Emicizumab: a novel drug in hemophilia A prophylaxis – a narrative review

ABSTRACT

Introduction

Hemophilia A is a genetically conditioned disease leading to hemostatic disorders due to factor VIII (FVIII) deficiency. The treatment of hemophilia has evolved throughout the past years and has significantly changed. One of the newest drugs for prophylactic treatment is the humanized bispecific IgG antibody – emicizumab, which binds with factor IXa and factor X, bridging those factors and thus mimicking the activity of factor VIII.

Areas covered

The literature search was done via the PubMed database, with the emphasis on clinical trials and case reports, describing the off-label emicizumab use. This review presents an extensive summary and considers the advantages and disadvantages (side-effects) of emicizumab, describing additional clinical situations, where emicizumab has been successfully used. In our review, we cover information about the mechanisms of action, indications, and efficacy and discuss some chosen case reports about off-label emicizumab use.

Expert Opinion

Its convenient administration method (subcutaneous) and frequency of injections (from once a week to once a month) makes it a more comfortable treatment, limiting injection–site reactions, hospital stays, costs of prophylaxis, and significantly increasing patients’ quality of life. Adverse effects are scarce and rarely serious – the most common ones are reactions at the injection-site and upper respiratory tract infections.

KEYWORDS:

• Hemophilia A
• emicizumab
• prophylaxis
• hemostasis
• antibody

Article highlights

• Thorough discussion concerning mechanisms of action, indications, adverse effects, efficacy, and off-label uses of emicizumab, showing its superiority over bypassing agents.

• Emicizumab is a novel non-replacement drug for hemophilia treatment, broadening the therapy possibilities of the disease.

• A number of advantages, which the drug is characterized by, make emicizumab a positive alternative to hemophilia A prophylaxis.

• Reducing costs of hemophilia therapy will improve the financial situations of health-care institutions all over the world.

• Off-label emicizumab use reports can supply scientists with new research perspectives and possible future indications, thus broadening their application.

Declaration of Interest

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Additional information

Funding

This paper was not funded.