As the BDD long-acting products come into the mainstream
of haemophilia care, however, it is likely that laboratories will need to offer a range of FVIII activity assays with the real possibility of requiring a different assay for each long-acting FVIII product. While this editorial dealt with FVIII products, the new generation FIX concentrates have similar issues. MM has acted as consultant to CSL Behring and NovoNordisk. He took part in an Advisory Panel organized by BPL and gave lectures and his institution has received honoraria for Baxter, Bayer, Biogen Idec, Biotest, Octapharma, Pfizer and SOBI. He has received travel support from Baxter and Bayer. FP has received honoraria Pirfenidone for participating as speaker at educational meetings organized by Novo Nordisk, CSL Behring, Bayer and Baxter. Research Grant: Novo Nordisk (for FXIII deficiency). “
“Prophylaxis, i.e. regular selleckchem preventive infusions of factor replacement therapy [Factor VIII (FVIII) or Factor IX (FIX)] [1,2], is widely accepted as the gold-standard treatment in children with severe haemophilia [3,4]. Long-term prophylaxis has proven effective in reducing bleeds and thus preventing haemophilic arthropathy [5–7]. Given the acceptance of prophylaxis as a mainstay of bleeding control and prevention in patients with haemophilia, it is surprising that unresolved differences remain concerning
regimen implementation and dosing schedules. Treatment of patients who develop inhibitors (antibodies) to factor replacement may include prophylaxis with bypass agents, 上海皓元医药股份有限公司 but few results are available in this field and evidence for efficacy are of the lowest level. Prevention
in patients with haemophilia’ was an international meeting held in Marseille, France, on 2 July 2009, that involved world-renowned experts based in Europe and North America who met to review country-specific variations in current prophylaxis practice, and discuss future directions in haemophilia management for patients with and without inhibitor. This supplement is a compilation of the presentations and discussions held at the meeting that highlight experience with haemophilia prophylaxis in a global context, while also reviewing current data and areas for ongoing research. Frequency of prophylaxis use in boys without inhibitor varies between countries, which may be a reflection of differences in timing of treatment initiation or intensity of treatment in the regimens used. In the first article of this supplement, Professors Rolf Ljung, Manuel Carcao and I discuss current prophylaxis treatment strategies employed in Swedish, Canadian and French centres. Using these examples, the authors outline areas of agreement (e.g. early prophylaxis) or debate (e.g. when to intensify treatment) that, with further refinement, could be used to optimize practice approach.