Potential use of stem cells as a therapy for cystinosis
AbstractCystinosis is an autosomal recessive metabolic disease that belongs to the family of lysosomal storage disorders (LSDs). Initial symptoms of cystinosis correspond to the renal Fanconi syndrome. Patients then develop chronic kidney disease and multi-organ failure due to accumulation of cystine in all tissue compartments. LSDs are commonly characterized by a defective activity of lysosomal enzymes. Hematopoietic stem and progenitor cell (HSPC) transplantation is a treatment option for several LSDs based on the premise that their progeny will integrate in the affected tissues and secrete the functional enzyme, which w...
Source: Pediatric Nephrology - April 19, 2019 Category: Urology & Nephrology Source Type: research

HNF1B nephropathy has a slow-progressive phenotype in childhood —with the exception of very early onset cases: results of the German Multicenter HNF1B Childhood Registry
AbstractBackgroundHNF1B gene mutations are an important cause of bilateral (cystic) dysplasia in children, complicated by chronic renal insufficiency. The clinical variability, the absence of genotype-phenotype correlations, and limited long-term data render counseling of affected families difficult.MethodsLongitudinal data of 62 children probands with genetically provenHNF1B nephropathy was obtained in a multicenter approach. Genetic family cascade screening was performed in 30/62 cases.ResultsEighty-seven percent of patients had bilateral dysplasia, 74% visible bilateral, and 16% unilateral renal cysts at the end of obse...
Source: Pediatric Nephrology - April 19, 2019 Category: Urology & Nephrology Source Type: research

Oxidative stress in autosomal dominant polycystic kidney disease: player and/or early predictor for disease progression?
AbstractAutosomal dominant polycystic kidney disease (ADPKD), caused by mutations inPKD1 orPKD2 genes, is the most common hereditary renal disease. Renal manifestations of ADPKD are gradual cyst development and kidney enlargement ultimately leading to end-stage renal disease. ADPKD also causes extrarenal manifestations, including endothelial dysfunction and hypertension. Both of these complications are linked with reduced nitric oxide levels related to excessive oxidative stress (OS). OS, defined as disturbances in the prooxidant/antioxidant balance, is harmful to cells due to the excessive generation of highly reactive ox...
Source: Pediatric Nephrology - April 19, 2019 Category: Urology & Nephrology Source Type: research

Haemodiafiltration use in children: data from the Italian Pediatric Dialysis Registry
ConclusionsOver the observation period, HDF use in Italy has been limited to roughly a quarter of patients on extracorporeal dialysis, in particular to those with high dialysis vintage, younger age or a long expected waiting time to renal transplantation. (Source: Pediatric Nephrology)
Source: Pediatric Nephrology - April 19, 2019 Category: Urology & Nephrology Source Type: research

Feeding modality is a barrier to adequate protein provision in children receiving continuous renal replacement therapy (CRRT)
AbstractBackgroundCritically ill children have a high prevalence of malnutrition. Children with acute kidney injury experience high rates of protein debt. Previous research has indicated that protein provision is positively associated with survival.MethodsThis was a prospective observational study of all patients receiving CRRT for greater than 48  h at our tertiary care institution. Patients with inborn errors of metabolism were excluded. Data collection included energy, protein, and fluid volume intakes, anthropometrics, feeding modality, and route of nutrition intake.ResultsForty-one patients 9  ±&thi...
Source: Pediatric Nephrology - April 19, 2019 Category: Urology & Nephrology Source Type: research

First-year profile of biomarkers for early detection of renal injury in infants with congenital urinary tract obstruction
AbstractBackgroundDiagnosis of renal function impairment and deterioration in congenital urinary tract obstruction (UTO) continues to be extremely challenging. Use of renal biomarkers in this setting may favor early renal injury detection, allowing for a reliable choice of optimal therapeutic options and prevention or minimization of definitive renal damage.MethodsThis longitudinal, prospective study analyzed the first-year profile of two serum renal biomarkers: creatinine (sCr) and cystatin C (sCyC); and six urinary renal biomarkers: neutrophil gelatinase-associated lipocalin (NGAL), kidney injury molecule-1 (KIM-1), tran...
Source: Pediatric Nephrology - April 19, 2019 Category: Urology & Nephrology Source Type: research

