Search

Background Infants with chronic kidney disease (CKD) form a vulnerable population who are highly prone to mineral and bone disorders (MBD) including biochemical abnormalities, growth retardation, bone deformities, and fractures. We present a position paper on the diagnosis and management of CKD-MBD in infants based on available evidence and the opinion of experts from the European Society for Paediatric Nephrology (ESPN) CKD-MBD and Dialysis working groups and the Pediatric Renal Nutrition Taskforce. Methods PICO (Patient, Intervention, Comparator, Outcomes) questions were generated, and relevant literature searches performed covering a population of infants below 2 years of age with CKD stages 2-5 or on dialysis. Clinical practice points (CPPs) were developed and leveled using the American Academy of Pediatrics grading matrix. A Delphi consensus approach was followed. Results We present 34 CPPs for diagnosis and management of CKD-MBD in infants, including dietary control of calcium and phosphate, and medications to prevent and treat CKD-MBD (native and active vitamin D, calcium supplementation, phosphate binders). Conclusion As there are few high-quality studies in this field, the strength of most statements is weak to moderate, and may need to be adapted to individual patient needs by the treating physician. Research recommendations to study key outcome measures in this unique population are suggested. Graphical Abstract, Author(s): Justine Bacchetta [sup.1] [sup.2] [sup.3] , Claus Peter Schmitt [sup.4] , Sevcan A. Bakkaloglu [sup.5] , Shelley Cleghorn [sup.6] , Maren Leifheit-Nestler [sup.7] , Agnieszka Prytula [sup.8] , Bruno [...]

Children requiring long-term kidney replacement therapy are a "rare disease" cohort. While the basic technical requirements for hemodialysis (HD) are similar in children and adults, key aspects of the child's cardiovascular anatomy and hemodynamic specifications must be considered. In this article, we describe the technical requirements for long-term HD therapy for children and the devices that are currently available around the world. We highlight the characteristics and major technical shortcomings of permanent central venous catheters, dialyzers, dialysis machines, and software available to clinicians who care for children. We show that currently available HD machines are not equipped with appropriately small circuits and sensitive control mechanisms to perform safe and effective HD in the youngest patients. Manufacturers limit their liability, and health regulatory agencies permit the use of devices, only in children according to the manufacturers' pre-specified weight limitations. Although registries show that 6–23% of children starting long-term HD weigh less than 15 kg, currently, there is only one long-term HD device that is cleared for use in children weighing 10 to 15 kg and none is available and labelled for use in children weighing less than 10 kg anywhere in the world. Thus, many children are being treated "off-label" and are subject to interventions delivered by medical devices that lack pediatric safety and efficacy data. Moreover, recent improvements in dialysis technology offered to adult patients are denied to most children. We, in turn, advocate for concerted action by pediatric nephrologists, industry, and health regulatory agencies to increase the development of dedicated HD machines and equipment for children. [ABSTRACT FROM AUTHOR]

Despite significant medical and technical improvements in the field of dialysis, the morbidity and mortality among patients with chronic kidney disease (CKD) stage 5 on dialysis remains extremely high. Hemodiafiltration (HDF), a dialysis method that combines the two main principles of hemodialysis (HD) and hemofiltration—diffusion and convection—has had a positive impact on survival when delivered with a high convective dose. Improved outcomes with HDF have been attributed to the following factors: HDF removes middle molecular weight uremic toxins including inflammatory cytokines, increases hemodynamic stability, and reduces inflammation and oxidative stress compared to conventional HD. Two randomized trials in adults have shown improved survival with HDF compared to high-flux HD. A large prospective cohort study in children has shown that HDF attenuated the progression of cardiovascular disease, improved bone turnover and growth, reduced inflammation, and improved blood pressure control compared to conventional HD. Importantly, children on HDF reported fewer headaches, dizziness, and cramps; had increased physical activity; and improved school attendance compared to those on HD. In this educational review, we discuss the technical aspects of HDF and results from pediatric studies, comparing outcomes on HDF vs. conventional HD. Convective volume, the cornerstone of treatment with HDF and a key determinant of outcomes in adult randomized trials, is discussed in detail, including the practical aspects of achieving an optimal convective volume. [ABSTRACT FROM AUTHOR]

