Stiripentol and Lumasiran as a Rescue Therapy for Oxalate Nephropathy Recurrence After Kidney Transplantation in an Adult Patient With Primary Hyperoxaluria Type 1

Yannis Lombardi; Pierre Isnard; Nathalie Chavarot; Sophie Chauvet; Frank Martinez; Éric Thervet; Dany Anglicheau; Alexandre Karras

doi:10.1053/j.ajkd.2022.12.005

Stiripentol and Lumasiran as a Rescue Therapy for Oxalate Nephropathy Recurrence After Kidney Transplantation in an Adult Patient With Primary Hyperoxaluria Type 1

Am J Kidney Dis. 2023 Jul;82(1):113-116. doi: 10.1053/j.ajkd.2022.12.005. Epub 2023 Jan 21.

Authors

Yannis Lombardi¹, Pierre Isnard², Nathalie Chavarot³, Sophie Chauvet⁴, Frank Martinez⁵, Éric Thervet⁴, Dany Anglicheau³, Alexandre Karras⁶

Affiliations

¹ Department of Nephrology, Hôpital Européen Georges Pompidou, Assistance Publique-Hôpitaux de Paris, Paris, France; Sorbonne Université, Paris, France.
² Department of Pathology, Hôpital Necker, Assistance Publique-Hôpitaux de Paris, Paris, France; Université de Paris, Paris, France.
³ Department of Kidney Transplantation, Hôpital Necker, Assistance Publique-Hôpitaux de Paris, Paris, France; Université de Paris, Paris, France.
⁴ Department of Nephrology, Hôpital Européen Georges Pompidou, Assistance Publique-Hôpitaux de Paris, Paris, France; Université de Paris, Paris, France.
⁵ Department of Kidney Transplantation, Hôpital Necker, Assistance Publique-Hôpitaux de Paris, Paris, France.
⁶ Department of Nephrology, Hôpital Européen Georges Pompidou, Assistance Publique-Hôpitaux de Paris, Paris, France; Université de Paris, Paris, France. Electronic address: [email protected].

PMID: 36693470
DOI: 10.1053/j.ajkd.2022.12.005

Abstract

Primary hyperoxaluria type 1 is a rare cause of kidney failure. Stiripentol, an inhibitor of lactate dehydrogenase A, and lumasiran, a small interfering RNA targeting glycolate oxidase, have been proposed as therapeutic options, but clinical data are scarce, especially in adults and transplanted patients. We describe the case of a 51-year-old patient with a biopsy-proven recurrence of oxalate nephropathy after a kidney-only transplantation. He received stiripentol and lumasiran without adverse events. Fourteen months after transplantation, graft function, serum, and urinary oxalate levels have remained stable, and kidney biopsy showed a complete regression of oxalate crystals. Further studies are needed to assess whether this strategy is effective and could replace liver-kidney transplantation.

Keywords: primary hyperoxaluria, kidney transplantation, recurrence, lumasiran.

Publication types

Case Reports

MeSH terms

Adult
Humans
Hyperoxaluria* / etiology
Hyperoxaluria, Primary* / complications
Hyperoxaluria, Primary* / surgery
Kidney Transplantation* / adverse effects
Male
Middle Aged
Oxalates
RNA, Small Interfering
Renal Insufficiency* / etiology

Substances

stiripentol
lumasiran
RNA, Small Interfering
Oxalates

Supplementary concepts

Primary hyperoxaluria type 1