Inhibitor development in haemophilia B: an orphan disease in need of attention. Review uri icon

Overview

abstract

  • Factor IX (FIX) inhibitors develop in 1.5-3% of haemophilia B patients. Due to its low incidence compared with that in haemophilia A, few comparable data exist on host and treatment-related risk factors, and immunological processes associated with FIX inhibitor development. Moreover, the safety and efficacy of bypass therapy as well as the outcome predictors of successful inhibitor eradication have been poorly characterised. The lack of a useful evidence-based approach to the diagnosis and management of FIX inhibitors complicates their significant morbidity due to the frequency of allergic reactions that often herald antibody development. This review discusses what is currently known about the epidemiology, natural history and immunology of anti-FIX antibody development. It addresses several special considerations in the approach to the treatment of bleeding and inhibitor eradication. A case is made for moving forward with an integrated international collaboration for the further study of the nature and treatment of this problem.

publication date

  • August 1, 2007

Research

keywords

  • Blood Coagulation Factor Inhibitors
  • Factor IX
  • Hemophilia B
  • Rare Diseases

Identity

Scopus Document Identifier

  • 34447128837

Digital Object Identifier (DOI)

  • 10.1111/j.1365-2141.2007.06657.x

PubMed ID

  • 17614818

Additional Document Info

volume

  • 138

issue

  • 3