Outcome of atypical hemolytic uremic syndrome: role of triggers and complement abnormalities in the response to C5 inhibition

Background Atypical-hemolytic uremic syndrome (aHUS) is a rare thrombotic microangiopathy often due to uncontrolled complement activation, characterized by high risk of end-stage kidney disease (ESKD). Eculizumab has improved the outcome, however, its efficacy varies among patients and its discontin...

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Published in:Journal of nephrology 2024-05, Vol.37 (4), p.1017-1026
Main Authors: Ardissino, Gianluigi, Cresseri, Donata, Mancuso, Maria Cristina, Capone, Valentina, Porcaro, Luigi, Amico, Valeria, Tangredi, Marianna, Grovetti, Elena, Griffini, Samantha, Castellano, Giuseppe, Montini, Giovanni, Consonni, Dario, Cugno, Massimo
Format: Article
Language:English
Subjects:
Medicine
Medicine & Public Health
Nephrology
Original Article
Urology
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Summary:Background Atypical-hemolytic uremic syndrome (aHUS) is a rare thrombotic microangiopathy often due to uncontrolled complement activation, characterized by high risk of end-stage kidney disease (ESKD). Eculizumab has improved the outcome, however, its efficacy varies among patients and its discontinuation is debated. Methods To identify characteristics associated with treatment response, we analyzed 244 aHUS patients referred to our center. Patients were classified according to the presence/absence of complement abnormalities and/or triggers at onset in 4 categories: (1) primary (complement abnormality without trigger), (2) secondary (trigger without complement abnormality), (3) combined (trigger and complement abnormality), (4) idiopathic (no trigger, no complement abnormality). Response to treatment was evaluated by comparing the response to eculizumab with that of conventional therapy. Short- and long-term outcomes were evaluated with the relapse rate after discontinuation of C5-inhibition. Results Patients had a better outcome with eculizumab compared to conventional treatment, with a response rate of 81.9% vs 56.9%, p  
ISSN:1724-6059
1724-6059
DOI:10.1007/s40620-023-01873-9