Real-Life Data of 2-Year Lumasiran Use in the DAILY-LUMA Cohort.

ABSTRACT:

Introduction

Lumasiran is a drug used in RNA-interference (RNAi) therapy for primary hyperoxaluria type 1 (PH1). Data on its efficacy and safety mainly come from industry-sponsored trials.

Methods

For postmarketing follow-up, French authorities requested a quasi-exhaustive retrospective and prospective study over 5 years for patients receiving lumasiran, requiring the inclusion of at least 90% of patients, named as the DAILY-LUMA cohort (NCT06225882). Here, we analyzed data from all patients who were not previously included in the industry-sponsored trials and had received lumasiran for at least 2 years.

Results

We included 38 patients, 22 from DAILY-A (i.e., estimated glomerular filtration rate (eGFR) > 45 ml/min per 1.73 m², age ≥ 6 years), 6 from DAILY-B

SUBMITTER: Sellier-Leclerc AL 

PROVIDER: S-EPMC12034872 | biostudies-literature | 2025 Apr

REPOSITORIES: biostudies-literature