Background Atypical hemolytic and uremic syndrome (aHUS), a thrombotic micro-angiopathy (TMA) caused by deregulation in the complement pathway, is sometimes due to the presence of anti-complement factor H (CFH) auto-antibodies. The 'standard' treatment for such aHUS combines plasma exchange therapy and immunosuppressive drugs. Eculizumab, a monoclonal antibody that blocks the terminal pathway of the complement cascade, could be an interesting alternative in association with an immunosuppressive treatment for maintenance regimen. Case-diagnosis/treatment We report on two children, diagnosed with mildly severe aHUS due to anti-CFH antibodies, who were treated with the association eculizumab-mycophenolate mofetil (MMF). Neither side effects nor relapses were observed during the 3 years of follow-up; MMF was even progressively tapered and withdrawn successfully in one patient. Conclusions The association of eculizumab and MMF appears to be an effective and safe option in pediatric cases of aHUS due to anti-CFH antibodies of mild severity., Author(s): Lucie Matrat [sup.1] , Justine Bacchetta [sup.1] [sup.2] , Bruno Ranchin [sup.1] , Corentin Tanné [sup.1] [sup.2] [sup.3] , Anne-Laure Sellier-Leclerc [sup.1] Author Affiliations: (1) grid.414103.3, Centre de Référence [...]

Introduction In nephropathic cystinosis (NC), adherence to cysteamine remains challenging; poor adherence is worsening the disease progression with a decline of kidney function and increase of extrarenal morbidities. Our objective was to describe adherence to cysteamine in NC patients, using electronic monitoring systems. Methods Patients with confirmed NC, aged > 4 years and receiving oral cysteamine (short acting or delayed release formulation as standard of care) from 3 French reference centers, were included. Adherence to treatment was primarily assessed as the percentage of days with a good adherence score, adherence score rating from 0 (poor) to 2 (good). A descriptive analysis was performed after 1-year follow-up. Results Seventeen patients (10 girls, median age: 13.9 (5.4-33.0) years) were included. Median age at diagnosis was 17.0 (3.0-76.9) months and age at start of cysteamine was 21.0 (15.5-116.3) months. Median daily dose of cysteamine was 1.05 (0.55-1.63) g/m.sup.2/day. Over the year, the median percentage of days with a good adherence score was 80 (1-99)% decreasing to 68 (1-99)% in patients > 11 years old. The median of average number of hours covered by treatment in a day was 22.5 (6.1-23.9) versus 14.9 (9.2-20.5) hours for delayed release versus short acting cysteamine. Conclusion Our data are the first describing a rather good adherence to cysteamine, decreasing in adolescents and adults. We described a potential interest of the delayed release formulation. Our data highlight the need for a multidisciplinary approach including therapeutic education and individualized approaches in NC patients transitioning to adulthood. Graphical abstract, Author(s): Segolene Gaillard [sup.1] [sup.2] , Laurent Roche [sup.2] [sup.4] , Sandrine Lemoine [sup.3] , Georges Deschênes [sup.5] , Denis Morin [sup.6] , Christine Vianey-Saban [sup.7] , Cécile Acquaviva-Bourdain [sup.7] [...]

Patients with Down syndrome (DS) are at risk of multiorgan dysfunction; kidney and urological impairment are common. This is due to a likely increased risk of congenital kidney and urological malformations (odds ratio of 4.5 compared to the general population in one study), more frequent associated comorbidities at risk of kidney dysfunction (such as prematurity in 9–24% of children, intrauterine growth retardation or low birth weight in 20%, and congenital heart disease in 44%), and more frequent lower urinary tract dysfunction (reported in 27–77% of children with DS). If present, malformations and comorbidities at risk of kidney dysfunction warrant regular kidney monitoring in addition to their treatment. Serum creatinine in children with DS has been shown to be higher than in the general population and asymptomatic hyperuricemia is reported in 12–33% of children or young adults with DS. Moreover cryptorchidism and testicular cancer are also more common and should be detected by clinical examination. Thus, persons with DS at risk of presenting kidney and urological impairment should be identified by prenatal ultrasonography, comorbidities at risk of kidney sequelae considered, and during regular medical follow-up, clinically examined and questioned to diagnose testicular anomalies and lower urinary tract dysfunction. This is of importance as such kidney and urological impairments are associated with impaired quality of life and mental health, and risk of kidney failure. [ABSTRACT FROM AUTHOR]

