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. 2025 Mar 11;21(1):31-35.
doi: 10.1684/ndt.2025.108.

Fine-tuning circulating oxalate levels to improve transplant strategies in primary hyperoxaluria: what is the ideal threshold in pediatrics?

Marisca Carlina Makosso Afiavi  1   2 , Anne-Laure Sellier-Leclerc  1 , Ariane Zaloszyc  3 , Sacha Flammier  1 , Aurélie De Mul  1 , Rouba Bechara  3 , Julie Bernardor  4 , Cécile Acquaviva-Bourdain  5 , Justine Bacchetta  1   6   7
Affiliations

Fine-tuning circulating oxalate levels to improve transplant strategies in primary hyperoxaluria: what is the ideal threshold in pediatrics?

Marisca Carlina Makosso Afiavi et al. Nephrol Ther. .

Abstract
in English, French

Background: Interfering RNA therapies (RNAi) have changed the management of patients with hyperoxaluria type 1 (PH1); data in dialysis remain scarce.

Results: A PH1 teenager undergoing intensive hemodiafiltration received lumasiran. POx levels almost halved during the loading phase (98 to 52 µmol/L), but rebound occurred when doses were quarterly-spaced, with POx at 94 µmol/L at 5 months. Lumasiran injections were therefore performed monthly, allowing adequate POx control (52 µmol/L) and isolated kidney transplantation. We also evaluated POx in 26 non-PH1 children with current dialysis techniques at a median(range) age of 10.9 (2.6-17.0) years, time on dialysis 14 (0-52) months, and POx 35 (8-125) µmol/L; residual diuresis was associated with lower POx. Circulating glycolate levels were normal in non-PH1 patients.

Conclusion: Intensification of lumasiran therapy is possible in dialysis and improves POx levels before kidney transplantation; POx levels in non-PH1 pediatrics patients in dialysis are provided to improve decision making in transplantation.

Introduction: Les thérapies par ARN interférent (ARNi) ont révolutionné la prise en charge des patients atteints d’hyperoxalurie de type 1 (PH1) ; les données en dialyse restent rares.

Résultats: Une adolescente PH1 sous hémodiafiltration intensive a reçu du lumasiran : les POx ont diminué de moitié pendant la phase d’attaque (98 à 52 µmol/L), avec un rebond à l’espacement trimestriel (POx à 94 µmol/L à 5 mois), conduisant à un rapprochement mensuel des doses. Nous avons également mesuré les POx chez 26 enfants dialysés non-PH1 à un âge médian (intervalle) de 10,9 (2,6-17,0) ans, une durée de dialyse de 14 (0-52) mois, et une POx de 35 (8-125) µmol/L. La diurèse résiduelle était associée à des POx plus basses.

Conclusion: L’intensification du traitement par lumasiran est possible en dialyse et améliore les POx avant la transplantation rénale ; les POx chez les patients pédiatriques non-PH1 en dialyse vont améliorer les décisions thérapeutiques.

Keywords: primary hyperoxaluria; dialysis; lumasiran; therapeutic intensification.

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