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Background <p>Adrenocorticotropic 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.</p> Methods <p>In 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 <i>P</i> &lt; 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 <i>P</i> &lt; 0.20 in univariate analysis were included in a penalized logistic regression with L1 penalty (LASSO) to identify independent indicators.</p> Results <p>Among 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, <i>P</i> &lt; 0.001) and frequent ACTH dose adjustments (one adjustment: OR = 0.153, 95% CI: 0.030–0.780, <i>P</i> = 0.024; ≥2 adjustments: OR = 0.251, 95% CI: 0.086–0.731, <i>P</i> = 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, <i>P</i> = 0.102).</p> Conclusions <p>The 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.</p>

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Treatment history as early indicators of short-term response to ACTH therapy in infantile epileptic spasms syndrome: a propensity score matching analysis

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2026年7月14日

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  • Kana Lin,
  • Guangyun Zhang,
  • Yiran Zhou,
  • Cuijin Wang,
  • Feng Han,
  • Li Liu,
  • Jie Liu,
  • Changhua Mou,
  • Yunqing Zhou,
  • Jiwen Wang,
  • Dawei Yuan,
  • Yingyan Wang

摘要

Background

Adrenocorticotropic 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.

Methods

In 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.

Results

Among 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).

Conclusions

The 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.