Background: Sodium (Na) balance is unexplored in dialyzed children. We assessed a simplified sodium balance (sNaB) and its correlates in pediatric patients receiving maintenance dialysis. Methods: Patients < 18 years old on hemodialysis (HD) or peritoneal dialysis (PD) in six European Pediatric Dialysis Working Group centers were recruited. sNaB was calculated from enteral Na, obtained by a 3-day diet diary, Na intake from medications, and 24-h urinary Na (uNa). Primary outcomes were systolic blood pressure and diastolic blood pressure standard deviation scores (SBP and DBP SDS), obtained by 24-h ambulatory blood pressure monitoring or office BP according to age, and interdialytic weight gain (IDWG). Results: Forty-one patients (31 HD), with a median age of 13.3 (IQR 5.2) years, were enrolled. Twelve patients (29.3%) received Na-containing drugs, accounting for 0.6 (0.7) mEq/kg/day. Median total Na intake was 1.5 (1.1) mEq/kg/day, corresponding to 60.6% of the maximum recommended daily intake for healthy children. Median uNa and sNaB were 0.6 (1.8) mEq/kg/day and 0.9 (1.7) mEq/kg/day, respectively. The strongest independent predictor of sNaB in the cohort was urine output. In patients receiving HD, sNaB correlated with IDWG, pre-HD DBP, and first-hour refill index, a volume index based on blood volume monitoring. sNaB was the strongest predictor of IDWG in multiple regression analysis (β = 0.63; p = 0.005). Neither SBP SDS nor DBP SDS correlated with sNaB. Conclusions: Na intake is higher than uNa in children on dialysis, and medications may be an important source of Na. sNaB is best predicted by urine output in the population, and it is a significant independent predictor of IDWG in children on HD. [ABSTRACT FROM AUTHOR]

Objective Mineral and bone disorders associated to chronic kidney disease (CKD-MBD) are a daily challenge for pediatric nephrologists, with a significant risk of long-term bone and vascular comorbidities. Methods This single-center study is a prospective transversal evaluation of pediatric CKD patients of our center, part of the European 4C study. In addition to clinical and biochemical data, vascular and bone evaluation was performed: 24-h blood pressure assessment, carotid intima-media thickness (cIMT), pulse wave velocity (PWV), and high-resolution peripheral quantitative computed tomography (HR-pQCT) at the ultra-distal tibia. Results are presented as median (range). Results At a median age of 12.9 years (10.2-17.9), SDS height of - 1.0 (- 3.3-1.2) and estimated glomerular filtration rate (eGFR) of 33 mL/min/1.73m.sup.2 (11-72), 32 patients (8 girls) were evaluated. Median calcium, phosphate, parathyroid hormone (PTH), and 25 OHD3 levels were 2.44 mmol/L (2.24-2.78), 1.43 mmol/L (1.0-2.7), 80 pg/mL (9-359), and 70 nmol/L (32-116), respectively. Bivariate Spearman and backward multivariable analyses showed that calcium and bone trabecular thickness (Tb.Th), were positively associated with diastolic and mean arterial blood pressure (both for the 24 h, day and night assessment), whereas PTH and vitamin D did not predict blood pressure. Conclusions We show that the greater the serum levels of calcium, the greater the (diastolic and mean) blood pressure; moreover, the greater the Tb. Th, the greater the (diastolic and mean) blood pressure. The role of calcium supplements to explain such findings in early pediatric CKD can be discussed., Author(s): Evgenia Preka [sup.1] , Bruno Ranchin [sup.1] , Anke Doyon [sup.2] , Melody Vierge [sup.1] , Tiphanie Ginhoux [sup.3] , Behrouz Kassai [sup.3] [sup.4] [sup.5] [sup.6] , Justine Bacchetta [...]

