Prolonging the Effects of Factor IX Replacement Therapy in Hemophilia B.

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Presentation transcript:

Prolonging the Effects of Factor IX Replacement Therapy in Hemophilia B

Introduction/Overview

Introduction/Overview (cont)

Importance of Prophylaxis, and Limitations on Its Application, in Hemophilia B

Key Unmet Need in Hemophilia B: Reducing Patient Burden of Disease

Improving Adherence in Hemophilia B

Potential Barriers to Adherence in Hemophilia Prophylaxis

Improving Adherence by Addressing the Barriers to Prophylaxis

Surgical Experience With EHL rFIX Replacement Therapy

Annualized Bleeding Rates: Results From the B-LONG Study

Inhibitor Development in EHL Molecules for Treatment of Hemophilia B

Annualized Bleeding Rates: Results From the Nonacog Beta Pegol Phase 3 Trial

The Importance of Maintaining Trough Levels to Prevent Breakthrough Bleeding

EHL Agents and Treatment Individualization

rFIX-FP Phase 3 Study in Hemophilia B: Study Design

rFIX-FP Phase 3 Study: AsBRs of Prophylaxis Patients

rFIX-FP Phase 3 Study: Safety Results

EHL Agents and Treatment Individualization

Phenotypic Variability Among PWH

Pharmacokinetic Characteristics of 3 EHL Agents

Evolution of FVIII/FIX Concentrates

Case Studies With Long-Acting FIX Therapies

Introduction/Overview

Case 1: 30-Year-Old Man Typically Treated on Demand

Case 2: Older Man With a Sedentary Lifestyle

Case 3: Patient Requiring Surgery

Case 4: Patient With a Frequent Bleeding Phenotype

Summary and Conclusions

Hemophilia B Treatment Goals

The Benefits of EHL Therapies in Hemophilia B

Transforming the Treatment Paradigm

Abbreviations

Abbreviations (cont)

Abbreviations (cont)