#5163 RATIONAL USE OF ECULIZUMAB IN SECONDARY ATYPICAL HAEMOLYTIC UREMIC SYNDROME

Abstract

Abstract Background and Aims Secondary atypical hemolytic uremic syndrome (secondary aHUS) is a heterogeneous group of thrombotic microangiopathies (TMA) associated with various underlying conditions. Unlike primary aHUS, there is still no hard evidence on the efficacy of complement blockade in treating secondary aHUS since the two main series in the literature that investigate this subject show discrepant results. Our work aims to reassess Eculizumab's efficacy in treating secondary aHUS. Method Observational, retrospective, single-center study, in which we analyzed the hematological and renal evolution of 24 patients diagnosed with secondary aHUS who received treatment with Eculizumab compared with a control cohort of 14 patients who did not receive Eculizumab. Complete renal response was defined as the recovery of renal function before the event, partial renal response as a recovery of 50% of lost glomerular filtration rate, and hematological response as normalization of hemoglobin and platelets. Results We found no statistically significant differences in baseline characteristics or disease severity between both groups. After a median of 5 doses of Eculizumab, the group of patients who received complement blockade presented a significant difference in renal response (complete in 47.8% of patients and partial in 17.4%) compared to the control cohort (complete response 14.3% and partial of 14.3%). Rates of hematological remission were similar in both groups (87% in the eculizumab cohort and 85.7% in the control cohort). Conclusion Rational and early use of Eculizumab in patients with secondary aHUS could be an effective and safe therapeutic option, guaranteeing better renal recovery compared to patients who do not receive complement blockade.