Background Post-transplant diabetes mellitus (PTDM) is a major complication of immunosuppressive therapy, with many risk factors reported in adults with renal transplantation. The objective of this study was to investigate potential non-genetic and genetic risk factors of PTDM in children with renal transplantation treated with tacrolimus. Methods A national database was screened for patients developing PTDM within 4 years following tacrolimus introduction. PTDM was defined as glucose disorder requiring anti-diabetic treatment. PTDM patients were matched to 'non-PTDM' control transplanted children according to age, gender, and duration of post-transplant follow-up. Patients were genotyped for six selected genetic variants in POR*28 (rs1057868), PPARa (rs4253728), CYP3A5 (rs776746), VDR (rs2228570 and rs731236), and ABCB1 (rs1045642) genes, implicated in glucose homeostasis and tacrolimus disposition. Results Among the 98 children with renal transplantation enrolled in this multicentre study, 18 developed PTDM. None of the clinical and biological parameters was significant between PTDM and control patients. Homozygous carriers of POR*28 or wild-type ABCB1 (rs1045642) gene variants were more frequent in PTDM than in control patients with differences close to significance (p = 0.114 and p = 0.066 respectively). A genetic score based on these variants demonstrated that POR*28/*28 and ABCB1 CC or CT genotype carriers were at a significantly higher risk of developing PTDM after renal transplantation. Conclusion Identification of PTDM risk factors should allow clinicians to allocate the best immunosuppressant for each patient with renal transplantation, and improve care for patients who are at a higher risk., Author(s): Pauline Lancia [sup.1] , Tiphaine Adam de Beaumais [sup.1] , Valéry Elie [sup.1] , Florentine Garaix [sup.2] , Marc Fila [sup.3] , François Nobili [sup.4] , Bruno Ranchin [sup.5] [...]

Background Childhood-onset systemic lupus erythematosus (cSLE) is rare, and considered more severe than its adult-onset counterpart. Lupus nephritis (LN) occurs more frequently in children, accounting for higher long-term morbidity and mortality compared with adults. Thus, reliable biological markers are needed to predict disease course. This study aimed to investigate the capacity of anti-C1q autoantibodies (Abs) to predict renal flare and global disease activity in cSLE patients, and association with disease activity and kidney involvement. Methods Twenty-eight patients with cSLE including 19 patients (68%) with a history of LN were included retrospectively. Anti-C1q Abs were analysed by ELISA at renal flare-up or in the quiescent phase of disease and compared with Farr dsDNA assay. Results Thirty-one flares occurred during follow-up: anti-C1q Abs were positive in 26 (84%), strongly associated with active disease status (p < 0.0001), and correlated with global disease activity score (p < 0.0001) and anti-dsDNA Abs presence (p < 0.0001). The specificity of anti-C1q Abs was higher than anti-dsDNA (73% vs 19%) in discriminating LN patients, whereas the receiver operating characteristic curves were not statistically different (0.83 ± 0.06 vs 0.78 ± 0.08 respectively), similar to C3 dosage. The presence of anti-C1q Abs at diagnosis was not predictive for global or renal flare. Introduction of a modified SLEDAI score excluding dsDNA Abs, demonstrated a stronger correlation of anti-C1q Abs titres with SLEDAI score in comparison with the Farr test. Conclusion Anti-C1q Abs seem very specific to flares, including LN in children, and their role in daily practice compared with the Farr dsDNA assay needs to be defined., Author(s): Cécile Picard [sup.1] [sup.2] , Jean-Christophe Lega [sup.2] [sup.3] [sup.4] , Bruno Ranchin [sup.2] [sup.5] , Pierre Cochat [sup.2] [sup.5] , Natalia Cabrera [sup.2] [sup.5] , Nicole Fabien [sup.2] [...]

