Advances in managing rare acquired bleeding disorders

ABSTRACT

Introduction

Rare acquired bleeding disorders include a wide spectrum of coagulopathies characterized by spontaneous or post-trauma and post-surgery hemorrhages in patients without a previous personal or family history of bleeding.

Areas covered

This review, based on a Medline/PubMed search during the last 20 years, will focus mainly on rare acquired bleeding disorders caused by autoantibodies against coagulation factors, including autoantibodies against factor VIII (acquired hemophilia A), von Willebrand factor (acquired von Willebrand syndrome) and other coagulation factors (factors V, X, XI, and XIII). The pathogenic, laboratory, and clinical features of these rare hemorrhagic conditions will be discussed, with particular attention to their management.

Expert opinion

The treatment of rare acquired bleeding disorders includes the control of bleeding and the elimination of the autoantibody and of the underlying disease, when present. As the bleeding clinical phenotype is often severe, the management of affected patients is particularly challenging. Thus, while an early diagnosis of the acquired coagulopathy is essential to start the most appropriate treatment and to improve patients’ outcomes, the support of specialized centers is equally important to provide a correct management of such complicated cases.

KEYWORDS:

• Acquired hemophilia A
• acquired von Willebrand syndrome
• autoantibodies
• bleeding
• clotting factors
• therapy

Article highlights box

• Rare acquired bleeding disorders, which are in the majority of cases caused by autoantibodies against clotting factors, include a wide spectrum of clinical manifestations ranging minimal or no bleeding to severe and life-threatening hemorrhagic events.

• Among rare acquired bleeding disorders, acquired hemophilia A (AHA) is the most clinically relevant.

• The mainstay of treatment of AHA is based on anti-hemorrhagic therapy with bypassing agents and inhibitor eradication using a combination of steroids and cytotoxic agents.

• The main goals of the management of acquired von Willebrand syndrome (AVWS) include the control of acute bleeding, the long-term remission, and the treatment of the underlying disorder.

• The incidence of autoantibodies against factor V (FV) has significantly declined following the introduction of recombinant thrombin products.

• Acquired factor X (FX) deficiency has also been reported, especially in association with amyloidosis.

• Acquired factor XI (FXI) deficiency is occasional and often associated with systemic lupus erythematosus.

• Among the rare acquired bleeding disorders other than AHA and AVWS, autoantibody-related acquired factor XIII (FXIII) deficiency is that with the most severe clinical bleeding.

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.