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A trial randomised 65 boys with severe haemophilia A, up to 30 months old at recruitment, to receiving prophylactic regular infusions of recombinant factor VIII every other day or to episodic treatment at the time of clinically evident joint haemorrhage. After a median follow-up of 49 months, at 6 years of age, 25 of the 27 boys who were randomised to prophylaxis and completed the trial had no damage to ankle, knee, or elbow joints, as assessed by magnetic resonance imaging, compared with 16 of 29 boys in the episodic treatment group. Boys randomised to prophylaxis also had fewer clinically evident joint haemorrhages (median 0.2 v 4.4 occurrences per participant per year) and fewer total haemorrhages (median 1.2 v 17.1 occurrences per participant per year).
Despite clinical benefits, which fit well with previous evidence, the major factor limiting widespread uptake is the high cost of recombinant factor VIII—estimated at an average of $300000 per patient per year. Still, the linked editorial (pp 603-5) reminds that, untreated, severe haemophilia A incurs great costs to the health systems later on in boys' lives, such as costs of replacing joints. In the absence of relevant trials and any formal cost effectiveness analyses, the optimal prophylaxis regimen still eludes us.us.