Efficacy of ACTH therapy in treating infantile epileptic spasm syndrome and its effect on Kramer score.

Objective: To compare the clinical efficacy and safety of adrenocorticotropic hormone (ACTH) combined with topiramate versus topiramate monotherapy in treating Infantile Epileptic Spasm Syndrome (IESS), and to explore the risk factors for relapse.

Methods: This retrospective cohort study included 185 IESS pediatric patients treated at Yan'an People's Hospital between April 2018 and January 2024. Patients were divided into a control group (n=95, topiramate monotherapy) and an observation group (n=90, ACTH combined with topiramate) based on their treatment regimen. Propensity score matching (PSM) was employed to balance baseline differences. Outcomes compared between the groups included spasm control rates, electroencephalogram (EEG) improvement, changes in functional scores, and incidence of adverse reaction. Multivariate logistic regression was used to identify independent risk factors for relapses.

Results: The observation group demonstrated significantly higher rates of complete spasm control (75.56% vs. 50.53%) and complete EEG improvement (76.67% vs. 54.74%) compared to the control group (P<0.01). Post-treatment, the observation group showed more significant improvements in developmental quotient (DQ), motor index (MI), and Kramer scores (P<0.01). Multivariate analysis identified structural etiology (OR=3.12), monotherapy (OR=2.54), and higher Kramer scores (OR=1.45) as independent risk factors for relapse (all P<0.01). The incidence of adverse reactions did not differ significantly between groups (34.44% vs. 29.47%, P=0.468).

Conclusion: ACTH combined with topiramate offers superior efficacy for spasm control, EEG improvement, and neurological function recovery compared to topiramate monotherapy, without increasing adverse reactions. Structural etiology, monotherapy, and Kramer scores are independent predictors for relapse.