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.

Download full-text PDF

Source
http://dx.doi.org/10.1053/j.ajkd.2022.12.005DOI Listing

Publication Analysis

Top Keywords

stiripentol lumasiran
8
oxalate nephropathy
8
primary hyperoxaluria
8
hyperoxaluria type
8
lumasiran rescue
4
rescue therapy
4
oxalate
4
therapy oxalate
4
nephropathy recurrence
4
recurrence kidney
4

Similar Publications

Article Synopsis
  • - Primary hyperoxalurias (PH) are rare genetic disorders that can lead to significant diagnostic delays, adversely affecting patient health outcomes.
  • - A study of 52 patients revealed that adults experienced much longer diagnostic delays (30 years on average) compared to children (1.2 years), with renal colic being the main symptom for adults.
  • - The findings highlight the need for greater awareness among healthcare professionals about these conditions to improve early detection and management, despite existing support structures for rare diseases.
View Article and Find Full Text PDF

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

July 2023

Department of Nephrology, Hôpital Européen Georges Pompidou, Assistance Publique-Hôpitaux de Paris, Paris, France; Université de Paris, Paris, France. Electronic address:

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.

View Article and Find Full Text PDF

New therapeutics for primary hyperoxaluria type 1.

Curr Opin Nephrol Hypertens

July 2022

Division of Nephrology and Hypertension.

Article Synopsis
  • Primary hyperoxaluria type 1 (PH1) is a rare genetic disorder leading to excess oxalate production, which can cause severe kidney issues.
  • Recent advancements in therapies, particularly RNA interference (RNAi) treatments like Lumasiran and Nedosiran, show promise in lowering oxalate levels in patients.
  • Other potential treatments and genetic editing tools are in development, aiming to reduce the necessity for combined kidney and liver transplants and improve patient outcomes.
View Article and Find Full Text PDF

Want AI Summaries of new PubMed Abstracts delivered to your In-box?

Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!