Nedosiran Dramatically Reduces Serum Oxalate in Dialysis-Dependent Primary Hyperoxaluria 1: A Compassionate Use Case Report.
Journal
Urology
ISSN: 1527-9995
Titre abrégé: Urology
Pays: United States
ID NLM: 0366151
Informations de publication
Date de publication:
Oct 2021
Oct 2021
Historique:
received:
25
01
2021
revised:
09
03
2021
accepted:
14
03
2021
pubmed:
29
3
2021
medline:
4
3
2022
entrez:
28
3
2021
Statut:
ppublish
Résumé
Primary hyperoxaluria 1 (PH1) is a devastating condition involving recurrent urolithiasis, early end-stage renal disease and multisystemic deposition of calcium oxalate crystals. Treatment options for PH1 are limited, inevitably requiring transplantation, usually combined kidney and liver transplant. Here we report successful compassionate use of Nedosiran, an RNA interference targeting lactate dehydrogenase, in an index patient. Monthly Nedosiran injections led to dramatically decreased plasma oxalate levels, decreased frequency of weekly hemodialysis sessions from 6 to 3, and deferral of combined kidney and liver transplant. Nedosiran represents a novel and impactful potential therapeutic for PH1 patients with end-stage renal disease.
Identifiants
pubmed: 33774044
pii: S0090-4295(21)00270-3
doi: 10.1016/j.urology.2021.03.014
pii:
doi:
Substances chimiques
Oxalates
0
nedosiran
0
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
e147-e149Informations de copyright
Copyright © 2021 Elsevier Inc. All rights reserved.