Background Clinical trial data are scarce for the use of prophylaxis in people with non-severe
haemophilia A. The HAVEN 6 study aims to assess safety and efficacy of emicizumab …

Objective Individuals with deficiency of adenosine deaminase 2 ( DADA 2), a recently recognized
autosomal recessive disease, present with various systemic vascular and inflammatory …

Newborns with hemophilia are at risk of intracranial hemorrhage, extracranial hemorrhage,
and other bleeding complications. The safe delivery of a healthy newborn with hemophilia is …

Extracellular vesicles (EVs) are nanosized membrane-bound particles released from all living
cells examined thus far. EVs can transfer information in the form of proteins, nucleic acids, …

Objective Our objective was to generate, optimize, and validate a self‐administered
pediatric bleeding questionnaire (Self‐PBQ) as a screening tool for von Willebrand disease (VWD) …

Inflammatory signals such as pathogen- and danger-associated molecular patterns have
been hypothesized as risk factors for the initiation of the anti–factor VIII (FVIII) immune …

Background: Emicizumab, a bispecific monoclonal antibody, bridges activated factor (F)IX
and FX, substituting for the function of missing activated FVIII in persons with hemophilia A (HA…

… Devonne Ryan and Paul Moorehead recruited the participants. … Paul Moorehead confirmed
the data analysis. Devonne Ryan drafted the manuscript. Roger Chafe and Paul Moorehead …

The development of neutralizing antibodies (inhibitors) against factor VIII (FVIII) is a major
complication of hemophilia A treatment. The sole clinical therapy to restore FVIII tolerance in …

Objective: There are no proven methods to predict the risk of clinically significant bleeding
in the PICU. A retrospective study identified platelet count as a risk marker for clinically …