The
primary hyperoxalurias (PHs) are a group of diseases characterized by
kidney stones,
nephrocalcinosis, and
chronic kidney disease. At stages of advanced
kidney disease, glomerular filtration of
oxalate becomes insufficient, plasma levels increase, and tissue deposition may occur.
Hemodialysis is often unable to overcome the excess hepatic
oxalate production. The current surgical management of
primary hyperoxaluria type 1 (PH1) is combined liver
kidney transplantation. In a subset of PH1 patients who respond to
pyridoxine, kidney-only
transplantation has been successfully performed. Recently, kidney-only
transplantation has also been performed in PH1 patients receiving a
small interfering RNA therapy called
lumasiran. This
drug targets the hepatic overproduction of
oxalate, making kidney-only
transplantation a potentially practical novel approach for managing PH1 patients with advanced
kidney disease. It is unknown if similar effects could be seen with a different
small interfering RNA agent called
nedosiran. This article will briefly review PH1, describe the
small interfering RNA therapies being used to treat PH, summarize the reported cases of kidney-only
transplantation performed with
lumasiran, and detail a case of kidney-only
transplantation performed in a PH1 patient receiving
nedosiran.