Empagliflozin in children with glycogen storage disease-associated inflammatory bowel disease: a prospective, single-arm, open-label clinical trial
Abstract Glycogen storage disease type Ib (GSD-Ib) is a rare inborn error of glycogen metabolism caused by mutations in SLC37A4. Patients with GSD-Ib are at high risk of developing inflammatory bowel disease (IBD). We evaluated the efficacy of empagliflozin, a renal sodium‒glucose cotransporter prot...
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| Format: | Article |
| Language: | English |
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Nature Portfolio
2024-04-01
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| Series: | Scientific Reports |
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| Online Access: | https://doi.org/10.1038/s41598-024-59320-z |
| _version_ | 1797199474825625600 |
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| author | Zhiling Li Xiaoyan Zhang Huan Chen Hanshi Zeng Jiaxing Wu Ying Wang Ni Ma Jiaoli Lan Yuxin Zhang Huilin Niu Lei Shang Xun Jiang Min Yang |
| author_facet | Zhiling Li Xiaoyan Zhang Huan Chen Hanshi Zeng Jiaxing Wu Ying Wang Ni Ma Jiaoli Lan Yuxin Zhang Huilin Niu Lei Shang Xun Jiang Min Yang |
| author_sort | Zhiling Li |
| collection | DOAJ |
| description | Abstract Glycogen storage disease type Ib (GSD-Ib) is a rare inborn error of glycogen metabolism caused by mutations in SLC37A4. Patients with GSD-Ib are at high risk of developing inflammatory bowel disease (IBD). We evaluated the efficacy of empagliflozin, a renal sodium‒glucose 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 ± 2.61 years. 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 weeks 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. |
| first_indexed | 2024-04-24T07:16:20Z |
| format | Article |
| id | doaj.art-74cbae30b7d046bd812460a9cbe3b10e |
| institution | Directory Open Access Journal |
| issn | 2045-2322 |
| language | English |
| last_indexed | 2024-04-24T07:16:20Z |
| publishDate | 2024-04-01 |
| publisher | Nature Portfolio |
| record_format | Article |
| series | Scientific Reports |
| spelling | doaj.art-74cbae30b7d046bd812460a9cbe3b10e2024-04-21T11:18:22ZengNature PortfolioScientific Reports2045-23222024-04-0114111010.1038/s41598-024-59320-zEmpagliflozin in children with glycogen storage disease-associated inflammatory bowel disease: a prospective, single-arm, open-label clinical trialZhiling Li0Xiaoyan Zhang1Huan Chen2Hanshi Zeng3Jiaxing Wu4Ying Wang5Ni Ma6Jiaoli Lan7Yuxin Zhang8Huilin Niu9Lei Shang10Xun Jiang11Min Yang12Department of Pediatrics, Guangdong Provincial People’s Hospital, Guangdong Academy of Medical Sciences, Southern Medical UniversityDepartment of Pediatrics, Guangdong Provincial People’s Hospital, Guangdong Academy of Medical Sciences, Southern Medical UniversityDepartment of Gastroenterology, Guangzhou Women and Children’s Medical Center, Guangzhou Medical UniversityDepartment of Pediatrics, Guangdong Provincial People’s Hospital, Guangdong Academy of Medical Sciences, Southern Medical UniversityDepartment of Pediatrics, Guangdong Provincial People’s Hospital, Guangdong Academy of Medical Sciences, Southern Medical UniversityDepartment of Pediatrics, Guangdong Provincial People’s Hospital, Guangdong Academy of Medical Sciences, Southern Medical UniversityDepartment of Pediatrics, Guangdong Provincial People’s Hospital, Guangdong Academy of Medical Sciences, Southern Medical UniversityDepartment of Pediatrics, Guangdong Provincial People’s Hospital, Guangdong Academy of Medical Sciences, Southern Medical UniversityDepartment of Pediatrics, Guangdong Provincial People’s Hospital, Guangdong Academy of Medical Sciences, Southern Medical UniversityDepartment of Pathology, Guangdong Provincial People’s Hospital, Guangdong Academy of Medical Sciences, Southern Medical UniversityDepartment of Health Statistics, School of Public Health, Fourth Military Medical UniversityDepartment of Pediatrics, The Second Affiliated Hospital, Fourth Military Medical UniversityDepartment of Pediatrics, Guangdong Provincial People’s Hospital, Guangdong Academy of Medical Sciences, Southern Medical UniversityAbstract Glycogen storage disease type Ib (GSD-Ib) is a rare inborn error of glycogen metabolism caused by mutations in SLC37A4. Patients with GSD-Ib are at high risk of developing inflammatory bowel disease (IBD). We evaluated the efficacy of empagliflozin, a renal sodium‒glucose 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 ± 2.61 years. 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 weeks 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.https://doi.org/10.1038/s41598-024-59320-zEmpagliflozinGlycogen storage diseaseInflammatory bowel diseaseClinical trialChildren |
| spellingShingle | Zhiling Li Xiaoyan Zhang Huan Chen Hanshi Zeng Jiaxing Wu Ying Wang Ni Ma Jiaoli Lan Yuxin Zhang Huilin Niu Lei Shang Xun Jiang Min Yang Empagliflozin in children with glycogen storage disease-associated inflammatory bowel disease: a prospective, single-arm, open-label clinical trial Scientific Reports Empagliflozin Glycogen storage disease Inflammatory bowel disease Clinical trial Children |
| title | Empagliflozin in children with glycogen storage disease-associated inflammatory bowel disease: a prospective, single-arm, open-label clinical trial |
| title_full | Empagliflozin in children with glycogen storage disease-associated inflammatory bowel disease: a prospective, single-arm, open-label clinical trial |
| title_fullStr | Empagliflozin in children with glycogen storage disease-associated inflammatory bowel disease: a prospective, single-arm, open-label clinical trial |
| title_full_unstemmed | Empagliflozin in children with glycogen storage disease-associated inflammatory bowel disease: a prospective, single-arm, open-label clinical trial |
| title_short | Empagliflozin in children with glycogen storage disease-associated inflammatory bowel disease: a prospective, single-arm, open-label clinical trial |
| title_sort | empagliflozin in children with glycogen storage disease associated inflammatory bowel disease a prospective single arm open label clinical trial |
| topic | Empagliflozin Glycogen storage disease Inflammatory bowel disease Clinical trial Children |
| url | https://doi.org/10.1038/s41598-024-59320-z |
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