Comparison of the efficacy of once- and twice-daily colchicine dosage in pediatric patients with familial Mediterranean fever - a randomized controlled noninferiority trial

Background: In this study, we examined the efficacy and safety of a once-daily dosage schema of colchicine compared with a twice-daily dosage schema in pediatric patients with familial Mediterranean fever (FMF). Methods: In this 24-week, multicenter, randomized controlled noninferiority trial, pediatric patients newly diagnosed with FMF carrying a homozygous or compound heterozygous mutation and not receiving any treatment were included. Patients were randomly assigned using a block randomization method to receive treatment with a once- or twice-daily dosage. Clinical and laboratory characteristics and medication side effects were recorded and compared between groups. The study was carried out in compliance with Good Clinical Practice and the Consolidated Standards for Reporting of Trials (CONSORT) statement. Results: A total of 92 patients were selected, and 79 patients completed the study. There were 42 patients in the once-daily dosage group and 37 in the twice-daily dosage group. The results indicated that the once-daily dosage was not inferior to the twice-daily dosage regarding decrease in attack frequency and duration as well as improvement in clinical findings and Mor severity scores. Alterations in laboratory findings indicating inflammation, such as erythrocyte sedimentation rate, C-reactive protein, and serum amyloid A, were similar in both groups. The rates of drug side effects were similar between the once- and twice-daily dosage groups, implying comparable safety of colchicine, with the exception of diarrhea, which was slightly higher in the once-daily dosage group. Conclusions: Using colchicine with either a once- or twice-daily dosage provides similar clinical and laboratory improvements. Considering both efficacy and safety, colchicine can be prescribed with a once-daily dosage. Trial Registration ID: ClinicalTrials.gov identifier NCT02602028. Registered 5 November 2015

A remote cause of anuria in a child

Acute renal injury is a rare complication of idiopathic nephrotic syndrome with mesPGN. Here we present a 2-year-old male patient with 4 days history of anuria, generalized edema and hypervolemia. Any evidence other than proteinuria and renal failure could not be identified with laboratory tests and doppler ultrasonography. Anuric presentation was thought to be related with rapidly progressive glomerulonephritis, diffuse mesangial sclerosis or acute tubular necrosis. However, renal biopsy revealed mesangial proliferative glomerulonephritis (mesPGN). Prednisolone 2 mg/kg/day was prescribed. Diuresis was started gradually and on the 10th day of disease, anuria was resolved and acute renal injury recovered without any sequel. This case is presented because of the incompatibility between clinical findings and histopathologic diagnosis. It is concluded that although rare, anuria and acute renal injury could be the presenting symptom of idiopathic nephrotic syndrome in childhood.&nbsp

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

Eculizumab therapy for aHUS associated with heterozygous factor I mutation

WOS: 000307274100179

Heterozygous factor H mutation presented as diarrhea-associated hemolytic uremic syndrome

WOS: 000307274100175