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

OBJECTIVES: To determine the dosage and factors influencing efficacy of adrenocorticotropic hormone (ACTH) for West syndrome. STUDY DESIGN: A 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. RESULTS: One 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. CONCLUSION: Synthetic 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.