Optimizing ADAMTS13 prophylaxis to reduce relapse and organ failure in congenital thrombotic thrombocytopenic purpura.

Congenital thrombotic thrombocytopenic purpura (cTTP) is caused by a severe inherited ADAMTS13 deficiency. While acute episodes are life-threatening, long-term burden of ischemic complications and effectiveness of prophylactic strategies remain underexplored. We conducted a 25-year national, multicenter study of 88 cTTP patients enrolled in the French Thrombotic Microangiopathy (TMA) registry.

[Management of congenital thrombotic thrombocytopenic purpura in the era of recombinant ADAMTS13 protein: Recommendations from the Reference Center for Thrombotic Microangiopathies (CNR-MAT)].

Thrombotic Thrombocytopenic Purpura (TTP) is a rare disease characterized by a severe deficiency of ADAMTS13, the specific protease that cleaves von Willebrand factor. The congenital form of TTP (cTTP) results from pathogenic variants of the ADAMTS13 gene. cTTP has two peaks of incidence: one in childhood and the other in adulthood, mainly in an obstetric context.

Obinutuzumab in Frequently Relapsing and Steroid-Dependent Nephrotic Syndrome in Children.

Background: B-cell depletion with rituximab induces sustained remission in children with steroid-dependent or frequently relapsing nephrotic syndrome. However, most patients relapse after B-cell recovery, and some patients do not achieve B-cell depletion. Obinutuzumab is a second-generation anti-CD20 antibody designed to overcome such situations in B-cell malignancies and was recently reported to be safe and effective in other autoimmune diseases affecting the kidneys.

Efficacy and Safety of Eculizumab in Pediatric Patients Affected by Shiga Toxin-Related Hemolytic and Uremic Syndrome: A Randomized, Placebo-Controlled Trial.

Significance Statement: Shiga toxin-related hemolytic uremic syndrome (STEC-HUS) is a serious condition, characterized by multiorgan thrombotic microangiopathy, mainly affecting children. Renal involvement is severe, with approximately half of patients requiring dialysis. So far, no specific treatment has been proven efficient in STEC-HUS.

Biallelic loss of function variants are responsible for neonatal systemic hypertension.

Background: Early-onset isolated systemic hypertension is a rare condition of unknown genetic origin. Renovascular, renal parenchymal diseases or aortic coarctation are the most common causes of secondary systemic hypertension in younger children and neonates. We investigated the genetic bases of early-onset isolated systemic hypertension.

Renal vein thrombosis in neonates: a case series of diagnosis, treatment and childhood kidney function follow-up.

Background: Neonatal renal vein thrombosis (NRVT) is a rare condition with little data available.

Methods: We retrospectively analyzed newborns diagnosed with NRVT admitted to 3 pediatric nephrology units in Paris from 2005 to 2020.

Results: Twenty-seven patients were analyzed (male = 59%).

Juvenile Neuropsychiatric Systemic Lupus Erythematosus: Identification of Novel Central Neuroinflammation Biomarkers.

Introduction: Juvenile systemic lupus erythematosus (j-SLE) is a rare chronic autoimmune disease affecting multiple organs. Ranging from minor features, such as headache or mild cognitive impairment, to serious and life-threatening presentations, j-neuropsychiatric SLE (j-NPSLE) is a therapeutic challenge. Thus, the diagnosis of NPSLE remains difficult, especially in pediatrics, with no specific biomarker of the disease yet validated.

Benefits of BNP/NT-proBNP serum level evaluation for dry weight adjustment in pediatric hemodialysis patients.

Background: Dry weight (DW) adjustment in children on hemodialysis (HD) can be challenging. It relies on clinical evaluation and additional supports. Our aim was to study the benefits of cardiac biomarker assessment, in addition to the more commonly used technique, bioimpedance spectroscopy (BIS), and clinical signs for DW prescription in pediatric HD patients.

Social Deprivation Is Associated With Lower Access to Pre-emptive Kidney Transplantation and More Urgent-Start Dialysis in the Pediatric Population.

Introduction: Socioeconomic status (SES) is recognized as an important determinant of kidney health. We aimed to evaluate the association of social deprivation with different indicators at kidney replacement therapy (KRT) initiation in the French pediatric metropolitan population.

