Executive summary of the NHLBI State of the Science (SOS) Workshop: Overview and next steps in generating a national blueprint for future research on factor VIM inhibitors

摘要

As elaborated by Sabatino et al, five decades of advances have brought us widespread availability of safe and effective haemophilia treatment. However, inhibitors, neutralizing alloantibodies to factor VIII, still occur in -25%-30% of persons with severe haemophilia A. Within the US, it is estimated that there are over 1000 individuals with a factor VIM inhibitor.2 Inhibitors are associated with increased risk from bleeding and twice the rate of hospitalization for a bleeding complication, increased morbidity from joint disease and significantly increased rate of death due to bleeding-related causes compared to those without inhibitors. Over the last 30 years, we have gained insights on risk factors for inhibitor development from retrospective and parallel-cohort studies as well as meta-analyses.3 However, there is still much to be learned about the basic mechanisms underlying this immune response and few studies that have significantly impacted inhibitor prevention and eradication.