Hereditary/Acquired Bleeding Disorders

Authors of a small multicenter international study report that plasma-derived von Willebrand factor/factor VIII (pdVWF/FVIII) was effective as on-demand and prophylaxis therapy in previously treated children under 12 years of age with severe hemophilia A. The authors said the results and pharmacokinetic profiles observed were comparable to those seen in adolescent and adult patients with severe hemophilia A, and concluded that overall, their findings “demonstrate a favorable benefit-risk profile for pdVWF/FVIII, and support its use to treat bleeding events in children with hemophilia A.”