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- Paul Giangrande.
- Oxford Haemophilia Centre and Thrombosis Unit, Churchill Hospital,OX3 7LJ, UK. paul.giangrande@ndm.ox.ac.uk
- Expert Opin Pharmacother. 2005 Aug 1; 6 (9): 1517-24.
AbstractHaemophilia B is an inherited bleeding disorder associated with a deficiency of coagulation factor IX. The hallmark of the severe phenotype is recurrent and spontaneous bleeding into joints, which can lead to joint deformity and arthritis at an early age. Recombinant factor IX is now increasingly regarded as the treatment of choice because it does not transmit human pathogens. All patients in the UK now receive this product exclusively. Conventional treatment now consists of the administration of recombinant factor IX concentrate on a prophylactic basis to prevent bleeds and, hence, minimise disability in the long term. Trials of gene therapy are also underway, but these are in the very early stages and will not be a realistic option for at least another 20 years.
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