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2015 ; 2015
(ä): 673195
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Improvement of Renal Function by Long-Term Sustained Eculizumab Treatment in a
Patient with Paroxysmal Nocturnal Hemoglobinuria
#MMPMID26124968
Ninomiya H
; Obara N
; Niiori-Onishi A
; Yokoyama Y
; Sakata-Yanagimoto M
; Hasegawa Y
; Chiba S
Case Rep Hematol
2015[]; 2015
(ä): 673195
PMID26124968
show ga
Chronic kidney disease (CKD) is one of the major manifestations of paroxysmal
nocturnal hemoglobinuria (PNH). CKD in PNH is induced mainly by intravascular
hemolysis of PNH-affected red blood cells (RBC) missing the
glycosylphosphatidylinositol-anchored proteins with complement-regulatory
activities, CD55 and CD59. CKD develops by heme absorption in the proximal
tubules resulting in the interstitial deposition of iron in the kidneys. We
administered eculizumab to a patient with PNH, who was one of 29 patients
enrolled in the AEGIS clinical trial, an open-label study of eculizumab in Japan.
The patient was complicated by stage 3 CKD with impaired estimated glomerular
filtration rate (eGFR), at grade G3b, and had obvious proteinuria (2-3+,
1-2?g/day). In a two-year extension to the 12-week AEGIS study, eGFR improved
significantly, and the eGFR has since been maintained at grade G2 without
proteinuria by sustained eculizumab treatment (>6 years). Renal function improved
and maintained by long-term sustained eculizumab treatment, presumably by
clearance of iron from the kidney as well as inhibition of the production of
anaphylatoxin C5a, even in advanced stages of CKD, is one of the benefits of
eculizumab treatment in PNH.