How does complement inhibition therapy affect the renal function of patients with paroxysmal nocturnal hemoglobinuria (PNH)?

Updated: May 20, 2021
  • Author: Emmanuel C Besa, MD; Chief Editor: Sara J Grethlein, MD, FACP  more...
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Chronic hemosiderosis and/or microvascular thrombosis from PNH causes kidney dysfunction or damage at an incidence of 65%, defined by stages of chronic kidney disease (CKD), in a large cohort of PNH patients. Eculizumab treatment was safe and well-tolerated in patients with Kidney dysfunction or damage and resulted in the likelihood of improvement as defined as categorical reduction in CKD stage (P < 0.001) compared with baseline and placebo (P = 0.04).

Improvement in kidney function was more commonly seen in those with less severe impairment. Improvements occurred quickly and were sustained for at least 18 months of treatment. Administration of eculizumab to patients with kidney dysfunction or damage was well tolerated and was usually associated with clinical improvement. [56]

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