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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