Table_1_Rational use of eculizumab in secondary atypical hemolytic uremic syndrome.docx

BackgroundSecondary 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 secondary aHUS, since the two main series that investigated this subject showed discrepant results. Our work aims to reassess the efficacy of eculizumab in treating secondary aHUS.MethodsObservational, retrospective, single-center study, in which we analyzed the hematological and renal evolution of 23 patients diagnosed with secondary aHUS who received treatment with eculizumab and compared them with a control cohort of 14 patients. 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.ResultsWe 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 52.3% of patients and partial in 23.8%) compared to the control cohort (complete response 14.3% and partial of 14.3%). Rates of hematological remission were similar in both groups (90.9% in the eculizumab cohort and 85.7% in the control cohort).ConclusionEarly and short-term use of eculizumab in patients with secondary aHUS could be an effective and safe therapeutic option, assuring better renal recovery compared to patients who do not receive complement blockade.</p

Differences in the mean annual eGFR slope by gender and presence of haematuria.

<p>Values are expressed as mean±SD.</p

Characteristics of patients according to the presence of haematuria.

<p>Results are expressed as mean ± SD, n(%) or median [IQR]. HP, Haemoproteinuria; P, proteinuria alone; eGFR, estimated glomerular filtration rate; PTH, parathyroid hormone; RAAS inhibitors, renin angiotensin aldosterone system inhibitors.</p><p>Characteristics of patients according to the presence of haematuria.</p

Multivariate analysis by Lineal Regression.

<p>Adjusted R² = 0.2907. PTH: parathyroid hormone.</p><p>Factors associated with the rate of eGFR deterioration.</p

Differences in the mean annual eGFR slope in patients according to proteinuria levels and presence of haematuria.

<p>Values are expressed as mean±SD.</p

Differences in the mean annual eGFR slope by age and presence of haematuria.

<p>NS, non significant. Values are expressed as mean±SD.</p