Asymptomatic bacteriuria in pediatric kidney transplant recipients: to treat or not to treat? A retrospective study
ConclusionsOur results are not in favor of systematic treatment of AB in pediatric KTR. Notably, limitation of antibiotic treatment is an urgent and important health issue in this population, in order to reduce multi-drug resistant bacteria emergence. (Source: Pediatric Nephrology)
Source: Pediatric Nephrology - April 19, 2019 Category: Urology & Nephrology Source Type: research

The population-based prevalence of hypertension and correlates of blood pressure among Australian children
ConclusionsUsing the AAP guidelines is likely to substantially increase the population prevalence of hypertension. The association between BMI and BP was strongest and non-linear for obese children, who should be the focus of interventional trials. (Source: Pediatric Nephrology)
Source: Pediatric Nephrology - April 19, 2019 Category: Urology & Nephrology Source Type: research

Patent ductus arteriosus is associated with acute kidney injury in the preterm infant
ConclusionsModerate-to-large PDA was strongly associated with all stages of AKI in preterm infants ≤ 28 weeks of gestational age. Effective NSAID treatment decreased the risk of severe but not mild AKI. These differential effects reflect the balance between the renal benefits of PDA closure and the risk of NSAID toxicity. (Source: Pediatric Nephrology)
Source: Pediatric Nephrology - April 19, 2019 Category: Urology & Nephrology Source Type: research

Oxidative stress in chronic kidney disease
AbstractOxidative stress (OS), defined as disturbances in the pro-/antioxidant balance, is harmful to cells due to the excessive generation of highly reactive oxygen (ROS) and nitrogen (RNS) species. When the balance is not disturbed, OS has a role in physiological adaptations and signal transduction. However, an excessive amount of ROS and RNS results in the oxidation of biological molecules such as lipids, proteins, and DNA. Oxidative stress has been reported in kidney disease, due to both antioxidant depletions as well as increased ROS production. The kidney is a highly metabolic organ, rich in oxidation reactions in mi...
Source: Pediatric Nephrology - April 19, 2019 Category: Urology & Nephrology Source Type: research

Factors associated with high-cost hospitalization for peritonitis in children receiving chronic peritoneal dialysis in the United States
AbstractBackgroundAlthough peritonitis causes significant morbidity and mortality in children receiving chronic peritoneal dialysis (CPD), little is known about costs associated with treatment.MethodsWe analyzed 246 peritonitis-related hospitalizations in the USA, linked by the Standardized Care to Improve Outcomes in Pediatric End Stage Renal Disease (SCOPE) and Pediatric Health Information Systems (PHIS) databases. Multivariable logistic regression was used to assess the relationship between high-cost hospitalizations (at or above the 75th percentile) and patient characteristics. Multivariable modeling was used to assess...
Source: Pediatric Nephrology - April 19, 2019 Category: Urology & Nephrology Source Type: research

Validation of standardized creatinine and cystatin C GFR estimating equations in a large multicentre European cohort of children
AbstractBackgroundMost validations of paediatric glomerular filtration rate (GFR) estimating equations using standardized creatinine (CR) and cystatin C (CYS) assays have comprised relatively small cohorts, which makes accuracy across subgroups of GFR, age, body mass index (BMI) and gender uncertain. To overcome this, a large cohort of children referred for GFR determination has been established from several European medical centres.MethodsThree thousand four hundred eight measurements of GFR (mGFR) using plasma clearance of exogenous substances were performed in 2218 children aged 2 –17 years. Validated equatio...
Source: Pediatric Nephrology - April 19, 2019 Category: Urology & Nephrology Source Type: research

Blood-borne viral infections in pediatric hemodialysis
AbstractHemodialysis patients are at increased risk for development of blood-borne viral infections. Human immunodeficiency virus (HIV), a once fatal infection, has become treatable, but continues to be associated with increased mortality. Hepatitis B and C viral infections can lead to acute and chronic hepatitis, cirrhosis, or hepatocellular carcinoma. Young children and immunocompromised patients are more likely to develop chronic disease leading to increased morbidity and mortality, as compared to the healthy population. The hemodialysis population is at increased risk of blood-borne viral infections as compared to the ...
Source: Pediatric Nephrology - April 19, 2019 Category: Urology & Nephrology Source Type: research

