Recent Advances in Haemophilia Management

Abstract
Haemophilia is a life-threatening bleeding disorder characterised by recurrent, spontaneous bleeding episodes into muscles and joints leading to the development of painful, disabling haemophilic arthropathy. Significant improvements in the management of patients with haemophilia have occurred in the last four decades with the widespread availability of safe and effective clotting factor concentrates. Modern treatment of haemophilia is still associated with significant morbidity and recent advances in the care of patients with haemophilia including the development of modified clotting factor concentrates with extended half-life hold promise to significantly reduce the burden treatment of this bleeding disorder. The benefits of sophisticated clinical registries documenting patient reported outcomes, improvements in the management of patients who develop antibodies to infused clotting factor concentrate and a review of the current status of gene therapy for haemophilia are examples of advances in haemophilia management and will be reviewed in this manuscript.

Source: Current Pediatrics Reports - March 28, 2015 Category: Pediatrics Source Type: research