Therapeutic efficacy and adverse effects of adrenocorticotropic hormone therapy in west syndrome: Differences in dosage of adrenocorticotropic hormone, onset of age, and cause

ObjectivesTo determine the dosage and factors influencing efficacy of adrenocorticotropic hormone (ACTH) for West syndrome.Study designA retrospective study of 135 patients receiving ACTH therapy with a synthetic analogue for initial effect, seizure outcome 1 year after therapy, and adverse effects. Efficacy and adverse effects were compared among the groups divided by clinical factors: dosage, treatment lag, onset age, and cause.ResultsOne hundred thirteen patients had seizure control with ACTH. For more than 1 year after ACTH, 59 remained seizure free. Adverse effects were observed in 57, and ACTH therapy was discontinued in 23. The lowest dosage group (0.0125 mg/kg/d) had fewer episodes of discontinuation (P < .05), whereas differences in efficacy between different dosages were insignificant. None of the clinical factors correlated with initial effect. The earlier-onset group (<4 months) showed unfavorable seizure outcome 1 year after ACTH (P < .01). The cryptogenic patients showed better seizure outcome (P < .05) compared with the symptomatic.ConclusionSynthetic ACTH therapy at a lower dosage is as effective as natural ACTH therapy at a higher dosage. Considering the adverse effects and the benefits for seizure control, the ACTH dosage of 0.0125 mg/kg/d (synthetic analogue) is more favorable than larger dosage.