Administration of lumasiran in a child with infantile oxalosis undergoing chronic peritoneal dialysis: A case report

Nephrologie & therapeutique Pub Date : 2025-06-11 DOI:10.1684/ndt.2025.121

Chloé Grosyeux, Arnaud Wiedemann, Marie-Christine Camoin-Schweitzer, Cécile Acquaviva-Bourdain, Noël Boussard, Justine Bacchetta, Isabelle Vrillon

{"title":"Administration of lumasiran in a child with infantile oxalosis undergoing chronic peritoneal dialysis: A case report","authors":"Chloé Grosyeux, Arnaud Wiedemann, Marie-Christine Camoin-Schweitzer, Cécile Acquaviva-Bourdain, Noël Boussard, Justine Bacchetta, Isabelle Vrillon","doi":"10.1684/ndt.2025.121","DOIUrl":null,"url":null,"abstract":"Background: Lumasiran is a small interfering RNA molecule indicated for the treatment of primary hyperoxaluria type 1 (PH1).Case diagnosis/treatment: We report a case of infantile PH1 in a 3-month-old girl admitted with anuria and kidney failure. Peritoneal dialysis (PD) and conservative treatment were immediately initiated. The first dose of lumasiran was administered at 12 months, while the patient was still on PD. Despite very high baseline plasma oxalate (POx) levels of around 200 µmol/L, treatment with lumasiran led to a nearly 50% reduction in POx levels, even during PD. Once the patient reached a sufficient body weight to safely initiate hemodialysis (HD), she was switched to HD; POx levels remained around 100 µmol/L, despite an intensive HD regimen.Conclusion: This is one of the first reported cases of lumasiran therapy during PD in an infant with PH1; even with this dialysis modality, POx levels can be decreased to a certain extent.","PeriodicalId":94153,"journal":{"name":"Nephrologie & therapeutique","volume":"21 2","pages":"1-4"},"PeriodicalIF":0.0000,"publicationDate":"2025-06-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Nephrologie & therapeutique","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1684/ndt.2025.121","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}

引用次数: 0

Abstract

Background: Lumasiran is a small interfering RNA molecule indicated for the treatment of primary hyperoxaluria type 1 (PH1).

Case diagnosis/treatment: We report a case of infantile PH1 in a 3-month-old girl admitted with anuria and kidney failure. Peritoneal dialysis (PD) and conservative treatment were immediately initiated. The first dose of lumasiran was administered at 12 months, while the patient was still on PD. Despite very high baseline plasma oxalate (POx) levels of around 200 µmol/L, treatment with lumasiran led to a nearly 50% reduction in POx levels, even during PD. Once the patient reached a sufficient body weight to safely initiate hemodialysis (HD), she was switched to HD; POx levels remained around 100 µmol/L, despite an intensive HD regimen.

Conclusion: This is one of the first reported cases of lumasiran therapy during PD in an infant with PH1; even with this dialysis modality, POx levels can be decreased to a certain extent.

查看原文本刊更多论文

鲁马西兰治疗慢性腹膜透析的草化病患儿1例

背景：Lumasiran是一种小干扰RNA分子，用于治疗原发性高草酸尿1型（PH1）。病例诊断/治疗：我们报告一例3个月大的女婴PH1无尿和肾衰竭入院。立即开始腹膜透析（PD）和保守治疗。第一次给药是在12个月时，当时患者仍在PD治疗。尽管基线血浆草酸（POx）水平非常高，约为200µmol/L，但lumasiran治疗导致POx水平降低了近50%，即使在PD期间也是如此。一旦患者达到足够的体重，可以安全地开始血液透析（HD），她就会切换到HD；尽管进行了强化HD治疗，但痘水平仍保持在100µmol/L左右。结论：这是首次报道的lumasiran治疗PH1婴儿PD的病例之一；即使采用这种透析方式，痘水平也能在一定程度上降低。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

求助全文

约1分钟内获得全文求助全文

来源期刊

Nephrologie & therapeutique

自引率

0.00%

发文量