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The case for treatment of monogenic SRNS with calcineurin inhibitors

Brandon M Lane1, Rasheed A Gbadegesin1

  • 1Department of Pediatrics, Division of Nephrology, Duke University Medical Center, Durham, North Carolina, USA; Duke Molecular Physiology Institute, Duke University School of Medicine, Durham, North Carolina, USA.

Kidney International
|April 21, 2023
PubMed

Insights

Calcineurin inhibitors (CNIs) show promise for treating children with monogenic steroid-resistant nephrotic syndrome, preserving kidney function in responders. Non-responders often experience kidney function decline, highlighting the need for biomarkers.

Area of Science:

  • Pediatric Nephrology
  • Genetics
  • Immunology

Background:

  • Monogenic steroid-resistant nephrotic syndrome (SRNS) lacks evidence-based treatment guidelines for pediatric patients.
  • Steroid-resistant nephrotic syndrome in children often leads to progressive kidney function loss.
  • Calcineurin inhibitors (CNIs) are used off-label, but their efficacy in monogenic forms is not well-established.

Purpose of the Study:

  • To evaluate the effectiveness of calcineurin inhibitor (CNI) treatment in children with monogenic steroid-resistant nephrotic syndrome.
  • To assess the long-term kidney function outcomes in CNI responders versus non-responders.
  • To underscore the need for identifying biomarkers predictive of CNI response in this population.

Main Methods:

  • Retrospective analysis of 141 pediatric patients diagnosed with monogenic steroid-resistant nephrotic syndrome.
  • Review of treatment data focusing on calcineurin inhibitor (CNI) administration and patient response.
  • Assessment of kidney function trajectories based on CNI treatment response.

Main Results:

  • 27.6% of pediatric patients with monogenic SRNS responded to calcineurin inhibitor (CNI) treatment.
  • 75% of CNI responders maintained stable kidney function during the study period.
  • Nearly all CNI non-responders experienced a progressive decline in kidney function.

Conclusions:

  • Calcineurin inhibitors (CNIs) represent a potential therapeutic option for select pediatric patients with monogenic steroid-resistant nephrotic syndrome.
  • CNI treatment appears to preserve kidney function in responders, while non-response is associated with disease progression.
  • Further research is crucial to identify biomarkers that predict CNI response and guide treatment decisions.

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