Year: 2024
Type: article
Study design: Observational Study
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: , 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.
Source: Kidney International Reports
Authors: Anne-Laure Sellier-Leclerc, Melissa Cloarec, Bertrand Knebelmann, Lise Allard, Olivia Boyer, Sylvie Cloarec, Claire Dossier, Moglie Le Quintrec, François Nobili, Thomas Stehlé, Isabelle Vrillon, Stéphane Burtey, Emilie Cornec-Le Gall, Marie Courbebaisse, Thierry Frouget, Arnaud Garnier, Thierry Krummel, Sandrine Lemoine, Catherine Monet-Didailler, Caroline Rousset-Rouvière, Amélie Ryckewaert, Adeline Schendel, Sacha Flammier, Cécile Acquaviva-Bourdain, Justine Bacchetta
Institutions: Hospices Civils de Lyon, Hôpital Necker-Enfants Malades, Université Paris Cité, Assistance Publique – Hôpitaux de Paris, Centre Hospitalier Universitaire de Bordeaux, Institut Necker Enfants Malades, Institut des Maladies Génétiques Imagine, Centre Hospitalier Universitaire de Tours, Hôpital Robert-Debré, Université de Montpellier, Centre Hospitalier Universitaire de Montpellier, Centre Hospitalier Universitaire de Besançon, Inserm, Université Paris-Est Créteil, Hôpitaux Universitaires Henri-Mondor, Institut Mondor de Recherche Biomédicale, Centre Hospitalier Régional et Universitaire de Nancy, Hôpital d'Enfants, Aix-Marseille Université, Institut National de Recherche pour l'Agriculture, l'Alimentation et l'Environnement, Hôpital de la Conception, Université de Bretagne Occidentale, Centre Hospitalier Régional Universitaire de Brest, Génétique, Génomique Fonctionnelle et Biotechnologies, Université de Bretagne Occidentale, Hôpital Européen Georges-Pompidou, Hôpital Pontchaillou, Centre Hospitalier Universitaire de Rennes, Centre hospitalier de Rouffach, Sociedad Chilena de Pediatria, Centre Hospitalier de la Côte Basque, Assistance Publique Hôpitaux de Marseille, Hôpital de la Timone
Language: English
Subfield: Pulmonary and Respiratory Medicine
Field: Medicine
Domain: Health Sciences
Open Access status: gold
Estimated APC paid: $2,800
OpenAlex ID: W4405905287