Background Practice patterns of eculizumab use are not well described. We examined
indications for, and outcomes of, eculizumab therapy in a tertiary care nephrology
center. Methods We used the "Vienna TMA cohort" and the hospital pharmacy database
at the Medical University of Vienna to identify patients that received eculizumab
treatment between 2012 and 2019. We describe clinical characteristics, details of
eculizumab use, and outcomes of patients with complement gene-variant mediated TMA
(cTMA), secondary TMA (sTMA) and C3 glomerulopathy (C3G). Results As of December 2019,
23 patients received complement blockade at the Division of Nephrology and Dialysis:
15 patients were diagnosed with cTMA, 6 patients with sTMA and 2 patients with C3G.
Causes of sTMA were bone marrow transplantation (n = 2), malignant hypertension, malignant
tumor, systemic lupus erythematosus, antiphospholipid syndrome and lung transplantation
(each n = 1). Across all indications, patients had a median age of 31 and were predominantly
female (78%) and the median duration of treatment was 227 days. Hematological recovery
was seen in most patients, while renal response was best in patients with cTMA. Adverse
events were recorded in 26%. Conclusions In summary, eculizumab is the treatment of
choice for cTMA patients that do not respond to plasma therapy. In patients with sTMA
and C3G, the response rates to therapy are much lower and therefore, the decision
to start therapy needs to be considered carefully. Graphic abstract
