To address many of these issues, the International Prophylaxis Study Group was formed in 2001 [35]. Pharmacokinetics have become a requisite for prophylactic

Selleck Obeticholic Acid dosing. During the 1990s, it could be shown that shortening of dose interval, keeping trough levels, reduced cost at sustained prophylactic efficacy [36, 37]. Even daily dosing has a potential to be feasible in some patients [38]. Pharmacokinetics have more recently been studied in larger international cohorts [39, 40] and the trend is to personalize dosing according to clinical response and individual pharmacokinetics. Long-acting FVIII and FIX concentrates are under study and have a potential to improve prophylaxis, either by using longer intervals than with traditional products, or by raising trough levels. learn more Longer intervals for dosing would improve convenience and compliance. Raising the trough levels has a potential to dramatically increase the long-term medical effect as even patients

receiving so-called high-dose prophylaxis have substantially reduced levels compared to haemostatically normal people. Cost remains the main hurdle for prophylaxis and therefore perhaps the most important wish for the future, irrespective of the type of concentrate used, is a price reduction Gene therapy and cure of haemophilia will totally change the history, but that is another story. The author stated that he had no interests which might be perceived as posing a conflict or bias. “
“Psychosocial outcomes are important in the perspective of boys with haemophilia. However, health-related quality of life (HRQoL) is based on self-report, and assumes adequate literacy. Yet, literacy

is rarely assessed prior to data collection. This study sought to identify criteria that might indicate the level of literacy of children being recruited for clinical trials and to develop a simple method to prescreen those whose literacy was uncertain. We developed a brief screening tool in the form of two stories, Phosphatidylinositol diacylglycerol-lyase at a grade 3 reading level, followed by comprehension questions. We applied the screening test to a sample of haemophilic boys between the ages of 7 and 13 years to assess their literacy. The data were analysed to determine the best criteria to use in identifying the ability to independently self-report for HRQoL studies. Twenty-four Brazilian boys (7.9–12.8) completed the testing. The results showed that 17 (70.8%) were literate (were able to both read and comprehend), and could complete a questionnaire without assistance. All boys over 11.0 years of age were sufficiently literate. Grade level was not found to be a helpful criterion. We recommend that all children under the age of 11.0 years be prescreened before providing self-reported HRQoL data. Those with limited literacy should be provided assistance to ensure comprehension of the questions. This is important to ensure high-quality data on HRQoL for future clinical trials.