Renal outcome with eculizumab in two diarrhea-associated hemolyticeuremic syndrome cases with severe neurologic involvement

İstanbul Bilim Üniversitesi, Tıp Fakültesi.The kidney and brain are the two target organs in patients with Shiga toxin-producing Escherichia coli-associated hemolytic–uremic syndrome (STEC-HUS). Activation of the alternative complement pathway occurs in patients with STEC-HUS. A monoclonal antibody (eculizumab) directed against C5 has been reported to be effective against severe neurologic involvement in patients with STEC-HUS. We report on two STEC-HUS children with severe neurologic involvement treated with eculizumab. Despite prompt resolution of initial neurologic findings upon treatment with eculizumab, proteinuria and hypertension persisted in these patients. The persistence of these two risk factors is particularly emphasized to discuss the long-term effects of eculizumab, especially on renal involvement

Evaluation of the Claria sharesource system from the perspectives of patient/caregiver, physician, and nurse in children undergoing automated peritoneal dialysis

Background Automated peritoneal dialysis (APD) is increasingly preferred worldwide. By using a software application (Homechoice with Claria sharesource system (CSS)) with a mod-M added to the APD device, details of the home dialysis treatment become visible for PD nurses and physicians, allowing for close supervision. We aimed to evaluate the perceptions of patients/caregivers, PD nurses, and physicians about the advantages and disadvantages of CSS

Complement gene mutations in children with C3 glomerulopathy: do they affect the response to mycophenolate mofetil?

Background: C3 glomerulopathy (C3G) is a complement-mediated disease. Although genetic studies are not required for diagnosis, they are valuable for treatment planning and prognosis prediction. The aim of this study is to investigate the clinical phenotypes, kidney survival, and response to mycophenolate mofetil (MMF) treatment in pediatric C3G patients with and without mutations in complement-related genes. Methods: Sixty pediatric C3G patients were included, divided into two groups based on complement-related gene mutations. Demographic and clinical-pathological findings, treatment modalities, and outcome data were compared, and Kaplan–Meier analysis was performed for kidney survival. Results: Out of the 60 patients, 17 had mutations. The most common mutation was in the CFH gene (47%). The mean age at diagnosis was higher in the group with mutation (12.9 ± 3.6 vs. 11.2 ± 4.1 years, p = 0.039). While the patients without mutation most frequently presented with nephritic syndrome (44.2%), the mutation group was most likely to have asymptomatic urinary abnormalities (47.1%, p = 0.043). Serum parameters and histopathological characteristics were similar, but hypoalbuminemia was more common in patients without mutation. During 45-month follow-up,10 patients progressed to chronic kidney disease stage 5 (CKD5), with 4 having genetic mutation. The time to develop CKD5 was longer in the mutation group but not significant. MMF treatment had no effect on progression in either group. Conclusions: This study is the largest pediatric C3G study examining the relationship between genotype and phenotype. We showed that the mutation group often presented with asymptomatic urinary abnormalities, was diagnosed relatively late but was not different from the without mutation group in terms of MMF treatment response and kidney survival. Graphical abstract: [Figure not available: see fulltext.