Efficacy and safety of empagliflozin in the treatment of glycogen storage disease-associated inflammatory bowel disease

Dan-Xia LIANG, Hao-Tian WU, Jing YANG, Min YANG

Chinese Journal of Contemporary Pediatrics ›› 2025, Vol. 27 ›› Issue (8) : 929-935.

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Chinese Journal of Contemporary Pediatrics ›› 2025, Vol. 27 ›› Issue (8) : 929-935. DOI: 10.7499/j.issn.1008-8830.2411030
CLINICAL RESEARCH

Efficacy and safety of empagliflozin in the treatment of glycogen storage disease-associated inflammatory bowel disease

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Abstract

Objective To investigate the efficacy and safety of empagliflozin in patients with glycogen storage disease (GSD)-associated inflammatory bowel disease (IBD). Methods A cross-sectional study was conducted, enrolling 25 patients with GSD-associated IBD who received empagliflozin treatment. General data, details of empagliflozin use, and adverse events were collected. Clinical symptoms and biochemical parameters before and after empagliflozin therapy were compared. Results Twenty-five patients with GSD-associated IBD were included, with a median age at diagnosis of 0.7 years, and a mean age at initiation of empagliflozin therapy of (11 ± 6) years. The initial dose of empagliflozin was (0.30 ± 0.13) mg/(kg·d), with a maintenance dose of (0.40 ± 0.21) mg/(kg·d), and a treatment duration of (34 ± 6) months. Seventy-eight percent (18/23) of patients' parents reported that empagliflozin therapy reduced the frequency of infections and oral ulcers, and increased neutrophil counts. Clinically, the number of patients with anorexia decreased from 12 to 5 after treatment, and 30% showed improved appetite (P<0.05). The numbers of patients with diarrhea, mucus/bloody stools, perianal disease, and oral ulcers decreased from 19, 9, 11, and 21 before treatment to 7, 1, 0, and 10 after treatment, respectively (P<0.05). Laboratory findings showed that absolute neutrophil counts increased, while platelet counts, lactate, and uric acid levels decreased significantly after empagliflozin treatment (P<0.05). Adverse reactions occurred in 7 patients (28%) during empagliflozin treatment. Two cases occurred in the treatment initiation phase, presenting as hypotension or profuse sweating with dehydration, along with urinary tract infections (UTIs); empagliflozin was discontinued in both cases. During the maintenance phase, 3 cases of UTIs and 2 cases of hypoglycemia (one with profuse sweating) were reported. Conclusions Empagliflozin therapy can increase neutrophil counts, reduce the incidence of infections and oral ulcers, alleviate diarrhea and abdominal pain, improve appetite, and ameliorate platelet count, lactate, and uric acid levels in patients with GSD-associated IBD, demonstrating significant clinical benefit. UTIs, hypoglycemia, hypotension, profuse sweating, and dehydration may be potential adverse reactions associated with empagliflozin therapy.

Key words

Glycogen storage disease-associated inflammatory bowel disease / Empagliflozin / Efficacy / Adverse event / Child

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Dan-Xia LIANG , Hao-Tian WU , Jing YANG , et al. Efficacy and safety of empagliflozin in the treatment of glycogen storage disease-associated inflammatory bowel disease[J]. Chinese Journal of Contemporary Pediatrics. 2025, 27(8): 929-935 https://doi.org/10.7499/j.issn.1008-8830.2411030

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