Predicting mortality and short-term outcomes of continuous kidney replacement therapies in neonates and infants.

Background: Continuous kidney replacement therapy (CKRT) has emerged as a valuable treatment option in critically ill neonates and infants with acute kidney injury (AKI) requiring dialysis. In this population, we apply Artificial Intelligence (AI) to identify factors influencing mortality and short-term adverse kidney outcomes.

Methods: The study involved neonates and infants included in the EurAKId registry (NCT02960867), who underwent CKRT treatment.

Exome analysis links kidney malformations to developmental disorders and reveals causal genes.

Congenital anomalies of the kidneys and urinary tract (CAKUT) are developmental disorders that commonly cause pediatric chronic kidney disease and mortality. We examine here rare coding variants in 248 CAKUT trios and 1742 singleton CAKUT cases and compare them to 22,258 controls. Diagnostic and candidate diagnostic variants are detected in 14.

SGLT2 inhibitors for kidney protection in children: expanding horizons beyond endocrinology.

For over two decades, kidney protection in children has relied on angiotensin-converting enzyme inhibitors (ACEi) and angiotensin receptor blockers (ARBs), which present significant limitations. Sodium-glucose cotransporter 2 inhibitors (SGLT2i), initially developed as antidiabetic agents, have demonstrated significant benefits in preserving kidney function in adults with chronic kidney disease (CKD), regardless of diabetes status. The pathophysiology of paediatric CKD differs from adult CKD, with congenital anomalies of the kidney and urinary tract (CAKUT) as the predominant cause.

Potential targeting of urokinase-type plasminogen activator receptor-formyl peptide receptor signaling to prevent recurrence in posttransplant primary podocytopathies.

Primary podocytopathies are a group of disorders characterized by nephrotic syndrome and frequent progression to kidney failure, with high rates of posttransplant recurrence. Increased expression of the urokinase-type plasminogen activator receptor (uPAR) has been associated with podocyte dysfunction in primary podocytopathies. uPAR can interact with formyl peptide receptors (FPRs) in podocytes, but the relevance of this signaling pathway in these diseases remains unclear.

Effects of steroid-resistant nephrotic syndrome serum on AA pathway in podocytes cultured in 3D in vitro glomerular model.

Steroid-resistant nephrotic syndrome (SRNS) is the most severe form of nephrotic syndrome, with genetic or unidentified immunological origins and rapidly progressing to the need for kidney replacement therapy. Lipotoxicity can affect podocytes inducing kidney damage. In this study, we evaluate the effects of SRNS serum on podocyte functionality and lipid metabolism.

Insights from ADPedKD, ERKReg and RaDaR registries provide a multi-national perspective on the presentation of childhood autosomal dominant polycystic kidney disease in high- and middle-income countries.

Data on the presentation of Autosomal Dominant Polycystic Kidney Disease (ADPKD) in children have been based on small/regional cohorts and practices regarding both asymptomatic screening in minors and genetic testing differ greatly between countries. To provide a global perspective, we analyzed over 2100 children and adolescents with ADPKD from 32 countries in six World Health Organization regions: 1060 children from the multi-national ADPedKD registry were compared to 269 pediatric patients from the United Kingdom (RaDaR) and 825 from the European Rare Kidney Disease Registry (ERKReg). Asymptomatic family screening was a common mode of presentation (48% in ADPedKD, 62% in ERKReg) with broad international variability (19%-75%), but fairly stable temporal trends in both registries with no correlation to genetic testing.

Time to remission in childhood steroid sensitive nephrotic syndrome: a change in perspective.

Time to remission (TTR) has been largely considered one of the predictive factors for the risk of relapse and steroid dependency in childhood steroid-sensitive nephrotic syndrome, yet conflicting opinions exist. However, the factors influencing TTR have never been studied. We performed a post-hoc analysis of the prospective pediatric cohort enrolled in a previous multicenter study (ClinicalTrials.

Efficacy of complement inhibition with pegcetacoplan in children with C3 glomerulopathy.

Background: C3 glomerulopathy (C3G) is a rare kidney disease due to a dysregulation of the alternative complement pathway, orphan of specific treatment. Pegcetacoplan is an inhibitor of the third complement component C3, currently on a phase III registration protocol in C3G. Here we describe our experience with the off-label use of pegcetacoplan in pediatric patients with C3G.

IPNA clinical practice recommendations on care of pediatric patients with pre-existing kidney disease during seasonal outbreak of COVID-19.

The coronavirus disease 2019 (COVID-19) pandemic, instigated by the severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), has profoundly impacted healthcare infrastructures around the globe. While children are usually asymptomatic or have mild symptoms, children with pre-existing kidney conditions require specialized attention. This pivotal report, championed by the International Pediatric Nephrology Association (IPNA), delivers precise and actionable recommendations tailored for pediatric patients with kidney ailments in this pandemic landscape.

