emerging hemofilia
DESCRIPTION
Emerging hemofiliaTRANSCRIPT
Moderator
Panelists
Learning Objectives
Prophylaxis: Why > 1%?
Optimal Use of Prophylaxis
Factor Product Modification
Extended Half-life Factor Products
Monitoring: Changing Landscape?
Advances in Monitoring Techniques
Outline
Case Presentation
Factors to Weigh in Choosing Treatment Options
Outline
Early Findings With Prophylaxis
Early Findings With Prophylaxis (cont)
Joint Outcome Study
Results of the Joint Outcome Study
ESPRIT Study
ESPRIT Study – Analyses
Outcome Measures – Raising the Bar
Outcome Measures – Raising the Bar (cont)
Outcome Measures – Raising the Bar (cont)
Outline
Ongoing Issues
When Should Prophylaxis Be Started?
Which Regimen Should Be Used?
Which Regimen Should Be Used? (cont)
Which Regimen Should Be Used? (cont)
Which Regimen Should Be Used? (cont)
Regimens by Regions
Regimens by Regions (cont)
Regimens by Regions (cont)
Outline
Ongoing Issues
What Needs To Be Investigated?
When Should Prophylaxis Stop?
When Should Prophylaxis Stop? (cont)
Take-home Messages
Return to Case
Case Discussion
Case Presentation
Outline
Current Outcomes
Half-life Extension of Biologics
Extended Half-life FIX Products
Extended Half-life FVIII Products (cont)
FVIII-VWF Interaction
rFVIIIFc
Phase 1 Trial of rFVIIIFc
rFVIIIFc Phase 3 Trial Results
rFVIIIFc Phase 3 in Children
rFVIIIFc Assay Field Study
BAY 94-9027
Phase 1 Study of BAY 94-9027
Phase 1 Study of BAY 94-9027 (cont)
Phase 2/3 Study of BAY 94-9027
BAY 94-9027 aPTT Study
N8-GP Phase 3 Trial
Glycopegylated FIX
Phase 3 Results With N9-GP
Clinical Trial Regimens
What’s Next?
Untested Regimens
Return to Case
Case Discussion
Case Presentation
Issues Involved in Switching
Duration of Outcome Assessment Needs To Be Long (Decades) and Hampers Clinical Studies
How to Make the Bridge From Standard to Extended Half-life Factor Products
Determining Dose
Effect of Half-life on FVIII Level Following Bolus Infusion
Relationship Between Predicted Time With FVIII:C < 1% and Joint Bleeds
Determining Thrombin Generation
Thrombin Generation Assay vs Chromogenic Method: FVIII Values ≤ 0.05 IU/mL
Correlation Between F8 Genotype and Bleeding Phenotype
Group Level vs Individual Level
Pharmacokinetics May Be the Key
Pharmacokinetic Dosing With Prophylactic Treatment
Concentrate Consumption in Dose Category
Using Extended Half-life Factor Products
T1/2 Extended: But What Is Most Important -- Dose Interval or Trough?
A Vision of Future Hemophilia Treatment: 2 Scenarios
How Many Samples Are Required?
Pharmacokinetics of Recombinant Factor IX
Factor VIII Pharmacokinetics
Monitoring
Arthropathy Is Individual, But Who Is Who?
Potential Use of Sensitive Biomarkers
Potential Biomarkers for Hemophilic Arthropathy
Genomics in Hemophilia:What Potential Does It Have?
Genomics and Hemophilic Arthropathy
Radiologic Classification of Hemophilic Arthropathy
MRI vs Ultrasound
Ultrasound vs MRI vs X-ray
Use of MSKUS
Impact of MSKUS
Assessment of Prophylaxis in Hemophilia: Summary
Case Revisited
Case Question Discussion
Case Discussion
