Emerging Clinical Insights Into Paroxysmal Nocturnal

Hemoglobinuria

Activity Goals

Overview

The Pathobiology of PNH

Terminal Complement Activation Renders RBCs Susceptible to

Lysis

Consequences of Chronic Hemolysis

and Free Hemoglobin

Symptoms of PNH

Common Signs and Symptoms May Reflect Risk of Organ

Dysfunction or Early Mortality

Incidence and Natural History of PNH

Identification of Patients at High Risk for Developing PNH

ICCS Guidelines for Diagnosis of PNH

Flow Cytometry to Identify the PNH Clone

Standard Diagnostic Test for PNH

Evolving Technology of Flow Cytometry

Prognosis of Patients With PNH

Risk of Early Mortality From Thrombosis

Clinical Signs and Symptoms

Multivariate Analysis of Risk Factors Affecting Thrombotic

Events

Hemolysis and Symptoms are Risk Factors for Thrombotic

Events

Historical Management of PNH

Treatment of PNH

Bone Marrow Transplant

PNH With Thrombosis After SCT

Eculizumab Blocks Terminal Complement Complex

Formation

TRIUMPH and SHEPHERD Studies

86% Reduction in LDH levels

TRIUMPH Study: Reduction in Transfusion Dependence Across all Subgroups

Relative Risk Reduction (92%) in Thrombotic Events

Reduction in Thrombotic Events

Eculizumab Improves Survival in

Patients With PNH

Improved Overall Survival With Eculizumab

Serious Infection-Related Treatment-Emergent Adverse

Events Eculizumab Therapy (n = 195)

Black Box Warning

Proposed Guideline for the Management of PNH

Summary

Summary (cont)

Abbreviations

Abbreviations (cont)

References

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