Recovery From Severe Heart Failure in a Patient With Primary Hyperoxaluria Type 1 After Treatment With Lumasiran, Pyridoxine, and Kidney Transplant

Abstract

Combined liver and kidney transplant has long been the only therapeutic option for patients with primary hyperoxaluria type 1 (PH1) and advanced chronic kidney disease. The development of lumasiran, a liver-directed RNA interference therapy, has led to effective reduction of hepatic oxalate production in patients with PH1. Despite reports of single cases successfully treated with isolated kidney transplant and lumasiran therapy, data are still scarce. Here, we report the case of a patient with PH1, severe cardiomyopathy, and chronic kidney disease, whose cardiac function improved remarkably after receiving an isolated kidney transplant and therapy with lumasiran and pyridoxine.