Modeling a biofluid-derived extracellular vesicle surface signature to differentiate pediatric idiopathic nephrotic syndrome clinical subgroups.

Idiopathic Nephrotic Syndrome (INS) is a common childhood glomerular disease requiring intense immunosuppressive drug treatments. Prediction of treatment response and the occurrence of relapses remains challenging. Biofluid-derived extracellular vesicles (EVs) may serve as novel liquid biopsies for INS classification and monitoring.

Structures, Organization, and Delivery of Kidney Care to Children Living in Low-Resource Settings.

Introduction: There is a disparity in the availability of health care for children in resource-constrained countries. The International Pediatric Nephrology Association (IPNA) commissioned an initiative exploring the challenges in the care of children with kidney disease in low- or middle-income countries (LMICs) with a focus on human, diagnostic, and therapeutic resources.

Methods: A survey was sent by e-mail to all members of IPNA and its affiliated regional or national societies residing in LMICs.

Factors influencing circuit lifetime in paediatric continuous kidney replacement therapies - results from the EurAKId registry.

Background: Continuous kidney replacement therapy (CKRT) has recently become the preferred kidney replacement modality for children with acute kidney injury (AKI). We hypothesise that CKRT technical parameters and treatment settings in addition to the clinical characteristics of patients may influence the circuit lifetime in children.

Methods: The study involved children included in the EurAKId registry (NCT02960867), who underwent CKRT treatment.

Lumasiran treatment in pediatric patients with PH1: real-world data within a compassionate use program in Italy.

Background: Primary hyperoxaluria (PH) is a rare, severe genetic disorder, characterized by increased urinary excretion of calcium oxalate, which is responsible for kidney damage and systemic clinical manifestations. Since the year 2020, a new molecule, lumasiran, based on RNA interference (RNAi) technology, has been added to the traditional therapeutic approach. The aim of this analysis was to define the baseline characteristics of a PH1 pediatric population treated with lumasiran in a compassionate-use program setting, and to evaluate the medium-term efficacy of this drug in the routine clinical setting.

Investigating the use of finerenone in children with chronic kidney disease and proteinuria: design of the FIONA and open-label extension studies.

Introduction: Proteinuria is a modifiable risk factor for chronic kidney disease (CKD) progression in children. Finerenone, a selective, non-steroidal, mineralocorticoid receptor antagonist (MRA) has been approved to treat adults with CKD associated with type 2 diabetes mellitus (T2DM) following results from the phase III clinical trials FIDELIO-DKD (NCT02540993) and FIGARO-DKD (NCT02545049). In a pre-specified pooled analysis of both studies (N = 13,026), finerenone was shown to have an acceptable safety profile and was efficacious in decreasing the risk of adverse kidney and cardiovascular outcomes and of proteinuria.

The relationship between urine heat shock protein 70 and congenital anomalies of the kidney and urinary tract: UTILISE study.

Background: Congenital anomalies of the kidney and urinary tract (CAKUT) are defined as structural malformations of the kidney and/or urinary tract. Heat shock proteins (HSPs) are expressed in the kidney in response to cellular changes, such as thermal, hemodynamic, osmotic, inflammatory, and mechanical stresses. This study aimed to assess uHSP70 levels during acute urinary tract infections (UTI) and non-infection periods in patients with CAKUT, and to evaluate whether uHSP70 is elevated in CAKUT subtypes.

Case Report: effect of lumasiran treatment in a late preterm baby with antenatal diagnosis of primary hyperoxaluria type 1.

Background: Primary hyperoxaluria type 1 (PH1) is a rare disease with autosomal recessive transmission, characterized by increased urinary excretion of oxalate, resulting in chronic kidney disease secondary to recurrent urolithiasis, nephrocalcinosis, and accumulation of oxalate in various organs and tissues (systemic oxalosis). Since 2020, an innovative pharmacological approach, namely, lumasiran, has been added to the therapeutic armamentarium (dialysis and liver-kidney transplantation). The purpose of this paper is to describe the effect of lumasiran initiated at 10 days of life in a newborn with prenatally diagnosed PH1.

Outcome of atypical hemolytic uremic syndrome: role of triggers and complement abnormalities in the response to C5 inhibition.

Background: Atypical-hemolytic uremic syndrome (aHUS) is a rare thrombotic microangiopathy often due to uncontrolled complement activation, characterized by high risk of end-stage kidney disease (ESKD). Eculizumab has improved the outcome, however, its efficacy varies among patients and its discontinuation is debated.

Methods: To identify characteristics associated with treatment response, we analyzed 244 aHUS patients referred to our center.