{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,19]],"date-time":"2026-05-19T21:09:47Z","timestamp":1779224987541,"version":"3.51.4"},"reference-count":18,"publisher":"Wiley","issue":"5","license":[{"start":{"date-parts":[[2026,2,28]],"date-time":"2026-02-28T00:00:00Z","timestamp":1772236800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"},{"start":{"date-parts":[[2026,2,28]],"date-time":"2026-02-28T00:00:00Z","timestamp":1772236800000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["J Intern Med"],"published-print":{"date-parts":[[2026,5]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Objective<\/jats:title>\n                    <jats:p>To compare the efficacy and safety of extended interval (Q3\u20134W) enzyme replacement therapy (ERT) versus standard biweekly (Q2W) ERT in clinically stable type 1 Gaucher disease (GD) patients.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>We emulated a target trial with a sequential trial design, using data from the French Gaucher Disease Registry. Eligible patients were treated for \u22652 years biweekly without clinical events. Every 3 months, switchers to Q3\u20134W were matched to Q2W patients by age, sex, referral center follow\u2010up, disease history (bone events, anemia, thrombocytopenia, splenectomy, and hepatosplenomegaly), and dose of ERT. The primary outcome was a composite of GD\u2010related events (bone events, anemia, and thrombocytopenia). A 10% non\u2010inferiority margin was prespecified. Secondary outcomes were biomarker changes and economic analyses.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Among 280 eligible GD patients, 63 switched to Q3\u20134W and were matched to a total of 215 Q2W patients, followed for an average of 6.3 years. No significant difference in the risk of clinical events was observed between groups (hazard ratio: 0.98 [95% confidence intervals (CI): 0.54\u20131.51]). During follow\u2010up, absolute risk difference remained below the 10% non\u2010inferiority threshold at all timepoints. Biomarkers remained stable or slightly decreased in the Q3\u20134W group. The dosing interval extension led to an average reduction of 55 infusions per patient, corresponding to approximately \u20ac450,000 saved per patient over 6 years.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion<\/jats:title>\n                    <jats:p>In stable GD1 patients, extending ERT administration to every 3\u20134 weeks was non\u2010inferior to the standard biweekly regimen, supporting personalized spacing strategies that may improve quality of life and reduce healthcare costs.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1111\/joim.70079","type":"journal-article","created":{"date-parts":[[2026,3,8]],"date-time":"2026-03-08T23:21:23Z","timestamp":1773012083000},"page":"604-614","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":1,"title":["Increased intervals in enzyme replacement therapy for stable type 1 Gaucher disease: A non\u2010inferiority sequential trial emulation"],"prefix":"10.1111","volume":"299","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-4924-093X","authenticated-orcid":false,"given":"Maxime","family":"Beydon","sequence":"first","affiliation":[{"name":"Service de M\u00e9decine Interne, Centre de r\u00e9f\u00e9rence des maladies lysosomales, Fili\u00e8re maladies h\u00e9r\u00e9ditaires du m\u00e9tabolisme, H\u00f4pital Beaujon, AP\u2010HP.Nord Universit\u00e9 Paris Cit\u00e9  Paris France"},{"name":"INSERM, Institut Pierre Louis d'Epid\u00e9miologie et de Sant\u00e9 Publique, Equipe PEPITES, AP\u2010HP, H\u00f4pital Piti\u00e9 Salp\u00eatri\u00e8re, D\u00e9partement de Sant\u00e9 Publique, Centre de Pharmaco\u00e9pid\u00e9miologie (Cephepi) Sorbonne Universit\u00e9  Paris France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"J\u00e9r\u00f4me","family":"Stirnemann","sequence":"additional","affiliation":[{"name":"Service de M\u00e9decine Interne G\u00e9n\u00e9rale H\u00f4pitaux Universitaires 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