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Emerging Clinical Insights Into Paroxysmal Nocturnal Hemoglobinuria

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.

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Emerging Clinical Insights Into Paroxysmal Nocturnal Hemoglobinuria

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  1. Emerging Clinical Insights Into Paroxysmal Nocturnal Hemoglobinuria

  2. Activity Goals

  3. Overview

  4. The Pathobiology of PNH

  5. Terminal Complement Activation Renders RBCs Susceptible to Lysis

  6. Consequences of Chronic Hemolysis and Free Hemoglobin

  7. Symptoms of PNH

  8. Common Signs and Symptoms May Reflect Risk of Organ Dysfunction or Early Mortality

  9. Incidence and Natural History of PNH

  10. Identification of Patients at High Risk for Developing PNH

  11. ICCS Guidelines for Diagnosis of PNH

  12. Flow Cytometry to Identify the PNH Clone

  13. Standard Diagnostic Test for PNH

  14. Evolving Technology of Flow Cytometry

  15. Prognosis of Patients With PNH

  16. Risk of Early Mortality From Thrombosis

  17. Clinical Signs and Symptoms

  18. Multivariate Analysis of Risk Factors Affecting Thrombotic Events

  19. Hemolysis and Symptoms are Risk Factors for Thrombotic Events

  20. Historical Management of PNH

  21. Treatment of PNH

  22. Bone Marrow Transplant

  23. PNH With Thrombosis After SCT

  24. Eculizumab Blocks Terminal Complement Complex Formation

  25. TRIUMPH and SHEPHERD Studies86% Reduction in LDH levels

  26. TRIUMPH Study: Reduction in Transfusion Dependence Across all Subgroups

  27. Relative Risk Reduction (92%) in Thrombotic Events

  28. Reduction in Thrombotic Events

  29. Eculizumab Improves Survival in Patients With PNH

  30. Improved Overall Survival With Eculizumab

  31. Serious Infection-Related Treatment-Emergent Adverse Events Eculizumab Therapy (n = 195)

  32. Black Box Warning

  33. Proposed Guideline for the Management of PNH

  34. Summary

  35. Summary (cont)

  36. Abbreviations

  37. Abbreviations (cont)

  38. References

  39. References (cont)

  40. References (cont)

  41. References (cont)

  42. References (cont)

  43. References (cont)

  44. References (cont)

  45. References (cont)

  46. References (cont)

  47. References (cont)

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