Indications

• Atypical Hemolytic Uremic Syndrome (see Hemolytic-Uremic Syndrome, [[Hemolytic-Uremic Syndrome]])
• Paroxysmal Nocturnal Hemoglobinuria (PNH) (see Paroxysmal Nocturnal Hemoglobinuria, [[Paroxysmal Nocturnal Hemoglobinuria]])
  • Licensed to treat PNH in 2007
  • Demonstrated to decrease need for PRBC transfusions in only 50% of cases (presumably due to extravascular hemolysis of cells opsonized by C3 fragments)
  • Extremely expensive (approximate $400k per year)
  • Requires lifelong therapy q14 days

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Pharmacology

• Human Monoclonal Antibody Against Complement C5
  • Inhibits the cleavage of C5 to C5a and C5b by C5 convertase -> inhibits the generation of the terminal complement complex C5b-9

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Administration

• IV Infusion
  • 600 mg qweek for the first 4 wks
  • 900 mg for the fifth dose 1 wk later
  • 900 mg q2 wks thereafter

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Adverse Effects

Infectious Adverse Effects

• Infection with Encapsulated Bacteria (see Bacteria and Fungi, [[Bacteria + Fungi]])
  • Neisseria Meningitidis (see Neisseria Meningitidis, [[Neisseria Meningitidis]])
• Nasopharyngitis
• Upper Respiratory Infection

Gastroenterologic Adverse Effects

• Diarrhea (see Diarrhea, [[Diarrhea]])
• Nausea/Vomiting (see Nausea and Vomiting, [[Nausea and Vomiting]])

Other Adverse Effects

• Back Pain
• Cough (see Cough, [[Cough]])
• Fatigue
• Headache (see Headache, [[Headache]])
• Hypertension (see Hypertension, [[Hypertension]])

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References

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