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

Francesca Taroni1, Licia Peruzzi2, Germana Longo3

  • 1Pediatric Nephrology Dialysis and Transplant Unit, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.

Clinical Kidney Journal
|May 14, 2024
PubMed

Insights

Lumasiran effectively reduced oxalate levels in children with primary hyperoxaluria (PH), demonstrating safety and efficacy in a real-world setting. Early treatment in pediatric PH patients shows promising results.

Area of Science:

  • Genetics and rare diseases
  • Pharmacology and drug development
  • Pediatric nephrology

Background:

  • Primary hyperoxaluria (PH) is a severe genetic disorder causing kidney damage due to excess oxalate excretion.
  • Lumasiran, an RNA interference (RNAi) therapy, offers a new treatment option since 2020.
  • Understanding lumasiran's real-world effectiveness in pediatric PH is crucial.

Purpose of the Study:

  • To characterize pediatric PH patients treated with lumasiran under a compassionate-use program.
  • To assess the medium-term efficacy and safety of lumasiran in routine clinical practice.
  • To evaluate lumasiran's impact on oxalate levels, kidney stones, and renal function.

Main Methods:

  • Retrospective observational analysis of nine pediatric PH patients.
  • Data collection included plasma/urine oxalate, kidney stone events, and renal function over a mean follow-up of 15.3 months.
  • Assessment of lumasiran's impact on urinary oxalate/creatinine ratio and plasma oxalate.

Main Results:

  • Significant reduction in urinary oxalate/creatinine ratio (median 51.2%) and plasma oxalate below supersaturation levels.
  • Most patients maintained stable kidney function and nephrocalcinosis status.
  • No lumasiran-related adverse events were reported; only one new stone event occurred.

Conclusions:

  • Lumasiran demonstrates significant efficacy in reducing oxalate burden in pediatric PH.
  • The drug is safe and effective for medium-term use in children.
  • Early administration of lumasiran in pediatric PH patients is particularly beneficial.
Abstract

Keywords:
lumasiranoutcomepediatric patientsprimary hyperoxaluria type 1

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