Endocrinological study of low-dose adrenocorticotropic hormone therapy without tapering in infantile epileptic spasms syndrome.

BACKGROUND

Adrenocorticotropic hormone (ACTH) therapy is effective for infantile epileptic spasms syndrome (IESS) and other types of refractory epilepsy; however, concerns remain regarding the risk of potential adrenal insufficiency, particularly when ACTH therapy is discontinued without dose tapering. Studies on comprehensive endocrinological evaluation of adrenocortical function following low-dose ACTH therapy without tapering are limited.

METHODS

We collected the data of patients who were diagnosed with IESS and received ACTH therapy using synthetic ACTH at 0.005-0.0125 mg/kg/day (equivalent to natural ACTH at 0.2-0.5 IU/kg/day) for 14 days without tapering, and underwent an ACTH stimulation test following ACTH therapy completion for adrenocortical function evaluation. Moreover, body weight, blood pressure, and electrolytes were measured before and after treatment.

RESULTS

A total of 11 patients, aged between 3 months and 1 year and 6 months, were enrolled. ACTH therapy was effective in all 11 patients. During the observation period, 3 patients developed seizure recurrence; however, none of the patients exhibited symptoms of adrenal insufficiency. The ACTH stimulation test confirmed normal adrenocortical function in all cases, with peak cortisol levels exceeding 20 μg/dL. After treatment, a significant increase in body weight and blood pressure were observed.

CONCLUSION

Low-dose ACTH therapy without tapering can be endocrinologically safe. The result provides useful information for reducing hospitalization duration and minimizing side effects. To validate these findings, further studies with larger cohorts are needed.