Atipik Hemolitik Üremik Sendromlu Hastaların Uzun Dönem Sonuçları Long-Term Outcomes of Patients with Atypical Hemolytic Uremic Syndrome

Aim: Atypical hemolytic uremic syndrome (aHUS) is a rare, heterogeneous life-threatening disease of alternative complement pathways. Hemolytic uremic syndrome (HUS) is the clinical triad of thrombocytopenia, anemia, and acute renal injury. It is aimed to evaluate long-term follow-up of patients with aHUS in the present study. Materials and methods: Eleven children diagnosed with aHUS were retrospectively evaluated. Demographic, clinical, and laboratory data and treatment details were reported. Results: A total of 11 patients were enrolled in this study. The mean age of patients at aHUS onset was 2.9±6 years. The mean follow-up time was 72 ± 4 months All patients had renal involvement. Extrarenal manifestations of aHUS were present in four patients. All patients had eculizumab treatment. Conclusion: Our study insight into the diagnosis and management of aHUS in our pediatric patients. More genetic testing is needed to improve the diagnosis of aHUS. We demonstrated the long-term efficacy and safety of eculizumab in our aHUS patients. Further studies are needed to determine the optimal time for discontinuation of eculizumab treatment.Aim: Atypical hemolytic uremic syndrome (aHUS) is a rare, heterogeneous life-threatening disease of alternative complement pathways. Hemolytic uremic syndrome (HUS) is the clinical triad of thrombocytopenia, anemia, and acute renal injury. It is aimed to evaluate long-term follow-up of patients with aHUS in the present study. Materials and methods: Eleven children diagnosed with aHUS were retrospectively evaluated. Demographic, clinical, and laboratory data and treatment details were reported. Results: A total of 11 patients were enrolled in this study. The mean age of patients at aHUS onset was 2.9±6 years. The mean follow-up time was 72 ± 4 months All patients had renal involvement. Extrarenal manifestations of aHUS were present in four patients. CFH mutations were detected in four patients. All patients had eculizumab treatment. Conclusion: Our study insight into the diagnosis and management of aHUS, which is a very rare disease, in our pediatric patients. Genetic testing is needed to improve the diagnosis of aHUS. We demonstrated the long-term efficacy and safety of eculizumab in our aHUS patients. Further studies are needed to determine the optimal time for discontinuation of eculizumab treatment.