Lumasiran PH1 RNA Interference — ESENeph MCQ

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HardRenal Stone DiseaseLumasiran PH1 RNA InterferenceESENeph

A 32-year-old man with recurrent calcium oxalate kidney stones is found to have primary hyperoxaluria type 1 on genetic testing (AGXT mutation). His eGFR is 55 mL/min/1.73m2. What novel RNA interference therapy is available for PH1?

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Correct answer: D — Lumasiran (targets glycolate oxidase via RNA interference)

Lumasiran is an RNA interference (siRNA) therapy that targets hepatic glycolate oxidase, reducing oxalate production upstream of the AGXT enzyme defect. NICE HST10 (2021) recommends Lumasiran for PH1 patients of all ages. The ILLUMINATE-A and ILLUMINATE-B trials showed dramatic reductions in urinary oxalate. It is administered subcutaneously monthly for 3 doses then quarterly. While it does not replace the need for supportive care, it significantly reduces disease burden and may delay or prevent the need for liver-kidney transplant.

Reference: NICE HST10 2021 – Lumasiran; ILLUMINATE-A Trial – NEJM 2021