SAFE Study: Safety of aPCC Following Emicizumab Prophylaxis

SAFE Study: Safety of APCCaPCC Following Emicizumab Prophylaxis

Inclusion Criteria: Moderately severe hemophilia A, defined as FVIII level <0.0205 IU/mL in the central laboratory prior tobefore development of an inhibitor Age ≥6 years of age at time of [...] -hour washout within 2 years of enrollment Parent/guardian (caregiverLegally henceforthAuthorized Representative) or the patient has provided written informed consent Adequate hema [...] hemophilia A excluding low VWF (>30% VWF:RCo or VWF:GP1bm) Had an active bleed requiring factor therapy at screening Previous or current treatment for thromboembolic disease or signs of thromboembolic disease (excluding previouspr [...] [...]

Number of infusionsserious adverse events Number of aPCCserious requiredbleeding to achieve hemostatic efficacy for treatmentepisodes Number of an acute bleeding episode, or preventionepisodes of bleedingthrombotic withevents emergentincluding andthrombotic non-emergentmicroangiopathy procedures(TMA)

NumberThrombin ofgeneration episodescapacity ofafter thrombotic events including thrombotic microangiopathyaPCC (TMAFEIBA) Number of serious adverse events Number of serious bleeding episodesinfusion

Hemophilia A (HA) is a congenital bleeding disorder caused by deficient or dysfunctional factor VIII (FVIII) which leads to bleeding correlated with factor deficiency severity. Patients with HA develop recurrent bleeds into joints and soft tissues that culminate into debilitating arthropathy and lon [...] The previous standard of care for high titer antibody eradic [...] concentrate (aPCC) given intravenously (IV) each week. The purposeoverall of the aPCC-emicizumab safety studyobjective is to prospectivelydetermine investigatewhether the safetythrombin andgeneration hemostaticassay efficacycan ofbe used t [...] [...]

SAFE Study: Safety of aPCCAPCC Following Emicizumab Prophylaxis