A prospective study on the treatment of infantile spasms with first-line topiramate followed by low-dose ACTH

SummaryPurposeA prospective study was conducted for the purpose of identifying the efficacy and safety of a protocol, first-line topiramate (TPM) followed by low-dose adrenocorticotropic hormone (ACTH) as the second drug, in the treatment of infantile spasms.Methods40 children newly diagnosed with infantile spasms between 2007 and 2008 were enrolled in this study. They received an initial dose of 0.5-1 mg/kg/day TPM, with 0.5-1 mg/kg/day ascending every 3-7 days up to the target dose within the first 1 month. Partial/nonresponders for TPM were subsequently added low-dose and short-duration ACTH as the second treatment. Both efficiency and side effects were evaluated during the follow-up period.ResultsInfants became spasms-free in 27 (67.5%) of all patients comprising 10 patients treated with TPM monotherapy and 17 with the combination of TPM and ACTH. Greater than 75% reduction in the frequency of spasms was found in 4 of all patients and at least 50% reduction in 4 patents as well. Side effects occurred in 29 (72.5%) patients; apart from 6 patients who needed moderately medical intervention, side effects throughout the trial were mild and well tolerated.ConclusionsThis prospective study demonstrated that, on the basis of primary TPM therapy, the combination treatment both TPM and low-dose ACTH was effective and available for the patients with infantile spasms.