Marina Noris, Miriam Galbusera, Sara Gastoldi, Paolo Macor, Federica Banterla, Elena Bresin, Claudio Tripodo, Serena Bettoni, Roberta Donadelli, Elisabetta Valoti, Francesco Tedesco, Alessandro Amore, Rosanna Coppo, Piero Ruggenenti, Eliana Gotti, Giuseppe Remuzzi
Atypical hemolytic-uremic syndrome (aHUS) is associated with genetic complement abnormalities/anti-complement factor H antibodies, which paved the way to treatment with eculizumab. We studied 44 aHUS patients and their relatives to (1) test new assays of complement activation, (2) verify whether such abnormality occurs also in unaffected mutation carriers, and (3) search for a tool for eculizumab titration. An abnormal circulating complement profile (low C3, high C5a, or SC5b-9) was found in 47% to 64% of patients, irrespective of disease phase...
September 11, 2014: Blood