Diagnosis, Prognosis, and Management of aHUS
Program Goals
TMA Disorders Are Now Classified by Molecular Mechanism
Hereditary Disorders Resulting in TMA
Acquired Causes of TMA
Complex Mechanisms May Contribute to Acquired TMA
Effects of Uncontrolled or Excessive Complement Activation
Complement-Mediated TMA (aHUS) Can Affect Multiple Organs, Tissues
Extrarenal Manifestations Are Common in aHUS
Keys to Diagnosing aHUS
No Role for Complement-Level Testing in the Diagnosis of aHUS
Challenges in Diagnosing aHUS
Complement-Amplifying Conditions May Unmask aHUS
Complement-Amplifying Conditions Commonly Unmask aHUS
aHUS and Complement-Amplifying Events
Unmasking the Diagnosis of Complement-Mediated TMA
Management Options for aHUS Have Been Limited
Challenges in Evaluating PI/PE Therapy
Response to Plasma Therapy for aHUS
Plasma Therapy for aHUS Is Clinically Inadequate
Recommendations of French Study Group for Defining PE/PI Failure
Potential for Significant Morbidity/Mortality Regardless of Overt Laboratory or Clinical Signs
Eculizumab: Humanized First-in-Class Anti-C5 Antibody
Eculizimab Dosing Schedule: Adults
Eculizumab Multinational Clinical Program Includes Broad aHUS Patient Population
Eculizumab Multinational, Multicenter Clinical Program of Prospective Trials in aHUS (N = 100)
Improvement in Hematologic Markers of Complement-mediated TMA Across All Studies at 26 weeks
Rapid and Sustained Improvement in Hematologic Markers of Complement-mediated TMA With or Without Identified Genetic Mutation
Early Eculizumab Therapy Associated With High Likelihood of Eliminating Dialysis
Prospective Trial of Eculizumab in Adults With aHUS
Safety Profile of Eculizumab in Prospective Trial of Adults With aHUS
Eculizumab and Serious Meningococcal Infections
Key Benefits of Eculizumab
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