A Unique Clinical Case of Using Emicizumab as an Effective Therapeutic Approach for Refractory Acquired Hemophilia

Introduction Acquired Hemophilia A (AHA) is a rare hemorrhagic disorder characterized by spontaneous bleeding resulting from autoantibodies targeting clotting factor VIII. While the standard treatment for AHA involves immunosuppressive therapy, a subset of patients remains unresponsive to these conventional approaches, necessitating alternative treatment strategies. Emicizumab, a bispecific antibody mimicking activated factor VIII, has demonstrated promise in managing congenital Hemophilia A. Nevertheless, limited evidence exists regarding its efficacy in treating AHA.

This case report seeks to provide compelling evidence supporting emicizumab as an effective alternative treatment for refractory AHA patients.

Method We present a case of a 30-year-old female patient with the anamnesis of undifferentiated connective tissue disease and psoriasis diagnosed with AHA at 6 months postpartum. At the time of diagnosis, the patient presented with extensive ecchymoses and heavy uterine bleeding. Factor VIII activity was 2,8% and the inhibitor level was 4,3 Bethesda unit. The patient also reported a family history of systemic lupus erythematosus diagnosed in her mother. Treatment with corticosteroid, hydroxychloroquine therapy, and plasmapheresis were ineffective, in addition to above mentioned unresolved bleeding manifestations she periodically developed hematuria. Emicizumab was administered subcutaneously at a weekly dosage of 3 mg/kg during the first 4 weeks, followed by 2 additional injections every 2 weeks in conjunction with methylprednisolone 8 mg for 3 weeks. Continuous monitoring was conducted through physical examinations and laboratory assessments over a six-month period.

Results Following the administration of emicizumab and during the subsequent six-month follow-up period after emizicumab discontinuation, the patient’s activated partial thromboplastin time (APTT) normalized from 96.1 to 27.8 seconds, factor VIII activity improved to 108-112% and inhibitor level decreased to 0,73-0,66 Bethesda unit. Over the course of the six-month follow-up period after stopping emizicumab, the patient remained free from any bleeding episodes and reported significant enhancement in her quality of life with no emicizumab-related adverse effects observed.

Conclusion Emicizumab emerges as a secure and efficacious therapeutic option for patients grappling with AHA who manifest resistance to conventional immunosuppressive interventions. Further investigations are warranted to deepen our comprehension of the intricate relationship between autoimmune disorders and acquired hemophilia. Nevertheless, this distinctive clinical case from Armenia contributes to the accumulating body of evidence affirming the therapeutic potential of emicizumab in addressing AHA. Notably, the patient's sustained remission without detectable antibodies and restored factor VIII levels post-treatment further distinguishes this clinical case.

Conflict of Interest

The authors declare no conflict of financial or personal interests.

Publication History

Article published online:
26 February 2024