In this review, we outline the standard of care for children in the UK with the most common major bleeding disorder, haemophilia, and how exciting new developments in therapy have the potential for further improvements in quality of life and clinical outcome. The combination of comprehensive specialist medical care, safer factor concentrates, earlier introduction of prophylaxis and patient-specific education has allowed the current generation of patients with haemophilia to grow into adulthood with excellent joint function, pursuing full-time employment with a good quality of life. We are entering an exciting new phase in paediatric haemophilia as potentially life-changing products appear on the scene taking a step towards achieving better, easier and personalised prophylaxis.
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Competing interests None declared.
Provenance and peer review Commissioned; externally peer reviewed.
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