FC010: Multicentric Experience with RNA Interference Medication (OXLUMO®) in Patients with Primary Hyperoxaluria Type 1

Abstract BACKGROUND AND AIMS Primary hyperoxaluria type 1 (PH1) is a rare metabolic disorder of the glyoxylate metabolism in the liver inducing severe endogenous oxalate overproduction. Massive hyperoxaluria leads to recurrent urolithiasis and/or nephrocalcinosis and often early end-stage kidney fai...

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Published in:Nephrology, dialysis, transplantation dialysis, transplantation, 2022-05, Vol.37 (Supplement_3)
Main Authors: Martin Higueras, Cristina, Santana Estupiñan, Raquel, Torres Ramírez, Armando, Stefanidis, Constantinos, Walli, Adam, B. Beck, Bodo, Hoppe, Bernd
Format: Article
Language:English
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Summary:Abstract BACKGROUND AND AIMS Primary hyperoxaluria type 1 (PH1) is a rare metabolic disorder of the glyoxylate metabolism in the liver inducing severe endogenous oxalate overproduction. Massive hyperoxaluria leads to recurrent urolithiasis and/or nephrocalcinosis and often early end-stage kidney failure. Current treatment options were scarce until the first RNA interference drug, Oxlumo® (Lumasiran, Alnylam Pharmaceuticals, USA) can now be prescribed. Oxlumo® targets the mRNA of glycolate oxidase and, thus, prevents its translation. This reduces production of glyoxylate (i.e. the precursor to oxalate) and thus oxalate production. However, glycolate production increases. METHOD We herein report our current clinical experience with s.c. administration of Oxlumo adhering to provide dosage recommendation. A total of 25 PH1 patients were included. Seven patients were on haemodialysis, 1 patient on peritoneal dialysis and 17 were on stable kidney function at start of Oxlumo medication (eGFR > 30 and 70 mL/min in all others). The male: female ratio was 15:10, and the age range was 1–62 years of age at start of treatment. In both groups plasma oxalate (Pox) and glycolate (Pglyc) were determined before the first and every next Oxlumo dosage. In non-dialysis patients, urinary oxalate (Uox) and urinary glycolate in 24 h urines were analysed at same interval plus monthly collection until month 6 of treatment and thereafter in 6 weeks’ intervals, if Uox did not reach near normalisation, or had increased again at end of dosing interval (3 monthly). Oxalate and glycolate were determined using ion chromatography/mass spectrometry in the same lab. RESULTS We now report data of those patients, who have received at least four dosages of Oxlumo (n = 15). Non-dialysis patients: Uox was normal (
ISSN:0931-0509
1460-2385
DOI:10.1093/ndt/gfac096.001