Kidney international reports · 2024
Real-Life Data of 2-Year Lumasiran Use in the DAILY-LUMA Cohort.
СтатьяНазвание и аннотация: Перевод пока не подготовлен
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Sellier-Leclerc Anne-Laure, Cloarec Melissa, Knebelmann Bertrand, Allard Lise, Boyer Olivia, Cloarec Sylvie, Dossier Claire, Le Quintrec Moglie, Nobili François, Stehlé Thomas, Vrillon Isabelle, Burtey Stéphane, Cornec-Le Gall Emilie, Courbebaisse Marie, Frouget Thierry, Garnier Arnaud, Krummel Thierry, Lemoine Sandrine, Monet-Didailler Catherine, Rousset-Rouvière Caroline, Ryckewaert Amélie, Schendel Adeline, Flammier Sacha, Acquaviva-Bourdain Cécile, Bacchetta Justine
Аннотация
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 (i.e., eGFR > 45 ml/min per 1.73 m2, age < 6 years), and 10 from DAILY-C (i.e., all ages, eGFR < 45 ml/min per 1.73 m2, 6 on dialysis). In DAILY-A and DAILY-B, decreased urinary oxalate-to-creatinine (UOx/creat) ratio, stable eGFR, and decrease in both nephrocalcinosis severity and stone numbers were observed, with a progressive tapering of conservative therapies. The decreased proportion of patients with nocturnal hydration and G-tubes overtime likely reflects improved quality of life. With a low number of patients - 2 patients on peritoneal dialysis and 3 patients with infantile oxalosis - the results are less conclusive for DAILY-C; however, in older patients, change in plasma oxalate (POx) levels is similar to previously published data. Tolerance was good with no severe side effects; injection site reactions, abdominal pain, and headaches were the main adverse events.
CONCLUSION: DAILY-LUMA is the largest cohort of patients receiving lumasiran in real life, confirming its safety and efficacy at 2 years.
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 (i.e., eGFR > 45 ml/min per 1.73 m2, age < 6 years), and 10 from DAILY-C (i.e., all ages, eGFR < 45 ml/min per 1.73 m2, 6 on dialysis). In DAILY-A and DAILY-B, decreased urinary oxalate-to-creatinine (UOx/creat) ratio, stable eGFR, and decrease in both nephrocalcinosis severity and stone numbers were observed, with a progressive tapering of conservative therapies. The decreased proportion of patients with nocturnal hydration and G-tubes overtime likely reflects improved quality of life. With a low number of patients - 2 patients on peritoneal dialysis and 3 patients with infantile oxalosis - the results are less conclusive for DAILY-C; however, in older patients, change in plasma oxalate (POx) levels is similar to previously published data. Tolerance was good with no severe side effects; injection site reactions, abdominal pain, and headaches were the main adverse events.
CONCLUSION: DAILY-LUMA is the largest cohort of patients receiving lumasiran in real life, confirming its safety and efficacy at 2 years.