Risk of cardiovascular involvement in pediatric patients with X-linked hypophosphatemia
AbstractObjectiveTo find out if cardiovascular alterations are present in pediatric patients with X-linked hypophosphatemia (XLH).Study designMulticentre prospective clinical study on pediatric patients included in the RenalTube database (www.renaltube.com) with genetically confirmed diagnosis of XLH by mutations in thePHEX gene. The study ’s protocol consisted of biochemical work-up, 24-h ambulatory blood pressure monitoring (ABPM), carotid ultrasonography, and echocardiogram. All patients were on chronic treatment with phosphate supplements and 1-hydroxy vitamin D metabolites.ResultsTwenty-four patients (17 females...
Source: Pediatric Nephrology - April 19, 2019 Category: Urology & Nephrology Source Type: research

Impaired renal function and fever of unknown origin in a patient with pediatric granulomatous arthritis: Questions
(Source: Pediatric Nephrology)
Source: Pediatric Nephrology - April 19, 2019 Category: Urology & Nephrology Source Type: research

Renal mass in a 2-year-old girl: Answers
(Source: Pediatric Nephrology)
Source: Pediatric Nephrology - April 19, 2019 Category: Urology & Nephrology Source Type: research

Impaired renal function and fever of unknown origin in a patient with pediatric granulomatous arthritis: Answers
(Source: Pediatric Nephrology)
Source: Pediatric Nephrology - April 19, 2019 Category: Urology & Nephrology Source Type: research

Spinal cord lesions in a pediatric patient with chronic kidney disease and review of literature: Answers
(Source: Pediatric Nephrology)
Source: Pediatric Nephrology - April 19, 2019 Category: Urology & Nephrology Source Type: research

Renal mass in a 2-year-old girl: Questions
(Source: Pediatric Nephrology)
Source: Pediatric Nephrology - April 19, 2019 Category: Urology & Nephrology Source Type: research

Spinal cord lesions in a pediatric patient with chronic kidney disease and review of literature: Questions
(Source: Pediatric Nephrology)
Source: Pediatric Nephrology - April 19, 2019 Category: Urology & Nephrology Source Type: research

Low-dose rituximab is no less effective for nephrotic syndrome measured by 12-month outcome
ConclusionsUse of a single low-dose regimen of rituximab in the management of frequently relapsing nephrotic syndrome does not affect the probability of relapse at 12  months or time to B cell reconstitution compared to a conventional higher dose. (Source: Pediatric Nephrology)
Source: Pediatric Nephrology - March 19, 2019 Category: Urology & Nephrology Source Type: research

Short stature in advanced pediatric CKD is associated with faster time to reduced kidney function after transplant
AbstractBackgroundAmong children who receive a kidney transplant, short stature is associated with a more complicated post-transplant course and increased mortality. Short stature prior to transplant may reflect the accumulated risk of multiple factors during chronic kidney disease (CKD); however, its relationship with post-transplant kidney function has not been well characterized.MethodsIn the Chronic Kidney Disease in Children (CKiD) cohort restricted to children who received a kidney transplant, short stature (i.e., growth failure) was defined  as age-sex-specific height 
Source: Pediatric Nephrology - March 19, 2019 Category: Urology & Nephrology Source Type: research

Optimal duration of treatment with eculizumab in atypical hemolytic uremic syndrome (aHUS) —a question to be addressed in a scientific way
(Source: Pediatric Nephrology)
Source: Pediatric Nephrology - March 19, 2019 Category: Urology & Nephrology Source Type: research

Neurocognitive and functional outcomes at 5  years of age after renal transplant in early childhood
ConclusionsOur data suggest that renal replacement therapies in young children are associated with acceptable developmental outcome. Programs to identify those with developmental delays and provide early intervention may allow achievement of the child ’s full potential. (Source: Pediatric Nephrology)
Source: Pediatric Nephrology - March 19, 2019 Category: Urology & Nephrology Source Type: research

