Treatment outcomes and ongoing pediatric trials in steroid-resistant nephrotic syndrome

Chris K Fan1, Heather S Jung1, Yasmine E Pang1

  • 1Division of Pediatric Nephrology, Department of Pediatrics, Emory University School of Medicine, Atlanta, GA, USA.

Insights

New therapies offer hope for children with steroid-resistant nephrotic syndrome (SRNS), a condition with high kidney failure risk. This review covers novel treatments and ongoing clinical trials for pediatric SRNS.

Area of Science:

  • Pediatric Nephrology
  • Genetics
  • Immunology

Background:

  • Steroid-resistant nephrotic syndrome (SRNS) in children presents significant challenges, with high rates of treatment resistance and progression to kidney failure.
  • Monogenic forms of SRNS and resistance to immunosuppressants pose the greatest risks for pediatric patients.
  • Understanding SRNS pathogenesis is crucial for developing targeted therapies.

Purpose of the Study:

  • To review contemporary findings from cohort studies on pediatric SRNS.
  • To summarize novel therapeutic agents and their specific mechanistic targets.
  • To present an overview of recent and ongoing clinical trials for pediatric SRNS.

Main Methods:

  • Literature review of contemporary cohort studies.
  • Analysis of novel therapeutic agents and their targets in SRNS.
  • Compilation of data from recent and ongoing clinical trials.

Main Results:

  • Advances in understanding SRNS pathogenesis have led to new targeted therapies.
  • Novel agents address genetic, immunologic, and metabolic pathways implicated in SRNS.
  • Eight recent and ongoing clinical trials are evaluating these novel approaches.

Conclusions:

  • Despite challenges, novel therapies show promise for pediatric SRNS.
  • Targeted treatments offer new hope for children at high risk of kidney failure.
  • Ongoing clinical trials are essential for advancing pediatric SRNS management.

Related Concept Videos

Nephrotic Syndrome II : Assessment and Medical Management01:26

Nephrotic Syndrome II : Assessment and Medical Management

IntroductionNephrotic syndrome is a kidney disorder marked by excessive protein loss in the urine, leading to various systemic complications. This condition often results from damage to the glomeruli—the kidney's filtering units—causing proteinuria, low blood protein levels, and fluid retention. Understanding the assessment, diagnosis, and management of nephrotic syndrome is essential for effective treatment and prevention of further kidney damage.AssessmentPatient History: Document...
257
Nephrotic Syndrome III : Nursing Management01:24

Nephrotic Syndrome III : Nursing Management

Nursing management for nephrotic syndrome adapts as the disease progresses, with strategies evolving to address advancing symptoms and complications.Early-Stage Management In the early stages, nursing interventions for nephrotic syndrome resemble those used in managing acute glomerulonephritis, focusing on symptom monitoring, fluid balance, and managing mild to moderate edema.Vital Signs: Regularly monitor blood pressure, pulse, respiratory rate, and temperature to promptly identify...
358
Nephrotic Syndrome I : Introduction01:24

Nephrotic Syndrome I : Introduction

Nephrotic Syndrome is a chronic kidney disorder defined by clinical findings such as severe proteinuria, hypoalbuminemia, hyperlipidemia, and edema. These symptoms result from damage to the glomeruli, the kidney’s filtering units, increasing their permeability to proteins.Definition and Meaning:Proteinuria, defined as the loss of more than 3.5 grams of protein per day in adults, is a crucial feature of nephrotic syndrome. This condition is often accompanied by edema, the accumulation of...
705
Pharmacokinetics in Pediatric Patients: Drug Excretion01:26

Pharmacokinetics in Pediatric Patients: Drug Excretion

In pediatric medicine, understanding the renal function and drug elimination nuances is crucial for administering safe and effective treatments. Newborns, in particular, display markedly slower renal functions than adults, profoundly affecting how drugs are cleared from their bodies. This slower drug clearance requires clinicians to extend the dosing intervals for many medications to prevent drug accumulation and toxicity while ensuring therapeutic efficacy.One key area where these adjustments...
284
Clinical Trials: Overview01:11

Clinical Trials: Overview

Clinical development focuses on how the drug will interact with the human body and encompasses four key phases of clinical trials, each serving a specific purpose in assessing the safety and effectiveness of new drugs. These phases overlap and build upon one another. Phase I involves a small group of healthy volunteers (typically 20-80 individuals) or, in cases where significant toxicity is expected, patients with the targeted disease, such as cancer or AIDS. The volunteers are tested for...
5.1K
Drugs for Treatment of Crohn's Disease in IBD Using Glucocorticoids01:21

Drugs for Treatment of Crohn's Disease in IBD Using Glucocorticoids

Glucocorticoids, a class of anti-inflammatory drugs, are pivotal in treating moderate to severe Crohn's disease by inducing remission. They exhibit their anti-inflammatory action by inhibiting the production of inflammatory cytokines such as tumor necrosis factor (TNF)-α, interleukin (IL)-1, and chemokines like IL-8. In addition, they reduce the expression of inflammatory cell adhesion molecules and inhibit gene transcription of nitric oxide synthase, phospholipase A2, cyclooxygenase-2...
538