Emicizumab for the treatment of acquired hemophilia A: Retrospective review of a single‐institution experience

Chen, Evan C.; Gibson, William J.; Temoczko, Paula; Connell, Nathan T.; Handin, Robert I.; Parnes, Aric

doi:10.1111/hae.14664

Cited by 10 publications

(2 citation statements)

References 19 publications

(56 reference statements)

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“…Recently, in several case reports and case series, the off-label use of emicizumab in AHA patients was reported [41][42][43][44][45][46]. Emicizumab is a recombinant, humanized and bispecific monoclonal antibody with FVIII mimetic activity, approved for the prophylaxis of congenital hemophilia A with and without the anti-FVIII inhibitor [47].…”

Section: Future Therapeutic Approachesmentioning

confidence: 99%

Acquired Hemophilia A: Bleeding Pattern and Hemostatic Therapeutic Strategies

Marino

2023

Medicina

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Acquired Hemophilia A (AHA) is a rare autoimmune disorder characterized by the onset of a sudden and unexpected bleeding episode in a patient with no personal or family history of bleeding diathesis, and with a typical laboratory feature, i.e., a prolonged activated partial thromboplastin time that is not otherwise explained. This bleeding disorder is caused by autoantibodies directed against the coagulation factor VIII (FVIII). AHA is idiopathic in 50% of cases and is secondary to well-defined diseases in the remaining 50%. AHA affects elderly patients although it has also been observed in the post-partum period. Bleeding manifestations are heterogeneous, ranging from mild to life-threatening bleeds involving limbs and organs. Severe bleeding with a significant decrease in hemoglobin levels must be promptly and adequately treated in order to avoid a worsening of the hemorrhages and their complications. According to international recommendations, the bypass agents (i.e., activated prothrombin complex concentrate and activated recombinant factor VII) and the replacement therapy with recombinant porcine FVIII are considered as the first-line therapy for bleeding control, due to their proven clinical efficacy. Plasma-derived or recombinant FVIII concentrates could be used as second-line treatments. Emicizumab may represent a valid and interesting therapeutic option for prophylaxis of bleeding recurrences.

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Section: Future Therapeutic Approachesmentioning

confidence: 99%

Acquired Hemophilia A: Bleeding Pattern and Hemostatic Therapeutic Strategies

Marino

2023

Medicina

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“…However, no randomized trials addressed the efficacy of emicizumab in AHA. To complement the limited published case series, 7–11 we share our experience of emicizumab as a haemostatic agent in patients with AHA.…”

Section: Introductionmentioning

confidence: 99%

Emicizumab for acquired haemophilia A: A case series

Engelen

Vandesande²,

Bent³

et al. 2023

Haemophilia

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Background Emicizumab is approved to prevent bleeding in patients with congenital haemophilia A with or without inhibitors. However, no randomized trials addressed the efficacy of emicizumab in acquired haemophilia A (AHA). Aims To report the clinical and biochemical response of emicizumab in AHA. Methods This single‐centre retrospective study included seven adults with AHA between November 2020 and May 2022. We collected patient characteristics, laboratory coagulation parameters, the use of haemostatic agents, bleeds and thrombotic events. Treatment was monitored using chromogenic FVIII assays. The assay with human reagents assesses both the emicizumab FVIII‐like‐activity and native patient FVIII‐activity. The assay with bovine reagents only measures the patients’ native FVIII‐activity as emicizumab does not bind to bovine reagents. Results Patients presented with spontaneous hematoma (n = 7), intramuscular bleeding (n = 2), haematuria (n = 2) and/or gastro‐intestinal bleeding (n = 2). Six patients had major bleedings. At diagnosis, APTT was prolonged (91 seconds, IQR 73–103), FVIII activity was 0% (IQR 0–1) and FVIII inhibitor 182 BU/mL (IQR 104–228). Emicizumab was administered weekly (3 mg/kg) for 4 weeks, and thereafter every 2 weeks until regression of the inhibitor. Three patients received activated FVIIa (cumulative dose of 1.7 mg/kg, IQR 1.2‐2.2). All bleedings were controlled after treatment initiation, without further bleeds. After starting emicizumab, FVIII‐like activity reached ≥5% at 12 days (IQR 7–14), whereas recovery of the intrinsic FVIII‐activity ≥5% occurred at 128 days (IQR 88–173), coinciding with the disappearance of the FVIII inhibitor. There were no safety issues. Conclusion In this AHA case series, no new clinically relevant bleeds were observed after initiation of emicizumab in conjunction with standard immunosuppressive therapy.

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Acquired hemophilia A as a disease of the elderly: A comprehensive review of epidemiology, pathogenesis, and novel therapy

Lehoczki,

Fekete,

Mikala

et al. 2024

GeroScience

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Acquired hemophilia A (AHA) is a rare autoimmune bleeding disorder characterized by the development of neutralizing autoantibodies (inhibitors) against coagulation factor VIII (FVIII). This review provides an in-depth exploration of AHA, covering its epidemiology, pathogenesis, clinical presentation, diagnosis, complications, and treatment strategies, focusing on recent advancements. AHA can manifest in both men and women with no prior bleeding history. The annual incidence is estimated to be approximately 1 case per million individuals in the general population. The incidence increases significantly with age: the incidence among individuals aged 60 years or older is approximately 3 to 4 cases per million individuals per year. Typically, patients present with an acquired bleeding disorder that is characterized by an isolated, prolonged activated partial thromboplastin time stemming from FVIII deficiency. Diagnosis relies on the detection of neutralizing antibodies using the Nijmegen-modified Bethesda assay. Hemostatic control strategies involve bypassing agents like recombinant activated factor VII, activated prothrombin complex concentrate, and recombinant porcine FVIII for bleeding patients. Emicizumab, a novel bypassing agent, exhibits several potential advantages. In the realm of immunosuppressive treatment for inhibitor eradication, the CyDRi regimen emerged as a remarkable advancement, significantly enhancing the outlook for the management of AHA even in the elderly frail population.

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Emicizumab for the treatment of acquired hemophilia A: Retrospective review of a single‐institution experience

Cited by 10 publications

References 19 publications

Acquired Hemophilia A: Bleeding Pattern and Hemostatic Therapeutic Strategies

Acquired Hemophilia A: Bleeding Pattern and Hemostatic Therapeutic Strategies

Emicizumab for acquired haemophilia A: A case series

Acquired hemophilia A as a disease of the elderly: A comprehensive review of epidemiology, pathogenesis, and novel therapy

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