Lumasiran in primary hyperoxaluria type 1: 73% responded at 3 months, more often in children

Design

Ongoing multicentre real-world cohort (Daily-Luma, France)

Population

76 patients with primary hyperoxaluria type 1, 54 children, 24 on dialysis

Primary outcome

Proportion reaching oxalate response criterion at 3 months

Effect

73% met the criterion; no significant genotype association; best response in children

Lumasiran is an RNA interference therapy for primary hyperoxaluria type 1. This analysis used the ongoing French Daily-Luma cohort of real-world use, with 76 patients (54 children), a median age of 13 at starting, and 24 on dialysis. The response criterion was normalisation or a fall of over 30% in urinary oxalate-to-creatinine ratio, or in plasma oxalate when eGFR was under 30.

At 3 months, 73% of patients met the criterion, using available data. Genotype, grouped by vitamin B6 response, was not significantly associated with response, though there was a trend to weaker response in vitamin B6-sensitive forms. Younger age at diagnosis and at starting lumasiran was associated with better response, with the best results in children.

The authors suggest continuing or starting pyridoxine in vitamin B6-sensitive patients even after lumasiran begins, and close follow-up with re-evaluation. This is a small cohort without a comparison group, so the pyridoxine suggestion is an opinion.

• Most patients (73%) reached the oxalate response criterion by 3 months.
• Response was best in children; age at starting predicted response.
• Genotype did not significantly predict response in this cohort.
• Consider keeping pyridoxine in vitamin B6-sensitive forms, as the authors suggest, and re-evaluate regularly.
• Availability and cost of lumasiran vary; check local access.

Why it matters

It starts to explain why a transformative therapy works better in some patients than others.

Don't overread it

A 76-patient real-world cohort with no controls; the genotype comparison is underpowered.

The statistics, in plain English

A response rate of 73% is calculated on patients with data available at 3 months, so missing data could shift the figure. With no comparison group, the cohort shows what happened to treated patients, not what an untreated group would have done.

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