Hemostatic therapy as a management strategy for acquired hemophilia: what does the future hold?

ABSTRACT

Introduction: Acquired hemophilia A (AHA) is a rare autoimmune disease caused by autoantibodies that bind and inactivate factor VIII (FVIII), predisposing to a potentially life-threatening bleeding.

Areas covered: The main epidemiological, clinical, laboratory and therapeutic features of AHA are critically discussed. In particular, we focus on the hemostatic management of AHA patients analyzing the currently available treatment options and showing the latest data on the innovative hemostatic agents still under investigation. Authors searched the Medline and PubMed electronic databases for publication on AHA in the last twenty years.

Expert opinion: While a rapid recognition of suspected cases of AHA is essential to make a correct diagnosis and appropriately and timely treat the hemorrhagic manifestations, the multidisciplinary approach to this challenging, rare and life-threatening bleeding disorder is of equal importance to improve patients’ outcome. Although promising, the safety and efficacy of the clinical use of emicizumab in AHA needs to be validated by trials including an adequate number of patients, before registering the drug also for this indication.

KEYWORDS:

• Acquired hemophilia A
• autoantibodies
• bleeding
• inhibitors

Article highlights

• Acquired hemophilia is a rare bleeding disorder, which affects most commonly elderly people, characterized in the great majority of the cases by autoantibodies against coagulation FVIII.

• The current management of AHA is aimed at eliminating the possible associated underlying disorder, eradicating the FVIII inhibitor, and treating the bleeding symptoms.

• Autoantibodies can be eradicated by immunosuppressive therapy including corticosteroids and cyclophosphamide.

• The hemostatic therapy of AHA includes the use of bypassing agents (i.e. rFVIIa and APCC) and FVIII concentrates (human FVIII and rpFVIII).

• The use of the monoclonal antibody emicizumab as hemostatic therapy in AHA is promising, although more safety and efficacy data are needed.

Declaration of interest

M Schiavulli has received consultancy and lecture fees from Bayer, CSL Behring, Kedrion, Roche/Chugai, and Sobi and has served on advisory boards for CSL Behring, Novo Nordisk, Roche/Chugai, and Sobi.

The authors have no other 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 apart from those disclosed.

Reviewer disclosures

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

Additional information

Funding

This paper was not funded.