Adrenocorticotropic hormone combined with vigabatrin as a second-line therapy for West syndrome

Abstract

Background

West syndrome is an epileptic encephalopathy for which combination therapies with adrenocorticotropic hormone and vigabatrin have emerged as new treatment options.

Objective

To evaluate the clinical and electroencephalographic remission rates, tolerability, and relapse rates in patients with West syndrome who failed primary treatment and underwent sequential therapy with vigabatrin and adrenocorticotropic hormone.

Methods

We included 39 patients with West syndrome from 2 specialized centers, aged 2 to 120 months. The patients were treated with intramuscular tetracosactide depot added to vigabatrin and were prospectively followed up for ≥ 1 year. The outcomes were clinical, and electroencephalographic remission rates at 7 and 30 days and 1 year following combined therapy initiation, progression to other epilepsy types, therapy tolerability, and relapse rates were recorded.

Results

Of the original sample, 71% of the subjects were boys, and 87% had a known etiology. The clinical and electroencephalographic remission rates were 46.1%, 94.8% (p = 0.001), and 74.1% (p = 0.01) at 7 and 30 days, and 1 year after the initiation of combined therapy, respectively. At the 1-year follow-up, adverse effects were observed in 86.0% and the relapse rate was 21.6%. After a median follow-up of 21 months, 73.6% of the patients developed epilepsy.

Conclusion

Combined therapy demonstrated a favorable efficacy profile in achieving clinical and electroencephalographic remission but was associated with significant seizure relapse rates in the medium term. Thus, it represents a feasible option for patients in whom initial treatment has failed.

Keywords

Spasms, Infantile - Adrenocorticotropic Hormone - Vigabatrin

Authors' Contributions

Conceptualization: LPS, SAA, ACSC; Data curation: LPS, DCBR, TST, SAA, ACSC; Formal analysis: LPS; Funding acquisition: LPS; Investigation: LPS; Methodology: LPS, TST, SAA, GLF, ACSC; Project administration: PS; Supervision: ACSC; Validation: GLF; Visualization: LPS; Writing – original draft: LPS, TST, SAA, ACSC; Writing – review & editing: LPS, DCBR, GLF, ACSC.

Data Availability Statement

All data generated or analyzed during the present study are included in this published article.

Editor-in-Chief: Hélio A. G. Teive; https://orcid.org/0000-0003-2305-1073.

Associate Editor: Lecio Figueira Pinto; https://orcid.org/0000-0002-2694-5120.

Publication History

Received: 31 August 2024

Accepted: 22 May 2025

Article published online:
20 August 2025

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