Treatment of infantile spasms with very high dose prednisolone before high dose adrenocorticotropic hormone

Summary Purpose This study investigated the short‐term response to a standardized hormonal therapy protocol for treatment of infantile spasms. Methods Twenty‐seven children with video electroencephalography (EEG)–confirmed infantile spasms received very high dose (8 mg/kg/day, max 60 mg/day) oral prednisolone for 2 weeks. Response (absence of both hypsarrhythmia and spasms) to prednisolone was ascertained by repeat overnight video‐EEG. Responders were tapered over 2 weeks and nonresponders were immediately transitioned to high dose (150 IU/m2/day) intramuscular adrenocorticotropic hormone (ACTH) for two additional weeks. Response was again determined by overnight video‐EEG after ACTH therapy. Key Findings Sixty‐three percent (17/27) of patients responded completely to prednisolone. Subsequently, 40% (4/10) of prednisolone nonresponders exhibited a complete response after an additional 2‐week course with ACTH. Among 27 subjects with median follow‐up of 13.5 months (interquartile range [IQR] 4.8–25.9), 12% (2/17) of prednisolone responders and 50% (2/4) of ACTH responders experienced a relapse between 2 and 9 months after initial response. Significance Very high dose prednisolone demonstrated significantly higher efficacy than previously reported for lower doses in prior studies. High dose ACTH may be superior to very high dose prednisolone, and in lieu of a definitive clinical trial, the choice between prednisolone and ACTH for initial treatment of infantile spasms remains controversial.