Experience with Mexican Pediatric Patients with Atypical Hemolytic Uremic Syndrome Treated with Eculizumab

Background: Currently there is no Mexican data on clinical characteristics and outcomes in pediatrics patients with atypical hemolytic uremic syndrome (aHUS) with genetic study. Eculizamb is a complement C5 inhibitor for the treatment of aHUS, a rare disease characterized by hemolytic anemia, thrombocytopenia and organ damage. Methods: This was an observational, non interventional, institution registry of patients diagnosed with aHUS, we describe the genetic variant, eGFR previous eculizumab and after (1,3,6,12 months) to the present and complete TMA response. Completed remission is hematologic normalization and renal improvement defined has increased of 25% of baseline. Results: Four pediatric patients, no one with family history of kidney disease, with a complete TMA at 6 months and present time of administration of eculizumab, renal improvement was a 100% in the first month in each subject. With a report of relapse on one patient associated to urinary tract infection Conclusion: Treatment with Eculizumab resulted in sustained maintenance of kidney function, no requirement of renal replacement therapy and outstanding hematological outcomes.Graph with glomerular filtration rate prior to the administration of eculizumb until the present time, showing a significant increase after the administration of a C5 inhibitor.Clinical characteristics and outcomes in pediatrics patients with atypical hemolytic uremic syndrome (aHUS) with genetic variant after treatment with eculizumab