Effectiveness of emicizumab in preventing life‐threatening bleeding complications in type 3 von Willebrand disease with inhibitors: A paediatric report.

The patient experienced a marked improvement of haemorrhagic episodes, which were represented by rarer, mild mucocutaneous bleedings, never requiring hospitalization (notably, he underwent 4 long-lasting hospitalization in the year before the start of treatment) or RBC transfusions. Keywords: bleeding disorders; childhood; emicizumab; factor VIII deficiency; inhibitors; von Willebrand disease EN bleeding disorders childhood emicizumab factor VIII deficiency inhibitors von Willebrand disease e495 e497 3 07/21/21 20210701 NES 210701 Type 3 von Willebrand disease (VWD), the rarest and most severe form of VWD, is an autosomal recessive haemorrhagic condition characterized by virtually undetectable levels of von Willebrand factor (VWF) in plasma, associated with deep deficiency of plasmatic factor VIII (FVIII), till to 1-5% of normal levels. Effectiveness of emicizumab in preventing life-threatening bleeding complications in type 3 von Willebrand disease with inhibitors: A paediatric report. [Extracted from the article]