emerging hemofilia

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