Methods: All patients with end-stage kidney disease (ESKD) who started KRT before 20 years old in France between 2002 and 2015 were included.

Specific immune biomarker monitoring in two children with severe IgA nephropathy and successful therapy with immunoadsorption in a rapidly progressive case.

Background: Childhood IgA nephropathy (cIgAN) is one of the most common primary glomerulonephritides with the potential to evolve to kidney failure. IgAN is an autoimmune disease involving 3 key factors: galactose-deficient IgA1 (Gd-IgA1), anti-IgA1 autoantibodies, and soluble (s)CD89 IgA Fc receptor. These molecules and immune complexes have been described recently as potential biomarkers of disease progression in childhood IgAN but their evolution in time under immunosuppressive treatment remains unknown.

Predicting others' social interaction preferences: What to do, for how long, and how often.

We present a three-dimensional what, how long, and how often framework to discuss three main inferences about other people's preferences for repeated social interactions over time in terms of: (1) what to do together, (2) how long to spend together on each occasion, and (3) how often to spend time together. For each dimension, we discuss when and how people make inferences about other people's preferences, as well as the consequences of making incorrect inferences. The three dimensions are conceptually independent; however, decisions made on one dimension can sometimes affect decisions made on others.

Anti-rituximab antibodies in pediatric steroid-dependent nephrotic syndrome.

Background: Rituximab is a chimeric anti-CD20 monoclonal antibody that induces sustained remission in children with steroid-dependent nephrotic syndrome. However, there is no consensus on the optimal regimen and monitoring of rituximab. In other autoimmune diseases, anti-rituximab antibodies (ARA) have been reported in 10-40% of patients, but their clinical relevance remains unclear.

Procalcitonin serum levels in stage 5 chronic kidney disease children on hemodialysis.

Background: Infections are responsible for morbidity and mortality in children on hemodialysis (HD). Procalcitonin (PCT) is rarely used in this population, even though it is an efficient biomarker of infection and sepsis. Our aim was to study PCT baseline level in uninfected children with stage 5 chronic kidney disease (CKD 5) on HD, and determine how to use it in this population.

A global antiB cell strategy combining obinutuzumab and daratumumab in severe pediatric nephrotic syndrome.

Background: Steroid-sensitive nephrotic syndrome (SSNS) is, in most patients, a chronic disease with 80% experiencing at least one relapse after first flare. B cell depletion using rituximab is effective in preventing relapse in steroid-dependent (SDNS) patients but fails to maintain long-term remission following B cell recovery, possibly due to development of autoreactive long-lived plasma cells. We investigated sequential combination of antiCD20 antibody targeting all B cell subsets, and antiCD38 antibody with high plasma cell cytotoxicity in patients with uncontrolled SDNS after failure of one or several attempts at B cell depletion.

A Successful Renal Transplant in a Pediatric Patient With Glanzmann Thrombasthenia and Hyperimmunization.

We report the case of a patient with type 2 Glanzmann thrombasthenia who underwent successful kidney transplant with his mother's kidney. He started dialysis at 13 months. The patient had been diagnosed with Glanzmann thrombasthenia at 9 years old, after hemorrhagic shock, during which multiple transfusions were required and hyperimmunization had developed.

Ultrasound dilution and thermodilution versus color Doppler ultrasound for arteriovenous fistula assessment in children on hemodialysis.

Background: This study is aimed at comparing ultrasound dilution (UD) and thermodilution (TD) with color Doppler ultrasound (CDU) for arteriovenous fistula (AVF) assessment in children on hemodialysis (HD).

Material And Methods: All patients were dialysed with the Fresenius 5008 HD machine. UD was performed using the Transonic device.

Asymptomatic bacteriuria in pediatric kidney transplant recipients: to treat or not to treat? A retrospective study.

Background: Urinary tract infections (UTI) are common infectious complications in kidney transplant recipients (KTR); asymptomatic bacteriuria (AB) is also frequent. It is unclear whether treatment of AB reduces subsequent UTI in KTR; no guideline is available in pediatric KTR. In this retrospective study, we analyzed the incidence of AB in pediatric KTR and the impact of screening and treating AB on the onset of subsequent UTI.