Jiang J J, Ma M S, Wei M, Qiu Z Q
Department of Pediatrics, Peking Union Medical College Hospital, Peking Union Medical College, Chinese Academy of Medical Sciences, Beijing 100730, China.
Zhonghua Er Ke Za Zhi. 2024 Jun 2;62(6):526-529. doi: 10.3760/cma.j.cn112140-20240301-00136.
To investigate the safety, efficacy and effective dose of empagliflozin in the treatment of glycogen storage disease type Ⅰb (GSD Ⅰb). This was a cross sectional study. A total of 28 children with GSDⅠb who started oral empagliflozin treatment from January 2021 to June 2023 in the WeChat group of patients with glycogen storage disease were selected as the study objects. Clinical data such as general situation, current situation of medication and adverse reactions of the children were collected through questionnaires from June 18 to 30, 2023. The differences of symptoms and laboratory tests before and after empagliflozin treatment were compared by using paired chi-square test and Wilcoxon signed rank sum test. Totally 28 children with GSD Ⅰb were from 12 different provinces, autonomous regions and municipalities in China. There were 14 males and 14 females. Empagliflozin treatment was started at the age of 4.8 (2.4, 10.8) years, the time of treatment was 14.5 (11.3, 21.5) months, the initial dosage was (0.23±0.11) mg/(kg·d), and the maintenance dosage was (0.28±0.12) mg/(kg·d). Empagliflozin showed positive effects on neutropenia, severity of inflammatory bowel disease like symptoms(=-3.702.65, both <0.05), The proportion of recurrent oral ulcers, recurrent bacterial infections and anemia was significantly lower than that before medication (18% (5/28) 46% (13/28), 14% (4/28) 46% (13/28), 21% (6/28) 46% (13/28), ²=4.05, 5.26, 3.05 all <0.05). Granulocyte colony-stimulating factor (GCSF) was once used in 5 children with GSD Ⅰb, all of them had completely stopped GCSF after empagliflozin treatment. The most common adverse events during empagliflozin treatment were hypoglycemia (5 children) and urinary infection (3 children). All 28 patients had no serious adverse reactions. Empagliflozin can increase the neutrophil count of children with GSD Ⅰb, and had a favorable effect on symptoms such as recurrent oral ulcers, and recurrent infection. The common adverse events during empagliflozin treatment were hypoglycemia and urinary infection.
探讨恩格列净治疗Ⅰb型糖原贮积病（GSDⅠb）的安全性、有效性及有效剂量。这是一项横断面研究。选取2021年1月至2023年6月在糖原贮积病患者微信群中开始口服恩格列净治疗的28例GSDⅠb患儿作为研究对象。于2023年6月18日至30日通过问卷调查收集患儿的一般情况、用药现状及不良反应等临床资料。采用配对卡方检验和Wilcoxon符号秩和检验比较恩格列净治疗前后症状及实验室检查的差异。28例GSDⅠb患儿来自中国12个不同的省、自治区、直辖市。男14例，女14例。恩格列净治疗起始年龄为4.8（2.4，10.8）岁，治疗时间为14.5（11.3，21.5）个月，初始剂量为（0.23±0.11）mg/（kg·d），维持剂量为（0.28±0.12）mg/（kg·d）。恩格列净对中性粒细胞减少症、类炎性肠病症状严重程度有积极作用（Z=-3.70，P=0.002，均<0.05），复发性口腔溃疡、复发性细菌感染及贫血的比例均显著低于用药前（18%（5/28）对46%（13/28），14%（4/28）对46%（13/28），21%（6/28）对46%（13/28），χ²=4.05、5.26、3.05，均<0.05）。5例GSDⅠb患儿曾使用过粒细胞集落刺激因子（GCSF），恩格列净治疗后均已完全停用GCSF。恩格列净治疗期间最常见的不良事件为低血糖（5例患儿）和泌尿系统感染（3例患儿）。28例患者均无严重不良反应。恩格列净可提高GSDⅠb患儿的中性粒细胞计数，对复发性口腔溃疡、复发性感染等症状有良好疗效。恩格列净治疗期间常见的不良事件为低血糖和泌尿系统感染。
