These genetic factors constitute the individual genetic risk profile of a haemophiliac patient. Understanding the pathomechanism of inhibitor formation might lead to development of preventive Doxorubicin supplier measures towards inhibitor development and improved treatment as it is recently discussed the application of a new, early prophylaxis regimen. “
“von Willebrand disease (VWD) is probably the most common congenital bleeding disorder while haemophilia, although relatively rare, remains the best characterized. Advances in the prevention and treatment of bleeding episodes in both conditions have translated into improved quality of life and survival

across all age groups. However, improved survival does not come without its own side effect. As patients advance in age, several previously unrecognized morbidities continue to manifest. In this supplement, we highlight the most relevant complications in the ageing population and overview current approaches to their management, while realizing areas of unmet needs. The other focus of the supplement is to shed light upon persisting challenges in the prophylactic treatment of bleeding episodes in patients with haemophilia and VWD, within all age groups. Since the middle of the

last century, clotting factor replacement for persons with haemophilia has evolved from plasma cryoprecipitate to plasma protein-free recombinant factor. Reactionary focus on pathogen BTK inhibitor solubility dmso transmission was addressed in the late 1980s with viral

reduction measures for plasma-derived clotting factor (pd-CF) and the development of recombinant clotting factor (rCF) concentrates. What has resurfaced is the unsolved dilemma of inhibitor development in a substantial number of individuals with haemophilia A, and possibly a differential inhibitor induction rate among recipients of pd-CF containing von Willebrand factor (VWF) and rCF. In addition, renewed attention on the profile of the inhibitor patient has incriminated genetic predispositions, timing and intensity of exposure to CFs and temporally associated MCE immunological danger signals. Cogent studies to address these questions, in the United States and internationally, include SIPPET, the CDC Inhibitor Study and the Haemophilia Inhibitor Genetics Study (HIGS). In addition, the means by which these inhibitors can be eradicated using ITI procedures are under investigation. Prophylactic treatment has been recommended for children with severe haemophilia in view of its efficacy in reducing the number of bleeds and therefore preventing the development of haemophilic arthropathy. Nonetheless, the ideal age for starting prophylaxis and the optimal dosing schedule, remain unclear.