Background Peritoneal dialysis (PD) remains the modality of choice in children, but there is no clear evidence to support a better outcome in children treated with PD. We aimed to assess factors that have an impact on the choice of dialysis modality in children and young adults in France and sought to determine the roles of medical factors and center practices. Methods We included all patients aged Results We included 806 patients starting RRT in 177 centers, 23 of which were specialized pediatric centers. Six hundred and one patients (74.6 %) started with hemodialysis (HD), whereas 205 (25.4 %) started with PD. A greater probability of PD was found in younger children, whereas starting the treatment in an emergency setting was associated with a low use of PD. We found a significant variability among centers that accounted for 43 % of the total variability. The probability of PD was higher in adult centers and was proportional to the rate of PD in the center. Conclusions Center practices are a major factor in the choice of dialysis modality. This raises concerns about patient and family choices and to what extent doctors may influence the final decision. Further pediatric studies focusing on children's and parents' wishes are needed to provide care as close as possible to patients' and families' expectations., Author(s): Julien Hogan [sup.1] [sup.2] , Bruno Ranchin [sup.3] , Marc Fila [sup.4] , Jérome Harambat [sup.5] , Saoussen Krid [sup.6] , Isabelle Vrillon [sup.7] , Gwenaelle Roussey [sup.8] , [...]

Background The management of tubulopathies after renal transplantation (RTx) may require high doses of sodium and bicarbonate, reducing the quality of life and therapeutic compliance of the patient. Some studies on adult patients have highlighted the benefits of fludrocortisone (fludro) in the treatment of severe tubulopathies. Methods This study was a retrospective review of the medical charts of 15 children, aged 12.4 (range 3.6-17.4) years who received fludro after RTx. Results With the administration of fludro, both sodium bicarbonate and chloride supplementation decreased, from 10 (range 0-14) to 0 (0-5) g/day, and from 9 (0-20) to 0 (0-3) g/day, respectively (both p Conclusions Based on our findings, fludro would appear to be an effective therapy in most cases of severe tubulopathy after RTx. Further prospective studies are required to validate this indication and to determine the optimal dose and timing of treatment to avoid side-effects as well as the clinical and biological follow-up. Keywords Bicarbonates * Fludrocortisone * Pediatrics * Renal transplantation * Sodium * Tubulopathy, Introduction Tubulopathy is frequent after renal transplantation (RTx), in association with metabolic acidosis, salt-wasting tubulopathy and (occasionally) hyperkalemia, corresponding at least partly to a certain degree of hypoaldosteronism despite normal [...]

Background C3 glomerulopathy (C3G) is characterized by predominant C3 deposits in glomeruli and dysregulation of the alternative pathway of complement. Half of C3G patients have a C3 nephritic factor (C3NeF). C3G incorporated entities with a range of features on microscopy including dense deposit diseases (DDD) and C3 glomerulonephritis (C3GN). The aim of this work was to study children cases of C3G associated with C3NeF. Methods We reviewed 18 cases of C3G with a childhood onset associated with C3NeF without identified mutations in CFH, CFI, and MCP genes. Results Clinical histories started with recurrent hematuria for seven patients, nephrotic syndrome for four, acute post infectious glomerulonephritis for three and acute renal failure for four. Twelve patients had a low C3 at first investigation. Kidney biopsy showed ten C3GN and eight DDD. Twenty-three percent of the patients tested presented elevated sC5b9. Seven patients relapsed 3 to 6 years after the onset. At the end of follow-up, two patients were under dialysis, 11 had a persistent proteinuria, five had none; four patients did not follow any treatment. Steroids were first used in 80% of cases. Conclusions C3NeF associated C3G has a heterogeneous presentation and outcome. Anti-proteinuric agents may control the disease during follow-up, even after nephrotic syndrome at the onset. The efficiency of immunosuppressive therapy remains questionable. Keywords Complement alternative pathway * C3 glomerulonephritis * Dense deposit disease * Membranoproliferative glomerulonephritis * C3 glomerulopathy, Introduction C3 glomerulopathy (C3G) is the term now used for C3 glomerulonephritis (C3GN) and dense deposit disease (DDD), both subtypes resulting from dysregulation of the alternative pathway (AP) of complement [...]