Clinical and histopathological prognostic factors affecting the renal outcomes in childhood ANCA-associated vasculitis
AbstractObjectiveAntineutrophil cytoplasmic antibody (ANCA) –associated vasculitides (AAV) are very rare in childhood with an increased risk of morbidity and mortality. We aimed to evaluate renal prognostic factors in childhood AAV from the perspective of ANCA serotype, histopathological classification, and five-factor score (FFS).MethodsPediatric AAV patients from 11 referral centers in Turkey had been included to the study. The demographics, clinical findings, AAV subtypes, outcomes, and FFS were evaluated retrospectively. Kidney biopsies were classified histopathologically.ResultsTotally, 39 patients were enrolled...
Source: Pediatric Nephrology - March 19, 2019 Category: Urology & Nephrology Source Type: research

The aminoglycoside geneticin permits translational readthrough of the CTNS W138X nonsense mutation in fibroblasts from patients with nephropathic cystinosis
ConclusionsReduction in intracellular cystine indicates that the CTNS protein produced is functional as a cystine transporter. Interestingly, similar effects were seen even in W138X compound heterozygotes. These studies establish proof-of-principle for the potential of aminoglycosides to treat cystinosis and possibly other monogenic diseases caused by nonsense mutations. (Source: Pediatric Nephrology)
Source: Pediatric Nephrology - March 19, 2019 Category: Urology & Nephrology Source Type: research

IgA nephropathy: is a new approach beyond proteinuria necessary?
(Source: Pediatric Nephrology)
Source: Pediatric Nephrology - March 19, 2019 Category: Urology & Nephrology Source Type: research

Predictors of grade 3 –5 vesicoureteral reflux in infants ≤ 2 months of age with pyelonephritis
This study aimed to assess predictors for grade 3 –5 vesicoureteral reflux (VUR) in infants ≤ 2 months of age admitted for first urinary tract infection (UTI).MethodsRetrospective cohort study of 195 infants ≤ 2 months admitted to a pediatric ward for first UTI between 2006 and 2017. Clinical, laboratory, and imaging data were collected from electronic medical charts. We examined associations between grade 3–5 VUR and different patient characteristics.ResultsTwenty infants (10%) were diagnosed with grade 3 –5 VUR; all had fever. Infants with grade 3–5 VUR had higher blo...
Source: Pediatric Nephrology - March 19, 2019 Category: Urology & Nephrology Source Type: research

Chronic kidney disease following twin-to-twin transfusion syndrome —long-term outcomes
ConclusionThis is the first long-term follow-up study demonstrating that CKD progressing to the need for RRT can develop after TTTS. Donor-twin status and neonatal AKI associated with adverse long-term outcomes warranting long-term surveillance in this group. (Source: Pediatric Nephrology)
Source: Pediatric Nephrology - March 19, 2019 Category: Urology & Nephrology Source Type: research

Pediatric intradialytic hypotension: recommendations from the Pediatric Continuous Renal Replacement Therapy (PCRRT) Workgroup
AbstractIntradialytic hypotension (IDH) is a common adverse event resulting in premature interruption of hemodialysis, and consequently, inadequate fluid and solute removal. IDH occurs in response to the reduction in blood volume during ultrafiltration and subsequent poor compensatory mechanisms due to abnormal cardiac function or autonomic or baroreceptor failure. Pediatric patients are inherently at risk for IDH due to the added difficulty of determining and attaining an accurate dry weight. While frequent blood pressure monitoring, dialysate sodium profiling, ultrafiltration-guided blood volume monitoring, dialysate coo...
Source: Pediatric Nephrology - March 19, 2019 Category: Urology & Nephrology Source Type: research

Lisinopril versus lisinopril and losartan for mild childhood IgA nephropathy: a randomized controlled trial (JSKDC01 study)
ConclusionsWe propose lisinopril monotherapy as treatment for childhood proteinuric mild IgA nephropathy as there are no advantages of combination therapy.Clinical trial registrationClinical trial registry, UMIN ID C000000006,https://www.umin.ac.jp. (Source: Pediatric Nephrology)
Source: Pediatric Nephrology - March 19, 2019 Category: Urology & Nephrology Source Type: research

