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Diagnosis, Prognosis, and Management of aHUS

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Presentation on theme: "Diagnosis, Prognosis, and Management of aHUS"— Presentation transcript:

1 Diagnosis, Prognosis, and Management of aHUS

2 Program Goals

3 TMA Disorders Are Now Classified by Molecular Mechanism

4 Hereditary Disorders Resulting in TMA

5 Acquired Causes of TMA

6 Complex Mechanisms May Contribute to Acquired TMA

7 Effects of Uncontrolled or Excessive Complement Activation

8 Complement-Mediated TMA (aHUS) Can Affect Multiple Organs, Tissues

9 Extrarenal Manifestations Are Common in aHUS

10 Keys to Diagnosing aHUS

11 No Role for Complement-Level Testing in the Diagnosis of aHUS

12 Challenges in Diagnosing aHUS

13 Complement-Amplifying Conditions May Unmask aHUS

14 Complement-Amplifying Conditions Commonly Unmask aHUS

15 aHUS and Complement-Amplifying Events

16 Unmasking the Diagnosis of Complement-Mediated TMA

17 Management Options for aHUS Have Been Limited

18 Challenges in Evaluating PI/PE Therapy

19 Response to Plasma Therapy for aHUS

20 Plasma Therapy for aHUS Is Clinically Inadequate

21 Recommendations of French Study Group for Defining PE/PI Failure

22 Potential for Significant Morbidity/Mortality Regardless of Overt Laboratory or Clinical Signs

23 Eculizumab: Humanized First-in-Class Anti-C5 Antibody

24 Eculizimab Dosing Schedule: Adults

25 Eculizumab Multinational Clinical Program Includes Broad aHUS Patient Population

26 Eculizumab Multinational, Multicenter Clinical Program of Prospective Trials in aHUS (N = 100)

27 Improvement in Hematologic Markers of Complement-mediated TMA Across All Studies at 26 weeks

28 Rapid and Sustained Improvement in Hematologic Markers of Complement-mediated TMA With or Without Identified Genetic Mutation

29 Early Eculizumab Therapy Associated With High Likelihood of Eliminating Dialysis

30 Prospective Trial of Eculizumab in Adults With aHUS

31 Safety Profile of Eculizumab in Prospective Trial of Adults With aHUS

32 Eculizumab and Serious Meningococcal Infections

33 Key Benefits of Eculizumab

34 Abbreviations

35 Abbreviations (cont)

36 Abbreviations (cont)

37 References

38 References (cont)

39 References (cont)

40 References (cont)

41 References (cont)

42 References (cont)

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45 References (cont)


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