Safety and efficacy of a recombinant factor IX (BAX326*) in pediatric previously-treated patients with hemophilia B
Objectives: This prospective clinical trial was conducted to assess the safety, hemostatic efficacy and pharmacokinetic (PK) profile of a recently developed recombinant factor IX (BAX326*) in pediatric previously-treated patients…
An Initiative to Implement Quality Improvement Measures for Hemophilia Treatment Centers
The effects of FXIa on clot formation and lysis in the thrombin generation assay
Objective: Assaying thrombin generation (TG) in real time using fluorogenic substrates has been a popular approach for developing a true global hemostasis assay. Benefits over other assays include assessment of global hemostasis…
Identification of Previously Unreported F8 and F9 Gene Mutations in Hemophilia Subjects From the Phase 3 Clinical Trials of rFVIIIFc and rFIXFc
Objective: Hemophilia A and B are X-linked bleeding disorders caused by the deficiency of clotting factor VIII or IX, respectively. Mutations in the F8 gene can result in hemophilia A while mutations in the F9 gene can lead to…
Mobilizing Patients Towards Positive Health Management Through Motivational Interviewing
Objective: Patient health outcomes are strongly correlated with management of their health condition. In chronic disease management, example hemophilia, a clinician often encounters situations where despite what the patient is…
Real-world dosing of factor in hemophilia A patients
Objective: To analyse real world FVIII dosing and treatment interval patterns in patients with haemophilia A. A secondary objective was to compare the observed dosing patterns with the dosing regimens for rFVIII and rFVIIIFc…
Real-world Dosing Patterns of Factor in Hemophilia B Patients
Objective: To analyse real-world FIX dosing and treatment interval patterns. A secondary objective was to compare the observed dosing patterns with the dosing regimens for FIX products evaluated in clinical studies. …
Global Assessment of Knowledge and Practices in the Diagnosis, Classification, and Management of Hemophilia among Pediatric Providers
Objective: Knowledge gaps among clinicians regarding diagnosis, classification, and/or management in hemophilia can potentially delay diagnosis/referral and lead to adverse clinical outcomes. A study was undertaken to identify…
Retrospective Database Analysis of the Prevalence of Cardiovascular Comorbidities in a US Patient Population with Hemophilia A: Confirmation of Findings
Objective: A previous retrospective study of the MarketScan® claims database reported increased prevalence and earlier onset of cardiovascular (CV) comorbidities in patients with hemophilia A compared with patients without…
Dosing Flexibility in Prophylaxis Regimens With Bayer’s Sucrose-Formulated Recombinant Factor VIII: Experience From Postmarketing Surveillance Studies
Objectives: Factor VIII (FVIII) prophylaxis regimens for severe hemophilia A that allow more flexible dosing than the standard 3-times-weekly regimen while maintaining efficacy may improve adherence. This analysis compared the…