Haptoglobin degradation product as a novel serum biomarker for hematopoietic stem cell transplant-associated thrombotic microangiopathy
AbstractBackgroundHematopoietic stem cell transplant (HSCT)-associated thrombotic microangiopathy (TA-TMA) is a well-known complication of HSCT and carries high risk of morbidity and mortality. A lack of consistent non-invasive diagnostic criteria can delay diagnosis and lead to irreversible organ damage.MethodsSerum samples of 100 patients that underwent HSCT at Cincinnati Children ’s Hospital were serially collected. Unbiased proteomic profiling by SELDI-TOF-MS was performed on serum from TA-TMA patients at baseline (pre-HSCT), 2 weeks before TMA diagnosis (pre-TMA), and at clinical TMA diagnosis. Two proteins...
Source: Pediatric Nephrology - March 19, 2019 Category: Urology & Nephrology Source Type: research

A breakthrough in readthrough? Could geneticin lead the way to effective treatment for cystinosis nonsense mutations?
(Source: Pediatric Nephrology)
Source: Pediatric Nephrology - March 19, 2019 Category: Urology & Nephrology Source Type: research

An unusual cause of nephrotic syndrome: Answers
(Source: Pediatric Nephrology)
Source: Pediatric Nephrology - March 19, 2019 Category: Urology & Nephrology Source Type: research

Dangerous hyperkalemia in a newborn: Answers
(Source: Pediatric Nephrology)
Source: Pediatric Nephrology - March 19, 2019 Category: Urology & Nephrology Source Type: research

Persistent fever in a pediatric renal transplant patient: Answers
(Source: Pediatric Nephrology)
Source: Pediatric Nephrology - March 19, 2019 Category: Urology & Nephrology Source Type: research

An unusual cause of nephrotic syndrome: Questions
(Source: Pediatric Nephrology)
Source: Pediatric Nephrology - March 19, 2019 Category: Urology & Nephrology Source Type: research

Dangerous hyperkalemia in a newborn: Questions
(Source: Pediatric Nephrology)
Source: Pediatric Nephrology - March 19, 2019 Category: Urology & Nephrology Source Type: research

Persistent fever in a pediatric renal transplant patient: Questions
(Source: Pediatric Nephrology)
Source: Pediatric Nephrology - March 19, 2019 Category: Urology & Nephrology Source Type: research

Endovascular intervention in the maintenance and rescue of paediatric arteriovenous fistulae for hemodialysis
ConclusionsRepeated interventions may be necessary to maintain AVF patency and avoid central venous catheters. This is the largest series reported to date. (Source: Pediatric Nephrology)
Source: Pediatric Nephrology - February 25, 2019 Category: Urology & Nephrology Source Type: research

Burden, access, and disparities in kidney disease
AbstractKidney disease is a global public health problem, affecting over 750 million persons worldwide. The burden of kidney disease varies substantially across the world, as does its detection and treatment. In many settings, rates of kidney disease and the provision of its care are defined by socio-economic, cultural, and political factors leading to significant disparities. World Kidney Day 2019 offers an opportunity to raise awareness of kidney disease and highlight disparities in its burden and current state of global capacity for prevention and management. Here, we highlight that many countries still lack access to b...
Source: Pediatric Nephrology - February 25, 2019 Category: Urology & Nephrology Source Type: research

Effects of long-term cysteamine treatment in patients with cystinosis
AbstractCystinosis is a rare autosomal-recessive lysosomal storage disease with high morbidity and mortality. It is caused by mutations in theCTNS gene that encodes the cystine transporter, cystinosin, which leads to lysosomal cystine accumulation. Patients with infantile nephropathic cystinosis, the most common and most severe clinical form of cystinosis, commonly present with renal Fanconi syndrome by 6 –12 months of age, and without specific treatment, almost all will develop end-stage renal disease (ESRD) by 10–12 years of age. Early corneal cystine crystal deposition is a hallmark of the disease....
Source: Pediatric Nephrology - February 25, 2019 Category: Urology & Nephrology Source Type: research

Management of chronic renal allograft dysfunction and when to re-transplant
AbstractDespite the advances in renal transplantation over the last decades, chronic allograft dysfunction remains the largest concern for patients, their families, clinicians and other members of the multi-disciplinary team. Although we have made progress in improving patient and renal allograft survival within the first year after transplantation, the rate of transplant failure with requirement for commencement of dialysis or re-transplantation has essentially remained unchanged. It is important that paediatric and adult nephrologists and transplant surgeons, not only manage their patients and their renal transplants but...
Source: Pediatric Nephrology - February 25, 2019 Category: Urology & Nephrology Source Type: research

