Diagnosis, therapeutic advances, and key recommendations for the management of factor X deficiency


Peyvandi F., Auerswald G., Austin S. K. , Liesner R., Kavakli K. , Roman M. T. A. , ...More

BLOOD REVIEWS, vol.50, 2021 (Journal Indexed in SCI) identifier identifier identifier

  • Publication Type: Article / Review
  • Volume: 50
  • Publication Date: 2021
  • Doi Number: 10.1016/j.blre.2021.100833
  • Title of Journal : BLOOD REVIEWS
  • Keywords: Diagnosis, Factor X deficiency, Plasma-derived factor X concentrate, Prothrombin complex concentrates, Rare bleeding disorders, Treatment, RARE BLEEDING DISORDERS, FRESH-FROZEN PLASMA, PROTHROMBIN COMPLEX CONCENTRATE, LIGHT-CHAIN AMYLOIDOSIS, COAGULATION DISORDERS, REPLACEMENT THERAPY, HEMOPHILIA, EFFICACY, PHARMACOKINETICS, MANIFESTATIONS

Abstract

Factor X deficiency is a rare coagulation disorder that can be hereditary or acquired. The typology and severity of the associated bleeding symptoms are highly heterogeneous, adding to the difficulties of diagnosis and man-agement. Evidence-based guidelines and reviews on factor X deficiency are generally limited to publications covering a range of rare bleeding disorders. Here we provide a comprehensive review of the literature on factor X deficiency, focusing on the hereditary form, and discuss the evolution in disease management and the evidence associated with available treatment options. Current recommendations advise clinicians to use single-factor replacement therapy for hereditary disease rather than multifactor therapies such as fresh frozen plasma, cry-oprecipitate, and prothrombin complex concentrates. Consensus in treatment guidelines is still urgently needed to ensure optimal management of patients with factor X deficiency across the spectrum of disease severity.