Abstracts

RATIONALE: Most Japanese pediatric neurologists attempt other treatments before prescribing ACTH therapy for West Syndrome (WS), and then, when necessary, treat patients with only low-dose synthetic ACTH to avoid serious adverse effects. In this multi-institutional study, we analyzed the initial effects, adverse effects and long-term outcome in patients treated with low-dose ACTH in Japan.
METHODS: We analyzed the medical records of 138 patients with WS, who were treated with low-dose ACTH therapy between 1989 and 1998.
RESULTS: At the end of ACTH therapy, excellent effect on seizures was noted in 106 out of 138 (76%) patients, good effect in 23 (17%), and poor effect in 9 (7%). Initial effects on EEG were excellent in 53 out of 138 (38%) patients, good in 76 (55%) and poor in 9 (7%). As for seizure prognosis at the time of follow-up, 51 out of 99 (52%) patients were seizure-free, and 48 (48%) patients had seizures. Mental outcome was normal in 6 out of 98 (6%) patients, mild MR in 16 (16%), moderate MR in 26 (27%) and severe MR in 50 (51%). The initial effects of ACTH on seizures and long-term outcome were not dose-dependent (daily dosage; 0.005-0.032 mg/kg, 0.2-1.28 IU/kg, total dosage; 0.1-0.87 mg/kg, 4-34.8 IU/kg). The severity of adverse effects correlated with total dosage of ACTH and the severity of brain shrinkage due to ACTH correlated well with the daily dosage and total dosage of ACTH.
CONCLUSIONS: Low-dose ACTH therapy is as effective for the treatment of WS as the higher doses used in previous studies. The dosage of ACTH used in the treatment of WS can be decreased as much as possible to avoid serious adverse effects.
Support: A grant from the Epilepsy Research Foundation.