Central systolic blood pressure and central pulse pressure predict left ventricular hypertrophy in hypertensive children
AbstractBackgroundCentral systolic and pulse pressures are stronger predictors of cardiovascular risk and hypertensive organ damage than brachial blood pressure. It is suggested that isolated systolic hypertension typically seen in adolescents is associated with normal central blood pressure and does not lead to organ damage and this phenomenon is called spurious hypertension.MethodsWe assessed the prevalence of spurious hypertension and analyzed utility of pulse wave analysis as determinant of hypertensive organ damage in 294 children (62 girls; 15.0  ± 2.4 years) diagnosed as primary hypertensio...
Source: Pediatric Nephrology - February 25, 2019 Category: Urology & Nephrology Source Type: research

Hemodialysis vascular access and subsequent transplantation: a report from the ESPN/ERA-EDTA Registry
ConclusionsCVC remains the most frequent type of vascular access in European children commencing HD. Our results suggest that the choice for CVC is influenced by the time of referral, rapid onset of end-stage renal disease, young age, and an expected short time to transplantation. The role of vascular access type on the pattern between living and deceased donation in subsequent transplantation requires further study. (Source: Pediatric Nephrology)
Source: Pediatric Nephrology - February 25, 2019 Category: Urology & Nephrology Source Type: research

Measurement of iron status in chronic kidney disease
AbstractAnemia is a common complication of chronic kidney disease (CKD) in children, and dysregulation of iron homeostasis plays a central role in its pathogenesis. Optimizing iron status is a prerequisite for effective treatment of anemia. Insufficient iron can lead to inappropriate escalation of the erythropoiesis-stimulating agent (ESA) dose, which is associated with adverse outcomes. Excess iron supplementation also has negative sequelae including free radical tissue damage and increased risk of systemic infection. Notwithstanding the importance of optimizing bioavailable iron for erythropoiesis for children with advan...
Source: Pediatric Nephrology - February 25, 2019 Category: Urology & Nephrology Source Type: research

Poor adherence to early childhood blood pressure measurement guidelines in a large pediatric healthcare system
AbstractBackgroundChildren who were born prematurely, those with a very low birthweight, or who have survived the neonatal intensive care unit (NICU) are at risk for the development of hypertension and chronic kidney disease (CKD), and thus require blood pressure screening less than 3  years of age, per American Academy of Pediatrics (AAP) 2004 and 2017 guidelines.MethodsWe reviewed the practice patterns of a large pediatric health care system and assessed adherence to the AAP clinical practice guidelines on blood pressure measurements in children less than 3  years of age for hypertension and CKD with the follow...
Source: Pediatric Nephrology - February 25, 2019 Category: Urology & Nephrology Source Type: research

Childhood onset steroid-sensitive nephrotic syndrome continues into adulthood
ConclusionIn general, one third of all patients with SSNS during childhood continue to have active disease during early adulthood, in particular patients with SD/FR continue to suffer from active disease. The present data illustrates that SSNS is not just a disease of childhood but persists in adulthood in a significant number of patients. (Source: Pediatric Nephrology)
Source: Pediatric Nephrology - February 25, 2019 Category: Urology & Nephrology Source Type: research

Correction to: An open-label, single-dose study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of cinacalcet in pediatric subjects aged 28  days to & lt;  6 years with chronic kidney disease receiving dialysis
The original version of this article unfortunately contained three mistakes. In Table 1, the last line under “Key Inclusion Criteria” should read “Normal or clinically acceptable ECGs at screening and at day − 1.” In addition, the abbreviation “IP” in the legend to Table 1 stands for “investigational product.” (Source: Pediatric Nephrology)
Source: Pediatric Nephrology - February 25, 2019 Category: Urology & Nephrology Source Type: research

An 8-year-old with genu valgum: Answers
(Source: Pediatric Nephrology)
Source: Pediatric Nephrology - February 25, 2019 Category: Urology & Nephrology Source Type: research