Background Deferasirox (DFX) is an oral iron chelator with an established dose-dependent efficacy in transfusion-related iron overload. Whereas emerging long-term data confirm the safety of the drug, with transient moderate elevation of serum creatinine level, several authors have reported renal tubular dysfunction. The aim of this study was to evaluate tubular and glomerular function before and after the initiation of DFX therapy in a pediatric patient population. Methods Ten children (4 girls, mean age 12.4 ± 3.9 years) enrolled in a routine blood transfusion program were treated with 24.8 [+ or -] 9.6 mg/kg per day of DFX, and renal function was assessed before and 17.2 ± 8.9 months after the initiation of DFX therapy. Results Prior to treatment with DFX, all patients had a normal glomerular function rate (GFR) (125 ± 15 ml/min per 1.73 [m.sup.2]) and normal tubular function. Following the initiation of DFX therapy, the GFR decreased by approximately 20% with one patient with a GFR of < 80 mL/min per 1.73 [m.sup.2] and seven patients with a GFR of Conclusions Renal toxicity is a frequent adverse event of DFX treatment, presenting as both glomerular and proximal dysfunction. A routine renal assessment is therefore required to prevent chronic kidney disease that may result from prolonged tubular injury. Keywords Children * Deferasirox * Drug nephrotoxicity * Iron-chelating agent * Glomerular filtration rate * Proximal tubular toxicity, Introduction Iron overload due to multiple red blood cell transfusions is a major concern in patients with thalassemia major, sickle cell disease, myelodysplastic syndromes, and other rare anemias [1]. Blood [...]

Background Autosomal dominant polycystic kidney disease (ADPKD) in children is often regarded as a benign condition. However, previous studies pointed out renal-related anomalies which may benefit from early appropriate treatments. This study was conducted to evaluate the prevalence and severity of early renal dysfunction in ADPKD children. Methods An extensive renal evaluation was performed in 52 consecutive ADPKD patients diagnosed either from prenatal screening or post-natal ultrasound (US) examination (54% males, mean age 10 [+ or -] 4 years [1-17]). Results Three patients had both systolic (SBP) and diastolic (DBP) blood pressure above the 95th percentile, one patient had a 'high normal' DBP, and one child was treated with an angiotensin-converting enzyme inhibitor (ACEI). The mean [+ or -] SD glomerular filtration rate (GFR ml/min per 1.73 [m.sup.2], inulin clearance) was 115 [+ or -] 26 [47-168] but six children (12%) had a GFR 135). Microalbuminuria (220 mg/mmol). Conclusions Early renal manifestations are frequent in ADPKD children, including hypertension in 6%, albuminuria in 58%, and decreased GFR in 12%. In conclusion, renal function in children with ADPKD should be regularly assessed in order to manage early renal dysfunction and even consider further therapeutic intervention. Keywords Autosomal dominant polycystic kidney disease * Children * Renal function * Glomerular filtration rate * Arterial hypertension * Microalbuminuria, Introduction Autosomal dominant polycystic kidney disease (ADPKD) is the most prevalent inherited renal disease. Despite the usual onset of end-stage kidney disease during adulthood, pre-symptomatic screening for ADPKD in children [...]

Bone damage in children with chronic kidney disease (CKD) is a challenge for pediatric nephrologists. Areal measurements of bone mineral density (BMD) by dual x-ray absorptiometry (DXA) have been routinely performed to assess bone mass but recent international guidelines have concluded that DXA was of less value in CKD. The aim of this study is to evaluate bone quality in CKD children using new bone imaging techniques in a pilot cross-sectional single-center study. We performed bone imaging (high-resolution peripheral quantitative computed tomography, HR-pQCT, XtremeCT, Scanco Medical AG, Switzerland), to assess compartmental volumetric BMD and trabecular microarchitecture in 22 CKD children and 19 controls. In seven younger patients (i.e., under 10 years of age), we performed bone texture analysis (BMA, D3A Medical Systems, France) in comparison to 15 healthy prepubertal controls. Among older children, CKD patients had significantly lower height and body weight without significant impairment of BMD and microarchitecture than healthy controls. In univariate analysis, there were significant correlations between cortical BMD and glomerular filtration rate (r =-0.46), age (r=0.60) and body mass index (r=0.67). In younger children, bone texture parameters were not different between patients and controls. Our results did not show significant differences between healthy controls and CKD children for compartmental bone densities and microarchitecture, but the small sample size and the heterogeneity of the CKD group require caution in the interpretation. Novel bone imaging techniques seem feasible in children, and further longitudinal studies are required to thoroughly explore long-term cardiovascular and bone consequences of phosphate-calcium metabolism deregulation during CKD. Keywords Bone imaging * Children * Chronic kidney Disease * Dialysis * HR-pQCT * Texture analysis, Introduction The deleterious effect of chronic kidney disease (CKD) on bone results from a combination of factors, e.g., vitamin D deficiency, hyperparathyroidism, hypogonadism, malnutrition, resistance to growth hormone and drug [...]

