Asli Kantar

Success of eculizumab in the treatment of atypical hemolytic uremic syndrome

BackgroundDisorders of complement regulation are the most important etiology of atypical hemolytic uremic syndrome (aHUS). Recent studies demonstrate that eculizumab is beneficial in long-term aHUS treatment. We present a series of children with aHUS resistant to/dependent on plasma exchange (PE) who were treated with eculizumab.MethodsThis was a retrospective study in which data were retrieved from the medical files of children who had received PE as treatment for aHUS. The data retrieved included age, sex, presenting symptoms, presence of diarrhea/vomiting, hospitalization duration, laboratory data on admission and follow-up, need for transfusion or dialysis, response to PE, response to eculizumab and outcome.ResultsOf the 15 children diagnosed with aHUS in 2011 and 2012 in our departments, ten were resistant to, or dependent on, plasma therapy and treated with eculizumab; these children were enrolled in the study. Three patients had relapses, and seven had a new diagnosis. Nine children had oliguria or anuria, and eight required dialysis. Hypertension was observed in six patients. Neurologic involvement developed in six patients, with the symptoms including seizures, loss of balance, vision loss and severe confusion. Five and five patients were resistant to and dependent on plasma therapy, respectively. Following the start of eculizumab treatment, all patients achieved full recovery of renal function and hematologic parameters.ConclusionsIn our ten pediatric patients with aHUS who did not respond to PE, eculizumab was a lifesaving therapy and improved their quality of life. Early eculizumab use was a rescue therapy for renal function. Our results show that eculizumab is an effective treatment for aHUS. However, more studies are needed on the long-term efficacy and safety of eculizumab in children with aHUS and to determine the optimal duration of treatment.

Neurocognitive Functions in Pediatric Renal Transplant Patients

Neurocognitive dysfunction is one of the major complications of chronic renal failure (CRF). Uremic state during CRF encompasses a wide spectrum of neurobehavioral and neurological disturbances. Recent studies showed that the pathophysiology of neurocognitive dysfunction in CRF is related to plasma levels of uremic solutes. Successful renal transplantation improves renal, metabolic, and endocrine functions and the quality of life. The aim of our study was to determine the state of neurocognitive function in pediatric renal transplant recipients. We prospectively performed a neurological examination and neuropsychological test battery (Bender-Gestalt Test, Cancellation Test, and Visual and Auditory Number Assay Test) in 20 pediatric renal transplant recipients between 6 and 16 years of age. Twenty healthy children and 20 children with CRF were included in the study as the control groups. Mean age of the renal transplant recipients was 13.50 ± 3.40 years old. Mean evaluation time after transplantation was 2.0 ± 0.5 years. Bender-Gestalt Test result was abnormal in 40% of patients. The results of the Cancellation Test and the Visual and Auditory Number Assay Test showed significant decline in pediatric renal transplant patients when compared with the control. We found that neurocognitive dysfunction was frequent in pediatric renal transplantation patients. Awareness of this potential problem may be helpful for early recognition and treatment. Our findings suggest that periodic neurocognitive assessments may be indicated in transplant recipients.

İdrar interlökin-13, CD80, CD28, matriks metaloproteinaz-2 ve granzim B‘nin çocukluk çağı minimal değişim nefrotik sendrom patogenezindeki rolleri

Objective: Minimal change disease (MCD) is the most common cause of nephrotic syndrome in childhood but its pathogenesis remains poorly understood. A T-cell-derived factor or factors were initially alleged as contributing to the disease pathogenesis. However, podocyte CD80 expression is now a commonly discussed theory. The aim of this study was to investigate the pathogenesis of MCD by determining urine interleukin-13, CD80, CD28, matrix metalloproteinase-2 (MMP-2), and granzyme B levels. Methods: Thirty patients and thirty healthy children were evaluated in this study. Six patients had biopsy proven MCD. The remaining patients were considered to have MCD because of their age at time of diagnosis; response to steroid treatment, absence of gross hematuria, hypocomplementemia or renal failure; and normal blood pressures in the active stage. The nephrotic-phase urine interleukin-13, CD80, CD28, MMP-2, and granzyme B levels of all patients were compared with the remission-stage urine levels of the same patients and control subjects. The urine samples were collected immediately before the application of immunosuppressive drugs or other treatment modalities. Results: Significantly higher interleukin-13, CD80, CD28, and MMP-2 levels were observed in the relapse period compared with both the remission period and control subjects. There was a significant positive correlation between the active-stage urine interleukin-13 and CD80 levels (r=0.619, p<0.001). Conclusion: Interleukin-13, CD80, CD28, and MMP-2 seem to have roles in the pathogenesis of MCD and using inhibitors of these molecules in treatment of steroid and immunosuppressive-resistant MCD cases can be thought. J Clin Exp Invest 2014; 5 (3): 354-361

Experience with continuous venovenous hemodiafiltration in four newborns: A case series and review of the literature

When conventional methods for treating complicated problems such as acute and chronic renal failure or metabolic diseases fail, the therapy of choice is peritoneal dialysis (PD) in neonatal period. However, in cases that involve technical difficulties, such as bulky lesions in the abdomen or complications from previous abdominal surgeries, it is not always possible to place a peritoneal catheter. In such situations, continuous venovenous hemodiafiltration (CVVHDF) can be effective. This case series presents our experience in 2013 with the administration of CVVHDF to four patients in our neonatal intensive care unit who could not undergo PD for various reasons.

PO-0716 Continuous Venovenous Hemodiafiltration Experience Of Four Newborns

Aim Conventional methods are the first treatment modalities of renal failure or metabolic diseases in newborns. If these modalities fail to treat, we start to use peritoneal dialysis (PD). Continuous venovenous hemodiafiltration (vvHDF) is used when PD can not be performed. Our continuous vvHDF experience of 4 patients in neonatal intensive care unit, is presented. Case1: A male term newborn infant, having mapple syrup urine disease with a high serum leucine value after PD could not be performed, vvHDF was successfully provided. He was discharged from our hospital on 34th postnatal day. Case2: A preterm newborn, having polycystic renal disease and could not use under PD and vvHDF was started on 13th postnatal day. He died due to ventilator associated pneumonia on 135th postnatal day. Case3: A term newborn, having “polycystic renal disease” and could not perform PD, was referred to our unit for continuous vvHDF administration on 3rd postnatal day. vvHDF application was continued until his 61st postnatal day. Case4: A term newborn was appealed to our emergency service, with dyspnea and supraventricular tachycardia was diagnosed on 13th postnatal day. After intervention, multiorgan failure developed in our patient. At his postnatal day 27, vvHDF was performed. The patient died because of ventilator associated pneumonia. Conclusion Continuous vvHDF application should be considered in the neonatal period, in cases where it is impossible to apply PD. Due to the technical difficulties in the neonatal period, such application is not common but it is also life saving.

PReS-FINAL-2214: Validation of new pediatric criteria in diagnosis of Familial Mediterranean Fever in children

Familial Mediterranean Fever (FMF) is the most common recurrent autoinflammatory fever syndrome. The diagnosis is based on clinical findings and supported by genetic analysis. Recently new set of diagnostic criteria were established for the diagnosis of familial Mediterranean fever (FMF) in childhood by Yalcinkaya et al.

PReS-FINAL-2087: Diagnosis of arthritis: history or laboratory?

Arthritis is a common rheumatologic problem of childhood. Physical examination and laboratory findings are important to clarify the etiology.