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Patients with GSD-Ib are at high risk of developing inflammatory bowel disease (IBD). We evaluated the efficacy of empagliflozin, a renal sodium\u2012glucose cotransporter protein 2 (SGLT2) inhibitor, on colonic mucosal healing in patients with GSD-associated IBD. A prospective, single-arm, open-label clinical trial enrolled eight patients with GSD-associated IBD from Guangdong Provincial People's Hospital in China from July 1, 2022 through December 31, 2023. Eight patients were enrolled with a mean age of 10.34\u2009\u00b1\u20092.61\u00a0years. Four male and four female. The endoscopic features included deep and large circular ulcers, inflammatory hyperplasia, obstruction and stenosis. The SES-CD score significantly decreased at week 48 compared with before empagliflozin. Six patients completed 48\u00a0weeks of empagliflozin therapy and endoscopy showed significant improvement or healing of mucosal ulcers, inflammatory hyperplasia, stenosis, and obstruction. One patient had severe sweating that required rehydration and developed a urinary tract infection. No serious or life-threatening adverse events. This study suggested that empagliflozin may promote colonic mucosal healing and reduce hyperplasia, stenosis, and obstruction in children with GSD-associated IBD.<\/jats:p>","DOI":"10.1038\/s41598-024-59320-z","type":"journal-article","created":{"date-parts":[[2024,4,16]],"date-time":"2024-04-16T06:43:15Z","timestamp":1713249795000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":8,"title":["Empagliflozin in children with glycogen storage disease-associated inflammatory bowel disease: a prospective, single-arm, open-label clinical trial"],"prefix":"10.1038","volume":"14","author":[{"given":"Zhiling","family":"Li","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xiaoyan","family":"Zhang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Huan","family":"Chen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hanshi","family":"Zeng","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jiaxing","family":"Wu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ying","family":"Wang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ni","family":"Ma","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jiaoli","family":"Lan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yuxin","family":"Zhang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Huilin","family":"Niu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lei","family":"Shang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xun","family":"Jiang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Min","family":"Yang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2024,4,15]]},"reference":[{"issue":"6","key":"59320_CR1","doi-asserted-by":"publisher","first-page":"1007","DOI":"10.1007\/s10545-018-0180-5","volume":"41","author":"JY Chou","year":"2018","unstructured":"Chou, J. Y. et al. Molecular biology and gene therapy for glycogen storage disease type Ib. J. Inherit. Metab. Dis. 41(6), 1007\u20131014 (2018).","journal-title":"J. Inherit. Metab. Dis."},{"issue":"12","key":"59320_CR2","doi-asserted-by":"publisher","first-page":"676","DOI":"10.1038\/nrendo.2010.189","volume":"6","author":"JY Chou","year":"2010","unstructured":"Chou, J. Y., Jun, H. S. & Mansfield, B. C. Glycogen storage disease type I and G6Pase-\u03b2 deficiency: Etiology and therapy. Nat. Rev. Endocrinol. 6(12), 676\u2013688 (2010).","journal-title":"Nat. Rev. Endocrinol."},{"issue":"3","key":"59320_CR3","doi-asserted-by":"publisher","first-page":"511","DOI":"10.1007\/s10545-014-9772-x","volume":"38","author":"JY Chou","year":"2015","unstructured":"Chou, J. Y., Jun, H. S. & Mansfield, B. C. Type I glycogen storage diseases: disorders of the glucose-6-phosphatase\/glucose-6-phosphate transporter complexes. J. Inherit. Metab Dis. 38(3), 511\u2013519 (2015).","journal-title":"J. Inherit. 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