With the advent of longer acting factor concentrates, prophylaxis regimens will almost certainly change. selleck kinase inhibitor This will involve changes in what trough levels are targeted and how frequently factor is administered. These products will cause investigators to consider the relative importance of trough vs. peak levels in the effectiveness of prophylaxis [14]. Changes in regimens may improve patients’ adherence to prophylaxis and patients’ quality of life. Definitions of the minimum infusion frequency
to still be considered prophylaxis will obviously change, as will definitions for full, intermediate, and low-dose prophylaxis. Finally, these long-acting factor concentrates will undoubtedly have cost repercussions and, given that these products will be substantially different from each other, they will raise important questions regarding how decisions about choosing one longer acting concentrate http://www.selleckchem.com/products/BAY-73-4506.html over another, and whether these products are interchangeable, are made. The following sections will deal with each of these implications of longer acting factor concentrates. With these newer concentrates, patients will have the option of lengthening the interval between infusions
while still achieving a factor trough level of >1%. Patients with severe haemophilia B who currently may take two infusions/week (104 infusions/year) might be just as protected from bleeds with perhaps one infusion every 1–3 weeks (18–52 infusions/year) [36]. Patients with severe haemophilia A might be able to receive two infusions/week (102/year) or one infusion every 3–5 days and still see more maintain a trough level of >1% [37]. This compares to current regimens, where on full-dose prophylaxis patients with severe haemophilia A will receive 156–182 infusions annually
(3–3.5 infusions/week). Decreasing the number of infusions should reduce the need for CVADs (and their consequent sequelae). A further benefit of decreasing the number of infusions is that when commencing patients on prophylaxis, fewer clinic visits will be required for those patients/families who are as yet unable to infuse factor at home. It will also reduce home care nurse visits where this is an option. All of this may translate into earlier start of prophylaxis, fewer missed doses, and overall better bleed protection. There may also be drawbacks to maximizing the interval between infusions, as it will result in patients having low factor levels for extended periods of time during which they may be physically active and at risk of bleeding. Until now, the relatively short half-lives of factor concentrates and their very high cost precluded patients maintaining trough levels during prophylaxis (even on full-dose prophylaxis) of much higher than 1%. Although such trough levels have been demonstrated to reduce the frequency of spontaneous bleeds, they certainly do not protect against traumatic bleeds where higher factor levels are required [38].