Abstract Primary hyperoxaluria type 1, the most common form of primary hyperoxaluria, is an autosomal recessive disorder caused by a deficiency of the liver-specific enzyme alanine: glyoxylate aminotransferase (AGT). This [...]

Abstract The occurrence of membranous nephropathy in pediatric series of systemic lupus erythematosus has been reported only rarely, probably due to a very low frequency. One hundred fifty-four children who [...]

Abstract Noncompliance is frequent in children and adolescents with nephrotic syndrome. Once suspected, noncompliance is difficult to confirm and often impossible to avoid. The standard oral glucocorticoid treatment for children [...]

Abstract Nephrotoxicity is a well-known adverse effect of cyclosporine A (CyA) treatment in children with steroid-dependent (SD) and steroid-resistant (SR) nephrotic syndrome (NS). We analyzed nine children (age: 3.3-15.7 years, [...]

Introduction: In nephropathic cystinosis (NC), adherence to cysteamine remains challenging; poor adherence is worsening the disease progression with a decline of kidney function and increase of extrarenal morbidities. Our objective was to describe adherence to cysteamine in NC patients, using electronic monitoring systems. Methods: Patients with confirmed NC, aged > 4 years and receiving oral cysteamine (short acting or delayed release formulation as standard of care) from 3 French reference centers, were included. Adherence to treatment was primarily assessed as the percentage of days with a good adherence score, adherence score rating from 0 (poor) to 2 (good). A descriptive analysis was performed after 1-year follow-up. Results: Seventeen patients (10 girls, median age: 13.9 (5.4–33.0) years) were included. Median age at diagnosis was 17.0 (3.0–76.9) months and age at start of cysteamine was 21.0 (15.5–116.3) months. Median daily dose of cysteamine was 1.05 (0.55–1.63) g/m2/day. Over the year, the median percentage of days with a good adherence score was 80 (1–99)% decreasing to 68 (1–99)% in patients > 11 years old. The median of average number of hours covered by treatment in a day was 22.5 (6.1–23.9) versus 14.9 (9.2–20.5) hours for delayed release versus short acting cysteamine. Conclusion: Our data are the first describing a rather good adherence to cysteamine, decreasing in adolescents and adults. We described a potential interest of the delayed release formulation. Our data highlight the need for a multidisciplinary approach including therapeutic education and individualized approaches in NC patients transitioning to adulthood. Graphical abstract [ABSTRACT FROM AUTHOR]

Background: COVID-19 was declared a global health emergency. Since children are less than 1% of reported cases, there is limited information to develop evidence-based practice recommendations. The objective of this study was to rapidly gather expert knowledge and experience to guide the care of children with chronic kidney disease during the COVID-19 pandemic. Methods: A four-round multi-center Delphi exercise was conducted among 13 centers in 11 European countries of the European Pediatric Dialysis Working Group (EPDWG) between March, 16th and 20th 2020. Results were analyzed using a mixed methods qualitative approach and descriptive statistics. Results: Thirteen COVID-19 specific topics of particular need for guidance were identified. Main themes encompassed testing strategies and results (n = 4), changes in use of current therapeutics (n = 3), preventive measurements of transmission and management of COVID-19 (n = 3), and changes in standard clinical care (n = 3). Patterns of center-specific responses varied according to regulations and to availability of guidelines. Conclusions: As limited quantitative evidence is available in real time during the rapid spread of the COVID-19 pandemic, qualitative expert knowledge and experience represent the best evidence available. This Delphi exercise demonstrates that use of mixed methodologies embedded in an established network of experts allowed prompt analysis of pediatric nephrologists' response to COVID-19 during this fast-emerging public health crisis. Such rapid sharing of knowledge and local practices is essential to timely and optimal guidance for medical management of specific patient groups in multi-country health care systems such as those of Europe and the US. [ABSTRACT FROM AUTHOR]