Abstracts

RATIONALE: Controversy remains as to the most effective dosage of ACTH for treatment of infantile spasms. Few prospective studies have evaluated long term outcome following high vs. low dose ACTH.
METHODS: Outcome was assessed in subjects enrolled in the [dsquote]High-dose, long-duration versus low-dose, short-duration corticotropin therapy for infantile spasms[dsquote] study, in which 50 subjects completed a prospective, randomized, single-blind study performed from 1988-93 at Baylor College of Medicine (J Pediatr 1994;124:803-6). Follow-up evaluation included history, neurological and developmental examination, and 1-2 hour awake/asleep EEG/video study. Physicians involved with clinical and EEG assessments were blinded regarding high vs. low dose treatment groups.
RESULTS: Follow-up was obtained in 25 of the 50 subjects who completed the initial study. There were no differences in demographic characteristics, etiology, or initial response to treatment between those who returned for follow-up and those who did not. Five subjects died after the initial study and prior to contact for this study (2 high dose, 3 low dose). Of the remaining 20 subjects, 12 received high and 8 received low dose ACTH. There were no group differences in etiology (2 patients from each group had cryptogenic etiology). Mean age at follow-up was 10.7 years (mean duration of follow-up 10.1 years). 40% of subjects currently experience recurrent seizures (42% in high dose, 37.5% in low dose groups). Neurodevelopmental status was abnormal in 85% (83% in high dose, 87.5% in low dose groups). EEG background was abnormal in 55% (67% in high dose, 37.5% in low dose groups), and epileptiform discharges were present in 65% (75% in high dose, 50% in low dose groups). There were only 3 patients (1 in high dose, 2 in low dose groups) who had EEGs within the range of normal variation.
CONCLUSIONS: Long term follow-up, assessed with clinical examination and EEG/video studies performed approximately 10 years following treatment with high vs. low dose ACTH, reveals no major differences between groups with regard to presence of recurrent seizures, neurodevelopmental status, EEG background, or presence of epileptiform discharges. Abnormal neurodevelopmental status was present in the majority of subjects, consistent with numerous earlier investigations, emphasizing the generally poor outcome following infantile spasms.
Support: Supported by grant No. NS 25884 from the National Institute of Neurological Disorders and Stroke and the Peter Kellaway, Ph.D. Endowment for Research.