Background: Acquired factor VIII deficiency is a rare coagulopathy caused by an autoantibody (inhibitor) to factor VIII (FVIII) leading to severe, potentially life-threatening hemorrhage. Disease management concentrates on permanently eradicating the autoantibody using immunosuppressants and achieving hemostasis, primarily with bypassing therapy and recombinant porcine FVIII. Emicizumab, a monoclonal antibody, designed to promote hemostasis in the congenital factor VIII deficient patient, has shown hemostatic efficacy in both congenital factor VIII deficient patients with and without inhibitors.

Aims: To assess the efficacy of emicizumab prophylaxis in prevention of bleeding symptoms in patients with acquired hemophilia.

Methods: Retrospective record review of a 57 year old female and a 90 year old male with multiple comorbidities diagnosed with acquired factor VIII at the Gulf States Hemophilia and Thrombophilia Center of Houston, Texas on standard and modified off label emicizumab prophylaxis.

Results: Average bleed event prior to emicizumab prophylaxis occurred twice a month. 1^st Patient (female) – Standard Protocol: Loading dose ~3 mg/kg/ dose subcutaneous once every 7 days for four doses. Maintenance ~1.5 mg/kg/dose subcutaneous once every 7 days. 2^nd Patient (male) – Texas Protocol: Loading dose ~1.5 mg/kg/dose subcutaneous once every 7 days for two doses. Maintenance dose ~1.5 mg/kg/dose subcutaneous once every 21 days. Peak inhibitor titer at start of protocol= female- 6.67 B.U. and male- 150.4 B.U. Following start of emicizumab, both Standard and Texas Protocol, immediate cessation of bleeding episodes were noted.

Conclusions: Emicizumab is a monoclonal antibody that promotes hemostasis without replacing factor VIII, while the use in acquired hemophilia is very limited these cases illustrate its efficacy and safety using standard and modified low dose prophylaxis (Texas Protocol) in acquired hemophilia patients with multiple comorbidities. Further studies in this disease are warranted.

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ISTH Congress Abstracts - https://abstracts.isth.org/abstract/prophylactic-potential-of-standard-and-modified-emicizumab-prophylaxis-in-2-patients-with-acquired-hemophilia-a-case-report/