| Abstract | BACKGROUND AND OBJECTIVES: Atypical hemolytic uremic syndrome (aHUS) is associated with a congenital or acquired dysregulation of the complement alternative pathway that leads to continuous complement activation on host cells causing inflammation and damage. Eculizumab, a humanized mAb against complement protein C5, inhibits activation of the terminal complement pathway. DESIGN, SETTING, PARTICIPANTS, & MEASUREMENTS: We report an adolescent with relapsing unclassified aHUS. On admission, a high plasma creatinine level indicated a poor prognosis, and hemodialysis had to be started. Plasma exchanges were initially effective against the microangiopathic hemolytic activity and allowed a temporary improvement of renal function with termination of hemodialysis after 7 wk. Subsequently, plasma exchanges (three times per week) failed to prevent ongoing aHUS activity and progressive renal failure. After 12 wk, aHUS treatment was switched to eculizumab. RESULTS: Eculizumab was effective in terminating the microangiopathic hemolytic process in two aHUS relapses; however, after normalization of complement activity, aHUS recurred and ultimately led to anuric end-stage renal failure. CONCLUSIONS: In this patient, complement inhibition by eculizumab temporarily terminated the microangiopathic hemolytic activity. Nevertheless, renal damage as a result of preceding and subsequent aHUS activity resulted in end-stage renal failure; therefore, therapeutic success may depend on early administration of eculizumab. The optimal duration of treatment may be variable and remains to be determined. |
| Authors | Christoph J Mache, Birgit Acham-Roschitz, Veronique Frémeaux-Bacchi, Michael Kirschfink, Peter F Zipfel, Siegfried Roedl, Udo Vester, Ekkehard Ring
(Affiliation: Department of Pediatrics, Medical University Graz, Auenbruggerplatz 30, Graz A-8036, Austria. christoph.mache at medunigraz.at)
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| Journal | Clinical journal of the American Society of Nephrology : CJASN
(Clin J Am Soc Nephrol)
Vol. 4
Issue 8
Pg. 1312-6
(Aug 2009)
ISSN: 1555-905X [Electronic] United States |
| PMID | 19556379
(Publication Type: Case Reports, Journal Article)
|
| Chemical References |
- Antibodies, Monoclonal
- Immunologic Factors
- eculizumab
|
| Topics |
- Adolescent
- Antibodies, Monoclonal
(administration & dosage)
- Complement Activation
(drug effects)
- Hemolytic-Uremic Syndrome
(complications, drug therapy, immunology)
- Humans
- Immunologic Factors
(administration & dosage)
- Kidney Failure, Chronic
(immunology, therapy)
- Male
- Plasma Exchange
- Recurrence
- Renal Dialysis
- Treatment Outcome
|
