J Thromb Haemost. 2020;18(6):1242-1255.
33. Shima M, Hanabusa H, Taki M, et al. Factor VIII-mimetic function of humanized bispecific antibody in hemophilia A. N Engl J Med. 2016;374(21):2044-2053.
34. 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.
35. Kotani N, Yoneyama K, Kawakami N, Shimuta T, Fukase H, Kawanishi T. Relative and absolute bioavailability study of emicizumab to bridge drug products and subcutaneous injection sites in healthy volunteers. Clin Pharmacol Drug Dev. 2019;8(6):702-712.
36. 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.
37. Kruse-Jarres R, Callaghan M, Croteau SE, et al. Surgical experience in two multicentre, openlabel phase 3 studies of emicizumab in persons with haemophilia a with inhibitors (haven 1 and haven 2) [abstract]. Br J Haematol. 2018; 181(suppl 1). Abstract 128.