4.5 Article

A new population pharmacokinetic model for recombinant factor IX-Fc fusion concentrate including young children with haemophilia B

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Summary: Haemophilia B is a rare genetic deficiency that can cause severe bleeding if untreated. The management of haemophilia B has improved in recent decades, with the development of new therapies. However, the evidence base for clinical guidelines is limited, and existing guidelines for haemophilia A cannot be extrapolated. To address this, an international Author Group developed a clinical practice framework based on a systematic review of the literature and clinical experience. The framework includes 29 recommendations for the management of haemophilia B in five areas. It is expected that this framework will complement existing guidelines and can be adapted for use in different regions and countries.

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