1,721,194 research outputs found
Prospective controlled studies on prophylaxis : an Italian approach
No full textThe benefits of prophylactic infusion of clotting factor in haemophilia A and B in delaying the onset of arthropathy are supported by retrospective data, but the possibility of selection bias and confounding factors cannot be ruled out by such studies. Many issues that have only been studied retrospectively require definitive data, including the optimal dose and infusion intervals, as well as when to start prophylaxis and when to stop it. Issues of cost-effectiveness of prophylaxis, quality of life and treatment satisfaction of patients on prophylaxis and their families also require prospective data. To advance the evidence-based approach in haemophilia treatment, ESPRIT (Evaluation Study on Prophylaxis: a Randomized Italian Trial), currently underway, is a prospective randomized 10-year study that will compare the efficacy and safety of prophylaxis and on-demand regimens in preventing joint deterioration and in reducing the number of bleeds in patients with severe haemophilia
VWF/FVIII concentrates in high-risk immunotolerance : the RESIST study
No full textImmune tolerance induction (ITI), through the regular infusion of coagulation factor concentrates over a time period ranging from 1 to more than 24 months, is successful in about 75% of high responders. Among the issues of ITI treatment that are still open, the choice of the product to use is one of the most difficult. In fact, common practice is to start with the same product that induced the inhibitory response, but recent findings indicated that plasma-derived products containing large amounts of von Willebrand factor (VWF) can play a positive role. Two retrospective cohorts in Germany and in France and one prospective cohort have shown a high rate of success when VWF/factor VIII (FVIII) products are used to induce ITI. For these reasons, two prospective studies have been planned to complement the international ITI study already started: an observational study in patients who had already experienced a failure with a VWF-free FVIII concentrate, called RESISTexp (experienced); a randomized, controlled study in patients who have never tried an ITI treatment before and at high risk to fail, called RESISTnaïve (naïve
Factor VIII safety : plasma-derived versus recombinant products
No full textIt is impossible to understand the importance of safety issues regarding the use of plasma-derived and recombinant clotting factor concentrates without looking back at the evolution of the management of haemophilia over the last 50 years. The treatment of people with haemophilia improved radically following the discovery by Judith Pool in the fi rst half of the 1960s of the possibility of concentrating antihaemophilic factors by cryoprecipitation of plasma1,2. Various techniques for further concentrating and purifying the coagulation factors were introduced such that these factors became increasingly available and easier to infuse3. This opened up a whole new world in which people with haemophilia could treat themselves more rapidly thus limiting bleeding into their joints, could treat themselves at home thus decreasing their dependence on the emergency room, could treat themselves prophylactically prior to activities that might cause bleeding, and could travel. This progress in the management of haemophilia has dramatically improved not only the life-expectancy of these patients but also their quality of lif
Long-term aspects of hemophilia B treatment : part II
No full textThis paper focuses on the benefits of prophylaxis, compared with on-demand therapy, in achieving long-term treatment goals in hemophilia patients. While primary treatment goals are to prevent joint damage and improve quality of life, other issues such as treatment costs-balanced against improvements in health status and direct and indirect costs of hemophilic arthropathy-need to be considered. The ESPRIT study (Evaluation Study on Prophylaxis: a Randomized Italian Trial), a 10-year trial comparing on-demand and prophylactic treatment in terms of efficacy, safety, and cost, is discussed. Early results support findings from other studies regarding the benefits of prophylaxis. Prospective pharmacoeconomic evaluations are needed to help define optimal treatment programs for patients with hemophilia A and
Immunogenicity of factor VIII concentrates in patients with hemophilia : a randomized clinical trial is warranted
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