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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