ArticleNeurological research and practice2026
Treatment history as early indicators of short-term response to ACTH therapy in infantile epileptic spasms syndrome: a propensity score matching analysis.
Article in Neurological research and practice, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
backgroundAdrenocorticotropic hormone (ACTH) is a first-line therapy for infantile epileptic spasms syndrome (IESS), along with oral steroids and vigabatrin. the UKISS/ICISS trials have established the superior short-term efficacy of hormonal therapy over vigabatrin and the potential benefit of combination therapy. However, early identification of short-term responders to ACTH remains a clinical challenge. This study investigated whether treatment history, including pre-treatment exposure and on-treatment events, can serve as early indicators of short-term response using propensity score matching (PSM) analysis.
methodsIn this retrospective cohort study, we analyzed IESS patients who received ACTH therapy at Shanghai Children's Medical Center from March 2015 to February 2024. Responders were defined as those achieving ≥ 50% spasm reduction. PSM was applied using covariates with P < 0.20 in pre-matching analysis to balance baseline characteristics between responders and non-responders. Treatment history variables were compared using Chi-square or Fisher's exact tests. Variables with P < 0.20 in univariate analysis were included in a penalized logistic regression with L1 penalty (LASSO) to identify independent indicators.
resultsAmong 162 enrolled patients, 110 (55 responders, 55 non-responders) were matched with well-balanced baselines. Multivariate analysis using LASSO regression identified the addition of new antiseizure medications (ASMs) during ACTH therapy (OR = 0.055, 95% CI: 0.012-0.250, P < 0.001) and frequent ACTH dose adjustments (one adjustment: OR = 0.153, 95% CI: 0.030-0.780, P = 0.024; ≥2 adjustments: OR = 0.251, 95% CI: 0.086-0.731, P = 0.012) as having strong associations with lower odds of good short-term response, despite wide confidence intervals. Importantly, these factors likely represent markers of refractory disease rather than independent early predictors. Prior steroid/ACTH use showed a non-significant trend toward higher odds of good response (OR = 3.203, 95% CI: 0.794-12.923, P = 0.102).
conclusionsThe addition of new ASMs and frequent dose adjustments during ACTH therapy might reflect early clinical concern for poor response and should prompt reassessment of treatment strategy. Recognizing these signals may facilitate timely clinical intervention.
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