This may facilitate much more active lifestyles (permitting greater levels of sports and work participation) while still maintaining a low risk of bleeding. Of course, it is not known what trough level to target. Presumably the higher the trough level targeted, the less risk of bleeding and the more active the patient

can be without the worry of bleeding but with the trade-off of higher cost and more infusions [39]. With these products, patients/families may opt to minimize the number of infusions (lengthening the interval between infusions) while still maintaining the trough level of >1%, or they may opt for more frequent dosing to achieve trough levels that are substantially higher than 1%. Ultimately, some Quizartinib patients might choose one or another or a hybrid depending on their lifestyle: lengthening the interval between infusions as much as possible may be preferred

in less active children/adults, very young patients (e.g. infants) just starting on prophylaxis, and those with poor venous access. More active patients, particularly those with good venous access, might choose to receive more frequent infusions to achieve higher trough levels. All of this leads to increased individualization of prophylaxis. Until Sotrastaurin mw now, with current concentrates, it has been recommended to infuse factor in the mornings [11]. This can be quite burdensome to families who are rushing

to get their children to school and themselves to work. With longer acting factor concentrates (particularly FIXs) there may be less to gain from morning administration. As these products can be given once every 1–2 weeks (some speculate perhaps even less frequently), there may be much to gain with respect to patient adherence if the day of the week chosen to administer the factor could be a non-work/non-school day (e.g. Sunday). Longer acting concentrates may result in patients currently treated on demand now opting to be on prophylaxis. In particular, patients with severe or moderate haemophilia B who are currently treated on demand may opt to receive one prophylactic Sclareol infusion every 2–3 weeks (18–26 infusions/year). Others who are currently not on prophylaxis may choose to be on prophylaxis during certain time periods: when travelling or during particular times of the year when they might be more physically active: e.g. summer. Currently, patients not on prophylaxis (some severe and most moderate) when they travel incur the risk of bleeding and then need medical attention. Such patients with haemophilia B may, with these products, choose to receive a dose of a longer acting factor prior to travelling, which would protect them while they are away. This might greatly relieve patient/family anxiety regarding experiencing a bleed while travelling.