Efficacy assessment and follow-up study with different small doses of adrenocorticotropic hormone in treating infantile spasms

Abstract

Objective To investigate the efficacy and optimal dose of adrenocorticotropic hormone(ACTH) in treatment of infantile spasms(IS). Methods Sixty patients with IS were randomly divided into group A (ACTH 20 IU/d, n=20), group B (ACTH 40 IU/d, n=20) and group C (Depakine syrup, n=20) in a prospective randomized study.Patients in group A and group B were given ACTH in the dose of 20 IU/d and 40 IU/d for 2 weeks, respectively.After 2 weeks of treatment, ACTH administration was replaced by oral prednisone if the seizure frequency decreased by 50.0% or less.Patients showing incomplete control of epilepsy(seizure frequency reduction less than 50.0%) or fai-ling to respond to treatment were continuously treated with the original amount of ACTH for another 2 weeks, then ACTH was replaced by oral prednisone.Patients in group C were treated with the combination regimen of oral clonazepam and Depakine syrup routinely.All participants were followed up by 0.5-3.0 years.The differences in clinical efficacy, changes in electroen cephalogram(EEG) and adverse reactions in 3 groups were compared and analyzed. Results After 4 weeks of treatment, the total effective rates(TER) and EEG remission rate (ERR) in group A [TER: 75.0%(15/20 cases), ERR: 40.0%(8/20 cases)] and group B [TER: 70.0%(14/20 cases), ARE: 45.0%(9/20 cases)] were significantly higher than those in group C[TER: 30.0%(6/20 cases), ERR: 10.0%(2/20 cases)](χ2=10.011, 6.624, all P 0.05). During follow-up study, the TER and ERR in group A [TER: 30.0%(16/20 cases), ERR: 35.3%(6/17 cases)] and group B [TER: 30.0%(6/20 cases), ARE: 37.5%(6/16 cases)] were also significantly higher than those in group C [TER: 5.0%(1/20 cases), ARE: 5.9%(1/17 cases)] (χ2=4.329, 6.455, all P<0.05), respectively.The incidence of adverse reactions in group A was lower than that in group B (χ2=3.956, P<0.05). Conclusions ACTH proves to be an effective drug for the treatment of IS.Administration of ACTH in the dose of 20 IU/d for 2 weeks seems to be an optimal treatment recipe with remarkable efficacy with fewer adverse effects and may be worthy of clinical trials in a larger cohort. Key words: Adrenocorticotropic hormone; Infantile spasms; Follow-up study