Participants in an international conference on the management of haemophilia patients with inhibitors developed a jointly authored summary of the findings and conclusions of the conference. Current knowledge of the genetic and immunologic mechanisms underlying inhibitor development was briefly summarized. Concerning the purported treatment-related risk factors, conference participants commented on the limitations of the available evidence and the need for more rigorous prospective research in a fully genotyped population. Other clinical considerations discussed included the unproved utility of routine surveillance, the need for assay standardization, the management of acute bleeding and approaches to joint disease prophylaxis and immune tolerance induction (ITI). A number of issues were identified as needing further investigation in larger prospective studies, ideally through international cooperation. Such studies should enroll cohorts that have been scrupulously defined in terms of mutation status and treatment exposure. Finally, conference participants urged their colleagues to participate in the currently ongoing international trials of ITI.
below the 70 th percentile and 1 (poor adherence) if above the 70 th percentile. ABR was also dichotomized into no bleeds (0 ABR), the true goal of prophylaxis, versus 1 or more bleeds (1+ ABR). Results: Reported ABR ranged from 0 to 54 (median 1). There was not a significant relationship between VERITAS-Pro score and ABR. There was a significantly greater percentage of patients experiencing one or more bleeds in the VERITAS-Pro poor adherence group (86%) than in the VERITAS-Pro good adherence group (62%). ConClusions: VERITAS-Pro scores reflecting adherence are significant predictors of ABR; individuals with poor adherence on the VERITAS-Pro are more likely to have at least 1 bleed per year versus those with good adherence. However, the high percentage of patients experiencing one or more bleeds in both groups indicates that factors other than adherence may impact annualized bleed rates.
scite is a Brooklyn-based organization that helps researchers better discover and understand research articles through Smart Citations–citations that display the context of the citation and describe whether the article provides supporting or contrasting evidence. scite is used by students and researchers from around the world and is funded in part by the National Science Foundation and the National Institute on Drug Abuse of the National Institutes of Health.