Fighting the epidemic of pediatric metabolic dysfunction-associated steatotic liver disease: Role of non-invasive diagnostics and early pharmacological intervention

Table 2 Summary of studies on pharmacotherapy for pediatric metabolic dysfunction-associated steatotic liver disease

Medication class	Study design & population	Primary endpoint	Efficacy summary	Safety/adverse events	Ref.
GLP-1RA (liraglutide, semaglutide)	Retrospective cohort, n = 42, age ≤ 18 years, MASLD diagnosis, GLP-1RA prescribed for obesity or T2DM[1]	ALT reduction at 6 months and end-of-treatment	Significant mean ALT reduction (-56 U/L at 6 months, -37 U/L at EOT, P < 0.05); greater effect in T2DM; improvements in AST, GGT, HbA1c, triglycerides; BMI stabilization, not reduction	Mild-moderate GI symptoms (nausea, vomiting, diarrhea); no serious adverse events reported	Tou and Panganiban[72], 2025
Metformin	Meta-analysis	ALT reduction	No significant improvement in ALT	GI upset, rare lactic acidosis	Gkiourtzis et al[62], 2023
Lifestyle/standard of care	Longitudinal cohort, n = 440, pediatric MASLD	Composite improvement (ALT, GGT, histology)	22% improved at 1 year, 31% at 2 years; 20% worsened; BMI and cholesterol changes most associated with outcomes	Not applicable	Newton et al[82], 2025
Resmetirom	No pediatric studies to date

GLP-1RA: Glucagon-like peptide 1 receptor agonists; T2DM: Type 2 diabetes mellitus; ALT: Alanine aminotransferase; GGT: Gamma glutamyl transferase; HbA1c: Glycated hemoglobin; BMI: Body mass index; GI: Gastrointestinal; MASLD: Metabolic dysfunction-associated steatotic liver disease.