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Primary Hyperoxaluria

Lumasiran effective in reducing hepatic oxalate production in primary hyperoxaluria type 1

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Lumasiran, an RNA interference medication, shows promise in reducing hepatic oxalate production in patients with primary hyperoxaluria type 1 (PH1), according to a study that indicates that lumasiran treatment is safe and efficient, with significant reductions in urine oxalate levels and potential improvements in renal function among patients with preserved kidney function.

The study, which involved 33 patients with PH1, including 20 with preserved kidney function and 13 on dialysis, analyzed data over a median treatment period of 18 months.

However, the study highlighted the importance of considering individual factors such as concurrent vitamin B6 (VB6) medication, as Uox levels differed accordingly. Additionally, while plasma oxalate (Pox) remained stable over time, nephrolithiasis persisted in some patients, albeit at lower rates, while nephrocalcinosis showed mixed outcomes.

Among patients on dialysis, lumasiran demonstrated effectiveness in reducing plasma oxalate (Pox) levels, particularly with monthly dosing. However, there were challenges in maintaining consistent reductions with quarterly dosing, suggesting a need for further optimization in this population.

The study also raised concerns about potential metabolic changes associated with lumasiran treatment, including increased urinary and plasma glycolate levels and decreased urine citrate, necessitating adjustments in alkali medication.

Reference
Martin-Higueras C, Borghese L, Torres A, et al. Multicenter Long-Term Real World Data on Treatment With Lumasiran in Patients With Primary Hyperoxaluria Type 1. Kidney Int Rep. 2023;9(1):114-133. doi: 10.1016/j.ekir.2023.10.004. PMID: 38312792; PMCID: PMC10831356.

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