Maxime Beydon, Jérôme Stirnemann, K. Yousfi, Samira Zebiche, Dalil Hamroun, A. Brassier, S. Pichard, L. Swiader, T. D. de Villemeur, Bénédicte Héron, Florence Dalbiès, Bérengère Cador, Anne-Sophie Guemann, Francis Gaches, B. Hivert, V. Leguy-Seguin, Agathe Masseau, Y. Pers, M. Pettazzoni, S. Bekri, Catherine Caillaud, Edouard Le Guillou, Marie Szymanowski, Leonardo Astudillo, Wladimir Mauhin, Yann Nadjar, Christine Serratrice, Marc G. Berger, Fabrice Camou, N. Belmatoug, Yann Nguyen
2026.2.28JOURNAL OF INTERNAL MEDICINE
tlooto Summary
In stable GD1 patients, extending ERT administration to every 3-4 weeks was non-inferior to the standard biweekly regimen, supporting personalized spacing strategies that may improve quality of life and reduce healthcare costs.
Abstract
To compare the efficacy and safety of extended interval (Q3–4W) enzyme replacement therapy (ERT) versus standard biweekly (Q2W) ERT in clinically stable type 1 Gaucher disease (GD) patients.
Citation format
BEYDON, Maxime, et al. Increased intervals in enzyme replacement therapy for stable type 1 gaucher disease: A non‐inferiority sequential trial emulation. JOURNAL OF INTERNAL MEDICINE, 2026, 299(5): 604–614.