Effectiveness and Safety of Different Once-Daily Doses of Adrenocorticotropic Hormone for Infantile Spasms.

Introduction: Adrenocorticotropic hormone (ACTH) has been commonly used as a first-line treatment for infantile spasms (IS), but its optimal dose and duration are still unclear. This study is the largest retrospective cohort to document the therapeutic efficacy and tolerability for three gradient doses of ACTH in IU/kg/day units in Chinese patients.

Objective: The aim of our study was to elucidate the effectiveness and safety of three different low doses and duration of ACTH treatment for IS in China.

Methods: We conducted a retrospective, chart review of IS cases that were treated with biologic short-acting ACTH and followed up for at least 6 months at a single center in China between June 2010 and June 2016. In total, 200 children met the inclusion criteria. Cases were divided into three groups according to dosage (1, 1.1-1.9, and 2-4 IU/kg/day). Furthermore, we divided the 2-4 IU/kg/day group into 2-3 and 3.1-4 IU/kg/day subgroups. All groups were evaluated for response rates, relapse rates, and adverse effects.

Results: Electroclinical remission by day 14 occurred in 41.4% of infants given 2-4 IU/kg/day and 36.4% of infants given 1.1-1.9 IU/kg/day, compared with only 14.7% of patients given 1 IU/kg/day (p = 0.004 and 0.03, respectively). Prolonging ACTH treatment for up to 28 days improved response by 24% in all 200 infants. Overall, 73.9% of infants receiving 2-4 IU/kg/day responded, significantly higher than the 52.7% responding to 1.1-1.9 IU/kg/day and the 23.5% responding to 1 IU/kg/day (p < 0.01). There was no significant difference in the number of relapses or adverse effects in the three groups. Moreover, in the 2-4 IU/kg/day group, 74.7% of children receiving 2-3 IU/kg/day of ACTH responded, compared with 70% who responded to 3.1-4 IU/kg/day (p = 0.78).

Conclusions: ACTH at a dosage of 2-3 IU/kg/day is superior to 1.1-1.9 and 1 IU/kg/day dosages, is as good as a 3.1-4 IU/kg/day dosage in terms of response rate, and causes no more adverse effects or relapses than other dosages. In addition, prolonging the duration of ACTH treatment can improve response.